CIRM 2.0; saving time, saves lives

It’s been almost ten years since the voters of California approved Proposition 71, creating the Stem Cell Agency and giving us $3 billion to fund stem cell research. So this is an appropriate time to look back and see what we have done with the money so far, the progress that’s been made, and where we are heading in the next ten years.

Over the next few weeks we’ll be taking a more detailed look at all these elements – it’s too much to cover in one blog – but let’s start with where we’re heading. At yesterday’s meeting of our governing Board, the Independent Citizens Oversight Committee, President & CEO, C. Randal Mills, Ph.D. charted a course for future funding.

Randy Mills, Stem Cell Agency President & CEO

Randy Mills, Stem Cell Agency President & CEO

Right now it can take up to two years for a project going into clinical trials to apply for and get funding from us. As Randy said in a news release we issued after the meeting, “That’s just unacceptable”:

Under what he is calling CIRM 2.0, Randy is proposing to trim that down dramatically:

“We are going to shorten that to just 120 days. But we’re not just making it faster, we’re also making it easier for companies or institutions with a therapy that is ready to go into clinical trials to be able to get funding for their project when they need it. Under this new system they will be able to apply anytime, and not have to try and shoehorn their needs into our application process.”

The goal is not just to make it easier to apply for funding, but also to get more, high quality applications. Right now there is pressure on companies to apply before they are really ready because they know if they miss a current application deadline it could be another year to 18 months before another award cycle comes around. Under CIRM 2.0 application will be accepted on a monthly basis, so applicants won’t have to worry about missing one deadline – they can just apply the following month. Applying when they are ready will increase the likelihood that the projects will be and of high quality.

And as Randy points out, if it works at the clinical stage of funding, it can work at every stage:

“Speeding up the process, at all stages of research, just makes sense. The faster that researchers can get access to the funds they need to do their work, the faster they are likely to be able to produce something that helps patients.”

The speeding up of the process doesn’t just involve companies and researchers being able to apply anytime, it also means that when they do apply they’ll have to have all the supporting documentation and studies on hand to show they are ready to go as soon as the Board approves funding.

In the past there was often a delay of six months or even more after an application had been approved for funding while research milestones were negotiated and agreements signed. Because CIRM 2.0 will involve identifying milestones much earlier in the application process that delay will disappear.

This new approach involves a complete overhaul of the way we currently work but we think it’s worth it. We plan to start by introducing these changes for the projects that are furthest along, those ready to go into clinical trials, but in time we intend extending this to cover everything we fund.

Making these changes will help us trim a two-year process down to just three months. That means any therapy that proves successful is getting to the patients who need it much sooner than it otherwise would. And with many of the diseases we are targeting, saving time means saving lives.

Stem cell stories that caught our eye: Some good news got a little overplayed on blindness and Alzheimer’s

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.

Stories on blindness show too much wide-eyed wonder. While our field got some very good news this week when Advanced Cell Technologies (ACT) published data on its first 18 patients treated for two blinding diseases, many of the news stories were a little too positive. The San Diego Union Tribune ran the story from Associated Press writer Maria Cheng who produced an appropriately measured piece. She led with the main point of this early-phase study—the cells implanted seem to be safe—and discussed “improved vision” in half the patients. She did not imply their sight came back to normal. Her third paragraph had a quote from a leading voice in the field Chris Mason of University College London:

“It’s a wonderful first step but it doesn’t prove that (stem cells) work.”

The ACT team implanted a type of cell called RPE cells made from embryonic stem cells. Those cells are damaged in the two forms of blindness tested in this trial, Stargardt’s macular dystrophy and age-related macular degeneration, the leading cause of blindness in the elderly. Some of the patients have been followed for three years after the cell transplants, which provides the best evidence to date that cells derived from embryonic stem cells can be safe. And some of the patients regained useful levels of vision, which with this small study you still have to consider other possible reasons for the improvement, but it is certainly a positive sign.

CIRM funds a team using a different approach to replacing the RPE cells in these patients and they expect to begin a clinical trial late this year

Stem cells create stronger bone with nanoparticles.   Getting a person’s own stem cells to repair bad breaks in their bones certainly seems more humane than hacking out a piece of healthy bone from some place else on their body and moving it to the damaged area. But our own stem cells often can’t mend anything more than minor breaks. So, a team from Keele University and the University of Nottingham in the U.K. laced magnetic nanoparticles with growth factors that stimulate stem cell growth and used external magnets to hold the particles at the site of injury after they were injected.

