A look back: CIRM funded trial aims to help patients suffering from chronic viral infections

Dr. Michael Pulsipher

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 look at a way of making blood stem cell transplants safer and more readily available

Blood stem cell transplants have provided lifechanging treatments to individuals.  This statement is observed firsthand in several patients in CIRM funded trials for X-linked Chronic Granulomatous Disease (X-CGD), Sickle Cell Disease (SCD), and Severe Combined Immunodeficiency (SCID).  The personal journeys of Evangelina Padilla-Vaccaro, Evie Junior, and Brenden Whittaker speak volumes for the potential this treatment holds.  In these trials, defective blood stem cells from the patient are corrected outside the body and then returned to the patient in a transplant procedure.

Unfortunately, there is still a certain degree of risk that accompanies this procedure.  Before a blood stem cell transplant can be performed,  diseased or defective blood stem cells in the patient’s bone marrow need to be removed using chemotherapy or radiation to make room for the transplant.  This leaves the patient temporarily without an immune system and at risk for a life-threatening viral infection.  Additionally, viral infections pose a serious risk to patients with immune deficiency disorders, with viruses accounting upwards of 40% of deaths in these patients.

That’s why in October 2017, the CIRM ICOC Board awarded $4.8M to fund a clinical trial conducted by Dr. Michael Pulsipher at the Children’s Hospital of Los Angeles.  Dr. Pulsipher and his team are using virus-specific T cells (VSTs), a special type of cell that plays an important role in the immune response, to treat immunosuppressed or immune deficient patients battling life-threatening viral infections.  This trial includes patients with persistent viral infections after having received a blood stem cell transplant as well as those with immune deficiency disorders that have not yet received a blood stem cell transplant.  The VSTs used in this trial specifically treat cytomegalovirus (CMV), Epstein-Barr virus (EBV), and adenovirus infections.  They are manufactured using cells from healthy donors and are banked so as to be readily available when needed. 

One challenge of receiving a stem cell transplant can be finding a patient and donor that are a close or identical match.  This is done by looking at specific human leukocyte antigens (HLA), which are protein molecules we inherit from our parents.  To give you an idea of how challenging this can be, you only have a 25% chance of being an HLA identical match with your sibling. 

Because VSTs are temporary soldiers that are administered to fight the viral infection and then disappear, Dr. Pulsipher and his team are using partially HLA-matched VSTs to treat patients in their trial.  Previous studies have indicated that partially HLA-matched T-cells can be effective in treating patients.  The availability of partially HLA-matched VST banks that can be used “off the shelf” improves accessibility and shortens the time for patients to receive VST therapy, which will save lives.

To learn more about Dr. Pulsipher’s work, please view the video below:

Anticipating the Future of Regenerative Medicine: CIRM’s Alpha Stem Cell Clinics Network

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 take a deeper dive into CIRM’s Alpha Stem Cell Clinics Network.  The following is written by Dr. Geoff Lomax, Senior Officer of CIRM Therapeutics and Strategic Infrastructure.

The year 2014 has been described as the regenerative medicine renaissance: the European Union approved its first stem cell-based therapy and the FDA authorized ViaCyte’s CIRM funded clinical trial for diabetes. A path forward for stem cell treatments had emerged and there was a growing pipeline of products moving towards the clinic. At the time, many in the field came to recognize the need for clinical trial sites with the expertise to manage this growing pipeline. Anticipating this demand, CIRM’s provided funding for a network of medical centers capable of supporting all aspect of regenerative medicine clinical trials. In 2015, the Alpha Stem Cell Clinics Network was launched to for this purpose.

The Alpha Clinics Network is comprised of leading California medical centers with specific expertise in delivering patient-centered stem cell and gene therapy treatments. UC San Diego, City of Hope, UC Irvine and UC Los Angeles were included in the initial launch, and UC San Francisco and UC Davis entered the network in 2017. Between 2015 and 2020 these sites supported 105 regenerative medicine clinical trials. Twenty-three were CIRM-funded clinical trials and the remaining 82 were sponsored by commercial companies or the Alpha Clinic site. These trials are addressing unmet medical needs for almost every disease where regenerative medicine is showing promise including blindness, blood disorders (e.g. sickle cell disease) cancer, diabetes, HIV/AIDS, neurological diseases among others.

