CIRM at Business of Personalized Medicine Summit

Exciting new technologies such as regenerative medicine, tissue engineering and gene therapy are already at the forefront of a new era of medicine. And today, CIRM’s own Business Development Officer, Neil Littman, moderated a panel titled The Impact of Next Generation Personalized Medicine Technologies: How Disruptive Tech Continues to Advance the Industry, at the annual Business of Personalized Medicine Summit.

BPMS Logo2014

The panel discussed the innovative technologies we have at our disposal today, and provided a glimpse into the future—highlighting promising therapies already in the clinic as well as technologies that may be available in 5 to 10 years. For example, Curt Herberts, Senior Director of Corporate Development & Strategy from Sangamo BioSciences, discussed Sangamo’s grant under CIRM’s Strategic Partnership II Award, which uses genome-editing technology for a one-time treatment for the blood disorder Beta-thalassemia.

Importantly, the panel delved into potential paradigm shifts in medical care that may arise as a result of these new technologies, and discussed how to translate these cutting-edge technologies into human clinical trials. Carlos Olguin, Head of Bio/nano/Programmable Matter Group, Autodesk and Dr. Kumar Sharma, who directs the Center for Renal Translational Medicine University of California, San Diego La Jolla, rounded out the panel.

Finally, Neil asked panel members to discuss the issues surrounding market adoption and the potential resistance to paradigm-shifting technologies, the final hurdle in the delivery of much-needed therapies to patients.

Stem Cell Stories that Caught our Eye: A Zebrafish’s Stripes, Stem Cell Sound Waves and the Dangers of Stem Cell Tourism

Here are some stem cell stories that caught our eye this past week. Some are groundbreaking science, others are of personal interest to us, and still others are just fun.

The zebrafish (Danio rerio) owes its name to a repeating pattern of blue stripes alternating with golden stripes. [Credit: MPI f. Developmental Biology/ P. Malhawar]

The zebrafish (Danio rerio) owes its name to a repeating pattern of blue stripes alternating with golden stripes. [Credit: MPI f. Developmental Biology/ P. Malhawar]

How the Zebrafish Got its Stripes. Scientists in Germany have identified the different pigment cells that emerge during embryonic development and that determine the signature-striped pattern on the skins of zebrafish—one of science’s most commonly studied model organisms. These results, published this week in the journal Science, will help researchers understand how patterns, from stripes to spots to everything in between, develop.

In the study, scientists at the Max Planck Institute for Developmental Biology mapped how three distinct pigment cells, called black cells, reflective silvery cells, and yellow cells emerge during development and arrange themselves into the characteristic stripes. While researchers knew these three cell types were involved in stripe formation, what they discovered here was that these cells form when the zebrafish is a mere embryo.

“We were surprised to observe such cell behaviors, as these were totally unexpected from what we knew about color pattern formation”, says Prateek Mahalwar, first author of the study, in a news release.

What most surprised the research team, according to the news release, was that the three cell types each travel across the embryo to form the skin from a different direction. According to Dr. Christiane Nüsslein-Volhard, the study’s senior author:

“These findings inform our way of thinking about color pattern formation in other fish, but also in animals which are not accessible to direct observation during development such as peacocks, tigers and zebras.”

Sound Waves Dispense Individual Stem Cells. It happens all the time in the lab: scientists need to isolate and study a single stem cell. The trick is, how best to do it. Many methods have been developed to achieve this goal, but now scientists at the Regenerative Medicine Institute (REMEDI) at NUI Galway and Irish start-up Poly-Pico Technologies Ltd. have pioneered the idea of using sound waves to isolate living stem cells, in this case from bone marrow, with what they call the Poly-Pico micro-drop dispensing device.

Poly-Pico Technologies Ltd., a start-up that was spun out from the University of Limerick in Ireland, has developed a device that uses sound energy to accurately dispense protein, antibodies and DNA at very low volumes. In this study, REMEDI scientists harnessed this same technology to dispense stem cells.

These results, while preliminary, could help improve our understanding of stem cell biology, as well as a number of additional applications. As Poly-Pico CEO Alan Crean commented in a news release:

“We are delighted to see this new technology opportunity emerge at the interface between biology and engineering. There are other exciting applications of Poly-Pico’s unique technology in, for example, drug screening and DNA amplification. Our objective here is to make our technology available to companies, and researchers, and add value to what they are doing. This is one example of such a success.”

