By turning stem cells into brain cells, Aspen Neuroscience hopes to rewind the progress of Parkinson’s disease – FierceBiotech
By daniellenierenberg
The idea of a cell therapy for Parkinsons disease starts out simple: Symptoms of the progressive disease are largely driven by the deaths of dopamine-producing neurons found deep within the brain. With lower levels of the neurotransmitter come the characteristic tremors, rigidity and slow movements.
By replacing those lost nerve cells with new dopamine producers, researchers hope to renew the brains connection to the bodys muscles and improve a persons overall motor function.
But in the brain, everything becomes more complicated. On top of the risk of immune system rejection that comes with any kind of living tissue transplant, its important to make sure the implanted cells function correctly and do not pick up any dangerous genetic mutations as they grow.
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Now, a new company, Aspen Neuroscience, aims to tackle both obstacles at once.
First, the startup hopes to avoid any harmful immune reactions by using a patients own cells as a starting point. Then, Aspen plans to implement a rigorous quality control program employing whole genome sequencing and artificial intelligence to make sure the cells stay in line as theyre processed and readied for the procedure.
And to do it, the San Diego-based company is starting out with $6.5 million in seed money plus an impressive roster of names.
They are led by neurology researcher Howard Federoff, previously vice chancellor for health affairs and CEO of the University of California, Irvine health system as well as the executive dean of medicine at Georgetown University. Hes joined by Aspen co-founder and stem cell scientist Jeanne Loring, founding director and professor emeritus of the Center for Regenerative Medicine at the Scripps Research Institute.
Meanwhile, the seed round was led by Domain Associates and Axon Ventures with additional backing from Alexandria Venture Investments, Arch Venture Partners, OrbiMed and Section 32.
Aspen looks to combine its expertise in stem cell biology, genomics and neurology to offer the first autologous cell therapy for Parkinsons diseasewhile others in the space have pursued allogeneic routes, or therapies derived from donors other than the patient.
The process starts with a culture of the patients skin cells, which are then genetically induced to become pluripotent stem cellsor cells capable of differentiating into any other cell type in the body. These are then chemically nudged further to transform into precursor versions of the dopamine-producing neurons, which are typically found in the midbrain and regions responsible for the movement of limbs.
We can say without any equivocation that we can produce the population of cells necessary to transplant, and in a short enough period of time to have a potential beneficial impact on the evolution of the disease, said Federoff, who has also served as chair of the NIHs Recombinant DNA Advisory Committee and helped lead the U.S. Parkinsons Disease Gene Therapy Study Group.
We envisage that this will set back the clock on patients who have Parkinsons, unlike any other therapy that we know of, he told FierceMedTech in an interview.
The number of cells needed would be much smaller compared to other cell therapies and cancer treatments. The healthy human brain contains only about 200,000 dopamine-producing nerve cells, split between its two hemispheres, while patients with Parkinsons disease have lost about 50% or more of those neurons.
Aspen aims to evaluate two doses: one that aims to replace about 60% to 65% of a persons normal cell complement and another larger treatment, Federoff said.
Those smaller doses, as well as starting with a patients donor cells, help make the treatment safer to produce by requiring fewer steps. Each cycle of cell division and multiplication to increase their numbers carries the risk of introducing genetic mutations.
As the cells are grown, they are consistently evaluated with data-driven techniques pioneered by Lorings laboratory. Using whole genome RNA sequencing, Aspen will match the cells up at every stage with a genetic barcode taken from each patient at the start. This will allow them to look for changes, duplications or deletions in the pluripotent stem cell genome.
If the cells harbor mutations that are cancer drivers, we don't want to put those into people, Loring said. The only way is to check the sequencing before we transplant them.
The cells used in the transplant procedure arent fully grown; as neuron progenitors, they mimic the development steps seen in the brain of a growing fetus after theyre placed in the body as they wire themselves up to other neural structures and begin to form new networks of their own.
We anticipate that they will manufacture and release dopamine in a manner that is consistent with synaptic neurotransmission and the process of communicating from cell-to-cell, said Federoff. They will take up dopamine from synapses when it has done its business, bring it back into the cell, and prepare it for another synaptic release.
These are not just dopamine pumps, theyre real neurons, added Loring. They will genuinely replace the cells that have been lost in every way.
Aspen plans to pursue two courses of therapy, for the two major types of Parkinsons disease. Their lead candidate is for idiopathic, or sporadic Parkinsons, while their second is a CRISPR-edited version of the therapy designed to address one of the diseases most common genetic mutations, linked to about 5% of cases.
This would not only aim to restart dopamine production in this orphan indication, but also restore the damaged enzyme GBA, which is seen as an underlying cause. Federoff and Loring expect their sequencing-based quality check system will also help catch any off-target edits linked to the use of CRISPR-Cas9.
The company has yet to secure permission from the FDA to officially launch clinical trials, but the agency has signed off on Aspens plans to prepare a trial-ready cohort of Parkinsons disease patients in the meantime. This would include the initial stages of recruitment and testing, including the selection of patients capable of having their skin cells made into pluripotent stem cells.
After it receives its go-ahead from the FDA, Aspen plans to hit the ground running,enrolling at least 176 participants in a phase 1/2 study that includes a randomized stage to determine clinical benefits.
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By turning stem cells into brain cells, Aspen Neuroscience hopes to rewind the progress of Parkinson's disease - FierceBiotech
CAMPUS: EGG DONATION – Artificial sperm cells to remove the genetic worries of sperm donation – ESHRE
By daniellenierenberg
A Campus meeting in November reviewed the arguments for and against donor conception, and the sometimes difficult ethical arguments raised by the prospect of a donor-conceived child. 'Artificial' sperm cells derived from testicular tissue or stem cells may resolve some of those arguments.
The problem is especially acute in cancers diagnosed in prepubertal boys in whom there are no sperm cells available for storage. Their only option for future fatherhood in the face of cancer treatment is adoption or donor sperm. And this, added Goossens, is not an exceptional problem. Incidence rates are around 17 cases per 100,000 population, with leukemia and CNS tumours the most commonly diagnosed. So the usual pathway to fertility preservation in these young cases is for the oncologist to warn of the risk to future fertility from the cancer treatments and refer to the fertility clinic. Biopsy of testicular tissue, of course, must be performed before any radio- or chemotherapy.
Goossens described two experimental techniques, spermatogonial stem cell retrieval and transplantation, and homotopic tissue grafting. The danger in the former procedure is a risk of introducing malignancy, so banked tissue must be free of malignant contamination. Experiments in mouse-to-mouse models have demonstrated spermatogenesis from tissue grafting, and most recently fully functional conception and delivery in a non-human primate (Grady). Similarly, experiments in mouse models with spermatogonial stem cell transplantation have so far proved efficient, with spontaneous pregnancy already possible.
Of course, the objective of this impressive experimental work is not merely a resolution to the question of genetic continuity in couples faced with third-party donation, but the future fertility and long-term quality of life of so many unfortunate young boys. Advances in cancer treatment have led to the increased survival of all children with cancer, and with it a new imperative for the restoration of their fertility. Not all cancer treatments cause complete testicular damage, but around one-third of children having treatment for pediatric cancers will end up infertile. Following the proof-of-concept study which saw the birth of Grady - in which testicular samples removed from prepubertal monkeys was frozen, thawed and regrafted under scrotal skin - the research group declared that their next logical step, with safety and feasibility apparent, is human trials.
1. Fayomi AP, Peters K, Sukhwani M, et al. Autologous grafting of cryopreserved prepubertal rhesus testis produces sperm and offspring. Science 2019; 363: 1314-1319.
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CAMPUS: EGG DONATION - Artificial sperm cells to remove the genetic worries of sperm donation - ESHRE
Got dry skin but no time for a facial? These hydration sheet masks are just as good – CNA
By daniellenierenberg
How do you know if your skin is dry? For starters, it feels rough to the touch and, in extreme cases, it may start flaking and you may even feel a tearing sensation.
The good news is that dryskin can be easily avoided, and the most efficient way to hydrate parched skin is with a hydrating sheet mask. All it takes is 15 to 20 minutes and youre on your way to plumped, glowing skin.
These days, hydration sheet masks are loaded with so much extra goodness vitamins, minerals, amino acids,organic acids, plant stem cells andpeptidesthat gointo the formulas that these can also help fight wrinkles, eliminate dark spots and lighten skin tone.
CHARLOTTE TILBURY INSTANT MAGIC FACIAL DRY SHEET MASK, S$76
Yes, this is a dry mask that you can get at Sephora. The magic all comes from the warmth of your skin: The bio-mimetic vector delivery system turns a combo of ingredients (crocus bulb extract, plant stem cells, peptides and vitamin B3) from solid to liquid.
Wear the dry mask by looping the hoops over each ear then simply activate the formula by massaging upwards to move the mask into place. After 15 minutes, take it off then gently tap the remaining essence into the skin. Youll soon notice a glow that is similar to the one you get after a super shiok facial.
But dont throw away the mask just yet the dry formula is engineered for reuse. In fact, you can use it three more times. Just slip it back (the formula side facing inward) into the resealable foil pouch for when you need it next. So really, thats four masks for the price of one.
UTENA PURESA SHEET MASK HYALURONIC ACID 5S, S$12.90
You can pick this up at Watsons. There are five masks to a pack and each one combines the benefits of hyaluronic acid and royal jelly extract, infused with a gel-like essence that works to hydrate skin like a jelly mask. To make the most of it, first smoothen the jelly bits onto the face before placing the sheet over the face.
But theres more: Peek into the packaging and youll see lots of jelly bits left. Apply these remaining bits anywhere else you want to hydrate. We personally like to spread it over the neck, the back of the palms and even the elbows. Yes, it also works as a body hydration gel.
SK II FACIAL TREATMENT MASK, S$127
Love SK IIs Facial Treatment Essence? Then youll love this sheet mask because its drenched with so much Facial Treatment Essence that it feels as if youve dunked your face into a tub of Pitera.
It contains 50 micro-nutrients like vitamins, minerals, amino acids and organic acids to condition skin's natural functions. Twenty minutes is all it takes to rehydrate, clarify complexion and have crystal clear skin.
LA MER THE TREATMENT LOTION HYDRATING MASK, S$45
Each sheet mask is infused with a full ounce of La Mers liquid energy skin hydrator and its equipped with Japanese skin-hugging technology that delivers a concentrated surge of healing hydration directly onto the skin to nourish and soften fine lines.
STARSKIN RED CARPET READY HYDRATING BIO-CELLULOSE SECOND SKIN FACE MASK, S$18
Another mask you can pick up at Sephora is one with a difference. Instead of prepping your face, you prep the mask massage the still-closed sachet to distribute the serum liquid evenly before opening.
The exclusive Bio Cellulose sheet a thin biodegradable microfibre is infused with a delicious cocktail of coconut juice, Amino acids and brown algae that work together to strengthen the skin barrier and promote moisture retention. Dull and dehydrated skin doesnt stand a chance.
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Got dry skin but no time for a facial? These hydration sheet masks are just as good - CNA
Gore, Mayo Clinic team up to deliver breakthrough medical therapies – Plastics Today
By daniellenierenberg
Materials science company W. L. Gore & Associates Inc. (Newark, DE) has announced a joint venture with the Mayo Clinic (Rochester, MN) to further develop a therapeutic system using stem cells and bio-absorbable scaffolds to treat a condition affecting patients with Crohns disease. Avobis Bio, based in Delaware, will draw on the expertise of scientists and medical professionals from both organizations to build on the encouraging results of an investigational treatment for perianal fistulas.
A debilitating condition that affects patients with Crohns disease, perianal fistulas are painful tunneling wounds connecting the anus to the skin, explained Gore in a press release. Few healing options exist, and patients endure multiple surgeries and ongoing risk of life-threatening complications, said Gore.
"Perianal fistulas are truly life-altering for Crohn's patients, and treatment options have eluded gastroenterologists and surgeons for years," added William Faubion Jr., MD, a Mayo Clinic gastroenterologist who specializes in inflammatory bowel diseases.
