A recent article in the journal Nature credits Stanford physician-neuroscientist Sergiu Pasca, MD, with blazing a trail toward a more profound understanding of early brain development, and of what can go wrong in the process, using a cell-based research innovation he named "assembloids."

In 2015, Pasca and his colleagues published a paper in Nature Methods describing a fascinating feat: His tinkering with induced pluripotent stem cells, or iPS cells -- former skin cells transformed so that they've acquired an almost magical capacity to generate all the tissues in the body -- had borne a three-dimensional product. From these "magic" iPS cells grew a complex conglomerate of cells capable of modeling specific organs.

Pasca's particular interest was in the brain, and in the experiments detailed in the study, his lab had caused human iPS cells to multiply and differentiate into small spherical clusters of brain tissue suspended in laboratory glassware.

These clusters recapitulated the architecture and physiology of the human cerebral cortex -- the outermost layer of brain tissue, critical to perception, cognition and action. Pasca named these clusters, which grew to several millimeters in diameter and contained millions of cells, "cortical spheroids." Today, researchers around the world are using similar methodology to create models, broadly known as "organoids," to study other parts of the human body.

Two years later, in a study published in Nature, Pasca upped the ante by, first, generating a second kind of neural spheroid -- this time, representative of a deeper part of the developing forebrain called the subpallium -- and, second, by growing this kind of spheroid in conjunction with cortical spheroids, in the same dish.

To the researchers' amazement, spheroids of both types fused together, with nerve cells from subpallial spheroids migrating and poking extensions into the cortical spheroids and establishing working connections with nerve cells of a different type in the latter spheroids, just as occurs in fetal development.

"It's amazing that these cells already self-organize and know what they need to do," Pasca marveled in "Brain Balls," an article I wrote for our magazine, Stanford Medicine, a few years ago.

Pasca sensibly dubbed the two-fused-spheroid combos "assembloids," the Nature recap notes.

But why stop at two? Pasca has since created three-element assembloids composed of spheroids representative of cerebral cortex, spinal cord and skeletal muscle in order to model the circuitry of voluntary movement. He's also shown that stimulating the "cerebral cortex" spheroid can result in contraction of the "muscle" spheroid. (This accomplishment was published in Cell in late 2020.) He has explored other assembloid combinations, as well, such as the fusing of cortical spheroids with spheroids representing the striatum, a brain structure implicated in regulating our movements and responses to rewarding and aversive stimuli.

Because each spheroid begins with skin cells, they can be grown on a personalized basis -- and can therefore be extracted from patients with neurological disorders known or suspected to spring from early developmental aberrations (such as autism or schizophrenia). The cells can then be used to create models to probe these disorders' molecular, cellular and circuit-based deviations from the pathways of normal brain development, allowing scientists to study the brain in way they could never do with a living patient.

From the Nature article:

Assembloids are now at the leading edge of stem-cell research. Scientists are using them to investigate early events in organ development as tools for studying not only psychiatric disorders, but other types of disease as well.

An assembloid is by no means a complete, working brain. But, the article notes, "Pasca stands by the aphorism that all models are wrong, and some are useful. 'There's been important progress in the field in a short period of time,' he says."

Photo courtesy of the Pasca laboratory

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Stanford neuroscientist's 'assembloids' pave the way for innovative brain research - Scope

Skin Stem Cells Comments Off on Stanford neuroscientist’s ‘assembloids’ pave the way for innovative brain research – Scope | October 5th, 2021

Fall is here, and now is the perfect time to upgrade your beauty routine for the cooler temperatures. Here are a few of our favorite products:

Parfums de Marly

The famed perfume house founded by Julien Sprecher will launch sensual Oriana on September 12th. Packaged in a hot pink bottle, the airy and dreamy fragrance is captivating withnotes of orange blossom, marshmallow, and Chantilly cream. Available on parfums-de-marly.com and Saks Fifth Avenue. $320.

ILONA

This innovative skincare brand continuously seeks out innovative ingredients and technologies from around the world and incorporates them into premium high-performance formulas like the BEYOND C Corrective Serum. This reincarnating serum is housed in a beautiful dual vessel package. One side is black, the other gold with each containing corrective technologies that reach maximum potency at the moment of unity.

The black side contains a novel probiotic and micronized niacin that fortifies skin defenses, intensifies cellular activity, quells inflammation, and amends blotchiness. The gold vessel contains a patented, hyper-potent, permeable form of vitamin C that helps brighten, fade age spots, even skin tone, and protect against oxidation. $132.

Heraux

This innovative product was created by Heraux Co-Founder Dr. Ben Van Handel, a Stem Cell Biologist at the University of Southern California and leading inflammaging researcher. Dr. Handel notes that Retinols are anti-inflammatory at the molecular level, acting as antioxidants and blocking hyperactivation of the immune system. HX-1, the proprietary ingredient in HerauxsMolecular Anti-Inflammaging Serum, acts to shield skin stem cells from pro-inflammaging stressors and concurrently supports the youthful function of stem cells. The regenerative program enabled by HX-1 promotes the secretion of collagen and elastin as well as improved skin barrier function.

Kimberly Fisher is a Pursuitist contributor. As a freelance writer and on-camera host, Kimberly has traveled the world and has published over 400 articles in over 44 publications including Sherman's Travel, Huffington Post, Just Luxe, Luxury Lifestyles UK, eHow, Examiner, Food Wine Travel Magazine, Luxe Beat, NiteGuide, Ocean View, and USA Today. Disclosure: Kimberly is employed by Remy Cointreau Americas.

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3 of the Best Fall Beauty Buys - Pursuitist

Skin Stem Cells Comments Off on 3 of the Best Fall Beauty Buys – Pursuitist | October 5th, 2021

Facial serums are skin care products that the skin can absorb rapidly. They are formulated to contain high doses of ingredients, including actives such as vitamin C and retinol. They are not moisturizers. Instead, they are an additional step in a skin care routine selected to address specific skin concerns.

While many facial serums make claims about their benefits, limited scientific data is available to support these claims. The potential uses and benefits of a serum will depend on the ingredients and their concentration.

Many serums use active ingredients such as:

Common issues face serums claim to address include acne, rosacea, and signs of aging such as wrinkles.

One study of a facial serum targeting fine lines and wrinkles found that after 12 weeks, women showed statistically significant improvements in:

Face serums are generally considered safe. They are available without a prescription. However, because face serums deliver concentrated doses of active ingredients such as vitamin C or hyaluronic acid, people should exercise caution when trying them for the first time.

Pregnant people should exercise caution when selecting skin care products. Using products made with concentrated forms of vitamin A, such as retinol, can harm a developing fetus. Pregnant people should avoid skin care products that contain retinoids or other harmful ingredients.

People who are already using prescription or high dosage skin care products should check with their healthcare providers before using face serums to ensure they are not taking in unhealthy levels of powerful ingredients, such as retinoids.

When introducing a new active ingredient to the skin, there is a possibility of side effects such as:

If a person experiences these symptoms, they should reduce the frequency and dosage of the serum or stop using it altogether if symptoms do not reduce.

The American Academy of Dermatology recommends trying just one new product, like a facial serum, at a time. Starting several new products all at once, particularly anti-aging products, can irritate the skin and make it difficult to determine which products are beneficial.

It is also helpful to perform a patch test for a week before using a new product on facial skin. A patch test involves applying small amounts of the new product on the inner forearm for a few days to check for reactions.

A skin serum will not be effective if it is not used properly. The best time to apply face serum is after cleaning the skin and before applying moisturizer.

Facial serums are concentrated, so people should only use a small amount to cover the facial skin.

Learn more about skin care routines here.

No single skin care product can address all skin health needs. To pick out the best face serum, a person should first decide what skin concern they want to address, such as wrinkles or acne.

Look for a face serum that is a good fit for individual concerns and needs. For example, a person should choose a face serum that suits their skin type, such as oily, dry, combination, or aging.

Some people may wish to find products that are hypoallergenic and noncomedogenic, which means they are less likely to cause allergic reactions or acne.

Products do not have to be expensive to be effective, and serums are available to suit various budgets.

Please note that the writer of this article has not tried these products. All information presented is purely research-based.

This is an oil-free vitamin C face serum designed for people with oily or blemish-prone skin.

SkinCeuticals claims this serum can reduce skin oiliness and wrinkles, and improve skin texture.

The ingredients include:

This serum costs $166 for a 30ml bottle.

Made with Avene Thermal Spring Water, this water-gel serum is safe for adolescents and adults.

It is suitable for oily skin and should be used after cleansing in the morning and evening.

Avena claims that this product can be used safely alongside other acne treatments and can reduce the appearance of pores.

This serum costs $28.00 for 30ml.

This antioxidant face serum is fragrance- and oil-free, and certified cruelty-free.

Drunk Elephant claims it can reduce the signs of sun damage and aging while hydrating the skin. It stays active on the skin for up to 72 hours and should be applied in the morning.

Customers must mix a powder and liquid to create the fresh activated serum.

This serum costs $78.00 for 28ml.

This face serum contains aloe, vitamin A, niacinamide, vitamin C, vitamin E, tea tree oil, and other vitamin and herbal ingredients.

Klur states that this serum may even out skin texture, helps calms inflammation, and increases skin clarity.

People should use it at night and no more than 3 times a week.

This serum costs $110.00 for 30ml.

This serum uses forms of retinoid that The Ordinary states may reduce the signs of aging better than other retinoid products, without causing irritation.

The company recommends people patch test the serum, before using it on facial skin. People should not use it in combination with other retinoid products.

Use only in the evening after cleansing, and refrigerate after opening.

This serum is vegan and free from oil, silicones, nuts, and gluten.

This low-cost serum sells for $9.80 for 60ml.

This cruelty-free skin serum contains milk protein, seaweed extract, and arnica. Ren claims it may calm and soothe the skin while reducing redness and irritation.

It should be used morning and/or night before moisturizing.

This serum costs $58.00 for 30ml.

True Botanicals claims that this anti-aging serum may reduce the appearance of fine lines and wrinkles.

It uses natural antioxidants such as chebula, elderberry, ginger, and echinacea to help strengthen the skins immunity to signs of aging.

After morning and evening cleansing, users can apply one half or full drop to their faces.

This serum costs $90.00 for 30ml.

Skin Authority claims that this lightweight serum reduces the appearance of fine lines, by plumping skin folds and smoothing skin texture.

This serum is designed for use after morning cleansing.

This serum costs $155.00 for 15ml.

Peach and Lily claims that this serum can brighten the skin and reduce the appearance of dark spots.

It contains various ingredients from botanical sources, including green tea extracts, mushrooms, and arbutin. It also contains niacinamide.

It is suitable for all skin types.

This serum costs $39.00 for 50ml.

SkinCeuticals claims that this serum may reduce the appearance of dark spots and improves skin tone.

Ingredients include:

It is suitable for people with all skin types.

This serum costs $98.00 for 30ml.

Bioderma claims that this face serum may increase the skins ability to retain moisture. Made specifically for people with dry skin, it is noncomedogenic, meaning it will not block pores.

People can use this serum in the morning and evening.

This serum costs $27.99 for 40ml.

This serum is suitable for use by people with all skin types. Eminence claims that this face serum helps reduce the appearance of sun-induced skin damage.

Its active ingredients include vitamin C and other antioxidants, which work together to brighten the skin and improve the appearance of fine lines and wrinkles.

This serum costs $110.00 for 30ml.

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12 of the best face serums 2021 - Medical News Today

Skin Stem Cells Comments Off on 12 of the best face serums 2021 – Medical News Today | October 5th, 2021

A man went to a clinic in Mannheim, Germany, where tests found that the rash on his hands and elbows was in fact leukaemia cutis, where the cancer cells are in the skin tissue

Image: NEJM)

A man suffering from a nasty skin rash on his hands and elbows that looked like psoriasis found that it was actually a form of leukaemia.

The 65-year-old went to a dermatologist in Mannheim, Germany, due to the red patches that had developed on the back of his hands and arms and he later found the worrying diagnosis that it was leukaemia cutis.

Tests done at the hospital showed that he had a high white-cell count along with a low number of platelets, it is reported.

The case was published in the New England Medical Journal where it said that a diagnosis of leukaemia cutis was made.

While extremely rare, the condition means that the leukaemia cells are in the skin tissue.

It is found to happen in around three percent of leukaemia cases where the rash has been found on skin including the arms, back or face.

Image:

Other leukaemia symptoms include fever, tiredness and susceptibility to infections.

But in the case of the 65-year-old in Germany, he only had the rash on his hands and elbow.

The New England Medical Journal stated: "A diagnosis of leukemia cutis was made, and the patient was urgently referred to the oncology clinic.

"The patient received a diagnosis of chronic myelomonocytic leukaemia and underwent stem-cell transplantation.

"Two weeks after the transplantation, the patients skin changes had resolved, and the cancer has been in remission since."

Leukaemia Care UK said that usually patients who have the cancer and find rashes do not have this particular condition.

It stated: Leukaemia cutis is very rare, occurring in only about three percent of total cases of leukaemia.

"With this in mind, it is unsurprising that most lesions seen in leukaemia patients are not leukaemia cutis.

In fact, most lesions (40%) seen in leukaemia patients are caused by other complications of leukaemia.

It continued: In the rare occasion that leukaemia cutis occurs, the patient will normally have already been diagnosed with leukaemia.

"However, in seven percent of cases of leukaemia cutis, the skin lesion is the very first symptom of a blood cancer. This is sometimes referred to as aleukaemic leukaemia cutis.

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Man discovers nasty red rash on his hands and elbows is potentially fatal - The Mirror

Skin Stem Cells Comments Off on Man discovers nasty red rash on his hands and elbows is potentially fatal – The Mirror | October 5th, 2021

Kyoto A drug discovered through a method using induced pluripotent stem cells, or iPS cells, from sufferers of amyotrophic lateral sclerosis (ALS) was effective in stopping the progression of the neurological disease in five out of nine patients in a clinical trial, a research team at Kyoto University said Thursday.

While drugs that slow down the progression of amyotrophic lateral sclerosis, also known as Lou Gehrigs disease, have been used in the past, this is the first time that a chronic myeloid leukemia drug called bosutinib has been found to stop its progression, according to the team.

Haruhisa Inoue, a professor of neurology at Kyoto University leading the team, said that larger clinical trials will be needed to determine if the drug can be put to practical use, but We are now looking at the possibility of being able to control ALS with the power of science.

The team reproduced the disease by culturing iPS cells derived from the skin of patients into motor neurons. They then tested a series of drug compounds on the cells to find that bosutinib was effective in slowing down the progression of ALS.

The drug was administered for around three months to nine patients who were in the early stages of the disease and showing signs of deterioration. Progression of the disease stopped in five patients during the treatment period, while four patients continued to deteriorate at the same pace as before.

A comparison of the blood samples from both sets of patients showed that they had differing amounts of a protein unique to nerve cells before the drug was administered. Researchers plan to conduct clinical trials with larger groups while adjusting the dosage and other conditions in the future.

There are about 9,000 people in Japan who suffer from ALS. The disease causes motor neurons to progressively die, resulting in paralysis, with many patients requiring a ventilator to stay alive. Its exact cause is unknown, and no fundamental treatments have been established.

In a time of both misinformation and too much information, quality journalism is more crucial than ever.By subscribing, you can help us get the story right.

PHOTO GALLERY (CLICK TO ENLARGE)

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Drug that stops ALS progression found using iPS cells from patients - The Japan Times

Skin Stem Cells Comments Off on Drug that stops ALS progression found using iPS cells from patients – The Japan Times | October 5th, 2021

Photo: Francis Collins, by NIH Image Gallery, via Flickr.

Francis Collins, Director of the National Institutes of Health (NIH),has just announcedhis intention to step down at the end of 2021 after more than 12 years heading the agency. The accolades are already rolling in. Noted evangelical political commentator David French, for example, rushed to praise Collins as a national treasure.

But Collinss real legacy is anything but praiseworthy, and the tendency of figures in the faith community to ignore his real record is far from admirable.

This year of all years should have made the truth about Francis Collins clear. Last month, documents were released suggesting that top National Institutes of Health (NIH) officials may haveliedwhen they denied that the NIH had funded gain of function research in Wuhan, China, that could have resulted in a pathogen that could infect humans.

After reviewing the documents, Rutgers University biologist Richard Ebright had a blistering response: The documents make it clear that assertions by the NIH Director, Francis Collins, and the NIAID Director, Anthony Fauci, that the NIH did not support gain-of-function research or potential pandemic pathogen enhancement are untruthful.

It was another blow to the reputation of Collins in a year when his agency has faced multiple scandals and controversies.

Among evangelical Christians and other people of faith in America, Collins has long been the equivalent of a rock star. But Collinss days of glory as a non-partisan role model, especially for the faith community, may be numbered and its not just because of the latest scandal over the origins of COVID-19.

In recent months, Collinss agency has become embroiled in controversies over its funding of stomach-churning medical experiments involving body parts harvested from aborted babies. The disclosures about the experiments followed Collinssrepealearlier this year of restrictions on the use of aborted fetal tissue in NIH-funded research.