The Dangers of Stem Cell Toursim. Finally, a story from ABC News Australia, in which they recount a woman’s terrifying encounter with an unproven stem cell technique.

In this story, Annie Levington, who has suffered from multiple scleoris (MS) since 2007, tells of her journey from Melbourne to Germany. She describes a frightening experience in which she paid $15,000 to have a stem cell transplant. But when she returned home to Australia, she saw no improvement in her MS—a neuroinflammatory disease that causes nerve cells to whither.

“They said I would feel the effects within the next three weeks to a year. And nothing – I had noticed nothing whatsoever. [My neurologist] sent me to a hematologist who checked my bloods and concluded there was no evidence whatsoever that I received a stem cell transplant.”

Sadly, Levington’s story is not unusual, though it is not as dreadful as other instances, in which patients have traveled thousands of miles to have treatments that not only don’t cure they condition—they actually cause deadly harm.

The reason that these unproven techniques are even being administered is based on a medical loophole that allows doctors to treat patients, both in Australia and overseas, with their own stem cells—even if that treatment is unsafe or unproven.

And while there have been some extreme cases of death or severe injury because of these treatments, experts warn that the most likely outcome of these untested treatments is similar to Levington’s—your health won’t improve, but your bank account will have dwindled.

Want to learn more about the dangers of stem cell tourism? Check out our Stem Cell Tourism Fact Sheet.

FDA gives Asterias green light to start CIRM-funded clinical trial in spinal cord injury

This morning Asterias Biotherapeutics announced that they have been cleared by the Food and Drug Administration (FDA) to start a clinical trial using stem cells to treat spinal cord injury. It’s great news, doubly so as we are funding that trial.


You can read more about the trial in a news release we just sent out.

This trial is a follow-on to the Geron trial that we funded back in 2010 that was halted after 5 patients, not because of any safety concerns but because of a change in Geron’s business strategy.

Katie Sharify was the fifth and final patient enrolled in that trial and treated with the stem cells. Like all of us she was disappointed when the trial was halted. And like all of us she is delighted that Asterias is now taking that work and building on it.

Here’s what Katie had to say when she heard the news:

“Of course, I’m very happy that the trial has been revived. Knowing that the FDA approved the continuation based on the safety data I was a part of is great news. As you know, the trial was halted 2 days before I received the stem cells. A big part of why I ended up participating was because I figured that once the study is revived a bigger sample size (even if just by 1 person) was more valuable than a smaller one. I never regretted my choice to participate but I have doubted whether my contribution actually meant anything. I think now I finally feel a sense of accomplishment because the trial is not only being continued but also progressing in the right direction as a higher dose is going to be used. A lot remains unknown about human embryonic stem cells and that’s exactly why this research is so important. The scientific community is going to have a much greater understanding of these stem cells from the data that will be collected throughout the study and I’m glad to have been a part of this advancement.”

World’s largest pharmaceutical company signs deal with ViaCyte supporting stem cell therapy for type 1 diabetes

It’s been a good week for ViaCyte, a good week for us here at the stem cell agency and potentially a great week for people with type 1 diabetes.

Earlier this week ViaCyte announced they have been given approval to start a clinical trial for their new approach to treating type 1 diabetes. Then today they announced that they have signed an agreement with Janssen Research & Development LLC and its affiliated investment fund, Johnson & Johnson Development Corporation (JJDC).

ViaCyte's President & CEO, Paul Laikind

ViaCyte’s President & CEO, Paul Laikind

Under this new agreement Janssen and JJDC will provide ViaCyte with $20 million with a future right to consider a longer-term transaction related to the product candidate that ViaCyte is developing for type 1 diabetes.

The agreement is a big deal because Janssen is a division of Johnson & Johnson, which just happens to be the largest pharmaceutical company in the world (they were also ranked the world’s most respected company by Barron’s Magazine in 2008, not a bad reputation to have). Companies like this have traditionally been shy about jumping into the stem cell arena, as they wanted to be sure that they had a good chance to see a return on any investment they made. Not surprising really. You don’t get to be as successful as they are by throwing your money away.

