Stem Cell Agency Board Approves Roadmap for Next Five Years

Dr. Maria Millan, CIRM’s President & CEO

It’s hard to get somewhere if you don’t know where you are going. Without a map you can’t plan a route to your destination. That’s why the CIRM Board approved a new Strategic Plan laying out a roadmap for the Stem Cell Agency for the next five years.

The plan builds on the achievements of Proposition 71, the voter approved ballot initiative that created the Agency in 2004, including:

  • Supporting 76 clinical trials.
  • Helping cure more than 40 children born with a rare, fatal immune disorder.
  • Creating the Alpha Clinics Network that specializes in the delivery of stem cell therapies to patients.
  • Training over 3000 students and scholars to become the future workforce of regenerative medicine.
  • Stimulating California’s economy with $10.7 Billion in additional sales revenue and the creation of 56,000 new jobs (between 2004-2018)

The passage of Proposition 14 in 2020 has positioned CIRM to continue to accelerate research from discovery to clinical; to drive innovative, real-world solutions resulting in transformative treatments for patients; and to ensure the affordability and accessibility of those treatments to a diverse community of patients in an equitable manner, including those often overlooked or underrepresented in the past.

“We achieved a lot in the last 15 years and this provides a solid foundation for our strategy to bring us to the new era of CIRM and to deliver the full potential of regenerative medicine, says Dr. Maria T. Millan, the President and CEO of CIRM. “This plan lays out a roadmap for us to overcome the challenges in developing transformative therapies and making them accessible and affordable in an equitable fashion to a diverse California. The plan will guide us in that work through the development of novel scientific endeavors, effective healthcare delivery models, and expanded education and training programs.”

The Strategic Plan is organized into three main themes:

  • Advance World Class Science – Foster a culture of collaborative science by creating knowledge networks and shared research tools and technologies that encourage and facilitate data and resource sharing.
  • Deliver Real World Solutions – Accelerate approval of therapies by optimizing our support models for CIRM-funded clinical trials with attention to including underserved communities; build the California Manufacturing Network to overcome manufacturing hurdles; and expand the Alpha Clinics network and create the Community Care Centers of Excellence to deliver therapies to a diverse patient population often in underserved communities.
  • Provide Opportunity for All – Build a racially, ethnically and experientially diverse and highly skilled workforce to support the growing regenerative medicine economy in California; deliver a roadmap for access and affordability of regenerative medicine for all California patients.

Reflecting these goals, CIRM’s new mission statement is: Accelerating world class science to deliver transformative regenerative medicine treatments in an equitable manner to a diverse California and world.

“We realize that these are ambitious goals but they are achievable,” says Dr. Millan, “If CIRM is going to continue to be a global leader in the field of regenerative medicine, and to live up to the faith shown in us by the people of California, we believe we have to aim high. We have a terrific team, a clear vision and a determination to fulfill our mission. And that’s what we intend to do.”

Sweating bullets and other stories from the front line

THIS BLOG IS ALSO AVAILABLE AS AN AUDIO CAST

When the COVID-19 pandemic hit and the 2020 election became one of the most contentious in living history it suddenly made trying to get a proposition on the ballot in California a lot harder. That meant the fate of Proposition 14, a ballot initiative refunding CIRM, California’s Stem Cell Agency, was in doubt. And if the agency went down, then a vital source of future funding for scientific research that could change and even save lives would also disappear.

It was a pretty nerve-racking time for all of us involved. We waited day after day after day after day before the election was finally called. Happily, it was in our favor. But only just!

In this podcast we talk to two of the key figures in this saga. Melissa King and Maria Bonneville. Melissa was part of the team that helped secure the votes needed to pass Proposition 14, and Maria helped keep CIRM on track to cope with whatever the outcome of the election was. 

I hope you enjoy this latest episode of our podcast ‘Talking ‘Bout (re)Generation.’

