Category Archives: Somatic Stem Cells

Osteonecrosis Treatment Market Benefit and Volume with Status and Prospect to 2026 – Crypto Journal


In a word,Osteonecrosis Treatment Marketreport provides elaborate statistics and analysis on the state of the industry; and may be a valuable supply of steerage and direction for corporations and people curious about the market.

Osteonecrosis is a disease in which bone cells dies or bones collapse due to lack of blood flow to the bones. It is also known as avascular necrosis, aseptic necrosis or ischemic necrosis. Osteonecrosis is most commonly developed in hip bone (femur) or knees, while less often in shoulder, wrist, ankle, hands, and feet. It can cause mild to severe pain and may lead to micro-fracture. Osteonecrosis can be diagnosed by using X-ray, CT scan, MRI, bone scan, and functional bone tests. Osteonecrosis treatment targets symptoms and reduces pain via medication or surgery in extreme cases. According to the National Organization for Rare Disorders, osteonecrosis is one of the rare diseases, where less than 1 in 2000 are only affected by this disorder. In 2017, Bone Therapeutics reported that around 170,000 patients were suffering from osteonecrosis in Europe, the U.S., and Japan. The advancements in treatment technologies and gene therapy and stem cell based osteonecrosis treatment are expected to propel growth of the osteonecrosis treatment market.

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Osteonecrosis Treatment Market Driver

Advanced Therapy Medicinal Products (ATMPs) to boost osteonecrosis treatment market. Advanced Therapy Medicinal Products is a class of innovative therapies that comprises of gene therapy, somatic cell therapy, and tissue-engineered products, which is expected to drive growth of osteonecrosis treatment market. Osteonecrosis usually affects young population and this significantly will contributes towards growth of osteonecrosis treatment market. For instance, according to the American College of Rheumatology, in 2017, around 10,000 to 20,000 people in the U.S. who suffered from osteonecrosis were between the ages of 20 and 50. Currently, the osteonecrosis non-surgical treatment (medication) is symptomatic treatment that targets the symptoms and try to cure the disease. Hence, emerging players in the field of bone disease treatment is gaining momentum by introducing gene regulation approach. Key players like Enzo Biochem, Inc. and Bone Therapeutics are aiming gene regulation and cell-based product treatment, which is expected to augment growth of osteonecrosis treatment market. For instance, PREOB manufactured by Bone Therapeutics, a cell based medicinal product derived from autologous bone marrow stem cells has been approved in the U.S., however, it is currently in phase III in Europe.

In spite of being a rare disease, osteonecrosis treatment market is expected to propel due to the prevalence of causative agent for osteonecrosis. Side effects of various medicines taken during cancer, HIV/AIDS, osteoarthritis, osteoporosis or blood disorders or medical treatment such as chemotherapy, radiation therapy, high-dosage of steroids or organ transplants may increases the chances of having osteonecrosis. Furthermore, few interventions such as Stanford Universitys sponsored project aims at evaluation of osteonecrosis before and after decompression surgery with ferumoxytol-enhanced MRI, which improve detection and allow to track transplanted bone marrow cells. This intervention is in phase 4 clinical trial and is expected to complete the project over the forecast period, which in turn will propel growth of the osteonecrosis treatment market.

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Osteonecrosis Treatment Market Regional Analysis

On the basis of region, the global osteonecrosis treatment market is segmented into North America, Latin America, Europe, Asia Pacific, Middle East, and Africa. North America is expected to hold a dominant position in the global osteonecrosis treatment market over the forecast period due to increasing prevalence pool of osteonecrosis, prior Food and Drug Administration (FDA) approval for the new treatments in this region. For instance, according to NCBI 2015, it is estimated that around 20,000 to 30,000 new patients are diagnosed with osteonecrosis annually in the U.S. Asia Pacific is also expected to witness faster growth in the global osteonecrosis treatment market over the forecast period, owing to increase in development activities by various key players in this region. For instance, Bone Therapeutics and Asahi Kasei Corporation signed a license agreement in 2017 for the development and commercialization of PREOB in Japan.

Osteonecrosis Treatment Market Competitor

Major players involved in osteonecrosis treatment market include Bone Therapeutics, Enzo Biochem Inc., and K-Stemcell Co Ltd. Hospitals, clinics, universities, and institutes are other major participants in the osteonecrosis treatment market.

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Osteonecrosis Treatment Market Benefit and Volume with Status and Prospect to 2026 - Crypto Journal

US Nobel laureates tell us what they think about cancer research, moonshots, the dark side, funding, meritocracy, herd mentality, Trump, and joy – The…

publication date: Nov. 15, 2019

William G. Kaelin Jr.

Sidney Farber Professor of Medicine,

Dana-Farber Cancer Institute, Brigham & Womens Hospital, Harvard Medical School

Gregg L. Semenza

Professor of genetic medicine,

Director of the Vascular Program, Institute for Cell Engineering, Johns Hopkins Medicine

William Kaelin and Gregg Semenza have a message for young scientists: do science for its own sakeand enjoy it.

What young trainees have to understand is that, at least for those of us who love science, getting to do science is a prize in and of itself, Kaelin, Sidney Farber professor of medicine at Dana-Farber Cancer Institute, Brigham & Womens Hospital, and Harvard Medical School, and a Howard Hughes Medical Institute investigator, said to The Cancer Letter. If your goal in science is simply to get prizes and to get recognition, you may be doing it for the wrong reason, and youll probably, frankly, wind up being a miserable person, because theres certainly some luck involved in winning prizes.

I think you have to take some joy in the day-to-day life of a scientist and try to do science because you love it.

Kaelin and Semenzaand Sir Peter Ratcliffe, director for the Target Discovery Institute within the Nuffield Department of Medicine at Oxford Universitywere awarded the 2019 Nobel Prize in Physiology or Medicine for their discoveries of how cells sense and adapt to oxygen availability (The Cancer Letter, Oct. 11).

Its so important to have a job thats exciting, said Semenza, professor of genetic medicine, and director of the Vascular Program in the Institute for Cell Engineering at Johns Hopkins Medicine. And a lot of people in our field, they say, When are you going to retire? Never. Why would I want to retire?

Of course, the greatest luck of all is if we actually are able to take something that weve learned and have it impact public healthand thats of course our ultimate goal. We may or may not be successful, but we at least feel that what weve learned might help other scientists get to that point.

Kaelin, Semenza, and P. James Peebles, professor emeritus and Albert Einstein Professor of Science at Princeton University, a recipient of the 2019 Nobel Prize in Physics, were honored at the Swedish Embassy in Washington, D.C. Nov. 13. They will receive the prize Dec. 10 in Stockholm.

Kaelin and Semenza said they were worried about the diminution of science in Trumps Washington.

If I was a young person hearing some of the nonsense coming out of Washington, I would wonder, Well, does my government still believe in science, and truth, and data-driven decision-making? Are scientists the good guys anymore, or are we now suddenly the bad guys, because were distrustful of expertise? Kaelin said.

I worry sometimes that now weve flipped over to the dark side, where maybe some young people think, Why would I follow this path if Im hearing, at best, mixed messages from people who make very important decisions that are going to affect my life?

