CIRM Builds Out World Class Team With 5 New hires

Kevin Marks, CIRM’s new General Counsel. Photo courtesy Modern-Counsel.com

Following the passage of Proposition 14 CIRM has hired five new employees to help increase the team’s ability to respond to new challenges and responsibilities.

Prop 14, which was approved by voters in November 2020, gives CIRM $5.5 billion in new funding. Those funds mean CIRM can once again fund research from Discovery, through Translational and Clinical, as well as create new education and training programs. Prop 14 also adds new areas of focus for the Stem Cell Agency including creating an Accessibility and Affordability Working Group, expanding the Alpha Stem Cell Clinic network and creating new Centers of Excellence in underserved parts of California. To meet those new responsibilities the Agency has hired a highly talented group of individuals. Those include:

Kevin Marks is CIRM’s new General Counsel. Kevin studied Russian at college and originally wanted to be a diplomat, but when that didn’t work out he turned to the law. He became a highly accomplished, skilled advisor with global and domestic expertise and a history of delivering innovative legal and business results. He has served as Vice President and Head of Legal and Compliance at Roche Molecular Solutions, VP and General Counsel at Roche Molecular Diagnostics and VP and General Counsel at Roche Palo Alto, LLC.

“We are so delighted to have Kevin Marks join CIRM as a member of our executive Leadership Team,” says Maria T. Millan, MD, CIRM’s President and CEO. “He brings unique qualifications and critical skills during the formative phase and launch of our new strategic plan for California’s $5.5B investment in stem cell, genomics and regenerative medicine research and therapy development. As general counsel, he will oversee the legal department, human resources, grants management and operations at the Agency. Kevin has an established track record with global and domestic expertise and a history of delivering innovative legal and business solutions.”

“He is revered by his colleagues as an exceptional leader in his profession and in the community. Kevin is known for developing people as well as programs, and for promoting racial, ethnic and gender diversity.”

“I am incredibly honored to be joining CIRM at this stage of its journey,” says Marks. “I see the opportunity to contribute to positive patient outcomes–especially those patients with unmet medical needs–by working towards accelerating stem cell research in California as a member of the CIRM team as rewarding and perfectly aligned with my professional and personal goals.”

Pouneh Simpson as Director of Finance. Pouneh comes to CIRM from the Governor’s Office of Emergency Services in California, where she served as the Recovery Financial Administration Chief. At OES she worked with local, state, and federal government stakeholders on disaster recovery planning, exercises, and grant administration, specifically, overseeing the grant processing of all disaster recovery activity.

Prior to that Pouneh worked as the Chief Financial Officer of the Veterans Homes, where she managed finances at eight Veterans Homes with over 2,800 positions and $365 million in General Fund support. She also led the writing of legislation, regulations, policies and procedures for Cal Vet, overhauling the business and financial portions of eight Veterans Homes.

Mitra Hooshmand, PhD. as Senior Science Officer for Special Projects and Initiatives. Mitra joins CIRM after more than five years of leadership experience at Americans for Cures, a stem cell advocacy group. During this time, she mobilized hundreds of stakeholders, from scientists to national and local patient advocacy organizations, to generate support for CIRM’s mission.

Mitra has a PhD. in Anatomy and Biology from the University of California at Irvine. She also worked as a Project Scientist at the Sue and Bill Gross Stem Cell Research Center at UC Irvine, where she conducted and published academic and industry-partnered research in studies investigating the therapeutic benefit of human neural stem cell transplantation in preclinical models of spinal cord injury and aging.

Vanessa Singh, as Human Resources Manager. Vanessa has 15 years of experience working for the state of California, working at the Departments of General Services, Insurance and Human Services. In those roles she gained experience in performing, processing, developing, implementing, and advising on many personnel aspects such as compensation, benefits, classifying positions, recruitment and hiring, salary structure (exempt and civil service), organization structure, and retirement.

When COVID struck Vanessa stepped up to help. She worked as a Case Investigator for San Bernardino Local Health Jurisdiction, Department of Public Health, doing contact tracing. She talked to people diagnosed with coronavirus and collected information on people they may have had close contact with who may have been exposed to the virus.

