Medeor Therapeutics Completes Enrollment in CIRM-Funded Clinical Trial for Kidney Transplant Patients

A CIRM-funded clinical trial to help kidney transplant patients avoid the need for anti-rejection or immunosuppressive medications has completed enrollment and transplantation of all patients.

Medeor Therapeutics’ MDR-101 Phase 3 multi-center clinical trial involved 30 patients; 20 of them were treated with MDR-101, and 10 control subjects were given standard care. CIRM awarded Medeor, based in South San Francisco, $18.8 million for this research in January 2018.

More than 650,000 Americans suffer from end-stage kidney disease – a life-threatening condition caused by the loss of kidney function. For these people the best treatment option is a kidney transplant from a genetically matched, living donor. Even matched patients, however, face a lifetime on immunosuppressive drugs to prevent their immune system from rejecting the transplanted organ. These drugs can be effective at preventing rejection, but they come at a cost. Because they are toxic these medications increase a transplant patient’s life-time risk of cancer, diabetes, heart disease and infections.

Medeor Therapeutics developed its MDR-101 therapy to reprogram the patient’s immune system to accept a transplanted kidney without the need for long term use of immunosuppression drugs.

The company takes peripheral blood stem cells from the organ donor and infuses them into the patient receiving the donor’s kidney. This creates a condition called “mixed chimerism” where immune cells from the donor help the patient’s immune system adapt to and tolerate the donor’s kidney. 

After a standard kidney transplant, the patient is given a combination of three anti-rejection medications which they typically have to remain on for the rest of their lives. However, the Medeor patients, by day 40 post-transplant, are only taking one medication and the hope is that immunosuppression is discontinued at the end of one year.

“Chronic kidney disease and kidney failure are a growing problem in the US, that’s why it’s so important that we find new ways to reduce the burden on patients and increase the odds of a successful transplant with long term benefit,” says Maria T. Millan, M.D., President and CEO of CIRM. “Medeor’s approach may not only reduce the likelihood of a patient’s body rejecting the transplanted organ, but it can also improve the quality of life for these people and reduce overall health care costs by eliminating the need to stay on these immunosuppressive medications for life.”

In an earlier Phase 2 trial, a majority of patients achieved mixed chimerism. Approximately 74 percent of those patients have been off all immunosuppressive drugs for more than two years, including some who continue to be off immunosuppressive medications 15 years after their surgery.

“Today’s news is a tremendous milestone not only for Medeor but for the entire transplant community. This is the first randomized, multi-center pivotal study designed specifically to stop the use of all immunosuppressive anti-rejection drugs post-transplant. This therapy can be a true game changer in our efforts to transform transplant outcomes and help patients live healthier lives,” said Dan Brennan, MD, Chief Medical Officer at Medeor Therapeutics.

If the results from this pivotal clinical trial show that MDR-101 is both safe and effective, Medeor may apply to the Food and Drug Administration (FDA) for approval to market their approach to other patients in the U.S.

Hitting our Goals: Accelerating to the finish line

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 #6 was Accelerate.

Ever wonder how long it takes for a drug or therapy to go from basic research to approval by the US Food and Drug Administration (FDA)? Around 12 years on average is the answer. That’s a long time. And it can take even longer for stem cell therapies to go that same distance.

There are a lot of reasons why it takes so long (safety being a hugely important element) but when we were sitting down in 2015 to put together our Strategic Plan we wanted to find a way to speed up that process, to go faster, without in any way reducing the focus on safety.

So, we set a goal of reducing the time it takes from identifying a stem cell therapy candidate to getting an Investigational New Drug (IND) approval from the FDA, which means it can be tested in a clinical trial. At the time it was taking us around eight years, so we decided to go big and try to reduce that time in half, to four years.

Then the question was how were we going to do that? Well, before we set the goal we did a tour of the major biomedical research institutions in California – you know, University of California Los Angeles (UCLA) UC San Francisco, Stanford etc. – and asked the researchers what would help them most. Almost without exception said “a clearing house”, a way to pair early stage investigators with later stage partners who possess the appropriate expertise and interest to advance the project to the next stage of development, e.g., helping a successful basic science investigator find a qualified partner for the project’s translational research phase.

So we set out to do that. But we didn’t stop there. We also created what we called Clinical Advisory Panels or CAPs. These consisted of a CIRM Science Officer with expertise on a particular area of research, an expert on the kind of research being done, and a Patient Representative. The idea was that CAPs would help guide and advise the research team, helping them overcome specific obstacles and get ready for a clinical trial. The Patient Representative could help the researchers understand what the needs of the patient community was, so that a trial could take those into account and be more likely to succeed. For us it wasn’t enough just to fund promising research, we were determined to do all we could to support the team behind the project to advance their work.