The innovative treatment involves harvesting a patients own mesenchymal stem cells, which then are populated on Gore's bioabsorbable polymer scaffold and surgically implanted in the fistula. A phase I clinical trial showed that 76% of patients healed within a year. If this outcome is validated in a larger trial, Gore said that it would dramatically exceed outcomes achieved with existing treatments.
This project is the initial focus of Avobis Bio, which describes its overall mission as an exploration of the use of mesenchymal stem cells combined with enabling bioabsorbable scaffolds that enhance the effectiveness of the cells in stimulating the body to heal.
Delivering a patients mesenchymal stem cells on a synthetic scaffold that biodegrades over time may be a first-of-its-kind medical therapy, noted Joe Carlson, a reporter at the Minneapolis-based Star Tribune reporting on the joint venture. If successful, Avobis Bio may one day offer a variety of tissue and organ-repair therapies combining Mayo's stem cell expertise and Gore's medical materials, he wrote.
Gore is perhaps best known to the public for its Gore-Tex outerwear, but the privately held $3.7 billion engineering and manufacturing firm sells products in an array of industries, including a line of medical devices designed to repair nonnatural holes in body organs, added Carlson. Mayo has used Gore-made devices for many years.
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Gore, Mayo Clinic team up to deliver breakthrough medical therapies - Plastics Today
Five recent drug target discoveries for pancreatic cancer – Drug Target Review
By daniellenierenberg
This article highlights some of the most recent drug target discoveries that could be used to develop and design a treatment for pancreatic cancer.
Scientists investigating pancreatic cancer have identified new targets which, with further research, could be the basis for developing future therapies. Listed below are five of the most recent target discoveries, in order of their journal publication dates, with the newest first.
Scientists at the Queen Mary University of London, UK and Zhengzhou University, China have developed a personalised vaccine system that may be able to delay the onset of pancreatic cancer.
Cells taken from mice, mutated chemically into pancreatic cancer cells and then infected with Adenovirus (AdV) as a prime or Vaccinia virus (VV) as a boost, create a vaccine product. The virus kills the cancerous cells in such a way that their antigens are released and are therefore able to prime the immune system to prevent pancreatic cancer returning.
Injection of the virus-infected cells into mice destined to develop pancreatic cancer doubled their survival rate, compared to their unvaccinated counterparts. The vaccine also delayed the onset of the condition in these mice.
Using cells from the recipient of the vaccine enables the immune system to respond to the exact antigens seen in tumour cells of the individual, resulting in a vaccine regime tailored to them.
Through this international collaboration, we have made progress towards the development of a prophylactic cancer vaccine against pancreatic cancer, said Professor Yaohe Wang, leader of the study, from Queen Mary University of London and the Sino-British Research Centre at Zhengzhou University in China.
Researchers at Sanford Burnham Prebys Medical Discovery Institute in the US have identified that a combination of two anti-cancer compounds, already approved for use to treat other cancers, shrank pancreatic tumours in mice.
Our study identifies a potential treatment combination that can immediately be tested against these aggressive tumours. We are already meeting with oncologists at Oregon Health & Science University, US to discuss how to advance this discovery into clinical evaluation, explained Dr Zeev Ronai, a professor in Sanford Burnham Prebys Tumor Initiation and Maintenance Program, also senior author of the study.
Scientists used L-asparaginase to starve pancreatic tumours of asparagine, an amino acid required by cells for protein synthesis. However, the tumour cells did not die, instead switching on a stress response pathway whereby they could produce asparagine themselves. Scientists then used an MEK inhibitor to block the stress response pathway, causing the pancreatic tumour to shrink.
L-asparaginase is already US Food and Drug Administration (FDA) approved to treat leukaemias and similarly the MEK inhibitor is approved for the treatment of solid tumours, including melanoma skin cancer.
This research lays the basis for the inhibition of pancreatic tumour growth by a combined synergistic attack based on asparagine restriction and MAPK signalling inhibition, says Dr Eytan Ruppin, chief of the Cancer Data Science Library at the National Cancer Institute (NCI) and co-author of the study.
Scientists from the Max Planck Institute for Biology of Ageing, Germany have identified that YME1L, a protease in the membrane of mitochondria, is activated when a cell uses glycolysis to produce energy anaerobically.
scientists were able to reduce tumour growth by switching off the glycolysis signalling pathway in the mitochondria
Cells adapt to oxygen deficiency by switching their energy supply to glycolysis, which ferments sugar without oxygen. This switch is often necessary in old age, as the cells in the body become poorly supplied with oxygen and nutrients.
Cancer cells can also face this problem; prior to angiogenesis, tumours are poorly perfused and so the tissue is deprived of oxygen. Oxidative stress in tumours drives the switch-on of multiple pathways. This includes the glycolysis pathway that alters the behaviour of the mitochondria to provide tumour cells with energy despite being starved of oxygen.
Scientists found that the YME1L protease is activated during the conversion to glycolysis. YME1L appears altered and breaks down various proteins in the organelles, preventing the formation of new mitochondria and causing the remaining organelles to change their metabolism. This process eventually stops as YME1L begins to degrade itself at high activity.
Researchers examined cancer cells originating from patients with pancreatic tumours and were able to reduce tumour growth by switching off the glycolysis signalling pathway in the mitochondria, with reproducible results both in the petri dish and in pancreatic tumours in mice.
There is currently no treatment available for pancreatic cancer. I believe that this protease can be a very interesting therapeutic target because we have seen that the signalling pathway is also active in human patients with pancreatic cancer, explained Thomas Langer, the Max Planck Director, continuing: However, there are no known substances that have an effect on this protease.
Researchers at the Crick Institute have identified cancer stem cells as a driver of pancreatic cancer growth. These cells can metastasise and differentiate into different tumour types to continue the spread of cancer.
Cancer stem cells appear at all stages of cancer growth so being able to identify where they are present could be vital in both targeting cancer and developing new treatments, according to the researchers. Analysis of gene expression in the cancer stem cells identified a protein, CD9, is present on tumour surfaces during development and when it is more established. This protein could therefore be used as a marker to help locate these cells.
A further development of the study established that this protein is not just a marker of cancer stem cells, but also promotes their malignant behaviour. By altering the amount of CD9 in tumour cells in mice, researchers found that reduced levels of this protein caused smaller tumours to form and increasing levels of CD9 created more aggressive cells able to form large tumours quickly.
These cells are vital to pancreatic cancer and if even just a few of them survive chemotherapy, the cancer is able to bounce back. We need to find effective ways to remove these cells and so stop them from fuelling cancer growth. However, we need more experiments to validate the importance of CD9 in human pancreatic cancer, says Victoria Wang, lead author and member of the Adult Stem Cell Laboratory at the Crick Institute.
A look into cancer stem cell metabolism also revealed CD9 increases the rate tumour cells take up glutamine, an amino acid which helps provide energy for cancer growth.
Now we know this protein is both linked to cancer stem cells and helps cancer growth, this could guide the development of new treatments that are targeted at the protein and so cut off the supply of glutamine to cancer stem cells, effectively starving the cancer, says Axel Behrens, corresponding author and group leader in the Adult Stem Cell Laboratory at the Crick Institute.
Scientists at Tel Aviv University, Israel have found that PJ34, a small molecule, causes human pancreatic cancer cells to self-destruct. The researchers tested PJ34 on xenografts (transplants) of human pancreatic tumours in mice.
this mechanism also exists in other types of cancer and therefore the treatment could be valuable for use on those resistant to current therapies
The mice were treated with a molecule called PJ34, which is permeable in the cell membrane but affects human cancer cells exclusively. This molecule causes an anomaly during the duplication of human cancer cells, provoking their rapid cell death. Thus, cell multiplication itself resulted in cell death in the treated cancer cells, explains Professor Malca Cohen-Armon, project lead at Tel Aviv Universitys Sackler Faculty of Medicine.
The treatment consisted of daily PJ34 injections for 14 days and four weeks later there was a relative drop of 90 percent in the number of cancer cells within the tumours of the mice. Cohen-Armon also noted there were no adverse side-effects observed in the mice.
This mechanism similarly exists in other types of cancer and therefore the treatment could be valuable for use on those resistant to current therapies. The molecule PJ34 is being tested in pre-clinical trials according to FDA regulations before clinical trials begin.
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Five recent drug target discoveries for pancreatic cancer - Drug Target Review
What its like to get laser hair removal as a black woman – Yahoo Lifestyle
By daniellenierenberg
Im hairythats just fact, and its something Ive always been aware of. Ill never forget being in summer camp as a kid, wearing shorts every day and becoming very aware that my legs were much hairier than those of the girls around me. Now, Im no longer ashamed of being hairyIve actually come to embrace it. But in the past two decades, Ive gotten very acquainted with epilators, including laser hair removal.
I got my first laser hair removal treatment two years ago. It seemed like everyone was getting it done, but as a black woman, I knew I wasnt everyone. For brown skin, laser hair removal isnt a spur-of-the-moment decision, a process that should be approached through a Groupon or the local nail salon (yes, some nail salons actually perform laser hair removal). For us, the process can be a bit more expensive and tedious, and should be approached with the utmost caution.
Why? Well, lasers target pigment, and due to the high content of melanin in our skin, risks of discoloration and hyper-pigmentation are astronomically higher. So thats why, according to Chris Karavolas, owner of Romeo And Juliette Laser Hair Removal, darker skin requires a completely different laser all together, and operators with much more experience. Darker skin complexions need to be careful because not all centers have the right lasers for dark skin, and even if they do they do, many do not have enough experience in treating dark skin, he says.
When it comes to those specialized lasers, there are two options. Its important to treat with an Nd:YAG laser, such as the Candela GentleYAG or GentleMax Pro, says Anne Chapas, M.D., medical director of Union Square Laser Dermatology in New York City. The wavelength of a YAG laser goes deeper into the skin than a diode laser and is less absorbed by the surrounding skin pigment, so it more successfully treats the stem cells of the hair follicle. Additionally, more treatments may be required than for fairer skin: Expect to receive at least six sessions, Chapas says.
Knowing all these things, I went into laser hair removal with cautious optimism. I chose to treat my Brazilian areayears of improper hair removal had left me with ingrown and severe discoloration, to the point that my wax lady refused to continue treating me because my skin had gotten so irritated. I was sure that I wanted to continue being hair-free down there, so I decided to get laser hair removal on my vagina area. I was nervous heading into my first appointment, so I made sure to ask the aesthetician at Romeo & Juliette in-depth questions about her experience with dark skin, as well as requesting to see photos of previous clients and inquiring about a patch test. Then, it was showtime.
I like to think that I have a pretty high pain tolerance. However, laser removal certainly made me question that belief. I wont lie and say that it didnt hurt. It did. It felt like being snapped, hard, by rubber bands, but it was quick, and the results I saw were almost immediate. I was advised to shave right before the treatment, in order to give the laser immediate access to the hair follicle, and because, well, nobody likes the smell of burning hair. After about 10 minutes, I was sent on my way and advised to come back in another six weeks. Laser hair removal requires a strict schedule and works in conjunction with the hair cycle for optimal results. Every appointment thereafter was just as easy, and now, completely through all six of my sessions, my hair is almost completely gone, with the occasional stubble appearing here and there.
So how do you know if your skin will need one of these specialized lasers? Different ethnicitiesregardless of skin tonereact differently to lasers. Your skin may appear to be a 3 or 4 on the Fitzpatrick scale, but if youre, say, Latin or Asian, it could react to the laser as a 6 would, Dr. Chapas says. So be sure that youre only going to facilities that have operators trained in dermatology, and are skilled enough to differentiate your skin tone.
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What its like to get laser hair removal as a black woman - Yahoo Lifestyle
Researchers Manage to Create Pig-Monkey Chimeras for the First Time – Tdnews
By daniellenierenberg
Researchers in China are laying claim to the first ever pig-monkey chimeras to be born in what they hope will be a breakthrough for biomedicine, not just fuel for your nightmares.
The worlds first monkey-pig hybrids have been born in a Chinese lab amid scientific attempts to grow human organs inside animals. The incredible experiment saw two chimera piglets born with DNA from both pigs and cynomolgus monkeys. However, the experiment was short-lived as the two piglets both died within a week.
According to the researchers, the baby piglets had genetic material from cynomolgus monkeys in their heart, liver, spleen, lung and skin.
