Stem Cell Awareness Day: Past, Present, Future

In 2008, the then California Governor Arnold Schwarzenegger  declared Sept. 25 to be Stem Cell Awareness Day. In the proclamation he said, ”The discoveries being made today in our Golden State will have a great impact on many around the world for generations to come.”

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Bob Klein (Left), Arnold Schwarzenegger (Middle), Don Reed (Right) in 2008.

In the years since, we have moved steadily towards turning those words into reality and using Stem Cell Awareness Day, now celebrated on the second Wednesday in October, as a symbol of the progress being made, not just in California but around the world.

Yesterday, for example, at a public event at UC Davis in Sacramento, Dr. Jan Nolta told an audience of patients, patient advocates, researchers and stem cell supporters that “we are part of a new era in medicine, one where it will one day be routine for prescriptions to be written for stem cell treatments for many different diseases.”

Those sentiments were echoed by Jonathan Thomas, Chair of the CIRM Board, who said:

“This is a time of truly extraodinary medical science.  We are lucky because, in our lifetime, we are going to see many of the biggest maladies plaguing people cured, in part because of developments in regenerative medicine. Every week you read about extraordinary developments in medicine and often those are here in California.”

In the early years Stem Cell Awareness Day was very much a creation of CIRM. We worked closely with our partners in academia and industry to host or stage events around the state. In 2009 for example, more than 40 CIRM grantees went to high schools in California, talking about stem cell research to more than 3,000 students. We also coordinated with researchers in Canada and Australia to create a global community of supporters.

We even hosted a poetry competition. No, really, we did. So, clearly not every idea we had back then was a winner.

These days CIRM doesn’t play as prominent a role in organizing these events for a very simple reason. We don’t have to. They have become such a popular part of the scientific calendar that individual institutions and schools organize their own events, without any pushing or prodding from us (though we are always happy to help when asked).

At UC Irvine this afternoon there is an Open House where you can take a self-guided tour of the facility, meet some of the scientists and watch lab demonstrations.

This weekend the UC  Berkeley’s Student Society of Stem Cell Research (SSSCR) is hosting its 5th annual Stem Cell Conference: Culturing a Stem Cell Community. This conference aims to bring together different aspects of stem cell research, from science to advocacy, to demonstrate the growth and success of the field. You can RSVP on Eventbrite (tickets cost a small fee of $7 or $12 including lunch to support the cost of the SSSCR conference)

The Gladstone Institutes in San Francisco just posted two new videos to its YouTube site:

In the early days of CIRM, Stem Cell Awareness Day was a valuable way for us to talk directly to the people of California – the ones who created CIRM. We felt it was important to let them know how their money was being spend and about the progress being made in stem cell research. And in the early years that progress was slower than all of us would have liked. Today, it’s a very different situation with CIRM now having funded 40 projects in clinical trials (and a goal of funding dozens more in the coming years) and with advances being made every day. We still reach out to our supporters and the patient advocate community but now we do it year round through our blog, social media and public events like the one yesterday at UC Davis.

While we are not as “hands on” as we were in the past we are still more than happy to provide tools for groups or organizations who want to hold their own stem cell awareness event – and it doesn’t have to be on October 11th, it can be any day of the year. Visit our Education Portal, Patient Resources page and video archive for various teaching tools.

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Stem Cell Patient Advocates, Scientists and Doctors Unite Around a Common Cause

Some phrases just bring a smile to your face: “It’s a girl/boy”, “Congratulations, you got the job”, and “Another beer sir?” (or maybe that last one is just me). One other phrase that makes me smile is “packed house”. That’s why I was smiling so much at our Patient Advocate Event at UC San Diego last week. The room was jammed with around 150 patients and patient advocates who had come to hear about the progress being made in stem cell research.

Jonathan Thomas, Chair of the CIRM governing Board, kicked off the event with a quick run-through of our research, focusing on our clinical trials. As we have now funded 29 clinical trials, it really was a quick run-through, but JT did focus on a couple of remarkable stories of cures for patients suffering from Severe Combined Immunodeficiency (SCID) and Chronic Granulomatous Disease.