A year unlike any other – a look back at one year post Prop 14

THIS BLOG IS ALSO AVAILABLE AS AN AUDIO CAST

State flag of California

2020 was, by any standards, a pretty wacky year. Pandemic. Political convulsions. And a huge amount of uncertainty as to the funding of life-saving therapies at CIRM. Happily those all turned out OK. We got vaccines to take care of COVID. The election was won fair and square (seriously). And Proposition 14 was approved by the voters of California, re-funding your favorite state Stem Cell Agency.

But for a while, quite a while, there was uncertainty surrounding our future. For a start, once the pandemic lockdown kicked in it was impossible for people to go out and collect the signatures needed to place Proposition 14 on the November ballot. So the organizers of the campaign reached out online, using petitions that people could print out and sign and mail in.

It worked. But even after getting all the signatures needed they faced problems such as how do you campaign to get something passed, when the normal channels are not available. The answer is you get very creative very quickly.

Bob Klein

Bob Klein, the driving force behind both Proposition 71 (the 2004 ballot initiative that created CIRM) and Proposition 14, says it was challenging:

“It was a real adventure. It’s always hard, you have a complicated message about stem cells and genetics and therapy and it’s always a challenge to get a million signatures for a ballot initiative but in the middle of a pandemic where we had to shut down the signature gathering at grocery stores and street corners, where we had to go to petitions that had to be sent to voters and get them to fill them out properly and send them back. And of course the state went into an economic recoil because of the pandemic and people were worried about the money.”

Challenging absolutely, but ultimately successful. On November 13, ten days after the election, Prop 14 was declared the winner.

As our President and CEO, Dr. Maria Millan says, we went from an agency getting ready to close its doors to one ramping up for a whole new adventure.

“We faced many challenges in 2020. CIRM’s continued existence was hinging on the passage of a new bond initiative and we began the year uncertain if it would even make it on the ballot.  We had a plan in place to wind down and close operations should additional funding not materialize.  During the unrest and challenges brought by 2020, and functioning in a virtual format, we retained our core group of talented individuals who were able to mobilize our emergency covid research funding round, continue to advance our important research programs and clinical trials and initiate the process of strategic planning in the event that CIRM was reauthorized through a new bond initiative. Fortunately, we planned for success and Proposition 14 passed against all odds!”

“When California said “Yes,” the CIRM team was positioned to launch the next Era of CIRM! We have recruited top talent to grow the team and have developed a new strategic plan and evolved our mission:  Accelerating world-class science to deliver transformative regenerative medicine treatments to a diverse California and worldwide in an equitable manner.” 

And since that close call we have been very busy. In the last year we have hired 16 new employees, everyone from a new General Counsel to the Director of Finance, and more are on the way as we ramp up our ability to turn our new vision into a reality.

We have also been working hard to ensure we could continue to fund groundbreaking research from the early-stage Discovery work, to testing therapies in patients in clinical trials. Altogether our Board has approved almost $250 million in 56 new awards since December 2020. That includes:

Clinical – $84M (9 awards)

Translational – $15M (3 awards)

Discovery – $13M (11 awards)

Education – $138M (33 awards)

We have also enrolled more than 360 new patients in clinical trials that we fund or that are being carried out in the CIRM Alpha Stem Cell Clinic network.

This is a good start, but we know we have a lot more work to do in the coming years.

The last year has flown by and brought more than its fair share of challenges. But the CIRM team has shown that it can rise to those, in person and remotely, and meet them head on. We are already looking forward to 2022. We’ve got a lot of work to do.

CIRM funds clinical trials targeting heart disease, stroke and childhood brain tumors

Gary Steinberg (Jonathan Sprague)

Heart disease and stroke are two of the leading causes of death and disability and for people who have experienced either their treatment options are very limited. Current therapies focus on dealing with the immediate impact of the attack, but there is nothing to deal with the longer-term impact. The CIRM Board hopes to change that by funding promising work for both conditions.

Dr. Gary Steinberg and his team at Stanford were awarded almost $12 million to conduct a clinical trial to test a therapy for motor disabilities caused by chronic ischemic stroke.  While “clot busting” therapies can treat strokes in their acute phase, immediately after they occur, these treatments can only be given within a few hours of the initial injury.  There are no approved therapies to treat chronic stroke, the disabilities that remain in the months and years after the initial brain attack.