The fact that they have decided that ViaCyte is a good investment reflects on the quality of the company, the years of hard work the people at ViaCyte have put in developing their therapy, and the impressive pre-clinical evidence that it works. It also reflects the fact that we helped fund the project, investing almost $40 million in the program, and get it to this point

In a news release we issued about the announcement our President and CEO, C. Randal Mills, said:

“This is excellent news as it demonstrates that pharmaceutical companies are recognizing stem cell therapies hold tremendous promise and need to be part of their development portfolio,” says C. Randal Mills, Ph.D., President and CEO of the stem cell agency. “This kind of serious financial commitment from industry is vital in helping get promising therapies like this through all the phases of clinical trials and, most importantly, to the patients in need.”

What’s nice is that this is not just a one-off deal. This is the third time this year that a large company has stepped in to make a deal with a company that we are funding.

In January Capricor Therapeutics signed a deal with Janssen Biotech that could ultimately be worth almost $340 million for its work using stem cells to treat people who have had a heart attack. The same month Sangamo, who we are funding to develop a treatment for beta-thalassemia, signed a potential $320 million agreement with Biogen Idec.

As Randy Mills said:

“Our goal at CIRM is to do everything we can to accelerate the development of successful therapies for people in need,” says Mills. “These kinds of agreements and investments help us do that, not only by adding an extra layer of funding for development, but also by validating the scientific and commercial potential of regenerative medicine.”

It’s great news for ViaCyte. It’s confirmation for us that we have been investing our money well in a promising therapy. But most of all it’s encouraging for anyone with type 1 diabetes, giving them a sense of hope that a new treatment could be on the horizon.

First of its kind stem cell production facility sets its sights on deadly childhood disease

We are used to hearing about immune suppression when transplanting organs or cells from one person to another. It’s a necessary step in preventing the body from attacking the transplanted material. Now Children’s Hospital of Orange County (CHOC) has just unveiled its newest tool to treat rare childhood diseases. Instead of focusing on immune suppression this focuses on immune-matching.

CHOC's new stem cell production facility

CHOC’s new stem cell production facility

CHOC has opened up a new stem cell production facility. It’s funded by CIRM and it’s a state-of-the-art mini clean room/manufacturing facility that will allow researchers to produce patient-specific cells for future immune-matching therapies.

“We are excited. We’ve been planning this for at least five years,” says Philip Schwartz, Ph.D., senior scientist at the CHOC Children’s Research Institute and managing director of the National Human Neural Stem Cell Resource.

“The major thing is that the footprint is much smaller than a traditional stem cell manufacturing facility, it’s all housed in one room so that keeps the cost down. The device we use to reproduce the cells is also much smaller so this set up doesn’t require multiple rooms and complex pass-throughs as you move from one room to another. All that meant the cost was only around $500,000 which is many times smaller than the more conventional facility.”

Dr. Schwartz is wasting little time putting the new facility to work. It’s already up and running and culturing cells for his work in developing a treatment for mucopolysaccharidosis (MPS-1), a rare neurodegenerative disease that usually kills children before the age of 10.

He is working on a kind of 1-2 punch approach to the disease. Using donated umbilical cord blood to help replace the child’s damaged immune system and then turning some of those blood stem cells into neural cells, the kind damaged by MPS-1, and transplanting those into the brain to repair and prevent further damage.

“This is a really interesting approach. Bone marrow transplants treat a neck down disease. Brain transplants treat a neck up disease. But conditions like MPS-1 are system wide and need both a neck down and neck up approach. Our approach could help combine those and because the cells are carefully matched also mean they won’t need to be on immune-suppressant therapy for life.”

Dr. Schwartz says animal studies using this two pronged approach have been very encouraging but he cautions there is still a lot of work to do before it would be ready for a clinical trial in people. However, if this approach is effective then it could be useful for more than just MPS-1:

“I have a high level of confidence that this will work and if it does work then we can use it in other conditions as well, such as Multiple Sclerosis. Some clinical studies show that MS patients with leukemia who got a bone marrow transplant also saw a decrease in their MS symptoms.”

