People who develop Parkinson's disease at a young age might have malfunctioning brain cells -- even before birth. A drug used to treat pre-cancers of the skin may help treat the condition, finds a new study. At least 500,000 people in the US are diagnosed with Parkinson's every year, a majority of them over the age of 60. But about 10% of them develop the condition young -- between 21 and 50 years. People develop the disease when the brain nerve cells that make dopamine -- a substance that helps coordinate muscle movement -- malfunction or die. Consequently, these patients experience difficulty moving due to stiff muscles and tremors. Most often, young-onset patients have a family history of Parkinsons disease.

"Young-onset Parkinson's is especially heartbreaking because it strikes people at the prime of life," said Dr. Michele Tagliati, director of the Movement Disorders Program, vice-chair, and professor in the Department of Neurology at Cedars-Sinai. "This exciting new research provides hope that one day we may be able to detect and take early action to prevent this disease in at-risk individuals," says Dr Tagliati, co-author of the study.

In this study, the team turned cells from these Parkinson's patients into a kind of stem cell, meaning they turned adult cells into an embryo-like state. These cells can be programmed into developing into any cell types, including muscles, nerves or heart, for instance. The team turned these stem cells into cells that produce dopamine and grew them in their lab.

"Our technique gave us a window back in time to see how well the dopamine neurons might have functioned from the very start of a patient's life," said senior author Dr. Clive Svendsen, director of the Cedars-Sinai Board of Governors Regenerative Medicine Institute and professor of Biomedical Sciences and Medicine at Cedars-Sinai.

When the team observed these cells, they saw an abnormal accumulation of a toxic protein called alpha-synuclein, which is seen in patients with most forms of Parkinson's disease. This accumulation could be the result of malfunctioning "trash cans".

These trash cans of the dopamine-producing cells called lysosomes are tasked with the breaking down and the disposing of proteins - but they failed to do so in young-onset Parkinson's patients. As a result, the toxic protein buildup ends up damaging dopamine-producing cells.

"The cells of the brain cannot dispose of the toxic protein called synuclein a hallmark of dying neurons in Parkinsons disease even before birth. This does not kill the neurons until much later in life though," the researchers tell MEA WorldWide (MEAWW). "Now we know that this starts so early in life we can think about ways to reduce this protein early and use this model as a way to detect whether the Parkinsons is starting," they add.

Further, the team also tested several drugs that might reverse the abnormality seen in these cells. They found that that one drug, dubbed PEP005, which is already approved by the Food and Drug Administration for treating precancers of the skin, proved effective in lab studies and mice. The drug brought down the levels of the toxic protein.

Encouraged by these positive results in the young-onset patients, the team is now testing whether these findings hold in patients who develop Parkinson's after the age of 50. "While we have shown our drug is effective in this cell model, it needs to be validated in actual patients before it is proven to be a treatment for Parkinsons. These studies are being planned," they add.

The study has been published in Nature Medicine.

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People who develop Parkinson's before 50 may have been born with damaged brain cells, says study - MEAWW

Skin Stem Cells Comments Off on People who develop Parkinson’s before 50 may have been born with damaged brain cells, says study – MEAWW | January 28th, 2020

The Cosmetic Skin Care report makes available a thoughtful overview of product specification, technology, product type and production analysis taking into account major factors such as revenue, cost, and gross margin. The report is sure to offer brilliant solution to the challenges and problems faced by industry. This business document comprises of extensive study about miscellaneous market segments and regions, emerging trends, major market drivers, challenges and opportunities in the market. This Cosmetic Skin Care business document also displays the key developments in the industry with respect to current scenario and the approaching advancements.

Global cosmetic skin care market is set to witness a substantial CAGR of 5.5% in the forecast period of 2019- 2026. The report contains data of the base year 2018 and historic year 2017. Increasing self-consciousness among population and rising demand for anti- aging skin care products are the factor for the market growth.

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Customize report of Global Cosmetic Skin Care Market as per customers requirement also available.Market Segmentations:Global Cosmetic Skin Care Market is segmented on the basis of

Market Segmentations in Details:By Product

By Application

By Gender

By Distribution Channel

By GeographyNorth America

Europe

Asia-Pacific

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Middle East & Africa

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Global cosmetic skin care market is highly fragmented and the major players have used various strategies such as new product launches, expansions, agreements, joint ventures, partnerships, acquisitions, and others to increase their footprints in this market. The report includes market shares of cosmetic skin care market for Global, Europe, North America, Asia-Pacific, South America and Middle East & Africa.

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Cosmetic Skin Care Market is Thriving with Rising Latest Trends by 2025 | Top Players- L'Oral, Unilever, New Avon Company, Este Lauder Companies,...

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Parkinsons disease is an illness that most often affects older people, but new research suggests it may actually be present in the brain right from birth and even earlier. Scientists from Cedars-Sinai have now found that in the brains of young-onset Parkinsons patients, malfunctioning neurons are always there but it takes 20 to 30 years for the symptoms to accumulate. Thankfully, a drug thats already on the market could help prevent the disease from taking hold if caught early enough.

Parkinsons disease primarily affects neurons in the brain that produce dopamine, eventually causing muscle weakness and stiffness, tremors, and balance problems. Most of the time, the disease is diagnosed in older people over the age of 60, but around 10 percent of cases occur in those aged between 21 and 50.

In a new study, scientists from Cedars-Sinai set out to investigate whether there were any early warning signs in the neurons of patients whod been diagnosed with Parkinsons before they turned 50. To do so, they created induced pluripotent stem cells (IPSCs) from young-onset Parkinsons patients, which can then be turned into almost any other cells in the body.

The researchers used the IPSCs to grow dopamine neurons in lab dishes. As they watched them develop, the team noticed that cell structures called lysosomes were malfunctioning. These structures are responsible for breaking down unneeded or worn-out proteins so when they dont work as well as they should, proteins begin to pile up. And one such protein that the team spotted in higher amounts is called alpha-synuclein, which is implicated in many forms of Parkinsons.

"Our technique gave us a window back in time to see how well the dopamine neurons might have functioned from the very start of a patients life, says Clive Svendsen, senior author of the study. "What we are seeing using this new model are the very first signs of young-onset Parkinsons. It appears that dopamine neurons in these individuals may continue to mishandle alpha-synuclein over a period of 20 or 30 years, causing Parkinsons symptoms to emerge.

Next up, the team investigated whether the condition could potentially be treated or even prevented. After testing a series of drugs, they found one that looked promising PEP005, which has already been approved by the FDA for use against skin precancers. The researchers found that PEP005 works to reduce the levels of alpha-synuclein, as well as another abnormally-abundant enzyme called protein kinase C, whose role in Parkinson's remains unclear.

The treatment looks promising, but for now its only been shown to work in mice and lab-grown cells, so it wont necessarily translate to human trials. The team plans to continue working on this, as well as figuring out how to adapt PEP005 for use in the brain at the moment, its only available as a topical gel, since it's for treating skin cancer.

The research was published in the journal Nature Medicine.

Source: Cedars-Sinai

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Study suggests Parkinson's present from birth and may be preventable - New Atlas

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What are progenitor cells?

Every cell in the human body, and that of other mammals, originates from stem cell precursors. Progenitor cells are descendants of stem cells that then further differentiate to create specialized cell types.There are many types of progenitor cells throughout the human body. Each progenitor cell is only capable of differentiating into cells that belong to the same tissue or organ. Some progenitor cells have one final target cell that they differentiate to, while others have the potential to terminate in more than one cell type.

Stem cells share two qualifying characteristics. Firstly, all stem cells have the potential to differentiate into multiple types of cells. Secondly, stem cells are capable of unlimited self-replication via asymmetric cell division, a process known as self-renewal.There are two broad categories of stem cells found in all mammals. The first are embryonic stem cells. These cells arise from the inner cell mass of the blastocyst in an early-stage embryo. Embryonic stem cells are the blueprint used to create every cell in the body. Because they can be used to create any type of cell, they are known as pluripotent.

The second type of stem cells found in mammals are adult stem cells (or somatic stem cells). Unlike pluripotent embryonic stem cells, adult stem cells are more limited in relation to the type of cells that they become. Unlike embryonic stem cells that could be used to create any cell, adult stem cells are limited to generating cell types within a specific lineage, such as blood cells or cells of the central nervous system. This level of differentiation potential is termed multipotent.

Stem cells create two types of progeny: more stem cells or progenitor cells. All progenitor cells are descendants of stem cells. When it comes to cell differentiation, they fall on the spectrum between stem cells and fully differentiated (mature) cells.

Whilst stem cells have indefinite replication (left) progenitor cells can at most differentiate into multiple types of specialized cell (right).

Function:

Cellular repair or maintenance

Cell Potency:

Multipotent, oligopotent, or unipotent

Self-renewal:

Limited

Origin:

Stem cells

Creates:

Further differentiated cells (either progenitor cells of mature/fully differentiated cells)

Progenitor cells are an intermediary step involved in the creation of mature cells in human tissues and organs, the blood, and the central nervous system.

The human central nervous system (CNS) contains three types of fully differentiated cells: neurons, astrocytes and oligodendrocytes. The latter two are collectively known as glial cells.Every neuron, oligodendrocyte and astrocyte in the CNS evolves from the differentiation of neural progenitor cells (NPCs). NPCs themselves are produced by multipotent neural stem cells (NSCs). Both NPCs and NSCs are termed neural precursor cells.Before the 1990s, it was believed that neurogenesis terminated early in life. More recent studies demonstrate that the brain contains stem cells that are capable of regenerating neurons and glial cells throughout the human lifecycle. These stem cells have only been found in certain brain regions, including the striatum and lateral ventricle.

Hematopoietic progenitor cells (HPCs) are an intermediate cell type in blood cell development. HPCs are immature cells that develop from hematopoietic stem cells, cells that can both self-renew and differentiate into hematopoietic progenitor cells. HPCs eventually differentiate into one of more than ten different types of mature blood cells.Hematopoietic progenitor cells are categorized based upon their cell potency, or their differentiation potential. As blood cells develop, their potency decreases.

First, hematopoietic stem cells differentiate into multipotent progenitor cells. Multipotent progenitor cells are those with the potential to differentiate into a subset of cell types. These cells then differentiate into either the common myeloid progenitor (CMP) or common lymphoid progenitor (CLP). Both CMPs and CLPs are types of oligopotent progenitor cells (progenitor cells that differentiate into only a few cell types).

CMPs and CLPs continue to differentiate along cell lines into lineage-restricted progenitor cells that become final, mature blood cells.Myeloid progenitor cells are precursors to the following types of blood cells:

Lymphoid progenitor cells (also known as lymphoblasts) are precursors to other mature blood cell types, including:

The primary role of progenitor cells is to replace dead or damaged cells. In this way, progenitor cells are necessary for repair after injury and as part of ongoing tissue maintenance. Progenitor cells also replenish blood cells and play a role in embryonic development.

Neural progenitor cells (NPCs) are being explored alongside neural stem cells for their potential to treat diseases of or injury to the central nervous system. A deeper understanding of how these cells function on a cellular and molecular basis is needed to progress from early experimental research to therapeutic use.NPCs are currently utilized in research conducted on CNS disorders, development, cell regeneration and degeneration, neuronal excitability, and therapy screening. When compared to induced pluripotent stem cells, which are cells reprogrammed into a pluripotent state, NPCs can cut down on time in some experiments.Hematopoietic progenitor cells and stem cells are being researched for their capacity to treat blood cell disorders. They are also currently used to help treat patients with a variety of malignant and non-malignant diseases via bone marrow transplants that deliver bone marrow and peripheral blood progenitor cells to patients. These procedures can assist patients in recovering from the damage caused by chemotherapy.Additionally, researchers are examining the potential of using progenitor cells to create a variety of tissues, such as blood vessels, heart valves, and electrically conductive tissue for the cardiovascular system.

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What are Progenitor Cells? Exploring Neural, Myeloid and Hematopoietic Progenitor Cells - Technology Networks

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Daratumumab: A Promising Option

Due to promising new data from several big clinical trials, its now believed that daratumumab can benefit patients with multiple myeloma regardless ofwhether theyre eligible to receive a stem cell transplant.