Collins has also stirred controversy with his increasingly shrill attacks on unvaccinated Americans and his support for harsh mandatory vaccination policies that will require the firing of employees who choose not to be vaccinated with a COVID-19 vaccine.

The former head of the Human Genome Project, Collins catapulted to fame (and the cover ofTimeMagazine) in 2000 with the announcement of a working draft of the sequence of the human genome.He then became a hero to many Christians with the publication in 2006 of his bookThe Language of God, which recounted his journey from atheism to Christianity.

In an increasingly polarized national environment, Collins is one of the rare heads of a major federal agency to serve under both Republican and Democratic Presidents. Appointed by President Obama to head the NIH in 2009, Collins continued in that role under President Trump and now President Biden. He has regularly drawn praise from lawmakers on both sides of the aisle. At gatherings of evangelical Christians, he has been known to strum the guitar and sing worship songs and receive the adoration of attendees. At one 2019 eventit was reportedthat conference participants lined up for selfies with him.

In 2020, Collins wasawarded the prestigious Templeton Prize, worth more than $1.3 million, for his work integrating science and faith. Previous recipients of the prize have included Mother Teresa, John Polkinghorne, Charles Colson, and Aleksandr Solzhenitsyn.

Among secular as well as religious journalists, Collins often receives what verges on fawning treatment. A writer forThe New Yorkergushedthat Collins is a model of geek cool. He likes big, noisy motorcycles, and, despite a mild manner, he is famously unself-conscious. At the unlikeliest moments, he will strap on a guitar and accompany himself in song, often a tune he has composed for the occasion.

This year, however, Collinss reputation has taken continued beatings, not just because of evasive answers about the role of the NIH in gain-of-function research in China, but also because of publicity around NIH-funded experiments that many Americans, especially people of faith, would find horrific.

In May, reports surfaced aboutmacabre NIH-funded experimentsthat utilized body parts collected fromaborted human fetuses to createhumanized mouse and rodent models with full-thickness human skin.For the experiments, researchersat the University of Pittsburghcut into tiny pieces human fetal spleen, thymus, and liver organs and then transplanted the tissues and hematopoietic stem cells into irradiated mice. Researchers also sliced off skin from the scalp of the aborted babies and then grafted the fetal skin onto the mice. In the words of the scientists: Full-thickness human fetal skin was processed via removal of excess fat tissues attached to the subcutaneous layer of the skin, then engrafted over the rib cage, where the mouse skin was previously excised.

The body parts used for these experiments were harvested from aborted human fetuses with a gestational age of 18-20 weeks. By that age, an unborn baby hasbrain wavesand abeating heart. He canhear sounds and move his limbs and eyes, and his digestion system has started to work.In other words, the human fetuses whose organs were harvested for this NIH-funded research were well-developed tiny humans, not blobs of undifferentiated cells.

In August, an additional project funded by the NIH came to light thanks to documents obtained in a Freedom of Information Act lawsuit by Judicial Watch and the Center for Medical Progress. The lawsuit was filed after Collinss NIH dragged its feet in responding.According to Judicial Watch, the documents show that theNIH has provided nearly $3 million in tax dollars to support a fetal organ harvesting operation by the University of Pittsburgh in its quest to become a Tissue Hub for human fetal tissue ranging from 6 to 42 [!] weeks gestation.

David Daleiden, president of the Center for Medical Progress,commented: The NIH grant application for just one of Pitts numerous experiments with aborted infants reads like an episode ofAmerican Horror Story. Infants in the womb, some old enough to be viable, are being aborted alive and killed for organ harvesting, in order to bring in millions of dollars in taxpayer funding.

Daleiden furtherallegedthat NIH funding was used to underwrite labor induction abortions, where the baby is pushed out of the mother whole and then killed to obtain the desired tissues. In other words, the NIH was facilitating a process where babies, some of the age of viability, [are] to be delivered alive, and then killing them by cutting their kidneys out.

Francis Collins self-identifies as an evangelical Christian, and most evangelicals as well as faithful Catholics regard abortion as the destruction of innocent human life.

So how has Collins responded to these revelations? With horror? With a pledge to investigate? With a promise to stop taxpayer funding of such research?

Since early August, Ive repeatedly tried to get Collins to answer questions about these NIH funded experiments using aborted fetal organs and tissues.Does Collins support this research funded by his agency? Does he have any ethical objections to it? How does he personally justify the research given his religious convictions? I repeatedly emailed these and other questions to the media contacts for the NIH Office of the Director.

The response? His office has refused to answer.

But its not just NIH research involving humans that has been raising controversy this year. In recent months, Collinss NIH has come under fire for fundingabusive medical experiments on dogsthat critics say were unnecessary as well as barbaric. The experiments were funded by the NIH division headed by Anthony Fauci, but that division is under the ultimate oversight of Collins. In late August, 15 members of Congresssent a letterraising questions about the research.

Again, I tried to get a response from Collins about this latest controversy. Again, he refused to respond to questions.

Ironically, while Collins is AWOL when it comes to answering basic questions about the research his agency is funding, he is more than willing to speak out on other topics, especially COVID-19, where he is now becoming a polarizing figure to many because ofincreasingly shrill advocacy of compulsory vaccination as well as his demonization of those who choose not to take a COVID-19 vaccine.

At the end of April,Collins claimedthat he would not require NIH employees to get vaccinated, and he seemed to argue for a positive approach of selling the benefits of vaccines rather than demonizing the unvaccinated or engaging in finger-wagging. Yet by early August his public posture had changed. He was nowcheerleading for compulsory vaccine requirementsimposed by private businesses as well as enthusiastically overseeing compulsory vaccinations for the very workers at the NIH that he earlier said would not face compulsory vaccination.

After President Bidens speech in September declaringwar on the unvaccinated, Collins ramped up his own rhetoric.In an appearance on MSNBC after Bidens speech, Collins firstsuggestedunvaccinated people were selfish, declaring that this is really an occasion to think about loving your neighbor, not just yourself.

Collins then branded both unvaccinated people and politicians who dont favor vaccine mandates as killers on the wrong side of history. Dismissing their views as merely a philosophical political argument that is part of the culture war, Collins complained that this philosophical political argument is killing people, including, Im sad to say, some children. We have to get past this if we really have a future as a nation.

I would like to say particularly to those leaders who are on the wrong side of this, what Lincoln said one time, Collins declared. Citizens, we will not escape history. Do you want to be looked at in the lens of that backward look ten years from now and defend what you did when in fact, we are losing tens of thousands of lives that didnt have to die?

A question for Collins: Is attacking your fellow citizens (including many fellow Christians) as heartless killers because they disagree with you on either vaccinations or vaccine mandates an example of loving your neighbor?

Whatever one thinks about COVID-19 vaccinations, Collinss over-the-top rhetoric demonizing those he disagrees with as killers is beyond the pale, especially for someone who wears his Christianity on his sleeve. As I have writtenelsewhere, his rhetoric is also based on several falsehoods.

So just how far is Collins willing to go to push coercive medicine? Thats an interesting question.

In my home state of Washington, the governor has issuedan emergency orderthat will compel private religious schools and day care centers as well as other private businesses to fire employees later this month if they wont get vaccinated. While there technically is a route for religious exemptions, it is so narrow and onerous that many religious people may not qualify.

Its now being suggested in some states that discharged employeeswont be able to get unemployment benefits. Perhaps the idea is that if the unvaccinated dont die of COVID they can die of starvation instead.

As if that werent bad enough, the unvaccinated face the denial of medical care. Also in my home state, the University of Washington medical system is now apparently denyingorgan transplantsto patients who are unvaccinated, even if those patients have a credible medical reason for not having the vaccinations.

This is pure, unadulterated social Darwinism: Brand a whole class of people as biologically unfit (in this case, the unvaccinated) and then make sure they cant receive medical care, hold jobs, or basically survive. Heap scorn on them, demonize them as killers, and stir up hatred against them so other people begin to abuse them. If Collins is truly concerned about the judgment of history, he should read a little more widely about the sorry results of demonizing entire classes of people as the enemies of society.

Let me be clear: I am not arguing here about whether people ought to get COVID-19 vaccinations, or whether those vaccinations are helpful. For the record, depending on ones risk profile, I think vaccinations are in a persons best interest. The issue is whether in the name of vaccination people should be stripped of their livelihoods, denied medical care, and demonized as enemies of society. In any morally sane universe, the policies being proposed are as immoral as they are unprecedented.

So does Francis Collins endorse depriving unvaccinated people of their right to work and to support their families? Does he endorse denying them unemployment insurance? Does he go even further and endorse denying medical care to the unvaccinated?

I again asked Collins media relations staff for answers. Again, crickets.

Although Collins likes to tout his personal faith, he appears to have very little concern for any sort of conscience rights of fellows religious believers who disagree with him.After all, he dutifully served in a previous administration that repeatedly weakenedconscience protectionsfor medical workers opposed to abortion and thatviolated federal lawby turning a blind eye when California mandated abortion coverage in all private insurance plans.

It might also be noted that as late as December 2020, Collins was still urging thatmost churches should not meet in person, even implying that they shouldnt do sountil the summer or fall of 2021.

And his current promotion of compulsory vaccinations seemingly has no qualifiers. At least, he isnt talking about any.

Collins hasconcededthat various COVID-19 vaccines used cell lines originally derived from an aborted fetus in either their production or testing, which is one reason some people have moral qualms about the vaccines. Yet you wont find Collins advocating for the conscience rights of these people. In fact, Collins has been silent in the face of attacks on religious exemptions for vaccines.

Reasonable people can disagree about whether the use of abortion-derived cell lines is a moral deal-killer for the vaccines. But thats the point: Reasonable people candisagreeabout what violates their conscience. The test of support for religious liberty is not whether you only support the right of people who agree with you.

Not being willing to stand up for the conscience rights of others to determine their own medical treatment is not morally neutral. It is a moral failure. In the words of theCatholic Bishops of Colorado, A person is morally required to obey his or her conscience, and others should respect the right of others to follow their conscience.

Alas, for those who have followed Francis Collins closely over the years, his current failures of moral leadership come as no surprise. As I will discuss in an upcoming article, Collinss career has been one long testament to moral cowardice and confusion.

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The Appalling Moral Failure of Francis Collins - Discovery Institute

Skin Stem Cells Comments Off on The Appalling Moral Failure of Francis Collins – Discovery Institute | October 5th, 2021

Introduction

The growing burden of ischemic heart disease (IHD) is a major public health issue. The most harmful type of IHD is acute myocardial infarction (MI), which leads to loss of tissue and impaired cardiac performance, accounting for two in five deaths in China.1 Timely revascularization after MI, including percutaneous coronary intervention, thrombolytic treatment and bypass surgery, is key to improving cardiac function and preventing post-infarction pathophysiological remodeling.2 However, these effective but invasive approaches cannot be used in all patients owing to their applicability, which is limited based on specific clinical characteristics, and the possibility of severe complications such as bleeding and reperfusion injury.2,3 Attempts to limit infarct size and improve prognosis using pharmacotherapy (including antiplatelet and antiarrhythmic drugs and angiotensin-converting enzyme inhibitors) without reperfusion has been proven generally inefficient, due to non-targeted drug distribution and side effects, and short half-life of some drugs.1,3,4 Consequently, many patients in which this approach is used still progress to cardiac hypertrophy and heart failure.1 Growth and rupture of atherosclerotic plaques and the ensuing thrombosis are the major causes of acute MI.4 Currently available interventions for atherosclerosis (AS) including statins can reduce acute MI, but the effects vary between individuals, and leave significant residual risks.58 Some chemotherapies, such as docetaxel9 and methotrexate,10,11 also seem to have beneficial effects in AS; however, systemic administration of these drugs is limited because of their adverse effects.12 The demand for safer and more efficient therapies and prevention strategies for MI is therefore increasing.

Several optimized strategies have so far been explored, one of which is the application of nanoparticles (NPs). These nanoscale particles have been widely used in the treatment of tumors and neural diseases.13,14 NPs enable delivery of therapeutic compounds to target sites with high spatial and temporal resolution, enhancement of tissue engineering processes and regulation of the behaviour of transplants such as stem cells. The application of NPs improves the therapeutic effects and minimizes the adverse effects of traditional or novel therapies, increasing the likelihood that they can be successfully translated to clinical settings.1518 However, research on NPs in this field is still in its infancy.5,1921 This review summarizes the latest NP-based strategies for managing acute MI, mostly published within the past 7 years, with a particular focus on effects and mechanisms rather than particle types, which have been extensively covered in other reviews (Figure 1). In addition, we offer an initial viewpoint on the value of function-based systems over those based on materials, and discuss future prospects in this field.

Figure 1 Overview of nanoparticle-based strategies for the treatment and prevention of myocardial infarction. Nanoparticles are capable of delivering therapeutic agents and nucleic acids in a stable and targeted manner, improving the properties of tissue engineering scaffolds, labeling transplanted cells and regulating cell behaviors, thus promoting the cardioprotective effects of traditional or novel therapies.

A multitude of NP types are currently under investigation, including lipid-based NPs, polymeric NPs, micelles, inorganic NPs, and exosomes. Virus can also be considered as NPs; however they will not be discussed in this review.22 NPs made from different materials show similar in vivo metabolic kinetic characteristics and protective effects on infarcted heart.19,20 Function-based NP types, oriented towards a specific purpose, may be preferable compared with traditional types, on account of their practicality in basic research and clinical translation. In this review, we discuss NPs used in the treatment and prevention of MI that fall into the following four categories: 1) circulation-stable nanocarriers (polymeric, lipid or inorganic particles); 2) targeted delivery vectors (magnetic or particles modified to improve target specificity); 3) enhancers of tissue engineering; and 4) regulators of cell behavior (Figure 1). We propose that the choice of each NP for any given application should be primarily based on the roles or mechanisms they perform.

Many NPs, whether composed of either naturally occurring or synthetic materials, act as nanocarriers to improve the circulating stability of therapeutic agents.15,16 Polymeric NPs comprise one of the most widely employed types, with excellent biocompatibility, tunable mechanical properties, and the ability be easily modified with therapeutic agents using a broad range of chemical techniques.23,24 The most commonly used polymer for these NPs is polylactide-co-glycolide (PLGA), which has Food and Drug Administration approval.25,26 Recently, there has been a therapeutic emphasis on polydopamine (PDA), from which several related nanomaterials have been created, including PDA NPs and PDA NP-knotted hydrogels.27,28 NPs made from polylactic acid (PLA),29,30 poly--caprolactone (PCL),31 polyoxalates,32 polyacrylonitrile,33 chitosan29,34 and hollow mesoporous organosilica35 have also been constructed and administered in vitro in cells and in vivo in animal models.

Lipid NPs or liposomes are also considered promising candidates for the delivery of therapeutic agents, due to their morphology, which is similar to that of cellular membranes and ability to carry both lipophilic and hydrophilic drugs. These non-toxic, non-immunogenic and biodegradable amphipathic nanocarriers can be designed to reduce capture by reticuloendothelial cells, increase circulation time, and achieve satisfactory targeting.36,37 Solid lipid NPs (SLNs) combine the advantages of polymeric NPs, fat emulsions, and liposomes, remaining in a solid state at room temperature. Active key components of SLNs are mainly physiological lipids, dispersed in aqueous solution containing a stabilizer (surfactant).38 Micelles are made by colloidal aggregation in a solution through self-assembly of amphiphilic polymers, or a simple lipidic layer of transfer vehicles;39 these have been used in cellular and molecular imaging40 and treatment41 for a long time.

Inorganic NPs used in basic IHD research are classified as metal, metal compounds, carbon,42 or silicon NPs;43 these are relatively inert, stable, and biocompatible. Gold (Au),44 silver (Ag)45 and copper (Cu)46 are commonly used materials in their production. These NPs can be delivered orally,47 or injected intravenously48 or intraperitoneally.56 However, they are more widely used to construct electrically conductive myocardial scaffolds in tissue engineering.49,50 Myocardial patches and scaffolds are promising therapeutic approaches to repairing heart tissue after IHD; incorporating conductive NPs can further improve functionality, introducing beneficial physical properties and electroconductivity. Some organic particles, such as liposomes anchored with poly(N-isopropylacrylamide)-based copolymer groups, are also suitable for the production of effective nanogels or patches for this purpose.37

Several metal compounds have been used for treatment of IHD.5154 The application of magnetic particles made from iron oxide has been of particular interest in recent research. These NPs are more prone to manipulation with an external magnetic field, and thus serve as powerful tools for targeted delivery of therapeutics. In addition, modification with targeted peptides or antibodies is another approach to the construction of targeted delivery systems.