It worked nicely in laboratory models as reported in the journal Stem Cells Translational Medicine, and reported on the web site benzinga. Now comes the hard step of proving it is safe to test in humans

Stem cells might end chronic shortage of blood platelets. Blood platelets—a staple of cancer therapy because they get depleted by chemotherapy and radiation—too often are in short supply. They can only set on the shelf for five days after a donation. If we could generate them from stem cells, they could be made on demand, but you’d have to make many different versions to match various peoples’ blood type. The latter has been a bit of a moot point since no one has been able to make clinical grade platelets from stem cells.

plateletsA paper published today by Advanced Cell Technologies may have solved the platelet production hurdle and the immune matching all at once. (ACT is having a good week.) They produced platelets in large quantities from reprogrammed iPS type stem cells without using any of the ingredients that make many iPS cells unusable for human therapy. And before they made the platelets, they deleted the gene in the stem cells responsible for the bulk of immune rejection. So, they may have created a so-called “universal” donor.

They published their method in Stem Cell Reports and Reuters picked up their press release. Let’s see if the claims hold up.

Alzheimer’s in a dish—for the second time. My old colleagues at Harvard got a little more credit than they deserved this week. Numerous outlets, including the Boston Globe, picked up a piece by The New York Times’ Gina Kolata crediting them with creating a model of Alzheimer’s in a lab dish for the first time. This was actually done by CIRM-grantee Lawrence Goldstein at the University of California, San Diego, a couple years ago.

But there were some significant differences in what the teams did do. Goldstein’s lab created iPS type stem cells from skin samples of patients who had a genetic form of the disease. They matured those into nerve cells and did see increased secretion of the two proteins, tau and amyloid-beta, found in the nerves of Alzheimer’s patients. But they did not see those proteins turn into the plaques and tangles thought to wreak havoc in the disease. The Harvard team did, which they attributed, in part, to growing the cells in a 3-dimensional gel that let the nerves grow more like they would normally.

The Harvard team, however, started with embryonic stem cells, matured them into nerves, and then artificially introduced the Alzheimer’s-associated gene. They have already begun using the model system to screen existing drugs for candidates that might be able to clear or prevent the plaques and tangles. But they introduced the gene in such a way the nerve cells over express the disease gene, so it is not certain the model will accurately predict successful therapies in patients.

Don Gibbons

Stem cell stories that caught our eye: fast track marketing in Japan, a 3D cell tour and autism

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.

Event showed great progress, but Japan nipping at our heals. The San Diego Union Tribune’s Brad Fikes seemed to be enjoying covering the Stem Cell Meeting on the Mesa in his own backyard in La Jolla. He stayed for the full two days of the industry Partnering Forum and when we chatted he said he had more good material than he could use. I was certainly willing to second the sentiment of the opening paragraph of the story he wrote:

“More than ever before, stem cell therapies appear poised to transform medicine — potentially curing heart disease, diabetes and paralyzing injuries, among other ailments.”

But the last portion of his piece was a little unsettling. He noted the frequent discussion at the meeting of Japan’s new fast track path for marketing stem cell therapies. The CEO of Athersys, one of the leading companies in the field, announced at the meeting that his company would be taking their lead product to Japan first for marketing, not the U.S.

Turning cell biology into a 3-D game. The ability to track cells has become one of the most important limiting factors in stem cell biology. We need to know where cells go when they are transplanted in the body, but even before that, we have found that the interaction of cells with their environment often determines if stem cell offspring do their jobs and we need to track cells to understand this.

Now a team at Drexel University that includes an expert in computer software and hardware used in gaming has

Researchers at Drexel touring a group of cells using 3D glasses.

Researchers at Drexel touring a group of cells using 3D glasses.

provided the field with an invaluable tool. They can label various cells with distinctive markers and follow their movements. More important they can use an elaborate software program to integrate individual slices of a tissue into a 3D sample that researchers can “tour” while wearing 3D glasses.

Red Orbit quoted Andrew Cohen the leader of the computer development team:

“It’s like Photoshop for cell biologists. The software outlines cells and blood vessels, keeping track of them as they’re dividing and moving around one another. This provides a wealth of information on the patterns of cell shape, motion and division. Visualization of the 3-D microscopy data together with the analysis results is a key step to measure and ultimately understand what drives these cells.”