Kevin McCormack

Putting the promise to the test: a new move to see if stem cell therapies can help injured athletes

One of the toughest questions we get asked, and we get asked this a lot, is a variation on the theme of “I have xxxx disease and want to know where I can get a stem cell therapy for it?” All too often, in fact pretty much all the time, we have to explain that there aren’t any therapies available, at least not yet, and that it might be a couple of years before any of the really promising projects we are funding are enrolling patients in a clinical trial.

Injured knee

But still the questions come in, fueled in part by all the clinics and centers out there claiming they can treat everything from rheumatoid arthritis to type 2 diabetes and Crohn’s disease. The biggest problem of course is that very few, if any, of these centers and physicians back up their claims with any evidence or studies to show that their treatments work. They have patient testimonials plastered all over their websites. They have lots of very reassuring sounding information, but no evidence or proof that anything they are doing will work.

Fortunately there are a growing number of researchers and reporters holding these clinics up to the light to see if what they are claiming could be true. In most cases the answer is a resounding “heck no.”

Just a week ago we told you about a couple of recent reports that looked at all the claims about using stem cells to treat sports injuries and whether there is anything to support claims by some cosmetic practitioners that they can use stem cell-based therapies to reverse the aging process (spoiler alert – there isn’t, otherwise I’d be first in line to try them out).

Cover of article about stem cells in Muscle and Medicine

Cover of article about stem cells in Muscle and Medicine

But now some mainstream media reporters are taking a closer look at claims these therapies are effective, particularly those associated with top-flight athletes. A recent issue of Muscle and Medicine – an online website that is part of the Sports Illustrated stable of publications – carried a really in-depth and thoughtful look at the use of stem cells to treat superstar athletes.

Writer Jenny Vrentas sets the tone in the opening paragraph saying:

“It may be the next big breakthrough in the treatment of sports ailments, but for now the use of such therapy is strictly limited in the U.S. – and questions about effectiveness outweigh the answers.”

Vrentas carries that questioning attitude throughout, highlighting some of the athletes who talk openly about procedures (we can’t really call them “treatments” because we don’t know if they actually treat anything) but also profiling orthopedic surgeon, James Andrews, who is a proponent of stem cells and is trying to do the kind of study necessary to see if these therapies work or not:

Andrews speaks carefully about the potential of stem-cell treatments. He’s hyper-aware of the danger of sensationalizing among his clientele of elite athletes, particularly since many questions remain—not the least of which is how well the treatments actually work. But the early returns have motivated him, as has seeing his top patients go abroad for therapy: “They don’t really know what they are getting,” he says. “Are they getting illegal stuff? We don’t have any control over it, so it’s something we needed to bring back and do in a controlled environment here.”

It’s an excellent example of the kind of reporting that can really help people, weekend warriors or anyone else, who are wondering whether stem cells might help them. It highlights the promise, but also underlines the fact that we need proof to back up that promise before it’s ready for prime time.

kevin mccormack 

Stem Cell Agency Funded Treatment for Type 1 Diabetes Takes a Big Step towards Clinical Trials

Even the best ideas can fail without a lot of support. One of the things we pride ourselves on at the Stem Cell Agency is nurturing really promising ideas for new therapies through sustained funding, giving them the support they need to turn that promise into reality. So it’s very gratifying today to hear that one project we have supported for many years, ViaCyte’s VC-01™ implantable device for treating type 1 diabetes, just took a big step towards being tested in patients.

ViaCyte has submitted what’s called an Investigational New Drug application (“IND”) with the Food and Drug Administration (FDA) asking permission to start a phase 1/2 clinical trial in patients. If the FDA says yes then ViaCyte hopes to start testing their device in patients before the end of the year.

We have invested almost $40 million in nurturing the project through the early, most basic research to see if this approach could be made to work, and then through more rigorous advanced research and testing in animals to make sure it’s safe and that it is effective.

As our Chairman, Jonathan Thomas, says in a press release we sent out announcing the news:

“We have been strong supporters of Viacyte for many years and it’s great to see that they are well on the way to starting a First-in-Human trial, hopefully in the next few months. This therapy’s growth from an idea to a potential treatment highlights CIRM’s commitment to following promising science at all stages of development.”