The appearance of a segment of society that can completely ignore facts and science, is really disturbing. Its really very disturbing, Semenza said. Certain elements of the government are fostering this attitude. I think its very dangerous and is a real threat to our society. Hopefully, that will be addressed in the next election.

Semenza and Kaelin spoke with Matthew Ong, associate editor, and Alex Carolan, a reporter, at the House of Sweden in Washington.

Alex Carolan:

What is your advice to the young scientists that you train?

Gregg L. Semenza:

Well, first of all, I tell people the life of a research scientist is fantastic. Unfortunately trainees they may too often hear their mentors complaining about difficulty getting grants, and it can all sound very negative. But scientific research is just a fantastic profession, because you get to follow your ideas and curiosity, wherever they lead. You get to exercise tremendous creativity. No one tells you what to do or how to do it. You make friends all over the world who share your passion for science.

Its fantastic, and I tell people, if you can have a job that takes advantage of something youre good at, makes you happy, and people will pay you for it, youve got it made. So many people have a job they do solely to support their family. They want to be done with it. Thats most of your lifeyour working life.

Its so important to have a job thats exciting. And a lot of people in our field, they say, When are you going to retire? Never. Why would I want to retire?

This is too much fun. Thats what trainees really need to understand what a fantastic profession it is, and how lucky we are. Of course, the greatest luck of all is if we actually are able to take something that weve learned and have it impact public healthand thats of course our ultimate goal. We may or may not be successful, but we at least feel that what weve learned might help other scientists get to that point.

Theres great satisfaction about that, too.

William G. Kaelin:

Well, one piece of advice I give them is to first of all, not pay too much attention to scientific prizes.

I think scientific prizes are obviously wonderful when they happen, but I think what young trainees have to understand is that, at least for those of us who love science, getting to do science is a prize in and of itself. Most people come to work because they have to put food on their table and a roof over their head.

I think, if youre the kind of person who enjoys science as I doI would come to work even if I didnt need the money, because most days it feels like Im playing rather than workingthen being a scientist is a gift. I think its a great privilege to come to work every day where you enjoy what you do, and its stimulating, and its fun.

I ask them to ask themselves whether they enjoy doing the science itself and whether they enjoy the small steps that you take, hopefully in succession, towards making meaningful breakthroughs and discoveries. I tell them to try to ask good questions and to be rigorous in the way they do their work and interpret their data, and to take some joy at the little successes along the way and, in particular, hopefully derive joy from understanding things that have never been understood before, because thats another prize in and of itself.

When you understand something thats never been understood before, especially when you look at the answer, and the answer strikes you as being beautiful, or elegant, or satisfying, thats a prize. And then, if youre really, really lucky and those discoveries generate new knowledge that touches patients, that again is a prize in and of itself.

I try to get them to think about doing science for the right reasons and not the wrong reasons. I warn them, if your goal in science is simply to get prizes and to get recognition, you may be doing it for the wrong reason and youll probably, frankly, wind up being a miserable person, because theres certainly some luck involved in winning prizes.

I think you have to take some joy in the day-to-day life of a scientist and try to do science because you love it. As I said, if thats already a prize and if you do your work well, youre very lucky and the stars align, you may also occasionally win prizes.

I tell them, try to get good training so they understand the mechanics of doing science, so they have a good armamentarium of techniques that theyre comfortable with, but far more important than the techniques, which you can always learn, I think is starting to develop some scientific instincts and intuition in terms of where the next great discovery might lie. Secondly, to really learn how to think clearly, critically, logically, so that you can hopefully design powerful experiments and interpret them correctly.

Matthew Ong:

Could you describe how your work has affected the understanding of cancer?


I would say that we occupy a minority position in the world of cancer research, because as you know, the prevailing paradigm is centered on somatic mutations in cancer cells, and understanding cancer progression simply as a matter of accumulation of mutations. Our focus is not on the changes in the DNA, but changes in the tumor microenvironment.

Again, the prevailing paradigm is: if its not mutated, its not important. Its not a bona fide therapeutic target. But what I would argue is that the most important targets cannot be mutated. Because when you mutate something, you lock it into a state, either on or off. And something like HIF-1 has to constantly be modulated.

Because you can go a hundred microns in a tumor, and you go from lots of oxygen to no oxygen. We know that this is really important, because cancer stem cells reside in the hypoxic niche. They can slowly divide and always give rise to another cancer stem cell, but also to a more differentiated cancer cell that can divide very rapidly, but only for a limited number of divisions.

All that cell has to do is migrate 100 microns from the hypoxic region to the well-oxygenated region around the blood vessel. It can divide like crazy. We think that most advanced cancers contain regions of intratumoral hypoxia for a reason. That is to say, its selected for. Because there are powerful selective forces and it would certainly select cancer cells to behave in a way that did not generate hypoxia.

This is really critical to the understanding of cancer pathogenesis and therapy, because all of the existing therapies are targeting dividing cells, which are well-oxygenated cells. Its the hypoxic cells, that are particularly resistant to those therapies. They survive the therapy, and those are the cells with stem cell properties.

Weve also been able to show most recently that those cells have also turned on a battery of genes that allows them to evade the immune system. These are the cells with the lethal phenotypethese are the cells that kill the patientsand there are no approved therapies targeting these cells.

And thats our mission. As I say, weve been swimming uphill for a long time. But we continue, and were more convinced than ever. Now that there is a drug in clinical trials that targets HIF-2 in kidney cancerhopefully soon well have a proof of principle. Encouraging results from a phase I trial have been published, but it only involved 50 patients. Obviously, the next 50 could be the opposite.

But its encouraging to see that. Were more convinced than ever that this is something thats really important that will actually make a difference in the treatment of advanced cancers, because, as you know, there are not many effective treatments available for advanced solid cancers.

We think that adding HIF inhibitors to existing therapies will make many of the existing therapies work better.


Well, Im a big believer in the power of genetics, including cancer genetics. We have the advantage now, of course, that in many cancers, we know the recurrent non-random mutations that contributed to those cancers.

Even as a postdoc, where I worked on retinoblastoma gene, I came to appreciate that a particularly powerful form of human cancer genetics is to use hereditary forms of cancer, because the definitive experiment, if you will, has already been done, right? Mutation in this gene does cause cancer.

That was one of the reasons why, when I started my own laboratory, I decided to work on the VHL gene, because it was pretty clear that germline mutations in the VHL gene cause specific forms of cancer and amongst those cancers was kidney cancer.

This was important to me, because back in the 80s, 90s, I would have said that many of the molecular advances and therapeutic advances were related to cancers that were interesting, but numerically not very common. It seemed to me, if we were going to make progress on cancer mortality, we had to start tackling the big bad common epithelial cancers.

Now, I will say, there was a time when people thought that solid tumors wouldnt succumb to molecular analysis, that they were just going to be too complicated, too heterogeneous, but fortunately, when I was a resident at Johns Hopkins, I went to a seminar that a young Bert Vogelstein gave, where he was showing that you could begin to study colon cancer using modern molecular techniques.