Daratumumab (also known by its brand name, Darzalex), is a type of drug called a targeted monoclonal antibody. It works by binding to a specific protein called CD38, which is found on the surface of multiple myeloma cells. Once the daratumumab attaches to these proteins on the surface of the cells, the bodys immune system identifies the need to attack and kill the multiple myeloma cells.

As Dr. Nina Shah,a hematologist at the University of California San Francisco, explains, patients receiving a stem cell transplant can benefit from the addition of daratumumab to a combination of the drugs Velcade, Revlimid and dexamethasone (a combination that doctors often abbreviate as Dara VRD).

If, on the other hand, a transplant isnt the right course of treatment for you, you may still be able to benefit from daratumumab when its used in whats called the upfront or first-line treatment setting (meaning as the first part of your treatment, before you receive other drugs) when combined withRevlimid and dexamethasone (a combination that doctors often abbreviate as DRD).

If youre not interested in a transplant, or maybe thats not in the works for you at the moment, you may consider daratumumab, Revlimid and dexamethasone, or DRD,' Dr. Shah explains.

One thing thats really gotten a lot of attention is not just three drugs, but four drugs, Dr. Shah says. She explains that a recent clinical trial called GRIFFIN showed that before a stem cell transplant, treatment with daratumumab in combination with Velcade, Revlimid and dexamethasone was more beneficial than treatment with Velcade, Revlimid and dexamethasone alone.

And when patients who received this combination before their transplant, then went on to receive additional daratumumab after their transplant, the benefit was even greater.

Preliminarily, at least, the data seems to indicate that the patients who got four drugs, then went on to a transplant and then got more daratumumab actually did better than the three drugs, Dr. Shah says.

Learn more about SurvivorNet's rigorous medical review process.

Dr. Nina Shah is a hematologist who specializes in the treatment of multiple myeloma, a type of cancer affecting the blood marrow. She treats patients at the Hematology and Blood and Marrow Transplant Clinic. Read More

Daratumumab (also known by its brand name, Darzalex), is a type of drug called a targeted monoclonal antibody. It works by binding to a specific protein called CD38, which is found on the surface of multiple myeloma cells. Once the daratumumab attaches to these proteins on the surface of the cells, the bodys immune system identifies the need to attack and kill the multiple myeloma cells.

If, on the other hand, a transplant isnt the right course of treatment for you, you may still be able to benefit from daratumumab when its used in whats called the upfront or first-line treatment setting (meaning as the first part of your treatment, before you receive other drugs) when combined withRevlimid and dexamethasone (a combination that doctors often abbreviate as DRD).

If youre not interested in a transplant, or maybe thats not in the works for you at the moment, you may consider daratumumab, Revlimid and dexamethasone, or DRD,' Dr. Shah explains.

One thing thats really gotten a lot of attention is not just three drugs, but four drugs, Dr. Shah says. She explains that a recent clinical trial called GRIFFIN showed that before a stem cell transplant, treatment with daratumumab in combination with Velcade, Revlimid and dexamethasone was more beneficial than treatment with Velcade, Revlimid and dexamethasone alone.

And when patients who received this combination before their transplant, then went on to receive additional daratumumab after their transplant, the benefit was even greater.

Preliminarily, at least, the data seems to indicate that the patients who got four drugs, then went on to a transplant and then got more daratumumab actually did better than the three drugs, Dr. Shah says.

Learn more about SurvivorNet's rigorous medical review process.

Dr. Nina Shah is a hematologist who specializes in the treatment of multiple myeloma, a type of cancer affecting the blood marrow. She treats patients at the Hematology and Blood and Marrow Transplant Clinic. Read More

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The Benefit of Adding Daratumumab to Multiple Myeloma Drug Combinations - SurvivorNet

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In our regular feature #TheBrand, Bazaars beauty team look into an exciting and efficacious brand taking the beauty industry by storm. This time, its a doctor-backed skincare line combining luxury with lasting results.

In the past, weve happily soaked up skincare advice from celebrities, supermodels and self-appointed influencers. (In fact, weve even bought into brands created by them.) But now, those of us looking to settle down with a serious skincare regime one that promises a healthy, resilient complexion for good are rightfully turning to doctors for direction.

As we become increasingly invested in our skincare, favouring proven formulations over zeitgeisty trends, the door has been opened for a host of dermatologists, surgeons and doctors to launch their own brands. Armed with the best qualifications in the business, these experts combine ingredients knowledge with confidence, ensuring maximum potency with minimal contraindications.

The latest brand in this formidable category is MZ Skin, founded by Dr. Maryam Zamani. Not only is she a leading oculoplastic surgeon (aka eye doctor), but she's also one of London's most in-demand aesthetic doctors, working out of the Cadogan Clinic in Chelsea.

With a background in medical science, Zamani is perfectly positioned to create clinically proven products that speak to womens needs, providing a direct path to the balanced and healthy skin were all hoping to obtain. Truly understanding the actives, how they interact with the skin and what they can achieve is imperative in formulating powerful results, she says.

While most dermatologist and doctor-led brands tend to sit on the cold, clinical side of the skincare fence, MZ Skin is a visibly luxurious affair. Most of the doctor ranges now are made by men for women, which often means we lose an important aspect of skincare and wellness, explains Zamani, who treasures the sense of ritual in her own routine, seeing it as a powerful self-care tool. Taking a few moments to do something that is good for you and feels good to do has compounded positive impact.

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Proven actives are, unsurprisingly, the focal point of MZ Skin here, you can find some of the most effective brightening, rejuvenating and repairing formulas around.

Expect familiar ingredients in optimum levels of potency, and always stabilised for longevity. Vitamin C, peptides, acids, ceramides, stem cells and most recently retinol form the basis. Everything is free from mineral oil, (a harmless yet useless filler ingredient), and controversial paraben preservatives.

Naturally, Zamanis Soothe & Smooth eye cream is a stand-out. Hyaluronic acid provides moisture while ceramides strengthen the skin barrier, but its the unusual addition of albazia bark extract that proves her skincare nous. Also known as Persian silk tree extract, it is said to encourage the skin to produce collagen and elastin, leading to less surface lines.

If youre looking for a quick fix, the Radiance & Renewal mask is worth a try, but the savviest shoppers will head instead for the Cleanse & Clarify cleanser. Ticking off two steps in one, it can be used nightly as a deep cleanser, or left to linger as a pre-event mask. The hefty dose of alpha-hydroxy acids sloughs away dead skin cells, leaving skin looking brighter immediately after use.

Several brands are investing in at-home LED technology now, but MZ Skins Light Therapy Golden Facial Device is one of the most advanced available outside of a professional setting, thanks to the impressive five shades of LED it emits.

Light emitting diodes send out specific wavelengths that are then absorbed by the skin," explains Zamani. Red and yellow light helps boost collagen production, while blue light kills bacteria that can lead to acne. Green LED can be absorbed by melanin in the skin to help improve the appearance of pigmentation.

But it's the inclusion of a fifth light setting that make's Zamani's device a true stand-out. White, or near-infrared light, penetrates remarkably deep into the dermis to promote wound healing and skin repair: a benefit scarcely found in at-home devices.

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Everything you need to know about MZ Skin products - harpersbazaar.com

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With so many groundbreaking medical advances being revealed to the world every single day, you would imagine there would be some advancement on the plethora of many female-prevalent diseases (think female cancers, Alzheimer's, depression, heart conditions etc.) that women are fighting every single day.

For Anna Villarreal and her team, there frankly wasn't enough being done. In turn, she developed a method that diagnoses these diseases earlier than traditional methods, using a pretty untraditional method in itself: through your menstrual blood.

Getting from point A to point B wasn't so easy though. Villarreal was battling a disease herself and through that experience. I wondered if there was a way to test menstrual blood for female specific diseases," she says. "Perhaps my situation could have been prevented or at least better managed. This led me to begin researching menstrual blood as a diagnostic source. For reasons the scientific and medical community do not fully understand, certain diseases impact women differently than men. The research shows that clinical trials have a disproportionate focus on male research subjects despite clear evidence that many diseases impact more women than men."

There's also no denying that gap in women's healthcare in clinical research involving female subjects - which is exactly what inspired Villarreal to launch her company, LifeStory Health. She says that, with my personal experience everything was brought full circle."

There is a challenge and a need in the medical community for more sex-specific research. I believe the omission of females as research subjects is putting women's health at risk and we need to fuel a conversation that will improve women's healthcare.,"

-Anna Villarreal

Her brand new biotech company is committed to changing the women's healthcare market through technology, innovation and vocalization and through extensive research and testing. She is working to develop the first ever, non-invasive, menstrual blood diagnostic and has partnered with a top Boston-area University on research and has won awards from The International Society for Pharmaceutical Engineering and Northeastern University's RISE.

How does it work exactly? Proteins are discovered in menstrual blood that can quickly and easily detect, manage and track diseases in women, resulting in diseases that can be earlier detected, treated and even prevented in the first place. The menstrual blood is easy to collect and since it's a relatively unexplored diagnostic it's honestly a really revolutionary concept, too.

So far, the reactions of this innovative research has been nothing but excitement. The reactions have been incredibly positive." she shares with SWAAY. Currently, menstrual blood is discarded as bio waste, but it could carry the potential for new breakthroughs in diagnosis. When I educate women on the lack of female subjects used in research and clinical trials, they are surprised and very excited at the prospect that LifeStory Health may provide a solution and the key to early detection."

To give a doctor's input, and a little bit more of an explanation as to why this really works, Dr. Pat Salber, MD, and Founder of The Doctor Weighs In comments: researchers have been studying stem cells derived from menstrual blood for more than a decade. Stem cells are cells that have the capability of differentiating into different types of tissues. There are two major types of stem cells, embryonic and adult. Adult stem cells have a more limited differentiation potential, but avoid the ethical issues that have surrounded research with embryonic stem cells. Stem cells from menstrual blood are adult stem cells."

These stem cells are so important when it comes to new findings. Stem cells serve as the backbone of research in the field of regenerative medicine the focus which is to grow tissues, such as skin, to repair burn and other types of serious skin wounds.

A certain type of stem cell, known as mesenchymal stem cells (MenSCs) derived from menstrual blood has been found to both grow well in the lab and have the capability to differentiate in various cell types, including skin. In addition to being used to grow tissues, their properties can be studied that will elucidate many different aspects of cell function," Dr. Salber explains.

To show the outpour of support for her efforts and this major girl power research, Villarreal remarks, women are volunteering their samples happily report the arrival of their periods by giving samples to our lab announcing de-identified sample number XXX arrived today!" It's a far cry from the stereotype of when it's that time of the month."

How are these collections being done? Although it might sound odd to collect menstrual blood, plastic cups have been developed to use in the collection process. This is similar to menstrual products, called menstrual cups, that have been on the market for many years," Dr. Salber says.

Equally shocking and innovative, this might be something that becomes more common practice in the future. And according to Dr. Salber, women may be able to not only use the menstrual blood for early detection, but be able to store the stem cells from it to help treat future diseases. Companies are working to commercialize the use of menstrual blood stem cells. One company, for example, is offering a patented service to store menstrual blood stem cells for use in tissue generation if the need arises."

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Divorce as Seen Through the Eyes of a Child - SWAAY

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LOS ANGELES Extending everyones life in a healthy fashion is one of many goals held by Peter Diamandis, a space, technology, aeronautics and medicine pioneer. But the new field known as longevity is of interest to everyone.

One hundred will be the new 60, he told his Abundance360 conference recently. The average human health span will increase by 10+ years this decade.

He, like others in Silicon Valley, believe that aging is a disease and the result of planned obsolescence, or the wearing down of, or damage to, certain critical mechanisms, sensors and functions within our bodies. Longevity research is about identifying the core problems to mitigate or reverse them.

The average human health span will increase by 10+ years this decade

Peter Diamandis

The exponential technologies of artificial intelligence, machine learning and computational heft have been harnessed, and have resulted in breakthroughs and clinical trials that are just a handful of years away from deployment on human patients. The main areas of research include: Stem cell supply restoration, regenerative medicine to regrow damaged cartilage, ligaments, tendons, bone, spinal cords and neural nerves; vaccine research against chronic diseases such as Alzheimers; and United Therapeutics that is developing technology to tackle the organ shortage for humans by genetically engineering organs grown in pigs.