Another strategy to protect cardiac performance after MI is the transplantation of cells; however, the beneficial effects of this are currently limited.58 Many NPs can improve the behavior of cells; in this context, they may stimulate cardioprotective potential. In particular, exosomes a major subgroup of extracellular vesicles (EVs) with a diameter of 30150nm, which are secreted via exocytosis55 represent novel, heterogeneous, biological NPs with an endogenous origin. They are able to carry a variety of proteins, lipids, nucleic acids, and other bioactive substances.5557 Mechanistic studies have confirmed that exosomes offer a cell-free strategy to rescue ischemic cardiomyocytes (CMs).59,60

The physical properties of NPs, including size, shape, and surface charge, impact on how biological processes behave, and consequently, responses in the body.61 The recommended definition of NPs in pharmaceutical technology and biomedicine includes a limitation that more than 50% of particles should be in a size distribution range of 10100 nm.39 However, this is not strictly distinguished in studies, so for the purposes of this review, we have relaxed this definition. Small NPs have a faster uptake and processing speed and longer blood circulation half-lives than larger ones; a decreased surface area results in increased reactivity to the microenvironment and greater speed of release of the compounds they carry.6163 However, an exception to this principle is that, among particles of less than 50 nm diameter, larger NPs have longer circulatory half-lives.64,65 NPs can be spherical, discoidal, tubular or dendritic.61,63 The impact of NP shape on uptake and clearance has also been revealed;66,67 for instance, spheres endocytose more easily,20 while micelles and filomicelles target aortic macrophages, B cells, and natural killer (NK) cells in the immune system more effectively than polymersomes.68 In terms of charge, cationic NPs are more likely to interact with cells than negatively charged or neutral particles because the mammalian cell membrane is negatively charged.62 As a result, positively charged particles are reported to be more likely to destabilize blood cell membranes and cause cell lysis.61 Additionally, the rate of drug release is largely determined by the diameter of the pore. Motivated by the idea, Palma-Chavez et al developed a multistage delivery system by encapsulating PLGA NPs in micron-sized PLGA outer shells.69

Some types of NPs, such as micelles, possess coreshell morphological structures: a core composed of hydrophobic block segments is surrounded by hydrophilic polymer blocks in a shell that stabilizes the entire micelle. The core provides enough space to accommodate compounds, while the shell protects drug molecules from hydrolysis and enzymatic degradation.36 Surface chemical composition largely governs the chemical interactions between NPs and molecules in the body. Appropriate surface coatings can create a defensive layer, protect encapsulated cargo, and affect biological behaviors. Coating with inert polymers like polyethylene glycol (PEG) is the most commonly used method, which hinders interactions with proteins, alters the composition of the protein corona, attenuate NP recognition by opsonins which tag particles for phagocytosis, and extend the half-life of particles.36,70 Additionally, PEG coating helps the therapeutic agents reach ischemic sites, because PEGylated macromolecules tend to diffuse in the interstitial space of the heart.71 Functionalization of gangliosides can further attenuate the immunogenicity of PEGylated liposomes without damaging therapeutic efficacy.72 Removal of detachable PEG conjugates in the microenvironment of the target sites improves capture by cells. Wang and colleagues synthesized PDA-coated tanshinone IIA NPs by spontaneous hydrophobic self-assembly.73 Polyethyleneimine (PEI) is capable of condensing nucleic acid and overcoming hamper of cell membrane. Therefore, modification with PEI is mainly used for the transport of DNA and RNA.74 Of note, despite their inertness, novel NPs composed of metals can also be modified with compounds such as PEG, thiols, and disulfides.48,75 Hydrogels mixed with peptide-coated Au NPs attain greater viscosity than hydrogels mixed with Au NPs.24

Targeted delivery is a primary goal in the development of nanocarriers. Passive targeting is based on enhanced permeability in ischemic heart tissue, which does not meet the needs of clinical application.76 This fact has prompted work on targeting agent modification and magnetic guidance. Conjugation with specific monoclonal antibodies is a feasible method for delivering drug payloads targeted to ischemic lesions. Copper sulfide (CuS) NPs coupled to antibodies targeting transient receptor potential vanilloid subfamily 1 (TRPV1), permit specific binding to vascular smooth muscle cells (SMCs), and can also act as a switch for photothermal activation of TRPV1 signaling.52 In another study conducted by Liu and colleagues, two types of antibodies, binding CD63 (expressed on the surface of exosomes) or myosin light chain (MLC, expressed on injured CMs) are utilized to allow NPs to capture exosomes and accumulate in ischemic heart tissue. These NPs have a unique structure comprising an ferroferric oxide core and PEG-decorated silica shell, which simultaneously enables magnetic manipulation and molecule conjugation via hydrazone bonds.21 Targeted peptides such as atrial natriuretic peptide (ANP),43 S2P peptide (plague-targeting peptide),77 and stearyl mannose (type 2 macrophage-targeting ligand)16 allow NPs to precisely target atherosclerotic tissue and ischemic heart lesions. Modification with EMMPRIN-binding peptide (AP9) has been shown to enable more rapid uptake of micelles by H9C2 myoblasts and primary CMs and to deliver drug payloads targeted to lesions in vivo.78,79 Another strategy for targeted nanocarriers is to produce cell mimetic carriers. Using the inflammatory response as a marker after MI,76 Boada and colleagues synthesized biomimetic NPs (leukosomes) by integrating membrane proteins purified from activated J774 macrophages into the phospholipid bilayer of NPs. Local chronic inflammatory lesions demonstrated overexpression of adhesion molecules, which bound leukosomes efficiently.80

The biocompatibility of NPs is difficult to predict because any interaction with molecules or cells can cause toxic effects. Generally, NPs remain in blood, but can also extravasate from vasculature with enhanced permeability, or accumulate in the mononuclear phagocyte system.81 Important causes of NP-associated toxicity include: oxidative stress injury and cell apoptosis secondary to the production of free radicals, lack of anti-oxidants, phagocytic cell responses, and the composition of some types of particles.61 Hepatotoxicity, nephrotoxicity and any other potential off-target organ damage caused by accumulation of particles, especially those with poor degradability and slow clearance, are also essential to explore in toxicity tests.82 Additionally, the evaluation of evoked immune responses according to the expression of inflammatory factors and stimulation of leukocytes in cell lines and animal models is also important.83

A few studies have reported NP-associated acute and chronic hazards in pharmacological applications, although some of these observations may be contentious. Specifically, aggregation of non-functionalized carbon nanotubes (CNTs) has been observed owing to inherent hydrophobicity of these particles.61 Aside from inflammation and T lymphocyte apoptosis, multi-walled CNTs can rupture cell membranes, resulting in macrophage cytotoxic effects.84,85 Silica NPs induce vascular endothelial dysfunction and promoted the release of proinflammatory and procoagulant factors, mediated by miR-451a negative regulation of the interleukin 6 receptor/signal transducer and activator of transcription/transcription factor (IL6R/STAT/TF) signaling pathway.8688 Metal NPs, such as Au and Ag, can also penetrate the cell membrane, increase oxidative stress and decrease cell viability.89,90 Consequently, exposure to Au may cause nephrotoxicity91 and reversible cardiac hypertrophy.92 El-Hussainy and colleagues observed myocardial dysfunction in rats given alumina NPs.93,94 Nemmar and colleagues investigated the toxicity of ultrasmall superparamagnetic iron oxide nanoparticles (SPIONs) administered intravenously, which resulted in cardiac oxidative stress and DNA damage as well as thrombosis.95 Cell-derived exosomes and a majority of natural polymers are considered relatively safe;83 however, Babiker and colleagues demonstrated that dendritic polyamidoamine NPs compromise recovery from ischemia/reperfusion (I/R) injury in isolated rat hearts.96 The effects of degradation byproducts are also of concern.83 An advantage of the nanoscale size of NPs is that their injection is unlikely to block the microvascular system; however, it remains controversial whether NPs give rise to arrhythmias.97 These factors highlight that examining the biocompatibility of NPs both in vitro and in vivo is a vital component of preclinical or clinical research.

NP toxicity depends on many parameters, including material composition, coating, size, shape, surface charges and concentration.39 For instance, larger particles seem to be more favorable from a toxicology standpoint.83 However, single-walled CNTs are considered more harmful than multi-walled CNTs, due to their smaller size resulting in less aggregation and increased uptake by macrophages.61 Cationic AuNPs are more toxic compared with anionic AuNPs, which appear to be nontoxic.98 Generally speaking, NP-associated toxicity can be lowered by functionalization with nontoxic surface molecules, stabilization and localization in the region of interest by using scaffolds.24,99 The toxicity of CNTs mediated by oxidative stress and inflammation was reduced using these strategies in several studies.24,100 Local application and targeted delivery also enabled dose reduction and concurrently decreased the incidence of adverse effects. Administration of therapeutic agents directly into the infarcted or peri-infarcted myocardium is a conventional approach with a low risk of inducing embolization.

NP is a suitable method for the administration of therapeutic agents in terms of the minimization of side effects, enhanced stability of cargo, and possibility of controlled delivery and release.76 Detailed information on the experimental design and results of the latest studies on the use of NPs as therapeutic vectors are provided in Table 1. Recently, several drugs approved for clinical use as immunosuppressants have been suggested as potentially effective cardioprotective agents. For example, NPs containing cyclosporine A inhibited apoptosis and inflammation in ischemic myocardium by improving mitochondrial function.25,101 Commercial methotrexate also showed minor cardioprotective effects; additionally, when loaded into lipid core NPs, adenosine bioavailability and echocardiographic and morphometric results were all improved a rats model of MI.102 Margulis and colleagues developed a method to fabricate NPs via a supercritical fluids setup, which loaded and transferred celecoxib, a lipophilic nonsteroidal anti-inflammatory drug, into the NPs. These celecoxib-containing NPs alleviated ejection function damage and ventricular dilation by inducing significant levels of neovascularization.103 Furthermore, a series of investigations indicated that drugs used for hypoglycemia (eg pioglitazone, exenatide and liraglutide)104106 and lipid lowering (statins)107 attenuate the progression of post-MI heart failure, and are therefore also potential therapeutic cargoes for NPs in the treatment of MI.

NP systems also offer an alternative method for delivering plant-derived therapeutic agents, most of which belong to traditional Chinese medicine. Its of vital importance because of the criticization on adverse reactions caused by direct injection of such complexes. Cheng and colleagues designed a dual-shell polymeric NP as a multistage, continuous, targeted vehicle of resveratrol, a reactive oxygen species (ROS) scavenger. Due to the severe oxide stress in areas of infarction, the proposed antioxidant-delivery NPs represent a new method to effectively treat MI. These NPs are modified with two peptides, targeting ischemic myocardium and mitochondria, respectively; cardioprotective effects have been confirmed in both hypoxia/reoxygenated (H/R) H9C2 cells and I/R rats.108 In addition, Dong and colleagues also demonstrated that puerarin-SLNs produced smaller areas of infarction in a MI rat model, evaluated by 2,3,5-triphenyltetrazolium chloride (TTC) staining. These particles were modified with cyclic arginyl-glycyl-aspartic acid peptide, a specific targeting moiety to v3 integrin receptors, which are highly expressed on endothelial cells (ECs) during angiogenesis.109 In a recent study, quercetin was loaded into mesoporous silica NPs, which enhanced the inhibition of cell apoptosis and oxidative stress, improving ventricular remodeling and promoting the recovery of cardiac function by activating the janus kinase 2 (JAK2)/STAT3 pathway.110 Similarly, curcuminpolymer NPs, administered by gavage, improved serum inflammatory cytokine levels compared with direct administration of curcumin.111

Translation of novel bioactive agents into clinical practice has been limited, owing to lack of sufficient bioavailability and systemic toxicity.76 Encapsulating small molecules such as 3i-1000 (an inhibitor of the GATA4NKX2-5 interaction),43 TAK-242 (inhibitor of toll-like receptor 4, TLR4)112 and C143 (inhibitor of ERK1/2)113 in NPs promotes myocardial repair after MI without the risk of uncontrolled and off-target adverse effects. Administration of vascular endothelial growth factor (VEGF) causes elevated vascular permeability and tissue edema. The cardioprotective effects of VEGF-loaded polymeric NPs injected either intravenously114 or intramyocardially115 eliminated vascular leakage due to promotion of lymphangiogenesis. Further studies have confirmed these results and add to the evidence that combined delivery of VEGF with other growth factors is recommended, since VEGF primarily drives the formation of new capillaries.116 Furthermore, in line with previous research, similar therapeutic effects have been demonstrated in studies using polymeric NPs loaded with stromal cell derived factor 1 (SDF-1) and insulin-like growth factor 1 (IGF-1).117,118

We also notice that some novel payloads in NPs-based therapy for MI have been studied. For example, deoxyribozyme-AuNP can silence tumor necrosis factor- (TNF-).119 A target that is implicated in irreversible heart damage after MI; its effects are mediated by free radical production, downregulation of contractile proteins, and initiation of pro-inflammatory cytokine cascades. Mesoporous iron oxide NPs containing the hydrogen sulfide donor compound diallyl trisulfide act as a platform for the controlled and sustained release of this therapeutic gas molecule. The application of these NPs at appropriate concentrations, resulted in the preservation of cardiac systolic performance without any observable detrimental effects on homeostasis in vivo.15

With increasing insight into the molecular mechanisms of MI, a particular emphasis on gene therapy has emerged. Gene expression can be modulated by DNA fragments, messenger RNA (mRNA), microRNA (miRNA) and small interfering RNA (siRNA), which thus represent new approaches for treating ischemia. Currently available nucleic acid delivery systems are mainly divided into viral and non-viral systems. However, virus-based approaches are limited by their potential for uncontrollable mutagenesis.36 From a clinical point of view, NP represents a suitable choice as novel non-viral nucleic acid vector, which could feasibly transfect in a stable, targeted, and sustained manner (as shown in Table 2).

Table 2 NPs-Based Nucleic Acid Delivery Systems for Treatment for MI Reported in the Last 7 Years

As a common gene vehicle, plasmids face the risk of being destroyed by DNase and immunoreactivity in the serum, and transduction in non-target organs.120 A recent study by Kim and colleagues aligns with current research trends focused on virus-free therapies, in which carboxymethylcellulose NPs were designed to transfer 5-azacytidine to halt proliferation, and deliver plasmid DNA containing GATA4, myocyte enhancer factor 2C (MEF2C), and TBX5 to induce reprogramming and cardiogenesis of mature normal human dermal fibroblasts.121 In a methodological study, lipidoid NPs were used to successfully deliver pseudouridine-modified mRNA, encoding enhanced green fluorescent protein.122

MiRNAs act as essential regulators of cellular processes through post-transcriptional suppression; increasing evidence reveals miRNAs play critical roles in cardiovascular diseases. An miRNA-transferring platform with self-accelerating nucleic acid release, containing a heparin core and an ethanolamine-modified poly(glycidyl methacrylate) shell, has been constructed and used as an efficient vector of miR-499, which inhibits cardiomyocyte apoptosis.123 Intravenous administration of anionic hyaluronan-sulfate NPs (mean diameter 130 nm) enable the stable delivery of miR-21 mimics, thus modulating the expression of TNF, transforming growth factor (TGF), and suppressor of cytokine signaling 1 (SOCS1). Consequently, these NPs switch the phenotype of macrophages from pro-inflammatory to reparative, promote neovascularization and reduced collagen deposition.124 Interestingly, silencing miR-21 using antagomiR-21a-5p in a nanoparticle formulation has also been shown to reduce expression of pro-inflammatory cytokines in vitro, and attenuate inflammation and fibrosis in mice with autoimmune myocarditis.125 A number of other potentially therapeutic miRNAs have also been successfully transferred to CMs in recent works, including miR-146a, miR-146b-5p, miR-181b, miR-199-3p, miR-214-3p, miR-194-5p and miR-122-5p.126128 Evaluation of angiogenesis, cardiac function, and scar size in these studies indicated that injectable miRNANPs can deliver miRNA to restore injured myocardium efficiently and safely. Yang and colleagues developed an in vivo miRNA delivery system incorporating a shear-thinning hydrogel and NPs characterized by surface presence of miRNA and cell-penetrating peptide (CPP).126 Additionally, angiotensin II type 1 receptor-targeting peptide-modified NPs serve as targeted carriers for anti-miR-1 antisense oligonucleotide, significantly reducing apoptosis and infarct size.129

SiRNAs inhibit gene expression by mediating mRNA cleavage in a sequence-specific manner, highlighting NP-based RNA interference as another viable approach to modulate cellular phenotype and attenuate cardiac failure. Dosta and colleagues demonstrated that poly(-amino ester) particles modified by adding lysine-/histidine-oligopeptides could represent a system for the transfer of siRNA.130 Studies have now revealed that chemokine CC motif ligand 2 (CCL2) and its cognate receptor CC chemokine receptor 2 (CCR2) promoted excessive Ly6Chigh inflammatory monocyte infiltration in infarcted area and aggravate myocardial injury.131 Photoluminescent mesoporous silicon nanoparticles (MSNPs) carrying siCCR2 have been reported to improve the effectiveness of transplanted mesenchymal stem cells (MSCs) in reducing myocardial remodeling after acute MI.131 Targeted transportation and enhanced uptake with minimum leakage improved the efficiency of delivery via NPs, significantly outperforming the control group. Taken together, these studies demonstrate that NPs act as promising drug delivery systems in the treatment of MI.