However, the success rate was significantly low as only 10 piglets were born from more than 4,000 implanted in sows.
From those, only two were chimeras in a result that has been deemed discouraging by other leading stem cell scientists.
Lead researcher Tang Hai at the State Key Laboratory of Stem Cell and Reproductive Biology in Beijing said: This is the first report of full-term pig-monkey chimeras.
Ms Hai confirmed that it was unclear why the piglets died, but the non-chimeric pigs died as well.
The Chinese research team suspect that the deaths could have been due to the IVF process instead than the chimerism
According to research study, stem cells from macaque monkeys were grown in a lab and then injected into pig embryos five days after fertilisation.
Ms Hai said that the next step in their research was to create healthy animals with a higher proportion of monkey cells before creating pigs in which one organ is composed almost entirely of primate cells.
The ultimate research goal is to grow human organs inside live animals as a way to resolve the crisis of organ transplantation.
University of California stem cell biologist Paul Knoepfler said: Given the extremely low chimeric efficiency and the deaths of all the animals, I see this as fairly discouraging.
Reaction to the study has prompted many social media users to question if this is a step too far.
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Researchers Manage to Create Pig-Monkey Chimeras for the First Time - Tdnews
Cell Harvesting Market a compound annual growth rate (CAGR) of 11.3% for the period of 2018-2023 – Crypto News Byte
By daniellenierenberg
Theglobal market for cell harvestingshould grow from $885 million in 2018 to reach $1.5 billion by 2023 at a compound annual growth rate (CAGR) of 11.3% for the period of 2018-2023.
Report Scope:
The scope of the report encompasses the major types of cell harvesting that have been used and the cell harvesting technologies that are being developed by industry, government agencies and nonprofits. It analyzes current market status, examines drivers on future markets and presents forecasts of growth over the next five years.
The report provides a summary of the market, including a market snapshot and profiles of key players in the cell harvesting market. It provides an exhaustive segmentation analysis of the market with in-depth information about each segment. The overview section of the report provides a description of market trends and market dynamics, including drivers, restraints and opportunities. it provides information about market developments and future trends that can be useful for organizations, including wholesalers and exporters. It provides market positionings of key players using yardsticks of revenue, product portfolio, and recent activities. It further includes strategies adopted by emerging market players with strategic recommendations for new market entrants. Readers will also find historical and current market sizes and a discussion of the markets future potential. The report will help market players and new entrants make informed decisions about the production and exports of goods and services.
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Report Includes:
41 data tables and 22 additional tables Description of segments and dynamics of the cell harvesting market Analyses of global market trends with data from 2017, 2018, and projections of compound annual growth rates (CAGRs) through 2023 Characterization and quantification of market potential for cell harvesting by type of harvesting, procedure, end user, component/equipment and region A brief study and intact information about the market development, and future trends that can be useful for the organizations involved in Elaboration on the influence of government regulations, current technology, and the economic factors that will shape the future marketplace Key patents analysis and new product developments in cell harvesting market Detailed profiles of major companies of the industry, including Becton, Dickinson and Co., Corning, Inc., Fluidigm Corp., General Electric Co., Perkinelmer, Inc., and Thermo Fisher Scientific, Inc.
Summary
Stem cells are unspecialized cells that have the ability to divide indefinitely and produce specialized cells. The appropriate physiological and experimental conditions provided to the unspecialized cells give rise to certain specialized cells, including nerve cells, heart muscle cells and blood cells. Stem cells can divide and renew themselves over long periods of time. These cells are extensively found in multicellular organisms, wherein mammals, there are two types of stem cells embryonic stem cells and adult stemcells. Embryonic stem cells are derived from a human embryo four or five days old that is in the blastocyst phase of development. Adult stem cells grow after the development of the embryo and are found in tissues such as bone marrow, brain, blood vessels, blood, skin, skeletal muscles and liver. Stemcell culture is the process of harvesting the exosomes and molecules released by the stem cells for the development of therapeuticsfor chronic diseases such as cancer and diabetes. The process is widely used in biomedical applications such as therapy, diagnosis and biological drug production. The global cell harvesting market is likely to witness a growth rate of REDACTED during the forecast period of 2018-2023.The value of global cell harvesting market was REDACTED in 2017 and is projected to reach REDACTED by 2023. Market growth is attributed to factors such as increasing R&D spending in cell-based research,the introduction of 3D cell culture technology, increasing government funding, and the growing prevalence of chronic diseases such as cancer and diabetes.
The growing incidence and prevalence of cancer is seen as one of the major factors contributing to the growth of the global cell harvesting market. According to the World Health Organization (WHO), cancer is the second-leading cause of mortality globally and was responsible for an estimated 9.6 million deaths in 2018. Therefore, there is an increasing need for effective cancer treatment solutions globally. Cell harvesting is the preferred method used in cancer cell-related studies including cancer cell databases (cancer cell lines), and other analyses and drug discovery in a microenvironment. The rising prevalence of such chronic diseases has led governments to provide R&D funding to research institutes and biotechnology companies to develop advanced therapeutics. Various 3D cell culture technologies have been developed by researchers and biotechnology companies such as Lonza Group and Thermo Fischer Scientific for research applications such as cancer drug discovery. The application of cell culture in cancer research is leading to more predictive models for research, drug discovery and regenerative medicine applications.
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Platelet-rich plasma (PRP) therapy, a new biotechnology solution that has a heightened interest among researchers in tissue engineering and cell-based therapies, has various applications in the treatment of tissue healing in tendinopathy, osteoarthritis and muscle injury. It has been conventionally employed in orthopedics, maxillofacial surgery, periodontal therapy and sports medicines. PRP therapy can be used in the treatment of fat grafting, acne scars, and hair regrowth.
Major factors driving market growth include increasing healthcare costs and the high rate of adoption for modern medicines in emerging economies such as China and India. It has been estimated that India will witness a CAGR of REDACTED in the cell harvesting market during the forecast period. The active participation of foreign pharmaceutical companies has tapped the Indian healthcare sector with a series of partnerships and mergers and acquisitions, which in turn is positively impacting the growth of the market in this region. Consistent development and clinical trials for stem cell therapies, plus contribution from the government and private sectors through investments and cohesive reimbursement policies in the development of cancer biomarkers, is further fueling market growth. InSweden, a research team at Lund University has developed a device to collect fluid and harvest stem mesenchymal stem cells (MSCs). The device is developed with 3D-printed bio-inert plastics which, when used by doctors, can result in the safe extraction of fluids (medical waste) from the patients body. The liquid is then passed through a gauze filter for purifying thoroughly and MSCs are separated from the fluid by centrifugation and are grown in culture.
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Cell Harvesting Market a compound annual growth rate (CAGR) of 11.3% for the period of 2018-2023 - Crypto News Byte
Syndax Pharmaceuticals Announces Plans to Commence Phase 2 Expansion Cohort of SNDX-6352 for the Treatment of Chronic Graft Versus Host Disease -…
By daniellenierenberg
WALTHAM, Mass., Dec. 10, 2019 /PRNewswire/ --Syndax Pharmaceuticals, Inc. ("Syndax," the "Company" or "we") (Nasdaq: SNDX), a clinical stage biopharmaceutical company developing an innovative pipeline of cancer therapies, today announced that it plans to commence a Phase 2 expansion cohort based on encouraging clinical activity and a well-tolerated safety profile observed to date in the ongoing Phase 1 dose escalation trial of SNDX-6352 in patients with chronic graft versus host disease (cGVHD). SNDX-6352 is the Company's anti-CSF-1R monoclonal antibody.
The ongoing Phase 1, open-label, modified 3+3 dose escalation trial is designed to evaluate the safety and preliminary efficacy of SNDX-6352 in up to 30 patients with cGVHD who have received at least two prior lines of therapy. As of a November 25, 2019 data cutoff date, a total of five patients, all of whom received prior treatment with ibrutinib, steroids, and a calcineurin inhibitor, have been enrolled across three dose cohorts: one patient was treated at 0.15 mg/kg every two weeks (Q2W, Cohort 1), one is receiving a dose of 0.5 mg/kg Q2W (Cohort 2), and three patients are receiving 1.0 mg/kg Q2W (Cohort 3).
Responses have been observed in all evaluable patients as of the data cutoff date, with no dose limiting toxicities (DLTs) reported. Among the three patients dosed in Cohort 3 (1.0 mg/kg Q2W), one patient recently cleared the DLT period and has not yet been evaluated for efficacy, two patients experienced a partial response, and all three patients remain on therapy. The patient in Cohort 2 experienced a partial response and is currently in their ninth month of treatment with SNDX-6352 following prior treatment with ibrutinib and both Jakafi (ruxolitinib) and KD025, two agents currently being investigated for the treatment of cGVHD. The first patient (Cohort 1) achieved a partial response but discontinued in their third cycle due to elevated LFTs attributed to progression in their liver cGVHD. Cohort 4, which will explore a 3.0 mg/kg Q2W dose, is now open for enrollment.
"The initial results from our Phase 1 trial underscore the potential of SNDX-6352 to serve as an effective therapy for patients with cGVHD who are lacking alternative options," said Briggs W. Morrison, M.D., Chief Executive Officer of Syndax. "We had not anticipated commenting on data from this initial trial until the second half of 2020, so it is quite encouraging to see the early signs of activity in patients with this difficult to treat disease. Based on these results, we have decided to advance into a Phase 2 expansion cohort to evaluate additional patients at the 1.0 mg/kg dose while we continue the dose escalation to 3.0 mg/kg. We continue to expect to present the Phase 1 trial results in the second half of 2020.
"Published preclinical data have demonstrated that CSF-1R blockade can prevent and treat disease in animal models of cGVHD1," said Peter Ordentlich, Ph.D., Chief Scientific Officer and Co-founder of Syndax."The initial data from our trial provide the first clinical evidence that targeting CSF-1R dependent macrophages may benefit patients with cGVHD."
To date, SNDX-6352 has been safe and well-tolerated, with no DLTs observed. Dose escalation is ongoing in the Phase 1 portion of the trial. The Phase 2 expansion cohort is expected to enroll up to 22 patients to further characterize the safety and efficacy at an initial dosing schedule of 1.0 mg/kg of SNDX-6352 administered every two weeks.
About Chronic Graft Versus Host Disease
Chronic graft versus host disease (cGVHD), an immune response of the donor-derived hematopoietic cells against recipient tissues, is a serious, potentially life-threatening complication of allogeneic hematopoietic stem cell transplantation (HSCT) which can last for years. cGVHD is estimated to develop in approximately 40% of transplant recipients, and affect approximately 14,000 patients in the US. 2-4 cGVHD typically manifests across multiple organ systems, with the skin and mucosa being commonly involved, and is characterized by the development of fibrotic tissue.
About SNDX-6352
SNDX-6352 is an investigational monoclonal antibody that targets colony stimulating factor-1 receptor, or CSF-1R, a cell surface protein thought to control the survival and function of monocytes and macrophages. In pre-clinical models, inhibition of signaling through the CSF-1 receptor has been shown to reduce the number of disease-mediating macrophages and the development of cutaneous and pulmonary chronic graft versus host disease (cGVHD), as well as to lead to the depletion of cells known as Tumor Associated Macrophages, or TAMS. SNDX-6352 is currently being evaluated in a Phase 1 multiple ascending dose clinical trial in cGVHD, and a Phase 1 multiple ascending dose clinical trial as monotherapy and in combination with Infinzi (durvalumab) in solid tumors. SNDX-6352 has the potential to treat a variety of solid tumor and immune-related diseases.
About Syndax Pharmaceuticals, Inc.
Syndax Pharmaceuticalsis a clinical stage biopharmaceutical company developing an innovative pipeline of cancer therapies. The Company's lead product candidate, entinostat, a once-weekly, oral, small molecule, class I HDAC inhibitor, is being tested in a Phase 3 combination trial with exemestane for treatment of advanced HR+, HER2- breast cancer and has been evaluated in combination with several approved PD-1/PD-(L)1 antagonists. The Company's pipeline also includes SNDX-6352, a monoclonal antibody that blocks the colony stimulating factor 1 (CSF-1) receptor, and SNDX-5613, a highly selective inhibitor of the MeninMLL binding interaction. For more information, please visitwww.syndax.comor follow the Company on TwitterandLinkedIn.