Claudette Mandac as Project Manager Review. Claudette has more than seven years’ experience with UCSF’s Human Research Protection Program. In that role she prepared protocols for scientific, regulatory and ethical review, pre-screening submissions to ensure they were complete and consistent, and then routing them to the appropriate reviewers for administrative, expedited or Committee review. She also managed an Institutional Review Board Committee, preparing and distributing protocols for review by designated scientific and nonscientific reviewers, coordinating meetings, recruiting and training members, and maintaining records of conflicts of interest. At UCSF she annually helped process up to 3,000 IRB modifications, continuing reviews, and post-approval safety reports for domestic and international socio-behavioral or biomedical research.

Claudette has two degrees from U.C. Berkeley; one in Arts and History and another in Science, Conservation and Resource Studies.

New technique maps out diversity and location of cells in tissue or tumor

Image Description: Alex Marson is part of a team of researchers who developed a new technique to map the specialized diversity and spatial location of individual cells within a tissue or tumor. Photo Credit: Anastasiia Sapon

All the cells in your body work together and each can have a different role. Their individual function not only depends on cell type, but can also depend on their specific location and surroundings.

A CIRM supported and collaborative study at the Gladstone Institutes, UC San Francisco (UCSF), and UC Berkeley has developed a more efficient method than ever before to simultaneously map the specialized diversity and spatial location of individual cells within a tissue or a tumor.

The technique is named XYZeq and involves segmenting a tissue into microscopic regions. Within each of these microscopic grids, each cell’s genetic information is analyzed in order to better understand how each particular cell functions relative to its spacial location.

For this study, the team obtained tissue from mice with liver and spleen tumors. A slice of tissue was then placed on a slide that divides the tissue into hundreds of “microwells” the size of a grain of salt. Each cell in the tissue gets tagged with a unique “molecular barcode” that represents the microwell it’s contained in, much like a zip code. The cells are then mixed up and assigned a second barcode to ensure that each cell within a given square can be individually identified, similar to a street address within a zip code. Finally, the genetic information in the form of RNA from each cell is analyzed. Once the results are obtained, both barcodes tell the researchers exactly where in the tissue it came from.

The team found that some cell types located near the liver tumor were not evenly spaced out. They also found immune cells and specific types of stem cells clustered in certain regions of the tumor. Additionally, certain stem cells had different levels of some RNA molecules depending on how far they resided from the tumor.

The researchers aren’t entirely sure what this pattern means, but they believe that it’s possible that signals generated by or near the tumor affect what nearby cells do.

In a press release, Alex Marson, M.D., Ph.D., a senior author of the study, elaborates on what the XYZeq technology could mean for disease modeling.

“I think we’re actually taking a step toward this being the way tissues are analyzed to diagnose, characterize, or study disease; this is the pathology of the future.”

The full results of the study were published in Science Advances.

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.

UCSF Nursing Professor Joins CIRM Board

Elena Flowers, PhD, RN, newest member of the CIRM Board: Photo courtesy UCSF

Elena Flowers, PhD, RN, an associate professor of physiological nursing at the University of California, San Francisco (UCSF) is joining the Board of the California Institute for Regenerative Medicine (CIRM), the state’s Stem Cell Agency.

Dr. Flowers was appointed to the Board by State Controller Betty T. Yee who said: “Ms. Flowers’ experience and express commitment to equitable health outcomes for California’s diverse communities will bring a valued perspective to the work ahead.”

Dr. Flowers is a member of the UCSF Institute for Human Genetics and the International Society of Nurses in Genetics. As a researcher her work focuses on genomics involving precision medicine and risk factors for cardiovascular health and type 2 diabetes. She is also a teacher and has lectured internationally on issues such as topics from racial disparities in Type 2 Diabetes to the implications of genomic technologies for the nursing workforce.

CIRM Board Chair, Jonathan Thomas, PhD, JD, welcomed the appointment: “Dr. Flowers brings a wealth of experience and expertise to our Board and, as a nurse, she will bring a different perspective to the work we do and help us in trying to better address the needs of underserved communities.” 

“I am honored to have the opportunity to serve the citizens of California in this capacity,” says Dr. Flowers. “CIRM has ambitious goals, seeking to improve upon common limitations of public research agencies by its commitment to delivering meaningful findings and ultimately treatments for patients as rapidly as possible. I’m particularly committed to improving inclusion and access to these treatments across the entire diverse California population.”

Dr. Flowers got her undergraduate degree at UC Davis and then served as a research assistant at Zuckerberg San Francisco General Hospital. She then went on to get her MS and Doctor of Philosophy degrees at the UCSF School of Nursing.

