Hatching disease in a dish: The new frontier in drug testing – Maclean’s

Over lunch at the Canadian Centre for Alternatives to Animal Methods (CCAAM), Charu Chandrasekera nonchalantly mentions one of the projects her team is working on. We are just printing some human liver tissue right now, she says.

Chandrasekera launched the CCAAM at the University of Windsor in 2017, with help from the schools vice-president of research and innovation, Michael Siu, and dean of science, Chris Houser. The centre promotes non-animal methods in biomedical research, education, and regulatory (chemical safety) testing. In October 2019, the centre received a million-dollar gift from the Eric S. Margolis Family Foundation, which Chandrasekera says was instrumental in establishing the state-of-the art research laboratory, and in launching a number of important initiatives.

Chandrasekera says the move away from animal testing to human-based research models isnt radical but inevitable. After many years working in biomedical research with mouse models of heart disease and diabetes, It became very obvious that the work I was doing was not translatable [to humans], she says. Nothing was really reproducible; there were so many discrepancies and contradictions, even among the top-notch researchers.

Ninety-five per cent of drugs tested to be safe and effective in animal models fail in human clinical trials, says Chandrasekera. Alzheimers disease99.6 per cent drug failure rate, she says. It has been cured in mice. But we dont even understand the molecular mechanisms of this disease in humans, much less a cure.

RELATED:I am mine: This is what Alzheimers is like at 41

Empirical evidence from across a whole host of biomedical science disciplines shows us that animal models are failing both science and human health, echoes Elisabeth Ormandy, co-founder and executive director of Animals in Science Policy Institute, a registered Canadian charity working to promote better science without animals. Animal models can falsely show that a drug is effective, she says. They can also falsely show no effect, in which case a drug that would have been shown to be effective in humans never gets advanced to human clinical trials.

The result, she says, is billions of public tax dollars being wasted on research using ineffective animal models, and diversion of precious research funding away from other lines of scientific inquiry that might hold greater promise in terms of predicting drug safety, risk, and effectiveness.

Those other promising lines of scientific inquiry, say Ormandy and Chandrasekera, are human biology-based models. We can use human cells and tissues from cadavers, biopsies, and explanted organs [from surgeries], says Chandrasekera. And we can also engineer them. With adult stem cell technology, you can take a small biopsylike two-to-three millimetres from a persons skinto create any cell type in your body, she says. And if that person has a disease, such as Alzheimers, it will still be present in these cells. These cells can then be assembled to form tissue-like structures called organoids, or engineered through 3D-bioprinting to create more complex tissues, all of which can be combined to create what has become known as disease-in-a-dish. At present,Chandrasekera iscreating diabetes-in-a-dish.

Further, those cells and tissues can also be placed onto computer chips the size of thumb drives, where a large number of drugs can be tested to select whats most appropriate for youpersonalized medicine based on your cells, your tissues, your biologynot mouse biology, Chandrasekera explained in her April 2019 TedX Talk. The goal of the scientific community at large is to create a human-on-a-chip to emulate human biology better than animals, she says, which I think will happen over the next decade.

Currently there is no data on the success rates of human biology-based methods, because there are no drugs that have been approved without animal testing, since animal testing was mandated by regulatory guidelines several decades ago, says Chandrasekera.

However, a growing body of scientific data and internationally approved guidelines in chemical safety testing, indicate that alternative methods are equal or superior to animal models in predicting human biology, Chandrasekera says. Even computer simulations are out-predicting animal-derived data.

RELATED:Health care cannot modernize unless health policy changes first

Ifdisease-in-a-dish and toxicity-on-a-chip effortscontinue to advance at a fast pace with a sense of urgencybacked by global scientific, financial, legislative, and ethical mandates, she says, we will come to a point where we can test drugs without relying on animals.

And while Chandrasekera is busy both in the lab and on the global stage promoting her work, she is also focused on enlightening future scientists. Shes working the development of courses and degrees to train the next generation, she says, to think outside the cage.

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Hatching disease in a dish: The new frontier in drug testing - Maclean's

BrainStorm Cell Therapeutics to make scientific presentations at the 30th International Symposium on ALS/MND – GlobeNewswire

NEW YORK, Nov. 26, 2019 (GLOBE NEWSWIRE) -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leader in the development of innovative autologous cellular therapies for highly debilitating neurodegenerative diseases, announced today that the Company is proud to be a gold sponsor of the 30th International Symposium on ALS/MND.

The symposium will take place December 4 6, 2019, at the Perth Convention and Exhibition Centre in Perth, Australia. The International Symposium on ALS/MND is a unique annual event that brings together leading international researchers and health and social care professionals to present and debate key innovations in their respective fields.

Ralph Kern MD MHSc, BrainStorms Chief Operating and Chief Medical Officer, will deliver a podium presentation: Modulation of innate immunity by MSC-NTF (NurOwn) cells correlates with ALS clinical outcomes, on December 4, from 11:50 12:10 pm AWST during the opening day Clinical Trials Session. In addition to the podium presentation, the Company will also present Poster 153: MSC-NTF Differentiation Increases the Neurotrophic Effects of MSC Cells: Live Imaging Analysis, that directly demonstrates the neuroprotective effects of NurOwn in a neuronal cell culture model.

Our fully-enrolled phase 3 clinical trial is one of the most advanced clinical programs in ALS, stated Chaim Lebovits, President and CEO of BrainStorm. He added, The International Symposium on ALS/MND is an important venue to update the community on our clinical and scientific efforts towards the advancement of therapies that may address the unmet needs of those living with ALS. BrainStorm Cell Therapeutics is proud to serve as a sponsor of this important annual symposium which underscores our commitment to the international community of ALS and MND patients, their families and their caregivers.

