Hitting our Goals: Scoring a half century

Way, way back in 2015 – seems like a lifetime ago doesn’t it – the team at CIRM sat down and planned out our Big 6 goals for the next five years. The end result was a Strategic Plan that was bold, ambitious and set us on course to do great things or kill ourselves trying. Well, looking back we can take some pride in saying we did a really fine job, hitting almost every goal and exceeding them in some cases. So, as we plan our next five-year Strategic Plan we thought it worthwhile to look back at where we started and what we achieved. Goal #2 was Expand.

Scientist preparing a sample vial for automated analysis in the lab.

When CIRM first started there was an internal report that said if we managed to help get one project into a clinical trial before we ran out of money we would be doing well. At the time that seemed quite reasonable. The field was still very much in its infancy and most of the projects we were funding, particularly in the early days, were Discovery or basic research projects.

But as the field advanced we got a little bolder. By 2010 we were funding not just our first clinical trial, but the first clinical trial in the world using embryonic stem cells. This was the Geron trial targeting spinal cord injury. Sadly the excitement didn’t last very long. After treating just five patients Geron pulled the plug on the trial, deciding that targeting cancer was a better bet.

Happily, Geron returned all the money we had loaned them, plus interest, so we were able to use that to fund more research. Soon enough we had a number of other promising candidates heading towards a meeting with the US Food and Drug Administration (FDA) to try and get permission to start a clinical trial.

By 2014, ten years after we began, we actually had ten projects either running or getting ready to start a clinical trial. We thought that was really good. But at CIRM, really good is never good enough.

For our Strategic Plan in 2015 we decided to shoot for the moon and aim to get another 50 clinical trials over the next five years. At the time it seemed, to be honest, a bit bonkers. How on earth were we going to do that. But then our Therapeutics team went a hunting!

In the past we had the luxury of mostly just waiting for people with promising projects to approach us for funding. With an ambitious goal of getting 50 more clinical trials, we couldn’t afford to wait. The Therapeutics team scouted around for promising projects, inside and outside California, inside and outside the US, and pitched them on the benefits of applying for funding. Slowly the numbers started to rise.

By the end of 2016 we had 12 new trials. In 2017 we were really cruising along, adding 16 more trials. 2018 there was another 14 and that was also the year we passed the 50 clinical trials total since CIRM was created. We celebrated at a Board meeting with a balloon and a cake (we’re a state agency, our budget doesn’t extend to confetti). Initially the inscription on the cake read ‘Congratulations: 50 Clinical Trails’. Happily, we were able to fix it before anyone noticed. But even with the spelling error, it would still have tasted just fine.

Patient advocate Rich Lajara with the Big Balloon celebration for funding 50 clinical trials

By the time we got to mid-2020 we were stuck on 47 and with time, and money, running out it looked like we might miss the goal. But then our team put in one last effort and with weeks to spare we funded four more clinical trials for a total of 51 (68 since we started in 2004).

So, the moral is dream big but work hard. Now let’s see what we can dream up for our next Strategic Plan.

Hitting our goals: regulatory reform

Way, way back in 2015 – seems like a lifetime ago doesn’t it – the team at CIRM sat down and planned out our Big 6 goals for the next five years. The end result was a Strategic Plan that was bold, ambitious and set us on course to do great things or kill ourselves trying. Well, looking back we can take some pride in saying we did a really fine job, hitting almost every goal and exceeding them in some cases. So, as we plan our next five-year Strategic Plan we thought it worthwhile to look back at where we started and what we achieved. We are going to start with Regulatory Reform.

The political landscape in 2015 was dramatically different than it is today. Compared to more conventional drugs and therapies stem cells were considered a new, and very different, approach to treating diseases and disorders. At the time the US Food and Drug Administration (FDA) was taking a very cautious approach to approving any stem cell therapies for a clinical trial.