His message was simple. We have come a long way, but we still have a long way to go to fulfill our mission of accelerating stem cell treatments to patients with unmet medical needs. We have a target of 40 new clinical trials by 2020 and JT stressed our determination to do everything we can to reach that goal.

David Higgins, Parkinson’s Disease Advocate and CIRM Board Member (Credit Cory Kozlovich, UCSD)

Next up was David Higgins, who has a unique perspective. David is a renowned scientist, he’s also the Patient Advocate for Parkinson’s disease on the CIRM Board, and he has Parkinson’s disease. David gave a heartfelt presentation on the changing role of the patient and their growing impact on health and science.

In the old days, David said, the patient was merely the recipient of whatever treatment a doctor determined was appropriate. Today, that relationship is much more like a partnership, with physician and patient working together to determine the best approach.

He said CIRM tries to live up to that model by engaging the voice of the patient and patient advocate at every stage of the approval process, from shaping concepts to assessing the scientific merits of a project and deciding whether to fund it, and then doing everything we can to help it succeed.

He said California can serve as the model, but that patients need to make their voices heard at the national level too, particularly in light of the proposed huge budget cuts for the National Institutes of Health.

Dr. Jennifer Braswell. (Credit Cory Kozlovich, UCSD)

U.C. San Diego’s Dr. Jennifer Braswell gave some great advice on clinical trials, focusing on learning how to tell a good trial from a questionable one, and the questions patients need to ask before agreeing to be part of one.

She said it has to:

  • Be at a highly regarded medical center
  • Be based on strong pre-clinical evidence
  • Involved well-informed and compassionate physicians and nurses
  • Acknowledge that it carries some risk.

“You all know that if it sounds too good to be true, it probably is. If someone says a clinical trial carries no risk that’s a red flag, you know that’s not true. There is risk. Good researchers work hard to reduce the risk as much as possible, but you cannot eliminate it completely.”

She said even sites such as www.clinicaltrials.gov – a list of all the clinical trials registered with the National Institutes of Health – have to be approached cautiously and that you should talk to your own physican before signing up for anything.

Finally, UC San Diego’s Dr. Catriona Jamieson talked about her research into blood cancers, and how her work would not have been possible without the support of CIRM. She also highlighted the growing number of trials being carried out at through the CIRM Alpha Stem Cell Clinic Network, which helps scientists and researchers share knowledge and resources, enabling them to improve the quality of the care they provide patients.

The audience asked the panelists some great questions about the need for;

  • A national patient database to make it easier to recruit people for clinical trials
  • For researchers to create a way of letting people know if they didn’t get into a clinical trial so the patients wouldn’t get their hopes up
  • For greater public education about physicians or clinics offering unproven therapies

Adrienne Shapiro, an advocate for sickle cell disease patients, asks a question at Thursday’s stem cell meeting in La Jolla. (Bradley J. Fikes)

The meeting showed the tremendous public interest in stem cell research, and the desire to move it ahead even faster.

This was the first of a series of free public events we are holding around California this year. Next up, Los Angeles. More details of that shortly.

Results are in: The Winners of our 2017 #StemCellResolution Campaign

We asked and you answered! In January, we launched our first Stem Cell Resolution campaign to raise awareness about the importance of stem cell research. We challenged scientists, students, institutes and the public to make a #StemCellResolution and share it on social media.

The goal of our campaign was to start a larger conversation about why stem cell research is important not just to advance science but to develop cures for diseases that currently have none.

Our campaign ran for the month of January, and we had global participation on multiple social media platforms including Twitter, Instagram, videos and blogs. Some resolutions involved answering important research questions while others involved empowering the public to pursue and understand scientific evidence to make their own informed decisions about the benefits of stem cell treatments for treating disease.

I was thoroughly impressed with everyone’s enthusiasm towards supporting and sharing this campaign that I plan to hold it again next year. But for now, I’ll announce the winners of our 2017 #StemCellResolution campaign. We picked the most inspiring resolution for each social media category and a few honorable mentions. The winner of each category will receive CIRM Stem Cell Champions t-shirts.

