A look back at the last year – but with our eyes firmly on the future

Randy

CIRM President & CEO Randy Mills doesn’t want “good”, he wants “better”

Better.

With that single word Randy Mills, our President and CEO, starts and ends his letter in our 2015 Annual Report and lays out the simple principle that guides the way we work at CIRM.

Better.

But better what?

“Better infrastructure to translate early stage ideas into groundbreaking clinical trials. Better regulatory practices to advance promising stem cell treatments more efficiently. Better treatments for patients in need.”

“Better” is also the standard everyone at CIRM holds themselves to. Getting better at what we do so we can fulfill our mission of accelerating stem cell treatments to patients with unmet medical needs.

The 2015 Annual Report highlights the achievements of the last year, detailing how we invested $135 million in 47 different projects at all levels of research. How our Board unanimously passed our new Strategic Plan, laying out an ambitious series of goals for the next five years from funding 50 new clinical trials, to creating a new regulatory process for stem cell therapies.

Snapshot of CIRM's 2015 Funding

The report offers a snapshot of where our money has gone this year, and how much we have left. It breaks down what percentage of our funding has gone to different diseases and how much we have spent on administration.

Jonathan Thomas, the Chair of our Board, takes a look back at where we started, 10 years ago, comparing what we did then (16 awards for a total of $12.5 million) to what we are doing today. His conclusion; we’re doing better.

But we still have a long way to go. And we are determined to get even better.

P.S. By the way we are changing the way we do our Annual Report. Our next one will come out on January 1, 2017. We figured it just made sense to take a look back at the last year as soon as the new year begins. It gives you a better (that word again) sense of what we did and where we  are heading. So look out for that, coming sooner than you think.

Dr. Deborah Deas joins CIRM Board

Deborah Deas has been appointed dean of the UCR School of Medicine

Deborah Deas, MD, MPH, UCR School of Medicine

Dr. Deborah Deas is clearly not someone who opts for the quiet life. If she were, she would have stayed home in Adams Run, the tiny town in rural South Carolina where she was born.

The website, NeighborhoodScout.com describes Adams Run (current population 1,492) as:

“One of the quietest neighborhoods in America. When you are here, you will find it to be very quiet. If quiet and peaceful are your cup of tea, you may have found a great place for you.”

Dr. Deas obviously wasn’t a tea drinker because she packed her bags and went off to college in Charleston. That was the first step on a journey that led the self-described “farmer’s daughter” to become an MD, then an MPH (Masters in Public Health), before assuming a leadership role at the Medical University of South Carolina (MUSC). More recently she headed to California’s Inland Empire where she was named the Dean and CEO for Clinical Affairs of the UC Riverside School of Medicine.

And now we are delighted to add to that list of achievements by announcing she is the newest member of the CIRM Board.

She was appointed to the Board by state Treasurer John Chiang who praised her for her:

“Passion to improve  health for underserved populations and to diversify the health care work force. She is committed to making the benefits of advanced medicine available to all Californians.”

 

In a news release our CIRM Board Chair, Jonathan Thomas, was equally fulsome in his praise and welcome to Dr. Deas.

 “We are delighted to have someone with Dr. Deas’ broad experience and expertise join us at CIRM. Her medical background and her commitment to diversity and inclusion are important qualities to bring to a Board that is striving to deliver stem cell treatments to patients, and to reflect the diversity of California.”

To say that she brings a broad array of skills and experience to the Board is something of an understatement. She is board certified in adult psychiatry, child and adolescent psychiatry and addiction psychiatry, and is widely regarded as a national leader in research into youth binge drinking, adolescent nicotine dependence, marijuana use and panic disorder, and pharmaceutical treatment of pediatric depressive disorder.

As if that wasn’t enough, she has also been named as one of the best doctors in the U.S. by U.S. News & World Report for the last eight years.