As of spring of 2020 the network had inked over $57 million in contracts with commercial sponsors. High demand for Alpha Clinics reflects the valuable human and technical resources they provide clinical trial sponsors. These resources include:

  • Skilled patient navigators to educate patients and their families about stem cell and gene therapy treatments and assist them through the clinical trial process.
  • Teams and facilities specialized in the manufacturing and/or processing of patients’ treatments. In some instances, multiple Alpha Clinic sites collaborate in manufacturing and delivery of a personalized treatment to the patient.
  • Nurses and clinicians with experience with regenerative medicine and research protocols to effectively deliver treatments and subsequently monitor the patients.

The multi- site collaborations are an example of how the network operates synergistically to accelerate the development of new treatments and clinical trials. For example, the UC San Francisco Alpha Clinic is collaborating with UC Berkeley and the UC Los Angeles Alpha Clinic to develop a CIRM-funded gene therapy for sickle cell disease. Each partner brings a unique expertise to the program that aims to correct a genetic mutilation in the patients’ blood stem cells to effectively cure the disease. Most recently, City of Hope has partnered with UC Irvine and UC San Diego as part of CIRM’s COVID-19 research program to study how certain immune system antibodies might be used as a treatment for respiratory disease in infected patients. In another COVID-19 study, UC Irvine and UC Davis are working with a commercial sponsor to evaluate a treatment for infected adults.

The examples above are a small sample of the variety of collaborations CIRM funding has enabled. As the Alpha Clinics track record grown, sponsors are increasingly coming to California to enable the success of their research programs. Sponsors with trials running across the country have noted a desire to expand their number of Alpha Clinic sties because they consistently perform at the highest level.

Back in 2014, it was hard to imagine over one hundred clinical trials would be served by the CIRM network in just five years. Fortunately, CIRM was able to draw on the knowledge of its internal team, external advisors and the ICOC to anticipate this need and provide California infrastructure to rise to the occasion.

Month of CIRM: Battling COVID-19

All this month we are using our blog and social media to highlight a new chapter in CIRM’s life, thanks to the people of California 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.

Dr. John Zaia, City of Hope stem cell researcher

The news that effective vaccines have been developed to help fight COVID-19 was a truly bright spot at the end of a very dark year. But it will be months, in some countries years, before we have enough vaccines to protect everyone. That’s why it’s so important to keep pushing for more effective ways to help people who get infected with the virus.

One of those ways is in a clinical study that CIRM is funding with City of Hope’s Dr. John Zaia. Dr. Zaia and his team, in partnership with the Translational Genomics Research Institute (TGen) in Flagstaff, Arizona, are using something called convalescent plasma to try and help people who have contracted the virus. Here’s the website they have created for the study.

Plasma is a part of our blood that carries proteins, called antibodies, that help defend our bodies against viral infections. When a patient recovers from COVID-19, their blood plasma contains antibodies against the virus. The hope is that those antibodies can now be used as a potential treatment for COVID-19 to help people who are newly infected. 

To carry out the study they are using clinical trial sites around California, including some of the CIRM Alpha Stem Cell Network clinics.

For the study to succeed they’ll first need people who have recovered from the virus to donate blood. That’s particularly appropriate in January because this is National Volunteer Blood Donor Month.

The team has three elements to their approach:

  • A rapid-response screening program to screen potential COVID-19 convalescent plasma donors, particularly in underserved communities.
  • A laboratory center that can analyze the anti-SARS-CoV-2 antibodies properties in COVID-19 convalescent plasma.
  • An analysis of the clinical course of the disease in COVID-19 patients to identify whether antibody properties correlate with clinical benefit of COVID-19 convalescent plasma.

There’s reason to believe this approach might work. A study published this week in the New England Journal of Medicine, found that blood plasma from people who have recovered from COVID-19 can help older adults and prevent them from getting seriously ill with the virus if they get the plasma within a few days of becoming infected.

We are used to thinking of blood donations as being used to help people after surgery or who have been in an accident. In this study the donations serve another purpose, but one that is no less important. The World Health Organization describes blood as “the most precious gift that anyone can give to another person — the gift of life. A decision to donate your blood can save a life, or even several if your blood is separated into its components — red cells, platelets and plasma.”