Ralph Kern, MD, stated, It is a privilege to present our innovative biomarker and preclinical research at the International Symposium on ALS/MND. He added, Every year, symposium participants gather together and discuss the opportunities and the challenges that we will face during the upcoming year. Research and medical breakthroughs for the ALS and MND community continue to make significant progress and we look forward to sharing our insights and engaging with colleagues from around the globe. The International Symposium on ALS/MND reminds us how far we have come in investigational therapies and how much more progress is still needed to bring patients a better and more promising future.

About NurOwn

NurOwn (autologous MSC-NTF) cells represent a promising investigational therapeutic approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo. MSCs are converted into MSC-NTF cells by growing them under patented conditions that induce the cells to secrete high levels of neurotrophic factors. Autologous MSC-NTF cells can effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression. BrainStorm has fully enrolled a Phase 3 pivotal trial of autologous MSC-NTF cells for the treatment of amyotrophic lateral sclerosis (ALS). BrainStorm also received U.S. FDA acceptance to initiate a Phase 2 open-label multicenter trial in progressive MS and enrollment began in March 2019.

About BrainStorm Cell Therapeutics Inc.

BrainStorm Cell Therapeutics Inc. is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. The Company holds the rights to clinical development and commercialization of the NurOwn technology platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement. Autologous MSC-NTF cells have received Orphan Drug status designation from the U.S. Food and Drug Administration (U.S. FDA) and the European Medicines Agency (EMA) in ALS. BrainStorm has fully enrolled a Phase 3 pivotal trial in ALS (NCT03280056), investigating repeat-administration of autologous MSC-NTF cells at six sites in the U.S., supported by a grant from the California Institute for Regenerative Medicine (CIRM CLIN2-0989). The pivotal study is intended to support a filing for U.S. FDA approval of autologous MSC-NTF cells in ALS. For more information, visit BrainStorm's website at http://www.brainstorm-cell.com.

The International Symposium on ALS/MND is a unique annual event that brings together leading international researchers and health and social care professionals to present and debate key innovations in their respective fields. The Symposium is planned as two parallel meetings, one on biomedical research and the other on advances in the care and management of people affected by ALS/MND. Joint sessions consider issues of mutual concern, challenging current views and practices.

Safe-Harbor Statements

Statements in this announcement other than historical data and information constitute "forward-looking statements" and involve risks and uncertainties that could cause BrainStorm Cell Therapeutics Inc.'s actual results to differ materially from those stated or implied by such forward-looking statements. Terms and phrases such as "may," "should," "would," "could," "will," "expect," "likely," "believe," "plan," "estimate," "predict," "potential," and similar terms and phrases are intended to identify these forward-looking statements. The potential risks and uncertainties include, without limitation, risks associated with BrainStorm's limited operating history, history of losses; minimal working capital, dependence on its license to Ramot's technology; ability to adequately protect the technology; dependence on key executives and on its scientific consultants; ability to obtain required regulatory approvals; and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available at http://www.sec.gov. These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance or achievements.

BRAINSTORM CONTACTS:Investors:Uri Yablonka, Chief Business OfficerBrainStorm Cell Therapeutics IncPhone: : +1-201-488-0460Email: uri@brainstorm-cell.com

Media:Sean LeousWestwicke/ICR PRPhone: +1.646.677.1839Email:sean.leous@icrinc.com

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BrainStorm Cell Therapeutics to make scientific presentations at the 30th International Symposium on ALS/MND - GlobeNewswire

Bedfordshire Police officer reunited with woman whose life he saved through stem cell donation – Bedford Today

PC Andrew Harris, who is based at the Police Headquarters in Kempston, signed up to the Anthony Nolan stem cell donor register in 2008 when he joined the Metropolitan Police, he has since transferred to Bedfordshire Police.

Beatrice, a mother of two, from Palm Springs, California, was diagnosed with Myelofibrosis at the age of 23. The condition is a rare type of blood cancer which leads to leukaemia.

She has been living with the condition for the majority of her adult life, managing the symptoms with medication and waiting for a donor match, as this is the only known treatment for this condition.

Her ethnic background Beatrice is half European and half Vietnamese - meant that the chance of finding a match was extremely unlikely, and after years of searching in 2008 she gave up on ever finding a donor to focus her time on taking care of her young children.

Over time her health began to deteriorate and she was in desperate need of a stem cell donation to save her life. So in 2015, when she was about to enter into palliative care, her health care provider ran one last check on the donor register and she received the news that there was a perfect genetic match on a donor list a police officer from the United Kingdom.

At the same time the news came through, Beatrices condition had deteriorated and she was very poorly, thankfully, her health improved enough to get through the transplant procedure.

The transplant was successful and after the two year waiting period, Beatrice and Andrew got in touch through Skype. They have stayed in touch over time and met in person in London a month ago.

PC Harris said: When I was contacted and informed about this match I didnt hesitate for a second. I joined the police because I always wanted to help people and this wasnt any different.

The preparation for the procedure was painless and was done by my doctor and a local nurse. I was given injections for a period of five days to release stem cells into my bloodstream.

After that my stem cells were collected through a special machine similar to a dialysis machine, which was filtering them out of my bloodstream. This was done under local anaesthesia and was completely painless.

If felt unreal when I finally got to meet Beatrice over Skype after the two year waiting period. There was a sense of pride from my side and it was an extremely emotional moment for both of us. Since then we have stayed in touch but we only got to meet in person last month.