Syndax's Cautionary Note on Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as "may," "will," "expect," "plan," "anticipate," "estimate," "intend," "believe" and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are intended to identify forward-looking statements. These forward-looking statements are based on Syndax's expectations and assumptions as of the date of this press release. Each of these forward-looking statements involves risks and uncertainties. Actual results may differ materially from these forward-looking statements. Forward-looking statements contained in this press release include, but are not limited to, statements about the progress, timing, clinical development and scope of clinical trials and the reporting of clinical data for Syndax's product candidates, and the potential use of our product candidates to treat various cancer indications. Many factors may cause differences between current expectations and actual results including unexpected safety or efficacy data observed during preclinical or clinical trials, clinical trial site activation or enrollment rates that are lower than expected, changes in expected or existing competition, changes in the regulatory environment, failure of Syndax's collaborators to support or advance collaborations or product candidates and unexpected litigation or other disputes. Other factors that may cause Syndax's actual results to differ from those expressed or implied in the forward-looking statements in this press release are discussed in Syndax's filings with the U.S. Securities and Exchange Commission, including the "Risk Factors" sections contained therein. Except as required by law, Syndax assumes no obligation to update any forward-looking statements contained herein to reflect any change in expectations, even as new information becomes available.
References
1. Alexander, KA. et al. J Clin Invest. 2014;124(10):42664280.
2.Kantar GVHD Expert Interviews N=8 interviews
3. SmartAnalyst 2017 SmartImmunology Insights chronic GVHD report.
4. Bachier, CR. et al. ASH annual meeting 2019; abstract #2109 Epidemiology and Real-World Treatment of Chronic Graft-Versus-Host Disease Post Allogeneic Hematopoietic Cell Transplantation: A U.S. Claims Analysis.
Syndax Contacts
Investor ContactMelissa ForstArgot Partnersmelissa@argotpartners.com212.600.1902
Media ContactCraig HeitGCI Healthcraig.Heit@gcihealth.com347.451.4733
SNDX-G
SOURCE Syndax Pharmaceuticals, Inc.
Pig-Monkey Chimeras Have Been Brought to Term For The First Time – ScienceAlert
By daniellenierenberg
Pigs engineered to have a small amount of monkey cells have been brought to full term and were even born alive, surviving for a few days after birth. Although the piglets died, it is claimed the experiment - performed in China - marks a major milestone for the future of lab-grown organs.
"This is the first report of full-term pig-monkey chimeras," Tang Hai of the State Key Laboratory of Stem Cell and Reproductive Biology in Beijing told New Scientist.
The research is part of an ongoing effort to develop animals - whether they are sheep or pigs - that can grow human organs we could then harvest for transplants, a process called xenogeneic organogenesis.
Research has been done on both pig and sheep embryos with transplanted human stem cells; in both cases, the embryos continued to develop until the experiment was deliberately terminated.
That's because, due to ethical concerns, these chimeras - organisms that incorporate the genetic material of another species - cannot be cultivated or studied in the later stages of embryonic development. Some scientists worry that some of the human stem cellscould end upin other parts of the animal or even in its brain, with unintended consequences.
For that reason, in this experiment the team used stem cells from crab-eating macaques (Macaca fascicularis). These were imbued with fluorescent proteins so that they would glow under fluorescent light, and derived to produce fluorescing embryonic cells.
These cells were then injected into over 4,000 five-day-old pig embryos fertilised using IVF; the modified pig embryos were subsequently implanted into sows.
This fiddly and painstaking work produced just 10 piglets that made it to full term and were born alive. And only two of these were chimeric, with between one in 1,000 and one in 10,000 functional monkey cells to pig cells.
The monkey cells had migrated to the heart, liver, lungs, spleen and skin of the piglet hosts, but were not found in other organs, such as testes and ovaries, due to the low rate of chimerism, the researchers said.
Sadly, before a week was out, the piglets died - not just the two chimeras, but the other eight normal piglets, too. Because all the pigs died, Hai told New Scientist, the cause of death likely had less to do with chimerism, and more to do with IVF - a procedure that is notoriously tricky in pigs.
The low chimerism rate is also somewhat discouraging. However, the researchers remain optimistic. Although the birth rate was low, and the pigs didn't survive, the team now has a wealth of data they can apply to future experiments.
The scientists are planning to try again, increasing the chimeric cell ratio. And they believe their data may help other scientists working in the field.
"Here, we have used monkey cells to explore the potential of reconstructing chimeric human organs in a large animal model," they wrote in their paper.
"We believe this work will facilitate the development of xenogeneic organogenesis by providing a better understanding of the processes of xenogeneic recognition, fate determination, and the proliferation and differentiation of primate stem cells during porcine development.
"The findings could pave the way toward overcoming the obstacles in the re-engineering of heterogeneous organs and achieve the ultimate goal of human organ reconstruction in a large animal."
The research has been published in Protein & Cell.
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Pig-Monkey Chimeras Have Been Brought to Term For The First Time - ScienceAlert
Top Florida Medical Spa, Amnion of Florida, Partners With Merakris Therapeutics to Advance Their Non-Surgical Treatment Options – Business Wire
By daniellenierenberg
ORLANDO, Fla.--(BUSINESS WIRE)--Amnion of Florida, a leading provider of alternative medicine utilizing cryopreserved placental cell allograft and advanced bioactive facial rejuvenation, is pleased to announce their vendor choice to round out regenerative anti-aging therapies.
Amnion announces a partnership with Merakris Therapeutics, LLC to advance the development of Merakris topical bioactive anti-aging hydrogel technology. Christopher Broderick, President, and Founder of Merakris Therapeutics stated, dedication to science-based outcomes is our primary focus, thus were delighted to be selected based upon our scientific approach to youth maintenance and rejuvenation technologies.
Amnion is focused on attracting women and men seeking affordable non-surgical options for youth maintenance via cell activated procedures, hair restoration, joint repair, dermal rejuvenation, and anti-aging treatments.
Our team of experienced medical professionals and aestheticians at Amnion are excited to utilize the Merakris Therapeutics product suite, including medical-grade, sterile filtered amniotic fluid serums and hydrogels at our newest Spa in Sanford, FL, said Eusebio Coterillo, President of Amnion.
In a constantly changing field, Amnion of Florida, under the guidance of the on-site medical staff, provides the highest level of quality products and procedures in cosmetic medicine. They offer cutting edge treatments that are proven by research, the use FDA cleared or registered products, and are widely published and peer endorsed.
More about Amnion of Florida
Amnion of Florida, based in Central Florida, is a leading provider of alternative medicine using cryopreserved placental cell transplants or allografts, processed from donated cellular birth tissue, which are natural alternatives to autologous regenerative medicine products. The primary function of our allogeneic regenerative treatments is to promote soft tissue joint/skin repair and regeneration mediated by growth factors and cells naturally found in placental tissue. These treatments have shown safety and efficacy in treating a variety of ailments including osteoarthritis, chronic ulcerative wounds, joint pain, skin rejuvenation, hair restoration, urinary incontinence, and ED. Learn more http://www.amnion.us.
More about Merakris Therapeutics, LLC
Merakris Therapeutics, based in Research Triangle Park, North Carolina, is focused on researching, developing, and marketing regenerative healthcare products. Merakris is pioneering commercially scalable biotherapeutic technologies derived from stem cells that have various clinical applications. Our vision is to improve global patient care and outcomes through the pioneering and innovation of acellular regenerative biotechnologies. Learn more at http://www.merakris.com.
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Top Florida Medical Spa, Amnion of Florida, Partners With Merakris Therapeutics to Advance Their Non-Surgical Treatment Options - Business Wire
Meet the world’s first pig-monkey hybrid – Free Press Journal
By daniellenierenberg
Beijing: In a world first, researchers have been able to produce pig-monkey hybrids in a Chinese laboratory, a media report said. Two piglets - having monkey tissues in their hearts, liver and skin - were born in the State Key Laboratory of Stem Cell and Reproductive Biology in Beijing but died within a week, the Mirror reported.
The announcement to this effect was made by Tang Hai. The experiment comes in the wake of Spanish scientist Juan Carlos Izpisua Belmonte's attempt to create pig-human hybrids two years ago. Tang and his team injected genetically modified monkey cells into more than 4,000 pig embryos before these were implanted into sows, the New Scientist magazine reported.
Only two of the 10 piglets thus born were hybrids - with tissues in the heart, liver, spleen, lung and skin, partly consisting of monkey cells. Experts suspected that the failure has to do with the IVF process.
Critics have now warned that the scientific development will create "disturbing" dilemmas over the ethics of human-animal hybrids. Doctor Angel Raya, director of the Barcelona Regenerative Medicine Centre, told the Spanish daily El Pais: "What happens if the stem cells escape and form human neurons in the brain of the animal? Would it have consciousness? And what happens if these stem cells turn into sperm cells?''
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Meet the world's first pig-monkey hybrid - Free Press Journal
Alpine Immune Sciences Presents ALPN-101 Phase 1 Healthy Volunteer Study Data and Details of Upcoming Phase I/II BALANCE GVHD Study at the 61st…
By daniellenierenberg
SEATTLE--(BUSINESS WIRE)--Alpine Immune Sciences, Inc. (NASDAQ:ALPN), a leading clinical-stage immunotherapy company focused on developing innovative treatments for cancer and autoimmune/inflammatory diseases, presented Phase 1 data yesterday from the healthy volunteer study of ALPN-101, a first-in-class dual CD28/ICOS antagonist, and details on its upcoming Phase 1/2 BALANCE study of ALPN-101 in steroid-resistant or steroid-refractory acute graft-versus-host disease (GVHD) at the 61st American Society of Hematology Annual Meeting (ASH) in Orlando, FL.
Jan Hillson, MD, Senior Vice President of Clinical Development at Alpine, presented An Open Label Study of ALPN-101, a First-in-Class Dual CD28/ICOS Antagonist, in Subjects with Steroid-Resistant or Steroid-Refractory Acute Graft Versus Host Disease (BALANCE) as part of the oral session, Chemical Biology and Experimental Therapeutics: Novel Compounds and Mechanisms of Action.
Highlights included:
Despite decades of intense research, GVHD remains a major cause of morbidity and mortality after hematopoietic stem cell transplantation, commented Sophie Paczesny, MD, PhD, Professor of Immunology and Pediatrics at Indiana University School of Medicine and lead of the Biomarkers Stem Cell Transplantation Program and one of Alpine's research collaborators. Current therapies are associated with significant toxicities or are simply insufficient to control the disease. CD28 and ICOS appear to be key pathways in the pathogenesis of GVHD, and the presented data with ALPN-101 appear uniquely strong. I look forward to the BALANCE study, which may demonstrate the therapeutic potential of ALPN-101.
About Graft Versus Host Disease (GVHD)
Graft versus host disease (GVHD) is the most common life-threatening complication of a hematopoietic cell transplant. It occurs when donor cells see recipient cells as foreign and attack them. Acute GVHD typically occurs within the early weeks and months after transplant, usually involving the skin, liver, and gastrointestinal tract. GVHD patients remain at risk of organ system damage and increased mortality due to the disease and to high dose glucocorticoids.
About ALPN-101
ALPN-101 is a novel Fc fusion protein of a human inducible T cell costimulator ligand (ICOSL) variant immunoglobulin domain (vIgD), and a first-in-class therapeutic designed to inhibit simultaneously the CD28 and ICOS inflammation pathways. CD28 and ICOS are closely-related costimulatory molecules with partially overlapping roles in T cell activation likely playing a role in multiple autoimmune and inflammatory diseases. In preclinical models of graft versus host disease, inflammatory arthritis, connective tissue disease, and multiple sclerosis, ALPN-101 demonstrates efficacy superior to agents blocking the CD28 CD80/86 or ICOS - ICOSL pathways alone.
About Alpine Immune Sciences, Inc.