You can view the full list of this year’s stem cell resolutions on our Storify.


Twitter

Winner: Hamideh Emrani (@HamidehEmrani)

Hamideh is a science and technology communicator and the founder of Emrani Communications. 

Honorable Mention: Christine Liu (@Christineliuart)

Christine is a neuroscience phd student at UC Berkeley and a science communicator and artist.

Instagram

Winner: Pedro Soria Jr. (@shadowtype)

Pedro is a former CIRM Bridges student who is conducting stem cell research in neural regeneration at Western University in Southern California.

My Stem Cell Resolution for 2017 is to create a social media page dedicated to educating, enlightening and disseminating information about past, current, and future stem cell related studies to the general public, as well as those in science, in order to bring to light the importance of stem cell research. My objective is to bring people together regardless of whether or not they Originate from the natural sciences and spark an interest in this emerging field. Coming from a family where I'm first generation Mexican American and the only scientist has shown me the importance of communication amongst those that have little knowledge of the natural world especially people that come from countries that aren't scientifically advanced. Both my parents are born and raised in Michoacan, Mexico, in a small mountain town called Ario de Rosales. Back in my parents day, most people were farmers that worked from sun rise to sunset in order to feed and provide for their families. Naturally, they had little time for education because of the need to survive but had a positive work ethic, which I was lucky to inherit. My parents came to America for an opportunity to improve their situations and provide for themselves and families back home. They worked so hard to obtain what they have and to give me the chance they never had, which I'm so deeply grateful for each and every day of my life!! I had always felt destined for more than mediocre and enjoy taking on challenges to improve myself mentally, physically and spiritually. As a stem cell scientist, it is my responsibility to share my knowledge with everyone I encounter in order to bring change to this world. I wouldn't be where I am if it weren't for the support of my family, friends, professors, colleagues and of course #CIRM . Please join me on this journey and spread the word to anyone that will listen because we're all on this ride together in one way or another. That is my #stemcellresolution #soriaclan #bringingchange #cellculture Look out for my social media page #cellculture for all your stem cell info and check out the @cirm_stemcells to see what this beautiful institute is doing this year!!! #StemCellResolution

A post shared by Pedro Soria Jr. (@shadowtype) on

Video

Winner: Samantha Yammine (@SamanthaZY)

Samantha Yammine is a science communicator and a PhD candidate in Dr. Derek van der Kooy’s lab at the University of Toronto. You can learn more about Sam and her research on her website. She also recently wrote a guest blog for CIRM about a Keystone stem cell conference that you can find here.

Honorable Mentions: Paul Knoepfler (@pknoepfler)

Paul is a biomedical scientist at UC Davis, a science writer, advocate, and cancer survivor. He writes a popular stem cell blog called the Niche.

Honorable Mention: Catia B (@apulgarita)

Catia is a PhD student at MIT and is conducting research on programming & stem cells. She is originally from Portugal and has a personal blog about traveling and the PhD lifestyle.

Honorable Mention: Gladstone trainees (@Gladstone_GO)

Gladstone students and postdocs stepped up to the challenge and filmed stem cell resolutions about their research.

Blog

Winner: Sophie Arthur (@SophArthur)

Soph is a PhD student in Southampton, K studying embryonic stem cell metabolism. Her goal is to find ways to maintain the pluripotent quality of stem cells. She has a personal science communications blog called Soph Talks Science.

 An excerpt from Soph’s blog is below. I highly recommend reading the entire piece as it is very engaging and inspiring!

“For my Stem Cell Resolution – I couldn’t decide on one, so instead, I’ve made 4! Oops!

First, I want to raise awareness that stem cell biology is as important as stem cell treatments! There is lots of hype over stem cell treatments across the globe, but I want to stress that there are only a handful that have actually been approved! I could very well be biased as I’m studying stem cells and their biological mechanisms that exist normally in our bodies – but I want to stress the importance of this work. Simple biology – as I think it will hold the key to all the future stem cell medicine! Once we know how stem cells work in our bodies we can exploit that to make the treatments, or even learn more about our normal development!

