Meshing a passion for medicine with stem cell research

Kevin Brown is an adventurer at heart. He was a pre-med student when he found a unique way to combine his passion for medicine in neuroscience with his natural inclination to explore and discover through scientific research.

A 2022 graduate of the California Institute for Regenerative Medicine’s (CIRM) Bridges to Stem Cell Research and Therapy Program, Kevin says the experience was a great opportunity to mesh his passion for medicine with his instinct for research in the stem cell/regenerative medicine field.

“I recognized the vital importance of having basic scientific training and thinking skills in relationship to being a good physician,” Kevin said, noting that these factors inspired him to apply to the Bridges program.

Started in 2009, the CIRM Bridges program provides paid regenerative medicine and stem cell research internships to students at universities and colleges that don’t have major stem cell research programs. Each Bridges internship includes thorough hands-on training and education in regenerative medicine and stem cell research, and direct patient engagement and outreach activities that engage California’s diverse communities. 

A Growing Field with Many Opportunities

Kevin recognizes that regenerative medicine is a growing field that offers great opportunity for exploration and discovery. It also is likely to have a strong impact on patient care in the field of neuroscience and many other areas of human health. Participating in the program has helped Kevin find a career path that excites both his passions for exploration and for patient care.

During his time in the Bridges program, Kevin attended California State University, San Marcos and he completed his program training at the Dorris Neuroscience Center at The Scripps Research Institute in San Diego.

“Medicine was all that was on my mind but as I got deeper into my research I fell more and more in love with the process of science and began seeing myself as a leader in this field in the future,” Kevin said. “I am now considering taking the MD-PhD path to satisfy both my yearning to leave a positive impact on the world through healthcare and continue venturing through the unknowns to hopefully create meaningful breakthroughs in science in the future.”

He credits his mentors—graduate student Anna Verduzco and principal investigator Dr. Hollis Cline—for helping him discover his future career path. The program and his mentors, he said, have made him a better student, a better scientist, and a better person overall.

“They helped mold the way that I approach scientific and life problems—curiosity at the forefront followed by openness to try something new.”

Overcoming Challenges

Kevin said one challenge of the internship was learning how to think, write, and communicate scientifically.

“The conventional way of learning and thinking in school is vastly different than the scientific way of thought,” Kevin said. “However, my lab partners were extremely helpful in helping me cultivate these skills by having me consistently talking through my experiments with them, do periodic write-ups on my progress, and give lab meeting presentations.”

Kevin clearly was an apt student of these lessons because at the conclusion of his internship, he offered a poster presentation at the Annual Biomedical Research Conference for Minoritized Scientists and won the 2022 Outstanding Presentation Award.

Paying It Forward

Today, Kevin is a full-time student at CSU San Marcos completing his Bachelor of Science in Biology with a concentration in Physiology. He is still a part-time research intern at The Scripps Research Institute in the Cline lab working toward understanding how stem cell derived brain cells can be used to study the intricacies of Alzheimer’s Disease in a genetic and cell-signaling context.

And he’s paying it forward for other students. “I am building a mentoring network to provide CIRM’s SPARK high school program interns with the necessary help and tools to transition into their college career feeling empowered and confident in their ability to succeed.”

Image courtesy The Cline Lab

He offers important advice regarding the field of stem cell and regenerative medicine research, and strong words for future explorers everywhere: be courageous, adaptable, and resilient.

“A lot of the work being done, especially within the context of regenerative medicine and neuroscience, has never been done before,” he said, noting that young pioneers in this space should be creative in their approach and not easily dissuaded by failure.

Kevin urges more people to become pioneers.

He adds, “Stem cell research is vital to the development of understanding how we can address the vast amount of diseases and conditions that impact humans.”

About the Bridges Program

The Bridges program is proud to claim 1,735 Bridges alumni, and more Bridges trainees are completing their internships in 2023. CIRM has 15 active Bridges programs throughout California, each with its own eligibility criteria and application process.