But the road to UC Riverside and CIRM hasn’t always been easy. In a first person perspective in Psychiatric News.

she said that at MUSC she was just one of two African Americans among the 500 residents in training:

“It was not uncommon for me to be mistaken by many for a social worker, a secretary, or a ward clerk despite wearing my white coat with Deborah Deas, M.D., written on it. This mistake was even made by some of my M.D. peers. I found that the best response was to ask, “And just why do you think I am a social worker?”

She says the lessons she learned from her parents and grandparents helped sustain her:

“They emphasized the importance of setting goals and keeping your eyes on the prize. Service was important, and the ways that one could serve were numerous. The notion that one should learn from others, as well as teach others, was as common as baked bread. My parents instilled in me that education is the key to a fruitful future and that it is something no one can take away from you.”

Her boss at UC Riverside, the Provost and Executive Vice Chancellor, Paul D’Anieri said Dr. Deas is a great addition to the CIRM Board:

“Deborah is a public servant at heart. Her own values and goals to help underserved patient populations align with the goals of CIRM to revolutionize medicine and bring new, innovative treatments to all patients who can benefit. I am confident that Dr. Deas’ service will have a lasting positive impact for CIRM and for the people of California.”

Dr. Deas ends her article in Psychiatric News saying:

“The farmer’s daughter has come a long way. I have stood on the shoulders of many, pushing forward with an abiding faith that there was nothing that I could not accomplish.”

She has indeed come a long way. We look forward to being a part of the next stage of her journey, and to her joining CIRM and bringing that “abiding faith” to our work.

 

 

Stem cell stories that caught our eye: turning on T cells; fixing our brains; progress and trends in stem cells; and one young man’s journey to recover from a devastating injury

Healthy_Human_T_Cell

A healthy T cell

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.

Directing the creation of T cells. To paraphrase the GOP Presidential nominee, any sane person LOVES, LOVES LOVES their T cells, in a HUGE way, so HUGE. They scamper around the body getting rid of viruses and the tiny cancers we all have in us all the time. A CIRM-funded team at CalTech has worked out the steps our genetic machinery must take to make more of them, a first step in letting physicians turn up the action of our immune systems.

We have known for some time the identity of the genetic switch that is the last, critical step in turning blood stem cells into T cells, but nothing in our body is as simple as a single on-off event. The Caltech team isolated four genetic factors in the path leading to that main switch and, somewhat unsuspected, they found out those four steps had to be activated sequentially, not all at the same time. They discovered the path by engineering mouse cells so that the main T cell switch, Bcl11b, glows under a microscope when it is turned on.

“We identify the contributions of four regulators of Bcl11b, which are all needed for its activation but carry out surprisingly different functions in enabling the gene to be turned on,” said Ellen Rothenberg, the senior author in a university press release picked up by Innovations Report. “It’s interesting–the gene still needs the full quorum of transcription factors, but we now find that it also needs them to work in the right order.”

Video primer on stem cells in the brain.  In conjunction with an article in its August issue, Scientific American posted a video from the Brain Forum in Switzerland of Elena Cattaneo of the University of Milan explaining the basics of adult versus pluripotent stem cells, and in particular how we are thinking about using them to repair diseases in the brain.

The 20-minute talk gives a brief review of pioneers who “stood alone in unmarked territory.” She asks how can stem cells be so powerful; and answers by saying they have lots of secrets and those secrets are what stem cell scientist like her are working to unravel.  She notes stem cells have never seen a brain, but if you show them a few factors they can become specialized nerves. After discussing collaborations in Europe to grow replacement dopamine neurons for Parkinson’s disease, she went on to describe her own effort to do the same thing in Huntington’s disease, but in this case create the striatal nerves lost in that disease.

The video closes with a discussion of how basic stem cell research can answer evolutionary questions, in particular how genetic changes allowed higher organisms to develop more complex nervous systems.

kelley and kent

CIRM Science Officers Kelly Shepard and Kent Fitzgerald

A stem cell review that hits close to home.  IEEE Pulse, a publication for scientists who mix engineering and medicine and biology, had one of their reporters interview two of our colleagues on CIRM’s science team. They asked senior science officers Kelly Shepard and Kent Fitzgerald to reflect on how the stem cell field has progressed based on their experience working to attract top researchers to apply for our grants and watching our panel of outside reviewers select the top 20 to 30 percent of each set of applicants.