That planted another seed in my mind. Again, when the VHL gene was cloned in 1993, there was clear genetic evidence that it played an important role in certain cancers, including kidney cancer. I now believed that you could study solid tumors using modern molecular techniques. Very quickly, it was shown, as you would predict, that in sporadic non-hereditary kidney cancers, the VHL gene also plays a role.

Fast forward, I think we now know that VHL is a negative regulator of HIF and HIF controls a number of genes, some of which almost certainly contribute to kidney carcinogenesis, including VEGF. We did the necessity and sufficiency experiments to show, that at least in the laboratory, kidney cancers lacking VHL were critically dependent on HIF and, specifically, HIF-2. Even in the 90s, when we showed that VHL regulated hypoxia-inducible genes like VEGF, we started arguing to our friends in the pharmaceutical industry that if the VEGF inhibitors they were developing were going to work anywhere, they were going to work in kidney cancer.

Thats turned out to be true. I think there are about seven approved VEGF inhibitors for the treatment of kidney cancer. Of course, theyre helpful in some other cancers as well, but I think their biggest benefit amongst the solid tumors is probably kidney cancer.

Its been very gratifying to work with Peloton Therapeutics, which was recently acquired by Merck, thats developing direct inhibitors of HIF-2, because I think you could argue that going after the master regulator would be more efficient than tackling any single downstream target of HIF-2. The HIF-2 inhibitor looks very promising, based on the phase II data. Its about to undergo phase III testing. At least Merck thought so too, because they purchased Peloton; right?

Less appreciated is the fact that, to their credit, Peloton also agreed to treat 51 patients with VHL disease who have never been treated before with any form of cancer medication. These are patients who have multiple small tumors. Because they have VHL disease, theyre often put in surveillance programs to try to avoid doing multiple surgeries, and so, theyll be put in careful surveillance programs. Fifty-one of these patients have now been treated with the HIF-2 inhibitor.

I dont think the data had been publicly presented yet, but if you look at the Facebook posts of the patients on the trial, it looks like theyre responding. This is extremely gratifying.


Can we talk about science policy for a moment? What do you think about the current state of federal funding for cancer? Whats good? Whats bad?


Well, Id say whats good is that in terms of a piece of the pie (meaning total federal research funding), its a pretty big piece.

Whats bad is that we could make a lot more progress if there was more. From a public health point of view, this is obviously a wise investment. Even from an economic point of view, its a wise investment. We know that these innovations will lead to new companies and new products.

We hope that if we can effectively treat people with cancer, that cancer care is going to be much less expensive. Because on the back end of that, theres a whole lot of expense.

If we can prevent patients from getting to that stage, thats going to have a really big impact on public health and how we utilize limited resources to take care of people with chronic diseases, as the population ages.

Thats one benefit of the Nobel Prize. It provides an illustration to the public of how basic science can lead to new treatments, how that process works, and why they should support it.

Because ultimately, its taxpayer dollars that are funding NIH, NSF and other granting agencies of the federal government that are the major sources of research funding for scientists here in the U.S.

My own opinion is that the focus should be on basic research funding, because we dont really know what discoveries will get us to new treatments for cancer. Likening cancer research to the Apollo Moon mission, I dont think is helpful.

We already had one war on cancer in the 70s, and now were just repeating this same rubric. I dont think its helpful.


I havent looked at the numbers recently, but it has certainly felt like its been flat for too long. I think that creates a lot of issues, because for example, I think study sections are pretty good at saying, This grant is in the bottom 50% versus the top 50%, and arguably, theyre okay at saying, This grant is in the top 20 or 30% versus in the bottom 70 or 80%.

I think where the system breaks down is when theyre asked to say, Is this an 8th percentile grant versus a 14th percentile grant? Because one is going to get funded and one isnt. I think that just puts too much stress on the peer review system and it also tells you that theres some very good grants that arent being funded. I think thats problem one.

I think problem two is, I would say, the secret sauce in American biomedical research for most of my life was saying, Lets let the private sector, meaning mostly the companies, fund the late-stage research and the applied research, what some people call the translational research, but lets let the public sector, largely the federal government, fund the early-stage basic sciencethe fundamental science, the mechanistic science that gets done early, because companies dont typically invest in that early stage work, because the timelines and deliverables are too unpredictable for them, and yet, over and over, they will say thats the one thing they count on us to do in academia, right?

They rely on that information, and often thats where the truly transformative discoveries come in the first place. I think having the public sector, again, largely the government, pump priming and investing in that early-stage work and letting the private sector be the harvesters or the beneficiaries of that new knowledge, that was a very powerful and useful formula.

But I think now, unfortunately, more and more investigators feel pressured to justify their work in terms of its potential clinical utility or impactfulness. I think thats sort of distorted the whole ecosystem.

Again, I tell people the next big breakthrough for pancreatic cancer might come from someone studying pancreatic cancer, but its just as likely, if not more likely, to come from someone either studying another cancer altogether or, frankly, someone who didnt even think they were studying cancer, but uncovered some new basic mechanism, maybe in some model organism, just trying to learn a new piece of biology, who could then come back and say, This was the key piece of the puzzle we were looking for, for say, pancreatic cancer.

So, I think its very shortsighted to hold people to, What are you going to do with this knowledge in the next five years? I think we have to maintain a longer view and understand that real progress comes by generating new knowledge, and you have to have scientists be free to follow their curiosity, and follow the road where it takes them, rather than just putting blinders on them and saying, Well, you promised us in year five you were going to be working on this, and this was going to be your deliverable. You know, thats the language of engineering. Thats not the language of science.


So, where are we in cancer research, and what are the opportunities that scientists and lawmakers should be capitalizing on at this point?


Of course, the first step is prevention. There should be more funding for prevention, because thats really where we can have tremendous impact. Stop smoking, prevent obesity, encourage exercise. These are major factors that impact on the likelihood of developing cancer. Prevention is critical.

Early detection is another revolution thats going to have a big impact, because if we can identify tumors when theyre still contained within the organ of origin, the chances of cure are much greater. Now, with powerful sequencing, its become possible to identify a few cells in the blood that carry telltale mutations that say theres a cancer growing in a particular organ.

Thats another critical area. There are companies now that are developing these new tests. Again, those need to be tested in a strict clinical way, and we have to be very careful about things being marketed that are not fact-basedmaking promises that that they cant fulfill.

And then, as I mentioned, funding basic research is critical, but also funding translational work, because ironically, when youre at the point when you think you know enough to develop a drug, it can be very hard to get research funding from the NIH because its not hypothesis driven. This idea that everything has to be hypothesis-driven is also not helpful.


So, I think we heard about the Big Bang. I really think the big bang in cancer was in 2000, when we had the first draft of the human genome. Because, of course, cancer to a first approximation is a disease of accumulated mutations in specific genes, and we didnt, until 2000, have the complete list of genes and their sequences.

So, its truly remarkable, all the things that were discovered before the year 2000, but as you know, things have really accelerated since 2000, because first, the human genome became available, and secondly, there was a precipitous drop in the cost of sequencing.