Alpine Immune Sciences, Inc. is committed to leading a new wave of immune therapeutics, creating potentially powerful multifunctional immunotherapies to improve patients lives via unique protein engineering technologies. Alpine has two lead programs. The first, ALPN-101 for autoimmune/inflammatory diseases, is a selective dual T cell costimulation blocker engineered to reduce pathogenic T and B cell immune responses by blocking ICOS and CD28. ALPN-101 has recently completed enrollment in a Phase 1 healthy volunteer trial. The second, ALPN-202 for cancer, is a conditional CD28 costimulator and dual checkpoint inhibitor. Alpine is backed by world-class research and development capabilities, a highly-productive scientific platform, and a proven management team. For more information, visit http://www.alpineimmunesciences.com.
Forward-Looking Statements
This release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, Section 21E of the Securities Exchange Act of 1934 and the Private Securities Litigation Reform Act of 1995. These forward-looking statements are not based on historical fact and include statements regarding our platform technology and potential therapies, the timing of and results from clinical trials and pre-clinical development activities, clinical and regulatory objectives and the timing thereof, expectations regarding the sufficiency of cash to fund operations, the potential efficacy, safety profile, future development plans, addressable market, regulatory success, and commercial potential of our product candidates, the timing of our public presentations and potential publication of future clinical data, the efficacy of our clinical trial designs, expectations regarding our ongoing collaborations, and our ability to successfully develop and achieve milestones in our development programs. Forward-looking statements generally include statements that are predictive in nature and depend upon or refer to future events or conditions and include words such as may, will, should, would, expect, plan, intend, and other similar expressions, among others. These forward-looking statements are based on current assumptions that involve risks, uncertainties, and other factors that may cause actual results, events, or developments to be materially different from those expressed or implied by such forward-looking statements. These risks and uncertainties, many of which are beyond our control, include, but are not limited to: clinical trials may not demonstrate safety and efficacy of any of our product candidates; our ongoing discovery and pre-clinical efforts may not yield additional product candidates; our discovery-stage and pre-clinical programs may not advance into the clinic or result in approved products; any of our product candidates may fail in development, may not receive required regulatory approvals, or may be delayed to a point where they are not commercially viable; we may not achieve additional milestones in our proprietary or partnered programs; the impact of competition; adverse conditions in the general domestic and global economic markets; as well as the other risks identified in our filings with the Securities and Exchange Commission. These forward-looking statements speak only as of the date hereof and we undertake no obligation to update forward-looking statements, and readers are cautioned not to place undue reliance on such forward-looking statements.
Secreted Immunomodulatory Proteins, SIP, Transmembrane Immunomodulatory Protein, TIP, Variant Ig Domain, vIgD and the Alpine logo are registered trademarks or trademarks of Alpine Immune Sciences, Inc. in various jurisdictions.
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Alpine Immune Sciences Presents ALPN-101 Phase 1 Healthy Volunteer Study Data and Details of Upcoming Phase I/II BALANCE GVHD Study at the 61st...
Harvard geneticist George Church’s goal: to protect humans from viruses, genetic diseases, and aging – 60 Minutes – CBS News
By daniellenierenberg
Our lives have been transformed by the information age. But what's coming next is likely to be more profound, call it the genetic information age. We have mapped the human genome and in just the last few years we have learned to read and write dna like software. And you're about to see a few breakthroughs-in-waiting that would transform human health. For a preview of this revolution in evolution we met George Church, a world leading geneticist, whose own DNA harbors many eccentricities and a few genes for genius.
We found George Church in here.Cory Smith: Most of these are frozen George. Little bits of George that we have edited all in different tubes.
Church threw himself into his work, literally. His DNA is in many of the experiments in his lab at Harvard Medical School. The fully assembled George Church is 6'5" and 65. He helped pioneer mapping the human genome and editing DNA. Today, his lab is working to make humans immune to all viruses, eliminate genetic diseases, and reverse the effects of time.
Scott Pelley: One of the things your lab is working on is reversing aging.
George Church: That's right.
Scott Pelley: How is that possible?
George Church: Reversing aging is one of these things that is easy to dismiss to say either we don't need it or is impossible or both.
Scott Pelley: Oh, we need it.
George Church: Okay. We need it. That's good. We can agree on that. Well, aging reversal is something that's been proven about eight different ways in animals where you can get, you know, faster reaction times or, you know, cognitive or repair of damaged tissues.
Scott Pelley: Proven eight different ways. Why isn't this available?
George Church: It is available to mice.
In lucky mice, Church's lab added multiple genes that improved heart and kidney function and levels of blood sugar. Now he's trying it in spaniels.
Scott Pelley: So is this gene editing to achieve age reversal?
George Church: This is adding genes. So, it's not really editing genes. It's, the gene function is going down, and so we're boosting it back up by putting in extra copies of the genes.
Scott Pelley: What's the time horizon on age reversal in humans?
George Church: That's in clinical trials right now in dogs. And so, that veterinary product might be a couple years away and then that takes another ten years to get through the human clinical trials.
Human trials of a personal kind made George Church an unlikely candidate to alter human evolution. Growing up in Florida, Church was dyslexic, with attention deficit, and frequently knocked out by narcolepsy.
Scott Pelley: What was it that made you imagine that you could be a scientist?
George Church: The thing that got me hooked was probably the New York World's Fair in 1964. I thought this is the way we should all be living. When I went back to Florida, I said, "I've been robbed," you know? "Where is it all?" So, I said, "Well, if they're not going to provide it, then I'm gonna provide it for myself."
With work and repetition, he beat his disabilities and developed a genius for crystallography, a daunting technique that renders 3D images of molecules through X-rays and math. But in graduate school at Duke, at the age of 20, his mania for the basic structures of life didn't leave time for the basic structure of life.
Scott Pelley: You were homeless for a time.
George Church: Yeah. Briefly.
Scott Pelley: Six months.
George Church: Six months.
Scott Pelley: And where were you sleeping when you were homeless?
George Church: Well, yeah. I wasn't sleeping that much. I was mostly working. I'm narcoleptic. So, I fall asleep sitting up anyway.
His devotion to crystallography was his undoing at Duke.
George Church: I was extremely excited about the research I was doing. And so, I would put in 100-plus hours a week on research and then pretty much didn't do anything else.
Scott Pelley: Not go to class.
George Church: I wouldn't go to class. Yeah.
Duke kicked him out with this letter wishing him well in a field other than biology. But, it turned out, Harvard needed a crystallographer. George Church has been here nearly 40 years. He employs around 100 scientists, about half-and-half men and women.
Scott Pelley: Who do you hire?
George Church: I hire people that are self-selecting, they see our beacon from a distance away. There are a lot of people that are a little, you know, might be considered a little odd. "Neuroatypicals," some of us are called.
Scott Pelley: "Neuroatypical?"
George Church: Right.
Scott Pelley: Unusual brains?
George Church: Right, yeah.
Parastoo Khoshakhlagh: One thing about George that is very significant is that he sees what you can't even see in yourself.
Parastoo Khoshakhlagh and Alex Ng are among the "neuroatypicals." They're engineering human organ tissue.
Cory Smith: I think he tries to promote no fear of failure. The only fear is not to try at all.
Cory Smith's project sped up DNA editing from altering three genes at a time to 13,000 at a time. Eriona Hysolli went to Siberia with Church to extract DNA from the bones of wooly mammoths. She's editing the genes into elephant DNA to bring the mammoth back from extinction.
Eriona Hysolli: We are laying the foundations, perhaps, of de-extinction projects to come.
Scott Pelley: De-extinction.
Eriona Hysolli: Yes.
Scott Pelley: I'm not sure that's a word in the dictionary yet.
Eriona Hysolli: Well, if it isn't, it should be.
Scott Pelley: You know there are people watching this interview who think that is playing God.
George Church: Well, it's playing engineer. I mean, humans have been playing engineer since the dawn of time.
Scott Pelley: The point is, some people believe that you're mucking about in things that shouldn't be disturbed.
George Church: I completely agree that we need to be very cautious. And the more powerful, or the more rapidly-moving the technology, the more cautious we need to be, the bigger the conversation involving lots of different disciplines, religion, ethics, government, art, and so forth. And to see what it's unintended consequences might be.
Church anticipates consequences with a full time ethicist in the lab and he spends a good deal of time thinking about genetic equity. Believing that genetic technology must be available to all, not just those who can afford it.
We saw one of those technologies in the hands of Alex Ng and Parastoo Khoshakhlagh. They showed us what they call "mini-brains," tiny dots with millions of cells each. They've proven that cells from a patient can be grown into any organ tissue, in a matter of days, so drugs can be tested on that patient's unique genome.
Scott Pelley: You said that you got these cells from George's skin? How does that work?
Alex Ng: We have a way to reprogram essentially, skin cells, back into a stem cell state. And we have technologies where now we can differentiate them into tissue such as brain tissue
Scott Pelley: So you went from George's skin cells, turned those into stem cells, and turned those into brain cells.
Alex Ng: Exactly. Exactly.
Scott Pelley: Simple as that.
Organs grown from a patient's own cells would eliminate the problem of rejection. Their goal is to prove the concept by growing full sized organs from Church's DNA.
George Church: It's considered more ethical for students to do experiments on their boss than vice versa and it's good to do it on me rather than some stranger because I'm as up to speed as you can be on the on the risks and the benefits. I'm properly consented. And I'm unlikely to change my mind.
Alex Ng: We have a joke in the lab, I mean, at some point, soon probably, we're going to have more of his cells outside of his body than he has himself.
Church's DNA is also used in experiments designed to make humans immune to all viruses.
George Church: We have a strategy by which we can make any cell or any organism resistant to all viruses by changing the genetic code. So if you change that code enough you now get something that is resistant to all viruses including viruses you never characterized before.
Scott Pelley: Because the viruses don't recognize it anymore?
George Church: They expect a certain code provided by the host that they replicate in. the virus would have to change so many parts of its DNA or RNA so that it can't change them all at once. So, it's not only dead. But it can't mutate to a new place where it could survive in a new host.
Yes, he's talking about the cure for the common cold and the end of waiting for organ transplants. It's long been known that pig organs could function in humans. Pig heart valves are routinely transplanted already. But pig viruses have kept surgeons from transplanting whole organs. Church's lab altered pig DNA and knocked out 62 pig viruses.
Scott Pelley: What organs might be transplanted from a pig to a human?
George Church: Heart, lung, kidney, liver, intestines, various parts of the eye, skin. All these things
Scott Pelley: What's the time horizon on transplanting pig organs into human beings?
George Church: you know, two to five years to get into clinical trials. And then again it could take ten years to get through the clinical trials.
Church is a role model for the next generation. He has co-founded more than 35 startups. Recently, investors put $100 million into the pig organ work. Another Church startup is a dating app that compares DNA and screens out matches that would result in a child with an inherited disease.
George Church: You wouldn't find out who you're not compatible with. You'll just find out who you are compatible with.
Scott Pelley: You're suggesting that if everyone has their genome sequenced and the correct matches are made, that all of these diseases could be eliminated?
George Church: Right. It's 7,000 diseases. It's about 5% of the population. It's about a trillion dollars a year, worldwide.
Church sees one of his own genetic differences as an advantage. Narcolepsy lulls him several times a day. But he wakes, still in the conversation, often, discovering inspiration in his twilight zone.
Scott Pelley: If somebody had sequenced your genome some years ago, you might not have made the grade in some way.
George Church: I mean, that's true. I would hope that society sees the benefit of diversity not just ancestral diversity, but in our abilities. There's no perfect person.
Despite imperfection, Church has co-authored 527 scientific papers and holds more than 50 patents. Proof that great minds do not think alike.
The best science can tell, it was about 4 billion years ago that self-replicating molecules set off the spark of biology. Now, humans hold the tools of evolution, but George Church remains in awe of the original mystery: how chemistry became life.
Scott Pelley: Is the most amazing thing about life, then, that it happened at all?
George Church: It is amazing in our current state of ignorance. We don't even know if it ever happened ever in the rest of the universe. it's awe-inspiring to know that it either happened billions of times, or it never happened. Both of those are mind boggling. It's amazing that you can have such complex structures that make copies of themselves. But it's very hard to do that with machines that we've built. So, we're engineers. But we're rather poor engineers compared to the pseudo engineering that is biological evolution.