Cally Templea, a leading expert from the Neural Stem Cell Institute in Rensselaer, NY, was also quoted about the power of this new tool to help stem cell biologists understand how stem cells interact with their environment:

“LEVER 3-D is amazing, it opens new vistas for understanding the stem cell niche.”

Autism linked to stem cell burst (in mice). The accelerated brain growth seen right after birth in many people with autism spectrum disorder has been linked to a burst of nerve stem cell division triggered by inflammation. The study at the University of California, Los Angeles, could explain why inflammation during pregnancy, either due to an autoimmune reaction or an infection, has been shown to be a risk factor for the disorder.

Health Canal posted the press release from the university that quoted the senior author of a paper in the journal Stem Cell Reports, Harley Kornblum:

“We have now shown that one way maternal inflammation could result in larger brains and, ultimately, autistic behavior, is through the activation of the neural stem cells that reside in the brain of all developing and adult mammals.”

The researchers gave pregnant mice a toxin found in bacteria and discovered that it triggered an excess production of nerve stem cells in their pups. This resulted in enlarged brains and behavior associated with autism, such as a reduced interest in interacting with other mice.

Little guy regrowing his head could help us. While a few species have the ability to regrow a severed body part, the tiny Hydractinia—commonly called snail fur—out does the rest in that it can regrow its head. BBC did a nice job of describing work at the University of Galway trying to explain how it accomplishes the feat and putting the work into perspective with other recent research findings.

After harvesting the creatures off the backs of hermit crabs the Galway team isolated embryonic stem cells from them, to which they attributed the ability to regrow something as complex as a head. The snail fur may be unique in that no other adult animal is believed to harbor embryonic stem cells. The researchers hope to use the tiny creature to learn how we might be able to turn on some ancestral regenerative capacity in humans.

Don Gibbons

Stem Cell Awareness Day and the Stem Cell Person of the Year

Today is Stem Cell Awareness Day, an event that seeks to bring together individuals and organizations around the world working to celebrate and raise awareness about the tremendous progress being made in stem cell research, and to ensure we remain focused on keeping that progress going until we have cures or treatments for people in need.

Paul Knoepfler

Paul Knoepfler

Today is also the day that our colleague, U.C. Davis stem cell researcher and avid blogger, Paul Knoepfler reveals the nominations for his annual Stem Cell Person of the Year award. It’s perfect timing, because many of those nominated have been pivotal in helping move the research forward, and in helping raise awareness about the field in general.

The rules for the competition are pretty simple. In Paul’s own words: “Who has been the single most important, influential person in the world of stem cells this year? Who has made the biggest positive contribution in 2014?”

This is the third year of the competition and Paul is not just a tireless champion of stem cell research, he’s also someone who puts his money where his mouth is. The prize for this year’s winner is $2,000, double that of the previous two years, and all that cash comes out of Paul’s own pocket. It’s a generous gesture from a tireless advocate. In fact those two qualities alone would suggest Paul qualifies to win his own competition. But he has too much integrity to ever even consider that.

So without further ado here is the list of nominations with quotes from some of the people who nominated them in green.

*Spoiler alert: a couple of those nominated are colleagues of mine here at the stem cell agency. I didn’t nominate them, but I can certainly testify to the fact that they deserve it.

Bernie Siegel – a long-time stem cell advocate who runs the yearly World Stem Cell Summit. “Has any one single person done more for the stem cell field?”

Chris Fasano: A principal investigator at the Neural Stem Cell Institute where he uses stem cells to study early nervous system development. Chris stands out for his energy, enthusiasm, dedication to the field, creativity and accomplishments.”

Diana DeGette, the Democratic Congresswoman from Colorado: a politician who has been working to pass important stem cell legislation. “Long time supporter of legislation to support stem cell research and regenerative medicine.”

Don Reed: long-time stem cell research advocate who played a key role in the success of Prop 71 and the creation of CIRM. “A tireless stem cell advocate always there to make a positive difference.”

Emmanuel (Ed) Baetge: Head of Nestlé Institute of Health Sciences at Nestlé Health Science and formerly CSO of Novocell/ViaCyte. “Ed was the driving force behind the development of Novocell (Viacyte’s) diabetes program using hESCs to develop a cell therapy for T1/2 patients…the phase 1 trial is enrolling this year. It has huge potential and Ed deserves the credit for his leadership and development of the technology as well as the company.”