So, now, I think, increasingly, we know the mutations that are responsible for specific forms of cancer. And as you know, theres a first generation of targeted agents emerging that are based on those genetic mutations.

But I think where we must get now are, first of all, we have to get to combination therapy. I mean, this is axiomatic, but I think if were going to deal with the resistance problem, we have to stop using targeted agents as single agents. We have to get to combining drugs that have distinct mechanisms of action, and the hope is, because they have distinct mechanisms of action, they wont be cross-resistant with one another and their toxicities will not overlap in a prohibitive way. So, we have to get, I think, to combination therapy.

And secondly, there are a lot of examples of cancer-causing mutations, where the protein product of those mutations is considered undruggable. So, we either have to come up with new ways to drug the undruggable, or we at least have to figure out the collateral vulnerabilities that are created by those mutations.

In some cases, we may not be able to directly target the genetic mutation, but at least we can target the vulnerabilities that are created by virtue of those mutations. And so, one paradigm for this, of course, is so-called synthetic lethality, where maybe mutation A makes you hyper-dependent on gene B. And so maybe the gene A mutations not druggable, but you can at least develop a drug against gene B. So, I think this is one area for the future.

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US Nobel laureates tell us what they think about cancer research, moonshots, the dark side, funding, meritocracy, herd mentality, Trump, and joy - The...

Youngstown State, IBM to offer high-tech training in the Mahoning Valley – Crain’s Cleveland Business

Researchers at Case Western Reserve University have won a number of grants this fall. Here's a look at some of the recently announced ones and the work those grants will be funding, all with links to full stories on the university's The Daily site.

1. First up, a new five-year, $2.2 million grant from Lubrizol Corp. will support STEM scholarships, internships, co-ops and joint research, a post said. The funding will also support programs focused on student research and women in science and engineering.

The joint research between CWRU and Lubrizol will focus on energy, human health, materials and sustainability.

"We are always interested in finding ways that the Case Western Reserve community can engage more fully with the industrial sector," university provost Ben Vinson III said in the post. "It is, along with our community and government partners, critical to the development of our students, our research endeavors and our innovation pathway. Working with the university's office of corporate relations, we are developing new strategies to deepen our industry collaborations that will include investment from our corporate partners to support programmatic areas across campus."

2. A five-year, $1.25 million grant will help the university better train developmental psychologists and speech language pathologists. The grant is from the U.S. Department of Education.

"Many children need extra help in their educational journey. Teachers cannot do it alone," Elizabeth Short, a professor in the Department of Psychological Sciences, said in a post. "Training professionals to provide supports is of paramount importance they are on the frontlines, providing the necessary help to optimize the development of children."

The project will emphasize the importance of working in teams, and the grant brings in both branches of the university's Department of Psychological Sciences: psychology and communication sciences.

3. A $425,000 grant from the U.S. Department of Justice will help Case Western Reserve's Begun Center for Violence Prevention Research and Education work with the city of Akron to analyze information in untested sexual assault kits. Identifying patterns of offender behavior could help the Akron Police Department respond to such assaults, a post said.

The team, led by research assistant professor Rachel Lovell, has also received two Department of Justice awards equaling $528,000 to continue similar work in Cuyahoga County.

4. CWRU and MetroHealth Medical Center researchers recently received more than $800,000 from the U.S. Department of Defense to study the experiences and needs of people with spinal cord injuries. Other partners include the United Spinal Association Northeast Ohio Chapter and the Louis Stokes Cleveland VA Medical Center.

The three-year project will focus on the first year of recovery, and researchers will interview veterans and civilians, as well as their caregivers, a post said.

5. Finally, researchers at the Case Western Reserve School of Medicine have received a grant of almost $700,000 through the National Institutes of Health Somatic Cell Genome Editing program. If researchers continue to meet NIH milestones, the grant amount could increase to $2.78 million, a post said.

The researchers are looking to develop strategies to deliver genome editing complexes directly to stem cells, which could change how certain diseases are treated.

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Youngstown State, IBM to offer high-tech training in the Mahoning Valley - Crain's Cleveland Business

A short guide to regulation for disruptive technologies – Lexology


Regulation, by necessity, introduces rigidity to otherwise flexible processes. Done proportionately, this can be an efficient societal device for preventing harm. At the same time, inherent regulatory rigidity creates particular challenges when the nature of the regulatory target changes quickly or unexpectedly.

Disruptive technologies in life sciences - a very dynamic field of activity are a good example of this. Disruptive technologies challenge the way a sector operates, and it is self-evident that (in most cases) this will also have an impact on the relevant normative framework. This effect is most visible in areas which have a direct impact on human life and wellbeing, as these areas are tightly (and often, rather specifically) regulated, and a failure to control a technology appropriately may lead to undesirable outcomes.

The dual purposes of preventing harm through proportionate regulation and maintaining trust in innovation mean that it is all the more important to ensure that regulation is adequately responsive and flexible to react to a disruptive technology. This can be a difficult line to tread, particularly in fields where research and development is also morally or ethically contentious.

We will illustrate the context and challenge of regulating disruptive technologies by discussing two specific case studies: artificial intelligence, and cell and gene therapy. In both cases, we suggest that the current regulatory framework in the UK strikes an appropriate balance between precaution and freedom of research, allowing for innovation subject to strict controls and licensing frameworks. There are, however, numerous challenges which need to be considered and addressed as these technologies advance. Regulators, policy makers and innovators working in this sector must continue to work together to ensure that responsible science is allowed to flourish.

Artificial intelligence

The science of making machines do things that would require intelligence if done by people (Definition of artificial intelligence from the Proposal for the Dartmouth Summer Research Project on Artificial Intelligence, 1955.)

Artificial intelligence (AI) technologies hold the potential to significantly improve health and care, providing faster and more accurate diagnosis, speedier treatments, and facilitating medical breakthroughs through drug discovery.

This is particularly the case in contexts where the pattern-recognition strengths of AI can be deployed to their fullest potential. Tasks such as the correct identification of tumour cells, recognition of areas of concern in medical imaging, and the processing of large amounts of genomic data can be carried out with much greater speed and accuracy by algorithms that learn from previous datasets, and develop their own datasets from which to learn from in the future. The ability to check a patients image or test result against all other available and comparable datasets is, at first glance, far superior to a clinicians ability to make an assessment on the basis of his or her experience.

At the same time, this does give rise to risk. For example, there is an inherent (and proven) risk that an algorithm which learns on the basis of historic human-generated data also takes on the biases that human decision-making has inevitably introduced. So how does regulation play a part in addressing this risk?

The first point to make is that no one body is solely responsible for regulating the adoption of AI technologies in the UK healthcare sector. A number of different regulatory bodies have a remit to oversee aspects of AI, including the Medicines and Healthcare products Regulatory Agency (MHRA) and the Information Commissioners Office (ICO). In addition, there are nonregulatory bodies which also play an important role, including the National Institute for Health and Care Excellence (NICE) and NHSX. However, no one institution has overall responsibility for policing, for example, the prevention of bias in AI algorithms. The most effective way of addressing this risk at present is to avoid exclusively automated decision-making so that the use of AI technologies in the clinical setting will focus instead on assisted decision-making and triage. The application of this approach will come down to individual healthcare payors and providers: in the absence of any direct regulation, it is left to them to decide how best to mitigate risk, and whether and if so how to apply nonbinding codes of conduct, such as the Department of Health and Social Cares code of conduct for data-driven technologies which seek to address the risk.

