CIRM board member Ysabel Duron appointed to National Cancer Advisory Board by President Biden

Ysabel Duron is an award-winning journalist, patient advocate, cancer survivor and board member of the California Institute for Regenerative Medicine (CIRM)

Her list of achievements continues to grow, as President Biden has appointed Duron to National Cancer Advisory Board (NCAB), which plays an important role in setting the course for the national cancer research program. 

The National Cancer Advisory Board will complement the Cancer Moonshot, which President Biden reignited a year ago to invest in research and development that will help advance breakthroughs to prevent, detect and treat diseases like cancer. 

“As a Latina, and a long-time patient and community advocate, it humbles me to join this roster of stellar new appointees,” Duron said. “I look forward to the challenge of amplifying the voices of racial and ethnic communities and other vulnerable populations.” 

Duron came into the cancer space after her own bout with Hodgkins Lymphoma in 1999. She covered her own cancer battle using her reporting skills to raise awareness about the disease.  

Over time, she turned a spotlight on the many disparities—lack of access, income inequality, language barriers, among other social determinants on health—that has exacerbated the disproportionate burden of cancer in Latino communities. 

In 2017, Ms. Duron founded The Latino Cancer Institute (TLCI), a nationwide network dedicated to developing and sharing best practice programs to enhance the work of Latino community service agencies, to provide collaboration with the global cancer research community, and drive policy to solve the issues and burden of Latinx/Hispanic cancer. 

In addition to her new appointment to the NCAB and role as Board member at CIRM, Duron also serves on the Institutional Review Board for the NIH/All of Us Research program. She also recently joined the newly launched American Cancer Society National Breast Cancer Roundtable

Read the official White House press release here.

Dr. Vito Imbasciani elected as Chair of California stem cell agency

Dr. Vito Imbasciani will be the new Chair of the California Institute for Regenerative Medicine (CIRM), the state agency created by voters in 2004 and funded again in 2020 to invest in stem cell and regenerative medicine research and treatments.

At January’s Board meeting, the agency’s 35-member Governing Board elected Imbasciani to the six-year term, replacing outgoing chair Jonathan Thomas, who has served in the position since 2011.

“Dr. Imbasciani’s experience across many relevant fronts will help him hit the ground running in guiding the Agency as it continues to grow its programs to bring treatments to patients with unmet medical needs,” Thomas said in welcoming Imbasciani to the role. “The agency, as well as the people of California and the world, will be well served by Imbasciani’s appointment as Chair of the CIRM Governing Board.”

Imbasciani expressed excitement in taking on the role, citing his extensive career in academia, government, military service and medicine.

“My experience has positioned me to champion the aims of CIRM, advocate for it cogently, and represent it responsibly before the public and their state and federal elected representatives,” Imbasciani said. “I look forward to the challenge of advancing the groundbreaking work of this Agency, at the same time nourishing the hopes for medical advances held by the citizens of our great State.”

Imbasciani has served as the Secretary of the California Department of Veterans Affairs (CalVet) since 2015. As Secretary, he created several new programs within the department, including forging eight independent California veteran homes into a unified system, establishing programs for veterans in state prisons, and supporting the 58 county veteran service offices.

In addition, Dr. Imbasciani has been a practicing urologic surgeon for 30 years, treating a mostly older population suffering from congenital and acquired conditions.

Dr. Imbasciani completed medical school at the University of Vermont College of Medicine, and his surgical and urologic residencies at Yale-New Haven Hospital and the West Haven VA Hospital in Connecticut. At the University of Vermont, he worked in the laboratory assisting in studies of neurodegenerative diseases.

He earned MA and PhD degrees from Cornell University, and was a Fulbright Scholar to Rome, Italy in 1973. He held academic teaching positions at the University of Florida, Cornell University and Middlebury College in Vermont.

He also served for 27 years as a surgeon in the United States Army Medical Corps, with four wartime deployments that exposed him to battlefield medicine and post-acute care.

Dr. Imbasciani also has a documented history in successful stem cell research advocacy. As an elected member of the Board of Directors of both the California Medical Association and the Los Angeles County Medical Association, he advocated for investments in basic stem cell research, and for the passage of Proposition 71, the ballot initiative that created CIRM. This included participating in activities aimed at educating the wider medical community in the long-term benefits of stem cell research.