If you are interested in applying, please visit this web page for more details about each program. If you have questions about the Bridges program, please email the CIRM Bridges director, Dr. Kelly Shepard at education@cirm.ca.gov. 

Why diversity in clinical trials is essential

The California Institute for Regenerative Medicine (CIRM) is proud to join fellow advocates of clinical trial diversity in applauding a new law that will allow the U.S. Food & Drug Administration (FDA) to require diverse representation in clinical trials. 

A clinical trial, as defined by the FDA, tests potential treatments in human volunteers to see whether they should be approved for wider use in the general population. CIRM has invested in 88 clinical trials to date.

In December 2022, Congress approved a bill that requires diversity action plans for clinical trials used by the FDA. This new law builds on draft guidance issued by the FDA in April 2022 and will move the draft forward to finalization and enforcement.  

The law follows a 2022 report issued by the National Academies of Sciences, Engineering and Medicine that starkly notes:  

“While progress has been made with representation of white women in clinical trials and clinical research, there has been little progress in the last three decades to increase participation of racial and ethnic minority population groups. This underrepresentation is compounding health disparities, with serious consequences for underrepresented groups and for the nation.”  

New Requirements for Clinical Trials

Under the law, clinical trial sponsors will be required to submit a diversity action plan to the FDA along with other important trial documents. The plans, according to the law, should contain:  

  • The sponsor’s goals for clinical study enrollment, disaggregated by age group, sex, and racial and ethnic characteristics.  
  • The rationale for these enrollment goals, including information about the disease or condition and its prevalence or incidence among various demographics.  
  • An explanation of how the sponsor intends to meet the goals, including demographic-specific outreach and enrollment strategies, inclusion and exclusion practices, and diversity training for study personnel. 

A Major Step Forward 

Requiring a clinical trial to expand the representation of diverse people is a major step forward to reverse systemic and structural social inequities in the health care system.  

In a study published in Nature Cell Biology, the authors, which included Dr. Maria Millan, CIRM’s President & CEO, summarized:  

“To address health disparities and facilitate increasingly personalized treatments, we need to develop new models for basic and disease research that reflect diverse ancestral backgrounds and sex and ensure that diverse populations are included among donors and research participants.” 

For example, low participation of Black Americans in clinical trials is well documented including by the JCO Oncology Practice.  The JCO reports that Black Americans constitute at least 13% of the general population in the United States, account for 22% of annual cancer cases, and succumb to prostate, stomach, uterine cancers, and multiple myeloma, at rates twice as high as white people. And yet, Black Americans reflect only 7% of those enrolled in cancer clinical trials.    

Addressing Diversity in Clinical Trials at CIRM 

CIRM requires plans for inclusion of diverse or underserved demographic groups in the clinical trials we fund.  

Proposals for funding (see samples here) must demonstrate an understanding of health disparities associated with the target indication of the study, and plans to:   

  • Include an inclusive group of participations by race, ethnicity, sex, gender, and age.  
  • Address any barriers to trial participation faced by underserved demographic groups.  
  • Guide, as needed, the cultural competency of study researchers.   

“We have incorporated the principles of promoting diversity, equity and inclusion in our research funding programs, education programs and future programs,” Dr. Millan says. “We believe this is essential to ensure that the therapies our support helps advance will reach all patients in need and in particular communities that are disproportionately affected and/or under-served.” 

To learn more about CIRM’s investments in clinical trials, visit this page on our website. To learn more about participating in a clinical trial, click here

Join the movement to fight rare diseases

Tomorrow, February 28th, is Rare Disease Day. It’s a day to remind ourselves of the millions of people, and their families, struggling with these diseases. These conditions are also called orphan diseases because, in many cases, drug companies were not interested in adopting them to develop treatments.

Here at the California Institute for Regenerative Medicine (CIRM), we understand the importance of funding research that impacts not just the most common diseases. In fact, 50% of all the projects we fund target a rare disease or condition such as: Retinitis pigmentosa, Sickle cell disease, Huntington’s disease, and Duchenne Muscular Dystrophy.