In her spare time she has no spare time because she is the mother of two young daughters.

CIRM-funded kidney transplant procedure eyeing faster approval

Kidney transplant surgery.

Medeor Therapeutics, which is running a CIRM-funded clinical trial to help people getting kidney transplants, just got some really good news. The US Food and Drug Administration (FDA) has just granted their product Regenerative Medicine Advanced Therapy (RMAT) designation. That’s a big deal because it means they may be able to apply for faster review and approval and get their therapy to more patients faster.

Here’s why that RMAT designation matters.

Over 650,000 Americans suffer from end-stage kidney disease – a life-threatening condition caused by the loss of kidney function. The best available treatment for these patients is a kidney transplant from a genetically matched living donor. However, patients who receive a transplant must take life-long immunosuppressive drugs to prevent their immune system from rejecting the transplanted organ. Over time, these drugs are toxic and can increase a patient’s risk of infection, heart disease, cancer and diabetes.  Despite these drugs, many patients still lose transplanted organs due to rejection.

To tackle this problem Medeor is developing a stem cell-based therapy called MDR-101. This is being tested in a Phase 3 clinical trial and it’s hoped it will eliminate the need for immunosuppressive drugs in genetically matched kidney transplant patients.

The company takes blood-forming stem cells and immune cells from the organ donor and infuses them into the patient receiving the donor’s kidney. Introducing the donor’s immune cells into the patient creates a condition called “mixed chimerism” where immune cells from the patient and the donor are able to co-exist. In this way, the patient’s immune system is able to adapt to and tolerate the donor’s kidney, potentially eliminating the need for the immunosuppressive drugs that are normally necessary to prevent transplant rejection.

So how does getting RMAT designation help that? Well, the FDA created the RMAT program to help speed up the development and review of regenerative medicine therapies that can treat, modify, reverse, or cure a serious condition. If MDR-101shows it is both safe and effective RMAT could help it get faster approval for wider use.

In a news release Giovanni Ferrara, President and CEO of Medeor, welcomed the news.

“This important designation underscores the tremendous unmet medical need for alternatives to today’s immunosuppressive therapies for transplantation. We have the potential to help people live longer, healthier lives without the need for high dose and chronic immunosuppression and we thank the FDA for this designation that will assist us progressing as efficiently as possible toward a commercially available product.”

This is the seventh CIRM-supported project that has been granted RMAT designation. The others are jCyte, Lineage, Humacyte, St. Jude’s/UCSF X-linked SCID, Poseida, Capricor

Building a progressive pipeline

Dr. Kelly Shepard

By Dr. Kelly Shepard

One of our favorite things to do at CIRM is deliver exciting news about CIRM projects. This usually entails discussion of recent discoveries that made headlines, or announcing the launch of a new CIRM-funded clinical trial …. tangible signs of progress towards addressing unmet medical needs through advances in stem technology.

But there are equally exciting signs of progress that are not always so obvious to the untrained eye-  those that we are privileged to witness behind the scenes at CIRM. These efforts don’t always lead to a splashy news article or even to a scientific publication, but they nonetheless drive the evolution of new ideas and can help steer the field away from futile lines of investigation. Dozens of such projects are navigating uncharted waters by filling knowledge gaps, breaking down technical barriers, and working closely with regulatory agencies to define novel and safe paths to the clinic.

These efforts can remain “hidden” because they are in the intermediate stages of the long, arduous and expensive journey from “bench to beside”.  For the pioneering projects that CIRM funds, this journey is unique and untrod, and can be fraught with false starts. But CIRM has developed tools to track the momentum of these programs and provide continuous support for those with the most promise. In so doing, we have watched projects evolve as they wend their way to the clinic. We wanted to share a few examples of how we do this with our readers, but first… a little background for our friends who are unfamiliar with the nuts and bolts of inventing new medicines.

A common metaphor for bringing scientific discoveries to market is a pipeline, which begins in a laboratory where a discovery occurs, and ends with government approval to commercialize a new medicine, after it is proven to be safe and effective. In between discovery and approval is a stage called “Translation”, where investigators develop ways to transition their “research level” processes to “clinically compatible” ones, which only utilize substances that are of certified quality for human use. 