How did we do. Pretty good I would have to say. For our Translational stage projects, the average amount of time it took for them to move to the CLIN1 stage, the last stage before a clinical trial, was 4.18 years. For our CLIN1 programs, 73 percent of those achieved their IND within 2 years, meaning they were then ready to actually start an FDA-sanctioned clinical trial.

Of course moving fast doesn’t guarantee that the therapy will ultimately prove effective. But for an agency whose mission is “to accelerate stem cell therapies to patients with unmet medical needs”, going slow is not an option.

CIRM funded stem cell therapy could one day help stroke and dementia patients

Image Description: Microscope images showing brain tissue that has been damaged by white matter stroke (left) and then repaired by the new glial cell therapy (right). Myelin (seen in red), is a substance that protects the connections between neurons and is lost due to white matter stroke. As seen at right, the glial cell therapy (green) restores lost myelin and improves connections in the brain. | Credit: UCLA Broad Stem Cell Research Center/Science Translational Medicine

Dementia is a general term that describes problems with memory, attention, communication, and physical coordination. One of the major causes of dementia is white matter strokes, which occurs when multiple strokes (i.e. a lack of blood supply to the brain) gradually damages the connecting areas of the brain (i.e. white matter).

Currently, there are no therapies capable of stopping the progression of white matter strokes or enhancing the brain’s limited ability to repair itself after they occur.

However, a CIRM-funded study ($2.09 million) conducted by S. Thomas Carmichael, M.D., Ph.D. and his team at UCLA showed that a one-time injection of an experimental stem cell therapy can repair brain damage and improve memory function in mice with conditions that mimic human strokes and dementia.

The therapy consists of glial cells, which are a special type of cell present in the central nervous system that surround and protect neurons. The glial cells are derived from induced pluripotent stem cells (iPSCS), stem cells that are derived from skin or blood cells through the process of reprogramming and have the ability to become virtually any type of cell.

Dr. Carmichael and his team injected the newly developed glial cells into the brains of mice that had damage similar to humans in the early to middle stages of dementia. The team found that the cell therapy traveled to the damaged areas of the brain and secreted chemicals that stimulated the brain’s own stem cells to start repairing the damage. This not only limited the progression of damage, but also enhanced the formation of new neural connections and increased the production of myelin, a fatty substance that covers and protects neurons.

In a press release from UCLA, Francesca Bosetti, Ph.D., Pharm.D., Program Director at the National Institute of Neurological Disorders and Strokes, was optimistic about what these findings could mean for patients with strokes or dementia.

“These preliminary results suggest that glial cell-based therapies may one day help combat the white matter damage that many stroke and vascular dementia patients suffer every year.”

Another interesting finding from this study is that even if the injected cells were eliminated a few months after they had been transplanted, the mice’s recovery was unaffected. The researchers believe that this indicates that the therapy primarily serves as a way to stimulate the brain’s own repair process.

In the same press release, Dr. Carmichael elaborates on this concept.

“Because the cell therapy is not directly repairing the brain, you don’t need to rely on the transplanted cells to persist in order for the treatment to be successful.”

The team is now conducting the additional studies necessary to apply to the Food and Drug Administration (FDA) for permission to test the therapy in a clinical trial in humans. If the therapy is shown to be safe and effective through clinical trials in humans, the team envisions that it could be used at hospitals as a one-time treatment for people with early signs of white matter stroke.

The full results of this study were published in Science Translational Medicine.

Regulated, Reputable and Reliable: FDA’s Taking Additional Steps to Advance Safe and Effective Regenerative Medicine Products

Peter Marks, M.D., Ph.D., Director, Center for Biologics Evaluation and Research

In February 2020, CIRM presented a series of benchmarks for the responsible delivery of stem cell and regenerative medicine products. These benchmarks are outlined in the publication Regulated, reliable and reputable: Protect patients with uniform standards for stem cell treatments. In a nutshell, CIRM advocates for the delivery of regenerative medicine products in a context where:

  • The product is authorized by the Food and Drug Administration (FDA) and is overseen by an IRB or ethics board,
  • The treatment is delivered by qualified doctors, nurses, and technicians,
  • Treatment occurs at a clinical treatment center with expertise in regenerative medicine, and
  • There is ongoing monitoring and follow-up of patients.