New tools are accelerating the development of new, tailor-made medicines at a fraction of todays costs. Alex Zhavoronkov of Insilico Medicine told the conference that drugs take 10 years and cost $3 billion to research and 90 per cent fail. But his company can test in 46 days using human tissue, then model, design and produce in weeks with the help of advanced computing.

In regenerative medicine, advances appear to be arriving relatively soon. For instance, Diamandis asked the audience if anyone was awaiting a knee replacement operation and suggested that they might be better off postponing these until 2021 when regenerative medicine innovator, Samumed LLC in San Diego, is expected to complete phase three clinical trials of cartilage regeneration.

Samumeds founder, Osman Kibar, said his company has successfully injected a protein that activates nearby stem cells into producing new cartilage in a knee or a new disc in a spine. Preliminary success has also occurred to regenerate muscle and neural cells, retinal cells, skin and hair. Not surprisingly, the private company just raised US$15.5 billion to continue research and product development.

Another hot area of early stage research is called epigenetic reprogramming or identifying how to reverse deficiencies in proteins, stem cells, chromosomes, genes that repair DNA and damaged cells. A leader in this field is David Sinclair, professor of genetics at the Harvard Medical School, whose new book Lifespan: Why We Age and Why We Dont Have To explains the science and offers advice.

Aging is a disease, and that disease is treatable, he said. As research progresses toward actual corrections or cures, there are also lifestyle habits that can slow down the aging process, or avert damage. For instance, he said humans should replicate some behaviour that their bodies were designed for. Obviously, exercising and sleep are necessary but so is eating less often. You should feel hungry regularly, he said.

Another condition that is useful to emulate is hormesis, a scientific term for what Neitzsche posited which was that that which does not kill us makes us stronger. Sinclair recommends stressing our bodies with temperature changes such as going from a hot sauna to rolling in the snow. This invigorates the bodys processes and cells.

Theres also xenohormesis or gaining benefits from eating plants that have been environmentally stressed, therefore contain more beneficial nutrients. For instance, drought-stressed or wild strawberries have better flavour but they also are enhanced with additional antioxidant capacity and phenol content.

The age of 100 is easily in sight now, said Diamandis. And kids born today can expect to live to 105.

Financial Post

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Diane Francis: Treating aging like a disease is the next big thing for science - Financial Post

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When Marie Antoinette was captured during the French Revolution, her hair reportedly turned white overnight. In more recent history, former U.S. Senator John McCain experienced severe injuries as a prisoner during the Vietnam Warand lost color in his hair.

For a long time, anecdotes have connected stressful experiences with hair-graying.

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Now, for the first time, Harvard University scientists have discovered exactly how the process plays out: stress activates nerves that are part of the fight-or-flight response, which in turn cause permanent damage to pigment-regenerating stem cells in hair follicles.

The work, published inNature, details the molecular mechanisms behind the longstanding biological puzzle.

Everyone has an anecdote to share about how stress affects their body, particularly in their skin and hairthe only tissues we can see from the outside, said senior authorYa-Chieh Hsu, the Alvin and Esta Star Associate Professor of Stem Cell and Regenerative Biology at Harvard. We wanted to understand if this connection is true, and if so, how stress leads to changes in diverse tissues. Hair pigmentation is such an accessible and tractable system to start with, and besides, we were genuinely curious to see if stress indeed leads to hair-graying.

Fingering the culprit

Because stress affects the whole body, researchers first had to narrow down which body system was responsible for connecting stress to hair color. The team first hypothesized that stress causes an immune attack on pigment-producing cells. However, when mice lacking immune cells still showed hair-graying, researchers turned to the hormone cortisol. Once more, it was a dead end.

Stress always elevates levels of the hormone cortisol in the body, so we thought that cortisol might play a role, Hsu said. But surprisingly, when we removed the adrenal gland from the mice so that they couldnt produce cortisol-like hormones, their hair still turned gray under stress.

After systematically eliminating different possibilities, researchers homed in on the sympathetic nervous system, which is responsible for the bodys fight-or-flight response.

Sympathetic nerves branch out into each hair follicle on the skin. The researchers found that stress causes these nerves to release the chemical norepinephrine, which gets taken up by nearby pigment-regenerating stem cells.

Permanent damage

In the hair follicle, certain stem cells act as a reservoir of pigment-producing cells. When hair regenerates, some of the stem cells convert into pigment-producing cells that color the hair.

Researchers found that the norepinephrine from sympathetic nerves causes the stem cells to activate excessively. The stem cells all convert into pigment-producing cells, prematurely depleting the reservoir.

When we started to study this, I expected that stress was bad for the body, but the detrimental impact of stress that we discovered was beyond what I imagined, Hsu said. After just a few days, all of the pigment-regenerating stem cells were lost. Once theyre gone, you cant regenerate pigments anymore. The damage is permanent.

The finding underscores the negative side effects of an otherwise protective evolutionary response, the researchers said.

Acute stress, particularly the fight-or-flight response, has been traditionally viewed to be beneficial for an animals survival. But in this case, acute stress causes permanent depletion of stem cells, said postdoctoral fellow Bing Zhang, the lead author of the study.

Answering a fundamental question

To connect stress with hair-graying, the researchers started with a whole-body response and progressively zoomed into individual organ systems, cell-to-cell interaction and, eventually, all the way down to molecular dynamics. The process required a variety of research tools along the way, including methods to manipulate organs, nerves and cell receptors.

To go from the highest level to the smallest detail, we collaborated with many scientists across a wide range of disciplines, using a combination of different approaches to solve a very fundamental biological question, Zhang said.

One of the study collaborators wasIsaac Chiu, assistant professor of immunology in the Blavatnik Institute at Harvard Medical School, who studies the interplay between the nervous and immune systems.

We know that peripheral neurons powerfully regulate organ function, blood vessels and immunity, but less is known about how they regulate stem cells, Chiu said.With this study, we now know that neurons can control stem cells and their function and can explain how they interact at the cellular and molecular levels to link stress with hair-graying.

The findings can help illuminate the broader effects of stress on various organs and tissues. This understanding will pave the way for new studies that seek to modify or block the damaging effects of stress.

By understanding precisely how stress affects stem cells that regenerate pigment, weve laid the groundwork for understanding how stress affects other tissues and organs in the body, Hsu said. Understanding how our tissues change under stress is the first critical step towards eventual treatment that can halt or revert the detrimental impact of stress. We still have a lot to learn in this area.

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Monday, January 27, 2020

Cancer patients could be treated with a one-size-fits-all therapy, following the discovery of an immune cell which kills all forms of the disease.

Researchers at Cardiff University have found a new type of killer T-cell, capable of recognising and destroying most human cancers while preserving healthy cells. The scientists discovered a method of killing prostate, breast, lung and other cancers in lab tests and say there is enormous potential for immunotherapies not previously thought to be possible.

Cardiff Universitys cancer findings came from scientists looking for unconventional ways in which the immune system naturally attacks tumours. They found, inside human blood, a T-cell that can scan the body for a threat, such as cancerous cells, and eliminate the danger while leaving healthy cells alone. The team described the work as at an early stage, but exciting.

T-cell cancer therapies are where immune cells are removed, modified and returned to the patients blood to seek and destroy cancer cells. The most widely-used, known as CAR-T, is personalised to the patient but combats only a handful of cancers and has not been successful in eliminating solid tumours - which account for the vast majority of cancers.

The Cardiff teams discovery involves a new type of T-cell receptor (TCR), which recognises a molecule present on the surface of a wide range of cancer cells as well as in many of the bodys normal cells and is, remarkably, able to distinguish between the two. In tests, T-cells equipped with the new TCR killed lung, skin, blood, colon, breast, bone, prostate, ovarian, kidney and cervical cancer cells.

Professor Andrew Sewell, the lead author on the study and an expert in T-cells from Cardiff Universitys School of Medicine, said it was highly unusual to find a TCR with such broad cancer specificity, raising the prospect of universal cancer therapy.

Prof Sewell said: We hope this new TCR may provide us with a different route to target and destroy a wide range of cancers in all individuals. Current TCR-based therapies can only be used in a minority of patients with a minority of cancers.

Cancer-targeting via MR1-restricted T-cells is an exciting new frontier - it raises the prospect of a one-size-fits-all cancer treatment; a single type of T-cell that could be capable of destroying many different types of cancers across the population. Previously nobody believed this could be possible.

Further experiments and safety testing are now underway, with the hope of trialling this new approach in patients towards the end of 2020. Prof Sewell added: There are plenty of hurdles to overcome; however, if this testing is successful, then I would hope this new treatment could be in use in patients in a few years time.

Cancer is the leading cause of all avoidable deaths in the UK. Breast cancer is the most common, followed jointly by prostate and lung cancer and then by bowel cancer. Obesity is now a bigger cause than smoking of some cancers, namely bowel, kidney, liver and ovarian cancer.

According to financial information business Defaqto*, 38 out of 51 health insurance products include cancer cover, with benefits ranging from breakthrough treatment not otherwise available on the NHS to hormone therapy, reconstructive surgery and stem cell therapy. To find the right cancer cover for your family, use our online comparison tool or speak with our team on 0800 862 0373.

Photo:Cardiff Universitys Professor Andrew Sewell, left, with Research Fellow Garry Dolton.

Credit: Cardiff University

* Data sourced on January 2, 2020.

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CHICAGO - Brian Wallach wasn't supposed to live to see his younger daughter's first birthday.

Diagnosed with amyotrophic lateral sclerosis (ALS), a terminal disease with no cure, doctors told him in 2017 that he might have six months to live.

Today, he's focused on being there for his daughter's future firsts: kindergarten drop-off, middle school dance, wedding day.

More than two years after his diagnosis, he has been lucky, he said, to experience relatively limited progression of his disease. After some balance issues, the Kenilworth resident now uses a cane - or, as he is careful to specify, a "cool walking stick" - to get around.

When Wallach was diagnosed, neither he nor his wife, Sandra Abrevaya, knew much about ALS, a neurodegenerative disease that affects nerve cells in the brain and the spinal cord, eventually paralyzing even the body's ability to breathe.

In response to Wallach's diagnosis, the couple, both 39, launched I AM ALS in 2019. Former staffers in the Obama White House, they marshaled lessons learned while campaigning - gathering information, forming consensus, considering the impossible possible - to build a force to mobilize hope and change for those facing a disease they say can and should be cured.

Rays of hope are beginning to emerge through an innovative trial that received FDA approval last week to test several drugs at the same time, a bipartisan congressional caucus, doubled federal funding, and support from groups like the Chan Zuckerberg Initiative, which gave the couple's organization a $453,000 grant in September.

"Last year we made hope a word that was OK to use," Wallach said. "This year we have to make hope real."

Audaciousness is the only option, the couple says, in their race against the clock.

Wallach logged 120,000 miles in the air last year, including traveling to Washington, D.C., in April, where he testified before Congress and asked legislators to amp up funding.

"Last year, every time someone said, 'Do you want to speak to us,' I said, 'yes.' Every time someone said, 'There's a meeting,' I said, 'I'm going.'" he said. "Every time there was anything, I said, 'Great, I'm on the plane.'"

Until October, when Wallach fell while exiting a Lyft in Boston after swinging a heavy backpack onto his back. Thirteen staples in his head later, and after terrifying Abrevaya with a phone call, the two agreed he wouldn't travel alone anymore. He's maintaining momentum for the cause with more hours in his home office and fewer in airports.

In December, I AM ALS debuted billboards around Times Square as part of its #CuresForAll campaign aimed at informing the public about the impact a cure or better treatment for a neurodegenerative disease can have on other diseases such as multiple sclerosis, Alzheimer's and Parkinson's. ALS patients and their families from states including Michigan, Maine and Colorado were in New York for the launch.

The billboards noted the number of people lost to ALS each day - 16 - with photographs of those who died in 2019. Days earlier, Pete Frates, a founder of the viral fundraiser the Ice Bucket Challenge, which raised $115 million, had died. He was 34.