It was amazing to meet her and her family. Beatrice is such a strong character and to think that only couple of years ago she and her children were preparing to enter palliative care.

"She is now committed to spreading awareness of how important it is to register as a donor. It is not only one life thats been saved; it is also her family and loved ones who get to keep their daughter, mum and friend.

Our force is working with the local charity and we invite them regularly to events, during which you can join the stem cell register. I would like to encourage everyone who is eligible to sign up; you can be someones last hope and save their life.

People aged between 16 and 30 who are in good general health can sign up to the Anthony Nolan register at http://www.anthonynolan.org.

The charity will send you a swab pack in the post, which you should return to the charity. Whenever a patient with blood cancer or a blood disorder needs a lifesaving stem cell transplant, Anthony Nolan searches the register, looking for someone who is a genetic match for that patient.

If you are found to be a match the charity will be in touch, and will ask you to donate if youre still healthy and happy to do so.

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Bedfordshire Police officer reunited with woman whose life he saved through stem cell donation - Bedford Today

Bedfordshire Police officer reunited with stem cell recipient – Cranfield and Marston Vale Chronicle

A Bedfordshire Police officer has been reunited with a woman whose life he saved after donating stem cells four years ago. Beatrice, a mother of two, from Palm Springs, California, was diagnosed with Myelofibrosis at the age of 23.

The condition is a rare type of blood cancer which leads to leukaemia.

She has been living with the condition for the majority of her adult life, managing the symptoms with medication and waiting for a donor match, as this is the only known treatment for this condition.

Her ethnic background Beatrice is half European and half Vietnamese meant that the chance of finding a match was extremely unlikely. In 2008, after years of searching, she gave up on ever finding a donor to focus her time on taking care of her young children. This was two months before PC Andrew Harris joined the register.

Over time her health began to deteriorate and she was in desperate need of a stem cell donation to save her life.

So in 2015, when she was about to enter into palliative care, her health care provider ran one last check on the donor register and she received the most welcome news. There was a perfect genetic match on a donor list a police officer, PC Harris, from the United Kingdom.

At the same time the news came through, Beatrices condition had deteriorated and she was very poorly. Thankfully, her health improved enough to get through the transplant procedure. The transplant was successful and after the two year waiting period, Beatrice and Andrew got in touch through Skype.

They have stayed in touch over time and met in person in London a month ago.

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Bedfordshire Police officer reunited with stem cell recipient - Cranfield and Marston Vale Chronicle

Keeping cells alive | Interviews – The Naked Scientists

One of the big challenges with making and sustaining organs in the lab is plumbing- getting nutrients in, and waste out. This is the job of the vascular system, that carries blood around the body and is vital to keeping tissues alive.Mark Skylarr-Scott and his colleagues at the Wyss Institute for Biologically Inspired Engineering at Harvard University have recently come up with a new technique for 3D printing large, vascularized human organ building blocks. The technique lays down tracks for blood vessels between clumps of cells to help keep tissues nourished. Using this method, theyve managed to keep clumps of heart cells alive for weeks, which would previously have been almost impossible. Mark took Katie Haylor through the process...

Mark- Step one would be prepare approximately one cup of stem cells. In a lab that involves using something called a bio-reactor to generate vast numbers of stem cells in three dimensions. We then need to instruct those cells to become heart cells. So we put the correct ingredients into this bio-reactor so that they are able to develop into a heart cell.

Mark- So stem cells, and then we provide chemicals that make the stem cells think that they're supposed to develop into beating heart cells. You know, this is a fairly standard process up to this point. And now if we have hundreds of millions of heart cells, a heart isn't a hundred million cells floating around in a bio-reactor. It's actually a solid organ that beats and you know, pumps out blood. We now need to think of a way to compile these together into a tissue.

Mark- So then step two, so we now have hundreds of millions of cells. At the moment, little pieces of tissue. So we actually, they're not single cells, they are in little clumps, about half a millimeter across. If we now take these little clumps and we put them in a centrifuge, we are able to sort of push them all together. We spin them down, they form, a little pellet of cells. Cool it on ice, so it's now at zero degrees.

Mark- We then come with a three D printer and inject gelatin - solid at room temperature and liquid when it's 37 degrees - in three dimensions into this group of cellular aggregates. Now, if this material were liquid, the gelatin would just sink and I wouldn't be able to create a 3D structure. If the material, if my cells were too solid, I would essentially be carving it like a turkey as I come in with a nozzle and 3D printer and inject gelatin in 3D. And this would also break the tissue. But because these cells are halfway between a liquid and a solid, I'm actually able to come in and lay material, this gelatin material in three dimensions and it will hold in place so that when I now raise the temperature, my cells all stick together. So now my tissue is become solid-like, and the gelatin that I printed melts and becomes liquid-like. If I now flush that gelatin out, I'm left with space. I'm left with channels that I can now connect a pump to those channels and actually keep the tissue alive, keep it perfused and viable.

Katie- So it's a bit like a Goldilocks porridge situation. And how does this compare to how a full scale heart would be vascularized?

Mark- This is very different in terms of the process of how we develop, but this is obviously because the goal of creating an organ for transplantation, you can't wait, you know, 20 years for an adult heart to develop, we need to be able to manufacture it quickly. So the process is very different.

Mark- In terms of the architecture, we similarly have blood vessels in our body and in our organ that start very large. The aorta comes from the heart and then it splits and it splits and you're down to what's called arterioles, little arteries. And then the arterioles become capillaries. And then the capillaries rejoin to form veins and then larger veins. Um, so this hierarchical arrangement of blood vessels we're able to reproduce, with a 3D printer - not necessarily at the resolution of capillaries, but certainly in terms of having these branched hierarchical networks.