CIRM President and CEO Dr. Maria T. Millan applauded Imbasciani’s appointment as Chair.

“Dr. Imbasciani’s experience as a state secretary, surgeon, professor, stem cell research advocate, and board member of various medical agencies and organizations makes him exceptionally well-suited to fill the role of ICOC Chair and to lead CIRM in accelerating world class science and treatments for a diverse California and the world. I look forward to working with him in his new role.”

Imbasciani will be sworn in and start on March 28, 2023.

Making transplants easier for kids, and charting a new approach to fighting solid tumors.

Every year California performs around 100 kidney transplants in children but, on average, around 50 of these patients will have their body reject the transplant. These children then have to undergo regular dialysis while waiting for a new organ. Even the successful transplants require a lifetime of immunosuppression medications. These medications can prevent rejection but they also increase the risk of infection, gastrointestinal disease, pancreatitis and cancer.

Dr. Alice Bertaina and her team at Stanford University were awarded $11,998,188 to test an approach that uses combined blood stem cell (HSC) and kidney transplantation with the goal to improve outcomes with kidney transplantation in children. This approach seeks to improve on the blood stem cell preparation through an immune-based purification process.

In this approach, the donor HSC are transplanted into the patient in order to prepare for the acceptance of the donor kidney once transplanted. Donor HSC give rise to cells and conditions that re-train the immune system to accept the kidney. This creates a “tolerance” to the transplanted kidney providing the opportunity to avoid long-term need for medications that suppress the immune system.

Pre-clinical data support the idea that this approach could enable the patient to stop taking any immunosuppression medications within 90 days of the surgery.

Dr. Maria T. Millan, President and CEO of CIRM, a former pediatric transplant surgeon and tolerance researcher states that “developing a way to ensure long-term success of organ transplantation by averting immune rejection while avoiding the side-effects of life-long immunosuppression medications would greatly benefit these children.”

The CIRM Board also awarded $7,141,843 to Dr. Ivan King and Tachyon Therapeutics, Inc to test a drug showing promise in blocking the proliferation of cancer stem cells in solid tumors such as colorectal and gastrointestinal cancer.

Patients with late-stage colorectal cancer are typically given chemotherapy to help stop or slow down the progression of the disease. However, even with this intervention survival rates are low, usually not more than two years.

Tachyon’s medication, called TACH101, is intended to target colorectal cancer (CRC) stem cells as well as the bulk tumor by blocking an enzyme called KDM4, which cancer stem cells need to grow and proliferate.

In the first phase of this trial Dr. King and his team will recruit patients with advanced or metastatic solid tumors to assess the safety of TACH101, and determine what is the safest maximum dose. In the second phase of the trial, patients with gastrointestinal tumors and colorectal cancer will be treated using the dose determined in the first phase, to determine how well the tumors respond to treatment.  

The CIRM Board also awarded $5,999,919 to Dr. Natalia Gomez-Ospina and her team at Stanford University for a late-stage preclinical program targeting Severe Mucopolysaccharidosis type 1, also known as Hurler syndrome. This is an inherited condition caused by a faulty gene. Children with Hurler syndrome lack an enzyme that the body needs to digest sugar. As a result, undigested sugar molecules build up in the body, causing progressive damage to the brain, heart, and other organs. There is no effective treatment and life expectancy for many of these children is only around ten years.

Dr. Gomez-Ospina will use the patient’s own blood stem cells that have been genetically edited to restore the missing enzyme. The goal of this preclinical program is to show the team can manufacture the needed cells, to complete safety studies and to apply to the US Food and Drug Administration for an Investigational New Drug (IND), the authorization needed to begin a clinical trial in people.

Finally the Board awarded $20,401,260 to five programs as part of its Translational program. The goal of the Translational program is to support promising stem cell-based or gene projects that accelerate completion of translational stage activities necessary for advancement to clinical study or broad end use. Those can include therapeutic candidates, diagnostic methods  or devices and novel tools that address critical bottlenecks in research.