The device is really quite ingenious. It is a thin plastic pouch that contains an immature form of pancreatic cells. When the device is implanted under the skin these cells become the different kinds of cells needed to regulate blood glucose levels. They are able to sense when blood glucose is high, and then secrete insulin to restore it to a healthy level. The truly impressive part is that the device has holes large enough to allow insulin to be pushed out, but too small to allow the body’s own immune system to get in and attack the device.

The goal of the first phase of this clinical trial, as with all phase 1 trials, is simply to show that the VC-01™ is safe. The second phase will also look at safety but also test it to see if it is helping patients, reducing their dependence on injected insulin. If the results from both those phases are encouraging, the next step is to test it in much larger numbers of patients to see just how effective it is.

But this first step, submitting an application to the FDA, is the starting point for all that. As our President and CEO C. Randal Mills said in our news release, getting to the starting line is often half the battle:

“This is good news for ViaCyte and is an encouraging sign of the progress they are making. Filing for an IND is a crucial step along the path to making a therapy available to patients and we’ll be working with them and supporting them every step of the way to try and make this happen as quickly, and as safely, as possible.”

You can read more about ViaCyte and our support for them on our website.

Stem Cell Stories that Caught our Eye: Gene Rx, New and Rejuvenated Blood Stem Cells and Budget Cuts

Here are some stem cell stories that caught our eye this past week. Some are groundbreaking science, others are of personal interest to us, and still others are just fun.

Tinkering with stem cell genes safe. Research at the Salk Institute provides some reassurance that using gene-editing techniques to correct disease-causing mutations in stem cells is safe. This type of intervention aims to give people a corrected version of a gene that can produce a functional protein to replace the bad one they were born with, such as the hemoglobin gene in sickle cell disease. The CIRM-funded Salk team made gene corrections with both of the two most common gene-editing techniques: using a virus to carry the correct gene into the cell, and using an enzyme to cut and splice the genes. The fear, the lead researcher said in Space Daily, has been that this gene manipulation would cause unwanted mutations. Instead the team found that the very small number of mutations in the edited cells did not exceed the number in normal cells growing in the lab for the same length of time.

Blood cells

This is great news for CIRM, since eight of our Disease Teams—all of which have the goal of moving therapies into the clinic—use gene modification techniques. These include efforts to correct the genetic mutation that causes sickle cell disease and beta thalassemia.

Interview with Nobelist on stem cell potential. The Raw Story ran an interview with Nobel Laureate Martin Evans about the field he helped to create when he first isolated mouse embryonic stem cells in 1981. He won the Nobel in 2007 for later work in which he used embryonic stem cells to create specific gene modifications in mice. He said we are “just scraping the surface” in unlocking the potential of stem cells to change medicine. He also addresses various aspects of reprogramming cells to become different types of tissue and provides a bit of advice to young scientists: “You should not believe in all that you read.”

Keep your blood stem cells acting young. Blood stem cells, like most of the adult stem cells in our various tissues, become less adept at doing their job of replenishing our tissues as we age. A team at New York’s Mount Sinai has fingered the decrease of a specific protein in older stem cells as the culprit. That protein, SIRT1, was not a surprise as it has been implicated in other aging research. When laboratory animals eat a severely calorie restricted diet and live longer, SIRT1 is active at a higher than normal level. So, it makes some sense that low levels of SIRT1 would be associated with conditions of aging. The team now wants to see if increasing SIRT1 levels can put the kick of youth back into older blood stem cells. The web portal Hospital Newspaper ran a story on the research that the team published in Stem Cell Reports.

Or use a new way to create blood cells. If you can’t get your own blood stem cells to behave like vigorous youthful cells another option is to get some new one. The problem is many folks cannot find a matching donor and previous attempts to grow them from earlier stage stem cells have not worked. Using either embryonic or reprogrammed iPS type stem cells to try to grow large quantities of blood-forming stem cells has always resulted in immature cells that cannot make all the blood cells and don’t readily take up residence—engraft—in the patient. Researchers at Cornell Medical College may have solved this problem by growing the stem cells in a more natural environment. They grew them in a bed of cells like those that would have surrounded them in blood vessels in a developing fetus. The resulting cells engrafted in mice and produced nearly all the components of blood. They had a few lingering problems with creating the immune system’s T cells, but got much closer than previous work. Device Space picked up the medical school’s press release.