Ellen Feigal; Vice President, Research and Development at the California Institute for Regenerative Medicine (CIRM). “At CIRM Dr. Feigal makes the Development Program happen.”

Ian mcNiece: Professor, Department of Stem Cell Transplantation,
Division of Cancer Medicine, The University of Texas MD Anderson
Cancer Center.

Janet Rossant: Senior Scientist in the Developmental & Stem Cell Biology Program and Chief of Research at The Hospital for Sick Children, Toronto

Jeanne Loring: Stem cell researcher and scholar, leading iPS cell clinical work in the pipeline for Parkinson’s Disease, and patient advocate. “Supportive to the advocacy community. Courage in supporting the challenge to WARF patents. Excellent scientist engaged in policy discussions.”

John Sinden: The co-founder of ReNeuron, a biotech company based in UK. “Thanks to his determination, and drive ReNeuron has a clinical approved stem cell product currently tested in two clinical trials.”

Judy Roberson: Long-time Huntington’s Disease patient advocate. “She makes concrete positive developments happen such as millions of dollars in research funding for HD.”

Leigh Turner: Outspoken advocate of evidence-based medicine in the stem cell field. “Gave an inspiringly frank talk about for-profit stem cell clinics flouting FDA regulations at ISSCR 2014.”

Malin Parmer: Associate Professor, Developmental and Regenerative Neurobiology, Lund University. Top neural regeneration scientist. “Young, hard worker who is doing very well”.

Masayo Takahashi: Stem cell researcher leading the team that is doing the first ever clinical study based on human iPS cells. “Creative and courageous clinical stem cell researcher.”

Mike West: He founded Geron and was CSO of Advanced Cell Technology before his current position as head of BioTime. “He has been a leader in our regen field for many years but he made a bold decision to resurrect Geron’s cell therapy for spinal cord injury this year and hopefully confirm the promise of hESC technology clinically.”

Patricia Olson: Executive Director of Scientific Activities at CIRM and active in CIRM scientific leadership from day 1. “A driving force in the stem cell field.”

Peter Zandstra: Professor at University of Toronto. Stem cell researcher. Canada Research Chair of Stem Cell Bioengineering. “Peter is a recognized pioneer and respected world leader at bringing concepts of scale-up, regulation, and industrialization to stem cell-derived cell therapy technologies.”

Pope Francis: Leader of Worldwide Catholic Church. “Strong supporter of adult stem cell biotechs and research”.

Richard Cohen: An MS patient in a clinical trial who has chronicled his experiences. An author and Meredith Vieira’s husband, father of three children. “Mr. Cohen is an inspiration to me and others because he’s very honest and doesn’t sugar-coat his struggles, losses, frustrations, anger, embarrassments, and he also shares his gains.”

Richard Garr: CEO of Neuralstem, conducting work on using stem cells for ALS and advocate of Right To Try laws. “Richard does two excellent things at once: making a treatment for ALS and connecting with ALS patients.”

Robert Lanza: CEO of Advanced Cell Technology, which has multiple ES cell-based clinical trials ongoing. “Visionary and practical so makes the impossible possible”.

Shoukhrat Mitalipov: Stem cell researcher who first successfully made nuclear transfer human ES cells by therapeutic cloning and developing oocyte transfer-based therapies for mitochondrial disorders. “Gutsy pioneer of new, game changing technologies.”

Susan Solomon: Co-Founder and CEO of The New York Stem Cell Foundation (NYSCF). Remarkably effective advocate for stem cell research. “not many leaders have created their own research laboratories and raised $100 million plus. Seriously, what an accomplishment!”

Takaho Endo: Senior Researcher at the Riken Center for Integrative Medical Sciences in Yokohama who published key genetic work on the possible origin of STAP cells. “Courage to publish this being from RIKEN.”

Ted Harada: Leading stem cell research advocate and very effective ALS patient advocate. “An Energizer Bunny for the ALS community and stem cell advocate”

Tory Williams: Stem cell advocate and author of the 2014 book, Inevitable Collision. Co-Founder and Executive Director of the Alabama Institute of Medicine (AIM). “A true hero who inspires and makes concrete things happen like AIM”.