One of the biggest changes has been a move from animal stem cell models to work with human stem cells, and because of CIRM’s dedicated and sustained funding through the voter initiative Proposition 71, California scientists have led the way in this change. Kelly described examples of how mouse and human systems are different and having data on human cells has been critical to moving toward therapies.

Kelly and Kent address several technology trends. They note how quickly stem cell scientists have wrapped their arms around the new trendy gene editing technology CRISPR and discuss ways it is being used in the field. They also discuss the important role of our recently developed ability to perform single cell analysis and other technologies like using vessels called exosomes that carry some of the same factors as stem cells without having to go through all the issues around transplanting whole cells.

“We’re really looking to move things from discovery to the clinic. CIRM has laid the foundation by establishing a good understanding of mechanistic biology and how stem cells work and is now taking the knowledge and applying it for the benefit of patients,” Kent said toward the end of the interview.

jake and family

Jake Javier and his family

Jake’s story: one young man’s journey to and through a stem cell transplant; As a former TV writer and producer I tend to be quite critical about the way TV news typically covers medical stories. But a recent story on KTVU, the Fox News affiliate here in the San Francisco Bay Area, showed how these stories can be done in a way that balances hope, and accuracy.

Reporter Julie Haener followed the story of Jake Javier – we have blogged about Jake before – a young man who broke his spine and was then given a stem cell transplant as part of the Asterias Biotherapeutics clinical trial that CIRM is funding.

It’s a touching story that highlights the difficulty treating these injuries, but also the hope that stem cell therapies holds out for people like Jake, and of course for his family too.

If you want to see how a TV story can be done well, this is a great example.

CIRM Board targets diabetes and kidney disease with big stem cell research awards

diabetes2

A recent study  estimated there may be more than 500 million people worldwide who have diabetes. That’s an astounding figure and makes diabetes one of the largest chronic disease epidemics in human history.

One of the most serious consequences of untreated or uncontrolled diabetes is kidney damage. That can lead to fatigue, weakness, confusion, kidney failure and even death. So two decisions taken by the CIRM Board today were good news for anyone already suffering from either diabetes or kidney disease. Or both.

The Board awarded almost $10 million to Humacyte to run a Phase 3 clinical trial of an artificial vein needed by people undergoing hemodialysis – that’s the most common form of dialysis for people with kidney damage. Hemodialysis helps clean out impurities and toxins from the blood. Without it waste will build up in the kidneys with devastating consequences.

The artificial vein is a kind of bioengineered blood vessel. It is implanted in the individual’s arm and, during dialysis, is connected to a machine to move the blood out of the body, through a filter, and then back into the body. The current synthetic version of the vein is effective but is prone to clotting and infections, and has to be removed regularly. All this puts the patient at risk.

Humacyte’s version – called a human acellular vessel or HAV – uses human cells from donated aortas that are then seeded onto a biodegradable scaffold and grown in the lab to form the artificial vein. When fully developed the structure is then “washed” to remove all the cellular tissue, leaving just a collagen tube. That is then implanted in the patient, and their own stem cells grow onto it, essentially turning it into their own tissue.

In earlier studies Humacyte’s HAV was shown to be safer and last longer than current versions. As our President and CEO, Randy Mills, said in a news release, that’s clearly good news for patients:

“This approach has the potential to dramatically improve our ability to care for people with kidney disease. Being able to reduce infections and clotting, and increase the quality of care the hemodialysis patients get could have a significant impact on not just the quality of their life but also the length of it.”

There are currently almost half a million Americans with kidney disease who are on dialysis. Having something that makes life easier, and hopefully safer, for them is a big plus.

The Humacyte trial is looking to enroll around 350 patients at three sites in California; Sacramento, Long Beach and Irvine.