Myocardial patches and scaffolds, consisting of either bioactive hydrogels or nanofibers, are minimally invasive, relatively localized, and targeted approaches to repair the heart after IHD. Those biomaterials must have an anisotropic structure, mechanical elasticity, electrical conductivity, and the ability to promote ischemic heart repair.132 A variety of NPs have been applied in this field, among which inorganic NPs have been the focus of most research efforts.42 These investigations of inorganic NPs can be divided into four categories based on their effects and the mechanisms involved, which are described in this section.

NPs enhance physical properties and electroconductivity, which is essential for the biomaterials to properly accommodate cardiac cells and subsequently resulted in cell retention, cell-cell coupling and robust synchronized beating behavior. CNTs are able to increase the required physical properties of scaffolds, such as maximum load, elastic modulus, and toughness.133,134 Gelatin methacrylate (GelMA) also has decreased impedance, hydrogel swelling ratio, and pore diameter, as well as increased Youngs modulus when combined with gold nanorods (AuNRs).135 Given this insight, highly electroconductive NPs have been increasingly investigated.34,99 Specifically, Ahadian and colleagues revealed that a higher integrated CNT concentration in gels resulted in greater conductivity.136 Zhou and colleagues verified the therapeutic effects of patches incorporating single-walled CNT for myocardial ischemia, which halted progressive cardiac dysfunction and regenerated the infarcted myocardium.137 Spherical AuNPs have also been shown to increase the conductivity of chitosan hydrogels in a concentration-dependent manner.138 Interestingly, silicon NPs mimic the effects of AuNRs without affecting conductivity or stiffness, as reported by Navaei and colleagues.139

Several studies demonstrate the effects of CNT on CM functions. When CMs are cultured on multi-walled CNT substrates or treated with CNT-integrated patches, these cells show spontaneous electrical activity.34,99,140 Brisa and colleagues functionalized reverse thermal gels with AuNPs, investigating the phenotype of CMs in vitro; the growth of cells with a CM phenotype was observed, along with gap junction formation.141 CMs exposed to AuNR-containing GelMa show higher affinity, leading to packed and uniform tissue structure.135 These conductive scaffolds also facilitate the robustness and synchrony of spontaneous beating in CMs without damaging their viability and metabolic activity.

Combined incorporation of inorganic NPs and cells represents a feasible strategy to promote therapeutic effects. Despite some reports on the cytotoxicity of Au,89,90 no significant loss of viability, metabolism, migration, or proliferation of MSCs in scaffolds containing AuNP is reported. A CNT-embedded, electrospun chitosan/polyvinyl alcohol mesh is reported to promote the differentiation of MSCs to CMs.142 In another approach, Baei and colleagues added AuNPs to chitosan thermosensitive hydrogels seeded with MSCs.138 There was a significant increase in expression of early and mature cardiac markers, indicating enhanced cardiomyogenic differentiation of MSCs compared to the matrix alone, while no difference in growth was observed. Gao et al created a fibrin scaffold, in which cells and AuNPs were suspended simultaneously; these bioactive patches were shown to promote left ventricular function and decrease infarct size and apoptosis in the periscar boarder zone myocardium in swine models of acute MI.97 These studies of AuNP-containing scaffolds demonstrated reduced infarct and fibrotic size, as well as facilitated angiogenesis and cardiac function, which can be attributed at least in part to the enhanced expression of connexin 43 and atrial natriuretic peptide, and activation of the integrin-linked kinase(ILK)/serine-threonine kinase (p-AKT)/GATA4 pathway.49,143,144 Scaffolds containing Ag NPs evoke M2 polarization of macrophages in vitro;145 which may also play a role in cardioprotective action because M2 macrophages are capable of promoting cardiac recovery via the secretion of anti-inflammatory cytokines, collagen deposition, and neovascularization.146

Similarly, CNT also act synergically with poly(N-isopropylacrylamide) scaffolds containing adipose-derived stem cells;147 significant improvement of cardiac function and increased implantation and proliferation of stem cells has been observed with these scaffolds, compared with scaffolds without CNT.147 Selenium NPs148 and titania NPs53 have been shown to improve the mechanical and conductive properties of chitosan patches, promoting their ability to support proliferation and the synchronous activity of cells growing on these patches.

Mounting evidence demonstrates the unique benefits of using cardiac scaffolds with magnetic NPs such as SPIONs; these benefits include, but are not limited to, significant improvements in cell proliferation149 and assembly of electrochemical junctions.150 Given that magnetic manipulation enhances the therapeutic efficacy of iron oxide NPs in cardiac scaffolds, Chouhan and colleagues designed a magnetic actuator device by incorporating magnetic iron oxide NPs (MIONs) in silk nanofibers; this resulted in more controlled drug release properties, as well as the promotion of proliferation and maturation in CMs.151 Magnetic NPs can be used to label induced pluripotent stem cell (iPSC)-derived CMs via conjugation with antibodies against signal-regulatory protein . Zwi-Dantsis and colleagues reported the construction of tailored cardiac tissue microstructures, achieved by orienting MION-labelled cells along the applied field to impart different shapes without any mechanical support.152 However, the interactions between and effects of NPs and cells in scaffolds, and the cardioprotective efficacy of patches in which NP-labelled cells are suspended, require further elucidation.

Polymeric nanomaterials have also been investigated in the context of cardiac bioengineering materials; for instance, water-swollen polymer NPs have been used to prepare nanogels. With a 3D structure containing cross-linked biopolymer networks, nanogels can encapsulate, protect, and deliver various agents.83,153 PDA-coated tanshinone IIA NPs suspended in a ROS-sensitive, injectable hydrogel via PDA-thiol bonds significantly improved cardiac performance, accompanied by inhibition of the expression of inflammation factors in rat model.73 After implanting cryogel patches consisting of GelMa and linked conductive polypyrrole NPs154 or scaffolds of electrospun GelMA/polycaprolactone with GelMA-polypyrrole NPs,155 left ventricular (LV) ejection fraction (EF) has been shown to increase, with a concurrent decrease in infarct size, in MI animal models.

Progenitor or stem cell-based therapy in the form of injections and engineered cardiac patches, discussed in the previous section, has been recognized as a promising strategy to improve the cardiac niche and ameliorate adverse remodeling processes and fibrosis after acute MI.56,156,157 However, poor survival and low engraftment rates for transplanted cells are still major challenges in this field.157 Among possible optimization strategies, combining NPs with stem cell therapy is of great interest (Table 3).

Table 3 Studies Combining NPs and Cell Therapy Reported in the Last 7 Years

Accumulating evidence has shown two main mechanisms for NP-loaded cell therapy in the context of MI treatment. Firstly, various NP types could efficiently improve survival and cell proliferation, modulating differentiation of implanted cells in the ischemic microenvironment.62,158 Specifically, electrically driven nanomanipulators could guide cardiomyogenic differentiation of MSCs: in a previous study, electroactuated gold NPs were administrated with pulsed electric field stimulation, and tube-like morphological alterations were observed, along with upregulation of cardiac specific markers.143 Adipose-derived stem cells that load PLGA-simvastatin NPs promoted differentiation of these cells into SMCs and ECs, and had cardioprotective effects in a mouse model of MI induced by left anterior descending ligation.17 Secondly, engraftment rate is another important factor affecting treatment efficacy in this context.159 Zhang and colleagues designed silica-coated, MION-labelled endothelial progenitor cells; intravenous administration of these cells in a rat model of MI significantly improved cardiac performance, as indicated by echocardiogram, morphological, and histological evidence, and neovascularization. This indicates magnetic guidance may potentially address the problem of low levels of stem cell retention, which has typically been observed.51 In particular, NPs can link the therapeutic cells to injured CMs, thereby promoting cell anchorage and engraftment. To this end, Cheng and colleagues established a magnetic, bifunctional cell connector by conjugating NPs with two antibodies: one against cell determinant (CD)45, which is expressed on bone marrow-derived stem cells, and one against MLC. The magnetic core of this NP also enabled physical enrichment in ischemic heart tissue using external magnets.160 More than one mechanism may be involved in a study. Chen and colleagues fabricated a sustained release carrier of insulin-like growth factor (IGF), a pro-survival agent, via in situ growth of Fe3O4 NPs on MSNPs. In this study, the NPs promoted both the survival and retention of MSCs, and intramyocardial injection of the NP-labeled MSCs was able to ameliorate functional and histological damage without any obvious toxicity in vivo.161 However, SPION labeling does not seem to improve therapeutic efficiency, as demonstrated by Wang and colleagues in a study using hypoxia-preconditioned SPION-labeled adipose-derived stem cells (ASCs).162

Primary criticisms of cell-based therapies include their potential immunogenicity, arrhythmogenicity and tumorigenicity. It is widely accepted that the beneficial effects of cell-based therapy are mainly attributable to paracrine effects rather than directly replenishing lost CMs;56 researchers are therefore investigating of cell-free approaches. Exosomes have attractive properties including stable transport, homing to target tissues or cells, and penetration of biological barriers, as well as being more biocompatible with lower immunogenicity than cell-based approaches. Interestingly, post-MI circulating exosomes serve as important cardioprotective messengers.163,164 Manipulating their biodistribution has proven to be a viable strategy to reduce infarct size, promoting angiogenesis and ejection functions.21 However, from a therapeutic standpoint, the lack of control over endogenous exosome production and cargo encapsulation limits the use of this naturally-present mechanism for therapeutic enhancement. The low purity and weak targeting of natural exosomes are two further obstacles to overcome before clinical application. Strategies to address these include finding robust sources; optimized isolation methods for higher yields, efficiency and purity; and improving therapeutic payloads. These have been systematically summarized in other reviews.165167

AS is considered a low-grade, chronic inflammatory disease, characterized by accumulation and deposition of cholesterol in arteries, as well as remodeling of the extracellular matrix in the intima and inner media.12,168 Inflammation of ECs, proliferation of SMCs, and recruitment of monocytes and macrophages play a critical role in the development of AS. NPs allow for the packaging of large amounts of therapeutic compounds in a compact nanostructure, specifically targeting pathological mechanisms and attenuating atherogenesis. Optimization of the loaded drug and NP target together lead to enhanced efficacy while minimizing side effects.169 In this section, we summarize recent breakthroughs in the order of pathological progression, as shown in Table 4.

Primary prevention refers to control of the risk factors of AS, one of which is hypertension.170 PLA NPs have been shown to improve the efficacy of aliskiren, the first oral direct renin inhibitor and the first in a new class of antihypertensive agents.29 Encapsulation in nanocarriers also renders the application of anandamide viable, which was once limited; recent research revealed that this new therapy could lower blood pressure and LV mass index in rats.171 Similar results were observed in a study in which angiotensinogen was silenced using small hairpin RNA.172 NPs may also help to make more anti-hypertensive drugs available, reduce side effects such as asthma, and lessen the effective dosage by providing sustained drug release over time. The link between AS and diabetes mellitus, which describes a group of metabolic disorders, has also been investigated in numerous studies.173 Possible mechanisms include oxidative stress, altered protein kinase signaling, and epigenetic modifications. Cetin and colleagues successfully constructed NP-based drug delivery systems for the administration of metformin, an oral antihyperglycemic agent with low oral bioavailability and short biological half-life.174 NPs are also promising tools for improving the oral bioavailability of insulin, which is of great interest because oral insulin will significantly increase patients compliance.175,176

The inflammatory hypothesis of AS is now widely established, making selective targeting and accumulation of NPs in inflammatory lesions attractive therapeutic strategies. Targeting macrophages in apoE-/- mice has been shown to result in decreased phagocytosis and suppression of inflammatory genes in lesional macrophages, thus lessening burden of atherosclerotic plaques.177 Tom and colleagues used NPs consisting of high-density lipoprotein (HDL), a known atheroprotective bionanomaterial, as carriers for TNF receptor-associated factor in mice, and observed reductions in both leukocyte recruitment and macrophage activation.178 Both single-walled CNT and HDL-NPs have a favorable safety profile. In a pathological context, activated endothelial tissue expresses more adhesion molecules, such as selectins, than usual. These molecules are thus potential targets for cardiovascular nanomedicine. Glycoprotein Ib (GPIb)179 and biotinylated Sialyl Lewis A (sLeA)69 specifically bind to selectins, leading to the accumulation of conjugated NPs in injured vessels; an in vitro study demonstrated that GPIb-conjugated NPs could bind to target surfaces, where they were taken up by activated ECs under shear stress conditions. In another study, Sager and colleagues simultaneously inhibited five adhesion molecules associated with leukocyte recruitment in post-MI apoE-/- mice. Inflammation in plaque and ischemic heart, rendering acute coronary events and post-MI complications less likely to occur.180 However, targeting inflammatory process may have heterogeneous effects in humans because the targeting moieties and target receptors may be overexpressed in several different pathologic conditions in addition to AS. Oxidation is another factor involved in the development of AS. Upregulation of endothelial nitric oxide synthase (eNOS) leads to vascular construction and other AS-promoting effects. Pechanova and colleagues observed that the application of PLA NPs resulted in larger decreases in NOS than direct administration.29

Aside from these processes, avoiding plaque rupture and thrombosis could be another therapeutic aim. Nakashiro and colleagues showed that delivering pioglitazone via NPs inhibited plaque rupture in apoE-/- mice.181 The integrin 3 is upregulated in angiogenic vasculature, which is ubiquitous in plaque ruptures, which may lead to MI.182 3 integrin-targeted NPs provide a site-specific drug delivery platform that has been shown to successfully stabilize plaques in rabbits.182 Ji and colleagues used NPs composed of albumin with an average diameter of 225.6 nm to deliver a plasmid containing the tissue-type plasminogen activator gene (t-PA); this system plays a role in preventing thrombosis in addition to attenuating intimal thickness and proliferation of vascular SMCs.183 NPs consisting of engineered amphipathic cationic peptide and serine/threonine protein kinase JNK2 siRNA also reduces thrombotic risk, plaque necrotic area, and vascular barrier disorder in mice given the equivalent of a 14-week western diet.184

Innovation and development of therapies based on NPs in recent years has led to significant advances towards complete repair of the injured myocardium following acute MI. Nevertheless, developing clinically relevant solutions remains difficult for several reasons. Firstly, as shown in tables, there is little consistency among studies regarding the characteristics of NPs, their payloads, and their methods of administration, as well as methods used for evaluating cardiac repair. It can be difficult to control characteristics such as the size of the synthesized particles in a narrow range, even within single studies. Such significant heterogeneity can lead to differences in observed results in repeated experiments, or under different conditions. Secondly, although many studies have focused on the health effects of unintentional exposure to NPs by inhalation or ingestion,185,186 most of the studies on medical applications of NPs have not reported on toxicity of NP systems until recently.73 Remarkably, there has not been a consensus on NP-associated adverse effects in existing reports, making assessments of biocompatibility a priority for NP characterization.

NPs have emerged as a powerful tool for controlling cell signaling pathways in regenerative strategies using novel therapeutics and drugs that are unsuitable for direct administration. One advantage of the application of NP systems is the ability to release the drug payload or regulate gene expression in a stable and controlled manner. Therefore, many otherwise serious side effects, such as sudden arrhythmic deaths resulting from persistent and uncontrolled expression of miRNA by viral vectors, may be completely avoided.187 More research is required to develop stable and efficient methods of NP production, improve encapsulation efficiency of drugs, and achieve satisfactory targeting. In particular, a greater focus on investigating NP-based switches, including optical, electrical and magnetic methods, has enabled the regulation of cell signaling, exemplified by the development of a CuS NP-based photothermal switch.52 Optimizing tissue engineering scaffolds containing conductive NPs is a promising strategy for the protection of the myocardium after ischemia by mimicking the myocardial extracellular matrix. Improvements in understanding of cardiac repair mechanisms, and how these biomaterials may interfere with them, is therefore urgently needed. Furthermore, heart repair is complex and involves many processes, including apoptosis, angiogenesis, inflammatory infiltration, and fibrosis. Therefore, novel treatments should be designed using NP-based integrative strategies based on these multiple different mechanisms. However, its important to highlight that synergistic effects of different drug payloads, NPs, and NPcell combined strategies should be addressed, as not all may be compatible with one another. Future research should focus on these aspects to translate NP-based therapeutic strategies for MI into practical and effective clinical use.

The authors report no conflicts of interest in this work.

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Therapy and Prevention Strategies for Myocardial Infarction | IJN - Dove Medical Press

Cardiac Stem Cells Comments Off on Therapy and Prevention Strategies for Myocardial Infarction | IJN – Dove Medical Press | October 5th, 2021

Learn how these advanced 3D tissue models generated on the Mantarray platform can improve the physiological relevance of preclinical cardiac and skeletal muscle models, accelerating the discovery of new medicines.

TORONTO (PRWEB) October 05, 2021

3D cellular models and organs-on-chips are poised to add tremendous value by providing human data earlier in the drug discovery pipeline. There is intense interest in adopting these 3D models in preclinical and translational research, but their complex implementation has remained a roadblock for many labs.

In this webinar, Curi Bio will present its Mantarray platform, which represents an easy-to-use, flexible, and scalable system for generating 3D EMTs at high-throughput with the ability to measure contractility in parallel. The platform features a novel method of casting 3D tissues that can be easily performed by nearly any cell biology researcher and can be readily adapted to a variety of cell lines and extracellular matrices. In addition, Mantarrays novel magnetic sensing modality permits contractility measurement of 24 tissues in parallel and in real time, while the cloud data analysis portal takes the guesswork out of analyzing and comparing results across experiments.