Investigators must also work out novel ways to manufacture the product at larger scale and transition the methods used for testing in animal models to those that can be implemented in human subjects.

A key milestone in Translation is the “preIND” (pre Investigational New Drug (IND) meeting, where an investigator presents data and plans to the US Food and Drug Administration (FDA) for feedback before next stage of development begins, the pivotal testing needed to show it is both safe and effective.

These “IND enabling studies” are rigorous but necessary to support an application for an IND and the initiation of clinical trials, beginning with phase 1 to assess safety in a small number of individuals, and phase 2, where an expanded group is evaluated to see if the therapy has any benefits for the patient. Phase 3 trials are studies of very large numbers of individuals to gain definitive evidence of safety and therapeutic effect, generally the last step before applying to the FDA for market approval. An image of the pipeline and the stages described are provided in our diagram below.

The pipeline can be notoriously long and tricky, with plenty of twists, turns, and unexpected obstacles along the way. Many more projects enter than emerge from this gauntlet, but as we see from these examples of ‘works in progress”, there is a lot of momentum building.

Caption for Graphic: This graphic shows the number of CIRM-funded projects and the stages they have progressed through multiple rounds of CIRM funding. For example, the topmost arrow shows that are about 19 projects at the translational stage of the pipeline that received earlier support through one of CIRM’s Discovery stage programs. Many of these efforts came out of our pre-2016 funding initiatives such as Early Translation, Basic Biology and New Faculty Awards. In another example, you can see that about 15 awards that were first funded by CIRM at the IND enabling stage have since progressed into a phase 1 or phase 2 clinical trials. While most of these efforts also originated in some of CIRM’s pre-2016 initiatives such as the Disease Team Awards, others have already progressed from CIRM’s newer programs that were launched as part of the “2.0” overhaul in 2016 (CLIN1).

The number of CIRM projects that have evolved and made their way down the pipeline with CIRM support is impressive, but it is clearly an under-representation, as there are other projects that have progressed outside of CIRM’s purview, which can make things trickier to verify.

We also track projects that have spun off or been licensed to commercial organizations, another very exciting form of “progression”. Perhaps those will contribute to another blog for another day! In the meantime, here are a just a few examples of some of the progressors that are depicted on the graphic.

Project: stem cell therapy to enhance bone healing in the elderly

– Currently funded stage: IND enabling development, CLIN1-11256 (Dr. Zhu, Ankasa Regenerative Therapeutics)

– Preceded by preIND-enabling studies, TRAN1-09270 (Dr. Zhu, Ankasa Regenerative Therapeutics)

– Preceded by discovery stage research grant TR1-01249 (Dr. Longaker and Dr. Helm, Stanford)

Project: embryonic stem cell derived neural cell therapy for Huntington Disease

– Currently funded stage: IND enabling development, CLIN1-10953 (Dr. Thompson, UC Irvine)

– Preceded by preIND-enabling studies, PC1-08117 (Dr. Thompson, UC Irvine)

– Preceded by discovery stage research grant (TR2-01841) (Dr. Thompson, UC Irvine)

Project: gene-modified hematopoietic stem cells for Artemis Deficient severe combined immunodeficiency (SCID)

– Currently funded stage: Phase 1 clinical trial CLIN2-10830 (Dr. Cowan, UC San Francisco)

– Preceded by IND enabling development, CLIN1-08363 (Dr. Puck, UC San Francisco)

– Preceded by discovery stage research grant, TR3-05535  (Dr. Cowan, UC San Francisco)

Project: retinal progenitor cell therapy for retinitis pigmentosa

– Currently funded stage: Phase 2 and 2b clinical trials, CLIN2-11472, CLIN2-09698 (Dr. Klassen, JCyte, Inc.)

– Preceded by IND enabling development, DR2A-05739 (Dr. Klassen, UC Irvine)

– Preceded by discovery stage research grant, TR2-01794 (Dr. Klassen, UC Irvine)

CIRM Funded Trial for Parkinson’s Treats First Patient

Dr. Krystof Bankiewicz

Brain Neurotherapy Bio, Inc. (BNB) is pleased to announce the treatment of the first patient in its Parkinson’s gene therapy study.  The CIRM-funded study, led by Dr. Krystof Bankiewicz, is one of the 64 clinical trials funded by the California state agency to date.