While not all people with diabetes are on dialysis, they all need help maintaining healthy blood sugar levels, particularly people with type 1 diabetes. That’s where the $3.9 million awarded to ViaCyte comes in.

We’re already funding a clinical trial with ViaCyte  using an implantable delivery system containing stem cell-derived cells that is designed to measure blood flow, detect when blood sugar is low, then secrete insulin to restore it to a healthy level.

This new program uses a similar device, called a PEC-Direct. Unlike the current clinical trial version, the PEC-Direct allows the patient’s blood vessels to directly connect, or vasularize, with the cells inside it. ViaCyte believes this will allow for a more robust engraftment of the stem cell-derived cells inside it and that those cells will be better able to produce the insulin the body needs.

Because it allows direct vascularization it means that people who get the delivery system  will also need to get chronic immune suppression to stop their body’s immune system attacking it. For that reason it will be used to treat patients with type 1 diabetes that are at high risk for acute complications such as severe hypoglycemic (low blood sugar) events associated with hypoglycemia unawareness syndrome.

In a news release Paul Laikind, Ph.D., President and CEO of ViaCyte, said this approach could help patients most at risk.

“This high-risk patient population is the same population that would be eligible for cadaver islet transplants, a procedure that can be highly effective but suffers from a severe lack of donor material. We believe PEC-Direct could overcome the limitations of islet transplant by providing an unlimited supply of cells, manufactured under cGMP conditions, and a safer, more optimal route of administration.”

The Board also approved more than $13.6 million in awards under our Discovery program. You can see the winners here.

 

CIRM-funded stem cell clinical trial for retinitis pigmentosa focuses on next stage

rp1

How retinitis pigmentosa erodes normal vision

The failure rate for clinical trials is depressingly high. A study from Tufts University in 2010  found that for small molecules – the substances that make up more than 90 percent of the drugs on the market today – the odds of getting from a Phase 1 trial to approval by the Food and Drug Administration are just 13 percent. For stem cell therapies the odds are even lower.

That’s why, whenever a stem cell therapy shows good results it’s an encouraging sign, particularly when that therapy is one that we at CIRM are funding. So we were more than a little happy to hear that Dr. Henry Klassen and his team at jCyte and the University of California, Irvine have apparently cleared the first hurdle with their treatment for retinitis pigmentosa (RP).

jCyte has announced that the first nine patients treated for RP have shown no serious side effects, and they are now planning the next phase of their Phase 1/2a safety trial.

In a news release Klassen, the co-founder of jCyte, said:

“We are pleased with the results. Retinitis pigmentosa is an incurable retinal disease that first impacts people’s night vision and then progressively robs them of sight altogether. This is an important milestone in our effort to treat these patients.”

The therapy involves injecting human retinal progenitor cells into one eye to help save the light sensing cells that are destroyed by the disease. This enables the researchers to compare the treated eye with the untreated eye to see if there are any changes or improvements in vision.

So far, the trial has undergone four separate reviews by the Data Safety Monitoring Board (DSMB), an independent group of experts that examines data from trials to ensure they meet all safety standards and that results show patients are not in jeopardy. Results from the first nine people treated are encouraging.

The approach this RP trial is taking has a couple of advantages. Often when transplanting organs or cells from one person into another, the recipient has to undergo some kind of immunosuppression, to stop their body rejecting the transplant. But earlier studies show that transplanting these kinds of progenitor cells into the eye doesn’t appear to cause any immunological response. That means patients in the study don’t have to undergo any immunosuppression. Because of that, the procedure is relatively simple to perform and can be done in a doctor’s office rather than a hospital. For the estimated 1.5 million people worldwide who have RP that could make getting treatment relatively easy.

Of course the big question now is not only was it safe – it appears to be – but does it work? Did any of those people treated experience improvements in their vision? We will share those results with you as soon as the researchers make them available.

Next step for the clinical trial is to recruit more patients, and treat them with a higher number of cells. There’s still a long way to go before we will know if this treatment works, if it either slows down, stops, or better still helps reverse some of the effects of RP. But this is a really encouraging first step.