Register for this webinar to hear an overview of the technology, along with application examples across various use cases, including:

Learn how these advanced 3D tissue models generated on the Mantarray platform can improve the physiological relevance of preclinical cardiac and skeletal muscle models, accelerating the discovery of new medicines.

Join Dr. Nicholas Geisse, Chief Science Officer at Curi Bio, for the live webinar on Friday, October 22, 2021 at 1pm EDT.

For more information, or to register for this event, visit Mantarray: Scalable Human-Relevant 3D-Engineered Cardiac and Skeletal Muscle Tissues for Safety and Efficacy Studies.

ABOUT XTALKS

Xtalks, powered by Honeycomb Worldwide Inc., is a leading provider of educational webinars to the global life science, food and medical device community. Every year, thousands of industry practitioners (from life science, food and medical device companies, private & academic research institutions, healthcare centers, etc.) turn to Xtalks for access to quality content. Xtalks helps Life Science professionals stay current with industry developments, trends and regulations. Xtalks webinars also provide perspectives on key issues from top industry thought leaders and service providers.

To learn more about Xtalks visit http://xtalks.comFor information about hosting a webinar visit http://xtalks.com/why-host-a-webinar/

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Mantarray: Scalable Human-relevant 3D Engineered Cardiac and Skeletal Muscle Tissues for Therapeutics Discovery Upcoming Webinar Hosted by Xtalks -...

Cardiac Stem Cells Comments Off on Mantarray: Scalable Human-relevant 3D Engineered Cardiac and Skeletal Muscle Tissues for Therapeutics Discovery Upcoming Webinar Hosted by Xtalks -… | October 5th, 2021

Today is International Heart Day, and Cosmos is looking back on the stories that make our hearts flutter.

Scientists have taken another step in the quest to create a Google map of the human body by putting together a detailed cellular and molecular map of the healthy heart.

An international team analysed almost half a million individual cells and cell nuclei from six different regions of the heart, obtained from 14 organ donors whose hearts were healthy but unsuitable for transplantation.

The result is theHeart Cell Atlas, which, shows the huge diversity of cells and reveals heart muscle cell types, cardiac protective immune cells and an intricate network of blood vessels. It also predicts how the cells communicate to keep the heart working.

Sometimes, the heart just stops for no perceivable reason. Sudden cardiac arrest (SCA) is a prevalent hidden killer, even for younger people: 40% of those who die from SCA are under 50 years old.

SCA is not as rare as we would like it to be, says cardiologist Elizabeth Paratz, whos undertaking her PhD at the Baker Heart and Diabetes Institute, Melbourne. In the last year in Victoria, 750 young people under 50 have suffered an SCA. This is almost exactly five times the road toll over the same time in this age group, yet we hear a lot more publicity about road fatalities in young people.

Paratz is researching the prevalence and causes of SCA, as well as looking at ways to diagnose it better. There are multiple causes of SCA, and theyre hard to pinpoint in young people.

The controversial use of stem cells to help patients recover from a heart attack may work, but not because it grows new heart muscle.

Research in mice has found that injecting stem cells into the heart triggers an immune response that makes the scar stronger and the heart beat more forcefully.

Thestudy, published in the journalNature, suggests the current practice of injecting stem cells into a patients blood may not be optimal: direct injection into the heart could be more effective.

In a preclinical trial on a beating human heart, researchers have found that a drug candidate developed from the venom of the worlds deadliest spider, the funnel web, may hold promise for heart attack treatment and transplants.

The researchers, led by Meredith Redd of the University of Queensland (UQ), and Sarah Scheuer of Victor Chang Cardiac Research Institute, tested a protein called Hi1a, found in the Fraser Island (Kgari) funnel web venom, on a beating heart that had been exposed to heart attack stresses.

After a heart attack, blood flow to the heart is reduced, resulting in a lack of oxygen to heart muscle, says Nathan Palpant of UQ, corresponding author of the paper.

The lack of oxygen causes the cell environment to become acidic, which combine to send a message for heart cells to die.

The Hi1a protein from spider venom blocks acid-sensing ion channels in the heart, so the death message is blocked, cell death is reduced, and we see improved heart cell survival.

The Chinese Finger Trap a tubular braided novelty beloved by kids and pranksters around the world provided the inspiration for a nifty bit of biotech that looks set to save sick kids a whole lot of heartache. Literally.

Pedro del Nido from Boston Childrens Hospital in the US heads a team that has designed a proof-of-concept device that promises to dramatically cut down on surgery for children with certain types of heart defects.

At present, kids with defective mitral and tricuspid heart valves must undergo surgery in which a corrective implant is installed. The problem, however, is that children grow: the heart increases in size, and requires at least one, and often several, further surgical interventions so that a correspondingly larger implant can be installed.

Needless to say, these repeated bouts of open-heart surgery are extremely traumatic and disruptive.

Now, however, Nido and Karp may have come up with an elegant and clever solution: an implant that grows with the organ.

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Five Heart Stories For International Heart Day - Cosmos

Cardiac Stem Cells Comments Off on Five Heart Stories For International Heart Day – Cosmos | October 5th, 2021

It is too late for conservation efforts to save the northern white rhinoceros, but with recent scientific advancements there may still be hope to bring back this beloved species. In a recently published paper, scientist Marisa Korody and her colleagues at San Diego Zoo Global (USA) and at the Department of Molecular Medicine at Scripps Research (USA) describe their exciting progress on using stem cells to revive the northern white rhino.

The northern white rhino is functionally extinct, meaning there are not enough of these rhinos left to save the species. In fact there are only two northern white rhinos left: a mother and a daughter. But for decades, scientists have preserved cell samples from 15 northern white rhinos containing enough genetic material to potentially bring this species back from the brink. These preserved samples hold fibroblast cells the type of skin cells that secrete collagen from white rhinos. With these scientists newly developed methods, fibroblast cells can be converted into something much more valuable: induced pluripotent stem cells. These stem cells can differentiate into any cell type in the body including heart cells, muscle cells, and reproductive cells.

In theory, by converting fibroblast cells into reproductive cells, scientists could create genetically unique rhino embryos. Alongside other assisted reproduction technologies, scientists could implant a new embryo into a closely-related southern white rhino, where the baby northern white rhino could develop as an otherwise normal pregnancy. By completing this process multiple times, scientists may be able to establish a stable population of northern white rhinos.

In 2011, this research team generated induced pluripotent stem cells from the samples of another endangered species, but unfortunately since this process was found to harm the recipient genomes, this method was largely unsuccessful. Despite this setback, in 2015 the authors met with colleagues worldwide to consider ways to save the northern white rhino, and they concluded that methods involving induced pluripotent stem cells may still be the most promising solution. Over the following years, the scientists worked to improve their methods, and these improvements are documented in their recent paper. These experiments represent the first step in a long-term plan to bring the northern white rhino back through assisted reproduction techniques.

Right from the start, the scientists faced a whole host of challenges. Through trial and error they modified the growth medium for the cells, optimizing it for rhinoceros cells. With their improved growth medium, scientists successfully generated induced pluripotent stem cell lines from 11 rhinoceros individuals. This has never been done before and represents a huge stride forward in the path to recovering this species.

Before trying to make their first rhino, the scientists needed to stress these induced pluripotent stem cells and sequence their genomes to determine if the cell quality is good enough to potentially produce new, viable rhinos. They maintained colonies of these cells in long-term cultures and exposed these colonies to different conditions to give insight into how resilient these cells could be. These tests demonstrated that long-term culture did not affect the potential for these cells to differentiate into cardiac lineage cells, confirming that these cells are stable long-term. The researchers also confirmed that these pluripotent cells could potentially produce gametes, the egg and sperm cells that are used for sexual reproduction. These advancements indicate that with these newly developed protocols, induced pluripotent stem cells are a promising tool that could someday help recover the northern white rhino.

Although this study includes some exciting results, there is still much work to do. For example, scientists must now sequence the genomes of the northern and southern white rhino so other researchers can analyze the stem cells ability to stay the same over time. Despite the work that still needs to be done, these promising advancements could someday help the northern white rhino population recover. This method may also work for saving other endangered or extinct species, as long as the genetic material needed is available. Long-term, these scientists plan to continue a series of experiments that could ultimately bring this beloved rhino, and potentially other endangered species, back from the brink of extinction.

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Stem cells may be the key to saving white rhinos from extinction - Sciworthy

Cardiac Stem Cells Comments Off on Stem cells may be the key to saving white rhinos from extinction – Sciworthy | October 5th, 2021

SYDNEY, Oct. 5, 2021 /PRNewswire/ -- Clinical stage drug development company Pharmaxis Ltd (ASX: PXS) today announced further positive results of data analysis from a phase 1c clinical trial (MF-101) studying its drug PXS-5505 in patients with the bone marrow cancer myelofibrosis for 28 days at three dosage levels.

Assessment with Pharmaxis' proprietary assays of the highest dose has shown inhibition of the target enzymes, LOX and LOXL2, at greater than 90% over a 24-hour period at day 7 and day 28. The trial safety committee has reviewed the results and having identified no safety signals, has cleared the study to progress to the phase 2 dose expansion phase where 24 patients will be treated at the highest dose twice a day for 6 months.

Pharmaxis CEO Gary Phillips said, "We are very pleased to have completed the dose escalation phase of this study with such clear and positive findings.We will now immediately progress to the phase 2 dose expansion study where we aim to show PXS-5505 is safe to be taken longer term with the disease modifying effects that we have seen in the pre-clinical models. The trial infrastructure and funding is in place and we are on track to complete the study by the end of 2022."

Independent, peer-reviewed research has demonstrated the upregulation of several lysyl oxidase family members in myelofibrosis.The level of inhibition of LOX achieved in the current study at all three doses significantly exceeds levels that caused disease modifying effects with PXS-5505 in pre-clinical models of myelofibrosis with improvements in blood cell count, diminished spleen size and reduced bone marrow fibrosis. LOXL2 was inhibited to a similar degree and based on pre-clinical work such high inhibition is likely replicated for other LOX family members (LOXL1, 3 and 4).[1] Study data can be viewed in the full announcement.

Commenting on the results of the trial, Dr Gabriela Hobbs, Assistant Professor, Medicine, Harvard Medical School & Clinical Director, Leukaemia, Massachusetts General Hospital said, "Despite improvements in the treatment of myelofibrosis, the only curative therapy remains an allogeneic stem cell transplantation, a therapy that many patients are not eligible for due to its morbidity and mortality. None of the drugs approved to date consistently or meaningfully alter the fibrosis that defines this disease. PXS-5505 has a novel mechanism of action by fully inhibiting all LOX enzymes. An attractive aspect of this drug is that so far in healthy controls and in this phase 1c study in myelofibrosis patients, the drug appears to be very well tolerated. This is meaningful as approved drugs and those that are undergoing study, are associated with abnormal low blood cell counts. Preliminary data thus far, demonstrate that PXS-5505 leads to a dramatic, >90% inhibition of LOX and LOXL2 at one week and 28 days. This confirms what's been shown in healthy controls as well as mouse models, that this drug can inhibit the LOX enzymes in patients. Inhibiting these enzymes is a novel approach to the treatment of myelofibrosis by preventing the deposition of fibrosis and ultimately reversing the fibrosis that characterizes this disease."

The phase 1c/2a trial MF-101 cleared by the FDA under the Investigational New Drug (IND) scheme aims to demonstrate that PXS-5505, the lead asset in Pharmaxis' drug discovery pipeline, is safe and effective as a monotherapy in myelofibrosis patients who are intolerant, unresponsive or ineligible for treatment with approved JAK inhibitor drugs. Trial sites will now open to recruit myelofibrosis patients into the 6-month phase 2 study in Australia, South Korea, Taiwan and the USA.

An effective pan-LOX inhibitor for myelofibrosis would open a market that is conservatively estimated at US$1 billion per annum.

While Pharmaxis' primary focus is the development of PXS-5505 for myelofibrosis, the drug also has potential in several other cancers including liver and pancreatic cancer where it aims to breakdown the fibrotic tissue in the tumour and enhance the effect of chemotherapy treatment.

Trial Design

Name of trial

PXS5505-MF-101: A phase 1/2a study to evaluate safety, pharmacokinetic and pharmacodynamic dose escalation and expansion study of PXS-5505 in patients with primary, post-polycythaemia vera or post-essential thrombocythemia myelofibrosis

Trial number

NCT04676529

Primary endpoint

To determine the safety of PXS-5505 in patients with myelofibrosis

Secondary endpoints

Blinding status

Open label

Placebo controlled

No

Trial design

Randomised, multicentre, 4 week duration phase 1 (dose escalation) followed by 6 month phase 2 (dose expansion)

Treatment route

Oral

Treatment frequency

Twice daily

Dose level

Dose escalation: three escalating doses

Dose expansion: one dose

Number of subjects

Dose escalation: minimum of three patients to maximum of 18 patients

Dose expansion: 24 patients

Subject selection criteria

Patients with primary or secondary myelofibrosis who are intolerant, unresponsive or ineligible for treatment with approved JAK inhibitor drugs

Trial locations

Dose escalation: Australia (2 sites) and South Korea (4 sites)

Dose expansion: Australia, Korea, Taiwan, USA

Commercial partners involved

No commercial partner

Reference: (1) doi.org/10.1002/ajh.23409

AUTHORISED FOR RELEASE TO ASX BY:

Pharmaxis Ltd Disclosure Committee. Contact: David McGarvey, Chief Financial Officer and Company Secretary: T +61 2 9454 7203, E david.mcgarvey@pharmaxis.com.au

Join the Pharmaxis mailing listhere

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About Pharmaxis

Pharmaxis Ltd is an Australian clinical stage drug development company developing drugs for inflammatory and fibrotic diseases, with a focus on myelofibrosis. The company has a highly productive drug discovery engine built on its expertise in the chemistry of amine oxidase inhibitors, with drug candidates in clinical trials. Pharmaxis has also developed two respiratory products which are approved and supplied in global markets, generating ongoing revenue.

Pharmaxis is developing its drug PXS-5505 for the bone marrow cancer myelofibrosis which causes a build up of scar tissue that leads to loss of production of red and white blood cells and platelets. The US Food and Drug Administration has granted Orphan Drug Designation to PXS-5055 for the treatment of myelofibrosis and permission under an Investigational Drug Application (IND) to progress a phase 1c/2 clinical trial that began recruitment in Q1 2021. PXS5505 is also being investigated as a potential treatment for other cancers such as liver and pancreatic cancer.

Other drug candidates being developed from Pharmaxis' amine oxidase chemistry platform are targeting fibrotic diseases such as kidney fibrosis, NASH, pulmonary fibrosis and cardiac fibrosis; fibrotic scarring from burns and other trauma; and inflammatory diseases such as Duchenne Muscular Dystrophy.

Pharmaxis has developed two products from its proprietary spray drying technology that are manufactured and exported from its Sydney facility; Bronchitol for cystic fibrosis, which is approved and marketed in the United States, Europe, Russia and Australia; and Aridol for the assessment of asthma, which is approved and marketed in the United States, Europe, Australia and Asia.

Pharmaxis is listed on the Australian Securities Exchange (PXS). Its head office, manufacturing and research facilities are in Sydney, Australia. http://www.pharmaxis.com.au

About PXS-5505

PXS-5505 is an orally taken drug that inhibits the lysyl oxidase family of enzymes, two members LOX and LOXL2 are strongly upregulated in human myelofibrosis. In pre-clinical models of myelofibrosis PXS-5505 reversed the bone marrow fibrosis that drives morbidity and mortality in myelofibrosis and reduced many of the abnormalities associated with this disease. It has already received IND approval and Orphan Drug Designation from the FDA.

Myelofibrosis is a disorder in which normal bone marrow tissue is gradually replaced with a fibrous scar-like material. Over time, this leads to progressive bone marrow failure. Under normal conditions, the bone marrow provides a fine network of fibres on which the stem cells can divide and grow. Specialised cells in the bone marrow known as fibroblasts make these fibres.

In myelofibrosis, chemicals released by high numbers of platelets and abnormal megakaryocytes (platelet forming cells) over-stimulate the fibroblasts. This results in the overgrowth of thick coarse fibres in the bone marrow, which gradually replace normal bone marrow tissue. Over time this destroys the normal bone marrow environment, preventing the production of adequate numbers of red cells, white cells and platelets. This results in anaemia, low platelet counts and the production of blood cells in areas outside the bone marrow for example in the spleen and liver, which become enlarged as a result.

Myelofibrosis can occur at any age but is usually diagnosed later in life, between the ages of 60 and 70 years. The cause of myelofibrosis remains largely unknown. It can be classified as either JAK2 mutation positive (having the JAK2 mutation) or negative (not having the JAK2 mutation).