Reliance on nonbinding codes of conduct as a substitute for regulation may not be ideal and can result in a lack of certainty. Equally, overlapping codes, rules and regulations also pose a risk, for example, as to how NICEs evidence standards framework for digital health technologies interacts with MHRA regulations concerning software as a medical device in relation to clinical evidence. The risk is lack of clarity; the mitigation is raising awareness.

Another challenge arises where regulation designed for a specific purpose is used for a new purpose, for example the application of MHRA regulations designed for traditional medical devices to software incorporating algorithms. A recent state of the nation survey on the use of AI in health and care revealed that half of all software developers were not intending to seek CE mark classification, with the most commonly cited reason being that they did not believe the medical device classification was applicable. It is essential that the sector raises awareness of these requirements, albeit that they are complex and sometimes impenetrable.

One significant area of concern is how existing laws relating to negligence, liability and insurance apply to the clinical use of AI whether in assisting decision-making about a patients treatment, or in the operation of medical devices. Currently, claims are almost always brought against the treating clinician or healthcare provider, but for a clinician using big data analysis as well as his or her own experience, where does the division of responsibility lie? If a patient is injured as a result of a malfunction in an AI-driven device, does liability lie with the manufacturer of the device, the programmer who wrote the code which operates the device, the clinical team, the hospital or all of the above? It remains to be seen whether this will give rise to novel constellations of liability, such as an increase in manufacturers liability or a change in statutory and wider insurance requirements.

One of the major areas of opportunity for AI-based technologies is biomedical research where the strengths of speed and range have huge potential. The extrapolation of the potential of certain compounds against huge databases of similar compounds is commercially powerful. The ability to quickly check clinical trial design against public registries of published results to avoid unnecessary duplication of human-based experimentation is ethically desirable. But as innovators seek to improve drug discovery using AI, it will be important to continue to keep under review laws relating to intellectual property and how they apply to AI-based technologies.

Cell and gene therapy

The area of cell and gene therapy is of particular significance, and great potential, in regenerative medicine. It has seen a decade-long genesis since its inception, and it does not immediately strike one as a field that meets the definition of a disruptive technology. At the same time, however, it provides a good illustration of how a technology may mature for a long time, or be repurposed in an unexpected way, before it becomes disruptive.

The field has come a long way since the first systematic trials in 1989, and by now, there are 17 FDA-approved cell and gene therapy products. Over and beyond technical questions of the safety of the vectors used for the manipulation of cells, there are few remaining ethical and legal issues in relation to somatic cell gene therapy for particularly debilitating conditions (i.e. where the manipulation does not lead to heritable genetic characteristics).

From a regulatory and ethical perspective, however, cell and gene therapy becomes more complex where germline gene therapy is used. The modification of the human germline is subject to significant debate and, in some jurisdictions, strongly prohibitive regulation. The advent of disruptive technologies such as CRISPR/Cas9 prime editing techniques, with their associated precision and purported safety, have already reignited the debate around the prohibition of germline manipulation, with some commentators calling for a relaxation of the regulation while others demand either a global ban or at least a moratorium.

Although the United Kingdom has a reputation of being a liberal jurisdiction for research, it is in fact very tightly regulated and only potentially permissive. UK law reects a compromise: we permit research (including research involving germline gene editing), but we subject such research to strict scrutiny, licensing and oversight, and we criminalise unlicensed research. That being said, the legislation is drafted in such a way as to facilitate a broad variety of research, including (again, potentially) the introduction of novel techniques, and few procedures are prohibited. Overall, this framework helps allay public and political concern about what is often controversial research and provides a degree of protection for researchers operating under a licence, facilitating innovation. Such a robust framework is particularly valuable when it comes to considering how best to address the clinical application of germline genome modication. In circumstances where UK law is comprehensive and clear in its application to gene editing, there is no merit or purpose in a moratorium or further restriction on the use of this technology as some have demanded.

Concluding remarks

The UK has a mature and robust regulatory framework governing research and development in life sciences. We have a successful history in regulating numerous disruptive and controversial new technologies, such as stem cell research, the creation of human-animal hybrids, the clinical use of preimplantation genetics, and mitochondrial donation all testaments to the strength of this framework and its capacity to adapt to accommodate new technologies. This success, however, has been built upon a vital foundation of open and accessible dialogue between innovators, parliamentarians, policy makers and the public, and it is to be hoped that a similar transparency will be maintained in the future. Such dialogue will also ensure that if there are gaps or restrictions in regulation that need to be addressed to avoid stifling innovation, these can be pre-empted.

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A short guide to regulation for disruptive technologies - Lexology

Cereal rust could lead to new wheat threat – Farm Weekly

SCIENTISTS have uncovered the origins of the world's deadliest strain of cereal rust disease which threatens global food security.

Researchers from Australia's national science agency, CSIRO, together with partners in the United States and South Africa, have solved a 20-year-old mystery with findings published last week in Nature Communications.

Their work shows that the devastating Ug99 strain of the wheat stem rust fungus (named for its discovery and naming in Uganda in 1999) was created when different rust strains simply fused to create a new hybrid strain.

This process is called somatic hybridisation and enables the fungi to merge their cells together and exchange genetic material without going through the complex sexual reproduction cycle.

The study found half of Ug99's genetic material came from a strain that has been in southern Africa for more than 100 years and also occurs in Australia.

The discovery shows that other crop-destroying rust strains could hybridise in other parts of the world - and scientists found evidence of this in their study.

It also means Ug99 could once again exchange genetic material with different pathogen strains to create a whole new enemy.

While it was proposed that rust strains could hybridise based on laboratory studies in the 1960s, this new research provides the first clear molecular evidence that this process generates new strains in nature.

Rusts are a common fungal disease of plants.

Globally they destroy more than $1 billion worth of crops each year.

Australian crops have largely been protected for the past 60 years by the breeding of rust-resistant crop varieties.

Group leader at CSIRO Dr Melania Figueroa said Ug99 was considered one of the most threatening of all rusts as it has managed to overcome many of the stem rust resistance genes used in wheat varieties and has evolved many variants.

"While outbreaks of Ug99 have so far been restricted to Africa and the Middle East, it has been estimated that a nationwide outbreak here could cost Australia up to $500 million in lost production and fungicide use in the first year," Dr Figueroa said.

"There is some good news, however, as the more you know your enemy, the more equipped you are to fight against it.

"Knowing how these pathogens come about means we can better predict how they are likely to change in the future and better determine which resistance genes can be bred into wheat varieties to give long-lasting protection."

Earlier this year, CSIRO worked with the University of Minnesota and the 2Blades Foundation to achieve good results in wheat resistance by stacking five resistance genes into the one wheat plant to combat wheat stem rust.