Related links:

Stem cell transplant offers Jake a glimpse of hope

Jake

Jake Javier surrounded by friends; Photo courtesy Julie Haener KTVU

On Thursday, July 7th, Jake Javier became the latest member of a very select group. Jake underwent a stem cell transplant for a spinal cord injury at Santa Clara Valley Medical Center here in the San Francisco Bay Area.

The therapy is part of the CIRM-funded clinical trial run by Asterias Biotherapeutics. For Asterias it meant it had hit a significant milestone (more on that later). But for Jake, it was something far more important. It was the start of a whole new phase in his life.

Jake seriously injured his spinal cord in a freak accident after diving into a swimming pool just one day before he was due to graduate from San Ramon Valley high school. Thanks, in part, to the efforts of the tireless patient advocate and stem cell champion Roman Reed, Jake was able to enroll in the Asterias trial.

astopc1The goal of the trial is to test the safety of transplanting three escalating doses of AST-OPC1 cells. These are a form of cell called oligodendrocyte progenitors, which are capable of becoming several different kinds of brain cells, some of which play a supporting role and help protect nerve cells in the central nervous system – the area damaged in spinal cord injury.

To be eligible, individuals have to have experienced a severe neck injury in the last 30 days, one that has left them with no sensation or movement below the level of their injury, and that means they have typically lost all lower limb function and most hand and arm function.

The first group of three patients was completed in August of last year. This group was primarily to test for safety, to make sure this approach was not going to cause any harm to patients. That’s why the individuals enrolled were given the relatively small dose of 2 million cells. So far none of the patients have experienced any serious side effects, and some have even shown some small improvements.

In contrast, the group Jake is in were given 10 million cells each. Jake was the fifth person treated in this group. That means Asterias can now start assessing the safety data from this group and, if there are no problems, can plan on enrolling people for group 3 in about two months. That group of patients will get 20 million cells.

It’s these two groups, Jakes and group 3, that are getting enough cells that it’s hoped they will see some therapeutic benefits.

In a news release, Steve Cartt, President and CEO of Asterias, said they are encouraged by the progress of the trial so far:

“Successful completion of enrollment and dosing of our first efficacy cohort receiving 10 million cells in our ongoing Phase 1/2a clinical study represents a critically important milestone in our AST-OPC1 clinical program for patients with complete cervical spinal cord injuries. In addition, while it is still very early in the development process and the patient numbers are quite small, we are encouraged by the upper extremity motor function improvements we have observed so far in patients previously enrolled and dosed in the very low dose two million cell cohort that had been designed purely to evaluate safety.”

 

jake and familyJake and his family are well aware that this treatment is not going to be a cure, that he won’t suddenly get up and walk again. But it could help him in other, important ways, such as possibly getting back some ability to move his hands.

The latest news is that Jake is doing well, that he experienced some minor problems after the surgery but is bouncing back and is in good spirits.

Jake’s mother Isabelle said this has been an overwhelming experience for the family, but they are getting through it thanks to the love and support of everyone who hears Jake’s story. She told CIRM:

 “We are all beyond thrilled to have an opportunity of this magnitude. Just the thought of Jake potentially getting the use of his hands back gives him massive hope. Jake has a strong desire to recover to the highest possible level. He is focused and dedicated to this process. You have done well to choose him for your research. He will make you proud.”

He already has.

Jake and Brady gear

New England Patriots star quarterback Tom Brady signed a ball and jersey for Jake after hearing about the accident


Related Links:

Accelerating the drive for new stem cell treatments

Acceleration

Acceleration is defined as the “increase in the rate or speed of something.” For us that “something” is new stem cell treatments for patients with unmet medical needs. Today our governing Board just approved a $15 million partnership with Quintiles to help us achieve that acceleration.

Quintiles was awarded the funding to create a new Accelerating Center. The goal of the center is to give stem cell researchers the support they need to help make their clinical trials successful.