Source: Australian Leukemia Foundation: https://www.leukaemia.org.au/disease-information/myeloproliferative-disorders/types-of-mpn/primary-myelofibrosis/

Forward-looking statements

Forwardlooking statements in this media release include statements regarding our expectations, beliefs, hopes, goals, intentions, initiatives or strategies, including statements regarding the potential of products and drug candidates. All forward-looking statements included in this media release are based upon information available to us as of the date hereof. Actual results, performance or achievements could be significantly different from those expressed in, or implied by, these forward-looking statements. These forward-looking statements are not guarantees or predictions of future results, levels of performance, and involve known and unknown risks, uncertainties and other factors, many of which are beyond our control, and which may cause actual results to differ materially from those expressed in the statements contained in this document. For example, despite our efforts there is no certainty that we will be successful in developing or partnering any of the products in our pipeline on commercially acceptable terms, in a timely fashion or at all. Except as required by law we undertake no obligation to update these forward-looking statements as a result of new information, future events or otherwise.

CONTACT:

Media: Felicity Moffatt: T +61 418 677 701, E felicity.moffatt@pharmaxis.com.au

Investor relations:Rudi Michelson (Monsoon Communications) T +61 411 402 737, E rudim@monsoon.com.au

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Pharmaxis Cleared To Progress To Phase 2 Bone Marrow Cancer Trial - WAGM

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Shahid Akhter, editor, ETHealthworld spoke to Dr. Dinesh Bhurani, Director, Department of Hemato-Oncology & Bone Marrow Transplant, Rajiv Gandhi Cancer Institute and Research Centre, to know about the progress of NPRD and the challenges associated with blood stem cell transplants.

How do you think the National Policy for Rare Diseases will impact the treatment of patients suffering from rare blood disorders? Will it help reduce the lag that we often see in policy and practice when it comes to healthcare systems?National Policy on Rare Diseases is a step-in right direction and must be welcomed by the Indian medical fraternity. It not only recognizes rare diseases for the first time in India but also has brought forward the possibility of affordable treatment for life-threatening rare diseases which were not previously covered under the national health program. The policy advocates access for treatment through center of excellences, crowd funding and financial assistance.

The NPRD in a bid to enable patients suffering from rare blood disorders has laid emphasis on the option of one-time curative treatment through hematopoietic stem cell transplant for diseases such as Severe Combined Immunodeficiency (SCID), Chronic Granulomatous disease, Wiskott Aldrich Syndrome, Osteopetrosis, and Fanconi Anaemia. By committing to provide a Rs. 20 lakhs cover for the one-time treatment cost of diseases falling under Group 1 through the umbrella scheme of Rashtriya Arogya Nidhi, the NPRD has attempted to provide coverage to almost 40 per cent of the population who are eligible under the Pradhan Mantri Jan Arogya Yojana. The NPRD as a policy that advocates affordable and accessible healthcare and has the potential to lead to the creation of a conducive healthcare ecosystem whereby multisectoral partnerships can collaboratively work towards reduction in the lag between policy and practice often seen otherwise, thereby leading people to live healthier and fuller lives.

Another reason for low number of donors in India is the misconception that stem cell donation comes with a cost to donor. This idea is completely misplaced and untrue as the cost of procedure starting from sample collection, donation and travel is free of cost, and covered under the cost of treatment of a patient for whom the donation is needed. Added to this is the fact that the number of organizations working in the country in the space of blood stem cell transplant is limited at best, thus awareness generation as compared to other health issues is nominal. However, the situation is gradually evolving and ICMR in its 2021 guidelines has gone on to recognize seven registries across the country as active stakeholders in this ecosystem. This recognition by ICMR will hopefully lead to greater awareness generation.

For blood stem cell transplant knowledge is key in establishing patient donor linkage, and by storing the requisite information with them, these registries do just that. Technology is a tool that has been successfully leveraged by stakeholders in the ecosystem to establish linkages. The Hap- E Search is one such tool that has been used by hospitals in the country to find donor matches for their patients. This software is perhaps one of the most enabling tools available to us in the ecosystem, as it helps find HLA matches not just in the country but across the world. This software is now being used by many government hospitals like AIIMS, Delhi and PGIMER Chandigarh. Once the matching donor is found via the HAP-E Search, the donor is encouraged to make the donation, provided counselling and support to donate blood stem cells, and post donation the stem cells are transported to the patients location.

The NPRD proposed crowdfunding and PPP models to ensure more patients availing treatment for rare diseases. How beneficial do you think such partnerships can be to enable blood stem cell transplant ecosystem?Treatment for rare diseases has been found to be expensive across the world. It is thus that despite stem cell transplants being a proven effective solution in the case of some blood disorders, affordability continues to be a challenge for patients and their families. With treatment costs ranging anywhere between Rs. 15-45 lakh, it remains out of reach for most patients in the country. Also, blood disorders, classified as rare, have limited infrastructure in health systems, networks, and subsidies for patients to access treatments are few. In such a scenario, crowdfunding is definitely a feasible option for patients that would ensure that they do not have to forego treatment due to a paucity of resources.

As per the NPRD, the money raised through crowdfunding would directly get credited to the treatment centre thus ensuring that there is adequate linkage. Further, the public private partnership model suggested by the government has enabled it to avail the support of non- governmental and not-for- profit agencies present in the country. This is truly commendable as not only will this ensure more patient donor linkage in the blood stem transplant ecosystem but will also lead to greater awareness generation and registrations of donors as well. One significant organization that has already partnered with the government in this arena is the DKMS BMST Foundation India. With over 50,000 blood stem cell donors registered with them, this organization has been steadily working towards enabling the ecosystem. In the case of rare diseases, it is imperative that stakeholders do not work in isolation and the government working alongside the private can lead to greater hope for many patients with greater amenities and facilities for treatment being made accessible to them.

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DBMR has added another report named Exosome therapeutic Market with information Tables for recorded and figure years addressed with Chats and Graphs spread through Pages with straightforward definite examination. The a-list report concentrates on broad assessment of the market development expectations and limitations. The systems range from new item dispatches, extensions, arrangements, joint endeavors, organizations, to acquisitions. This report includes profound information and data on what the markets definition, characterizations, applications, and commitment and furthermore clarifies the drivers and restrictions of the market which is gotten from SWOT investigation. Worldwide market examination report serves a great deal for the business and presents with answer for the hardest business questions. While making Exosome therapeutic Market report, examination and investigation has been completed with one stage or the mix of a few stages relying on the business and customer necessities.

Market definition canvassed in the predominant Exosome therapeutic Market advertising report investigates the market drivers that show factors causing ascend in the market development and market limitations which demonstrate the components causing fall in the market development. It helps clients or other market members to know about the issues they might confront while working in this market throughout a more extended timeframe. This statistical surveying report additionally concentrates on utilization of market, central participants included, deals, value, income and portion of the overall industry with volume and an incentive for every area. The greatness and straightforwardness proceeded in Exosome therapeutic Market business research report makes acquire the trust and dependence of part organizations and clients.

Global Exosome Therapeutic Market By Type (Natural Exosomes, Hybrid Exosomes), Source (Dendritic Cells, Mesenchymal Stem Cells, Blood, Milk, Body Fluids, Saliva, Urine Others), Therapy (Immunotherapy, Gene Therapy, Chemotherapy), Transporting Capacity (Bio Macromolecules, Small Molecules), Application (Oncology, Neurology, Metabolic Disorders, Cardiac Disorders, Blood Disorders, Inflammatory Disorders, Gynecology Disorders, Organ Transplantation, Others), Route of administration (Oral, Parenteral), End User (Hospitals, Diagnostic Centers, Research & Academic Institutes), Geography (North America, Europe, Asia-Pacific and Latin America)

Market Analysis and Insights:Global Exosome Therapeutic Market

Exosome therapeutic market is expected to gain market growth in the forecast period of 2019 to 2026. Data Bridge Market Research analyses that the market is growing with a CAGR of 21.9% in the forecast period of 2019 to 2026 and expected to reach USD 31,691.52 million by 2026 from USD 6,500.00 million in 2018. Increasing prevalence of lyme disease, chronic inflammation, autoimmune disease and other chronic degenerative diseases are the factors for the market growth.

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Exosomes are used to transfer RNA, DNA, and proteins to other cells in the body by making alteration in the function of the target cells. Increasing research activities in exosome therapeutic is augmenting the market growth as demand for exosome therapeutic has increased among healthcare professionals.

Increased number of exosome therapeutics as compared to the past few years will accelerate the market growth. Companies are receiving funding for exosome therapeutic research and clinical trials. For instance, In September 2018, EXOCOBIO has raised USD 27 million in its series B funding. The company has raised USD 46 million as series a funding in April 2017. The series B funding will help the company to set up GMP-compliant exosome industrial facilities to enhance production of exosomes to commercialize in cosmetics and pharmaceutical industry.

Increasing demand for anti-aging therapies will also drive the market. Unmet medical needs such as very few therapeutic are approved by the regulatory authority for the treatment in comparison to the demand in global exosome therapeutics market will hamper the market growth market. Availability of various exosome isolation and purification techniques is further creates new opportunities for exosome therapeutics as they will help company in isolation and purification of exosomes from dendritic cells, mesenchymal stem cells, blood, milk, body fluids, saliva, and urine and from others sources. Such policies support exosome therapeutic market growth in the forecast period to 2019-2026.

This exosome therapeutic market report provides details of market share, new developments, and product pipeline analysis, impact of domestic and localised market players, analyses opportunities in terms of emerging revenue pockets, changes in market regulations, product approvals, strategic decisions, product launches, geographic expansions, and technological innovations in the market. To understand the analysis and the market scenario contact us for anAnalyst Brief, our team will help you create a revenue impact solution to achieve your desired goal.

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Competitive Landscape and Exosome Therapeutic Market Share Analysis

Global exosome therapeutic market competitive landscape provides details by competitor. Details included are company overview, company financials, revenue generated, market potential, investment in research and development, new market initiatives, global presence, production sites and facilities, company strengths and weaknesses, product launch, product trials pipelines, concept cars, product approvals, patents, product width and breadth, application dominance, technology lifeline curve. The above data points provided are only related to the companys focus related to global exosome therapeutic market.

The major players covered in the report are evox THERAPEUTICS, EXOCOBIO, Exopharm, AEGLE Therapeutics, United Therapeutics Corporation, Codiak BioSciences, Jazz Pharmaceuticals, Inc., Boehringer Ingelheim International GmbH, ReNeuron Group plc, Capricor Therapeutics, Avalon Globocare Corp., CREATIVE MEDICAL TECHNOLOGY HOLDINGS INC., Stem Cells Group among other players domestic and global. Exosome therapeutic market share data is available for Global, North America, Europe, Asia-Pacific, and Latin America separately. DBMR analysts understand competitive strengths and provide competitive analysis for each competitor separately.

Many joint ventures and developments are also initiated by the companies worldwide which are also accelerating the global exosome therapeutic market.

For instance,

Partnership, joint ventures and other strategies enhances the company market share with increased coverage and presence. It also provides the benefit for organisation to improve their offering for exosome therapeutics through expanded model range.

Global Exosome Therapeutic Market Scope and Market Size

Global exosome therapeutic market is segmented of the basis of type, source, therapy, transporting capacity, application, route of administration and end user. The growth among segments helps you analyse niche pockets of growth and strategies to approach the market and determine your core application areas and the difference in your target markets.

Based on type, the market is segmented into natural exosomes and hybrid exosomes. Natural exosomes are dominating in the market because natural exosomes are used in various biological and pathological processes as well as natural exosomes has many advantages such as good biocompatibility and reduced clearance rate compare than hybrid exosomes.

Exosome is an extracellular vesicle which is released from cells, particularly from stem cells. Exosome functions as vehicle for particular proteins and genetic information and other cells. Exosome plays a vital role in the rejuvenation and communication of all the cells in our body while not themselves being cells at all. Research has projected that communication between cells is significant in maintenance of healthy cellular terrain. Chronic disease, age, genetic disorders and environmental factors can affect stem cells communication with other cells and can lead to distribution in the healing process. The growth of the global exosome therapeutic market reflects global and country-wide increase in prevalence of autoimmune disease, chronic inflammation, Lyme disease and chronic degenerative diseases, along with increasing demand for anti-aging therapies. Additionally major factors expected to contribute in growth of the global exosome therapeutic market in future are emerging therapeutic value of exosome, availability of various exosome isolation and purification techniques, technological advancements in exosome and rising healthcare infrastructure.

Rising demand of exosome therapeutic across the globe as exosome therapeutic is expected to be one of the most prominent therapies for autoimmune disease, chronic inflammation, Lyme disease and chronic degenerative diseases treatment, according to clinical researches exosomes help to processes regulation within the body during treatment of autoimmune disease, chronic inflammation, Lyme disease and chronic degenerative diseases. This factor has increased the research activities in exosome therapeutic development around the world for exosome therapeutic. Hence, this factor is leading the clinician and researches to shift towards exosome therapeutic. In the current scenario the exosome therapeutic are highly used in treatment of autoimmune disease, chronic inflammation, Lyme disease and chronic degenerative diseases and as anti-aging therapy as it Exosomes has proliferation of fibroblast cells which is significant in maintenance of skin elasticity and strength.

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Exosome therapeutic Market Country Level Analysis

The global exosome therapeutic market is analysed and market size information is provided by country by type, source, therapy, transporting capacity, application, route of administration and end user as referenced above.

The countries covered in the exosome therapeutic market report are U.S. and Mexico in North America, Turkey in Europe, South Korea, Australia, Hong Kong in the Asia-Pacific, Argentina, Colombia, Peru, Chile, Ecuador, Venezuela, Panama, Dominican Republic, El Salvador, Paraguay, Costa Rica, Puerto Rico, Nicaragua, Uruguay as part of Latin America.

Country Level Analysis, By Type

North America dominates the exosome therapeutic market as the U.S. is leader in exosome therapeutic manufacturing as well as research activities required for exosome therapeutics. At present time Stem Cells Group holding shares around 60.00%. In addition global exosomes therapeutics manufacturers like EXOCOBIO, evox THERAPEUTICS and others are intensifying their efforts in China. The Europe region is expected to grow with the highest growth rate in the forecast period of 2019 to 2026 because of increasing research activities in exosome therapeutic by population.

The country section of the report also provides individual market impacting factors and changes in regulation in the market domestically that impacts the current and future trends of the market. Data points such as new sales, replacement sales, country demographics, regulatory acts and import-export tariffs are some of the major pointers used to forecast the market scenario for individual countries. Also, presence and availability of global brands and their challenges faced due to large or scarce competition from local and domestic brands, impact of sales channels are considered while providing forecast analysis of the country data.

Huge Investment by Automakers for Exosome Therapeutics and New Technology Penetration

Global exosome therapeutic market also provides you with detailed market analysis for every country growth in pharma industry with exosome therapeutic sales, impact of technological development in exosome therapeutic and changes in regulatory scenarios with their support for the exosome therapeutic market. The data is available for historic period 2010 to 2017.

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Exosome therapeutic Market Report- Trends Key Programs Analysis and Competitive Landscape and Forecast 2028 Amite Tangy Digest - Amite Tangy Digest

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Trained as a chemist, biophysicist, internist, and cardiologist, Mark Yeager is eager to propel the Frost Institute for Chemistry and Molecular Science into a leading research center.

Even in his youth Mark Yeager could picture the door to his future. Scuffed, chipped, and almost black from layers of varnish, the old, wooden door had a frosted window with five words stenciled in glossy black: Laboratory of Dr. Mark Yeager.

Yet Yeager, the inaugural executive director of the University of Miamis Frost Institute for Chemistry and Molecular Science (FICMS), is quite happy that his new lab in the 94,000-square-foot building slated to open late next year wont even have a door. The $60 million facilitys open floor plan was designed to encourage the free flow of people and ideasand help transform the University into one of the worlds premier research centers for improving the health of humans and that of our planet.

That is the vision, but its not a fantastical vision, said Yeager, a distinguished biophysicist and cardiologist whose top priority is attracting a diverse and elite group of scientists as the institutes first faculty. It is achievable, and it will happen because the University has not wavered in its commitment to elevate STEM (science, technology, engineering, and mathematics) to advance scientific discovery. Theres something going on here thats organic and alive and excitingand Im thrilled to be part of it.

Yeager, whose own groundbreaking research focuses on the molecular causes of heart disease and viral infections, trained as a chemist at Carnegie-Mellon University, as a physician and biophysicist at Yale University and as an internist and cardiologist at Stanford University. He spent two decades at Scripps Research in California, where he established his first independent laboratory, served as the director of research in cardiology, and helped launch the Skaggs Clinical Scholars Program in Translational Research. He has also served as a consultant and scientific and clinical advisor to several biotech companies.

Now he is transitioning to the University from the University of Virginia School of Medicine (UVA), where he chaired the Department of Molecular Biophysics and Biochemistry for nearly a dozen years and helped establish the Sheridan G. Snyder Translational Research Building. At UVA, he also established one of the nations five regional centers for cryo-electron microscopy (cryoEM)the technique he advanced for flash-freezing, imaging, and studying proteins and other macromolecules in their near-natural state.