On April 21 of 2021, Dr. Peter Marks, Director of the Center for Biologics Evaluation and Research, indicated the FDA’s intent to ensure new regenerative medicine products are FDA-authorized. Specifically, the FDA will require product developers to obtain an Investigational New Drug or IND authorization. In his news release Dr. Marks says the agency is willing to exercise more enforcement of these rules should clinics or therapy producers fail to follow these guidelines.

“These regenerative medicine products are not without risk and are often marketed by clinics as being safe and effective for the treatment of a wide range of diseases or conditions, even though they haven’t been adequately studied in clinical trials. We’ve said previously and want to reiterate here – there is no room for manufacturers, clinics, or health care practitioners to place patients at risk through products that violate the law, including by not having an IND in effect or an approved biologics license. We will continue to take action regarding unlawfully marketed products.”

IND authorization is particularly important as the agency pays close attention to how the product is produced and whether there is a scientific rationale and potential clinical evidence that it may be effective against the specific disease condition. All CIRM-funded clinical trials and all trials conducted in the CIRM Alpha Stem Cell Clinics Network must have IND authorization.

Regenerative medicine products are generally created from human cells or tissues. These products are frequently referred to as “living medicines.” The “living” nature of these products is what contributes to their remarkable potential to relieve, stop or reverse disease in a durable or sustainable manner.

The risk with unregulated products is that there is no assurance that they have been  produced in a quality controlled process or manner  where all components of the  injected material have been well characterized and studied for safety and efficacy for a given disease as well as a specific site in the body. In addition, there is no way to ensure that unregulated products meet standards or quality specifications such as ensuring that they have the active and beneficial component while making sure that they do not include harmful contaminants..  There have been documented examples of patients being severely injured by unregulated and inadequately characterized products. For example, in 2017 three Florida women were blinded by an unauthorized product.  Dr. George Daley, a stem cell expert and the Dean of Harvard Medical School, described the clinic operators as “charlatans peddling the modern equivalent of snake oil.”

To receive FDA authorization, detailed scientific data and well controlled clinical data are required to ensure safety and a demonstration that  the product is safe has the potential to improve or resolve the patient’s disease condition.

While it seems both important and self-evident that stem cell products be safe and effective and supported by evidence they can impact the patient’s disease condition, that doesn’t always happen. Unfortunately, too many patients have experienced unnecessary medical risks and financial harm from unauthorized treatments. CIRM applauds the FDA for taking additional steps to advance regenerative medicine products where the clinical benefits of such therapies outweigh any potential harms.

Hitting our goals: Making good progress

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 #5 was Advance.

A dictionary definition of progression is “The act of moving forward or proceeding in a course.” That’s precisely what we set out to do when we set one of the goals in our 2015 Strategic Plan. We wanted to do all that we could to make sure the work we were funding could advance to the next stage. The goal we set was:

Advance: Increase projects advancing to the next stage of development by 50%.

The first question we faced was what did we mean by progression and how were we going to measure it? The answer basically boiled down to this: when a CIRM award completes one stage of research and gets CIRM funding to move on to the next stage or to develop a second generation of the same device or therapy.

In the pre-2016 days we’d had some success, on average getting around nine progression events every year. But if we were going to increase that by 50 percent we knew we had to step up our game and offer some incentives so that the team behind a successful project had a reason, other than just scientific curiosity, to try and move their research to the next level.

So, we created a series of linkages between the different stages of research, so the product of each successful investment was the prerequisite for the next stage of development for the research or technology.

We changed the way we funded projects, going from offering awards on an irregular basis to having them happen according to a pre-defined schedule with each program type offered multiple times a year. This meant potential applicants knew when the next opportunity to apply would come, enabling them to prepare and file at the time that was best for them and not just because we said so. We also timed these schedules so that programs could progress from one stage to the next without interruption.

But that’s not all. We recognized that some people may be great scientists at one level but didn’t have the experience or expertise to carry their project forward. So, we created both an Accelerating Center and Translating Center to help them do that. The Translating Center helped projects do the work necessary to get ready to apply to the US Food and Drug Administration (FDA) for permission to start a clinical trial. The Accelerating Center helped the team prepare that application for the trial and then plan how that trial would be carried out.

Creating these two centers had an additional benefit; it meant the work that did progress did so faster and was of a higher quality than it might otherwise have been.

Putting all those new building blocks in place meant a lot of work for the CIRM team, on top of their normal duties. But, as always, the team rose to the challenge. By the end of December 2020, a total of 74 projects had advanced or progressed to the next level, an increase of 100 percent on our pre-2016 days.