The successful applicants are:

APPLICATIONTITLEPRINCIPAL INVESTIGATOR – INSTITUTIONAMOUNT  
TRAN4-14124Cell Villages and Clinical Trial in a Dish with Pooled iPSC-CMs for Drug DiscoveryNikesh Kotecha — Greenstone Biosciences  $1,350,000
TRAN1-14003Specific Targeting Hypoxia Metastatic Breast Tumor with Allogeneic Off-the-Shelf Anti-EGFR CAR NK Cells Expressing an ODD domain of HIF-1αJianhua Yu — Beckman Research Institute of City of Hope  $6,036,002  
TRAN1-13983CRISPR/Cas9-mediated gene editing of Hematopoietic
stem and progenitor cells for Friedreich’s ataxia
Stephanie Cherqui — University of California, San Diego  $4,846,579
TRAN1-13997Development of a Gene Therapy for the Treatment of
Pitt Hopkins Syndrome (PHS) – Translating from Animal Proof of Concept to Support Pre-IND Meeting
Allyson Berent — Mahzi Therapeutics  $4,000,000
TRAN1-13996Overcoming resistance to standard CD19-targeted CAR
T using a novel triple antigen targeted vector
William J Murphy — University of California, Davis  $4,168,679

Sweet 16 and counting for stem cell clinical trial

Dr. Judy Shizuru: Photo courtesy Jasper Therapeutics

Over the years the California Institute for Regenerative Medicine (CIRM) has invested a lot in helping children born with severe combined immunodeficiency (SCID), a fatal immune disorder. And we have seen great results with some researchers reporting a 95 percent success rate in curing these children.

Now there’s more encouraging news from a CIRM-funded clinical trial with Jasper Therapeutics. They have announced that they have tested their approach in 16 patients, with encouraging results and no serious adverse events.

Let’s back up a little. Children born with SCID have no functioning immune system, so even a simple infection can prove life threatening. Left untreated, children with SCID often die in the first few years of life. Several of the approaches CIRM has funded use the child’s own blood stem cells to help fix the problem. But at Jasper Therapeutics they are using another approach. They use a bone marrow or hematopoietic stem cell transplant (HCT).   This replaces the child’s own blood supply with one that is free of the SCID mutation, which helps restore their immune system.

However, there’s a problem. Most bone marrow transplants use chemotherapy or radiation to destroy the patient’s own unhealthy blood stem cells and make room for the new, healthy ones. It can be effective, but it is also toxic and complex and can only be performed by specialized teams in major medical centers, making access particularly difficult for poor and underserved communities.

To get around that problem Jasper Therapeutics is using an antibody called JSP191 – developed with CIRM funding – that directs the patient’s own immune cells to kill diseased blood stem cells, creating room to transplant new, healthy cells. To date the therapy has already been tested in 16 SCID patients.

In addition to treating 16 patients treated without any apparent problems,  Jasper has also been granted Fast Track Designation by the US Food and Drug Administration. This can help speed up the review of treatments that target serious unmet conditions. They’ve also been granted both Orphan and Rare Pediatric Disease designations. Orphan drug designation qualifies sponsors for incentives such as tax credits for clinical trials. Rare Pediatric Disease designation means that if the FDA does eventually approve JSP191, then Jasper can apply to receive a priority review of an application to use the product for a different disease, such as someone who is getting a bone marrow transplant for sickle cell disease or severe auto immune diseases.

In a news release, Ronald Martell, President and CEO of Jasper Therapeutics said:

“The FDA’s Fast Track designation granted for JSP191 in Severe Combined Immunodeficiency (SCID) reinforces the large unmet medical need for patients with this serious disease. Along with its previous designations of Orphan and Rare Pediatric Disease for JSP191, the FDA’s Fast Track recognizes JSP191’s potential role in improving clinical outcomes for SCID patients, many of whom are too fragile to tolerate the toxic chemotherapy doses typically used in a transplant.”

Why the future of regenerative medicine depends on students getting a living wage

The headline in the journal Nature was intended to grab attention and it definitely did that. It read: ‘The scandal of researchers paid less than a living wage’ The rest of the article built on that saying “The cost-of-living crisis is a fundamental threat for PhD scholars and early-career researchers. They need to be paid properly.”