Dr. Steinberg will use embryonic stem cells that have been turned into neural stem cells (NSCs), a kind of stem cell that can form different cell types found in the brain.  In a surgical procedure, the team will inject the NSCs directly into the brains of chronic stroke patients.  While the ultimate goal of the therapy is to restore loss of movement in patients, this is just the first step in clinical trials for the therapy.  This first-in-human trial will evaluate the therapy for safety and feasibility and look for signs that it is helping patients.

Another Stanford researcher, Dr. Crystal Mackall, was also awarded almost $12 million to conduct a clinical trial to test a treatment for children and young adults with glioma, a devastating, aggressive brain tumor that occurs primarily in children and young adults and originates in the brain.  Such tumors are uniformly fatal and are the leading cause of childhood brain tumor-related death. Radiation therapy is a current treatment option, but it only extends survival by a few months.

Dr. Crystal Mackall and her team will modify a patient’s own T cells, an immune system cell that can destroy foreign or abnormal cells.  The T cells will be modified with a protein called chimeric antigen receptor (CAR), which will give the newly created CAR-T cells the ability to identify and destroy the brain tumor cells.  The CAR-T cells will be re-introduced back into patients and the therapy will be evaluated for safety and efficacy.

Joseph Wu Stanford

Stanford made it three in a row with the award of almost $7 million to Dr. Joe Wu to test a therapy for left-sided heart failure resulting from a heart attack.  The major issue with this disease is that after a large number of heart muscle cells are killed or damaged by a heart attack, the adult heart has little ability to repair or replace these cells.  Thus, rather than being able to replenish its supply of muscle cells, the heart forms a scar that can ultimately cause it to fail.  

Dr. Wu will use human embryonic stem cells (hESCs) to generate cardiomyocytes (CM), a type of cell that makes up the heart muscle.  The newly created hESC-CMs will then be administered to patients at the site of the heart muscle damage in a first-in-human trial.  This initial trial will evaluate the safety and feasibility of the therapy, and the effect upon heart function will also be examined.  The ultimate aim of this approach is to improve heart function for patients suffering from heart failure.

“We are pleased to add these clinical trials to CIRM’s portfolio,” says Maria T. Millan, M.D., President and CEO of CIRM.  “Because of the reauthorization of CIRM under Proposition 14, we have now directly funded 75 clinical trials.  The three grants approved bring forward regenerative medicine clinical trials for brain tumors, stroke, and heart failure, debilitating and fatal conditions where there are currently no definitive therapies or cures.”

A conversation with Bob Klein about the past, present and future of CIRM

Bob Klein

Anyone who knows anything about CIRM knows about Bob Klein. He’s the main author and driving force behind both Proposition 71 and Proposition 14, the voter-approved ballot initiatives that first created and then refunded CIRM. It’s safe to say that without Bob there’d be no CIRM.

Recently we had the great good fortune to sit down with Bob to chat about the challenges of getting a proposition on the ballot in a time of pandemic and electoral pandemonium, what he thinks CIRM’s biggest achievements are (so far) and what his future plans are.

You can hear that conversation in the latest episode of our podcast, “Talking ’bout (re) Generation”.

Enjoy.

CIRM Board Approves New Clinical Trial for ALS

This past Friday the governing Board of the California Institute for Regenerative Medicine (CIRM) awarded $11.99 million to Cedars-Sinai to fund a clinical trial for amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease. 

ALS is a neurodegenerative disease that results in the death of nerve cells in the brain and spinal cord, causing the muscles in the body to gradually weaken, leading to loss of limb function, difficulty breathing, paralysis, and eventually death.  There are medications that can slow down the progression of ALS, but unfortunately there is no cure for the disease.

Clive Svendsen, Ph.D., executive director of Cedars-Sinai’s Board of Governors Regenerative Medicine Institute, and his team will be conducting a trial that uses a combined cell and gene therapy approach as a treatment for ALS.  The trial builds upon the Stem Cell Agency’s first ALS trial, also conducted by Cedars-Sinai and Svendsen.