The campaign was also shared on social media. The posts expressed the suffering and loss nationwide: a mother wrote about her son who was diagnosed at 20 and died at 28; a son posted in honor of his dad; Colorado Rep. Jason Crow posted a message honoring his cousin.

It's time, the couple said, to switch ALS conversations from a diagnosis rooted in darkness to the faces of people bravely moving forward. They want to speed development of potential cures and give patients more access to experimental treatments.

That's not an unreasonable goal, said Sabrina Paganoni, a faculty member at The Sean M. Healey & AMG Center for ALS at Mass General in Boston, which plans to test at least five different medications for ALS at the same time, a first for the disease and something she said could be a huge turning point.

On Wednesday, the Healey Center announced it received FDA approval to move forward with testing the first three drugs: Zilucoplan, Verdiperstat and CNM-Au8. Similar to how cancer drugs are already tested, this gives patients access to more treatments and allows researchers to quickly collect data and accelerate the pace toward a cure.

"This is a very exciting time in the history of ALS," Paganoni said. "I think this is going to be the decade when ALS is changed from a rapidly fatal disease to a more chronic disease that we can manage."

For years, Steve Perrin, the chief executive officer at the ALS Therapy Development Institute, has monitored clinical trials for ALS. So far, he said, the two drugs approved by the FDA, Radicava and Rilutek, are "a very marginal slowing down of disease."

This year, he said the quality of drugs going into trials seems improved. He is excited about several trials, including one studying stem cells and another testing a drug to potentially slow progression in some patients.

"As a patient you want to see something measurable, and I don't mean measurable in days," he said. "If I'm a patient, I want to see something, and I want hope for myself and my family. I want something that is going to slow the disease down so I can watch my kids growing up, I can watch them graduate from college, I can watch them marry."

But that takes resources.

"We are in a time when we can reasonably say that there's going to be new treatments available," Paganoni said. "But we need more funding and support, so all of this can happen, and happen soon."

Nearly every moment feels like a push-pull for Wallach and Abrevaya.

Do they spend more precious minutes with their two daughters, ages 4 and 2, or do they spend time away, among strangers - on a plane, in a researcher's office, walking the halls of Congress - with the hope that those minutes will, someday, result in time banked to create more family memories.

"The hardest balance, if I'm honest, is, I love every minute I have with them," Wallach said about his daughters, "but I also feel this pressing sense of, I need to be working towards a goal of actually finding a cure."

"We're doing that so we have a shot at a real future together," Abrevaya said about their time spent traveling and advocating.

At home, when the family heads for the door, the toddlers reach for their father's shoes, and they get his walking stick.

"While that both fills your heart with joy and appreciation, it's also painful that your toddlers are being put in this position," Abrevaya said.

The parents guard normalcy. They take their daughters to swim at the neighborhood pool and on vacation with friends. Wallach wishes he could lift them above his head to touch the ceiling, like their uncle can. But he can lie on the floor and play with them; he can listen to them belt out songs on their purple karaoke machine.

They find ways to lighten a heavy subject. On New Year's Eve, the two danced in a video on the foundation's Instagram, singing into hairbrushes, and Wallach promised to get an "ALS: You Gone" tattoo if 20,000 people donated $10 to a Healey Center research fundraiser. It raised $40,000 in 24 hours, Wallach said. No matter the outcome, he plans to get the tattoo.

The couple, who both work full-time jobs - Abrevaya is the president of nonprofit Thrive, Wallach works at law firm Skadden, Arps, Slate, Meagher & Flom - want more research, to create a patient navigation system, and to gather signatures for a letter asking new FDA commissioner Stephen Hahn to speed ALS patients' access to possible treatments.

And they keep looking for light. But it takes work.

Changing life with ALS for Wallach, and for other patients and their families, requires bold action from people with the power to make change: politicians, researchers, philanthropists.

As they meet others with ALS, they welcome new friends and face the pain of losing some.

"It does make you uniquely urgent in what you do," Wallach said. "You push because you have to. You push because you know that the time that we have is precious, and that you want to see 20 years from now. And know that you can make that happen."

Wallach often shares moments about his ALS journey on Twitter with his 40,000 followers. Recently, he shared something he wasn't sure he should. It was a time he was unable to find light.

On a recent night, he woke up to pain he's had for the past few months, radiating from his right hip to his right calf.

He clutched a stuffed llama his daughter gave him. And he began to cry.

"I cried because of the pain. I cried because I couldn't be the father to my girls I dreamed of being," he wrote. "I cried because I couldn't be the husband to my wife I dream of being. Because I saw the future zooming ahead, and for a brief moment I wondered if I would be a part of it."

His wife heard him crying that night. She asked what was wrong. And he said maybe they would be better off if he left, living instead in an assisted living facility. Their daughters, he told her, could have a dad who could do everything he dreamed of doing.

She looked at him in the dark. "You are my light," she said. "You are their light. The only way you are leaving us is if you die in my arms, and we aren't going to let that happen for a long, long, long time."

Distributed by Tribune Content Agency, LLC.

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In a race against terminal illness, former Obama staffer with ALS and his wife find new hope a year later - PostBulletin.com

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Submerged in liquid nitrogen vapor at a temperature of minus 175 degrees Celsius, hundreds of thousands of stem cells from all over Europe bide their time in large steel barrels on the outskirts of Warsaw.

Present in blood drawn from the umbilical cord of a newborn baby, stem cells can help cure serious blood-related illnesses like leukemias and lymphomas, as well as genetic conditions and immune system deficits.

Polish umbilical cord blood bank PBKM/FamiCord became the industrys leader in Europe after Swiss firm Cryo-Save went bankrupt early last year.

It is also the fifth largest in the world, according to its management, after two companies in the United States, a Chinese firm and one based in Singapore.

Since the first cord blood transplant was performed in France in 1988, the sector has significantly progressed, fuelling hopes.

Health insurance

Mum-of-two Teresa Przeborowska has firsthand experience.

At five years old, her son Michal was diagnosed with lymphoblastic leukemia and needed a bone marrow transplant, the entrepreneur from northern Poland said.

The most compatible donor was his younger sister, Magdalena.

When she was born, her parents had a bag of her cord blood stored at PBKM.

More than three years later, doctors injected his sisters stem cells into Michals bloodstream.

It was not quite enough for Michals needs but nicely supplemented harvested bone marrow.

As a result, Michal, who is nine, is now flourishing, both intellectually and physically, his mum told AFP.

A cord blood transplant has become an alternative to a bone marrow transplant when there is no donor available, with a lower risk of complications.

Stem cells taken from umbilical cord blood are like those taken from bone marrow, capable of producing all blood cells: red cells, platelets and immune system cells.

When used, stem cells are first concentrated, then injected into the patient. Once transfused, they produce new cells of every kind.

At the PBKM laboratory, each container holds up to 10,000 blood bags Safe and secure, they wait to be used in the future, its head, Krzysztof Machaj, said.

The bank holds around 440,000 samples, not including those from Cryo-Save, he said.

If the need arises, the blood will be ready to use without the whole process of looking for a compatible donor and running blood tests, the biologist told AFP.

For families who have paid an initial nearly 600 euros (around P34,000) and then an annual 120 euros (around P7,000) to have the blood taken from their newborns umbilical cords preserved for around 20 years, it is a kind of health insurance promising faster and more effective treatment if illness strikes.

But researchers also warn against unrealistic expectations.

Beauty products

Hematologist Wieslaw Jedrzejczak, a bone marrow pioneer in Poland, describes promoters of the treatment as sellers of hope, who make promises that are either impossible to realize in the near future or downright impossible to realize at all for biological reasons.

He compares them to makers of beauty products who swear their cream will rejuvenate the client by 20 years.

Various research is being done on the possibility of using the stem cells to treat other diseases, notably nervous disorders. But the EuroStemCell scientist network warns that the research is not yet conclusive.

There is a list of almost 80 diseases for which stem cells could prove beneficial, U.S. hematologist Roger Mrowiec, who heads the clinical laboratory of the cord blood program Vitalant in New Jersey, told AFP.

But given the present state of medicine, they are effective only for around a dozen of them, like leukemia or cerebral palsy, he said.

Its not true, as its written sometimes, that we can already use them to fight Parkinsons disease or Alzheimers disease or diabetes.

EuroStemCell also cautions against private blood banks that advertise services to parents suggesting they should pay to freeze their childs cord blood in case its needed later in life.

Studies show it is highly unlikely that the cord blood will ever be used for their child, the network said.

It also pointed out that there could be a risk of the childs cells not being useable anyway without reintroducing the same illness.

Some countries, such as Belgium and France, are cautious and ban the storage of cord blood for private purposes. Most E.U. countries however permit it while imposing strict controls.

Rapid growth

In the early 2000s, Swiss company Cryo-Save enjoyed rapid growth.

Greeks, Hungarians, Italians, Spaniards and Swiss stored blood from their newborns with the company for 20 years on payment of 2,500 euros (around P140,000) upfront.

When the firm was forced to close in early 2019, clients were left wondering where their stem cells would end up.

Under a kind of back-up agreement, the samples of some 250,000 European families were transferred for storage at PBKM.

The Polish firm, founded in 2002 with 2 million zlotys (around P26 million), has also grown quickly.

Present under the FamiCord brand in several countries, PBKM has some 35% of the European market, excluding Cryo-Save assets.

Over the last 15 months, outside investors have contributed 63 million euros to the firm, PBKMs chief executive Jakub Baran told AFP.

But the company has not escaped controversy: the Polityka weekly recently published a critical investigative report on several private clinics that offer what was described as expensive treatment involving stem cells held by PBKM.IB/JB

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Europe's guardian of stem cells and hopes, real and unrealistic - INQUIRER.net

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UCLA Broad Stem Cell Research Center/Nature Medicine

At left, image shows white blood cells (red) from one of the X-CGD clinical trial participants before gene therapy. At right, after gene therapy, white blood cells from the same patient show the presence of the chemicals (blue) needed to attack and destroy bacteria and fungus.

UCLA researchers are part of an international team that reported the use of a stem cell gene therapy to treat nine people with the rare, inherited blood disease known as X-linked chronic granulomatous disease, or X-CGD. Six of those patients are now in remission and have stopped other treatments. Before now, people with X-CGD which causes recurrent infections, prolonged hospitalizations for treatment, and a shortened lifespan had to rely on bone marrow donations for a chance at remission.

With this gene therapy, you can use a patients own stem cells instead of donor cells for a transplant, said Dr. Donald Kohn, a member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA and a senior author of the new paper, published today in the journal Nature Medicine. This means the cells are perfectly matched to the patient and it should be a much safer transplant, without the risks of rejection.

People with chronic granulomatous disease, or CGD, have a genetic mutation in one of five genes that help white blood cells attack and destroy bacteria and fungus using a burst of chemicals. Without this defensive chemical burst, patients with the disease are much more susceptible to infections than most people. The infections can be severe to life-threatening, including infections of the skin or bone and abscesses in organs such as lungs, liver or brain. The most common form of CGD is a subtype called X-CGD, which affects only males and is caused by a mutation in a gene found on the X-chromosome.

Other than treating infections as they occur and taking rotating courses of preventive antibiotics, the only treatment option for people with CGD is to receive a bone marrow transplant from a healthy matched donor. Bone marrow contains stem cells called hematopoietic, or blood-forming, stem cells, which produce white blood cells. Bone marrow from a healthy donor can produce functioning white blood cells that effectively ward off infection. But it can be difficult to identify a healthy matched bone marrow donor and the recovery from the transplant can have complications such as graft versus host disease, and risks of infection and transplant rejection.

Patients can certainly get better with these bone marrow transplants, but it requires finding a matched donor and even with a match, there are risks, Kohn said. Patients must take anti-rejection drugs for six to 12 months so that their bodies dont attack the foreign bone marrow.

In the new approach, Kohn teamed up with collaborators at the United Kingdoms National Health Service, France-based Genethon, the U.S. National Institute of Allergy and Infectious Diseases at the National Institutes of Health, and Boston Childrens Hospital. The researchers removed hematopoietic stem cells from X-CGD patients and modified the cells in the laboratory to correct the genetic mutation. Then, the patients own genetically modified stem cells now healthy and able to produce white blood cells that can make the immune-boosting burst of chemicals were transplanted back into their own bodies. While the approach is new in X-CGD, Kohn previously pioneered a similar stem cell gene therapy to effectively cure a form of severe combined immune deficiency (also known as bubble baby disease) in more than 50 babies.