As our President and CEO Randy Mills said in a news release:

randy-at-podium1CIRM President Randy Mills addresses the CIRM Board

“Many scientists are brilliant researchers but have little experience or expertise in running a clinical trial; this Accelerating Center means they don’t have to develop those skills; we provide them for them. This partnership with Quintiles means that scientists don’t have to learn how to manage patient enrollment or how to create a data base to manage the results. Instead they are free to focus on what they do best, namely science.”

How does it work? Well, if a researcher has a promising therapy and approval from the US Food and Drug Administration (FDA) to start a clinical trial, the Accelerating Center helps them get that trial off the ground. It helps them find the patients they need, get those patients consented and ready for the trial, and then helps manage the trial and the data from the trial.

The devil is in the details

Managing those details can be a key factor in determining whether a clinical trial is going to be successful. Last year, a study in the New England Journal of Medicine listed the main reasons why clinical trials fail.

Among the reasons are:

  • Poor study design: Selecting the wrong patients, the wrong dosing and the wrong endpoint, as well as bad data and bad site management cause severe problems.
  • Poor management: A project manager who does not have enough experience in costing and conducting clinical trials will lead to weak planning, with no clear and real timelines, and to ultimate failure.

We hope our partnership with Quintiles in this Accelerating Center will help researchers avoid those and the other pitfalls. As the world’s largest provider of biopharmaceutical development and commercial outsourcing services, Quintiles has a lot of experience and expertise in this area. On its Twitter page it’s slogan is “Better, smarter, faster trials” so I think we made a smart choice.

When Randy Mills first pitched this idea to the Board, he said that he is a great believer in “not asking fish to learn how to fly, they should just do what they do best”.

The Accelerating Center means scientists can do what they do best, and we hope that leads to what patients need most; treatments and cures.


Related Links:

Helping stem cells sleep can boost their power to heal

Mouse muscle

Mighty mouse muscle cells

We are often told that sleep is one of the most important elements of a healthy lifestyle, that it helps in the healing and repair of our heart and blood vessels – among other things.

It turns out that sleep, or something very similar, is equally important for stem cells, helping them retain their power or potency, which is a measure of their effectiveness and efficiency in generating the mature adult cells that are needed to repair damage. Now researchers from Stanford, with a little help from CIRM, have found a way to help stem cells get the necessary rest before kicking in to action. This could pave the way for a whole new approach to treating a variety of genetic disorders such as muscular dystrophy.

Inside out

One problem that has slowed down the development of stem cell therapies has been the inability to manipulate stem cells outside of the body, without reducing their potency. In the body these cells can remain quiescent or dormant for years until called in to action to repair an injury. That’s because they are found in a specialized environment or niche, one that has very particular physical, chemical and biological properties. However, once the stem cells are removed from that niche and placed in a dish in the lab they become active and start proliferating and changing into other kinds of cells.

You might think that’s good, because we want those stem cells to change and mature, but in this case we don’t, at least not yet. We want them to wait till we return them to the body to do their magic. Changing too soon means they have less power to do that.

Researchers at Stanford may have found a way to stop that happening, by creating an environment in the lab that more closely resembles that in the body, so the stem cells remain dormant longer.

As senior author, Thomas Rando, said in a Stanford news release, they have found a way to keep the stem cells dormant longer:

Dr. Thomas Rando, Stanford

Dr. Thomas Rando, Stanford

“Normally these stem cells like to cuddle right up against their native muscle fibers. When we disrupt that interaction, the cells are activated and begin to divide and become less stemlike. But now we’ve designed an artificial substrate that, to the cells, looks, smells and feels like a real muscle fiber. When we also bathe these fibers in the appropriate factors, we find that the stem cells maintain high-potency and regenerative capacity.”

Creating an artificial home

When mouse muscle stem cells (MuSCs) are removed from the mouse they lose their potency after just two days. So the Stanford team set out to identify what elements in the mouse niche helped the cells remain dormant. They identified the molecular signature of the quiescent MuSCs and used that to help screen different compounds to see which ones could help keep those cells dormant, even after they were removed from the mouse and collected in a lab dish.