It is exciting to see the progress being made on the evolution of our Frost Institutes, starting with Data Science and Computing and now the emergence of Chemistry and Molecular Science. We are fortunate to have Mark overseeing our Frost Institute for Chemistry and Molecular Science and working across the entire institutionhis interdisciplinary knowledge and perspective on chemistry are essential for our success, said Jeffrey Duerk, executive vice president for academic affairs and provost. Mark brings a wealth of knowledge and experience to the University of Miami and we are looking forward to his impactful leadership continuing as we move forward.

Yeager said he knew he was making the right career move on his first visit to the University last November. Although the COVID-19 pandemic had curtailed in-person learning and suspended new construction, he heard the unmistakable sound of heavy equipment as he walked past the royal palms and fountain at the end of Memorial Drive, where the five-story FICMS now stands.

I could see an excavation area and heard a cacophony of construction noise where I had a hunch the institute should be, he recalled. That told me that the University was all in. They had made this commitment to fortify STEM and to do transformational science and nothing was going to stop them. In spite of the pandemic, it was all systems go.

The Universitys longtime benefactors, Phillip and Patricia Frost, enabled that commitment in 2017, when they announced their landmark $100 million gift to establish the Frost Institutes for Science and Engineering, now a key initiative of the Roadmap to Our New Centurythe strategic plan guiding the University toward its centennial mark. The umbrella organization for a group of multidisciplinary research centers patterned after the National Institutes of Health and its network of affiliated institutes, the Frost Institutes were envisioned to translate interdisciplinary research into solutions for real-world problems.

Though Yeager officially started his new role on June 1, he has been heavily involved in planning the FICMS' interior for months. He recently placed a $20 million order to equip the facility with five different electron microscopy instruments that chemists, molecular scientists, and engineers will use to explore the molecular structure of exquisitely beam-sensitive soft materials like proteins, hard materials such as metal alloys, as well as nanomaterials comprised of soft and hard components. Along with the buildings state-of-the-art technology and the Universitys research infrastructure, hes confident its location in the heart of the Coral Gables campus will help him recruit a diverse and elite group of scientists who are exploring challenging avenues of impactful researchsomething he has been driven to do almost his entire life.

An occasional songwriter, guitar player, and jogger who in his younger days ran 18 marathons, Yeager was always fascinated by scientific discoveries that illuminated unknown and unseen worlds. A child of the Sputnik era who began entering science fairs in junior high, he began forging his own career as a physician-scientist while in high school in Colorado Springs, Colorado, where his father, an agricultural economist, settled his family after a number of job-related moves.

Inspired by an experiment in Scientific American magazine, he convinced physicians in the therapeutic radiology department at Penrose Hospital to irradiate his fruit flies so he could compare the effects of administering different doses of radiation on their eye pigments. Delivered in Styrofoam cups, his experiments on what is now called dose fractionationand used to reduce tissue damage during cancer treatmentswon him first place in the U.S. Department of Agricultures 1967 International Science Fair and a research stint in an insect toxicology lab in Berkeley, California.

The following summer, when Yeager returned to Penrose Hospital to work as an orderly, he realized that he loved patient care as much as laboratory research and began plotting how he could pursue both careers.

I just got incredible satisfaction from helping patients get out of bed and into a wheelchair, transfer to a gurney, learn to use crutches, recalled Yeager, who joins the University as one of its 100 Talents for 100 Years, a Roadmap initiative to add 100 new endowed chairs to the faculty by the Universitys 2025 centennial. But I also loved chemistry. I loved physics. I loved too many things.

After earning his undergraduate degree in chemistry from Carnegie-Mellon, he was accepted to the Medical Scientist Training Program at Yale University, where, along with his medical degree, he earned his masters degree and doctorate in molecular biophysics and biochemistry. There, he encountered the first of many trailblazing scientists, including two future Nobel laureates, who would influence his lifes work. His Ph.D. advisor, Lubert Stryer, was particularly influential. Stryer authored a premier textbook of biochemistry, pioneered fluorescence-based techniques to explore the motions of biological macromolecules, and made fundamental discoveries on the molecular basis of vision. Yeagers graduate work on rhodopsin, a photoreceptor membrane protein, triggered his fascination with elucidating the molecular bases for such diseases as sudden cardiac death, heart attacks, HIV-1, and other viral infections.

Yeager completed his medical residency and specialized fellowship training in cardiovascular medicine at Stanford University Medical Center, where he managed the pre- and post-operative care of heart transplant patients and wrote 13 chapters in the book Handbook of Difficult Diagnoses.

He also continued exploring cellular biology in the laboratory of Nigel Unwin, who had collaborated with future Nobel laureate Richard Henderson to pioneer the use of cryoEM to determine the molecular structure of membrane proteinsand inspired Yeagers groundbreaking research on gap junction channels. The electrical conduits that connect every cell in the body to its neighbor, gap junction channels play a critical role in maintaining the normal heartbeat.

That research, which Yeager continued at Scripps and at UVA, explained how gap junction channels behave in their normal state, and during an injured state, such as a heart attack. His quest to answer another question particularly relevant todayhow viruses enter host cells, replicate, and assemble infectious particlesis exemplified by his breakthrough research on the assembly, structure, and maturation of HIV-1, the virus that causes AIDS.

Today, those insights, which Yeager humbly calls a few bricks in the edifice of science, hold important clues for developing new, more effective therapies to prevent HIV-1 infection, repair injured tissue, and treat cancer and cardiovascular diseasethe kind of impactful research that the FICMS was designed to advance with collaborative partners across the University, and beyond.

As a pioneer in the field of cryo-transmission electron microscopy, a forefront technology in materials and biological research, Marks expertise and knowledge will position the University as aleader in these cutting-edge fields, said Leonidas Bachas, dean of the College of Arts and Sciences who served as the initial interim director of the FICMS. We look forward to having him lead the Frost Institute for Chemistry and Molecular Science as we continue to advance the sciences, innovate, and expand research collaborations with our faculty and industry partners.

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Distinguished physician-scientist takes the helm of first Frost Institute - University of Miami

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A genetic syndrome that affects bones

Osteogenesis Imperfecta (OI) is a hereditary disorder occurring in 1:10,000 births and characterised by osteopenia (bone loss) and skeletal fragility (fractures). Secondary features include short stature, skeletal deformities, blue sclera and dentinogenesis imperfect. (1) There is a large clinical variability in OI, and severity ranges from mild to lethal, based on radiological characteristics. Genetically, OI is a collagen-related syndrome. Type I collagen is a heterotrimeric helical structure synthesized by bone-forming cells (osteoblasts), and it constitutes the most abundant protein of the skeletal organic matrix. (2) Synthesis of type I collagen is a complex process. (3) Collagen molecules are cross-linked into fibrils (which confer tensile strength to the bones). Those are then mineralised by hydroxy-apatites (which provides compressive strength) and assembled into fibres.

Dominant mutations in either the COL1A1 or the COLA1A2 genes are responsible for up to 90% of all OI cases. These mutations (more than 1,000 of which have been identified) lead to impairment of collagen structure and production, which in either quantitative or qualitative bone extracellular matrix (ECM) defects. Mutations affecting ECM structure have serious health consequences because the skeleton protects visceral organs and the central nervous system and provides structural support. Bones also store fat in the yellow bone marrow found within the medullary cavity, whilst the red marrow located at the end of long bones is the site of haematopoiesis. In addition, the ECM constitutes a reservoir of phosphate, calcium, and growth factors, and is involved in trapping dangerous molecules.

Stem cell therapy for OI aims to improve bone quality by harnessing the ability of mesenchymal stem cells (MSC) to differentiate into osteoblasts, with the rationale that donor cells would engraft into bones, produce normal collagen and function as a cell replacement. Stem cells have, therefore, been proposed for the treatment of OI (4) and, in particular, prenatal foetal stem cell therapy (foetal stem cells injected into a foetus, i.e. foetal-to-foetal) approach, which offers a promising route to effective treatment. (5) Human foetal stem cells are more primitive than stem cells isolated from adult tissues and present advantageous characteristics compared to their adult counterparts, i.e. they possess a higher level of plasticity, differentiate more readily into specific lineages, grow faster, senesce later, express higher levels of adhesion molecules, and are smaller in size. (6,7) Prenatal cell therapy capitalises on the small size of the foetus and its immunological naivete. In addition, stem cells delivered in utero benefit from the expansion of endogenous stem cells and may prevent organ injury before irreversible damage. (8)

However, human foetal stem cells used are isolated from either foetal blood drawn by cardiac puncture, either during termination of pregnancy or during ongoing pregnancy, albeit using an invasive procedure associated with a high risk of morbidity and mortality for both the foetus and the mother (9). Foetal cells can also be isolated from the first-trimester liver (following termination of pregnancy) and such cells are currently used in The Boost Brittle Bones Before Birth (BOOSTB4) clinical trial, which aims to investigate the safety and efficacy of transplanting foetal derived MSCs prenatally and/or in early postnatal life to treat severe Osteogenesis Imperfecta (OI) (10). Alternatively, foetal stem cells can be isolated during ongoing pregnancy from the amniotic fluid, either during mid-trimester amniocentesis or at birth (11,12) or from the chorionic villi of the placenta during first-trimester chorionic villi sampling (13).

We have demonstrated that human fetal stem cells isolated from first trimester blood possess superior osteogenic differentiation potential compared to adult stem cells isolated from bone marrow and to fetal stem cells isolated from first trimester liver. We showed that in utero transplantation of these cells in an experimental model of severe OI resulted in a drastic 75% decrease in fracture rate incidence and skeletal brittleness, and improvement of bone strength and quality.(14) A similar outcome was obtained using placenta-derived foetal stem cells (15) and amniotic fluid stem cells following perinatal transplantation into experimental models. (16,17)

Understanding the mechanisms of action of donor cells will enable the engineering of donor cells with superior efficacy to stimulate bone formation and strengthen the skeleton. Despite their potential to differentiate down the osteogenic lineage, there is little evidence that donor cells contribute to regenerating bones through direct differentiation, due to the very low level of donor cell engraftment reported in all our studies. When placed in an osteogenic microenvironment in vitro, foetal stem cells readily differentiate into osteoblasts and produce wild type collagen molecules. However, there are insufficient proofs that collagen molecules of donor cell origin contribute to the formation of the host bone ECM to confer superior resistance to fracture.

It is now well accepted that stem cells can influence the behaviour of target cells through the release of paracrine factors and, therefore, contribute to tissue regeneration indirectly. We have indeed recently shown that donor stem cells stimulate the differentiation of resident osteoblasts, which were unable to fully mature in the absence of stem cell treatment. (16,17) We are now focusing our efforts on understanding the precise molecular mechanisms by which donor cells improve skeletal health to counteract bone fragility caused by various OI-causative mutations.

References

Please note: This is a commercial profile

2019. This work is licensed under aCC BY 4.0 license.

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Stem cell & gene therapy to treat osteogenesis imperfecta: hype or hope - Open Access Government

Bone Marrow Stem Cells Comments Off on Stem cell & gene therapy to treat osteogenesis imperfecta: hype or hope – Open Access Government | October 5th, 2021

Myles Glass has spent the past several years living life on the sidelines in a wheelchair, wishing for a better day. That day came in November 2020.

INDIANAPOLIS A 13-year-old boy living with sickle cell disease has been given a second chance at life, thanks to his mother.

Myles Glass has been through more in his young life than most adults. For the past few years, Glass has spent his days in and out of Riley Hospital for Children.

"[I] kind of have to look on the bright side of things. Being in the hospital, I meet new nurses and kids who go through what I go through. It's kind of hard to go through that at my age," Glass said.

He was diagnosed with sickle cell disease as a newborn. According to the Centers for Disease Control and Prevention, African Americans make up the largest number of people with the disease in the U.S.

Sickle cell disease is an inherited condition that impacts red blood cells and causes pain, infections and extreme fatigue. These symptoms keep Glass from doing things he loves.

"For him, it's kind of like we have to have him in a bubble," said his mother, Melissa Sanders.

Glass has spent the past several years living life on the sidelines in a wheelchair, wishing for a better day.

"[I would] hope that one day, I can do what kids do, like playing football and basketball," Glass said.

That day came in November 2020 when his mother donated bone marrow for a stem cell transplant, curing him of sickle cell disease.

"I was able to give him a second life with being a donor so that he can somewhat be a normal kid," Sanders said.

Riley Hospital for Children Dr. Seethal Jacob, who has been working with Glass and his family, said one baby every two minutes is born with sickle cell disease. She also said studies show there is a clear disparity for funding for this disease.

"There's been a lot of neglect when it comes to the disease itself. I think it's important to pay attention to the population it affects. I think that likely tells the story why sickle cell disease has been a neglected disease for so long," Jacob said.

Despite his challenges, Glass is staying positive and making strides in his physical therapy at Riley Hospital for Children.

"He's already been through harder things than most people will ever go through. I think anything else in life is going to be a piece of cake," said his physical therapist, Sarah Johnson.

"This gives me a glimpse of hope that even though you may have been diagnosed with this disease, it's not the end of the world," Sanders said.

For Glass, this is just the beginning. He hopes his story encourages other people living with sickle cell disease to keep moving forward.

"I know it's hard now, but you'll get through it. You'll be able to do what kids do your own age," Glass said.

Click here for more information on sickle cell disease and treatment options.

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Indianapolis mother gives 13-year-old son with sickle cell disease a 2nd chance at life - WTHR

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SYDNEY, Oct. 5, 2021 /PRNewswire/ -- Clinical stage drug development company Pharmaxis Ltd (ASX: PXS) today announced further positive results of data analysis from a phase 1c clinical trial (MF-101) studying its drug PXS-5505 in patients with the bone marrow cancer myelofibrosis for 28 days at three dosage levels.

Assessment with Pharmaxis' proprietary assays of the highest dose has shown inhibition of the target enzymes, LOX and LOXL2, at greater than 90% over a 24-hour period at day 7 and day 28. The trial safety committee has reviewed the results and having identified no safety signals, has cleared the study to progress to the phase 2 dose expansion phase where 24 patients will be treated at the highest dose twice a day for 6 months.

Pharmaxis CEO Gary Phillips said, "We are very pleased to have completed the dose escalation phase of this study with such clear and positive findings.We will now immediately progress to the phase 2 dose expansion study where we aim to show PXS-5505 is safe to be taken longer term with the disease modifying effects that we have seen in the pre-clinical models. The trial infrastructure and funding is in place and we are on track to complete the study by the end of 2022."

Independent, peer-reviewed research has demonstrated the upregulation of several lysyl oxidase family members in myelofibrosis.The level of inhibition of LOX achieved in the current study at all three doses significantly exceeds levels that caused disease modifying effects with PXS-5505 in pre-clinical models of myelofibrosis with improvements in blood cell count, diminished spleen size and reduced bone marrow fibrosis. LOXL2 was inhibited to a similar degree and based on pre-clinical work such high inhibition is likely replicated for other LOX family members (LOXL1, 3 and 4).[1] Study data can be viewed in the full announcement.

Commenting on the results of the trial, Dr Gabriela Hobbs, Assistant Professor, Medicine, Harvard Medical School & Clinical Director, Leukaemia, Massachusetts General Hospital said, "Despite improvements in the treatment of myelofibrosis, the only curative therapy remains an allogeneic stem cell transplantation, a therapy that many patients are not eligible for due to its morbidity and mortality. None of the drugs approved to date consistently or meaningfully alter the fibrosis that defines this disease. PXS-5505 has a novel mechanism of action by fully inhibiting all LOX enzymes. An attractive aspect of this drug is that so far in healthy controls and in this phase 1c study in myelofibrosis patients, the drug appears to be very well tolerated. This is meaningful as approved drugs and those that are undergoing study, are associated with abnormal low blood cell counts. Preliminary data thus far, demonstrate that PXS-5505 leads to a dramatic, >90% inhibition of LOX and LOXL2 at one week and 28 days. This confirms what's been shown in healthy controls as well as mouse models, that this drug can inhibit the LOX enzymes in patients. Inhibiting these enzymes is a novel approach to the treatment of myelofibrosis by preventing the deposition of fibrosis and ultimately reversing the fibrosis that characterizes this disease."

The phase 1c/2a trial MF-101 cleared by the FDA under the Investigational New Drug (IND) scheme aims to demonstrate that PXS-5505, the lead asset in Pharmaxis' drug discovery pipeline, is safe and effective as a monotherapy in myelofibrosis patients who are intolerant, unresponsive or ineligible for treatment with approved JAK inhibitor drugs. Trial sites will now open to recruit myelofibrosis patients into the 6-month phase 2 study in Australia, South Korea, Taiwan and the USA.

An effective pan-LOX inhibitor for myelofibrosis would open a market that is conservatively estimated at US$1 billion per annum.

While Pharmaxis' primary focus is the development of PXS-5505 for myelofibrosis, the drug also has potential in several other cancers including liver and pancreatic cancer where it aims to breakdown the fibrotic tissue in the tumour and enhance the effect of chemotherapy treatment.