The viral delivery system for the X-CGD gene therapy was developed and fine-tuned by Professor Adrian Thrashers team at Great Ormond Street Hospital, or GOSH, in London, who collaborated with Kohn. The patients ranged in age from 2 to 27 years old; four were treated at GOSH and five were treated in the U.S., including one patient at UCLA Health.

Two people in the new study died within three months of receiving the treatment due to severe infections that they had already been battling before gene therapy. The seven surviving patients were followed for 12 to 36 months after receiving the stem cell gene therapy. All remained free of new CGD-related infections, and six of the seven have been able to discontinue their usual preventive antibiotics.

None of the patients had complications that you might normally see from donor cells and the results were as good as youd get from a donor transplant or better, Kohn said.

An additional four patients have been treated since the new paper was written; all are currently free of new CGD-related infections and no complications have arisen.

Orchard Therapeutics, a biotechnology company of which Kohn is a scientific co-founder, acquired the rights to the X-CGD investigational gene therapy from Genethon. Orchard will work with regulators in the U.S. and Europe to carry out a larger clinical trial to further study this innovative treatment. The aim is to apply for regulatory approval to make the treatment commercially available, Kohn said.

Kohn and his colleagues plan to develop similar treatments for the other forms of CGD caused by four other genetic mutations that affect the same immune function as X-CGD.

Beyond CGD, there are also other diseases caused by proteins missing in white blood cells that could be treated in similar ways, Kohn said.

The research was supported by grants from the California Institute for Regenerative Medicine; the National Heart, Lung and Blood Institute and the National Institute of Allergy and Infectious Diseases, both at the National Institutes of Health; the Wellcome Trust; Boston Childrens Hospital; the National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre; the Institute for Health Research Biomedical Research Centre at University College London Hospitals NHS Foundation Trust and University College London; the Great Ormond Street Hospital Childrens Charity; the AFM-Tlthon, French Muscular Dystrophy Association; and the European Commission through the Net4CGDconsortium.

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Global Cell Therapy Technologies Marketwas valued US$ 12 billion in 2018 and is expected to reach US$ 35 billion by 2026, at CAGR of 12.14 %during forecast period.

The objective of the report is to present comprehensive assessment projections with a suitable set of assumptions and methodology. The report helps in understanding Global Cell Therapy Technologies Market dynamics, structure by identifying and analyzing the market segments and projecting the global market size. Further, the report also focuses on the competitive analysis of key players by product, price, financial position, growth strategies, and regional presence. To understand the market dynamics and by region, the report has covered the PEST analysis by region and key economies across the globe, which are supposed to have an impact on market in forecast period. PORTERs analysis, and SVOR analysis of the market as well as detailed SWOT analysis of key players has been done to analyze their strategies. The report will to address all questions of shareholders to prioritize the efforts and investment in the near future to the emerging segment in the Global Cell Therapy Technologies Market.

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Global Cell Therapy Technologies Market: OverviewCell therapy is a transplantation of live human cells to replace or repair damaged tissue and/or cells. With the help of new technologies, limitless imagination, and innovative products, many different types of cells may be used as part of a therapy or treatment for different types of diseases and conditions. Celltherapy technologies plays key role in the practice of medicine such as old fashioned bone marrow transplants is replaced by Hematopoietic stem cell transplantation, capacity of cells in drug discovery. Cell therapy overlap with different therapies like, gene therapy, tissue engineering, cancer vaccines, regenerative medicine, and drug delivery. Establishment of cell banking facilities and production, storage, and characterization of cells are increasing volumetric capabilities of the cell therapy market globally. Initiation of constructive guidelines for cell therapy manufacturing and proven effectiveness of products, these are primary growth stimulants of the market.

Global Cell Therapy Technologies Market: Drivers and RestraintsThe growth of cell therapy technologies market is highly driven by, increasing demand for clinical trials on oncology-oriented cell-based therapy, demand for advanced cell therapy instruments is increasing, owing to its affordability and sustainability, government and private organization , investing more funds in cell-based research therapy for life-style diseases such as diabetes, decrease in prices of stem cell therapies are leading to increased tendency of buyers towards cell therapy, existing companies are collaborating with research institute in order to best fit into regulatory model for cell therapies.Moreover, Healthcare practitioners uses stem cells obtained from bone marrow or blood for treatment of patients with cancer, blood disorders, and immune-related disorders and Development in cell banking facilities and resultant expansion of production, storage, and characterization of cells, these factors will drive the market of cell therapy technologies during forecast period.

On the other hand, the high cost of cell-based research and some ethical issue & legally controversial, are expected to hamper market growth of Cell Therapy Technologies during the forecast period

AJune 2016, there were around 351 companies across the U.S. that were engaged in advertising unauthorized stem cell treatments at their clinics. Such clinics boosted the revenue in this market.in August 2017, the U.S. FDA announced increased enforcement of regulations and oversight of clinics involved in practicing unapproved stem cell therapies. This might hamper the revenue generation during the forecast period; nevertheless, it will allow safe and effective use of stem cell therapies.

Global Cell Therapy Technologies Market: Segmentation AnalysisOn the basis of product, the consumables segment had largest market share in 2018 and is expected to drive the cell therapy instruments market during forecast period at XX % CAGR owing to the huge demand for consumables in cell-based experiments and cancer research and increasing number of new product launches and consumables are essential for every step of cell processing. This is further expected to drive their adoption in the market. These factors will boost the market of Cell Therapy Technologies Market in upcoming years.

On the basis of process, the cell processing had largest market share in 2018 and is expected to grow at the highest CAGR during the forecast period owing to in cell processing stage,a use of cell therapy instruments and media at highest rate, mainly in culture media processing. This is a major factor will drive the market share during forecast period.

Global Cell Therapy Technologies Market: Regional AnalysisNorth America to held largest market share of the cell therapy technologies in 2018 and expected to grow at highest CAGR during forecast period owing to increasing R&D programs in the pharmaceutical and biotechnology industries. North America followed by Europe, Asia Pacific and Rest of the world (Row).

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Scope of Global Cell Therapy Technologies Market

Global Cell Therapy Technologies Market, by Product

Consumables Equipment Systems & SoftwareGlobal Cell Therapy Technologies Market, by Cell Type

Human Cells Animal CellsGlobal Cell Therapy Technologies Market, by Process Stages

Cell Processing Cell Preservation, Distribution, and Handling Process Monitoring and Quality ControlGlobal Cell Therapy Technologies Market, by End Users

Life Science Research Companies Research InstitutesGlobal Cell Therapy Technologies Market, by Region

North America Europe Asia Pacific Middle East & Africa South America

Key players operating in the Global Cell Therapy Technologies Market

Beckman Coulter, Inc. Becton Dickinson and Company GE Healthcare Lonza Merck KGaA MiltenyiBiotec STEMCELL Technologies, Inc. Terumo BCT, Inc. Thermo Fisher Scientific, Inc. Sartorius AG

Browse Full Report with Facts and Figures of Cell Therapy Technologies Market Report at:https://www.maximizemarketresearch.com/market-report/global-cell-therapy-technologies-market/31531/

MAJOR TOC OF THE REPORT

Chapter One: Cell Therapy Technologies Market Overview

Chapter Two: Manufacturers Profiles

Chapter Three: Global Cell Therapy Technologies Market Competition, by Players

Chapter Four: Global Cell Therapy Technologies Market Size by Regions

Chapter Five: North America Cell Therapy Technologies Revenue by Countries

Chapter Six: Europe Cell Therapy Technologies Revenue by Countries

Chapter Seven: Asia-Pacific Cell Therapy Technologies Revenue by Countries

Chapter Eight: South America Cell Therapy Technologies Revenue by Countries

Chapter Nine: Middle East and Africa Revenue Cell Therapy Technologies by Countries

Chapter Ten: Global Cell Therapy Technologies Market Segment by Type

Chapter Eleven: Global Cell Therapy Technologies Market Segment by Application

Chapter Twelve: Global Cell Therapy Technologies Market Size Forecast (2019-2026)

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Global Cell Therapy Technologies Market : Industry Analysis and Forecast (2018-2026) - Expedition 99

Bone Marrow Stem Cells Comments Off on Global Cell Therapy Technologies Market : Industry Analysis and Forecast (2018-2026) – Expedition 99 | January 27th, 2020

The hearts ability to beat normally over a lifetime is predicated on the synchronized work of proteins embedded in the cells of the heart muscle.

Like a fleet of molecular motors that get turned on and off, these proteins cause the heart cells to contract, then force them to relax, beat after life-sustaining beat.

Now a study led by researchers at Harvard Medical School, Brigham and Womens Hospital and the University of Oxford shows that when too many of the hearts molecular motor units get switched on and too few remain off, the heart muscle begins to contract excessively and fails to relax normally, leading to its gradual overexertion, thickening and failure.

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Results of the work, published Jan. 27 inCirculation,reveal that this balancing act is an evolutionary mechanism conserved across species to regulate heart muscle contraction by controlling the activity of a protein called myosin, the main contractile protein of the heart muscle.

The findingsbased on experiments with human, mouse and squirrel heart cellsalso demonstrate that when this mechanism goes awry it sets off a molecular cascade that leads to cardiac muscle over-exertion and culminates in the development of hypertrophic cardiomyopathy (HCM), the mostcommon genetic diseaseof the heartand aleading causeof sudden cardiac death in young people and athletes.

Our findings offer a unifying explanation for the heart muscle pathology seen in hypertrophic cardiomyopathy that leads to heart muscle dysfunction and, eventually, causes the most common clinical manifestations of the condition, said senior authorChristine Seidman, professor of genetics in the Blavatnik Institute at Harvard Medical School, a cardiologist at Brigham and Womens Hospital and a Howard Hughes Medical InstituteInvestigator.

Importantly, the experiments showed that treatment with an experimental small-molecule drug restored the balance of myosin arrangements and normalized the contraction and relaxation of both human and mouse cardiac cells that carried the two most common gene mutations responsible for nearly half of all HCM cases worldwide.

If confirmed in further experiments, the results can inform the design of therapies that halt disease progression and prevent complications.

Correcting the underlying molecular defect and normalizing the function of heart muscle cells could transform treatment options, which are currently limited to alleviating symptoms and preventing worst-case scenarios such as life-threatening rhythm disturbances and heart failure, said study first authorChristopher Toepfer,who performed the work as a postdoctoral researcher in Seidmans lab and is now a joint fellow in the Radcliffe Department of Medicine at the University of Oxford.

Some of the current therapies used for HCM include medications to relieve symptoms, surgery to shave the enlarged heart muscle or the implantation of cardioverter defibrillators that shock the heart back into rhythm if its electrical activity ceases or goes haywire. None of these therapies address the underlying cause of the disease.

Imbalance in the motor fleet

Myosin initiates contraction by cross-linking with other proteins to propel the cell into motion. In the current study, the researchers traced the epicenter of mischief down to an imbalance in the ratio of myosin molecule arrangements inside heart cells. Cells containing HCM mutations had too many molecules ready to spring into action and too few myosin molecules idling standby, resulting in stronger contractions and poor relaxation of the cells.

An earlier study by the same team found that under normal conditions, the ratio between on and off myosin molecules in mouse heart cells is around 2-to-3. However, the new study shows that this ratio is off balance in heart cells that harbor HCM mutations, with disproportionately more molecules in active versus inactive states.

In an initial set of experiments, the investigators analyzed heart cells obtained from a breed of hibernating squirrel as a model to reflect extremes in physiologic demands during normal activity and hibernation. Cells obtained from squirrels in hibernationwhen their heart rate slows down to about six beats per minutecontained 10 percent more off myosin molecules than the heart cells of active squirrels, whose heart rate averages 340 beats per minute.

We believe this is one example of natures elegant way of conserving cardiac muscle energy in mammals during dormancy and periods of deficient resources, Toepfer said.

