CIRM 2.0: How to Build a Better Stem Cell Agency and Speed up Treatments to Patients

Change is never easy. We all get used to doing things in a certain way and it can sometimes be difficult to realize that the way we have chosen, while it may have worked well at one time is perhaps not the best way to achieve our goals at this time. Well, change is coming to the stem cell agency.

CIRM_LogoColor2_L_web_540x216

It’s not surprising that our new President & CEO, C. Randal Mills, Ph.D., would want to introduce some of his own ideas about how best to run the agency in the current moment of stem cell science. After all, it’s those ideas that landed him the job in the first place. Now Randy wants us to develop a clearer focus, one that is more aligned with his 4-point criteria for assessing everything we do.

  1. Will it speed up treatments to patients
  2. Will it increase the likelihood of successful treatments for patients
  3. Does it target an unmet medical need
  4. Is it efficient.

That new focus begins with re-imagining how we can be most effective in the way we fund research. Right now we put out what’s called an RFA or Request for Application, telling people who have promising projects in a particular area of stem cell research to submit an application and if they are successful they’ll get up to $20 million, depending on the kind of project.

The problem is, we often have long gaps between each round of funding and so a company or institution with a promising therapy will sometimes have to wait as much as a couple of years before they can apply again. If they do wait and are successful in their application it could still be another year or two before they are able to gain actual funding and begin a clinical trial. But when lives are at stake, you can’t afford to wait that long. So we’re looking at ways of speeding things up, making it easier for the best science to get the funds needed when they are needed.

At our Board meeting yesterday Randy outlined some broad concepts about what he wants to do and how it can be done. It’s part of his vision for the agency, a new focus that he is calling CIRM 2.0 (with acknowledgments to Dr. Paul Knoepfler who coined the term earlier this year)

As with any simple idea it’s really complicated. We need to achieve greater speed, to streamline the way we do things, without sacrificing the quality of the review process because we need to ensure that we only fund the best science.

In the months to come, as the precise details about these proposed changes are fine tuned, the Board will hear in greater detail how this will work and, as always, it will be up to them to decide if they think it’s a good idea.

Either way it will start a conversation about how we can become more efficient and more effective at living up to our mission, of accelerating therapies that target patients with unmet medical needs. And that always has to be a good thing.

For more details about the other big events at yesterday’s Board meeting, including awarding $16 million to ViaCyte to help it advance its promising therapy for type 1 diabetes, you can read the news release posted on our website.

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

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.

 

 

 

 

 

A Cool New Way of Raising Funds and Awareness

Raising money to help fight a disease is tough. Trying to raise awareness about the disease can be just as tough. Doing both together is positively masochistic; an experience that is often as rewarding as dumping a bucket of ice cold water over your head.

Have you taken the ALS Ice Bucket Challenge?

Have you taken the ALS Ice Bucket Challenge?

And that’s precisely what a growing number of people around the country are doing to raise awareness about—and money for research into—Amyotrophic Lateral Sclerosis (ALS) also known as Lou Gehrig’s disease. They are dumping buckets of ice-cold water on their head.

It’s called, not surprisingly, the Ice Bucket Challenge. The idea behind it is simple. You dare someone you know to dump a bucket of ice cold water over their head within the next 24 hours or make a donation to help fight ALS. Once the person you have challenged either completes the challenge or makes a donation they then challenge other people—usually three other people—to do the same. And of course there’s nothing stopping you both dumping the water on yourself and making a donation.

The idea started out with people who had ALS and their friends and family but it has quickly spread. Celebrities such as Facebook’s Mark Zuckerberg, singer/actor Justin Timberlake, TV newsman Matt Lauer and even New Jersey Governor Chris Christie have all taken the Challenge. In fact the campaign has gone viral with videos and pictures of people taking the Challenge popping up on social media – Facebook and Instagram in particular – at a bewildering rate.

It’s more than just an opportunity to laugh at a potential Presidential candidate taking a self-inflicted cold shower it’s also raising a ton of money. The ALS Association says it raised $4 million in donations between the end of July and August 12th. That’s more than three and a half times more than it raised during the same period last year. They have also added more than 70,000 new donors to their cause.