Mark- This is actually important for transplantation. If a surgeon wants to be able to connect tissue to the patient, they don't want to have a hundred different tubes that they need to suture to connect to be able to feed that tissue. They want a single inlet that will then split, you know, and feed the full volume of the tissue and then a single outlet that they can plumb into.

Mark- I'd say the advantage of our method here is these organoids that are being developed, they really leverage biology's natural ability to make complex structures on their own. The instructions for generating the sort of patterns that you see in organs, of course, it's in our DNA. Organoid protocols, they take advantage of that to form these tissues that can exhibit amazingly complicated architectures that self assemble. They develop on their own for free essentially. So because at the smallest scale we have all of these structures already in place in the organoids, if we can now compile hundreds of thousands of these organoids together into a larger tissue that we can keep alive, we hope that through 3D printing, we get the large scale structures and vasculature necessary to keep it alive, but through developmental biology and the fact that the stem cell derived organoids that have all the microstructure already present, that we get the small scale structure in place as well.

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Keeping cells alive | Interviews - The Naked Scientists

Dana-Farber joins with leading Boston teaching hospitals and universities – Mirage News

Some patients who have not responded to traditional medicines are now experiencing remarkable recoveries thanks to next-generation immunotherapies. These therapies equip a patients own immune cells to recognize, target, and destroy cancer cells. To do this, the patients cells are collected, modified, and re-introduced into their body a complex procedure currently available to only a small number of people. With major innovations underway, this fast-moving area of science is set to expand the pool of patients who will respond to immunotherapies and other emerging medicines. But there is a bottleneck in the discovery pipeline. Manufacturing backlogs are slowing the production of cells that are essential to research, holding up the availability of new treatments headed for the clinic.

To address these challenges, a group of Massachusetts academic, healthcare, biotech, and biopharma industry leaders have come together to establish a new center.

The new center for advanced biological innovation and manufacturing will explore and cultivate innovations in cell and gene therapy, advance biologic innovation and manufacturing, and accelerate developments in immunotherapy, cell therapies, gene editing, and other technologies that carry the promise of lasting impact on human health globally and boosting the local economy. By fostering collaboration and innovation, it holds the promise of speeding innovation and broadening the universe of patients that can be served by these emerging therapies.

Leaders from Dana-Farber, Harvard University, Massachusetts Institute of Technology (MIT), Fujifilm Diosynth Biotechnologies (FDB), GE Healthcare Life Sciences, Alexandria Real Estate Equities, Inc., will comprise the Board of Directors, while other contributing members include Beth Israel Deaconess Medical Center, Boston Childrens Hospital, Brigham and Womens Hospital, Massachusetts General Hospital, MilliporeSigma, and the Commonwealth of Massachusetts.

The $50 million center will be an independent non-profit organization located in the greater Boston area and will be named, along with incorporation, in the new year. The expectation is that this will be an independent, separate nonprofit corporation.

Scientific breakthroughs in cellular, immune and gene therapies from just the past few years are now saving lives and represent a truly revolutionary time in medicine, said Laurie H. Glimcher, MD, president and CEO of Dana-Farber Cancer Institute. By bringing together the talent that exists only in the Massachusetts life sciences ecosystem and fostering collaboration, this new manufacturing center will help to extend the benefit of these technologies to more patients and accelerate discoveries to effectively treat more diseases.

Home to a dense concentration of world-leading universities, hospitals, large pharmaceutical companies and small biotech firms, Massachusetts is at the forefront of biomedicine. These organizations are redefining traditional ideas about biomedicine and rapidly advancing discoveries from lab to clinic.

The overarching mission of the newly established consortium is to catalyze the development of transformative therapies by shortening the path between research and clinical application. The consortium will harness world-leading expertise to propel forward fast-emerging and promising science, the cost and risks of which are daunting for any single institution to tackle alone. By housing institutions with strengths in each link in the chain of innovation within one facility, the partners believe new innovations in both science and manufacturing will speed the introduction of new medicines to patients.

The ability of scientists to modify cells for therapeutic application, and to alter disease-causing genes, has ushered in a new era in biomedicine. Some of these potential therapies are entering clinical trials, others will soon be in the clinic, and still more are in early stages of investigation. There is strong motivation and acute need to translate these emergent approaches to clinical use. More than 60,000 patients globally are currently participating in clinical trials for new cell and gene therapies, including gene editing.

Currently, major obstacles and bottlenecks to getting new treatments into the clinic include production specifically, the pressure placed on highly skilled contract manufacturers to deliver customized cells and viral vectors of high quality and regulatory compliance to labs throughout the region. Because of the backlog, scientists may need to wait as long as 18 months for essential products they need to carry out research.

The center will offer three critical services to the Massachusetts life science ecosystem.

It will provide preferred access to a new manufacturing facility at favorable pricing, reducing the wait and cost for researchers at universities, hospitals and start-ups. The facility offers pharma-grade good manufacturing practices (GMP) manufacturing capacity in approximately eight cleanrooms for the production of cell and viral vector products and other related raw materials that may be used for phase 1 or phase 2 clinical trials.

The facility will have a shared innovation space where scientists from universities, hospitals, and industry can work side-by-side with dedicated, experienced, professional staff. This will be a unique opportunity to refine new methods rapidly, readying them for first-in-patient clinical trials. With access to manufacturing within the same space, the center will cultivate a community of experts across sectors who share a goal of serving patients, and who are dedicated to innovating collectively in both manufacturing processes and drug development.