A survey of CIRM stakeholders found that 70% said the FDA was “the biggest impediment for the development of stem cell treatments.” One therapy, touted by the FDA as a success story, had such a high clinical development hurdle placed on it that by the time it was finally approved, five years later, its market potential had significantly eroded and the product failed commercially. As one stakeholder said: “Is perfect becoming the enemy of better?”

So, we set ourselves a goal of establishing a new regulatory paradigm, working with Congress, academia, industry, and patients, to bring about real change at the FDA and to find ways to win faster approval for promising stem cell therapies, without in any way endangering patients.

It seemed rather ambitious at the time, but achieving that goal happened much faster than any of us anticipated. With a sustained campaign by CIRM and other industry leaders, working with the patient advocacy groups, the FDA, Congress, and President Obama, the 21st Century Cures Act was signed into law on December 13, 2016.

President Obama signs the 21st Century Cures Act.
Photo courtesy of NBC News

The law did something quite radical; it made the perspectives of patients an integral part of the FDA’s decision-making and approval process in the development of drugs, biological products and devices. And it sped up the review process by:

In a way the FDA took its foot off the brake but didn’t hit the accelerator, so the process moved faster, but in a safe, manageable way.

Fast forward to today and eight projects that CIRM funds have been granted RMAT designation. We have become allies with the FDA in helping advance the field. We have created a unique partnership with the National Heart, Lung and Blood Institute (NHLBI) to support the Cure Sickle Cell initiative and accelerate the development of cell and gene therapies for sickle cell disease.

The landscape has changed since we set a goal of regulatory reform. We still have work to do. But now we are all working together to achieve the change we all believe is both needed and possible.

A word from our Chair, several in fact

In 2005, the New Oxford American Dictionary named “podcast” its word of the year. At the time a podcast was something many had heard of but not that many actually tuned in to. My how times have changed. Now there are some two million podcasts to chose from, at least according to the New York Times, and who am I to question them.

Yesterday, in the same New York Times, TV writer Margaret Lyons, wrote about how the pandemic helped turn her from TV to podcasts: “Much in the way I grew to prefer an old-fashioned phone call to a video chat, podcasts, not television, became my go-to medium in quarantine. With their shorter lead times and intimate production values, they felt more immediate and more relevant than ever before.”

I mention this because an old colleague of ours at CIRM, Neil Littman, has just launched his own podcast and the first guest on it was Jonathan Thomas, Chair of the CIRM Board. Their conversation ranged from CIRM’s past to the future of the regenerative field as a whole, with a few interesting diversions along the way. It’s fun listening. And as Margaret Lyons said it might be more immediate and more relevant than ever before.

Charting a course for the future

A new home for stem cell research?

Have you ever been at a party where someone says “hey, I’ve got a good idea” and then before you know it everyone in the room is adding to it with ideas and suggestions of their own and suddenly you find yourself with 27 pages of notes, all of them really great ideas. No, me neither. At least, not until yesterday when we held the first meeting of our Scientific Strategy Advisory Panel.

This is a group that was set up as part of Proposition 14, the ballot initiative that refunded CIRM last November (thanks again everyone who voted for that). The idea was to create a panel of world class scientists and regulatory experts to help guide and advise our Board on how to advance our mission. It’s a pretty impressive group too. You can see who is on the SSAP here.  

The meeting involved some CIRM grantees talking a little about their work but mostly highlighting problems or obstacles they considered key issues for the future of the field as a whole. And that’s where the ideas and suggestions really started flowing hard and fast.

It started out innocently enough with Dr. Amander Clark of UCLA talking about some of the needs for Discovery or basic research. She advocated for a consortium approach (this quickly became a theme for many other experts) with researchers collaborating and sharing data and findings to help move the field along.

She also called for greater diversity in research, including collecting diverse cell samples at the basic research level, so that if a program advanced to later stages the findings would be relevant to a wide cross section of society rather than just a narrow group.