 Honorable Mention: Stacey Johnson (@msstaceyerin)

Stacey is the Director of Communications and Marketing for CCRM, the Centre for Commercialization of Regenerative Medicine in Canada. She also is a regular contributor to CCRM’s Signals Blog.

“Since I’m not a scientist, a student or a patient, but I regularly communicate about stem cells to raise awareness and educate the public, my #stemcellresolution is to use this forum to spread the news – what I do best – about this fun and important challenge.”

Read Stacey’s full blog here.


 Thank you and see you next year!

Science communications is a vital tool that scientists and science enthusiasts need to leverage now more than ever to support stem cell research. Speaking out through social media or blogs is a great way to do this, and I want to congratulate all those that participated this year. I’m grateful for your support!

We look forward to doing this again next year and this time, you’ll have an entire year to ponder your next #StemCellResolution.

Cured by Stem Cells

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To get anywhere you need a good map, and you need to check it constantly to make sure you are still on the right path and haven’t strayed off course. A year ago the CIRM Board gave us a map, a Strategic Plan, that laid out our course for the next five years. Our Annual Report for 2016, now online, is our way of checking that we are still on the right path.

I think, without wishing to boast, that it’s safe to say not only are we on target, but we might even be a little bit ahead of schedule.

The Annual Report is chock full of facts and figures but at the heart of it are the stories of the people who are the focus of all that we do, the patients. We profile six patients and one patient advocate, each of whom has an extraordinary story to tell, and each of whom exemplifies the importance of the work we support.

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Brenden Whittaker: Cured

Two stand out for one simple reason, they were both cured of life-threatening conditions. Now, cured is not a word we use lightly. The stem cell field has been rife with hyperbole over the years so we are always very cautious in the way we talk about the impact of treatments. But in these two cases there is no need to hold back: Evangelina Padilla Vaccaro and Brenden Whittaker have been cured.

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Evangelina: Cured

 

In the coming weeks we’ll feature our conversations with all those profiled in the Annual Report, giving you a better idea of the impact the stem cell treatments have had on their lives and the lives of their family. But today we just wanted to give a broad overview of the Annual Report.

The Strategic Plan was very specific in the goals it laid out for us. As an agency we had six big goals, but each Team within the agency, and each individual within those teams had their own goals. They were our own mini-maps if you like, to help us keep track of where we were individually, knowing that every time an individual met a goal they helped the Team get closer to meeting its goals.

As you read through the report you’ll see we did a pretty good job of meeting our targets. In fact, we missed only one and we’re hoping to make up for that early in 2017.

But good as 2016 was, we know that to truly fulfill our mission of accelerating treatments to patients with unmet medical needs we are going to have do equally well, if not even better, in 2017.

That work starts today.

 

Asterias’ stem cell clinical trial shows encouraging results for spinal cord injury patients

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Jake Javier; Asterias spinal cord injury clinical trial participant

When researchers are carrying out a clinical trial they have two goals: first, show that it is safe (the old “do no harm” maxim) and second, show it works. One without the other doesn’t do anyone any good in the long run.

A few weeks ago Asterias Biotherapeutics showed that their CIRM-funded stem cell therapy for spinal cord injuries appeared to be safe. Now their data suggests it’s working. And that is a pretty exciting combination.

Asterias announced the news at the annual scientific meeting of the International Spinal Cord Society in Vienna, Austria. These results cover five people who got a transplant of 10 million cells. While the language is muted, the implications are very encouraging:

“While early in the study, with only 4 of the 5 patients in the cohort having reached 90 days after dosing, all patients have shown at least one motor level of improvement so far and the efficacy target of 2 of 5 patients in the cohort achieving two motor levels of improvement on at least one side of their body has already been achieved.”