Museum exhibit explaining stem cell super heroes opens in Canada today, due in California in 2016

7108285_origAn international touring exhibit using super hero cells as guides to explain the many roles of stem cells in our lives opens today at the Sherbrooke Museum of Nature and Science in Canada. Its five-year tour will include further displays in Canada, the United Kingdom and three stops on California—the San Francisco Bay area, Los Angeles and San Diego—in 2016.

Super Cell logoDesigned for the general public, with a special eye to children, the exhibit uses hands-on and interactive modules to show just how important stem cells are not only to our early development but also to our daily lives. CIRM was a partner in the development of the exhibit, but the primary mover behind it has been Canada’s Stem Cell Network, and within the network, Lisa Willemse who has really pushed its two-year gestation.

The earliest steps in the development involved visits to children in schools to tease out their points of interest. In a press release she explained some of what they learned:

“How does a lizard grow a new tail? Where does disease come from? How do we start little and get big? These were the kinds of questions the kids asked us, which shows a real interest in the mysteries of the body—mysteries that are largely the domain of stem cells.”

“Much of it is easy to explain, once they understand that stem cells have the ability to make all the kinds of cells in the body. For example, you can tell them that every second, stem cells in your bone marrow make about 2 million new red blood cells. You snap your fingers, and just like that, another 2 million cells were made. Soon they all start snapping their fingers, knowing that every time they do it, something remarkable and vital to life has happened in their own body.”

In Canada, the four modules have explanations in English and French. In California, they will be in English and Spanish. In Spanish the exhibit title “Super Cells: The Power of Stem Cells” becomes Celulas Fantasticas: El Poder Del Las Celulas Madre. I love the concept of a mother cell.

Additional partners in the project included the Centre for Commercialization of Regenerative Medicine in Canada and the UK’s Cell Therapy Catapult.

Don Gibbons

Stem cell stories that caught our eye: a good review at the NY Times, expanding cord blood and leukemia

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.

Review paints picture of the field today.
A writer I have respected for many years, Karen Weintraub, wrote a nice review of the current state of stem cell clinical trials in the Tuesday Science Times in the New York Times. She discusses the steady, methodical progress being made:

“Researchers have been slowly learning how to best use stem cells, what types to use and how to deliver them to the body — findings that are not singularly transformational, but progressive and pragmatic.”

She quotes our senior VP Ellen Feigal about the safety seen so far in clinical trials and notes that CIRM should have 10 clinical trials enrolling patients by the end of the year. She also covers the dangers of clinics offering unproven therapies and the power of using iPS-type stem cells to model diseases in the laboratory. Overall, a nicely balanced piece.

Making mitochondrial disease and 3-parent embryos personal. A little newspaper in Oregon called the Willamet Week has published a story that makes the issues around so-called “three-parent” babies very personal. The controversial procedure aims to allow women with rare mitochondrial diseases to have normal children.

Mitochondria, known as the powerhouses of the cell, have the unusual trait of being the only part of the cell besides the nucleus to have any DNA. It is these few genes in the mitochondria that we inherit solely from our mothers because when the DNA from the egg and sperm fuse, the mother’s mitochondria stay in the fluid outside the nucleus. So, to avoid passing along faulty mitochondrial genes, a team in Oregon devised a way to insert the DNA from the mother’s nucleus into a donor egg that had its nucleus removed, a process called nuclear transfer.

Guided by a microscope researchers insert the nucleus from one woman into the egg of another

Guided by a microscope researchers insert the nucleus from one woman into the egg of another

The paper provides a long read—nearly 4,000 words—that goes into great detail about the procedure, the ethics, the research team’s views on the ethics, and the personal story of a patient living with a disease of exhaustion she calls “mitochondrial crash.” The writer lets the patient have the last word on ethics:

“To me it’s win-win because you’re not messing with God’s child. You’re just taking out the bad parts. I don’t want to pick out a blond-haired, blue-eyed tall kid, picking your child’s traits, but to rule out a potentially lethal chronic illness brings in a whole different story.”


Cord blood might now save more adult cancer patients.
Umbilical cord blood is a literal lifesaver for many pediatric cancer patients allowing them to withstand harsh chemotherapy and be rescued by the stem cells in the cord blood. But the procedure is used in few adults because the vast majority of cord blood samples don’t have enough stem cell for an adult requiring the use of two cord samples and doubling the chance for potentially deadly immune reactions.