This goal of creating fully functional blood stem cells is sufficiently important but vexing to the research community that CIRM organized an international workshop on the topic. You can read the resulting whitepaper “Breaking the Bottleneck.”

Donors needed to power discovery. With federal support for research shrinking many institutions are relying more and more on donors to fund the research that leads to discoveries and eventually therapies. The New York based web publication Capital Playbook painted a picture of the deficit citing a 22 percent reduction in the inflation-adjusted budget for the National Institutes of Health since 2003. It goes on to quote senior scientists fearing the loss of a generation of scientists. A great comment came from my friend and former colleague David Scadden, co-director of the Harvard Stem Cell Institute. “They are seeing their senior mentors spending more and more time writing grants and going hat in hand. That’s not a good way to inspire the best and brightest.”

Don Gibbons

Argentina Soccer Star Pins his World Cup Final Hopes on Stem Cells

I suppose we should have expected it. Every time there is a big sporting event stem cells seem to come into the conversation. So it’s not surprising that the World Cup in Brazil, the biggest sporting event on the planet, was bound to somehow, in some way, involve stem cells. And it has.

Argentina’s speedy attacker, Angel Di Maria, suffered a torn hamstring in the game against Belgium. He was initially ruled out for the rest of the tournament but then came news that he was hoping to be able to play in the final – if his team made it, which they have – by getting a stem cell therapy.

Angel Di Maria: Photo courtesy Fanny Schertzer

Angel Di Maria: Photo courtesy Fanny Schertzer

Now, as often happens in instances like this, the reports have been light on specifics although there are some hints in the media that it might involve the use of stem cells taken from Di Maria’s own fat tissue or from his blood.

The web site Inside Spanish Football mentioned that another player, Atletico Madrid’s Diego Costa, underwent a similar procedure to try and recover from an injury before a recent championship game. The web site described it this way:

“The medical procedure is used to regenerate damaged cells using the patient’s own healthy cells, with the primary object being to reduce inflammation and repair the torn muscle tissue.”

Not surprisingly, because famous athletes are involved, the therapy is getting a lot of exposure in the media. The same thing occurred when Peyton Manning, the quarterback for another kind of football team, the Denver Broncos, got a stem cell treatment for a neck injury; and when Yankee’s baseball pitcher C. C. Sabathia underwent a stem cell treatment for a knee injury. We blogged about both of these instances.

The problem with the coverage is that the media typically does a good job of explaining what the therapy is designed to do, but then fails to mention that none of these therapies have been tested or proven to work in a clinical trial. It gives the impression that this is a routine therapy for an injury. It’s not. It is, in every sense, experimental. And therein lies the problem. While the treatment may be safe there’s also a chance it is not. While it may be effective to some extent, we really have no way of knowing.

Another confounding factor in all this is that alongside the stem cell therapy, Di Maria is also getting intensive traditional therapy – ice, kinesiology, electro pulse stimulation and some rehabilitation exercises in the swimming pool. So even if Di Maria does beat the odds and return in time for the World Cup Final, we really won’t know if it was the stem cells, the traditional therapy, or both that worked.

And that’s the real problem here. It’s not that a professional athlete is doing everything he can to be ready for the biggest game of his life – that’s to be expected – but that the media doesn’t dig a little deeper to see if there’s any evidence this approach could work. By failing to do that they leave the playing field open to other “clinics” to offer this same kind of therapy to anyone; clinics who will promote their treatment “as used by” and give the impression that if it helped Argentina win the World Cup, or at least come in second, then it can certainly help you bounce back from your injury.

So next time you read about a superstar athlete turning to stem cells for a miracle cure don’t assume that it will help them. The odds are it won’t. Sports are fun. But your health is nothing to play around with.

Before considering any stem cell treatment, we highly suggest looking at educational information for patients provided by the International Society for Stem Cell Research, the world’s leading stem cell research organization. Their printable, take-it-along Patient Handbook identifies questions any patient should ask. It would be a good idea to review answers with a physician you trust.

kevin mccormack

Stem Cell Stories that Caught our Eye: Multiple Sclerosis, Diabetes, Cornea Repair and of Course, New Stem Cells too Good to be True

Here are some stem cell stories that caught our eye this past week. Some are groundbreaking science, others are of personal interest to us, and still others are just fun.