The center will provide a platform for workforce development and training in a rapidly growing field, where there is a critical need for people with specialized skills.

The modular design of the new facility will make it easier for users to adapt quickly to changes in technology. Such flexibility will remove barriers to accessing promising innovations that emerge from improved methods involving gene manipulation, gene editing, oligonucleotides, peptides, and new methods and discoveries as they arise.

While there are many commercial contract manufacturing organizations, shared lab spaces, and even small manufacturing spaces at universities and hospitals in the U.S., this is a first-of-its-kind facility in three respects. First, for its intention to produce both cell and viral vector products within a single physical space. Second, for its unique partnerships between industry, academia, and leading area hospitals. Finally, for its partners aspirations to provide services to researchers and start-ups that will advance this new area of medicine through collaboration.

This powerful collaboration embodies the deep and broad world-class expertise in multiple disciplines that exists across this region, said Harvard President Larry Bacow. We are privileged to be part of this collaborative initiative. It will advance scientific discovery, reaffirm the regions global leadership in the life sciences, and bring forward life-saving and life-changing therapies that will make a difference for people around the world.

The broad question that we were trying to address was, How can we best position our region to be preeminent in the life sciences in the decades to come?' said Alan M. Garber, Harvards Provost, who helped conceive of the project more than two years ago and has shepherded it since then. We have a vibrant life sciences community, with some of the worlds greatest hospitals, universities, and life sciences companies of all kinds. We also have a strong financial sector that helps to spawn and support new companies. So the elements for rapid progress in the life sciences particularly in the application of the life sciences to human health are all here. But with such a rapid pace of innovation, its easy to fall behind. We wanted to make sure that would not happen here.

MIT researchers are developing innovative approaches to cell and gene therapy, designing new concepts for such biopharmaceutical medicines as well as new processes to manufacture these products and qualify them for clinical use, said MIT Provost Martin A. Schmidt. A shared facility to de-risk this innovation, including production, will facilitate even stronger collaborations among local universities, hospitals, and companies and ultimately, such a facility can help speed impact and access for patients. MIT appreciates Harvards lead in convening exploration of this opportunity for the Commonwealth.

Richard McCullough, Harvards vice provost for research and professor of materials science and engineering, who helped lead the project, said, the power of facilitys partners will accelerate therapeutic discoveries and have the ability to advance biologics from the lab to the bedside.

Its an exciting time for the life sciences industry with cell and gene therapies in position to revolutionize the global healthcare system. While these therapies are promising, challenges in manufacturing, access and cost must be addressed so they can reach their full potential. Initiatives such as the center are important because they bring together key life sciences stakeholders together to share their capabilities, knowledge and expertise to collaborate and accelerate innovation, said Emmanuel Ligner, CEO and President of GE Healthcare Life Sciences.

We are very proud to be part of this unparalleled consortium to create an innovative and collaborative center involving advanced technologies as well as next-generation manufacturing. The highly respected partner institutions have the scientific talent and the engineering capabilities to deliver truly novel therapies to patients suffering today from serious and life-threatening diseases and also to design the next-generation processes that will accelerate the translation of tomorrows cost-effective, lifesaving medicines from bench to bedside, said Joel S. Marcus, executive chairman and founder, Alexandria Real Estate Equities, Inc. and Alexandria Venture Investments.

We are excited to be a founding member of this consortia. Partnering to get medicines to patients is what we are all about. The opportunity to do this in collaboration with everyone that has come together to make this a reality is something that really meets our core purpose to deliver tomorrows medicines as a partner for life, said Martin Meeson, President & COO, FUJFILM Diosynth Biotechnologies USA.

Massachusetts new center for advanced biological innovation and manufacturing will focus first on emergent areas such as cell therapies and gene therapies, and other advanced therapy medicinal products. Cell therapies that help a patients own immune system target cancer cells have been remarkably successful. One example is CAR-T cell therapy, in which a patients own T cells are modified to identify and attack cancer cells in the blood more easily. But immunotherapy is not restricted to treating cancers. Scientists are finding new ways to harness the immune system to treat a broadspectrum of diseases, including type 1 diabetes and many others. Cell therapies more broadly harnessing unique properties of adult stem cells, for example are under wide consideration for regenerative medicine, including joint tissue repair and neurodegeneration.

Gene therapies offer new hope to patients, often children, who suffer from debilitating inherited diseases. They involve introducing, removing, or changing a targeted gene within a patients cells. The goal is to make the patients cells produce disease-fighting proteins, or to stop them from producing disease-causing versions of a protein. Gene-editing research is progressing very rapidly, but there is a marked shortage of capability for manufacturing the gene delivery vectors.

Hospitals need to be able to create customized therapeutics for their patients, but most do not have manufacturing facilities on-site. Beyond the constraint of limited facilities to produce potential new treatments, much technological innovation is required to produce these medicines more efficiently in terms of time, labor, and cost and in accordance with regulatory guidance. The new center would be equipped to handle some of this work for technology innovation and early stage clinical trial-scale production, which would directly help bring promising solutions to patients sooner.

We need more manufacturing capability in order to translate our work, especially in the stem cell field, said Leonard Zon, MD, director of the Stem Cell Research Program at Boston Childrens Hospital. For academic investigators who want to see their basic science advance into the clinic space, its important to have a manufacturing facility collaborate on protocols. Researchers can then exchange information directly with the facility, optimizing protocols and working smarter.