That plasma could help in developing more effective treatments against the virus. Because until we have enough vaccines for everyone, we are still going to need as much help as we can get in fighting COVID-19. The recent surge in cases throughout the US and Europe are a reminder that this virus is far from under control. We have already lost far too many people. So, if you have recently recovered from the virus, or know someone who has, consider donating blood to this study. It could prove to be a lifesaver.

For more information about the study and how you can be part of it, click here.

Month of CIRM: Reviewing Review

Dr. Gil Sambrano, Vice President Portfolio & Review

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 take a look at our Review team.

Many people who have to drive every day don’t really think about what’s going on under the hood of their car. As long as the engine works and gets them from A to B, they’re happy. I think the same is true about CIRM’s Review team. Many people don’t really think about all the moving parts that go into reviewing a promising new stem cell therapy.

But that’s a shame, because they are really missing out on watching a truly impressive engine at work.

Just consider the simple fact that since CIRM started about 4,000 companies, groups and individuals have applied to us for funding. Just take a moment to consider that number. Four thousand. Then consider that at no time have there been more than 5 people working in the review team. That’s right. Just 5 people. And more recently there have been substantially fewer. That’s a lot of projects and not a lot of people to review them. So how do they do it? Easy. They’re brilliant.

First, as applications come in they are scrutinized to make sure they meet specific eligibility requirements; do they involve stem cells, is the application complete, is it the right stage of research, is the budget they are proposing appropriate for the work they want to do etc. If they pass that initial appraisal, they then move on to the second round, the Grants Working Group or GWG.

The GWG consists of independent scientific experts from all over the US, all over the world in fact. However, none are from California because we want to ensure there are no possible conflicts of interest. When I say experts, I do mean experts. These are among the top in their field and are highly sought after to do reviews with the National Institutes of Health etc.

Mark Noble, PhD, the Director of the Stem Cell and Regenerative Medicine Institute at the University of Rochester, is a long-time member of the GWG. He says it’s a unique group of people:

“It’s a wonderful scientific education because you come to these meetings and someone is putting in a grant on diabetes and someone’s putting in a  grant on repairing the damage to the heart or spinal cord injury or they have a device that will allow you to transplant cells better and there are people  in the room that are able to talk knowledgeably about each of these areas and understand how this plays into medicine and how it might work in terms of actual financial development and how it might work in the corporate sphere and how it fits in to unmet medical needs . I don’t know of any comparable review panels like this that have such a broad remit and bring together such a breadth of expertise which means that every review panel you come to you are getting a scientific education on all these different areas, which is great.”

The GWG reviews the projects for scientific merit: does the proposal seem plausible, does the team proposing it have the experience and expertise to do the work etc. The reviewers put in a lot of work ahead of time, not just reviewing the application, but looking at previous studies to see if the new application has evidence to support what this team hope to do, to compare it to other efforts in the same field. There are disagreements, but also a huge amount of respect for each other.

Once the GWG makes its recommendations on which projects to fund and which ones not to, the applications move to the CIRM Board, which has the final say on all funding decisions. The Board is given detailed summaries of each project, along with the recommendations of the GWG and our own CIRM Review team. But the Board is not told the identity of any of the applicants, those are kept secret to avoid even the appearance of any conflict of interest.

The Board is not required to follow the recommendations of the GWG, though they usually do. But the Board is also able to fund projects that the GWG didn’t place in the top tier of applications. They have done this on several occasions, often when the application targeted a disease or disorder that wasn’t currently part of the agency’s portfolio.

So that’s how Review works. The team, led by Dr. Gil Sambrano, does extraordinary work with little fanfare or fuss. But without them CIRM would be a far less effective agency.

The passage of Proposition 14 means we now have a chance to resume full funding of research, which means our Review team is going to be busier than ever. They have already started making changes to the application requirements. To help let researchers know what those changes are we are holding a Zoom webinar tomorrow, Thursday, at noon PST. If you would like to watch you can find it on our YouTube channel. And if you have questions you would like to ask send them to info@cirm.ca.gov

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.

Building Bridges to a Brighter Future – Celebrating 11 Years of Workforce Development

By: Dr. Kelly Shepard, Associate Director, Discovery and Translation, CIRM

CIRM 2020 Bridges Conference via Zoom

Every July, CIRM is thrilled to announce the arrival a new generation of stem cell scientists who are ready to hit the ground running as laboratory technicians, educators, communicators, or future leaders of their chosen profession. These diverse and remarkable individuals are the latest graduates of the CIRM Bridges Program, which provides students the opportunity to take coursework at California state schools and community colleges and conduct stem cell research at top universities and industry labs. The culmination of this experience is an annual conference where students are able to network with their peers and share their research outcomes with one another.