Parkinson’s is a neurodegenerative movement disorder that affects one million people in the U.S alone and leads to shaking, stiffness, and problems with walking, balance, and coordination.  It is caused by the breakdown and death of dopaminergic neurons, special nerve cells in the brain responsible for the production of dopamine, a chemical messenger that is crucial for normal brain activity.

The patient was treated at The Ohio State University Wexner Medical Center with a gene therapy designed to promote the production of a protein called GDNF, which is best known for its ability to protect dopaminergic neurons, the kind of cell damaged by Parkinson’s. The treatment seeks to increase dopamine production in the brain, alleviating Parkinson’s symptoms and potentially slowing down the disease progress.

“We are pleased to support this multi-institution California collaboration with Ohio State to take a novel first-in-human gene therapy into a clinical trial for Parkinson’s Disease.” says Maria T. Millan, M.D., President and CEO of CIRM.  “This is the culmination of years of scientific research by the Bankiewicz team to improve upon previous attempts to translate the potential therapeutic effect of GDNF to the neurons damaged in the disease. We join the Parkinson’s community in following the outcome of this vital research opportunity.”

CIRM Board Member and patient advocate David Higgins, Ph.D. is also excited about this latest development.  For Dr. Higgins, advocating for Parkinson’s is a very personal journey since he, his grandmother, and his uncle were diagnosed with the disease.

“Our best chance for developing better treatments for Parkinson’s is to test as many logical approaches as possible. CIRM encourages out-of-the-box thinking by providing funding for novel approaches. The Parkinson’s community is a-buzz with excitement about the GDNF approach and looks to CIRM to identify, fund, and promote these kinds of programs.”

In a news release Dr. Sandra Kostyk, director of the Movement Disorders Division at Ohio State Wexner Medical Center said this approach involves infusing a gene therapy solution deep into a part of the brain affected by Parkinson’s: “This is a onetime treatment strategy that could have ongoing lifelong benefits. Though it’s hoped that this treatment will slow disease progression, we don’t expect this strategy to completely stop or cure all aspects of the disease. We’re cautiously optimistic as this research effort moves forward.” 

Other trial sites located in California that are currently recruiting patients are the University of California, Irvine (UCI) and the University of California, San Francisco (UCSF). Specifically, the Irvine trial site is using the UCI Alpha Stem Cell Clinic, one of five leading medical centers throughout California that make up the CIRM Alpha Stem Cell Clinic (ASSC) Network.  The ASSC Network specializes in the delivery of stem cell therapies by providing world-class, state of the art infrastructure to support clinical research.

For more information on the trial and enrollment eligibility, you can directly contact the study coordinators by email at the trial sites listed:

  1. The Ohio State University: OSUgenetherapyresearch@osumc.edu
  2. University of California, San Francisco: GDNF@ucsf.edu
  3. University of California, Irvine: chewbc@hs.uci.edu

CIRM joins forces with US Department of Defense to fight COVID

Photo courtesy of Gabrielle Lurie / San Francisco Chronicle / Polaris

Small state agencies like CIRM don’t normally get to partner with a behemoth like the Department of Defense (DOD), but these are not normal times. Far from it. That’s why we are both joining forces with the National Institutes of Health to fund a clinical trial that hopes to help patients on a ventilator battling a sometime fatal condition that attacks their lungs.

The study is being run by Dr. Michael Matthay from U.C. San Francisco. CIRM awarded Dr. Matthay $750,000 to help expand an existing trial and to partner with U.C. Davis to bring in more patients, particularly from underserved communities.

This approach uses mesenchymal stem cells (MSCs) taken from bone marrow to help patients with a condition called acute respiratory distress syndrome (ARDS). This occurs when the virus attacks the lungs.

In an article in UCSF News, Dr. Matthay says the impact can be devastating.

“Tiny air spaces in the lungs fill up with fluid and prevent normal oxygen uptake in the lungs. That’s why the patient has respiratory failure. Usually these patients have to be intubated and treated with a mechanical ventilator.”

Many patients don’t survive. Dr. Matthay estimates that as many as 60 percent of COVID-19 patients who get ARDS die.

This is a Phase 2 double blind clinical trial which means that half the 120 patients who are enrolled will get MSCs (which come from young, health donors) and the other half will get a placebo. Neither the patients getting treated nor the doctors and nurses treating them will know who gets what.