Buddy system gets stem cells to stick around. The type of stem cell most likely to be used in a clinical trial today is the mesenchymal stem cell (MSC) found in fat and bone marrow. It is also the type of stem cell most likely to produce vaguely positive or downright disappointing results. In most situations they die within a few days of being transplanted, so the only impact they can have is from the various protein signals they secrete that may trigger the body’s own natural healing processes. They never live up to their stem cell potential to form new adult tissue. A team at Harvard looked at their natural environment and found they most often live near a second type of cell called an endothelial colony-forming cell. When the team transplanted the two cells together they found the MSCs survived for weeks and matured into appropriate adult tissue. Genetic Engineering & Biotechnology News had a nice interview with members of the team about their work that appeared this week in the Proceedings of the National Academy of Sciences.

Fat cells (yellow) descended from transplanted stem cells (green) inside a mouse 28 days after co-transplantation with “buddy cells”  [Courtesy Children’s Hospital]

Fat cells (yellow) descended from transplanted stem cells (green) inside a mouse 28 days after co-transplantation with “buddy cells”
[Courtesy Children’s Hospital]

Master switch for creating brain insulation.
Researchers know how to take a skin cell from a patient, turn it into an iPS type stem cell and then turn those cells into the type of intermediate cell that can become the myelin that insulates our nerves and is lost in Multiple Sclerosis. The problem: the process takes way too long to be a feasible therapy. To get enough of these middleman cells called oligodendrocyte progenitors for a therapy can take as much as a year. Neural stem cells naturally mature into multiple intermediate cells, but prefer to become the progenitors for neurons, which would not help an MS patient. A team at the University of Buffalo looked to see what genetic switches were active in neuron progenitors versus those for myelin. They found that just one of these switches could push the early nerve stem cells to the myelin middlemen. That genetic factor, SOX10, instantly becomes a candidate for a path to a more efficient therapy. Again, Genetic Engineering & Biotechnology News did the best of several write-ups of this work that was published in the Proceedings of the National Academy of Sciences.

You can read about CIRM’s projects working on a cure for MS on our Multiple Sclerosis Fact Sheet.

Can gut be taught to make insulin. Earlier work at Columbia University had shown that in mice you can turn off a single gene and get normal gut cells to secrete insulin and to do so in response to sugar in the bloodstream. Now the team has made the often difficult transition of moving from mouse results to humans, or in this case human gut cells in a dish. They matured human stem cells into gut tissue and then shut down the one gene. The resulting cells produced insulin in response to sugar in their environment. The research published in Nature Communication got coverage on a few sites including HealthDay.

Early success in cornea repair poised to get even better. One of the stem cell field’s early successes has been work pioneered in Italy using a type of stem cell found in the cornea of the eye. When a patient has the cornea of one eye damaged they harvest these cells, called limbal stem cells, from the healthy eye and transplant them to the damaged eye. It often works quite well, but not always and the success has been correlated with how many actual limbal stem cells are among the cells transplanted. It has been difficult to sort out and purify the stem cells until now. A team from three Harvard affiliated hospitals has found a marker that let them transplant purer human limbal stem cells into mice and they saw consistent regrowth of damaged corneas. RedOrbit wrote up the research that was published in Nature.

STAP stem cell retraction everywhere. When Japanese and American researchers published a new, simple method for creating stem cells in January it got way more news coverage than an unconfirmed and unconventional piece of research should have. Most of that coverage failed to include the caveat that the work needed to be replicated to confirm the findings. In less than six months, the research community quickly reported repeated failures to replicate the work and more recently found outright errors in the published papers. When the journal that published the work, Nature, formally retracted the papers this week it was good to see that this “oops-ignore-our-first-article” seemed to get equal play. To show the reach of this news, I have included the Associated Press version from the tiny Logansport Pharos Tribune, which averages about 12 pages a day and is the closest real newspaper to the tiny Indiana town where I grew up.

Don Gibbons