Genetically engineered stem cells will be transplanted into the motor cortex, an area of the brain responsible for voluntary movements.  These transplanted cells then become astrocytes, a type of support cell that help keep nerve cells functioning.  The astrocytes have been genetically altered to deliver high doses of a growth factor which has been shown to protect nerve cells.  The goal of this approach is to protect the upper motor neurons controlling muscle function and meaningfully improve the quality of life for ALS patients.

“ALS is a devastating disease that attacks the spinal cord and brain and results in the progressive loss of the ability to move, to swallow and eventually to breathe. ” says Maria T. Millan, M.D., President and CEO of CIRM.  “This clinical trial builds on Dr. Svendsen’s work previously funded by CIRM. We are fortunate to be able to support this important work, which was made possible by California citizens who voted to reauthorize CIRM under Proposition 14 this past November.”

Month of CIRM – Our Therapeutics Team Goes Hunting

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 have a guest blog by CIRM Senior Science Officer Lisa Kadyk, outlining how she and her colleagues actively search for the best science to fund.

Lisa Kadyk, Ph.D.

Hi everyone,

This is Lisa Kadyk, a Science Officer from the CIRM Therapeutics team, here to tell you about some of the work our team does to support the CIRM mission of accelerating stem cell treatments to patients with unmet medical needs.  Our job involves seeking out and recruiting great scientists to apply to CIRM and supporting those we fund.

Therapeutics team members manage both the awards that fund the final preclinical studies required before testing a therapeutic in a clinical trial (CLIN1), and the awards that fund the clinical trials themselves (CLIN2). 

I mentioned above that we actively recruit new applicants for our CLIN1 and CLIN2 awards – which is not an activity that is typical of most funding agencies – so why and how do we do this?  

It all comes down to our mission of accelerating the development of therapies to help patients with unmet medical needs.  It turns out that there are many potential applicants developing cutting edge therapies who don’t know much or anything about CIRM, and the ways we can help them with getting those therapies to the clinic and through clinical trials.    So, to bridge this gap, we Science Officers attend scientific conferences, read the scientific literature and meet regularly with each other to stay abreast of new therapeutic approaches being developed in both academia and industry, with the goal of identifying and reaching out to potential applicants about what CIRM has to offer. 

What are some of the things we tell potential applicants about how partnering with CIRM can help accelerate their programs?   First of all, due to the efforts of a very efficient Review team, CIRM is probably the fastest in the business for the time between application and potential funding.  It can be as short as three months for a CLIN1 or CLIN2 application to be reviewed by the external Grants Working Group and approved by the CIRM Board, whereas the NIH (for example) estimates it takes seven to ten months to fund an application.   Second, we have frequent application deadlines (monthly for CLIN1 and CLIN2), so we are always available when the applicant is ready to apply.  Third, we have other accelerating mechanisms in place to help grantees once they’ve received funding, such as the CIRM Alpha Stem Cell Clinics network of six clinical sites throughout California (more efficient clinical trial processes and patient recruitment) and Clinical Advisory Panels (CAPs) – that provide technical, clinical or regulatory expertise as well as patient advocate guidance to the grantee.  Finally, we Science Officers do our best to help every step of the way, from application through grant closeout.

We now feel confident that our recruitment efforts, combined with CIRM’s more efficient funding pipeline and review processes, are accelerating development of new therapies.  Back in 2016, a new CIRM Strategic Plan included the goal of recruiting 50 successful (i.e., funded) clinical trial applicants within five years.  This goal seemed like quite a stretch, since CIRM had funded fewer than 20 clinical trials in the previous ten years.  Fast-forward to the end of 2020, and CIRM had funded 51 new trials in those five years, for a grand total of 68 trials.    

Now, with the passage of Proposition 14 this past November, we are looking forward to bringing more cell and gene therapeutic candidates into clinical trials.   If you are developing one yourself, feel free to let us know… or don’t be surprised if you hear from us!  

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