This latest research is the result of a collaboration between scientists from CSIRO, the University of Minnesota, University of the Free State and Australian National University.

The breakthrough came as Dr Figueroa's group was sequencing Ug99 (then at the University of Minnesota) and at the same time a CSIRO team led by Dr Peter Dodds was sequencing Pgt 21 in Australia.

Pgt21 is a rust strain that was first seen in South Africa in the 1920s and believed to have been carried to Australia in the 1950s by wind currents.

When the two groups compared results, they found the two pathogens share an almost identical nucleus and therefore half of their DNA.

"This discovery will make it possible to develop better methods to screen for varieties with strong resistance to disease," Dr Figueroa said.

"There was an element of serendipity at play in this work.

"We never expected that Ug99 and an Australian isolate might be related but only through a multi-continental collaboration was it possible to make the connections needed to achieve this discovery."

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Cereal rust could lead to new wheat threat - Farm Weekly

Tooth Regeneration Industry 2019 Research on Market Sales, Revenue, Top Companies and Future Development – TheFinanceTime

Latest Report Available at Analytical Research Cognizance, Tooth Regeneration Market provides pin-point analysis for changing competitive dynamics and a forward looking perspective on different factors driving or restraining industry growth.

Tooth regeneration is a stem cell based regenerative medicine procedure in the field of tissue engineering and stem cell biology to replace damaged or lost teeth by redrawing them from autologous stem cells. As a source of the new bioengineered teeth somatic stem cells are collected and reprogrammed to induced pluripotent stem cells which can be placed in the dental lamina directly or placed in a reabsorb able biopolymer in the shape of the new tooth.

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Scope of the Report:The global Tooth Regeneration market is valued at xx million USD in 2018 and is expected to reach xx million USD by the end of 2024, growing at a CAGR of xx% between 2019 and 2024.The Asia-Pacific will occupy for more market share in following years, especially in China, also fast growing India and Southeast Asia regions.North America, especially The United States, will still play an important role which cannot be ignored. Any changes from United States might affect the development trend of Tooth Regeneration.Europe also play important roles in global market, with market size of xx million USD in 2019 and will be xx million USD in 2024, with a CAGR of xx%.This report studies the Tooth Regeneration market status and outlook of Global and major regions, from angles of players, countries, product types and end industries; this report analyzes the top players in global market, and splits the Tooth Regeneration market by product type and applications/end industries.

Market Segment by Companies, this report coversUnileverStraumannDentsply Sirona3MZimmer BiometOcata TherapeuticsIntegra LifeSciencesDatum Dental

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Market Segment by Regions, regional analysis coversNorth America (United States, Canada and Mexico)Europe (Germany, France, UK, Russia and Italy)Asia-Pacific (China, Japan, Korea, India and Southeast Asia)South America (Brazil, Argentina, Colombia)Middle East and Africa (Saudi Arabia, UAE, Egypt, Nigeria and South Africa)

Market Segment by Type, coversDentinDental PulpTooth Enamel

Market Segment by Applications, can be divided intoHospitalsDental ClinicsOthers

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Some Points from TOC:

Chapter One: Tooth Regeneration Market Overview

1.1 Product Overview and Scope of Tooth Regeneration

1.2 Classification of Tooth Regeneration by Types

1.2.1 Global Tooth Regeneration Revenue Comparison by Types (2019-2024)

1.2.2 Global Tooth Regeneration Revenue Market Share by Types in 2018

1.2.3 Dentin

1.2.4 Dental Pulp

1.2.5 Tooth Enamel

1.3 Global Tooth Regeneration Market by Application

1.3.1 Global Tooth Regeneration Market Size and Market Share Comparison by Applications (2014-2024)

1.3.2 Hospitals

1.3.3 Dental Clinics

1.3.4 Others

1.4 Global Tooth Regeneration Market by Regions

1.4.1 Global Tooth Regeneration Market Size (Million USD) Comparison by Regions (2014-2024)

1.4.1 North America (USA, Canada and Mexico) Tooth Regeneration Status and Prospect (2014-2024)

Chapter Two: Manufacturers Profiles

2.1 Unilever

2.1.1 Business Overview

2.1.2 Tooth Regeneration Type and Applications Product A Product B

2.1.3 Unilever Tooth Regeneration Revenue, Gross Margin and Market Share (2017-2018)

2.2 Straumann

2.2.1 Business Overview

2.2.2 Tooth Regeneration Type and Applications Product A Product B

2.2.3 Straumann Tooth Regeneration Revenue, Gross Margin and Market Share (2017-2018)

2.3 Dentsply Sirona

2.3.1 Business Overview

2.3.2 Tooth Regeneration Type and Applications

Chapter Three: Global Tooth Regeneration Market Competition, by Players

3.1 Global Tooth Regeneration Revenue and Share by Players (2014-2019)

3.2 Market Concentration Rate

3.2.1 Top 5 Tooth Regeneration Players Market Share

3.2.2 Top 10 Tooth Regeneration Players Market Share

3.3 Market Competition Trend

Chapter Four: Global Tooth Regeneration Market Size by Regions

4.1 Global Tooth Regeneration Revenue and Market Share by Regions

4.2 North America Tooth Regeneration Revenue and Growth Rate (2014-2019)

4.3 Europe Tooth Regeneration Revenue and Growth Rate (2014-2019)

4.4 Asia-Pacific Tooth Regeneration Revenue and Growth Rate (2014-2019)

4.5 South America Tooth Regeneration Revenue and Growth Rate (2014-2019)

4.6 Middle East and Africa Tooth Regeneration Revenue and Growth Rate (2014-2019)

Chapter Five: North America Tooth Regeneration Revenue by Countries

5.1 North America Tooth Regeneration Revenue by Countries (2014-2019)

5.2 USA Tooth Regeneration Revenue and Growth Rate (2014-2019)

5.3 Canada Tooth Regeneration Revenue and Growth Rate (2014-2019)

5.4 Mexico Tooth Regeneration Revenue and Growth Rate (2014-2019)

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Stem Cell Therapy Market : Opportunities and Challenges – MENAFN.COM

(MENAFN - GetNews) Emerging countries (such as South Korea, India, and China) are supporting their respective domestic stem cell industry through various regulatory reforms that support commercialization and research activities related to stem cell therapy.

The globalstem cell therapy marketis estimated to reach USD 145.8 million by 2021, growing at a CAGR of 11.0% during the forecast period

Supportive regulations across developing countries

Emerging countries (such as South Korea, India, and China) are supporting their respective domestic stem cell industry through various regulatory reforms that support commercialization and research activities related to stem cell therapy.

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Due to flexible policies regarding stem cell research, South Korea has made significant advancement in this industry. The less stringent and relatively fast approval process of clinical trials has enabled South Korean manufacturers to commercialize their stem cell therapy products. Researchers in the country have successfully developed stem cells that are a perfect genetic match to patients of all races, genders, and so forth. Due to progress in therapeutic cloning, they can efficiently produce stem cells tailored to the individual and with a low risk of immunological rejection.