Next, researchers looked at cardiac muscle cells from mice harboring the two most common gene defects seen in HCM. As expected, these cells had altered ratios of on and off myosin reserves.The researchers also analyzed myosin ratios in two types of human heart cells: Stem cell-derived human heart cells engineered in the lab to carry HCM mutations and cells obtained from the excised cardiac muscle tissue of patients with HCM. Both had out-of-balance ratios in their active and inactive myosin molecules.

Further experiments showed that this imbalance perturbed the cells normal contraction and relaxation cycle. Cells harboring HCM mutations contained too many on myosin molecules and contracted more forcefully but relaxed poorly. In the process, the study showed, these cells gobbled up excessive amounts of ATP, the cellular fuel that sustains the work of each cell in our body. And because oxygen is necessary for ATP production, the mutated cells also devoured more oxygen than normal cells, the study showed. To sustain their energy demands, these cells turned to breaking down sugar molecules and fatty acids, which is a sign of altered metabolism, the researchers said.

Taken together, our findings map out the molecular mechanisms that give rise to the cardinal features of the disease, Seidman said. They can help explain how chronically overexerted heart cells with high energy consumption in a state of metabolic stress can, over time,lead to a thickened heart muscle that contracts and relaxes abnormally and eventually becomes prone to arrhythmias, dysfunction and failure.

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Revving the Engine - Harvard Medical School

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Biomedical researchers from Texas Tech University Health Sciences Center El Paso (TTUHSC El Paso) and The University of Texas at El Paso (UTEP) are collaborating to develop artificial mini-hearts using 3D bioprinting technology for space.

These heart-tissue structures will be sent to the International Space Station (ISS) to gain insight into how microgravity affects the function of the human heart, particularly in regards to the health condition known as cardiac atrophy.

The artificial mini-heart, otherwise known as a cardiac organoid, will be produced using a combination of human stem cells and 3D bioprinting. The project, which began in September 2019, will take course over the next three years. It is funded by the National Science Foundation (NSF) and the space stations U.S. National Laboratory.

TTUHSC El Paso faculty scientist Munmun Chattopadhyay, Ph.D., a researcher on the project, states:

Knowledge gathered from this study could be used to develop technologies and therapeutic strategies to better combat tissue atrophy experienced by astronauts, as well as open the door for improved treatments for people who suffer from serious heart issues due to illness.

How does microgravity affect our hearts?

Researchers taking part in the project are Dr. Chattopadhyay and UTEP biomedical engineer Binata Joddar, Ph.D. Dr. Chattopadhyay is an assistant professor in TTUHSC El Pasos Center of Emphasis in Diabetes and Metabolism, part of the Paul L. Foster School of Medicines Department of Molecular and Translational Medicine. Dr. Joddar is an assistant professor in the UTEP College of Engineering and leads research in the universitys Inspired Materials and Stem Cell-Based Tissue Engineering Laboratory.

Together, the researchers will collaborate to 3D bioprint small cardiac organoids using human stem cells. These heart-tissue structures will then be sent to the ISS, where they will be exposed to the near-weightless environment of the orbiting space station. The researchers hope that this will provide a better understanding of cardiac atrophy, which is a reduction and weakening of heart tissue, leading to difficulty pumping blood to the body. This condition commonly affects astronauts who spend long periods of time in microgravity, which causes significant problems as a weakened heart muscle can lead to symptoms such as fainting, irregular heartbeat, heart valve problems, and even heart failure.

Cardiac atrophy and a related condition, cardiac fibrosis, is a very big problem in our community. People suffering from diseases such as diabetes, muscular dystrophy and cancer, and conditions such as sepsis and congestive heart failure, often experience cardiac dysfunction and tissue damage, comments Dr. Chattopadhyay.

The first phase of the project will focus on research design. During this stage, taking place over the first year, Dr. Joddar will use 3D printing to fabricate the cardiac organoids. This will be achieved by coupling cardiac cells in physiological ratios to mimic heart tissue. Moving on to the second year, the researchers will be preparing the organoid payload for a rocket launch and mission in space. The third and final year of the project will center on analyzing the data from the experiment once the organoids have been returned to Earth.

Additionally, Dr. Chattopadhyay and Dr. Joddars project will provide an educational opportunity for the El Paso community. A workshop for K-12 students will be set up engaging young minds in the local area around the subject of tissue engineering, with focus placed on projects taking place on the space station. A seminar will also be provided for medical students, interns and residents to enable a discussion regarding the benefits and challenges of transitioning research from Earth-based laboratories into space.

3D bioprinting aboard the ISS

The TTUHSC El Paso and UTEP collaborative research project is one of just five research proposals selected by the NSF and ISS National Lab in 2019 as part of the organizations collaboration on tissue-engineering research funding. The NSF awarded Dr. Chattopadhyay $256,892 and Dr. Joddar $259,350 for their roles in the project.

A number of 3D bioprinting research projects have taken place aboard the ISS, as companies and organizations seek further understanding of how space flight affects astronauts.

For example, Russian bio-technical research laboratory 3D Bioprinting Solutions developed its Organ.Aut magnetic 3D bioprinter to study how living organisms are affected by long flights in outer space. In 2018, it was delivered to the ISS onboard the Soyuz MS-11 manned spacecraft following a previous failed launch from the Soyuz MS-10 spaceflight. In late 2019 it was announced that the company was able to 3D bioprint bone tissue in zero gravity aboard the ISS using the Organ.Aut. The experiment is part of a plan to create bone implants for astronaut transplantation during long-term interplanetary expeditions.

Additionally, the 3D BioFabrication Facility (BFF) bioprinter from nScrypt, a Florida-based 3D printing system manufacturer, and spaceflight equipment developer Techshot is also onboard the ISS. Delivered to the ISS aboard the SpaceX CRS-18 cargo mission in 2019, the system is capable of manufacturing human tissue in microgravity conditions. It was sent to the ISS in order to facilitate the production of self-supporting tissues that could lead to the development of therapeutic treatments.

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Featured image shows the ISS Exterior. Photo via Roscosmos/ NASA/TTUHSC El Paso.

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El Paso scientists to deliver 3D bioprinted miniature hearts to the ISS - 3D Printing Industry

Cardiac Stem Cells Comments Off on El Paso scientists to deliver 3D bioprinted miniature hearts to the ISS – 3D Printing Industry | January 27th, 2020

MAPLE GROVE, Minn., Jan. 27, 2020 /PRNewswire/ --StemoniX, a biotech company revolutionizing how new medicines are discovered, announced today that its Director of Applications, Oivin Guichert, Ph.D., will deliver a podium presentation highlighting the company's microBrain technology at the SLAS (Society for Laboratory Automation and Screening) 2020 International Conference & Exhibition at the San Diego Convention Center, Jan. 27-29, 2020. The presentation will be featured as part of the Assay Development and Screening Session during the annual meeting.

During the podium presentation, entitled "New innovation to solve unmet needs: Implementing human induced pluripotent stem cell-derived neural spheroids as a robust screening platform for phenotypic-based central nervous system drug discovery," Dr. Guichert will detail how performing a high-throughput functional screening assay on StemoniX's human induced pluripotent stem cell (iPSC)-derived 3D neural spheroid platform demonstrated the ability to identify a wide range of hits spanning multiple target areas. He will highlight how this model could provide relevant human platforms for disease-specific drug discovery to help overcome traditional hurdles of CNS-targeted drug discovery and development efforts.

Ping Yeh, co-founder and CEO of StemoniX, said: "The SLAS 2020 International Conference & Exhibitionis an ideal event to showcase the value potential of our microOrgan platform and AnalytiX data management and analytical software. As presented by Dr. Guichert and in the six posters, microBrain, microHeart, microPancreas and AnalytiX offer the potential to reshape how drugs are discovered and developed by providing the opportunity to go from model to molecule to validated drug in a fraction of the time and cost required with traditional methods. This includes the near-term potential to identify and advance novel therapeutic targets for Rett syndrome by leveraging our groundbreaking in vitro microBrain model in partnership with AI drug discovery pioneer, Atomwise."

Podium Presentation Details

Title:

New innovation to solve unmet needs: Implementing human induced pluripotent stem cell-derived neural spheroids as a robust screening platform for phenotypic-based central nervous system drug discovery

Session:

Assay Development and Screening

Event

SLAS 2020 International Conference & Exhibition

Date:

Tuesday, January 28, 2020

Time:

4:00 4:30 p.m. PST

Location:

San Diego Convention Center

Room/Location:

6C

Poster Presentations:

About StemoniXStemoniX is accelerating the discovery of new medicines to treat challenging diseases via the world's first ready-to-use assay plates containing living human microOrgans, including electrophysiologically active neural (microBrain) and cardiac (microHeart) cells. Predictive, accurate, and consistent, StemoniX's products combined with its proprietary data management and analytical tools (AnalytiX) are revolutionizing traditional drug discovery and development by radically improving the speed, accuracy and costs required to identify new drugs and conduct initial human cell toxicity and efficacy testing. Through its Discovery as a Service offering, the company partners with organizations to screen compounds as well as to create customized microOrgan models and assays tailored to specific discovery and toxicity needs. Visit http://www.stemonix.com to learn how StemoniX is helping global institutions humanize drug discovery and development to bring the most promising medicines to patients.

Tiberend Strategic Advisors, Inc.

Investor Contact:Maureen McEnroe, CFA+1.212.375.2664mmcenroe@tiberend.com

Media Contact:Ingrid Mezo+1.646.604.5150imezo@tiberend.com

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StemoniX's microBrain to be Featured in Podium Presentation at SLAS 2020 International Conference & Exhibition - Crow River Media

Cardiac Stem Cells Comments Off on StemoniX’s microBrain to be Featured in Podium Presentation at SLAS 2020 International Conference & Exhibition – Crow River Media | January 27th, 2020

Bones are an important part of the musculoskeletal system. This article, the first in a two-part series on the skeletal system, reviews the anatomy and physiology of bone

The skeletal system is formed of bones and cartilage, which are connected by ligaments to form a framework for the remainder of the body tissues. This article, the first in a two-part series on the structure and function of the skeletal system, reviews the anatomy and physiology of bone. Understanding the structure and purpose of the bone allows nurses to understand common pathophysiology and consider the most-appropriate steps to improve musculoskeletal health.

Citation: Walker J (2020) Skeletal system 1: the anatomy and physiology of bones. Nursing Times [online]; 116: 2, 38-42.

Author: Jennie Walker is principal lecturer, Nottingham Trent University.

The skeletal system is composed of bones and cartilage connected by ligaments to form a framework for the rest of the body tissues. There are two parts to the skeleton:

As well as contributing to the bodys overall shape, the skeletal system has several key functions, including:

Bones are a site of attachment for ligaments and tendons, providing a skeletal framework that can produce movement through the coordinated use of levers, muscles, tendons and ligaments. The bones act as levers, while the muscles generate the forces responsible for moving the bones.

Bones provide protective boundaries for soft organs: the cranium around the brain, the vertebral column surrounding the spinal cord, the ribcage containing the heart and lungs, and the pelvis protecting the urogenital organs.

As the main reservoirs for minerals in the body, bones contain approximately 99% of the bodys calcium, 85% of its phosphate and 50% of its magnesium (Bartl and Bartl, 2017). They are essential in maintaining homoeostasis of minerals in the blood with minerals stored in the bone are released in response to the bodys demands, with levels maintained and regulated by hormones, such as parathyroid hormone.

Blood cells are formed from haemopoietic stem cells present in red bone marrow. Babies are born with only red bone marrow; over time this is replaced by yellow marrow due to a decrease in erythropoietin, the hormone responsible for stimulating the production of erythrocytes (red blood cells) in the bone marrow. By adulthood, the amount of red marrow has halved, and this reduces further to around 30% in older age (Robson and Syndercombe Court, 2018).

Yellow bone marrow (Fig 1) acts as a potential energy reserve for the body; it consists largely of adipose cells, which store triglycerides (a type of lipid that occurs naturally in the blood) (Tortora and Derrickson, 2009).