Produced by Henry Schuster. Associate producer, Rachael Morehouse. Broadcast associate, Ian Flickinger.
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Harvard geneticist George Church's goal: to protect humans from viruses, genetic diseases, and aging - 60 Minutes - CBS News
Get The Gloss Beauty & Wellness Awards 2019: The winners & commended – Get The Gloss
By daniellenierenberg
Each day until Christmas we'll be announcing an award winner - think of it as a virtual advent calendar. Today its the turn of Best Hair Tool - brushes at the ready
Wednesday 27 November saw some of the biggest names in beauty and wellness gather at Get The Gloss' HQ with the sole purpose of whittling down the 450 awards entries we received.
Our judges blazed through the day swatching, spritzing and swirling, putting the products through their paces, and after hours spent poring over the latest and greatest beauty releases, our panel of esteemed judges narrowed down over 450 beauty products to a shortlist and a selection of worthy winners. From cult names to little-known brands, your beauty wish list is about to get a whole lot longer.
Check back here and on our Instagram @getthegloss each day to discover our very worthy winners...
Winner: Manta x Zoe Irwin, 25
More than just a hair brush, this ergonomically shaped tool feels like a mini head massage every time you use it - our judges loved the way it stimulated the scalp in ways that other handheld brushes dont.
Commended: Hair story Brush, 11
To be used in conjunction with our Best Health Hair Saviour winner New Wash, this helps rinse hair product thoroughly, and promotes blood circulation for less hair loss, as well as effectively removing dandruff and protecting the scalp from scratchy fingernails.
Winner: ZENii Stem Cell Renewal Day Cream SPF 30, 70
As opposed to a pure SPF product this is moisturiser combined with a high SPF; its comfortable enough to wear all day, with editorial director Victoria Woodhall saying: It feels lovely, quite amazing and very soft.
While it may be expensive, the hefty price tag comes backed up with powerful ingredients including citrus stem cells and hyaluronic acid.
Commended: Altruist Sunscreen SPF50, 7.50 for 2x100ml
For such a fabulous price point, you really cant go wrong with this. Its lightweight with founder Sarah Vine commenting: Thats very nice, I would actually wear that - big words!
We like the story behind it too - the price point is so reasonable as it was launched by a dermatologist who wanted to make sunscreen accessible to all to reduce skin cancer diagnoses.
Winner: Code8 Highlight HD Palette, 24
For a touch of summer in the chilliest months, you cant go wrong with this two-toned palette, that offers the perfect amount of shimmer.
Commended: Beuti Skincare Pomegranate Glow 3in1 Enzyme Cleanser flash mask, 55.00
We love a multi-purpose product, so this was an instant hit around the judging table. The gel-balm hybrid can be used as a cleanser or a mask for a mini-facial, depending on what youre after.
Winner: WooWoo Tame it! Hair Removal Cream, 6.75
A far cry from the hair removal creams of yesteryear, this has none of the unpleasant scent you might remember and leaves skin soft and smooth. It almost makes hair removal cool - no mean feat!
Highly commended: MegsMenopause Blossom Balm, 10
Targeting vaginal dryness, this intimate skin moisturiser makes menopause a whole lot more comfortable - plus, the bottle is made from 96% recyclable materials.
Winner: Votary Rose Geranium and Apricot Cleansing Oil, 45
When Ateh Jewel says she uses a product every night, you sit up and listen. Something of a cult item in beauty circles, this cleansing oil goes on like velvet and leaves your skin feeling like silk.
Highly commended: Jane Scrivner Nourishing Cleanser, 46
With a creamy and delicious texture, this removes every scrap of makeup without being harsh on the skin.
Highly commended: PAI Light Work Rosehip Cleansing Oil, 36
As the name says, this cleansing oil is light as air, with the rosehip leaving a pleasing glow on the skin.
Winner: 001 Cryopress Ice Facial Massager, 75
After a full-on day of judging, this ice-cold facial massager was a breath of fresh air. Kept between -8 and 2.5 degrees Celcius, rolling this over your face increases blood circulation, tightening, firming and lifting the skin. Get The Gloss founder Susannah Taylor commented how amazing it made her skin feel, and if it's good enough for her...
Highly commended: Hayou Beauty Restorer, 38
This jade facial massage tool might not look like much, but it sure packs a punch. Simply sweep it over your face for a massage that not only improves circulation but can also soothe headaches and release tension. Our editorial director Victoria Woodhall commented that she loves it because it transforms your skin from the inside out.
Winner: Hairstory New Wash, 44
When a product is loved by Jonathan Van Ness, you just know it's going to be good - and our judges were definitely in agreement on this one. Darren Fowler commented that he loved the idea of a biodegradable cleanser that does everything in one - a product to use in place of shampoo which strips excess oil, dirt and styling products, but leaves behind all the goodness your hair needs to be happy and healthy.
Highly commended: L'Oreal Professionnel Vitamino Colour Shampoo, 12.90
Expert judges Cher Webb and Susannah Taylor both already use this in their day-to-day lives, so we were excited to give it a whirl - and we weren't disappointed by this trusted and well-known shampoo. Designed for coloured hair, it leaves locks shiny and vibrant, without weighing it down.
Winner: IT Cosmetics Superhero Mascara, 20
A stalwart in makeup artists' bags, Superhero Mascara boasts serious staying power, with our judges impressed by how a little goes a long way - serious bang for your buck with this one. Judge Cher Webb said she always carried it in her kit.
Highly commended: Lottie London Power Foil, 5.20
Launched in 2014, Lottie London is a relatively new player in the game. This eyeshadow wowed the panel thanks to the highly pigmented formula that creates a three-dimensional illusion on the eyes - Christmas party makeup isn't complete without it. Judge and hairstylist Darren Fowler said he'd love to see this on a model when he's on a shoot.
Watch this space tomorrow for another winner's announcement and follow us on Instagram @getthegloss for updates.
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Get The Gloss Beauty & Wellness Awards 2019: The winners & commended - Get The Gloss
Stem Cells – The Hastings Center
By daniellenierenberg
By Insoo Hyun
Stem cells are undifferentiated cells that have the capacity to renew themselves and to specialize into various cell types, such as blood, muscle, and nerve cells. Embryonic stem cells, derived from five-day-old embryos, eventually give rise to all the different cells and organ systems of the embryo. Embryonic stem cells are pluripotent, because they are capable of differentiating along each of the three germ layers of cells in the embryo, as well as producing the germ line (sperm and eggs). The three germ layers are the ectoderm (skin, nerves, brain), the mesoderm (bone, muscle), and the endoderm (lungs, digestive system).
During later stages of human development, minute quantities of more mature stem cells can be found in most tissue and organ systems, such as bone marrow, the skin, and the gut. These are somatic stem cells, responsible for renewing and repairing the bodys specialized cells. Although the lay public often refers to them as adult stem cells, researchers prefer to call them multipotent because they are less versatile than pluripotent stem cells, and because they are present from the fetal stage of development and beyond. Multipotent stem cells can only differentiate into cells related to the tissue or organ systems from which they originated for instance, multipotent blood stem cells in bonemarrow can develop into different types of blood cells, but not into nerve cells or heart cells.
While multipotent stem cell research has been around for nearly 50 years and has led to clinical therapies for leukemia and other blood disorders, the field of human embryonic stem cell research is still relatively new, and basic discoveries have yet to be directly transitioned into clinical treatments. Human embryonic stem cells were first isolated and maintained in culture in 1998 by James Thomson and colleagues at the University of Wisconsin. Since then, more than a thousand different isolateslines of self-renewing embryonic stem cellshave been created and shared by researchers worldwide.
The main ethical and policy issues with stem cells concern the derivation and use of embryonic stem cells for research. A vocal minority of Americans objects to the destruction of embryos that occurs when stem cells are derived. Embryonic stem cell research is especially controversial for those who believe that five-day-old preimplantation human embryos should not be destroyed no matter how valuable the research may be for society.
To bypass this ethical controversy, the Presidents Council on Bioethics recommended in 2005 that alternative sources of pluripotent stem cells be pursued. Some alternatives have been developed, most notably, the induced pluripotent stem (iPS) cells human skin cells and other body cells reprogrammed to behave like embryonic cells. But embryonic stem cell research will remain needed because there are some questions only they have the potential to answer.
Embryonic stem cells are necessary for several aims of scientific and biomedical research. They include addressing fundamental questions in developmental biology, such as how primitive cells differentiate into more specialized cells and how different organ systems first come into being. By increasing our knowledge of human development, embryonic stem cells may also help us better understand the causes of fetal deformations.
Other important applications lie in the areas of disease research and targeted drug development. By deriving and studying embryonic or other pluripotent stem cells that are genetically-matched to diseases such as Parkinsons disease and juvenile diabetes, researchers are able to map out the developmental course of complex medical conditions to understand how, when, and why diseased specialized cells fail to function properly in patients. Such disease-in-a-dish model systems provide researchers with a powerful new way to study genetic diseases. Furthermore, researchers can aggressively test the safety and efficacy of new, targeted drug interventions on tissue cultures of living human cells derived from disease-specific embryonic stem cells. This method of testing can reduce the risks associated with human subjects research.
One possible way of deriving disease-specific stem cells is through a technique called somatic cell nuclear transfer (SCNT), otherwise known as research cloning. By replacing the DNA of an unfertilized egg with the DNA of a cell from a patients body, researchers are able to produce embryonic stem cells that are genetically-matched to the patient and his or her particular disease. SCNT, however, is technically challenging and requires the collection of high-quality human eggs from female research volunteers, who must be asked to undergo physically burdensome procedures to extract eggs.
A much more widespread and simpler technique for creating disease-specific stem cells was pioneered in 2006 by Shinya Yamanaka and colleagues in Kyoto, Japan. They took mouse skin cells and used retroviruses to insert four genes into them to to create iPS cells. In 2007, teams led by Yamanaka, James Thomson, and George Daley each used similar techniques to create human iPS cells. The iPS cell approach is promising because disease-specific stem cells could be created using skin or blood samples from patients and because, unlike SCNT, it does not require the procurement of human eggs for research.
However, despite these advances, scientists do not believe iPS cells can replace human embryonic stem cells in research. For one, embryonic stem cells must be used as controls to assess the behavior and full scientific potential of iPS cells. Furthermore, iPS cells may not be able to answer some important questions about early human development. And safety is a major issue for iPS cell research aimed at clinical applications, since the cell reprogramming process can cause harmful mutations in the stem cells, increasing the risk of cancer. In light of these and other concerns, iPS cells may perhaps prove to be most useful in their potential to expand our overall understanding of stem cell biology, the net effect of which will provide the best hope of discovering new therapies for patients.
Many who oppose embryonic stem cell research believe for religious or other personal reasons that all preimplantation embryos have a moral standing equal to living persons. On the other hand, those who support embryonic stem cell research point out that not all religious traditions grant full moral standing to early-stage human embryos.
According to Jewish, Islamic, Hindu, and Buddhist traditions, as well as many Western Christian views, moral standing arrives much later during the gestation process, with some views maintaining that the fetus must first reach a stage of viability where it would be capable of living outside the womb. Living in a pluralistic society such as ours, supporters argue, means having to tolerate differences in religious and personal convictions over such theoretical matters as when, during development, moral standing first appears.
Other critics of embryonic stem cell research believe that all preimplantation embryos have the potential to become full-fledged human beings and that they should never have this potential destroyed. In response, stem cell supporters argue that it is simply false that all early-stage embryos have the potential for complete human life many fertility clinic embryos are of poor quality and therefore not capable of producing a pregnancy (although they may yield stem cells). Similarly, as many as 75% to 80% of all embryos created through intercourse fail to implant. Furthermore, no embryos have the potential for full human life until they are implanted in a womans uterus, and until this essential step is taken an embryos potential exists only in the most abstract and hypothetical sense.
Despite the controversies, embryonic stem cell research continues to proceed rapidly around the world, with strong public funding in many countries. In the U.S., federal money for embryonic stem cell research is available only for stem cell lines that are on the National Institutes of Health stem cell registry. However, no federal funds may be used to derive human embryonic stem cell lines; NIH funds may only be used to study embryonic stem cells that were derived using other funding sources.