This collaboration represents an exciting opportunity to harness the collective efforts of leading academic, industrial and clinical institutions to further explore exciting new technologies and therapies that are inspiring scientists and offering new hope to our patients, says Peter L. Slavin, MD, MGH president. New scientific fields like regenerative medicine, gene editing and immunotherapy are unlocking clues to understanding disease which can lead to better treatments and ultimately, richer, more healthy lives for our patients and their families.

Our mission at Beth Israel Deaconess Medical Center is to provide extraordinary care supported by world-class research and education, said Peter J. Healy, president of Beth Israel Deaconess Medical Center. We are happy to be a founding member of this innovative consortium, which will allow us to work collaboratively across the diverse health care ecosystem. Together, we will propel the fields of cell therapy, gene therapy and gene editing forward with the shared goal of transforming how we care for patients right here in Boston and around the world.

Boston is an epicenter of biomedical research and innovation, said Brigham Health president Elizabeth G. Nabel, MD. In furthering the Brighams commitment to advancing development and delivery of cell and gene therapies, this unique collaboration is an opportunity to accelerate the pace and broaden the manufacturing capacity for therapies that have the potential to significantly improve patient outcomes.

Never before have we had so many breakthroughs available in the clinic. However, it can take up to 30 days, needle to needle, to deliver a CAR-T therapy to a patient, and that does not take into account any of the bottlenecks in the supply chain that could occur along the way. It is our collective responsibility to eliminate any barriers to making these life-saving medicines accessible to patients everywhere, said Udit Batra, CEO, MilliporeSigma.

The Commonwealths life sciences ecosystem is thriving because of the strength of the academic, research and industry partners that call Massachusetts home, and their commitment to collaboration, said Secretary of Housing and Economic Development Mike Kennealy. Combining a manufacturing facility, co-working labs, and workforce development and training in this first-in-the-nation center will boost the regional economy, create jobs and accelerate the delivery of next-generation therapies.

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Dana-Farber joins with leading Boston teaching hospitals and universities - Mirage News

Stem cells’ role in medicine and research – The Medium

What are stem cells and what role can they play in medicine andresearch? Stem cell research offers exciting possibilities in terms ofregenerative medicine. However, there are ethical controversies and challengesimpeding the fields advancement. In this article, The Medium presents a briefoverview of the unique abilities, applications, and challenges of stem cells.

According tothe National Institute of Health, stem cells are able to develop into manydifferent cell types in the body during early life and growth. When stem cellsdivide, the new cell can become another stem cell or it can become aspecialized cell such as a muscle cell or a brain cell. Stem cells provide newcells for the body as it grows and replaces damaged or lost specialized cells.The two unique properties of stem cells are that the stem cells can dividemultiple times to produce new cells, and as they divide, the stem cells cangenerate other types of cells found in the body.

In organs suchas the gut and the bone marrow (the soft tissue inside most bones), stem cellsroutinely divide to replace damaged tissue. However, in other organs such asthe heart, stem cells require certain physiological conditions to facilitate celldivision.

Stem cells canbe divided into two categories: embryonic stem cells and adult stem cells.Embryonic stem cells are derived from a blastocystan early stage of embryodevelopment. The blastocyst contains the trophectoderm, which will eventuallyform the placenta, and the inner cell mass, which will develop into the embryo,and later into the organism. Stem cells taken from the inner cell mass arepluripotentthey can develop into any cell type in the body. The embryonic stemcells used in research are sourced from unused embryos that were a result of anin vitro fertilization procedure and were donated for scientific research.

Adult stemcells also have the ability to divide into more than one cell type; however,they are often restricted to certain types of cells. For example, an adult stemcell found in the liver will only divide into more liver cells. In 2006, ShinyaYamanaka, a Japanese stem cell researcher, discovered how to program inducedpluripotent stem cells (iPSCs). iPSCs are adult cells which have beengenetically reprogrammed into a pluripotent embryonic stem cell-like state.Yamanaka won the Nobel Prize for Physiology or Medicine alongside Englishdevelopmental biologist Sir John Gurdon in 2012 for this important discovery.

There arenumerous ways in which stem cells can be used. Firstly, human embryonic stemcells can provide information as to how cells divide into tissues and organs.Abnormal cell division can cause cancer and birth defects, and therefore, amore comprehensive understanding of the processes underlying cell division maysuggest new therapy strategies. Another beneficial avenue involves drug testingas new medications could be tested on cells developed from stem cells in thelab. However, a challenge for researchers is to create an environment identicalto the conditions found in the human body.

Finally, stemcells present exciting possibilities in cell-based therapies and regenerativemedicine. Instead of relying on a limited supply of donated organs and tissuesto replace damaged and destroyed ones, stem cells could be directed to developinto the desired cell type and treat diseases such as heart disease, diabetes,and spinal cord injuries. For example, healthy heart muscle cells could begenerated from stem cells in a laboratory and transplanted into an individualwith heart disease. However, there is still research and testing which needs tobe conducted before researchers can confirm how to effectively and safely usestem cells to treat serious disease.

As explainedby the University of Rochesters medical centre, there are several challengesassociated with stem cells. Researchers first need to learn about how embryonicstem cells develop so that they can control the type of cells generated fromstem cells. Scientists also need to determine how to ensure that the cellsdeveloped from stem cells in the lab are not rejected by the human body. Adultpluripotent stem cells are found in small amounts in the human body and arehard to grow in the lab. There are also numerous ethical issues surrounding theuse of embryonic stem cells as some individuals believe that using cells froman unused blastocyst and consequently, rendering it incapable to develop intoan organism, is similar to destroying an unborn child. Others argue that theblastocyst is not a child yet as it needs to be imbedded into the mothersuterus wall before it has the chance to develop into a fetus. Supporters ofembryonic stem cell research also say that many surplus blastocysts aredestroyed in fertility clinics and can be better used to research medicaltreatments which could save peoples lives.