Interestingly this trial did not get started as a response to COVID-19. In fact, it’s the result of years of work by Dr. Matthay and his team hoping to see if MSC’s could help people who have ARDs as a result of trauma, bacterial or other infection. They first started treating patients earlier this year when most people still considered the coronavirus a distant threat.

“We started the study in January 2020, and then COVID-19 hit, so we have been enrolling patients over the last eight months. Most of the patients we’ve enrolled in the trial have ended up having severe viral pneumonia from COVID.”

So far CIRM has funded 17 different projects targeting COVID-19. You can read about those in our Press Release section.

Saying farewell to an old friend

There are some people who, when you think of them, always bring a smile to your face. Dr. Bert Lubin was one of those people. Sadly, we lost Bert to brain cancer two days ago. But the impact he had, not just as an advocate for stem cell research but as a pioneer in sickle cell disease research and a champion for children’s health, will live on.

Bert had a number of official titles but probably the one he was most proud of was President & CEO of Children’s Hospital Oakland (now UCSF Benioff Children’s Hospital Oakland). But it wasn’t the title that he cared about, it was the opportunity it gave him to make a difference in the life of children in Oakland, to create a program to find new treatments and cures for a life-threatening disease. And he has made a difference.

As I started to write this tribute to Bert, I thought about who I should ask for a quote. And then I realized I had the perfect person. Bert himself. I was fortunate enough to interview him in December 2018, when he decided to step down after eight years on the CIRM Board.  As always, he had his own positive spin on that, saying: “I don’t see myself leaving. I’m just repurposing what is my role in CIRM. I’m recycling and reinventing.”

And Bert was always full of invention.

He grew up in Bellevue, a small town outside Pittsburgh, PA. His parents ran a fruit and vegetable market there and, growing up, Bert often worked in the store. It wasn’t something he enjoyed but he said he learned some valuable lessons.

“I think what happened in my childhood is that I learned how to sell. I am a salesman. I hated working in that store, I hated it, but I liked the communication with people, they trusted me, I could sell things and they were good things. Like Christmas. I’m Jewish, we were the only Jews in that community, and at Christmas we sold Christmas trees, but the trees were sometimes crooked and they were $2.99 a tree so I convinced families that I could go to their house and set the tree so it looked straight and I helped them decorate it and they loved it.”

He said, thinking back on his life it’s almost as if there were a plan, even if he wasn’t aware of it.

“I started thinking about that more recently, I started wondering how did this even happen? I’m not a religious person but it’s almost like there’s some fate. How did I get there? It’s not that I planned it that way and it’s certainly not that my parents planned it because I was the first in my family to go to high school let alone college. My parents, when I went to medical school and then decided I wanted to spend more time in an academic direction, they were upset. They wanted me to go into practice in a community that I grew up in and be economically secure and not be on the fringe in what an academic life is like.”

And then, fate stepped in and brought him to the San Francisco Bay Area.

“What happened was, I was at the University of Pennsylvania having trained at Boston Children’s and Philadelphia Children’s, where I had started a sickle cell disease program, and was asked to look at a job in southern California to start a sickle cell program there. So, I flew to San Francisco because a lot of people I’d studied with were now working at UCSF and I thought it would be fun to see them before going down to southern California. They took me out to dinner and showed me around and I said this place is beautiful, I can play tennis out here all year round, there’s lots of music – I love jazz – and they said ‘you know Bert, have you looked at Oakland Children’s hospital? We want to start a sickle cell program center, but the patients are all in Oakland and the patient population that would be served is in Oakland. But if you came out to the Bay Area we could partner with you to start that program. 

“So, when I walked in the door here (at Oakland) and said ‘I want to create this northern California sickle cell center with UC’ the staff that was here said ‘you know we’re not a research hospital, we are a community based hospital’. I said, ‘I’m not saying you shouldn’t be that but I’m trying to create an opportunity here’ and they said to me ‘as long as you don’t ask for any money you can go and do whatever you want’.

‘They recognized that I had this fire in me to really create something that was novel. And the warmth and community commitment from this place is something that attracted me and then allowed me to build on that.

“For example, when I became the director of the research program we had $500,000 in NIH grants and when I left we had $60 million. We just grew. Why did we grow? Because we cared about the faculty and the community. We had a lovely facility, which was actually the home of the Black Panther party. It was the Black Panthers who started screening for sickle cell on street corners here in Oakland, and they were the start of the national sickle cell act so there’s a history here and I like that history.