Despite the lack of full federal commitment to funding embryonic stem cell research in the U.S., there are wide-ranging national regulatory standards. The National Academy of Sciences established guidelines in 2005 for the conduct of human embryonic stem cell research. (See Resources.) According to these guidelines, all privately and publicly funded scientists working with embryonic stem cells should have their research proposals approved by local embryonic stem cell research oversight (ESCRO) committees. ESCRO committees are to include basic scientists, physicians, ethicists, legal experts, and community members to look at stem-cell-specific issues relating to the proposed research. These committees are also to work with local ethics review boards to ensure that the donors of embryos and other human materials are treated fairly and have given their voluntary informed consent to stem cell research teams. Although these guidelines are voluntarily, universities and other research centers have widely accepted them.
At the global level, in 2016 the International Society for Stem Cell Research (ISSCR) released a comprehensive set of professional guidelines for human stem cell research, spanning both bench and clinical stem cell research. (See Resources.) Unlike the NAS guidelines, the ISSCR guidelines go beyond American standards, adding, for example, the recommendation that stem cell lines be banked and freely distributed to researchers around the world to facilitate the fields progress on just and reasonable terms.The potential for over-commercialization and restrictive patenting practices is a major problem facing the stem cell field today, which may delay or reduce the broad public benefit of stem cell research. The promise of broad public benefit is one of thejustifying conditions for conducting stem cell research; without the real and substantial possibility for public benefit, stem cell research loses one of its most important moral foundations.
However, providing useful stem-cell-based therapies in the future is not a simple proposition, either. Developing a roadmap to bring stem cell research into the clinic will involve many complex steps, which the new ISSCR guidelines help address. They include:
These and other difficult issues must be sorted out if stem cell research in all its forms is to fulfill its promise.
STEM CELL GLOSSARY
Newer ethical issues in stem cell research go far beyond the embryo debate, since they encompass all stem cell types, not just human embryonic stem cells, and because they involve human subjects who, despite what one may think about the moral status of preimplantation embryos, are unequivocally moral persons. No other emerging issue better encapsulates the above concern than the growing phenomenon of stem cell tourism. At present, stem cell-based therapies are the clinical standard of care for only afew conditions, such as hematopoietic stem cell transplants for leukemia and epithelial stem cell-based treatments for burns and corneal disorders. Unfortunately, some unscrupulous clinicians around the world are exploiting patients hopes by purporting to provide for large sums of money effective stem cell therapies for many other conditions. These so-called stem cell clinics advance claims about their proffered stem cell therapies without credible scientific rationale, transparency, oversight, or patient protections.
The administration of unproven stem cell interventions outside of carefully regulated research protocols endangers patients and jeopardizes the legitimate progress of translational stem cell scientific research. Patients who travel for unproven stem cell therapies put themselves at risk of physical and financial harm.
The ISSCR guidelines are a good point for thinking about this important problem. The guidelines allow for exceptional circumstances in which clinicians might attempt medically innovative care in a very small number of seriously ill patients, subject to stringent oversight criteria. These criteria include: independent peer review of the proposed innovative procedure and its scientific rationale; institutional accountability; rigorous informed consent and close patient monitoring; transparency; timely adverse event reporting; and a commitment by clinician-scientists to move to a formal clinical trial in a timely manner after experience with at most a few patients. By juxtaposing some current stem cell clinics against the standards outlined in the ISSCR guidelines, one may easily identify some clinics shortcomings and call into question the legitimacy of their purported claims of providing innovative care to patients.
Moving beyond past debates about embryo status to issues concerning the uses of all varieties of stem cells, one can begin to focus the bioethical discourse on areas that have a much broader consensus base of shared values, such as patient and research subject protections and justice. Justice may also call on regulatory and oversight bodies to include a greater involvement of community and patient advocates in the oversight of research. Dealing with the bioethics of stem cell research demands that we wrestle with these and other tough questions.
Insoo Hyun, PhD, is an associate professor of bioethics at Case Western Reserve University.
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Stem Cells - The Hastings Center
Antibiotic in human skin can beat superbugs. Now scientists have a way to put it to use – ThePrint
By daniellenierenberg
Text Size:A- A+
New Delhi: A team of scientists in Bengaluru has claimed that it was able to stimulate skin cells to secrete naturally occurring antibiotics that can help treat superbugs or drug-resistant bacteria.
Antibiotic drugs used to treat infections are increasingly becoming ineffective globally, with bacteria having developed resistance to them over time.
According to the latest study published last monthin journal Cell Reports, scientists have discovered a way to stimulate skin cells to secrete antimicrobial peptides (AMPs).
AMPs target and kill bacteria in such variable ways that few bacteria ever become resistant to these molecules. This makes them uniquely suited to treating antibiotic-resistant bacteria, also called superbugs.
So far, artificially creating effective AMPs for use as antibiotic had not been possible.The new discovery has the potential to treat and prevent infections for post-surgery wounds, especially in diabetic patients and those with weakened immune systems, the researchers said in a statement Thursday.
The team comprises scientists from the National Centre for Biological Sciences, an affiliate of the Tata Institute of Fundamental Research, the Institute for Stem Cell Science and Regenerative Medicine (inStem) and the R&D department of Unilever, all based in Bengaluru.
Also read: Worlds most-isolated city catches NASAs attention as it hunts for innovative tech
To develop new strategies to deal with antimicrobial resistance, scientists from inStem and Unilever probed the cellular mechanisms that regulate the release of AMPs.
Apart from their role as natural antibiotics, AMPs are also known to be involved in wound healing.
Amitabha Majumdar, a researcher at Unilever, hypothesised that the same machinery used to release AMPs during wound-healing could be harnessed to control AMP release from skin cells for treating or preventing infections.
To test this, Majumdar worked with Colin Jamora, a researcher at inStems Centre for Inflammation and Tissue Homeostasis, whose group works extensively on the mechanisms of wound-healing in skin.
The team found a new signalling pathway for long-term release of AMPs from skin cells. Usually, AMPs are released to fight off bacterial infections when direct contact between skin epidermal cells and bacteria occurs. This process is triggered by a reduction in the levels of a protein called caspase-8.
The scientists found that using molecular techniques to reduce caspase-8 is enough to trigger the release of stored AMP from skin cells.
Their study showed that just by modulating caspase-8 levels in the skin, AMP release can be controlled to prevent a whole spectrum of infections.
This may be especially useful for diabetics and patients with weakened immune systems who are highly susceptible to bacterial, yeast, fungal, and viral infections in post-surgery wounds.
Over the last century, bacteria have evolved so much to protect themselves against antibiotics that the World Health Organisation (WHO) fears humankind may soon slip back into a situation similar to the pre-antibiotic era.
According to a WHO report, the death toll caused by antimicrobial resistance is estimated to rise to 10 million annually by 2050 with India carrying one of the largest burdens of drug-resistant pathogens worldwide.
Also read: Gene editing might alter our DNA, but at the cost of our humanity
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Antibiotic in human skin can beat superbugs. Now scientists have a way to put it to use - ThePrint
MaaT Pharma Announces the Presentation of Positive Data with Its Lead Microbiome Biotherapeutic in Intestinal-Predominant Acute…
By daniellenierenberg
LYON, France--(BUSINESS WIRE)--MaaT Pharma announced today that leading hemato-oncological experts presented clinical data on the compassionate use of MaaT Pharmas lead full-ecosystem microbiome restoration biotherapeutic, MaaT013. The data included eight patients that developed gastrointestinal-predominant, acute Graft-versus-Host-Disease (GI aGvHD) after receiving an allogeneic Hematopoietic Stem Cell Transplantation (allo-HSCT) to treat their hematologic malignancies. All patients were positively impacted by the MaaT013 treatment, with three patients achieving complete response. GvHD, a condition where the transplant donors immune cells attack the patients tissues, is one of the most serious complications of allo-HSCT, and its acute GI form is fatal in most cases. MaaT013 features a consistently high diversity and richness of microbial species in their natural environment. It aims to restore the symbiotic relationship between microbes in the gut and the immune system of the patient to correct the responsiveness and tolerance (homeostasis) of immune functions and thereby contain GI GvHD. The results were presented in a poster presentation on December 7, 2019 during the 61st American Society of Hematology (ASH) Annual Meeting and Exposition held in Orlando, Florida.
The GI aGvHD patients who were treated with MaaT013 had a very poor prognosis with no other therapeutic options. The results following MaaT013 administration showed a positive impact on all patients, commented Professor Mohamad Mohty, MD, PhD, Head of the Hematology and Cellular Therapy Department at Sorbonne University, Saint Antoine Hospital in Paris. The most impressive results were seen in those patients who achieved a complete response and who were able to taper and stop using steroids and other immunosuppressants without relapse of gastrointestinal symptoms.
In the presented evaluation, eight patients with a median age of 67 were treated for classical aGvHD, late-onset aGvHD or aGvHD with overlap syndrome that were either steroid-resistant or steroid-dependent following stem cell transplantation. These patients had previously been treated with and failed up to five lines of systemic therapy for aGvHD. Each patient received at least one and up to three doses of MaaT013 and treatment response was evaluated seven days after each administration and on day 28 after the first dose. Based on the best response to the treatment, all eight patients experienced at least a partial response with three patients achieving complete response, two patients with very good partial response and three patients with partial response. Overall, the data demonstrated that reintroduction of a full-ecosystem microbiota provided therapeutic effect and was tolerated in a satisfactory manner in these patients.
Herv Affagard, Co-founder and CEO of MaaT Pharma added, We provided our cGMP-manufactured lead biologic drug, MaaT013, to hospitals as part of a compassionate use program to give GI GvHD patients a therapeutic option where there are no other available treatments after steroids and additional lines of treatment. These findings indicate that reestablishing the gut microbiome improved outcomes in these patients.
Moreover, MaaT Pharma is currently conducting the HERACLES Phase II clinical trial (NCT03359980) to evaluate the safety and efficacy of MaaT013 in steroid-refractory, GI aGvHD patients, with more than half of the patients enrolled.
To date, a total of 46 patients with GI GvHD have been treated with MaaT013, including patients under compassionate use and patients enrolled in the Phase II clinical trial. MaaT Pharma is actively developing an oral formulation of MaaT013 (a capsule, MaaT033) to provide easier administration for patients while delivering a similar effect of regenerating the microbial ecosystem with the goal of restoring immune homeostasis in the gut.
The poster can be viewed on the companys website under News.
About HERACLES
The HERACLES study is a multi-center, single-arm, open-label study, enrolling 32 patients to evaluate the efficacy and safety of MaaT Pharmas lead microbiome restoration drug candidate, MaaT013, in steroid-resistant, gastrointestinal-predominant aGvHD patients. Acute GvHD is a serious, often fatal syndrome typically involving the gut, skin, and liver. Treatments up to now focused largely on suppressing the immune reaction that is induced by the donor cells derived from the hematopoietic stem cell graft reacting against the host. These strategies have remained clinically unsuccessful in most cases, with mortality rates around 80% after twelve months in steroid-resistant cases. Patients with hematological malignancies receive multiple courses of chemotherapy, antibiotics, and ultimately conditioning before HSCT, which are known to severely impact the gut microbial composition.
About MaaT013
MaaT013 is the first full-ecosystem, off-the-shelf, reproducible, enema formulation manufactured using MaaT Pharmas integrated Microbiome Restoration Biotherapeutic (MMRB) platform. The product has a stability of up to 24 months and is characterized by a high diversity and consistent richness of microbial species derived from pooled healthy donors and manufactured at the companys centralized European cGMP production facility. MaaT013 has been granted Orphan Drug Designation by the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) and is already being administered in compassionate use.
About MaaT Pharma
MaaT Pharma, a clinical stage company, has established the most complete approach to restoring patient-microbiome symbiosis to improve survival outcomes in life-threatening diseases. Committed to treating blood cancers and Graft-versus-Host-Disease, a serious complication of allogeneic stem cell transplantation, MaaT Pharma has already achieved proof of concept in acute myeloid leukemia patients. Supporting the further expansion of our pipeline into improving outcomes of immunotherapy in solid tumors, we have built a powerful discovery and analysis platform, GutPrint, to evaluate drug candidates, determine novel disease targets and identify biomarkers for microbiome-related conditions. Our biotherapeutics are produced under the strictest cGMP manufacturing and quality control process to safely deliver the full diversity and functionality of the microbiome. MaaT Pharma benefits from the commitment of world-leading scientists and established relationships with regulators to spearhead microbiome treatment integration into clinical practice.