Students canlearn more about stem cells in BIO380H5: Human Development. Furthermore, Dr.Ted Erlicks lab at UTM is researching how complex neural circuits developfrom an initial population of stem cells. Stem cell research offers promisingavenues of treating diseases and understanding how humans develop. However,there is still a substantial amount of research which needs to be conducted andethical concerns which need to be appropriately addressed and resolved.

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Stem cells' role in medicine and research - The Medium

Modulating the expression and activity of the potassium-chloride cotransporter KCC2 – SFARI News

The proper regulation of neural excitatory/inhibitory (E/I) balance has been a subject of intense study in autism spectrum disorder (ASD) and other neurodevelopmental disorders. One particular focus has been the progressive increase in chloride ion extrusion from neurons as development proceeds, which is critical for the developmental switch in GABA function from excitatory to inhibitory. Three new studies with potential therapeutic implications shed new light on how the expression and function of KCC2 (a neuron-specific K+/Cl cotransporter that plays an important role in this process) is regulated.

Previous in vitro studies have shown that KCC2 activity is substantially modulated by phosphorylation at two particular threonine residues. In two new papers partly supported by a SFARI Pilot Award, SFARI Investigator Kristopher Kahle and Igor Medina developed a knockin mouse model of constitute phosphorylation at these two key threonine residues. Mice that were homozygous for these mutations died within 12 hours after birth, highlighting that precise phosphoregulation of these sites is essential for postnatal survival. By contrast, heterozygous mice were viable, allowing for an examination of subsequent neurodevelopmental effects. Among the findings was that this constitutively phosphorylated version of KCC2 prevented the normal increase in its activity during development. They associated this reduced KCC2 activity with reduced GABAergic inhibition, an enhanced E/I ratio, reduced seizure threshold, impaired social interaction and additional effects on respiration and locomotion.

In a separate study, SFARI Bridge to Independence awardee Xin Tang, together with SFARI Investigators Rudolf Jaenisch and Mriganka Sur, carried out a high-throughput screen for Food and Drug Administration (FDA)-approved drugs that might act to boost KCC2 expression in neurons derived from human embryonic stem cells. Several such compounds were identified, including those that are inhibitors of the tyrosine kinase FLT3 and the GSK-3 pathway. Of note, a few of these compounds were able to rescue phenotypes associated with Rett syndrome in both MECP2-null human neurons and Mecp2 mutant mice, including respiratory and locomotion phenotypes in the latter.

These findings give investigators new tools with which to explore KCC2 function during brain development and potentially to manipulate its activity for therapeutic benefit.

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Modulating the expression and activity of the potassium-chloride cotransporter KCC2 - SFARI News

Stem Cell Assay Market Demand with Leading Key Players and New Investment Opportunities Emerge To Augment Segments in Sector By 2025 – The Denton…

Stem Cell Assay Market: Snapshot

Stem cell assay refers to the procedure of measuring the potency of antineoplastic drugs, on the basis of their capability of retarding the growth of human tumor cells. The assay consists of qualitative or quantitative analysis or testing of affected tissues and tumors, wherein their toxicity, impurity, and other aspects are studied.

With the growing number of successful stem cell therapy treatment cases, the global market for stem cell assays will gain substantial momentum. A number of research and development projects are lending a hand to the growth of the market. For instance, the University of Washingtons Institute for Stem Cell and Regenerative Medicine (ISCRM) has attempted to manipulate stem cells to heal eye, kidney, and heart injuries. A number of diseases such as Alzheimers, spinal cord injury, Parkinsons, diabetes, stroke, retinal disease, cancer, rheumatoid arthritis, and neurological diseases can be successfully treated via stem cell therapy. Therefore, stem cell assays will exhibit growing demand.

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Another key development in the stem cell assay market is the development of innovative stem cell therapies. In April 2017, for instance, the first participant in an innovative clinical trial at the University of Wisconsin School of Medicine and Public Health was successfully treated with stem cell therapy. CardiAMP, the investigational therapy, has been designed to direct a large dose of the patients own bone-marrow cells to the point of cardiac injury, stimulating the natural healing response of the body.

Newer areas of application in medicine are being explored constantly. Consequently, stem cell assays are likely to play a key role in the formulation of treatments of a number of diseases.

Global Stem Cell Assay Market: Overview

The increasing investment in research and development of novel therapeutics owing to the rising incidence of chronic diseases has led to immense growth in the global stem cell assay market. In the next couple of years, the market is expected to spawn into a multi-billion dollar industry as healthcare sector and governments around the world increase their research spending.

The report analyzes the prevalent opportunities for the markets growth and those that companies should capitalize in the near future to strengthen their position in the market. It presents insights into the growth drivers and lists down the major restraints. Additionally, the report gauges the effect of Porters five forces on the overall stem cell assay market.

Global Stem Cell Assay Market: Key Market Segments

For the purpose of the study, the report segments the global stem cell assay market based on various parameters. For instance, in terms of assay type, the market can be segmented into isolation and purification, viability, cell identification, differentiation, proliferation, apoptosis, and function. By kit, the market can be bifurcated into human embryonic stem cell kits and adult stem cell kits. Based on instruments, flow cytometer, cell imaging systems, automated cell counter, and micro electrode arrays could be the key market segments.