Bone matrix has three main components:

Organic matrix (osteoid) is made up of approximately 90% type-I collagen fibres and 10% other proteins, such as glycoprotein, osteocalcin, and proteoglycans (Bartl and Bartl, 2017). It forms the framework for bones, which are hardened through the deposit of the calcium and other minerals around the fibres (Robson and Syndercombe Court, 2018).

Mineral salts are first deposited between the gaps in the collagen layers with once these spaces are filled, minerals accumulate around the collagen fibres, crystallising and causing the tissue to harden; this process is called ossification (Tortora and Derrickson, 2009). The hardness of the bone depends on the type and quantity of the minerals available for the body to use; hydroxyapatite is one of the main minerals present in bones.

While bones need sufficient minerals to strengthen them, they also need to prevent being broken by maintaining sufficient flexibility to withstand the daily forces exerted on them. This flexibility and tensile strength of bone is derived from the collagen fibres. Over-mineralisation of the fibres or impaired collagen production can increase the brittleness of bones as with the genetic disorder osteogenesis imperfecta and increase bone fragility (Ralston and McInnes, 2014).

Bone architecture is made up of two types of bone tissue:

Also known as compact bone, this dense outer layer provides support and protection for the inner cancellous structure. Cortical bone comprises three elements:

The periosteum is a tough, fibrous outer membrane. It is highly vascular and almost completely covers the bone, except for the surfaces that form joints; these are covered by hyaline cartilage. Tendons and ligaments attach to the outer layer of the periosteum, whereas the inner layer contains osteoblasts (bone-forming cells) and osteoclasts (bone-resorbing cells) responsible for bone remodelling.

The function of the periosteum is to:

It also contains Volkmanns canals, small channels running perpendicular to the diaphysis of the bone (Fig 1); these convey blood vessels, lymph vessels and nerves from the periosteal surface through to the intracortical layer. The periosteum has numerous sensory fibres, so bone injuries (such as fractures or tumours) can be extremely painful (Drake et al, 2019).

The intracortical bone is organised into structural units, referred to as osteons or Haversian systems (Fig 2). These are cylindrical structures, composed of concentric layers of bone called lamellae, whose structure contributes to the strength of the cortical bone. Osteocytes (mature bone cells) sit in the small spaces between the concentric layers of lamellae, which are known as lacunae. Canaliculi are microscopic canals between the lacunae, in which the osteocytes are networked to each other by filamentous extensions. In the centre of each osteon is a central (Haversian) canal through which the blood vessels, lymph vessels and nerves pass. These central canals tend to run parallel to the axis of the bone; Volkmanns canals connect adjacent osteons and the blood vessels of the central canals with the periosteum.

The endosteum consists of a thin layer of connective tissue that lines the inside of the cortical surface (Bartl and Bartl, 2017) (Fig1).

Also known as spongy bone, cancellous bone is found in the outer cortical layer. It is formed of lamellae arranged in an irregular lattice structure of trabeculae, which gives a honeycomb appearance. The large gaps between the trabeculae help make the bones lighter, and so easier to mobilise.

Trabeculae are characteristically oriented along the lines of stress to help resist forces and reduce the risk of fracture (Tortora and Derrickson, 2009). The closer the trabecular structures are spaced, the greater the stability and structure of the bone (Bartl and Bartl, 2017). Red or yellow bone marrow exists in these spaces (Robson and Syndercombe Court, 2018). Red bone marrow in adults is found in the ribs, sternum, vertebrae and ends of long bones (Tortora and Derrickson, 2009); it is haemopoietic tissue, which produces erythrocytes, leucocytes (white blood cells) and platelets.

Bone and marrow are highly vascularised and account for approximately 10-20% of cardiac output (Bartl and Bartl, 2017). Blood vessels in bone are necessary for nearly all skeletal functions, including the delivery of oxygen and nutrients, homoeostasis and repair (Tomlinson and Silva, 2013). The blood supply in long bones is derived from the nutrient artery and the periosteal, epiphyseal and metaphyseal arteries (Iyer, 2019).

Each artery is also accompanied by nerve fibres, which branch into the marrow cavities. Arteries are the main source of blood and nutrients for long bones, entering through the nutrient foramen, then dividing into ascending and descending branches. The ends of long bones are supplied by the metaphyseal and epiphyseal arteries, which arise from the arteries from the associated joint (Bartl and Bartl, 2017).

If the blood supply to bone is disrupted, it can result in the death of bone tissue (osteonecrosis). A common example is following a fracture to the femoral neck, which disrupts the blood supply to the femoral head and causes the bone tissue to become necrotic. The femoral head structure then collapses, causing pain and dysfunction.

Bones begin to form in utero in the first eight weeks following fertilisation (Moini, 2019). The embryonic skeleton is first formed of mesenchyme (connective tissue) structures; this primitive skeleton is referred to as the skeletal template. These structures are then developed into bone, either through intramembranous ossification or endochondral ossification (replacing cartilage with bone).

Bones are classified according to their shape (Box1). Flat bones develop from membrane (membrane models) and sesamoid bones from tendon (tendon models) (Waugh and Grant, 2018). The term intra-membranous ossification describes the direct conversion of mesenchyme structures to bone, in which the fibrous tissues become ossified as the mesenchymal stem cells differentiate into osteoblasts. The osteoblasts then start to lay down bone matrix, which becomes ossified to form new bone.

Box 1. Types of bones

Long bones typically longer than they are wide (such as humerus, radius, tibia, femur), they comprise a diaphysis (shaft) and epiphyses at the distal and proximal ends, joining at the metaphysis. In growing bone, this is the site where growth occurs and is known as the epiphyseal growth plate. Most long bones are located in the appendicular skeleton and function as levers to produce movement

Short bones small and roughly cube-shaped, these contain mainly cancellous bone, with a thin outer layer of cortical bone (such as the bones in the hands and tarsal bones in the feet)

Flat bones thin and usually slightly curved, typically containing a thin layer of cancellous bone surrounded by cortical bone (examples include the skull, ribs and scapula). Most are located in the axial skeleton and offer protection to underlying structures

Irregular bones bones that do not fit in other categories because they have a range of different characteristics. They are formed of cancellous bone, with an outer layer of cortical bone (for example, the vertebrae and the pelvis)

Sesamoid bones round or oval bones (such as the patella), which develop in tendons

Long, short and irregular bones develop from an initial model of hyaline cartilage (cartilage models). Once the cartilage model has been formed, the osteoblasts gradually replace the cartilage with bone matrix through endochondral ossification (Robson and Syndercombe Court, 2018). Mineralisation starts at the centre of the cartilage structure, which is known as the primary ossification centre. Secondary ossification centres also form at the epiphyses (epiphyseal growth plates) (Danning, 2019). The epiphyseal growth plate is composed of hyaline cartilage and has four regions (Fig3):

Resting or quiescent zone situated closest to the epiphysis, this is composed of small scattered chondrocytes with a low proliferation rate and anchors the growth plate to the epiphysis;

Growth or proliferation zone this area has larger chondrocytes, arranged like stacks of coins, which divide and are responsible for the longitudinal growth of the bone;

Hypertrophic zone this consists of large maturing chondrocytes, which migrate towards the metaphysis. There is no new growth at this layer;

Calcification zone this final zone of the growth plate is only a few cells thick. Through the process of endochondral ossification, the cells in this zone become ossified and form part of the new diaphysis (Tortora and Derrickson, 2009).

Bones are not fully developed at birth, and continue to form until skeletal maturity is reached. By the end of adolescence around 90% of adult bone is formed and skeletal maturity occurs at around 20-25 years, although this can vary depending on geographical location and socio-economic conditions; for example, malnutrition may delay bone maturity (Drake et al, 2019; Bartl and Bartl, 2017). In rare cases, a genetic mutation can disrupt cartilage development, and therefore the development of bone. This can result in reduced growth and short stature and is known as achondroplasia.

The human growth hormone (somatotropin) is the main stimulus for growth at the epiphyseal growth plates. During puberty, levels of sex hormones (oestrogen and testosterone) increase, which stops cell division within the growth plate. As the chondrocytes in the proliferation zone stop dividing, the growth plate thins and eventually calcifies, and longitudinal bone growth stops (Ralston and McInnes, 2014). Males are on average taller than females because male puberty tends to occur later, so male bones have more time to grow (Waugh and Grant, 2018). Over-secretion of human growth hormone during childhood can produce gigantism, whereby the person is taller and heavier than usually expected, while over-secretion in adults results in a condition called acromegaly.

If there is a fracture in the epiphyseal growth plate while bones are still growing, this can subsequently inhibit bone growth, resulting in reduced bone formation and the bone being shorter. It may also cause misalignment of the joint surfaces and cause a predisposition to developing secondary arthritis later in life. A discrepancy in leg length can lead to pelvic obliquity, with subsequent scoliosis caused by trying to compensate for the difference.

Once bone has formed and matured, it undergoes constant remodelling by osteoclasts and osteoblasts, whereby old bone tissue is replaced by new bone tissue (Fig4). Bone remodelling has several functions, including mobilisation of calcium and other minerals from the skeletal tissue to maintain serum homoeostasis, replacing old tissue and repairing damaged bone, as well as helping the body adapt to different forces, loads and stress applied to the skeleton.

Calcium plays a significant role in the body and is required for muscle contraction, nerve conduction, cell division and blood coagulation. As only 1% of the bodys calcium is in the blood, the skeleton acts as storage facility, releasing calcium in response to the bodys demands. Serum calcium levels are tightly regulated by two hormones, which work antagonistically to maintain homoeostasis. Calcitonin facilitates the deposition of calcium to bone, lowering the serum levels, whereas the parathyroid hormone stimulates the release of calcium from bone, raising the serum calcium levels.

Osteoclasts are large multinucleated cells typically found at sites where there is active bone growth, repair or remodelling, such as around the periosteum, within the endosteum and in the removal of calluses formed during fracture healing (Waugh and Grant, 2018). The osteoclast cell membrane has numerous folds that face the surface of the bone and osteoclasts break down bone tissue by secreting lysosomal enzymes and acids into the space between the ruffled membrane (Robson and Syndercombe Court, 2018). These enzymes dissolve the minerals and some of the bone matrix. The minerals are released from the bone matrix into the extracellular space and the rest of the matrix is phagocytosed and metabolised in the cytoplasm of the osteoclasts (Bartl and Bartl, 2017). Once the area of bone has been resorbed, the osteoclasts move on, while the osteoblasts move in to rebuild the bone matrix.

Osteoblasts synthesise collagen fibres and other organic components that make up the bone matrix. They also secrete alkaline phosphatase, which initiates calcification through the deposit of calcium and other minerals around the matrix (Robson and Syndercombe Court, 2018). As the osteoblasts deposit new bone tissue around themselves, they become trapped in pockets of bone called lacunae. Once this happens, the cells differentiate into osteocytes, which are mature bone cells that no longer secrete bone matrix.

The remodelling process is achieved through the balanced activity of osteoclasts and osteoblasts. If bone is built without the appropriate balance of osteocytes, it results in abnormally thick bone or bony spurs. Conversely, too much tissue loss or calcium depletion can lead to fragile bone that is more susceptible to fracture. The larger surface area of cancellous bones is associated with a higher remodelling rate than cortical bone (Bartl and Bartl, 2017), which means osteoporosis is more evident in bones with a high proportion of cancellous bone, such as the head/neck of femur or vertebral bones (Robson and Syndercombe Court, 2018). Changes in the remodelling balance may also occur due to pathological conditions, such as Pagets disease of bone, a condition characterised by focal areas of increased and disorganised bone remodelling affecting one or more bones. Typical features on X-ray include focal patches of lysis or sclerosis, cortical thickening, disorganised trabeculae and trabecular thickening.

As the body ages, bone may lose some of its strength and elasticity, making it more susceptible to fracture. This is due to the loss of mineral in the matrix and a reduction in the flexibility of the collagen.

Adequate intake of vitamins and minerals is essential for optimum bone formation and ongoing bone health. Two of the most important are calcium and vitamin D, but many others are needed to keep bones strong and healthy (Box2).