If you have no idea where to start with your skin care, these new kits make it dummy-proof – Well+Good
By daniellenierenberg
Putting together a skin-care routine from scratch can be a daunting task. Usually, it involves a lot of scrolling through websites wondering where to begin. According to dermatologists, the basics of a good routine include a cleanser, an antioxidant serum, a retinol, a moisturizer, and a sunscreen. Even with that in mind, putting together a multi-step regimen can often feel overwhelming, especially if youre new to the game.
The good news? The holiday shopping season means skin-care kits that are filled with the products you need for a lot less. From cleansers to serums to moisturizers, these babies have got it alljust add some sunscreen into the mix and youll be good to go, all with the single click of the add to cart button. Shop the best skin-care sets below, and get ready to have your skin-care routine down pat.
Achieve the #topshelf of your dreams with this set, which puts mini versions of the brands cult faves into one place. Youll get a Milky Jelly Cleanser, Priming Rich Moisturizer, Future Dew and Super Bounce Serums, andtwo Balm Dotcoms. Your Instagram feed (and your skin) will look lit.
Every serum your skin could possibly need all in one place. Theres the cult-fave Good Genes for chemical exfoliation, C.E.O. Glow Vitamin C for brightening, and A+ High Dose Retinol for stimulating cell turnover. Its also got an eye cream and a moisturizer, so all you need to bring to a party is a cleanser (which derms say you can totally get at the drugstore) and youll be good to go.
This deal seems almost too good to be true. Its gotallof our favorite Fresh products, including the cleanser and lip balm that half of our editors swear by, plus a moisturizer, face mask, and eye cream. You skin will slurp nourishing ingredients like rose, lotus, and black tea right up, and your lips will feel kissable well past New Years Eve.
A great skin-care routine has both exfoliating and hydrating elements, which this kit offers in spades. Theres a cleanser to help prep your skin, AHA Facial Radiance Pads to slough away dead skin cells, and a hydrating hyaluronic acid serum and colloidal oatmeal cream to keep skin moisturized. To keep your lips from feeling left out, theres also a petroleum lip balm thatll soothe away any chapping.
Get your hands on Sephoras seven favorite skin-care products, all in one place. Its a great way to discover clean brands you may not have tried yet by way of their bestselling products, which do everything from exfoliate to hydrate to soothe your skin. A few of my personal favorite picks from this kit? Biossance Squalane + Vitamin C Rose Oil, Farmacy Honeymoon Glow AHA resurfacing night serum, and Youth to the People Superberry Hydrate + Glow Dream Mask.
The best part about The Ordinary? The products are as affordable as they are efficaciousall year round. The Daily Set has everything your dry skin needs, including a hydrating gentle cleanser, a 2 percent hyaluronic acid serum, and an ultra-hydrating moisturizer.
If youve heard everyone in your group chat raving about Drunk Elephant for the last few years and have yet to try it for yourself, consider this your best point of entry. This set includes travel-sized versions of the brands best sellers, including the C-Firma serum, T.L.C. Framboos Glycolic Night Serum, and Protini Polypeptide Cream (plus, my personal favorite sunscreen, Umbra Tinte Physical Daily Defense SPF 30). Its a great way to sample a huge selection of the line before deciding which products are investing in full sizes of. Or, if youre already a Drunk Elephant devotee, this is a great way to keep your routine with you everywhere you go.
Protecting your skin barrier is critical to healthy skin, and thats the priority of this full-service set. The gentle cleanser will get rid of makeup and grime, and the lotion and lip balm help seal in moisture. Theres also an antioxidant treatment that reduces redness and blotchiness, plus irritation-soothing creams for your body and hands.
This line is the K-beauty holy grail for hydration, and is perfect for combatting the effects that cold weather has on your skin during the early months of the year. Each of the productsfrom the cream cleanser to the essence to the moisture cream to the sleep maskare all formulated with ingredients to lock in moisture and keep your complexion from feeling parched. Plus, the Lip Sleeping Mask is a treat for chapped lips at all hours of the day and night.
Ask any derm the holy-grail serums that everyone should have in their routine, and theyll tell you Vitamin C, hyaluronic acid, and retinol. This kit has all three of them, plus a caffeine-infused eye cream that will make you look instantly more awake.
As far as skin care goes, you really cant go wrong with a dermatologist-developed line. Our editors are huge fans of all things from Dr. Dennis Gross, and this kit will allow you to try his cosmetically elegant products like a ferulic and retinol overnight serum and eye cream, a vitamin C and collagen serum, and a best-in-class AHA/BHA Peel. As a whole, the set is meant to target fine lines and wrinkles, all while hydrating and brightening in the process.
Hardly surprising, but a lot of Dermstores favorite products areour favorite products, too. In this kit, youll receive picks from Naturopathica, SkinMedica, Bioderma, Boscia, and First Aid Beauty, among others, that make up an entire multi-step regimen. If youre not quite sure about going all-in on a single brand, this is a great way to try a lot of different things all at the same time for aseriouslydiscounted price.
Finding the right routine for acne can be a lot of trial and error, but this medical-grade kit does a lot of the legwork for you. Its got all of the best acne-fighting ingredients like Benzoyl Peroxide and Retinol, plus a niacinamide-packed moisturizer to reduce any irritation from the treatments.
A fan-favorite brand among both French girls and beauty editors, this oh-so-simple three step routine is perfect for anyone looking to streamline what theyre doing to their skin. Theres a micellar water, which acts as a gentle cleanser, a sensitive-skin-friendly moisturizer, and a puffiness-reducing eye cream to top it all off.
This kit has everything you need for cleansing, hydrating and brightening your skin, the combination of which will leave it looking radiant. Theres a stem cell cleanser, three different serums that have hyaluronic acid and vitamins C, A, and E, plus a mask for nights when your face needs a little bit more love.
To help brighten skin, you first need to slough away the old, dead skin cells that have been accumulating on your complexion. Start with a lactic acid-spiked resurfacing treatment and then slather on the potent vitamin C-laced brightening serum. Top it all off with pure argan oil (the ingredient the brand is known for) to moisturize it.
Heres what a dermatologists skin-care routine looks like:
Looking to buy beauty gifts for someone else this holiday season? Consider this hi-def makeup mirror, or one of the products our readers swear by for bringing dry hair back to life.
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If you have no idea where to start with your skin care, these new kits make it dummy-proof - Well+Good
5 Innovations From the Science of Senses Now. Powered by – Now. Powered by Northrop Grumman.
By daniellenierenberg
Millions of people around the world have some form of sight or hearing loss, have no sense of smell or taste or have lost limbs, taking away their sense of touch. Fortunately, the science of senses is the most advanced its ever been. Biotech researchers are developing methods that merge humans and machines in ways that could restore human abilities to hear, see, taste, smell and touch. From neuro-prosthetic limbs that mimic touch to bionic eyes and smart glasses that restore sights, the innovations could drastically improve the quality of life of people around the world.
Some of the most advanced technology developed around the science of senses comes from the field of prosthetic limbs, where researchers are finding ways to connect tissue to metal. Systems called brain-machine interfaces literally wire robotic limbs to a persons nervous system. Two of the latest achievements were reported in July 2019 in the journal Science Robotics.
In the first, a team from the University of Utah connected a robotic hand and partial forearm to the remaining nerves in the mans arm. The man trained his brain to control the motion of the hand. At the same time, artificial zaps sent to the robotic hand were designed to mimic the skins natural response patterns to touch. Remarkably, the man could more easily discriminate between small and large objects as well as soft and hard items while blindfolded and wearing headphones. Another team, based at the National University of Singapore, engineered flexible, electronic skin that contains artificial nerves that transmit signals 1,000 times faster than nerves in human skin. The skin is able to sense temperature, pressure and humidity and is also durable enough to function even if it is scratched or damaged.
Since the mid-1980s, a tiny electronic device called a cochlear implant has been providing the sense of sound to hundreds of thousands of people worldwide, according to the National Institutes of Health. Part of the implant is surgically placed under the skin behind the ear, with another part attached in the same position externally. A third part is inserted inside the ear canal. Unlike a hearing aid that amplifies sound, a cochlear implant senses sounds and converts them into an electric signal that it uses to stimulate a persons auditory nerve. Even people who are profoundly deaf can learn to discern sounds as long as some fraction of their nerve still functions.
But cochlear implants are not perfect. They are only capable of sensing and transmitting part of a sound waves full audio spectrum, producing a sound that has a metallic quality. That can make it difficult to filter out background noise, such as a crowd conversations or traffic. In 2019, a team from the University of Greenwich in England reported on new research that improves upon this technology, reports MedicalXpress. It deconstructs sounds from the environment and then reconstructs them with 90% to 100% percent efficiency. This means patients will be able to better distinguish noises from background sounds.
Smell loss, called anosmia, affects about 5% of the general population, according to the Massachusetts Eye and Ear Infirmary. The condition may be the result of something temporary, such as a sinus infection or swelling or polyps in the nasal cavity or it could be the result of damage to the sensory nerves. Permanent loss of smell can impact daily enjoyment of life and even affect safety. The inability of smelling smoke or natural gas could put someone in harms way.
Although there is no proven therapy, researchers at the Massachusetts Eye and Ear have, for the first time, invented a device that stimulates different smells. Their technology, which they reported in 2018 in the International Forum of Allergy & Rhinology, uses an array of tiny electrodes to send an electrical signal to the olfactory bulb, a structure in the brain involved in smell. In a small experiment, the scientists created different electrical stimulation in five patients, producing smells similar to onions and antiseptic as well as sour and fruity aromas. Although the innovation is still in the early stage, it demonstrates a possible path forward for a cochlear implant for the nose, the scientists say.
Although smell is connected to taste, its the receptor cells on the taste buds of a persons tongue that discern sweet, salty, sour, bitter or savory flavors. Medical procedures inside the mouth or ear can alter a persons taste, as can head trauma or ear infections, according to MedicineNet. Scientists have made a couple of attempts to solve the problem with technology. Back in 2013, a team from the National University of Singapore developed a taste simulator that used a kind of electronic tongue depressor to simulate taste sensations, New Scientist reported. Later, another team at City University of London invented a similar device called Taste Buddy that also stimulated taste buds to alter the flavor of foods, reported Digital Trends.
Unfortunately, neither gadget went beyond the research lab. For now, solutions may lie within human DNA. Lynnette McCluskey, a neurobiologist at the Medical College of Georgia at Augusta University, and her team are investigating whether a protein called interleukin-1, or IL-1, secreted during an injury could help rebuild a persons sense of taste. The protein promotes inflammation and also helps regulate nerve growth. In 2018, she and her colleagues received grant money to study whether manipulating the proteins after an injury could help the nerves associated with taste recover faster, reports MedicalXpress. It could take a few more years to find out.
Worldwide, 36 million people are legally blind, according to Nature. Some biotechnological solutions, such as growing stem cells into those that can repair damage to the retina or using techniques from gene therapy to correct genetic defects, are showing promising results. But technology is also playing a big role.
A bionic eye, called the Argus II, is a retinal prosthesis system that, since its development in early 2000, has restored some vision capabilities to more than 300 people. Its reserved for people who have no vision or almost no vision due to a genetic condition called retinitis pigmentosa. Patients undergo surgery, in which a tiny electronic device is attached to the persons retina. Its connected wirelessly to a pair of smart glasses that have a portable video-processing unit that project images from the outside world onto the persons retina. Clinical trials done in 2015 showed that visual function improved in 90% of people wearing the prosthesis and that 80% of patients reported improved quality of life, according to the American Academy of Ophthalmology.
Advances in technology are allowing machines to merge with the human body. Coupled with our growing ability to correct genetic defects or repair cellular damage, the science of senses is moving into the future. One day all humans could move through the world with all five of their senses intact seeing the unseen, hearing the unheard and tasting, touching and smelling new wonders that evoke all of the pleasures of being alive.
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