A team at the University of Montreal screened more than 5,000 molecules looking for one that would let them expand the number of stem cells from one sample in the lab. They hit upon one that they say could allow a 10-fold increase in the number of single cord samples suitable for adults. They expect to begin clinical trials in December.

Science News ran a brief review of the work and the blog Science 2.0 ran the university’s press release with a bit more detail.

Trial begins with cancer drug named for CIRM
Researchers at the University of California, San Diego, announced this week that they had begun a clinical trial with leukemia patients using a drug named for our agency cirmtuzumab. This molecule, in the class of drugs called antibodies, disables a protein that cancer stem cells use to accelerate the growth of cancer.

This trial, for patients with recurrence of their chronic lymphocytic leukemia, became the third CIRM funded team this month announcing plans to start clinical trials. In addition to our blog post the San Diego Union Tribune wrote about the latest trial, and we issued press releases on the trials for spinal cord injury and diabetes.

Don Gibbons

New formula a more efficient way to reprogram adult cells to become like embryonic stem cells

Shinya Yamanaka won the Nobel Prize for developing a recipe of genetic factors that can turn back the clock of adult cells and make them behave like embryonic stem cells. But he would be the first to tell you his recipe ultimately may not be the best one for making these stem cells called iPS cells.

Virtually from the day he published his groundbreaking work, teams around the world have tried to develop new formulas that get around some problems with the original. One issue is the low efficiency of getting true stem cells. Another is the high rate of genetic aberrations that can be produced in the resulting stem cells.

Now, a team pairing researchers at the Hebrew University in Jerusalem and the Whitehead Institute in Cambridge, Massachusetts, has published a new recipe that seems to yield many more true stem cells, ones that are called pluripotent because they can make all cell types. The new cells also seem to have fewer genetic alterations, which could make them safer for clinical use in people.

They made the improved cells by moving from OSKM to SNEL—from the original genetic factors, Oct4, Sox2, Klf4 and Myc, to Sall4, Nanog, Esrrb and Lin28. An elaborate computer analysis of the function of genes helped them come up with the formula.

This work used mouse cells, so up next on their agenda is coming up with a similar formula that works in human cells. HealthCanal ran the university’s press release and Genetic Engineering & Biotechnology News ran a slightly more technical analysis of the work.

Don Gibbons

Stem cell stories that caught our eye: heart stem cells, lizard tails and mapping progress in the field

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.

Could cells in arteries be elusive heart stem cells?
Our hearts have a modest limited ability to regenerate and repair themselves, suggesting we must have a few heart stem cells. But no one has figured out where those cells hang out. Now, a team at Vanderbilt in Nashville has shown that cells in the lining of the heart’s arteries can contribute to new heart muscle.

They made the discovery using a labeling technique that let them tag those cells, called endothelial cells, and show that the same tag showed up in new muscle in the heart. This suggests those cells have the properties of heart stem cells.

The finding also suggests that coronary heart disease, where plaque builds up inside the arteries, could damage the heart with a one-two-punch. Besides narrowing the artery it may also make it more difficult to mobilize these heart stem cells that reside inside the artery lining. The research published in Cell Reports was written up by Genetic Engineering & Biotechnology News.

Secrets of the lizard’s tail. Most folks who have spent any time watching nature programing on TV have seen the handy trick of the green anole lizard. If a predator catches it by the tail it can shed its tail and grow a new one. A team at Arizona State University has uncovered the genetic recipe for how the lizard pulls off this trick.

anole_5They analyzed various segments of tails as they were regrowing to see which genes were turned on that would not normally be turned on in adult tissue. They identified 325 genes. The beauty of the finding is 302 of those genes have matching genes in humans. Those genes become immediate candidates for research into finding ways to allow humans to regrow lost or damaged tissue.

Discover did a nice job of explaining how this lizard is a better model for human comparisons than other animals such as salamanders and fish that can also regrow body parts but use a very different process. And the university press release offers a bit more detail of what the team did.

Review maps where the field is going. Six leaders in the stem cell field wrote a review in the journal Science this week of what to expect in the next few years from research with pluripotent stem cells—those stem cells that can become any tissue in the body, both embryonic stem cells and reprogrammed iPS type stem cells. The authors included researchers from the University of Rochester, the University of Pittsburgh, Harvard, and the University of Wisconsin.