What does that mean for the people treated? A lot. Remember these are people who qualified for this clinical trial because of an injury that left them pretty much paralyzed from the chest down. Seeing an improvement of two motor levels means they are regaining some use of their arms, hands and fingers, and that means they are regaining the ability to do things like feeding, dressing and bathing themselves. In effect, it is not only improving their quality of life but it is also giving them a chance to lead an independent life.

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Kris Boesen, Asterias clinical trial participant

One of those patients is Kris Boesen who regained the use of his arms and hands after becoming the first patient in this trial to get a transplant of 10 million cells. We blogged about Kris here

Asterias says of the 5 patients who got 10 million cells, 4 are now 90 days out from their transplant. Of those:

  • All four have improved one motor level on at least one side
  • 2 patients have improved two motor levels on one side
  • One has improved two motor levels on both sides

What’s also encouraging is that none of the people treated experienced any serious side effects or adverse events from the transplant or the temporary use of immunosuppressive drugs.

Steve Cartt, CEO of Asterias, was understandably happy with the news and that it allows them to move to the next phase:

“We are quite encouraged by this first look at efficacy results and look forward to reporting six-month efficacy data as planned in January 2017.  We have also just recently been cleared to begin enrolling a new cohort and administering to these new patients a much higher dose of 20 million cells.  We look forward to begin evaluating efficacy results in this higher-dose cohort in the coming months as well.”

People with spinal cord injuries can regain some function spontaneously so no one is yet leaping to the conclusion that all the progress in this trial is due to the stem cells. But to see all of the patients in the 10 million stem cell group do well is at the very least a positive sign. Now the hope is that these folks will continue to do well, and that the next group of people who get a 20 million cell transplant will also see improvements.

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Roman Reed, spinal cord injury patient advocate

While the team at Asterias were being cautiously optimistic, Roman Reed, whose foundation helped fund the early research that led to this clinical trial, was much less subdued in his response. He was positively giddy:

“If one patient only improves out of the five, it can be an outlier, but with everyone improving out of the five this is legit, this is real. Cures are happening!”

 

Why is a cell therapy that restores sight to the blind against the law?

FDA

A lot of people are frustrated with the US Food and Drug Administration (FDA) and its woefully slow process for approving stem cell therapies. That’s one of the reasons why we started the CIRM Stem Cell Champions campaign, to gather as many like-minded supporters of stem cell research as possible and help to change the way the FDA works, to create a more efficient approval process.

You can read more about that campaign and watch a short video on what being a Stem Cell Champion involves (hint: not very much).

Now Randy Mills, our President and CEO, has teamed up with former US Senator Bill Frist to explain precisely why the FDA needs to change the way it regulates stem cells, and to offer a simple way to create the system that will best serve the needs of patients.

This Op Ed appeared on Fox News’ online Opinion section on Friday, May 20th.


Cell therapy reversed blindness for 47,000 patients in 2015. So why is it against the law?

By C. Randal Mills Ph.D., Sen. Bill Frist M.D.

As medical miracles go, restoring sight to the blind is right up there. A mother seeing her baby for the first time, or a child being able to count the stars is a beautiful gift, and its value cannot be overstated. Last year 47,000 Americans received that gift and had their blindness reversed through the transplantation of cells from a corneal donor’s final selfless act.

It is safe, it is effective, and because it is curative, it is a relatively cost effective procedure. It is medicine at its most beautiful. And according to FDA regulations, the distribution of this cell therapy is in violation of federal law.

That’s right. The regulation says that no matter how competent the surgeon, the FDA must first approve cells from donated corneas as if they were a drug—a process that takes over a decade and can costs billions of dollars — all for a practice that has been successfully restoring sight for more than 50 years. And this is only one example.

The good news: the FDA doesn’t always adhere to its regulations and has not in this case.

The bad news: inconsistent enforcement creates uncertainty, deterring innovation for other unmet medical needs such as arthritis, back pain, and diabetic ulcers.

How did a country known for pioneering medical breakthroughs get here?