“Then I got a sense of the opportunities that stem cell therapies would have for a variety of things, certainly including sickle cell disease, and I thought if there’s a chance to be on the CIRM Board, as an advocate for that sickle cell community, I think I’d be a good spokesperson. So, I applied. I just thought this was an exciting opportunity.

“I thought it was a natural fit for me to add some value, I only want to be on something where I think I add value.”

Bert added value to everything he did. And everyone he met felt valued by him. He was a mentor to so many people, young physicians and nurses, students starting out on their careers. And he was a friend to those in need.

He was an extraordinary man and we are grateful that we were able to call him a colleague, and a friend, for as long as we did.

When Burt stepped down from Children’s his colleagues put together this video about his life and times. It seems appropriate to share it again and remind ourselves of the gift that he was to everyone fortunate enough to know him.

CIRM Board Approves Clinical Trials Targeting COVID-19 and Sickle Cell Disease

Coronavirus particles, illustration.

Today the governing Board of the California Institute for Regenerative Medicine (CIRM) approved new clinical trials for COVID-19 and sickle cell disease (SCD) and two earlier stage projects to develop therapies for COVID-19.

Dr. Michael Mathay, of the University of California at San Francisco, was awarded $750,000 for a clinical trial testing the use of Mesenchymal Stromal Cells for respiratory failure from Acute Respiratory Distress Syndrome (ARDS). In ARDS, patients’ lungs fill up with fluid and are unable to supply their body with adequate amounts of oxygen. It is a life-threatening condition and a major cause of acute respiratory failure. This will be a double-blind, randomized, placebo-controlled trial with an emphasis on treating patients from under-served communities.

This award will allow Dr. Matthay to expand his current Phase 2 trial to additional underserved communities through the UC Davis site.

“Dr. Matthay indicated in his public comments that 12 patients with COVID-related ARDS have already been enrolled in San Francisco and this funding will allow him to enroll more patients suffering from COVID- associated severe lung injury,” says Dr. Maria T. Millan, CIRM’s President & CEO. “CIRM, in addition to the NIH and the Department of Defense, has supported Dr. Matthay’s work in ARDS and this additional funding will allow him to enroll more COVID-19 patients into this Phase 2 blinded randomized controlled trial and expand the trial to 120 patients.”

The Board also approved two early stage research projects targeting COVID-19.

  • Dr. Stuart Lipton at Scripps Research Institute was awarded $150,000 to develop a drug that is both anti-viral and protects the brain against coronavirus-related damage.
  • Justin Ichida at the University of Southern California was also awarded $150,00 to determine if a drug called a kinase inhibitor can protect stem cells in the lungs, which are selectively infected and killed by the novel coronavirus.

“COVID-19 attacks so many parts of the body, including the lungs and the brain, that it is important for us to develop approaches that help protect and repair these vital organs,” says Dr. Millan. “These teams are extremely experienced and highly renowned, and we are hopeful the work they do will provide answers that will help patients battling the virus.”

The Board also awarded Dr. Pierre Caudrelier from ExcellThera $2 million to conduct a clinical trial to treat sickle cell disease patients

SCD is an inherited blood disorder caused by a single gene mutation that results in the production of “sickle” shaped red blood cells. It affects an estimated 100,000 people, mostly African American, in the US and can lead to multiple organ damage as well as reduced quality of life and life expectancy.  Although blood stem cell transplantation can cure SCD fewer than 20% of patients have access to this option due to issues with donor matching and availability.

Dr. Caudrelier is using umbilical cord stem cells from healthy donors, which could help solve the issue of matching and availability. In order to generate enough blood stem cells for transplantation, Dr. Caudrelier will be using a small molecule to expand these blood stem cells. These cells would then be transplanted into twelve children and young adults with SCD and the treatment would be monitored for safety and to see if it is helping the patients.

“CIRM is committed to finding a cure for sickle cell disease, the most common inherited blood disorder in the U.S. that results in unpredictable pain crisis, end organ damage, shortened life expectancy and financial hardship for our often-underserved black community” says Dr. Millan. “That’s why we have committed tens of millions of dollars to fund scientifically sound, innovative approaches to treat sickle cell disease. We are pleased to be able to support this cell therapy program in addition to the gene therapy approaches we are supporting in partnership with the National Heart, Lung and Blood Institute of the NIH.”