In terms of application, the market can be segmented into drug discovery and development, clinical research, and regenerative medicine and therapy. The growth witnessed across the aforementioned application segments will be influenced by the increasing incidence of chronic ailments which will translate into the rising demand for regenerative medicines. Finally, based on end users, research institutes and industry research constitute the key market segments.

The report includes a detailed assessment of the various factors influencing the markets expansion across its key segments. The ones holding the most lucrative prospects are analyzed, and the factors restraining its trajectory across key segments are also discussed at length.

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Global Stem Cell Assay Market: Regional Analysis

Regionally, the market is expected to witness heightened demand in the developed countries across Europe and North America. The increasing incidence of chronic ailments and the subsequently expanding patient population are the chief drivers of the stem cell assay market in North America. Besides this, the market is also expected to witness lucrative opportunities in Asia Pacific and Rest of the World.

Global Stem Cell Assay Market: Vendor Landscape

A major inclusion in the report is the detailed assessment of the markets vendor landscape. For the purpose of the study the report therefore profiles some of the leading players having influence on the overall market dynamics. It also conducts SWOT analysis to study the strengths and weaknesses of the companies profiled and identify threats and opportunities that these enterprises are forecast to witness over the course of the reports forecast period.

Some of the most prominent enterprises operating in the global stem cell assay market are Bio-Rad Laboratories, Inc (U.S.), Thermo Fisher Scientific Inc. (U.S.), GE Healthcare (U.K.), Hemogenix Inc. (U.S.), Promega Corporation (U.S.), Bio-Techne Corporation (U.S.), Merck KGaA (Germany), STEMCELL Technologies Inc. (CA), Cell Biolabs, Inc. (U.S.), and Cellular Dynamics International, Inc. (U.S.).

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Stem Cell Assay Market Demand with Leading Key Players and New Investment Opportunities Emerge To Augment Segments in Sector By 2025 - The Denton...

Stem Cells Market 2019 Global Growth Analysis and Forecast Report by 2025 – Markets Gazette 24

New York, November 26, 2019: The global stem cells market is expected to grow at an incredible CAGR of 25.5% from 2018to 2024and reach a market value of US$ 467 billion by 2024. The emergence of Induced Pluripotent Stem (iPS) cells as an alternative to ESCs (embryonic stem cells), growth of developing markets, and evolution of new stem cell therapies represent promising growth opportunities for leading players in this sector.

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Due to the increased funding from Government and Private sector and rising global awareness about stem cell therapies and research are the main factors which are driving this market. A surge in therapeutic research activities funded by governments across the world has immensely propelled the global stem cells market. However, the high cost of stem cell treatment and stringent government regulations against the harvesting of stem cells are expected to restrain the growth of the global stem cells market.

This report will definitely help you make well informed decisions related to the stem cell market. The stem cell therapy market includes large number of players that are involved in development of stem cell therapies of the treatment of various diseases. Mesoblast Ltd. (Australia), Aastrom Biosciences, Inc. (U.S.), Celgene Corporation (U.S.), and StemCells, Inc. (U.S.) are the key players involved in the development of stem cell therapies across the globe.

This market research report categorizes the stem cell therapy market into the following segments and sub-segments:

The Global Stem Cell Market this market is segmented on the basis of Mode of Therapy, Therapeutic Applications and Geography.

By Mode of Therapy this market is segmented on the basis of Allogeneic Stem Cell Therapy Market and Autologous Stem Cell Therapy Market. Allogeneic Stem Cell Therapy Market this market is segmented on the basis of CVS Diseases, CNS Diseases, GIT diseases, Eye Diseases, Musculoskeletal Disorders, Metabolic Diseases, Immune System Diseases, Wounds and Injuries and Others. Autologous Stem Cell Therapy Market this market is segmented on the basis of GIT Diseases, Musculoskeletal Disorders, CVS Diseases, CNS Diseases, Wounds and Injuries and Others. By Therapeutic Applications this market is segmented on the basis of Musculoskeletal Disorders, Metabolic Diseases, Immune System Diseases, GIT Diseases, Eye Diseases, CVS Diseases, CNS Diseases, Wounds and Injuries and Others.

By Regional Analysis this market is segmented on the basis of North America, Europe, Asia-Pacific and Rest of the World.

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Table of Contents

1 INTRODUCTION

2 Research Methodology

2.1 Research Data2.1.1 Secondary Data2.1.1.1 Key Data From Secondary Sources2.1.2 Primary Data2.1.2.1 Key Data From Primary Sources2.1.2.2 Breakdown of Primaries2.2 Market Size Estimation2.2.1 Bottom-Up Approach2.2.2 Top-Down Approach2.3 Market Breakdown and Data Triangulation2.4 Research Assumptions

3 Executive Summary

4 Premium Insights

5 Market Overview

6 Industry Insights

7 Global Stem Cell Therapy Market, By Type

8 Global Stem Cell Therapy Market, By Therapeutic Application

9 Global Stem Cell Therapy Market, By Cell Source

10 Stem Cell Therapy Market, By Region

11 Competitive Landscape

12 Company Profiles

12.1 Introduction

12.1.1 Geographic Benchmarking

12.2 Osiris Therapeutics, Inc.

12.3 Medipost Co., Ltd.

12.4 Anterogen Co., Ltd.

12.5 Pharmicell Co., Ltd.

12.6 Holostem Terapie Avanzate Srl

12.7 JCR Pharmaceuticals Co., Ltd.

12.8 Nuvasive, Inc.

12.9 RTI Surgical, Inc.

12.10 Allosource

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Stem Cells Market 2019 Global Growth Analysis and Forecast Report by 2025 - Markets Gazette 24