So, just how poorly are these researchers – PhD candidates and postdoctoral students – paid? Well, according to one survey salaries for PhD students in the biological sciences are below the cost of living at almost every institution in the United States. And imagine trying to live on a sub-standard income in a state as expensive as California?

The outrage is fueled by a survey of more than 3,200 students, three quarters of whom are PhD candidates. Around 85% of the students said inflation is making things even worse and almost half said it was making it hard to complete their courses.

The situation isn’t any better in other countries. In the UK, PhD students often get the equivalent of just $20,400, and that’s after getting a recent big boost of more than $2,000 per year. It’s no wonder English students organized protests calling for better funding. Students in Ireland also staged protests, saying the money they get simply isn’t enough.

The Nature Editorial said this isn’t just a matter of inconvenience for the students, it’s a threat to the future of science: “If students don’t have the resources to support themselves, they can’t put their full efforts into their training and development. And if their stipends aren’t keeping pace with rising rents and the cost of groceries and fuel, any gaps will only grow with time — with devastating results for the ability of research to attract the best talent.”

That’s one of the reasons the California Institute for Regenerative Medicine (CIRM) tries to make sure all the students in its internship programs have enough money to live on. We know it’s hard to focus on work if you are hungry or worried that you don’t have enough money to pay your bills.

When our Board approved a new internship program, called COMPASS (Creating Opportunities through Mentorship and Partnership Across Stem Cell Science) they made sure that enough money was included to cover students living expenses, course fees and even travel to scientific conferences. The Board allocated more than $58,000 a year to support each students, many of whom will come from poor or low-income communities and might not otherwise be able to afford to stay in school.

For our Bridges students, many of whom are also from low-income communities or are the first in their family to attend college, the Board allocated each one around $72,000 worth of support per year.

We know that the future of regenerative medicine in California depends on having a skilled, well-trained, diverse workforce. That doesn’t just mean PhDs doing the research, it also means the technicians and support staff that can help with manufacturing etc. Without a living wage that makes this possible many students will drop out and the field as a whole will struggle. Those most affected will be students from poor backgrounds or from disadvantaged and historically marginalized communities.

We need to support these students in every way we can. If we don’t provide enough financial support for these students to succeed, the field as a whole will be a lot poorer.

Patient Advocacy is its own reward

It’s always nice to be told you are doing a good job. It’s even nicer when it’s unexpected. That’s certainly the case when we, the Communications Team at the California Institute for Regenerative Medicine, found out we’d been named as a finalist for the Patient Advocacy Award (non-profit category) as part of the Phacilitate Advanced Therapies Awards.

To be honest, we didn’t even know we’d been nominated. But who cares. We are now in the final. And we are in good company. Our friends at Americans for Cures, were also nominated. They are advocates for stem cell research in California and were hugely instrumental in getting Proposition 14 passed in 2020, that’s the voter initiative that refunded CIRM with $5.5 billion.

The other finalists are the Alliance for Cancer Gene Therapy and the Rare Advocacy Movement.

While we may focus on different areas we all share a common goal, a desire to ensure that the voice of the patient is front and center in all that we do. At CIRM we have patient advocates on our Board and on the panel of experts who review applications for our funding. We have patient advocates helping guide the clinical trials we fund. And now, as we expand our efforts to reach out in every community in California, we have patients and patient advocates guiding that work as well.

We do this work because it’s important and because, without the support of the patient advocacy community, we wouldn’t be here.

It’s an old cliché that when you are in this position you say, “it’s an honor just to be nominated.” But in this case, it’s true.

Dr. Deborah Deas and Ysabel Duron recognized for their contributions to advancing public health

Dr. Deborah Deas

The California Institute for Regenerative Medicine (CIRM) has two reasons to celebrate today.

Earlier this month, Dr. Deborah Deas was elected as a member of the National Academy of Medicine, or NAM. Membership in the academy is one of the highest national honors in health and medicine.

Dr. Deas is the vice chancellor of health sciences and the Mark and Pam Rubin Dean of the UCR School of Medicine, as well as a member of CIRM’s governing Board.