The medical tourism industry in Mexico is growing rapidly. The country has been a popular destination for stem cell treatments. A significant number of Americans and Canadians travel to Mexico to avail lower-priced treatments that are unavailable in their countries due to regulatory policies. Many private companies in Mexico offer stem cell treatments. However, these treatments are not approved by the Federal Commission for the Protection against Sanitary Risk (COFEPRIS Mexican government regulatory authority for the manufacture and commercialization of drugs and medical products); they are also not considered as registered clinical trials. Yet, many of these treatments are offered as proven and effective therapies. COFEPRIS does not have standards or guidelines for evaluating, authorizing, and monitoring research and therapeutic activities involving human tissues and cells.

Many other developing countries have allowed the use of ESCs for R & D purpose.

n In March 2005, the Brazilian government passed a legislation that allows the use of in vitro fertilized embryos that have been frozen for more than three years.

n In India, it is permissible to establish new human ESC lines from spare embryos by obtaining approval from regulatory authorities.

n In China, regulatory guidelines (established in 2013) permit the use of embryos for stem cell research only if the embryos are obtained from in vitro fertilization (IVF); fetal cells from abortions; somatic cell nuclear transfer (SCNT); or voluntarily donated germ line cells.

Such supportive regulations across developing countries will provide significant growth opportunities for local as well as international players in the stem cell therapy market.

Technical limitations related to production scale-up

Due to technical difficulties faced at various manufacturing stages such as stem cell identification, isolation, storage, and preservation, it becomes difficult for a manufacturer to scale-up the production. Limited manufacturing capability for production scale-up is expected to hinder the large-scale manufacturing of stem cells. The scaling-up process is also affected due to the development process adopted during the research phase. Typically, less attention is paid to the scalability of production and the cost associated with it. Different stem cells require different environments for proliferation and differentiation, potentially including mechanical stimuli or flow conditions, variable gas tensions, chemical gradients, 3D frameworks, and supporting-cell paracrine signaling. These factors depend on the source and type of stem cell.

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There is a requirement for a basic stem-cell manufacturing solution which supports the production of a variety of stem cells and provides optimal environment. With current technological advancements, it is possible that a manufacturing solution can be programmed to control some factors; however, it is very difficult to provide a production system that fits all solutions. There are also concerns regarding the possibility of cells to adapt to different, more large-scale culture conditions while retaining safety and therapeutic efficacy. These limitations form a key challenge in the stem cell therapy market.

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Stem Cell Therapy Market : Opportunities and Challenges - MENAFN.COM

Cell Therapy Market Forecast to 2025 | Analysis by Regions, Type, Application, and Top Key Players like Dendreon, Mesoblast, Vericel, Antibe…

Cell therapy involves the administration of somatic cell preparations for the treatment of diseases or traumatic damages. The objective of this study is to provide long term treatment through a single injection of therapeutic cells. Growing aging patient population, the rise in cell therapy transplantations globally, and rising disease awareness drive the growth of the global cell therapy market. However, stringent regulatory policies may hamper the growth of the market.

The Global Cell Therapy Market is estimated to grow at a CAGR of +13% during the forecast period.

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Rising funding from government as well as private organizations to support cell therapy clinical trials, introduction of effective guidelines for cell therapy manufacturing, and proven effectiveness of products are some of the primary growth stimulants for the market. In addition, declining prices of stem cell therapies are leading to increased inclination of buyers towards cell therapy.

Top Companies Profiled in this Report includes, Dendreon, Mesoblast, Vericel, Antibe Therapeutics, Astellas Pharma, Bellicum Pharmaceuticals, BIOCELLULAR THERAPIES, BioTissue Technologies, BrainStorm Cell Therapeutics, CRC for Cell Therapy Manufacturing, Living Cell Technologies, Opexa Therapeutics, Synergistic Technologies, Tessa Therapeutics, VistaGen Therapeutics, SDRMI and Xcelthera

Cell Therapy Market Segmentation by Regions/Countries:

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Global Cell Therapy Market Research Report

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Detection of Latent HSCs Fated to Progress to Blast Phase in Myelofibrosis Patients Several Years Before Blast Transformation – DocWire News

Blast phase (BP) fated clones often appear several years before blast transformation and can be traced back to hematopoietic stem cells (HSCs), according to the findings of a study presented at the 12th International Congress on Myeloproliferative Neoplasms.

Researchers studied nine myelofibrosis (MF) patients who progressed to BP and had previously aggregated chronic phase (CP) and BP samples, with an average time interval between collections between 1.5 to 6.5 years.

The time interval between CP and BP collections ranged between 1.5 to 6.6 years. In four patients, additional CP samples were available from intervening time points. Whole genome sequencing (WGS) of leukemic blasts, the MPN clone, and germline control (T-cells/buccal DNA) were performed to identify somatic mutations. The researchers detected mutations at BP in 21 ML genes with an average of 5.3 (range 2-8) genes mutated per patient. Genes that were mutated in 2 or more BP samples included SRSF2 (n=5), ASXL1 (n=4), TET2 (n=4), IDH1/2 (n=4), RUNX1 (n=4), NRAS (n=4), KRAS (n=2), U2AF1 (n=2), PHF6 (n=2), and STAG2 (n=2).

The results showed that BP-specific ML gene mutations could be detected at low frequencies in one or more cell populations several years before BP diagnosis. The study authors wrote that, importantly, these low frequency mutations were detected within the HSC population from several patients, indicating that BP-fated clones derived from an HSC. This finding is being verified by targeted sequencing of additional BP-specific mutations identified by WGS (average of 300 variants per patient, range 37 to 659).

The authors added that: Identification of BP-fated clones that are latent strongly suggests that mechanisms beyond the acquisition of somatic mutations in ML genes are necessary to effectively promote full leukemic transformation.

Ho J, et al. Detection of Latent HSCs Fated to Progress to Blast Phase in Myelofibrosis Patients Several Years Before Blast Transformation. Presented at the 12th International Congress on Myeloproliferative Neoplasms; October 24-25, 2019; New York, NY.

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Detection of Latent HSCs Fated to Progress to Blast Phase in Myelofibrosis Patients Several Years Before Blast Transformation - DocWire News

Stem cell therapy is for animals too – SciTech Europa

Stem cell therapy for animals has seen breakthroughs

Stem cell therapy is increasingly becoming a more mainstream form of medicine. Usually applied to humans, the use of this regenerative treatment is now also being extended to animals including cats and dogs. Regenerative medicine, particularly stem cell treatment has seen many advancements in recent years with some groundbreaking studies coming to light.

Taking the cells from bone marrow, umbilical cords, blood or fat, stem cells can grow to become any kind of cell and the treatment has seen many successes in animals. The regenerative therapy has been useful particularly for treatment of spinal cord and bone injuries as well as problems with tendons, ligaments and joints.

Expanded Potential Stem Cells (EPSCs) have been obtained from pig embryos for the first time. The cells offer groundbreaking potential for studying embryonic development and producing transnational research in genomics and regenerative medicine, biotechnology and agriculture.