Dr. Clark also said that as well as supporting research into neurodegenerative diseases, such as Alzheimer’s and Parkinson’s, there needed to be a greater emphasis on neurological conditions such as autism, bipolar disorder and other mental health problems.

(CIRM is already committed to both increasing diversity at all levels of research and expanding mental health research so this was welcome confirmation we are on the right track).

Dr. Mike McCun called for CIRM to take a leadership role in funding fetal tissue research, things the federal government can’t or won’t support, saying this could really help in developing an understanding of prenatal diseases.

Dr. Christine Mummery, President of ISSCR, advocated for support for early embryo research to deepen our understanding of early human development and also help with issues of infertility.

Then the ideas started coming really fast:

  • There’s a need for knowledge networks to share information in real-time not months later after results are published.
  • We need standardization across the field to make it easier to compare study results.
  • We need automation to reduce inconsistency in things like feeding and growing cells, manufacturing cells etc.
  • Equitable access to CRISPR gene-editing treatments, particularly for underserved communities and for rare diseases where big pharmaceutical companies are less likely to invest the money needed to develop a treatment.
  • Do a better job of developing combination therapies – involving stem cells and more traditional medications.

One idea that seemed to generate a lot of enthusiasm – perhaps as much due to the name that Patrik Brundin of the Van Andel Institute gave it – was the creation of a CIRM Hotel California, a place where researchers could go to learn new techniques, to share ideas, to collaborate and maybe take a nice cold drink by the pool (OK, I just made that last bit up to see if you were paying attention).

The meeting was remarkable not just for the flood of ideas, but also for its sense of collegiality.  Peter Marks, the director of the Food and Drug Administration’s Center for Biologics Evaluation and Research (FDA-CBER) captured that sense perfectly when he said the point of everyone working together, collaborating, sharing information and data, is to get these projects over the finish line. The more we work together, the more we will succeed.

Everything you wanted to know about COVID vaccines but never got a chance to ask

All this month we are using our blog and social media to highlight a new chapter in CIRM’s life, thanks to the voters approving Proposition 14. We are looking back at what we have done since we were created in 2004, and also looking forward to the future. Today we feature a rare treat, an interview with Moderna’s Dr. Derrick Rossi.

Moderna co-founder Dr. Derrick Rossi

It’s not often you get a chance to sit down with one of the key figures in the fight against the coronavirus and get to pick his brain about the best ways to beat it. We were fortunate enough to do that on Wednesday, talking to Dr. Derrick Rossi, the co-founder of Moderna, about the vaccine his company has developed.

CIRM’s President and CEO, Dr. Maria Millan, was able to chat to Dr. Rossi for one hour about his background (he got support from CIRM in his early post-doctoral research at Stanford) and how he and his colleagues were able to develop the COVID-19 vaccine, how the vaccine works, how effective it is, how it performs against new variations of the virus.

He also told us what he would have become if this science job hadn’t worked out.

All in all it was a fascinating conversation with someone whose work is offering a sense of hope for millions of people around the world.

If you missed it first time around you can watch it here.

Progress in the fight against Sickle Cell Disease

Marissa Cors, sickle cell disease patient advocate

Last November Marissa Cors, a patient advocate in the fight against Sickle Cell Disease (SCD), told the Stem Cellar “A stem cell cure will end generations of guilt, suffering, pain and early death. It will give SCD families relief from the financial, emotional and spiritual burden of caring someone living with SCD. It will give all of us an opportunity to have a normal life. Go to school, go to work, live with confidence.” With each passing month it seems we are getting closer to that day.

CIRM is funding four clinical trials targeting SCD and another project we are supporting has just been given the green light by the Food and Drug Administration to start a clinical trial. Clearly progress is being made.