While the Bridges program has been operating in full force for 11 years now, 2020 brought some new challenges to everyone in the form of a global pandemic. Shelter in place orders- cancellation of in person classes- travel restrictions…. these are only a few of the factors that have touched our lives in recent months. But sometimes challenges bring opportunities and a new way of doing things. Through the collective efforts of program directors, institutional officials, mentors and students, the 2020 Bridges alumni were able to complete their training requirements at their institutions and present their research at the Annual Bridges Conference, which was conducted virtually this year. While visiting students posters via Zoom, we at CIRM were thrilled to learn that many of them already had jobs waiting for them or had been accepted into PhD or MD programs, similar to alumni from previous years, which now number over 1400.

While we cannot predict all of the twists and turns that life may bring us, we can be confident that scientific research and discovery will remain essential to creating a brighter future, and that Bridges alumni will be there to help us navigate it.

Perseverance: from theory to therapy. Our story over the last year – and a half

Some of the stars of our Annual Report

It’s been a long time coming. Eighteen months to be precise. Which is a peculiarly long time for an Annual Report. The world is certainly a very different place today than when we started, and yet our core mission hasn’t changed at all, except to spring into action to make our own contribution to fighting the coronavirus.

This latest CIRM Annual Reportcovers 2019 through June 30, 2020. Why? Well, as you probably know we are running out of money and could be funding our last new awards by the end of this year. So, we wanted to produce as complete a picture of our achievements as we could – keeping in mind that we might not be around to produce a report next year.

Dr. Catriona Jamieson, UC San Diego physician and researcher

It’s a pretty jam-packed report. It covers everything from the 14 new clinical trials we have funded this year, including three specifically focused on COVID-19. It looks at the extraordinary researchers that we fund and the progress they have made, and the billions of additional dollars our funding has helped leverage for California. But at the heart of it, and at the heart of everything we do, are the patients. They’re the reason we are here. They are the reason we do what we do.

Byron Jenkins, former Naval fighter pilot who battled back from his own fight with multiple myeloma

There are stories of people like Byron Jenkins who almost died from multiple myeloma but is now back leading a full, active life with his family thanks to a CIRM-funded therapy with Poseida. There is Jordan Janz, a young man who once depended on taking 56 pills a day to keep his rare disease, cystinosis, under control but is now hoping a stem cell therapy developed by Dr. Stephanie Cherqui and her team at UC San Diego will make that something of the past.

Jordan Janz and Dr. Stephanie Cherqui

These individuals are remarkable on so many levels, not the least because they were willing to be among the first people ever to try these therapies. They are pioneers in every sense of the word.

Sneha Santosh, former CIRM Bridges student and now a researcher with Novo Nordisk

There is a lot of information in the report, charting the work we have done over the last 18 months. But it’s also a celebration of everyone who made it possible, and our way of saying thank you to the people of California who gave us this incredible honor and opportunity to do this work.

We hope you enjoy it.

A new voice and vision added to CIRM Board

UC Davis School of Medicine Dean, Dr. Allison Brashear: Photo courtesy UCD

We have a new member on the CIRM Board – Dr. Allison Brashear is the Dean of the UC Davis School of Medicine, overseeing one of the nation’s top research, academic and medical training institutions.

Dr. Brashear is an internationally known researcher in movement disorders and an expert in ATP1A3-related diseases, a spectrum of rare neurologic disorders.

Before joining UC Davis, Dr. Brashear was professor and chair of the Department of Neurology for 14 years at Wake Forest School of Medicine.

She serves on the American Board of Psychiatry and Neurology, and has served on the boards of the American Neurological Association and the American Academy of Neurology, where she was instrumental in crafting a leadership program for women, now expanded to include leadership development for minorities.

You can read more about her background in this news release. But we wanted to get a sense of what motivates and inspires Dr. Brashear. So we asked her. And she told us.

Dean Brashear being sworn in – virtually – on the CIRM Board. Top left, James Harrision CIRM General Counsel; Jonathan Thomas, CIRM Board Chair; Dr. Brashear; and Dr. Jim Kovach, Director of Industry Alliances at UC Davis, alternative Board member.