The cells have been efficiently derived from pig preimplantation embryos and a new culture medium developed in Hong Kong and Cambridge enabled researchers from the FLI to establish permanent embryonic stem cell lines. The cells have been discovered in a collaboration between research groups from the Institute of Farm Animal Genetics at the Friedrich-Loeffler-Institut (FLI) in Mariensee, Germany, the Wellcome Trust Sanger Institute in Cambridge, UK and the University of Hong Kong, Li Ka Shing Faculty of Medicine, School of Biomedical Sciences.

Embryonic stem cells (ESC) are derived from the inner cells of very early embryos, the so-called blastocysts. Embryonic stem cells are all-rounders and can develop into various cell types of the body in the culture dish. This characteristic is called pluripotency. Previous attempts to establish pluripotent embryonic stem cell lines from farm animals such as pigs or cattle have resulted in cell lines that have not really fulfilled all properties of pluripotency and were therefore called ES-like.

Dr Monika Nowak-Imialek of the FLI said: Our porcine EPSCs isolated from pig embryos are the first well-characterized cell lines worldwide. EPSCs great potential to develop into any type of cell provides important implications for developmental biology, regenerative medicine, organ transplantation, disease modelling and screening for drugs.

The stem cells can renew themselves meaning they can be kept in culture indefinitely, and also show the typical morphology and gene expression patterns of embryonic stem cells. Somatic cells have a limited lifespan, so these new stem cells are much better suited for long selection processes. It has been shown that these porcine stem cell lines can easily be modified with new genome editing techniques such as CRISPR/Cas, which is particularly interesting for the generation of porcine disease models.

The EPSCs have a high capacity to develop not only into numerous cell types of the organism, but also into extraembryonic tissue, the trophoblasts, making them very unique and lending them their name. This capacity could prove valuable for the future promising organoid technology, where organ-like small cell aggregations are grown in 3D aggregates that can be used for research into early embryo development, various disease models and testing of new drugs in petri dishes. In addition, the authors were able to show that trophoblast stem cells can be generated from their porcine stem cells, offering a unique possibility to investigate functions or diseases of the placenta in vitro.

A major hurdle to using neural stem cells derived from genetically different donors to replace damaged or destroyed tissues, such as in a spinal cord injury, has been the persistent rejection of the introduced material (cells), necessitating the use of complex drugs and techniques to suppress the hosts immune response.

Earlier this year, an international team led by scientists at University of California San Diego School of Medicine successfully grafted induced pluripotent stem cell (iPSC)-derived neural precursor cells back into the spinal cords of genetically identical adult pigs with no immunosuppression efforts. The grafted cells survived long-term, displayed differentiated functionality and caused no tumours.

The researchers also demonstrated that the same cells showed similar long-term survival in adult pigs with different genetic backgrounds after only short course use of immunosuppressive treatment once injected into injured spinal cord.

Senior author of the paper Martin Marsala, MD, professor in the Department of Anesthesiology at UC San Diego School of Medicine said: The promise of iPSCs is huge, but so too have been the challenges. In this study, weve demonstrated an alternate approach.

We took skin cells from an adult pig, an animal species with strong similarities to humans in spinal cord and central nervous system anatomy and function, reprogrammed them back to stem cells, then induced them to become neural precursor cells (NPCs), destined to become nerve cells. Because they are syngeneic genetically identical with the cell-graft recipient pig they are immunologically compatible. They grow and differentiate with no immunosuppression required.

Co-author Samuel Pfaff, PhD, professor and Howard Hughes Medical Institute Investigator at Salk Institute for Biological Studies, said: Using RNA sequencing and innovative bioinformatic methods to deconvolute the RNAs species-of-origin, the research team demonstrated that pig iPSC-derived neural precursors safely acquire the genetic characteristics of mature CNS tissue even after transplantation into rat brains.

NPCs were grafted into the spinal cords of syngeneic non-injured pigs with no immunosuppression finding that the cells survived and differentiated into neurons and supporting glial cells at all observed time points. The grafted neurons were detected functioning seven months after transplantation.

Then researchers grafted NPCs into genetically dissimilar pigs with chronic spinal cord injuries, followed by a transient four-week regimen of immunosuppression drugs again finding long-term cell survival and maturation.

Marsala continued: Our current experiments are focusing on generation and testing of clinical grade human iPSCs, which is the ultimate source of cells to be used in future clinical trials for treatment of spinal cord and central nervous system injuries in a syngeneic or allogeneic setting.

Because long-term post-grafting periods between one and two years are required to achieve a full grafted cells-induced treatment effect, the elimination of immunosuppressive treatment will substantially increase our chances in achieving more robust functional improvement in spinal trauma patients receiving iPSC-derived NPCs.

In our current clinical cell-replacement trials, immunosuppression is required to achieve the survival of allogeneic cell grafts. The elimination of immunosuppression requirement by using syngeneic cell grafts would represent a major step forward said co-author Joseph Ciacci, MD, a neurosurgeon at UC San Diego Health and professor of surgery at UC San Diego School of Medicine.

Other recent advancements include the advancement toward having a long-lasting repair caulk for blood vessels. A new method has been for generating endothelial cells, which make up the lining of blood vessels, from human induced pluripotent stem cells. When endothelial cells are surrounded by a supportive gel and implanted into mice with damaged blood vessels, they become part of the animals blood vessels, surviving for more than 10 months.

The research was carried out by stem cell researchers at Emory University School of Medicine and could form the basis of a treatment for peripheral artery disease, derived from a patients own cells.

Young-sup Yoon, MD, PhD, who led the team, said: We tried several different gels before finding the best one. This is the part that is my dream come true: the endothelial cells are really contributing to endogenous vessels.

When cells are implanted on their own, many of them die quickly, and the main therapeutic benefits are from growth factors they secrete. When these endothelial cells are delivered in a gel, they are protected. It takes several weeks for most of them to migrate to vessels and incorporate into them.

Other groups had done this type of thing before, but the main point is that all of the culture components we used would be compatible with clinical applications.

This research is particularly successful as previous attempts to achieve the same effect elsewhere had implanted cells lasting only a few days to weeks, using mostly adult stem cells, such as mesenchymal stem cells or endothelial progenitor cells. The scientists also designed a gel to mimic the supportive effects of the extracellular matrix. When encapsulated by the gel, cells could survive oxidative stress inflicted by hydrogen peroxide that killed unprotected cells. The gel is biodegradable, disappearing over the course of several weeks.

The scientists tested the effects of the encapsulated cells by injecting them into mice with hindlimb ischemia (restricted blood flow in the leg), a model of peripheral artery disease.

After 4 weeks, the density of blood vessels was highest in mice implanted with gel-encapsulated endothelial cells. The mice were nude, meaning genetically immunodeficient, facilitating acceptance of human cells.

The scientists found that implanted cells produce pro-angiogenic and vasculogenic growth factors. In addition, protection by the gel augmented and prolonged the cells ability to contribute directly to blood vessels. To visualise the implanted cells, they were labelled beforehand with a red dye, while functioning blood vessels were labelled by infusing a green dye into living animals. Implanted cells incorporated into vessels, with the highest degree of incorporation occurring at 10 months.

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Stem cell therapy is for animals too - SciTech Europa