Yesterday we got a chance to see that progress. We held a Zoom event featuring Marissa Cors and other key figures in the fight against SCD, CIRM Science Officer Dr. Ingrid Caras and Evie Junior. Evie is a pioneer in this struggle, having lived with sickle cell all his life but now hoping to live his life free of the disease. He is five months past a treatment that holds out the hope of eradicating the distorted blood cells that cause such devastation to people with the disease.

You can listen to his story, and hear about the other progress being made. Here’s a recording of the Zoom event.

You can also join Marissa every week on her live event on Facebook, Sickle Cell Experience Live.

A Month of CIRM: Where we’ve been, where we’re going

All this month we are using our blog and social media to highlight a new chapter in CIRM’s life, thanks to the voters approving Proposition 14. We are looking back at what we have done since we were created in 2004, and also looking forward to the future. We kick off this event with a letter from our the Chair of our Board, Jonathan Thomas.

When voters approved Proposition 14 last November, they gave the Stem Cell Agency a new lease on life and a chance to finish the work we began with the approval of Proposition 71 in 2004. It’s a great honor and privilege. It’s also a great responsibility. But I think looking back at what we have achieved over the last 16 years shows we are well positioned to seize the moment and take CIRM and regenerative medicine to the next level and beyond.

When we started, we were told that if we managed to get one project into a clinical trial by the time our money ran out we would have done a good job. As of this moment we have 68 clinical trials that we have funded plus another 31 projects in clinical trials where we helped fund crucial early stage research. That inexorable march to therapies and cures will resume when we take up our first round of Clinical applications under Prop 14 in March.

But while clinical stage projects are the end game, where we see if therapies really work and are safe in people, there’s so much more that we have achieved since we were created. We have invested $900 million in  basic research, creating a pipeline of the most promising stem cell research programs, as well as investing heavily on so-called “translational” projects, which move projects from basic science to where they’re ready to apply to the Food and Drug Administration (FDA) to begin clinical trials.

We have funded more than 1,000 projects, with each one giving us valuable information to help advance the science. Our funding has helped attract some of the best stem cell scientists in the world to California and, because we only fund research in California, it has persuaded many companies to either move here or open offices here to be eligible for our support. We have helped create the Alpha Stem Cell Clinics, a network of leading medical centers around the state that have the experience and expertise to deliver stem cell therapies to patients. All of those have made California a global center in the field.

That result is producing big benefits for the state. An independent Economic Impact Analysis reported that by the end of 2018 we had already helped generate an extra $10.7 billion in new sales revenue and taxes for California, hundreds of millions more in federal taxes and created more than 56,000 new jobs.

As if that wasn’t enough, we have also:

  • Helped develop the largest iPSC research bank in the world.
  • Created the CIRM Center of Excellence in Stem Cell Genomics to accelerate fundamental understanding of human biology and disease mechanisms.
  • Helped fund the construction of 12 world class stem cell institutes throughout the state.
  • Reached a unique partnership with the National Heart, Lung and Blood Institutes to find a cure for sickle cell disease.
  • Used our support for stem cell research to leverage an additional $12 billion in private funding for the field.
  • Enrolled more than 2700 patients in CIRM funded clinical trials

In many ways our work is just beginning. We have laid the groundwork, helped enable an extraordinary community of researchers and dramatically accelerated the field. Now we want to get those therapies (and many more) over the finish line and get them approved by the FDA so they can become available to many more people around the state, the country and the world.

We also know that we have to make these therapies available to all people, regardless of their background and ability to pay. We have to ensure that underserved communities, who were often left out of research in the past, are an integral part of this work and are included in every aspect of that research, particularly clinical trials. That’s why we now require anyone applying to us for funding to commit to engaging with underserved communities and to have a written plan to show how they are going to do that.

Over the coming month, you will hear more about some of the remarkable things we have managed to achieve so far and get a better sense of what we hope to do in the future. We know there will be challenges ahead and that not everything we do or support will work. But we also know that with the team we have built at CIRM, the brilliant research community in California and the passion and drive of the patient advocate community we will live up to the responsibility the people of California placed in us when they approved Proposition 14.