They whittled down the number of potential compounds involved in this process from 50 to 10, and then tested these in different combinations until they found a formulation that kept the stem cells quiescent for at least 2 days outside of the mouse.

But that was just the start. Next they experimented with different kinds of engineered muscle fibers, to simulate the physical environment inside the mouse niche. After testing various materials, they found that the one with the greatest elasticity was the most effective and used that to create a kind of scaffold for the stem cells.

The big test

The artificial niche they created clearly worked in helping keep the MuSCs in a dormant state outside of the mouse. But would they work when transplanted back into the mouse? To answer this question they tested these stem cells to see if they retained their ability to self-renew and to change into other kinds of cells in the mouse. The good news is they did, and were far more effective at both than MuSCs that had not been stored in the artificial niche.

So, great news for mice but what about people, would this same approach work with human muscle stem cells (hMuSCs)? They next tested this approach using hMuSCs and found that the hMuSCs cultured on the artificial niche were more effective at both self-renewal and retaining their potency than hMuSCs kept in more conventional conditions, at least in the lab.

In the study, published in the journal Nature Biotechnology, the researchers say this finding could help overcome some of the challenges that have slowed down the development of effective therapies:

“Research on MuSCs, hematopoietic stem cells and neural stem cells has shown that very small numbers of quiescent stem cells, even single cells, can replace vast amounts of tissue; culture systems that that maintain stem cell quiescence may allow these findings to be translated to clinical practice. In addition, the possibility of culturing hMuSCs for longer time periods without loss of potency in order to correct mutations associated with genetic disorders, such as muscular dystrophy, followed by transplantation of the corrected cells to replace the pathogenic tissue may enable improved stem cell therapeutics for muscle disorders.”

What’s the big idea? Or in this case, what’s the 19 big ideas?

supermarket magazineHave you ever stood in line in a supermarket checkout line and browsed through the magazines stacked conveniently at eye level? (of course you have, we all have). They are always filled with attention-grabbing headlines like “5 Ways to a Slimmer You by Christmas” or “Ten Tips for Rock Hard Abs” (that one doesn’t work by the way).

So with those headlines in mind I was tempted to headline our latest Board meeting as: “19 Big Stem Cell Ideas That Could Change Your Life!”. And in truth, some of them might.

The Board voted to invest more than $4 million in funding for 19 big ideas as part of CIRM’s Discovery Inception program. The goal of Inception is to provide seed funding for great, early-stage ideas that may impact the field of human stem cell research but need a little support to test if they work. If they do work out, the money will also enable the researchers to gather the data they’ll need to apply for larger funding opportunities, from CIRM and other institutions, in the future

The applicants were told they didn’t have to have any data to support their belief that the idea would work, but they did have to have a strong scientific rational for why it might

As our President and CEO Randy Mills said in a news release, this is a program that encourages innovative ideas.

Randy Mills, Stem Cell Agency President & CEO

Randy Mills, CIRM President & CEO

“This is a program supporting early stage ideas that have the potential to be ground breaking. We asked scientists to pitch us their best new ideas, things they want to test but that are hard to get funding for. We know not all of these will pan out, but those that do succeed have the potential to advance our understanding of stem cells and hopefully lead to treatments in the future.”

So what are some of these “big” ideas? (Here’s where you can find the full list of those approved for funding and descriptions of what they involve). But here are some highlights.

Alysson Muotri at UC San Diego has identified some anti-retroviral drugs – already approved by the Food and Drug Administration (FDA) – that could help stop inflammation in the brain. This kind of inflammation is an important component in several diseases such as Alzheimer’s, autism, Parkinson’s, Lupus and Multiple Sclerosis. Alysson wants to find out why and how these drugs helps reduce inflammation and how it works. If he is successful it is possible that patients suffering from brain inflammation could immediately benefit from some already available anti-retroviral drugs.

Stanley Carmichael at UC Los Angeles wants to use induced pluripotent stem (iPS) cells – these are adult cells that have been genetically re-programmed so they are capable of becoming any cell in the body – to see if they can help repair the damage caused by a stroke. With stroke the leading cause of adult disability in the US, there is clearly a big need for this kind of big idea.