Trial Design

Name of trial

PXS5505-MF-101: A phase 1/2a study to evaluate safety, pharmacokinetic and pharmacodynamic dose escalation and expansion study of PXS-5505 in patients with primary, post-polycythaemia vera or post-essential thrombocythemia myelofibrosis

Trial number

NCT04676529

Primary endpoint

To determine the safety of PXS-5505 in patients with myelofibrosis

Secondary endpoints

Blinding status

Open label

Placebo controlled

No

Trial design

Randomised, multicentre, 4 week duration phase 1 (dose escalation) followed by 6 month phase 2 (dose expansion)

Treatment route

Oral

Treatment frequency

Twice daily

Dose level

Dose escalation: three escalating doses

Dose expansion: one dose

Number of subjects

Dose escalation: minimum of three patients to maximum of 18 patients

Dose expansion: 24 patients

Subject selection criteria

Patients with primary or secondary myelofibrosis who are intolerant, unresponsive or ineligible for treatment with approved JAK inhibitor drugs

Trial locations

Dose escalation: Australia (2 sites) and South Korea (4 sites)

Dose expansion: Australia, Korea, Taiwan, USA

Commercial partners involved

No commercial partner

Reference: (1) doi.org/10.1002/ajh.23409

AUTHORISED FOR RELEASE TO ASX BY:

Pharmaxis Ltd Disclosure Committee. Contact: David McGarvey, Chief Financial Officer and Company Secretary: T +61 2 9454 7203, E [emailprotected]

Join the Pharmaxis mailing listhere

Follow us on LinkedInand Twitter

About Pharmaxis

Pharmaxis Ltd is an Australian clinical stage drug development company developing drugs for inflammatory and fibrotic diseases, with a focus on myelofibrosis. The company has a highly productive drug discovery engine built on its expertise in the chemistry of amine oxidase inhibitors, with drug candidates in clinical trials. Pharmaxis has also developed two respiratory products which are approved and supplied in global markets, generating ongoing revenue.

Pharmaxis is developing its drug PXS-5505 for the bone marrow cancer myelofibrosis which causes a build up of scar tissue that leads to loss of production of red and white blood cells and platelets. The US Food and Drug Administration has granted Orphan Drug Designation to PXS-5055 for the treatment of myelofibrosis and permission under an Investigational Drug Application (IND) to progress a phase 1c/2 clinical trial that began recruitment in Q1 2021. PXS5505 is also being investigated as a potential treatment for other cancers such as liver and pancreatic cancer.

Other drug candidates being developed from Pharmaxis' amine oxidase chemistry platform are targeting fibrotic diseases such as kidney fibrosis, NASH, pulmonary fibrosis and cardiac fibrosis; fibrotic scarring from burns and other trauma; and inflammatory diseases such as Duchenne Muscular Dystrophy.

Pharmaxis has developed two products from its proprietary spray drying technology that are manufactured and exported from its Sydney facility; Bronchitol for cystic fibrosis, which is approved and marketed in the United States, Europe, Russia and Australia; and Aridol for the assessment of asthma, which is approved and marketed in the United States, Europe, Australia and Asia.

Pharmaxis is listed on the Australian Securities Exchange (PXS). Its head office, manufacturing and research facilities are in Sydney, Australia. http://www.pharmaxis.com.au

About PXS-5505

PXS-5505 is an orally taken drug that inhibits the lysyl oxidase family of enzymes, two members LOX and LOXL2 are strongly upregulated in human myelofibrosis. In pre-clinical models of myelofibrosis PXS-5505 reversed the bone marrow fibrosis that drives morbidity and mortality in myelofibrosis and reduced many of the abnormalities associated with this disease. It has already received IND approval and Orphan Drug Designation from the FDA.

About Myelofibrosis

Myelofibrosis is a disorder in which normal bone marrow tissue is gradually replaced with a fibrous scar-like material. Over time, this leads to progressive bone marrow failure. Under normal conditions, the bone marrow provides a fine network of fibres on which the stem cells can divide and grow. Specialised cells in the bone marrow known as fibroblasts make these fibres.

In myelofibrosis, chemicals released by high numbers of platelets and abnormal megakaryocytes (platelet forming cells) over-stimulate the fibroblasts. This results in the overgrowth of thick coarse fibres in the bone marrow, which gradually replace normal bone marrow tissue. Over time this destroys the normal bone marrow environment, preventing the production of adequate numbers of red cells, white cells and platelets. This results in anaemia, low platelet counts and the production of blood cells in areas outside the bone marrow for example in the spleen and liver, which become enlarged as a result.

Myelofibrosis can occur at any age but is usually diagnosed later in life, between the ages of 60 and 70 years. The cause of myelofibrosis remains largely unknown. It can be classified as either JAK2 mutation positive (having the JAK2 mutation) or negative (not having the JAK2 mutation).

Source: Australian Leukemia Foundation: https://www.leukaemia.org.au/disease-information/myeloproliferative-disorders/types-of-mpn/primary-myelofibrosis/

Forward-looking statements

Forwardlooking statements in this media release include statements regarding our expectations, beliefs, hopes, goals, intentions, initiatives or strategies, including statements regarding the potential of products and drug candidates. All forward-looking statements included in this media release are based upon information available to us as of the date hereof. Actual results, performance or achievements could be significantly different from those expressed in, or implied by, these forward-looking statements. These forward-looking statements are not guarantees or predictions of future results, levels of performance, and involve known and unknown risks, uncertainties and other factors, many of which are beyond our control, and which may cause actual results to differ materially from those expressed in the statements contained in this document. For example, despite our efforts there is no certainty that we will be successful in developing or partnering any of the products in our pipeline on commercially acceptable terms, in a timely fashion or at all. Except as required by law we undertake no obligation to update these forward-looking statements as a result of new information, future events or otherwise.

CONTACT:

Media: Felicity Moffatt: T +61 418 677 701, E [emailprotected]

Investor relations:Rudi Michelson (Monsoon Communications) T +61 411 402 737, E [emailprotected]

SOURCE Pharmaxis Limited

http://www.pharmaxis.com.au

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Pharmaxis Cleared To Progress To Phase 2 Bone Marrow Cancer Trial - PRNewswire

Bone Marrow Stem Cells Comments Off on Pharmaxis Cleared To Progress To Phase 2 Bone Marrow Cancer Trial – PRNewswire | October 5th, 2021

TUCSON, Ariz., Oct. 5, 2021 /PRNewswire/ --On November 15th, 2021, healthcare professionals and the general public are invited to participate in World Cord Blood Day 2021 (www.WorldCordBloodDay.org) via a free online conference and live educational events being held around the globe. Registration is now open (free, public welcome).

Cord blood is the blood left in the umbilical cord and placenta following the birth of a child. It is rich in life-saving stem cells. While cord blood has been used for over 30 years, Covid-19 has renewed interest in this medical resource given its unique regenerative qualities and the fact that most cord blood currently stored was collected prior to the pandemic. These units are naturally Covid-free, an advantage over many other stem cell sources. Yet, cord blood is still thrown away as medical waste in the majority of births worldwide. Education is key to changing this practice and World Cord Blood Day 2021 will provide the perfect opportunity for OBGYNs, midwives, transplant doctors, nurses, parents and students to learn about this vital medical resource.

During World Cord Blood Day 2021, participants will learn how cord blood is used to treat over 80 life-threatening diseases such as leukemia and lymphoma, bone marrow failure, immune deficiency diseases and inherited blood disorders such as thalassemia and sickle cell disease. Leading transplant doctors and researchers will also highlight cord blood's role in the emerging fields of gene therapy and regenerative medicine to potentially treat cerebral palsy, autism, stroke and more.

Organized by Save the Cord Foundation, a 501c3 non-profit, World Cord Blood Day 2021 is officially sponsored by QuickSTAT Global Life Science Logistics, recognized leader in medical shipping and healthcare logistics. Inspiring Partners include Be the Match (NMDP), World Marrow Donor Association (WMDA-Netcord), AABB Center for Cellular Therapies, Cord Blood Association, and the Foundation for the Accreditation of Cellular Therapy (FACT).

"QuickSTAT, part of Kuehne+Nagel, is proud to sponsor the 5th annual World Cord Blood Day to help support and educate the healthcare community and expectant parents about the life-saving value of cord blood stem cells. We're excited to play a role in the research and development of cord blood derivative therapies by providing logistics supply chain solutions to cord blood, biotech and pharmaceutical companies worldwide," said Monroe Burgess, VP Life Science Commercial Marketing, QuickSTAT.

Visit http://www.WorldCordBloodDay.org to learn how you can participate. Show your support on social media: @CordBloodDay, #WorldCordBloodDay, #WCBD21

About Save the Cord FoundationSave the Cord Foundation (a 501c3 non-profit) was established to advance cord blood education providing non-commercial information to health professionals and the public regarding methods for saving cord blood, as well as current applications and the latest research. http://www.SaveTheCordFoundation.org.

About QuickSTAT Global Life Science LogisticsEvery day, QuickSTAT, a part of Kuehne+Nagel, safely and reliably moves thousands of critical shipments around the world. For over forty years, QuickSTAT has been entrusted with transporting human organs and tissue for transplant or research, blood, blood products, cord blood, bone marrow, medical devices, and personalized medicine, 24/7/365. QuickSTAT's specially trained experts work with hospitals, laboratories, blood banks and medical processing centers, and utilize the safest routes to ensure integrity, temperature control and chain of custody throughout the transportation process. Learn more at http://www.quickstat.aero.

Contact:Charis Ober(520) 419-0269[emailprotected]

SOURCE Save the Cord Foundation

http://www.SaveTheCordFoundation.org

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Ready to Treat Over 80 Life-Threatening Diseases, Discover the Potential of Cord Blood during World Cord Blood Day 2021 - PRNewswire

Bone Marrow Stem Cells Comments Off on Ready to Treat Over 80 Life-Threatening Diseases, Discover the Potential of Cord Blood during World Cord Blood Day 2021 – PRNewswire | October 5th, 2021

A student has completed the London Marathon alongside the stem cell donor who saved her life.

icky Lawrence, 21, from Moseley, Birmingham, was diagnosed with severe aplastic anaemia in 2008, when she was eight years old, a condition in which the bone marrow does not produce an adequate number of new blood cells.

Thanks to Elliott Brock, a physiotherapist from Mersea Island, Essex, Ms Lawrence received a transplant that same year.

Ms Lawrence sent Mr Brock a letter in 2015 and the pair met for the first time.

Fast forward to 2021 and they have just completed the London Marathon in support of Anthony Nolan.

Ms Lawrence, who is in her fourth year of a medical degree at Newcastle University, told the PA news agency that completing the marathon was absolutely amazing.

She said: Crossing the finish line was so emotional, not just because wed run 26 miles, but running 26 miles alongside the man who saved your life is a pretty big feat.

Ms Lawrence added: A big slogan of Anthony Nolan is without your support, there is no cure.

Without Elliott donating his stem cells to a stranger, I would not be here. I wouldnt have made it to Christmas. I would never have had the opportunities Ive had to go to university, to study abroad, to play hockey.

Him donating his stem cells gave me a second life and there are still so many people that need a transplant that are not finding the matches they need, especially among the ethnic minority community.

Unfortunately if you are of ethnic minority background, you only have a 37% chance of finding a match.

Mr Brock, 42, who wore a mask and cape during the race, said: That was a tongue of check nod [to the fact that the] easiest way to be called a hero is to donate your bone marrow.

I cannot emphasise to people enough that it is pain-free.

He added: It was just a day of celebration for London to celebrate having their marathon back.

The crowds were amazing and obviously to be side-by-side with the girl whose life, through the amazing work of Anthony Nolan, I managed to save sort of 13 years ago was just surreal really.

Its a lovely story of how my simple act made such a massive difference and we are able to celebrate it so many years after.

Anthony Nolan chief executive Henny Braund said: We are so grateful to Vicky and Elliott for running to raise funds and awareness of Anthony Nolan and the lifesaving work that we do.

Every day five Vickys, patients with blood cancer or a blood disorder, start their search for an Elliott.

If youre aged 16-30 and in good health, please consider joining the Anthony Nolan stem cell register. You could potentially save a life.

More information on how to join the stem cell register can be found at: http://www.anthonynolan.org/help-save-a-life/join-stem-cell-register

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Student completes London Marathon with the man who saved her life - Independent.ie

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StemExpress to use utilize the Thermo Fisher Accula rapid PCR testing system to provide event attendees with accurate results in 30 minutes.

Published: Oct. 5, 2021 at 2:33 PM CDT|Updated: 4 hours ago

SACRAMENTO, Calif., Oct. 5, 2021 /PRNewswire/ --StemExpress is proud to announce that they will be the official COVID-19 testing provider for 2021's Meeting on the Mesa, a hybrid event bringing together great minds in the cell and gene biotech sphere. It has partnered with Alliance for Regenerative Medicine to comply with the newly implemented California state COVID-19 vaccination and testing policy regarding gatherings with 1,000 or more attendees. This partnership will allow the vital in-person networking aspect of the event to commence while protecting the health and safety of participants and attendees.

In-person networking commences at the 2021 Cell and Gene Meeting on the Mesa with COVID-19 testing options provided by StemExpress.

As a leading global provider of human biospecimen products, StemExpress understands the incredible impact that Meeting on the Mesa has on the industry and has been a proud participant for many years. For over a decade, StemExpress has provided the cell and gene industry with vital research products and holds valued partnerships with many of this year's participants. As such, it understands the immense value that in-person networking provides and is excited to help bring this element back to the meeting safely and responsibly.

StemExpress has been a trusted provider of widescale COVID-19 testing solutions since early 2020 - providing testing for government agencies, public health departments, private sector organizations, and the public nationwide. For Meeting on the Mesa, StemExpress is offering convenient testing options for unvaccinated attendees and those traveling from outside of the country. Options will include take-home RT-PCR COVID Self-Testing Kits and on-site, rapid PCR testing for the duration of the event. The self-testing kit option allows attendees to test for COVID in the days leading up to the event for a seamless admission and the days following the event to confirm they haven't been exposed. The on-site rapid testing option utilizes the new Thermo Fisher Accula, offering in-person testing at the event with results in around 30 minutes. StemExpress is excited to bring these state-of-the-art COVID testing solutions to the frontlines of the Cell & Gene industry to allow for safe in-person connections.

The StemExpress partnership with Alliance for Regenerative Medicine seeks to empower the entire cell and gene industry with a long-awaited opportunity to return to traditional networking practices. It is well known that innovation doesn't exist in a vacuum - allowing great minds to come together is a sure way to spur scientific growth and advance cutting-edge research, giving hope for future cures.

Cell and Gene Meeting on the Mesa will take place October 12th, 2021, through October 14th, 2021, at Park Hyatt Aviara,7100 Aviara Resort Drive Carlsbad, CA 92011. To learn more about the event, please visit MeetingOnTheMesa.com.

For more information about COVID testing solutions for businesses and events, visit https://www.stemexpress.com/covid-19-testing/.

About StemExpress:

Founded in 2010 and headquartered in Sacramento, California, StemExpress is a leading global biospecimen provider of human primary cells, stem cells, bone marrow, cord blood, peripheral blood, and disease-state products. Its products are used for research and development, clinical trials, and commercial production of cell and gene therapies by academic, biotech, diagnostic, pharmaceutical, and contract research organizations (CRO's).

StemExpress has over a dozen global distribution partners and seven (7) brick-and-mortar cellular clinics in the United States, outfitted with GMP certified laboratories. StemExpress runs its own non-profit supporting STEM initiatives, college and high school internships, and women-led organizations. It is registered with the U.S. Food and Drug Administration (FDA) and is continuously expanding its network of healthcare partnerships, which currently includes over 50 hospitals in Europe and 3 US healthcare systems - encompassing 31 hospitals, 35 outpatient facilities, and over 200 individual practices and clinics.

StemExpress has been ranked by Inc. 500 as one of the fastest-growing companies in the U.S.

About the Alliance for Regenerative Medicine:

The Alliance for Regenerative Medicine (ARM) is the leading international advocacy organization dedicated to realizing the promise of regenerative medicines and advanced therapies. ARM promotes legislative, regulatory, reimbursement and manufacturing initiatives to advance this innovative and transformative sector, which includes cell therapies, gene therapies and tissue-based therapies. Early products to market have demonstrated profound, durable and potentially curative benefits that are already helping thousands of patients worldwide, many of whom have no other viable treatment options. Hundreds of additional product candidates contribute to a robust pipeline of potentially life-changing regenerative medicines and advanced therapies. In its 12-year history, ARM has become the voice of the sector, representing the interests of 400+ members worldwide, including small and large companies, academic research institutions, major medical centers and patient groups. To learn more about ARM or to become a member, visit http://www.alliancerm.org.

Media Contact: Anthony Tucker, atucker@stemexpress.com

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The above press release was provided courtesy of PRNewswire. The views, opinions and statements in the press release are not endorsed by Gray Media Group nor do they necessarily state or reflect those of Gray Media Group, Inc.

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StemExpress Partners with the Alliance for Regenerative Medicine to Provide COVID-19 Testing for the Cell and Gene Meeting on the Mesa - WSAW

Bone Marrow Stem Cells Comments Off on StemExpress Partners with the Alliance for Regenerative Medicine to Provide COVID-19 Testing for the Cell and Gene Meeting on the Mesa – WSAW | October 5th, 2021