That money goes to research into finding new treatments for ALS because right now there is no effective therapy at all. It also goes to help people living with this nasty, debilitating and ultimately deadly disease.

In a blog on the ALS website Barbara Newhouse, the President and CEO of the ALS Association said:

“We have never seen anything like this in the history of the disease. We couldn’t be more thrilled with the level of compassion, generosity and sense of humor that people are exhibiting as they take part in this impactful viral initiative.”

What I love about this is not just that it is raising awareness and funds for a truly worthwhile cause but that it also shows how a little bit of creativity can create so much more interest in a disease, and the people suffering from it, than any amount of well-meaning, more traditional attempts at education.

At the Stem Cell Agency we have worked closely with our friends in the ALS Association for many years and they do terrific work (you can read about our funding on our ALS Fact Sheet). But it’s a relatively rare condition – only affecting some 30,000 people in the U.S. at any one time – so it always struggles to get people’s attention compared to bigger diseases such as Alzheimer’s or stroke. But with this campaign they have changed that. They have taken a simple idea, a simple challenge, and used it to open people’s eyes to what they can do to help fight back against a deadly disease.

I find that really refreshing. As refreshing as a bucket of water over my own head.

Kevin McCormack

A life long battle with Parkinson’s disease earns Dr. David Higgins a place on our Board

David Higgins, Ph.D., the new member of the stem cell agency Board as the Patient Advocate for Parkinson's disease

David Higgins, Ph.D., the new member of the stem cell agency Board as the Patient Advocate for Parkinson’s disease

As a child David Higgins imagined that the reason his grandmother wouldn’t talk to him was because of something he did, some fault of his. In time he came to realize that the reason was because his grandmother had Parkinson’s disease and she had trouble communicating with, not just him, but with everyone. It was the start of his life-long relationship with the disease, one that led to his own diagnosis with Parkinson’s a few years ago, and that has now led to him becoming the newest member of our governing Board, the Independent Citizens Oversight Committee.

David, now Dr. Higgins, was appointed by State Controller John Chiang. In his official letter announcing the news – which you can also find in our news release – he said:

“Diagnosed with Parkinson’s in 2011, his experience with the disease and its impact on a personal level brings a distinct perspective to the ICOC. Dr. Higgins also brings a broad range of experience from the biotechnology field. As a trained molecular biologist, his involvement in drug development and business operations places him in a unique position, understanding both science and process.”

David says he learned many important lessons from his grandmother, including the power of medical research:

“She was in one of the first L-dopa trials (this is a drug that converts into dopamine in the brain, to help counter the disease). I saw how that therapy helped change her life and it gave me my first lesson in how science and scientific research could change someone’s life.”

David’s mother was also diagnosed with, and later died from, a Parkinson’s-like form of dementia. His own diagnosis only served to further reinforce his commitment to doing something to fight the disease.

“I probably had symptoms that I was suspicious of for about two years before I was diagnosed. It was a mixed bag because as a scientist I finally had a label for what was going on and I thought at least now I can start fighting it. But the other side of me said “Oh my God I know exactly where this is going.”

As a research scientist he has held positions at several biotech companies, most recently as Head of US Operations for BioMedica, Inc., a UK-based gene therapy company. He also became a highly active Patient Advocate, and is now the California Assistant State Director with the Parkinson’s Action Network, as well as the new President of the Board of Directors of the Parkinson’s Association in San Diego.

David says he was completely stunned when he was asked if he would like to be on the CIRM Board. But the Chair of our Board, Jonathan Thomas, Ph.D., J.D., says it’s clear that he will be a terrific addition to the team:

“He is a powerful and passionate advocate not just for people with Parkinson’s disease but for science in general. He understands on a personal level how important it is for the public to support research trying to find new treatments and cures for currently incurable diseases.”