Holger Willenbring at UC San Francisco wants to use stem cells to create a kind of mini liver, one that can help patients whose own liver is being destroyed by disease. The mini livers could, theoretically, help stabilize a person’s own liver function until a transplant donor becomes available or even help them avoid the need for liver transplantation in the first place. Considering that every year, one in five patients on the US transplant waiting list will die or become too sick for transplantation, this kind of research could have enormous life-saving implications.

We know not all of these ideas will work out. But all of them will help deepen our understanding of how stem cells work and what they can, and can’t, do. Even the best ideas start out small. Our funding gives them a chance to become something truly big.


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A Dream made me change my mind. Almost.

Dream Alliance

Dream Alliance: photo courtesy Daily Telegraph, UK

On Friday I was faced with the real possibility that a horse had made an ass out of me.

Over the years we have written many articles about the risks of unproven stem cell therapies, treatments that have not yet been shown in clinical trials to be safe and effective. Often we have highlighted the cases of high profile athletes who have undergone stem cell treatments for injuries when there is little evidence that the treatments they are getting work.

Well, on Friday I saw an athlete who bounced back from a potentially career-ending injury to enjoy an amazing career thanks to a stem cell treatment. I wondered if I was going to have to revise my thoughts on this topic. Then my wife pointed out to me that the athlete was a horse.

We had been watching the movie Dark Horse, a truly delightful true story about a group of working class people in a Welsh mining village who bred and raised a horse that went on to great success as a race horse – often beating out thoroughbreds that were worth millions of dollars.

 

At one point the horse, Dream Alliance, suffered an almost fatal injury. Everyone assumed his career was over. But thanks to a stem cell treatment he was able to return to the track and became the first horse to win a major race after undergoing stem cell surgery.

It shouldn’t be too surprising that stem cells can help heal serious injuries in horses, the researchers at UC Davis have been using them to help treat horses for years – with great success. The danger comes in then assuming that just because stem cells work for horses, they’ll work for people. And that if they can cure one kind of injury, why not another.

That thought was driven home to me on Saturday when I was giving a talk to a support group for ALS or Lou Gehrig’s disease. ALS is a nasty, rapidly progressive disease that attacks the motor nerve cells in the brain and spinal cord, destroying a person’s ability to move, eat, speak or breath.

One person asked about a clinic they had been talking to which claimed it might be able to help them. The clinic takes fat from the person with ALS, isolates the stem cells in the fat and injects it back into the person. The clinic claims it’s been very effective in treating injuries such as torn muscles, and that it also works for other problems like Parkinson’s so it might help someone with ALS.

And that’s the problem. We hear about one success story that seems to prove stem cells can do amazing things, and then we are tempted to hope that if it works for one kind of injury, it might work for another, or even for a neurodegenerative disease.

And hope doesn’t come cheap. The cost of the procedure was almost $10,000.

If you have a disease like ALS for which there is no cure, and where the life expectancy is between two to five years, you can understand why someone would be tempted to try anything, no matter how implausible. What is hard is when you have to tell them that without any proof that it works, and little scientific rational as to why it would work, that it’s hard to recommend they try using their own fat cells to treat their ALS.

At CIRM we are investing more than $56.5 million in 21 different projects targeting ALS.   We are hopeful one of them, Clive Svendsen’s research at Cedars-Sinai Medical Center,  will soon get approval from the FDA to start a clinical trial.

Much as we would like to believe in miracles, medical breakthroughs usually only come after years of hard, methodical work. It would be great if injecting your own fat-derived stem cells into your body could cure you of all manner of ailments. But there’s no evidence to suggest it will.

The movie Dark Horse shows that for one horse, for one group of people in a small Welsh mining village, stem cells helped create a happy ending. We are hoping stem cells will one day offer the same sense of hope and possibility for people battling deadly diseases like ALS. But that day is not yet here.