Amongst many other honors, Dr. Deas is recognized for being a national contributor to addressing health disparities through diversifying the physician workforce, especially around the shortage of Black males in medicine.

“I was ecstatic to learn that I was elected. It will allow me to have a greater voice at the national level in science as well as in diversity, equity, and inclusion. I’m also so pleased about what we are doing at CIRM, and this is such a great opportunity to not only represent myself but also the UC system as well as CIRM.”

Ysabel Duron (pictured on left) at While House Cancer Moonshot event.

Simultaneously, another Board member, founder and President of the Latino Cancer Institute Ysabel Duron was asked to join the American Cancer Society (ACS) National Breast Cancer Roundtable (NBCRT).

Last week, Ms. Duron attended the event at the white house with First Lady Dr. Jill Biden, where she announced the launch of NBCRT.

The ACS NBCRT is a national coalition working to accelerate progress across the breast cancer continuum through strategic partnerships to eliminate disparities and reduce mortality. The ACS NBCRT works to ensure all women have access to quality screening and treatment, including Black women and women in other historically excluded communities, to address the social and emotional needs of patients and their families.

“I feel both honored to join the ACS NBCRT and the weight of this responsibility and obligation to those who suffer and die from this horrific disease every day. I am also committed, during the critical next steps in determining initiatives to propose, to spotlight the gaps and needs in education, quality care and access to the most advanced diagnostics and treatment for Latina and other underserved populations.”

CIRM Board Approves Funding for New Clinical Trial Targeting Brain Tumors

The governing Board of the California Institute for Regenerative Medicine (CIRM) has awarded almost $12 million to carry out a clinical trial targeting brain tumors.

This brings the total number of CIRM funded clinical trials to 83.  

$11,999,984 was awarded to Dr. Jana Portnow at the Beckman Research Institute of City of Hope. They are using Neural stem cells (NSCs) as a form of delivery vehicle to carry a cancer-killing virus that specifically targets brain tumor cells.

Glioblastoma is the most common malignant primary brain tumor in adults and each year about 12,000 Americans are diagnosed. The 5-year survival rate is only about 10%.

The current standard of care involves surgically removing the tumor followed by radiation, chemotherapy, and alternating electric field therapy. Despite these treatments, survival remains low.

The award to Dr. Portnow will fund a clinical trial to assess the safety and effectiveness of this stem cell-based treatment for Glioblastoma.

The Board also awarded $3,111,467 to Dr. Boris Minev of Calidi Biotherapeutics. This award is in the form of a CLIN1 grant, with the goal of completing the testing needed to apply to the Food and Drug Administration (FDA) for permission to start a clinical trial in people.

This project uses donor fat-derived mesenchymal stem cells that have been loaded with oncolytic virus to target metastatic melanoma, triple negative breast cancer, and advanced head & neck squamous cell carcinoma.

“There are few options for patients with advanced solid tumor cancers such as glioblastoma, melanoma, breast cancer, and head & neck cancer,” says Maria T. Millan, M.D., President and CEO of CIRM. “Surgical resection, chemotherapy and radiation are largely  ineffective in advanced cases and survival typically is measured in months. These new awards will support novel approaches to address the unmet medical needs of patients with these devastating cancers.”

The CIRM Board also voted to approve awarding $71,949,539 to expand the CIRM Alpha Clinics Network. The current network consists of six sites and the Board approved continued funding for those and added an additional three sites. The funding is to last five years.

The goal of the Alpha Clinics award is to expand existing capacities for delivering stem cell, gene therapies and other advanced treatment to patients. They also serve as a competency hub for regenerative medicine training, clinical research, and the delivery of approved treatments.

Each applicant was required to submit a plan for Diversity, Equity and Inclusion to support and facilitate outreach and study participation by underserved and disproportionately affected populations in the clinical trials they serve.