Box 2. Vitamins and minerals needed for bone health

Key nutritional requirements for bone health include minerals such as calcium and phosphorus, as well as smaller qualities of fluoride, manganese, and iron (Robson and Syndercombe Court, 2018). Calcium, phosphorus and vitamin D are essential for effective bone mineralisation. Vitamin D promotes calcium absorption in the intestines, and deficiency in calcium or vitamin D can predispose an individual to ineffective mineralisation and increased risk of developing conditions such as osteoporosis and osteomalacia.

Other key vitamins for healthy bones include vitamin A for osteoblast function and vitamin C for collagen synthesis (Waugh and Grant, 2018).

Physical exercise, in particular weight-bearing exercise, is important in maintaining or increasing bone mineral density and the overall quality and strength of the bone. This is because osteoblasts are stimulated by load-bearing exercise and so bones subjected to mechanical stresses undergo a higher rate of bone remodelling. Reduced skeletal loading is associated with an increased risk of developing osteoporosis (Robson and Syndercombe Court, 2018).

Bones are an important part of the musculoskeletal system and serve many core functions, as well as supporting the bodys structure and facilitating movement. Bone is a dynamic structure, which is continually remodelled in response to stresses placed on the body. Changes to this remodelling process, or inadequate intake of nutrients, can result in changes to bone structure that may predispose the body to increased risk of fracture. Part2 of this series will review the structure and function of the skeletal system.

Bartl R, Bartl C (2017) Structure and architecture of bone. In: Bone Disorder: Biology, Diagnosis, Prevention, Therapy.

Danning CL (2019) Structure and function of the musculoskeletal system. In: Banasik JL, Copstead L-EC (eds) Pathophysiology. St Louis, MO: Elsevier.

Drake RL et al (eds) (2019) Grays Anatomy for Students. London: Elsevier.

Iyer KM (2019) Anatomy of bone, fracture, and fracture healing. In: Iyer KM, Khan WS (eds) General Principles of Orthopedics and Trauma. London: Springer.

Moini J (2019) Bone tissues and the skeletal system. In: Anatomy and Physiology for Health Professionals. Burlington, MA: Jones and Bartlett.

Ralston SH, McInnes IB (2014) Rheumatology and bone disease. In: Walker BR et al (eds) Davidsons Principles and Practice of Medicine. Edinburgh: Churchill Livingstone.

Robson L, Syndercombe Court D (2018) Bone, muscle, skin and connective tissue. In: Naish J, Syndercombe Court D (eds) Medical Sciences. London: Elsevier

Tomlinson RE, Silva MJ (2013) Skeletal blood flow in bone repair and maintenance. Bone Research; 1: 4, 311-322.

Tortora GJ, Derrickson B (2009) The skeletal system: bone tissue. In: Principles of Anatomy and Physiology. Chichester: John Wiley & Sons.

Waugh A, Grant A (2018) The musculoskeletal system. In: Ross & Wilson Anatomy and Physiology in Health and Illness. London: Elsevier.

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Skeletal system 1: the anatomy and physiology of bones - Nursing Times

Cardiac Stem Cells Comments Off on Skeletal system 1: the anatomy and physiology of bones – Nursing Times | January 27th, 2020

OSAKA An Osaka University team said it has carried out the worlds first transplant of cardiac muscle cells created from iPS cells in a physician-initiated clinical trial.

In the clinical project to verify the safety and efficacy of the therapy using induced pluripotent stem cells, Yoshiki Sawa, a professor in the universitys cardiovascular surgery unit, and colleagues aim to transplant heart muscle cell sheets into 10 patients suffering from serious heart malfunction caused by ischemic cardiomyopathy.

The cells on the degradable sheets attached to the surface of the patients hearts are expected to grow to secrete a protein that can regenerate blood vessels and improve cardiac function. The iPS cells have already been derived from healthy donors blood cells and stored.

The researchers said Monday they decided to conduct a clinical trial instead of a clinical study in hopes of obtaining approval from the health ministry for clinical applications as soon as possible.

The trial involves stringently evaluating risks, particularly cancer possibilities, and the efficacy of transplanting some 100 million cells per patient that may include tumor cells.

This is the second iPS cell-based clinical trial in Japan. The first was conducted on eye disease patients by the Riken research institute.

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Osaka University transplants iPS cell-based heart cells in world's first clinical trial - The Japan Times

IPS Cell Therapy Comments Off on Osaka University transplants iPS cell-based heart cells in world’s first clinical trial – The Japan Times | January 27th, 2020

CHICAGO Brian Wallach wasnt supposed to live to see his younger daughters first birthday.

Diagnosed with amyotrophic lateral sclerosis (ALS), a terminal disease with no cure, doctors told him in 2017 that he might have six months to live.

Today, hes focused on being there for his daughters future firsts: kindergarten drop-off, middle school dance, wedding day.

More than two years after his diagnosis, he has been lucky, he said, to experience relatively limited progression of his disease. After some balance issues, the Kenilworth resident now uses a cane or, as he is careful to specify, a cool walking stick to get around.

When Wallach was diagnosed, neither he nor his wife, Sandra Abrevaya, knew much about ALS, a neurodegenerative disease that affects nerve cells in the brain and the spinal cord, eventually paralyzing even the bodys ability to breathe.

In response to Wallachs diagnosis, the couple, both 39, launched I AM ALS in 2019. Former staffers in the Obama White House, they marshaled lessons learned while campaigning gathering information, forming consensus, considering the impossible possible to build a force to mobilize hope and change for those facing a disease they say can and should be cured.

Rays of hope are beginning to emerge through an innovative trial that received FDA approval last week to test several drugs at the same time, a bipartisan congressional caucus, doubled federal funding, and support from groups like the Chan Zuckerberg Initiative, which gave the couples organization a $453,000 grant in September.

Last year we made hope a word that was OK to use, Wallach said. This year we have to make hope real.

Audaciousness is the only option, the couple says, in their race against the clock.

Wallach logged 120,000 miles in the air last year, including traveling to Washington, D.C., in April, where he testified before Congress and asked legislators to amp up funding.

Last year, every time someone said, Do you want to speak to us, I said, yes. Every time someone said, Theres a meeting, I said, Im going. he said. Every time there was anything, I said, Great, Im on the plane.

Until October, when Wallach fell while exiting a Lyft in Boston after swinging a heavy backpack onto his back. Thirteen staples in his head later, and after terrifying Abrevaya with a phone call, the two agreed he wouldnt travel alone anymore. Hes maintaining momentum for the cause with more hours in his home office and fewer in airports.

In December, I AM ALS debuted billboards around Times Square as part of its #CuresForAll campaign aimed at informing the public about the impact a cure or better treatment for a neurodegenerative disease can have on other diseases such as multiple sclerosis, Alzheimers and Parkinsons. ALS patients and their families from states including Michigan, Maine and Colorado were in New York for the launch.

The billboards noted the number of people lost to ALS each day 16 with photographs of those who died in 2019. Days earlier, Pete Frates, a founder of the viral fundraiser the Ice Bucket Challenge, which raised $115 million, had died. He was 34.

The campaign was also shared on social media. The posts expressed the suffering and loss nationwide: a mother wrote about her son who was diagnosed at 20 and died at 28; a son posted in honor of his dad; Colorado Rep. Jason Crow posted a message honoring his cousin.

Its time, the couple said, to switch ALS conversations from a diagnosis rooted in darkness to the faces of people bravely moving forward. They want to speed development of potential cures and give patients more access to experimental treatments.

Thats not an unreasonable goal, said Sabrina Paganoni, a faculty member at The Sean M. Healey & AMG Center for ALS at Mass General in Boston, which plans to test at least five different medications for ALS at the same time, a first for the disease and something she said could be a huge turning point.

On Wednesday, the Healey Center announced it received FDA approval to move forward with testing the first three drugs: Zilucoplan, Verdiperstat and CNM-Au8. Similar to how cancer drugs are already tested, this gives patients access to more treatments and allows researchers to quickly collect data and accelerate the pace toward a cure.

This is a very exciting time in the history of ALS, Paganoni said. I think this is going to be the decade when ALS is changed from a rapidly fatal disease to a more chronic disease that we can manage.

For years, Steve Perrin, the chief executive officer at the ALS Therapy Development Institute, has monitored clinical trials for ALS. So far, he said, the two drugs approved by the FDA, Radicava and Rilutek, are a very marginal slowing down of disease.

This year, he said the quality of drugs going into trials seems improved. He is excited about several trials, including one studying stem cells and another testing a drug to potentially slow progression in some patients.

As a patient you want to see something measurable, and I dont mean measurable in days, he said. If Im a patient, I want to see something, and I want hope for myself and my family. I want something that is going to slow the disease down so I can watch my kids growing up, I can watch them graduate from college, I can watch them marry.

But that takes resources.

We are in a time when we can reasonably say that theres going to be new treatments available, Paganoni said. But we need more funding and support, so all of this can happen, and happen soon.

Nearly every moment feels like a push-pull for Wallach and Abrevaya.

Do they spend more precious minutes with their two daughters, ages 4 and 2, or do they spend time away, among strangers on a plane, in a researchers office, walking the halls of Congress with the hope that those minutes will, someday, result in time banked to create more family memories.

The hardest balance, if Im honest, is, I love every minute I have with them, Wallach said about his daughters, but I also feel this pressing sense of, I need to be working towards a goal of actually finding a cure.

Were doing that so we have a shot at a real future together, Abrevaya said about their time spent traveling and advocating.

At home, when the family heads for the door, the toddlers reach for their fathers shoes, and they get his walking stick.

While that both fills your heart with joy and appreciation, its also painful that your toddlers are being put in this position, Abrevaya said.

The parents guard normalcy. They take their daughters to swim at the neighborhood pool and on vacation with friends. Wallach wishes he could lift them above his head to touch the ceiling, like their uncle can. But he can lie on the floor and play with them; he can listen to them belt out songs on their purple karaoke machine.

They find ways to lighten a heavy subject. On New Years Eve, the two danced in a video on the foundations Instagram, singing into hairbrushes, and Wallach promised to get an ALS: You Gone tattoo if 20,000 people donated $10 to a Healey Center research fundraiser. It raised $40,000 in 24 hours, Wallach said. No matter the outcome, he plans to get the tattoo.

The couple, who both work full-time jobs Abrevaya is the president of nonprofit Thrive, Wallach works at law firm Skadden, Arps, Slate, Meagher & Flom want more research, to create a patient navigation system, and to gather signatures for a letter asking new FDA commissioner Stephen Hahn to speed ALS patients access to possible treatments.

And they keep looking for light. But it takes work.

Changing life with ALS for Wallach, and for other patients and their families, requires bold action from people with the power to make change: politicians, researchers, philanthropists.

As they meet others with ALS, they welcome new friends and face the pain of losing some.

It does make you uniquely urgent in what you do, Wallach said. You push because you have to. You push because you know that the time that we have is precious, and that you want to see 20 years from now. And know that you can make that happen.

(EDITORS: STORY CAN END HERE)

Wallach often shares moments about his ALS journey on Twitter with his 40,000 followers. Recently, he shared something he wasnt sure he should. It was a time he was unable to find light.

On a recent night, he woke up to pain hes had for the past few months, radiating from his right hip to his right calf.

He clutched a stuffed llama his daughter gave him. And he began to cry.

I cried because of the pain. I cried because I couldnt be the father to my girls I dreamed of being, he wrote. I cried because I couldnt be the husband to my wife I dream of being. Because I saw the future zooming ahead, and for a brief moment I wondered if I would be a part of it.

His wife heard him crying that night. She asked what was wrong. And he said maybe they would be better off if he left, living instead in an assisted living facility. Their daughters, he told her, could have a dad who could do everything he dreamed of doing.

She looked at him in the dark. You are my light, she said. You are their light. The only way you are leaving us is if you die in my arms, and we arent going to let that happen for a long, long, long time.

Finally, he smiled.

2020 Chicago Tribune

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PHOTOS (for help with images, contact 312-222-4194): ALS-BATTLE

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In a race against terminal illness, former Obama staffer with ALS and his wife find new hope a year later - Bryan-College Station Eagle

Spinal Cord Stem Cells Comments Off on In a race against terminal illness, former Obama staffer with ALS and his wife find new hope a year later – Bryan-College Station Eagle | January 26th, 2020