Appropriate regulation of living cells that treat disease is inherently complex. Some therapies, like corneal cell transplants, are well-understood. Others are far more sophisticated and can involve forcing cells to change from one type to another, cutting out defective genes, and growing cells in culture to expand their numbers into the billions. Although this may sound like science fiction, it’s the type of very real science that will revolutionize the practice of medicine. And it is a challenging spectrum to regulate.

Unfortunately, what we have today amounts to a regulatory light switch for cell therapy; one that is either OFF or ON. For some cell therapies there is essentially no pre-market regulation. But at some point of added complexity, often arbitrarily decided by the FDA, the switch flips to ON and the cell becomes a drug in the minds of the Agency. And the consequences could not be more profound.

A product can be introduced through the OFF pathway in days with no FDA review and at very little cost. The ON pathway on the other hand, takes 10-20 years and can cost over a billion dollars. For cell therapy, there is no in between.

It is not possible to regulate the continuum of cell therapies fairly and effectively by using this binary approach. The system is broken and is impeding the hunt for safe and effective treatments for suffering patients.

Why? Because sensible people don’t invest significant capital gambling that the FDA will give them a pass out of its rules. They evaluate the time and cost of development assuming they will be forced down the ON pathway. They also assume that this arbitrary approach to regulation will (and often does) work against them by allowing a competitor to enter the market through the OFF pathway, placing them at a prohibitive disadvantage. The results speak for themselves. After 15 years under this paradigm we have had only a few cell therapies approved, all commercial disasters.

This is because the ON-OFF approach fails to adequately account for the difference in cell therapy complexity. To better understand, imagine this methodology applied to the regulation of automobiles. The government might permit low tech cars, say the Model T, to be sold without pre-market regulation. But if a manufacturer wanted to improve the vehicle by adding air conditioning, a radio or other such feature, the car would be subject to massive pre-market regulation. And not just on the new feature. Instead, the addition of the new feature would trigger a bumper-to-bumper evaluation of the entire car, increasing its development cost from basically nothing to that of a Lamborghini. The result would be streets full of hot, radio-less go-karts, except for a few ultra-high-end sports cars whose manufacturers are now defunct because they were never able to recoup the disproportionate costs of satisfying the regulatory system. This is what we see with cell therapies today: progress that is sluggish at best.

How can we move forward?

Ironically, the FDA identified a solution to the problem. In order to account for the broad spectrum inherent to cell therapy, in the late 90’s the FDA proposed a progressive, risk-based approach. The higher the risk, the greater the regulation. This guards against under regulation that might put patients at risk and prevents overregulation that can disincentivize the development of new or improved products.

In the FDA’s own words, the regulation they proposed would abide by a few basic principles:

  • “Under this tiered, risk-based approach, we propose to exert only the type of government regulation necessary to protect the public health.”
  • “The regulation of different types of human cells… will be commensurate with the public health risks…”
  • “These planned improvements will increase the safety of human cells… while encouraging the development of new products.”

It was a remarkably common sense approach that would have balanced safety with the need for innovation over an exceptionally broad range of technological complexity and risk.

It would have.

Unfortunately, the regulatory framework that was promised was never delivered, and it is time to resuscitate it. The burden placed on the development of cell therapies must accurately reflect the risks; must be balanced against the very real consequences of doing nothing (patients continuing to suffer); and must be consistently and fairly applied. In short, the FDA had it right and we need to give them the tools to deliver the regulatory paradigm they originally envisioned.

If we fix this highly fixable problem, we can create a system that will drive new innovations and better outcomes. Europe and Japan have already acted and are seeing the benefits. People with great ideas are coming off the bench, and game changing therapies are entering practice. While challenging the status quo does not sit well with some, particularly those who stand to prosper from the built-in barriers to entry the current structure provides, in the United States we have a responsibility to do better for patients and fix this broken system.

Randal Mills, Ph.D., is the President and CEO of the California Institute for Regenerative Medicine

William “Bill” H. Frist, M.D. is a nationally-acclaimed heart and lung transplant surgeon, former U.S. Senate Majority Leader, and chairman of the Executive Board of the health service private equity firm Cressey & Company.