The successful applicants are:

ApplicationProgram TitleInstitution/Principal InvestigatorAmount awarded
INFR4-13579The Stanford Alpha Stem Cell ClinicStanford University – Matthew Porteus  $7,997,246  
INFR4-13581UCSF Alpha Stem Cell ClinicU.C. San Francisco – Mark Walters  $7,994,347  
INFR4-13586A comprehensive stem cell and gene therapy clinic to
advance new therapies for a diverse patient
population in California  
Cedars-Sinai Medical Center – Michael Lewis  $7,957,966    
INFR4-13587The City of Hope Alpha Clinic: A roadmap for equitable and inclusive access to regenerative medicine therapies for all Californians  City of Hope – Leo Wang  $8,000,000
INFR4-13596Alpha Stem Cell Clinic for Northern and Central California  U.C. Davis – Mehrdad Abedi  $7,999,997  
INFR4-13685Expansion of the Alpha Stem Cell and Gene Therapy Clinic at UCLA  U.C. Los Angeles – Noah Federman  $8,000,000
INFR4-13878Alpha Clinic Network Expansion for Cell and Gene Therapies  University of Southern California – Thomas Buchanan  $7,999,983  
INFR4-13952A hub and spoke community model to equitably deliver regenerative medicine therapies to diverse populations across four California counties  U.C. Irvine – Daniela Bota  $8,000,000
INFR4-13597UC San Diego Health CIRM Alpha Stem Cell Clinic  U.C. San Diego – Catriona Jamieson  $8,000,000

The Board also unanimously, and enthusiastically, approved the election of Maria Gonzalez Bonneville to be the next Vice Chair of the Board. Ms. Bonneville, the current Vice President of Public Outreach and Board Governance at CIRM, was nominated by all four constitutional officers: the Governor, the Lieutenant Governor, the Treasurer and the Controller.

In supporting the nomination, Board member Ysabel Duron said: “I don’t think we could do better than taking on Maria Gonzalez Bonneville as the Vice Chair. She is well educated as far as CIRM goes. She has a great track record; she is empathetic and caring and will be a good steward for the taxpayers to ensure the work we do serves them well.”

In her letter to the Board applying for the position, Ms. Bonneville said: “CIRM is a unique agency with a large board and a long history. With my institutional knowledge and my understanding of CIRM’s internal workings and processes, I can serve as a resource for the new Chair. I have worked hand-in-hand with both the Chair and Vice Chair in setting agendas, prioritizing work, driving policy, and advising accordingly.  I have worked hard to build trusted relationships with all of you so that I could learn and understand what areas were of the most interest and where I could help shed light on those particular programs or initiatives. I have also worked closely with Maria Millan for the last decade, and greatly enjoy our working relationship. In short, I believe I provide a level of continuity and expertise that benefits the board and helps in times of transition.”

In accepting the position Ms. Bonneville said: “I am truly honored to be elected as the Vice Chair for the CIRM Board. I have been a part of CIRM for 11 years and am deeply committed to the mission and this new role gives me an opportunity to help support and advance that work at an exciting time in the Agency’s life. There are many challenges ahead of us but knowing the Board and the CIRM team I feel confident we will be able to meet them, and I look forward to helping us reach our goals.”

Ms. Bonneville will officially take office in January 2023.

The vote for the new Chair of CIRM will take place at the Board meeting on December 15th.

Judy Chou, Ph.D., Appointed to Governing Board of California’s Stem Cell & Gene Therapy Agency

Judy Chou, Ph.D.

Judy Chou, Ph.D. has been appointed to the Independent Citizens’ Oversight Committee (ICOC), the governing Board of the California Institute for Regenerative Medicine (CIRM).

Dr. Chou is the President, CEO and a member of the Board of Directors of AltruBio, Inc. a clinical stage biotech company that is focused on developing novel antibody therapeutics for the treatment of immune inflammatory diseases.

“I am excited to join the ICOC leveraging my experience both as a scientist in the the biopharmaceutical industry and as a corporate executive to support the research and funding of life changing medicines for patients in need,” said Dr. Chou.

Dr. Chou has more than 20 years experience in drug development and biomanufacturing. Before joining AltruBio she headed the global Biotech organization at Bayer Pharmaceuticals. At Bayer she oversaw the development, manufacturing and distribution of the company’s more than $3 billion product portfolio. She also oversaw more than 2,000 employees and led the drug development and launch activities for the biologics pipeline. In addition, she also served as the site head for Bayer’s facility in Berkeley, California, the company’s largest manufacturing site in the U.S.