Inspiring new documentary about stem cell research

Poster for the documentary “Ending Disease”

2020 has been, to say the very least, a difficult and challenging year for all of us. But while the focus of the world has, understandably, been on the coronavirus there was also some really promising advances in stem cell research. Those advances are captured in a great new documentary called Ending Disease.

The documentary is by Emmy award-winning filmmaker Joe Gantz. In it he follows ten people who are facing life-threatening or life-changing diseases and injuries and who turn to pioneering stem cell therapies for help.

It’s an inspiring documentary, one that reminds you of the real need for new treatments and the tremendous hope and promise of stem cell therapies. Here’s a look at a trailer for Ending Disease.

You can see an exclusive screening of Ending Disease on Friday, January 8th, 2021 at 5:00pm PST.

After the livestream, there will be a live Q&A session where former members of the successful Proposition 14 campaign team – which refunded CIRM with an additional $5.5 billion – will be joined by CIRM’s President and CEO Dr. Maria Millan, talking about what lies ahead for CIRM and the future of stem cell research.

To purchase a ticket, click here. It only costs $12 and 50% of the ticket sales proceeds will go to Americans for Cures to help them continue to advocate for the advancement of stem cell research, and more importantly, for the patients and families to whom stem cell research provides so much hope.

If you need any extra persuading that it’s something you should definitely put on our calendar, here’s a letter from the film maker Joe Gantz.

I am the director of the documentary Ending Disease: The Stem Cell, Anti-Cancer T-Cell, & Antibody Revolution In Medicine, a film that will help inform people about the progress that’s been made in this field and how people with their lives on the line are now able to benefit from these new regenerative therapies. 

I was granted unprecedented access to ten of the first generation of clinical trials using stem cell and regenerative medicine to treat and cure many of the most devastating diseases and conditions including: brain cancer, breast cancer, leukemia and lymphoma, HIV, repairing a broken spinal cord, retinitis pigmentosa and SCID. The results are truly inspiring.

This is personal for me.  After spending four years making this documentary, I was diagnosed with bladder cancer. Upon diagnosis, I immediately felt the same desperation as millions of families who are in search of a medical breakthrough. I understood, on a personal level, what the patients we followed in the film all knew: when you are diagnosed with a disease, there is a narrow window of time in which you can effectively seek a life-saving treatment or cure. If treatment becomes available outside of that window, then it is too late. However, Ending Disease shows that with continued support for regenerative medicine, we can create a near future in which one-time cures and highly mitigating therapies are available to patients for a whole host of diseases.

Best regards,

Joe

Much to be Thankful for

It’s traditional this time of year to send messages of gratitude to friends and family and colleagues. And we certainly have much to be thankful for.

Thanks to the voters of California, who passed Proposition 14, we have a bright, and busy, future. We have $5.5 billion to continue our mission of accelerating stem cell treatments to patients with unmet medical needs.

That means the pipeline of promising projects that we have supported from an early stage can now apply to us to help take that work out of the lab and into people.

It means research areas, particularly early-stage work, where we had to reduce our funding as we ran out of money can now look forward to increased support.

It means we can do more to bring this research, and it’s potential benefits, to communities that in the past were overlooked.

We have so many people to thank for all this. The scientists who do the work and championed our cause at the ballot box. The voters of California who once again showed their support for and faith in science. And the patients and patient advocates, the reason we were created and the reason we come to work every day.

As Dr. Maria Millan, our President & CEO, said in a letter to our team; “We are continually faced by great opportunities brilliantly disguised as insoluble problems.”  Here’s to the opportunities made possible by CIRM and for its continuation made possible by Prop 14!”

And none of this would be possible without the support of all of you. And for that we are truly Thankful.

From everyone at CIRM, we wish you a happy, peaceful and safe Thanksgiving.