When we were laying out the goals we said that “The full implementation of these programs will create the chassis of a machine that provides a continuous, predictable, and timely pathway for the discovery and development of promising stem cell treatments.” Thanks to the voter approved Proposition 14 we now have the fund to help those treatments realize that promise.

Three UC’s Join Forces to Launch CRISPR Clinical Trial Targeting Sickle Cell Disease

Sickle shaped red blood cells

The University of California, San Francisco (UCSF), in collaboration with UC Berkeley (UCB) and UC Los Angeles (UCLA), have been given permission by the US Food and Drug Administration (FDA) to launch a first-in-human clinical trial using CRISPR technology as a gene-editing technique to cure Sickle Cell Disease.

This research has been funded by CIRM from the early stages and, in a co-funding partnership with theNational Heart, Lung, and Blood Institute under the Cure Sickle Cell initiatve, CIRM supported the work that allowed this program to gain FDA permission to proceed into clinical trials.    

Sickle Cell Disease is a blood disorder that affects around 100,000 people, mostly Black and Latinx people in the US. It is caused by a single genetic mutation that results in the production of “sickle” shaped red blood cells. Normal red blood cells are round and smooth and flow easily through blood vessels. But the sickle-shaped ones are rigid and brittle and clump together, clogging vessels and causing painful crisis episodes, recurrent hospitalization, multi-organ damage and mini-strokes.    

The three UC’s have combined their respective expertise to bring this program forward.

The CRISPR-Cas9 technology was developed by UC Berkeley’s Nobel laureate Jennifer Doudna, PhD. UCLA is a collaborating site, with expertise in genetic analysis and cell manufacturing and UCSF Benioff Children’s Hospital Oakland is the lead clinical center, leveraging its renowned expertise in cord blood and marrow transplantation and in gene therapy for sickle cell disease.

The approach involves retrieving blood stem cells from the patient and, using a technique involving electrical pulses, these cells are treated to correct the mutation using CRISPR technology. The corrected cells will then be transplanted back into the patient.

Dr. Mark Walters

In a news release, UCSF’s Dr. Mark Walters, the principal investigator of the project, says using this new gene-editing approach could be a game-changer. “This therapy has the potential to transform sickle cell disease care by producing an accessible, curative treatment that is safer than the current therapy of stem cell transplant from a healthy bone marrow donor. If this is successfully applied in young patients, it has the potential to prevent irreversible complications of the disease. Based on our experience with bone marrow transplants, we predict that correcting 20% of the genes should be sufficient to out-compete the native sickle cells and have a strong clinical benefit.”

Dr. Maria T. Millan, President & CEO of CIRM, said this collaborative approach can be a model for tackling other diseases. “When we entered into our partnership with the NHLBI we hoped that combining our resources and expertise could accelerate the development of cell and gene therapies for SCD. And now to see these three UC institutions collaborating on bringing this therapy to patients is truly exciting and highlights how working together we can achieve far more than just operating individually.”

The 4-year study will include six adults and three adolescents with severe sickle cell disease. It is planned to begin this summer in Oakland and Los Angeles.

The three UCs combined to produce a video to accompany news about the trial. Here it is:

Prime Time for Rocket

Rocket Pharmaceuticals, a company that specializes in developing genetic therapies for rare childhood disorders, just got a big boost from the European Medicines Agency (EMA). They were given a Priority Medicines (PRIME) designation for their therapy for Leukocyte Adhesion Deficiency-1 (LAD-1).

CIRM is funding ($6.56 million) Rocket’s clinical trial for LAD-I, an immune disorder that leaves patients vulnerable to repeated infections that often results in death within the first two years of life. The therapy involves taking some of the child’s own blood stem cells and, in the lab, correcting the mutation that causes LAD-I, then returning those cells to the patient. Hopefully those blood stem cells then create a new, healthy blood supply and repair the immune system.

The therapy, called RP-L201, is already showing promise in the clinical trial, hence the PRIME designation. The program was set up to help speed up development and evaluation of therapies that could help patients who have limited treatment options. Getting a PRIME designation means it is considered a priority by EMA and could reach patients sooner.

In the US, Rocket has won similar recognition from the Food and Drug Administration (FDA) and has been granted Regenerative Medicine Advanced Therapy (RMAT), Rare Pediatric Disease, and Fast Track designations.

In a news release Kinnari Patel, President and Chief Operating Officer of Rocket, said the designation showed that regulators understand the urgent need to develop a therapy for patients with LAD-1. “More than half of LAD-I patients suffer with a severe variant in which mortality occurs in up to 75% of young children who don’t receive a successful bone marrow transplant by the age of two. Securing all possible accelerated designations will enable us to collaborate with both the FDA and EMA to speed the development and delivery of a potential treatment for these patients.  We look forward to sharing initial Phase 2 data from our potentially registration-enabling LAD-I trial in the second quarter of 2021.”