David says his personal experience with the disease has clearly influenced his life but does not mean he will focus on that alone:

“One thing I feel strongly about is that, yes I’m the Parkinson’s Patient Advocate on the Board, but first and foremost I’m an Advocate for everyone and I want to make sure that we spend our money wisely, and that we use our resources to identify and nurture the most promising stem cell projects across all target diseases.”

He says he takes up his new role with mixed emotions:

“I am thrilled to be a part of the CIRM governing Board, but I am also humbled to follow in the footsteps of Joan Samuelson, who has been CIRM’s Parkinson’s Patient Advocate from the beginning. She is a hard act to follow.”

Dr. Thomas echoed those sentiments saying:

“Joan set the gold standard for patient advocacy. She was a part of the agency from the very first and her passion, dedication and commitment to helping others was an inspiration to all of us. Joan was always willing to ask tough questions and push us all to do more to speed up the development of new therapies. We are all better for her service, and she will be missed.”

We are delighted to have David join us and we’re looking forward to working with him in the years to come.

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 

Bridging the gap: helping create a new generation of stem cell scientists

Inspiration comes in many different shapes and sizes, but when you see it there is no mistaking it. And when you meet and talk to the students in our Bridges program you find inspiration in each and every one of them.

The program is designed to train the next generation of stem cell scientists, bridging (hence the name) the gap between undergraduate and Master’s level training in research. But it’s so much more than just a recruiting and training program because one of the goals of Bridges is to find students who are often overlooked for opportunities like this: students who may be the first in their family to go to college, who don’t come from a wealthy family or fancy school. These students seize the opportunity with both hands and their sense of delight at being given a chance, and enthusiasm for the work is exciting and infectious.

We held our annual Bridges Trainee Meeting in Burlingame this week, a chance for all the students in the program to come together, listen to lectures from world-class stem cell researchers, and show their posters describing the work they have done over the past year.

At first many of them seem a little shy but once you ask them about their experiences their enthusiasm simply bubbles over. Shayda Kianfar graduated from Berkeley City College and is now studying at the University of California, Berkeley. She says she was accepted into the program even though she had no prior lab experience:

“This has given me an amazing experience. To be surrounded by so many incredible people, to have great mentors is life changing. You learn so many new skills and it opens your eyes. I hadn’t thought about stem cell work before but now I would love to do this. It’s so exciting.”

Kevin Martinez talks to fellow Bridges student David James

Kevin Martinez talks to fellow Bridges student David James

Kevin Martinez graduated from San Francisco State University and says getting a chance to work with extraordinary researchers like Thea Tlsty, Ph.D., at the University of California, San Francisco, was incredible. Kevin got to work with Tlsty and her team on their discovery that certain rare cells extracted from adult breast tissue can be instructed to become different types of cells – a discovery that could have important potential for regenerative medicine.

He says what surprised him most of all was how much independence they gave him, he wasn’t treated like a student but like a colleague:

“They trained me and gave me the experience and opportunity to do amazing work. This is great training for a career either in academia or industry because they teach you how to do research independently, but to also work as part of a team.”

Eleanor Kim, spent her year at City of Hope near Los Angeles. She focused on leukemia stem cells (LCS), testing different medications to see if they could be effective at preventing recurrence of the leukemia or the speed with which it spreads.

Bridges student Eleanor Kim

Bridges student Eleanor Kim

Eleanor was a pre-med student who hadn’t really thought about research until she found out about the Bridges program. Now she’s set her sights on becoming an MD/PhD:

“This got me much more interested in the biology of cancers, what is driving them, what controls them. I want to be able to talk to my patients about what is happening to them but also to be able to do research that might be able to help them.”

Eleanor says she also learned a valuable lesson about the need for a good night’s sleep:

“I learned that you have to work hard but that you also can’t work to the point where you are sleep deprived. This is such detail-oriented work that being sleepy can lead to mistakes and one mistake can set you back days.”

Each Bridges student has their own story; each brings their own unique perspective to their work and to the field. You can hear some of our students talk about how important this opportunity was for them, and how it has changed them in so many ways.

kevin mccormack