The main hurdles researchers are working to overcome involve maturing the stem cells to the right adult tissue, making sure they are purely those cells, and getting them to integrate with the patient’s own tissue after transplant. They note progress is each of these areas, but in most cases much more work needs to be done.

The University of Rochester put out a press release detailing their faculty member’s contribution to the paper focusing on neural diseases. He suggests that complex diseases that impact multiple types of cells, such as Alzheimer’s, would be the most difficult to treat with stem cells. But diseases impacting a single type of nerve cell, such as Huntington’s, Parkinson’s and multiple sclerosis would be the first to benefit from cells generated from pluripotent stem cells. HealthCanal picked up the university’s release.

Don Gibbons

Tiny transparent zebra fish yields big clue to black box of Alzheimer’s disease

The PR folks at the Flanders Institute for Biotechnology in Belgium produced an unusual press release to describe recent work there published in Developmental Cell. They devoted the first half to the marvels of their animal model the zebra fish.

zebrafish1For those who have only seen these nearly transparent little guys in a home aquarium the story provides a nice explanation for why they are such popular lab models. It is not unusual to walk into a lab with dozens of small fish tanks holding thousands of zebra fish. A couple key reasons: their DNA matches 90 percent of ours and the guys reproduce quickly, just three months after birth.

Nerve stem cells, key players to brain development in the embryo, become few in number in adults. More important, those few we have left seem to be less active when we need them most, when Alzheimer’s disease or other neurodegenerative disease destroys some of our existing nerves. Evgenia Salta at the Institute used the fish to try to discern why.

We have known for some time that the genes in a pathway known as Notch regulate the ability of nerve stem cells to mature into adult nerves. But we don’t know why that goes awry in disease. She focused on a genetic regulatory molecule called a microRNA that is known to be in abnormally low supply in cells from patients with Alzheimer’s.

When they manipulated the fish to lower the levels of this microRNA, the nerve stem cells in the fish failed to mature properly into nerve cells. In the press release published on ScienceDaily Salta is quoted saying:

“To our surprise, the reduced activity of miRNS-132 in the zebra fish blocks the further ripening of the stem cell into nerves cells. This new knowledge about the molecular signaling pathway that underlies this process gives us an insight into the exact blocking mechanism. Thanks to this work in zebra fish, we can now examine in detail what exactly goes wrong in the brains of patients with Alzheimer’s disease.”

You can read about CIRMM-funded projects seeking solutions to Alzheimer’s Disease on our fact sheet.

Don Gibbons

CIRM funded therapy for type 1 diabetes gets FDA approval for clinical trial

diabetes

It’s always nice to start the week off with some good news and we got this week off to a great start with some great news. ViaCyte has been given the green light to start a clinical trial with its therapy for type 1 diabetes, a program we are funding.

ViaCyte applied to the Food and Drug Administration for approval in mid-July, a process that can sometimes take months. They got their approval in a matter of weeks, which, considering the device they are using is so novel and complicated, is a really significant achievement.

As the Chairman of our governing Board, Jonathan Thomas, J.D., Ph.D., noted in a press release we sent out about the news:

“This is a therapy that we have funded from its earliest days so it’s exciting to see that it is now ready to start a First-in-Human trial. Reaching this milestone is a tribute to years of hard work by the team at ViaCyte, but also to the vision of the people of California who created the stem cell agency to support work like this. That vision is one step closer to being realized.”

So what is this new approach that ViaCyte is trying? Well, in type 1 diabetes the pancreas no longer produces the insulin our bodies need to regulate blood sugar levels. That can increase your risk of heart disease, stroke, kidney failure, blindness, even death. ViaCyte has developed a thin plastic pouch, containing an immature form of pancreatic cells, to mimic the blood glucose regulating function of the pancreas. When the device is implanted under the skin these cells are designed to become the insulin-producing and other cells needed to regulate blood glucose levels. It is believed that these cells will be able to sense when blood glucose is high, and then secrete insulin to restore it to a healthy level.

It’s fascinating science but more than that, it’s a really promising program that has the potential to end reliance on daily testing and injections of insulin for people with type 1 diabetes. It could dramatically change their lives.

Of course this is just one step along the way and, encouraging as it is, it is also important to place it in context. This is the first time it’s being tried in people. In all the pre-clinical testing it’s looked promising, but this is the only test that really counts, seeing if it works in patients with type 1 diabetes. Now we get to find out.