“We are honored and delighted to have Dr. Chou take a seat on the Board,” says Jonathan Thomas, Ph.D., J.D., Chair of the CIRM Board. “She has a remarkable career in academia, industry and in promoting diversity, equity and inclusion and will be an invaluable addition to the ICOC. We are very much looking forward to working with her.”

Dr. Chou also has had leadership roles at Pfizer, Medivation Inc., Genentech and Wyeth Biopharma. She has won several awards and in 2018 was the recipient of the Most Influential Women in Business award by the San Francisco Business Times. She is currently an advisor at the UC Berkeley Engineering School and is working to promote diversity and inclusion through her advisory board position at Silicon Valley Women in Engineering.

Dr. Chou obtained her Ph.D., at Yale, her post-doctoral training at the Max-Planck Institute in Germany and was a research faculty member at Harvard University Medical School focusing on cell biology and neuroscience.

Dr. Chou was appointed to the CIRM Board by State Treasurer Fiona Ma, as the Executive Officer of a Commercial Life Science entity. She replaces Dave Martin.

State Stem Cell & Gene Therapy Agency Sets up Support Program to Help Patients Participate in Clinical Trials

For many patients battling deadly diseases, getting access to a clinical trial can be life-saving, but it can also be very challenging. Today the governing Board of the California Institute for Regenerative Medicine (CIRM) approved a concept plan to make it financially and logistically easier for patients to take part in CIRM-funded clinical trials.

The plan will create a Patient Support Program (PSP) to provide support to California patients being evaluated or enrolled in CIRM-supported clinical trials, with a particular emphasis on helping underserved populations.

“Helping scientists develop stem cell and gene therapies is just part of what we do at CIRM. If those clinical trials and resulting therapies are not accessible to the people of California, who are making all this possible, then we have not fulfilled our mission.” says Maria T. Millan, M.D., President and CEO of CIRM.

The Patient Support Plan will offer a range of services including:

  • Clinical trial navigation, directing patients to appropriate CIRM-supported clinical trials.
  • Logistical support for patients being evaluated or enrolled in clinical trials.
  • Financial support for under resourced and underserved populations in CIRM-supported clinical trials, including the CIRM Patient Assistance Fund (PAF).  This support includes transportation/travel expenses, such as gasoline, tolls, parking, airfare, taxi, train, lodging, and meals during travel.
  • Providing nurse navigator support for the psychosocial, emotional, and practical needs of patients and their families.

The funds for the PSP are set aside under Proposition 14, the voter-approved initiative that re-funded CIRM in 2020. Under Prop 14 CIRM money that CIRM grantees earn from licensing, inventions or technologies is to be spent “offsetting the costs of providing treatments and cures arising from institute-funded research to California patients who have insufficient means to purchase such treatment or cure, including the reimbursement of patient-qualified costs for research participants.”

Currently, the CIRM Licensing Revenues and Royalties Fund has a balance of $15.6 million derived from royalty payments.

“The patient support program and financial resources will not only help patients in need, it will also help increase the likelihood that these clinical trials will succeed,” says Sean Turbeville, Ph.D., Vice President of Medical Affairs and Policy at CIRM. “We know cell and gene therapies can be particularly challenging for patients and their families. The financial challenges, the long-distance traveling, extended evaluation, and family commitments can make it difficult to enroll and retain patients. The aim of the PSP is to change that.”

The overall objective of this funding opportunity is to establish a statewide program that, over five years, is expected to support hundreds of patients in need as they participate in the growing number of CIRM-supported clinical trials. The program is expected to cost between $300,000 to $500,000 a year. That money will come from the Medical Affairs budget and not out of the patient assistance fund.

The first phase of the program will identify an organization, through a competitive process, that has the expertise to provide patient support services including:

  • Maintaining a call and support center.
  • Assessing patient eligibility for financial assistance.
  • Reporting to CIRM on patients needs and center performance

 You can find more information about the Patient Support Program on our website here and here.