That trial has now completed enrolling patients (nine altogether) but their treatments are not yet complete. LAD-1 patients with severe disease have low levels of a key protein called CD18, usually less than 2%. Of the first three patients treated in this trial CD18 levels are all higher than the 4-10% threshold considered necessary for these children to survive into adulthood. Another encouraging sign is that there were no serious side effects from the therapy.

Obviously there is still a long way to go before we know if this therapy really works, but the PRIME designation – along with the similar ones in the US – are recognition that this is a very promising start.

Going the extra mile to save a patient’s life

You can tell an awful lot about a company by the people it hires and the ability it gives them to do their job in an ethical, principled way. By that measure Rocket Pharma is a pretty darn cool company.

Rocket Pharma is running a CIRM-funded clinical trial for Leukocyte Adhesion Deficiency-I (LAD-I), a rare genetic immune disorder that leaves patients vulnerable to repeated infections that often results in death within the first two years of life. The therapy involves taking some of the child’s own blood stem cells and, in the lab, correcting the mutation that causes LAD-I, then returning those cells to the patient. Hopefully those blood stem cells then create a new, healthy blood supply and repair the immune system.

So far, they have treated the majority of the nine patients in this Phase 1/2 clinical trial. Here’s the story of three of those children, all from the same family. Every patient’s path to the treatment has been uniquely challenging. For one family, it’s been a long, rough road, but one that shows how committed Rocket Pharma (Rocket) is to helping people in need.

The patient, a young girl, is from India. The family has already lost one child to what was almost certainly LAD-I, and now they faced the very real prospect of losing their daughter too. She had already suffered numerous infections and the future looked bleak. Fortunately, the team at Rocket heard about her and decided they wanted to help enroll her in their clinical trial.

Dr. Gayatri Rao, Rocket Pharmaceuticals

Dr. Gayatri Rao, the Global Program Head for the LAD-I therapy, this patient was about 6 months old when they heard about her: “She had already been in and out of the hospital numerous times so the family were really interested in enrolling the patient. But getting the family to the US was daunting.”

Over the course of several months, the team at Rocket helped navigate the complicated immigration process. Because the parents and child would need to make several trips to the US for treatment and follow-up exams they would need multiple-entry visas. “Just to get all the paper work necessary was a monumental task. Everything had to be translated because the family didn’t speak English. By the time the family flew to Delhi for their visa interview they had a dossier that filled a 3 inch binder.”  Rocket worked closely with partners in India to provide the family on-the-ground support every step of the way.  To help ensure the family received the visas they needed, Rocket also reached out to members of Congress and six members wrote in support of the family’s application.

Finally, everything fell into place. The family had the visas, all the travel arrangements were made. The Rocket team had even found an apartment near the UCLA campus where the family would stay during the treatment and stocked it with Indian food.

But on the eve of their flight to the US, the coronavirus pandemic hit. International flights were cancelled. Borders were closed. A year of work was put on hold and, more important, the little girl’s life hung in the balance.

Over the course of the next few months the little girl suffered several infections and had to be hospitalized. The family caught COVID and had to undergo quarantine till they recovered. But still the Rocket team kept working on a plan to bring them to the US. Finally, in late January, as vaccines became available and international flights opened up once again, the family were able to come to the US. One west-coast based Rocket team member even made sure that upon arriving to the apartment in UCLA, there was a home-cooked meal, a kitchen stocked with groceries, and handmade cards welcoming them to help transition the family into their new temporary “home.” They are now in living in that apartment near UCLA, waiting for the treatment to start.

Gayatri says it would have been easy to say: “this is too hard” and try to find another patient in the trial, but no one at Rocket wanted to do that: “Once a patient gets identified, we feel like we know them and the team feels invested in doing everything we can for them. We know it may not work out. But at the end of the day, we recognize that this child often has no other choices, and that motivates us to keep going despite the challenges.  If anything, this experience has taught us that with persistence and creativity, we can surmount these challenges.”

Maybe doing the right thing brings its own rewards, because this earlier this month Rocket was granted Regenerative Medicine Advanced Therapy (RMAT) designation for their treatment for LAD-I. This is a big deal because it means the therapy has already shown it appears to be safe and potentially beneficial to patients, so the designation means that if it continues to be safe and effective it may be eligible for a faster, more streamlined approval process. And that means it can get to the patients who need it, outside of a clinical trial, faster.

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.

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.