When did you get interested in science? Was this always something you knew you wanted to do?

I loved math and science in middle school and continued with science in college. I grew up hearing my parents talk about caring for patients and the impact you could have on them and their family’s lives. My father is a pulmonologist and my mother was a Ph.D. in marriage and family therapy. Together they taught me the value of patient-centered care. 

My mother was a tremendous advocate for women. When I was in middle school she took my friend and I to the state legislature and we watched the ERA (Equal Rights Amendment) debates. It’s a powerful memory but not always flattering about what people thought at the time. So, from an early age I really became a strong advocate for women, to make sure women had opportunities and that we were an inclusive culture wherever I was. 

As a woman going into a male dominated field, how did you manage to push past the skeptics and doubters to succeed? 

Early on I recognized the need to work with senior faculty who would give me an opportunity to lead and learn.  I became a chair of neurology at Wake Forest when I was 44 and was the only woman chair for 4 years. When I was appointed to the Wake Forest Baptist Medical Center Board of Directors as one of two faculty, I was the only woman. I learned early on that it was important to have sponsorship from senior leaders to succeed. I learned that, when opportunities presented themselves, to say “yes.” This is how I became the lead investigator into ATP1A3 related diseases in 1991. That project, now 11 years funded by the NIH, is one that is led by me and three other women. 

It’s still not uncommon for me to walk into a room and be the only woman. And so, making sure that there is appropriate support for women leaders is really key.

Did you have mentors to help you along the way – what was their advice to you?

I prefer the term sponsorship. Mentors advise – which is important, but more important is the role of the sponsor. A sponsor goes out of their way to advance another career. This can be a public call-out, a well-placed phone call or giving a resident what ends up being a new pathway of research.  I appreciate the many sponsors in my life, and that includes men and women.  I aspire to be a similar sponsor. This is my way to pay it forward. 

How do you sponsor others to help them overcome barriers, etc.? 

I advise women to get extra leadership training, learn about money and to make sure to have a network of advocates. I also remind them to say thank you to those who pave the way. 

I think it goes down to the message that you meet these key people in your life and they go the extra mile to help you and you, as a leader, need to take that opportunity and really just launch from it. Along the way I found I really wanted to bring people in and grow them and that was the best part of being chair of the department and one of the reasons I wanted to be a dean. When faculty join our health system I want to set them up to succeed. Celebrating others’ success with them is a great feeling. Fostering these successes is how we can be a catalyst to research and care innovations that improve health, which is at the heart of our work.  

These are interesting times to head a major university, what advice and encouragement do you have for students just starting out who face their first year “at university” at home? 

Every change brings opportunity. University at home is hard – interpersonal relationships are so important to learning and we miss that when we are on Zoom. I advise students and faculty to nurture those social connections.

When you are not working what do you do for fun?  

I hang out with my husband and our two rescue dogs. We are making plans to go explore California when the COVID-19 pandemic settles down. We had our two adult children home during the shutdown, but both are back at school on the East Coast. 

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 Expands Efforts of COVID-19 Program

Today the governing Board of the California Institute for Regenerative Medicine (CIRM) expanded efforts related to the $5 million in emergency funding for the CIRM COVID-19 program.

The new guidelines mean that inception discovery projects (DISC1), whose goal is developing new and transformational ideas, will now be eligible for CIRM COVID-19 funding.  These projects can receive up to $150,000 and must have data to confirm or reject their hypothesis within 6 months. In addition to this, quest discovery projects (DISC2), which promote the discovery of new technologies that could be translated to enable broad use, can now receive up to $250,000 in funding.

The Board approved using $1 million from the program in supplemental support for CIRM-funded COVID-19 clinical trials. Under the change an existing clinical trial can receive up to $250,000 in additional funding but must demonstrate sufficient progress and specific activities in order to be eligible.  The Board will also require that all clinical trial projects include a plan for outreach and study participation by underserved and disproportionately affected populations.

The Board also strongly encouraged those that meet the stem cell component for vaccine development for COVID-19 to apply for funding.

“We continue to receive large amounts of inquiries and applications to the COVID-19 program announcement,” said Maria T. Millan, M.D., President and CEO of CIRM. “The amendments passed by our Board today will provide additional opportunities for CIRM to support novel vaccine development, fundamental discoveries and the acceleration of clinical programs.”