Over the years, CIRM has invested millions of dollars in helping children born with severe combined immunodeficiency (SCID), including $12 million to test a newly designed therapy in a clinical trial at UC San Francisco.

Children born with SCID have no functioning immune system so even a simple infection can prove life-threatening or fatal. We recently shared an update from one of the young patients in the trial.

Additionally, last December, the CIRM governing Board awarded $4,048,253 to Dr. Joseph Anderson and his team at UC Davis to develop a blood stem cell gene therapy for the treatment of Tay-Sachs disease.

Tay-Sachs disease is a rare genetic disorder where a deficiency in the Hex A gene results in excessive accumulation of certain fats in the brain and nerve cells and causes progressive dysfunction.  

There are several forms of Tay-Sachs disease, including an infant, juvenile, and adult forms. Over a hundred mutations in the disease-causing Hex A gene have been identified that result in enzyme disfunction. There are currently no effective therapies or cures for Tay-Sachs. 

The irony of rare diseases is that a lot of people have them. The total number of Americans living with a rare disease is estimated at between 25-30 million. Two-thirds of these patients are children.

Right now, individual disease programs tend to try individual approaches to developing a treatment, which is time consuming and expensive. That’s why this past summer, CIRM signed a Memorandum of Understanding (MOU) with the Foundation for the National Institutes of Health (FNIH) to join the Bespoke Gene Therapy Consortium (BGTC).

BGTC is a public-private partnership, managed by FNIH, that brings together the National Institutes of Health (NIH), the U.S. Food and Drug Administration (FDA), and multiple public and private sector organizations to streamline the development and delivery of gene therapies for rare diseases.

“At CIRM we have funded several projects using gene therapy to help treat, and even cure, people with rare diseases such as severe combined immunodeficiency,” says Dr. Maria T. Millan, the President and CEO of CIRM. “But even an agency with our resources can only do so much. This agreement with the Bespoke Gene Therapy Consortium will enable us to be part of a bigger partnership, one that can advance the field, overcome obstacles and lead to breakthroughs for many rare diseases.”

CIRM is proud to fund and spread awareness of rare diseases and invites you to watch this video about how they affect families around the world.

Investing in stem cell and gene therapy treatments for HIV

A recent article in Nature shared the news about a 53-year-old man in Germany who was declared free of HIV after receiving virus-resistant cells. 

The man—referred to as the “Düsseldorf patient”—was diagnosed with acute myeloid leukemia and underwent a stem cell transplant in 2013 that replaced his bone marrow cells with HIV-resistant stem cells from a donor.  

In the five years following the procedure, virologist Björn-Erik Jensen and his team at Düsseldorf University Hospital in Germany continued to monitor the patient. They continued to find immune cells that reacted to HIV in his body, which suggested that his body was not clear of the virus. 

In 2018, the patient stopped taking antiretroviral therapy (ART), a treatment for HIV that reduces a person’s viral load to an undetectable level. His body has remained HIV-free since then, indicating that the stem cell transplant worked.  

Similar stem cell treatments have been used to treat others living with HIV, including a patient in 2007 and another patient in 2019

The article in Nature states that the procedure is unlikely to be used widely in its current form due to the associated risks, including the chance that an individual will reject a donor’s marrow.  

Scientists continue to test stem cells as a treatment for HIV, including methods in which cells are taken from a person’s own body and genetically modified, eliminating the need for donor cells. 

CIRM’s Commitment to Investing in Treatments for HIV

The news of the Düsseldorf patient shows the importance of continued stem cell and gene therapy research to find treatments for HIV.  

At the California Institute for Regenerative Medicine (CIRM), we have invested more than $85 million in the search for stem cell and gene therapy treatments for HIV/AIDS, ranging from basic Discovery research to clinical trials.  

Recent CIRM investments include a study at UC Davis health, in which researchers take a patient’s own white blood cells, called T-cells, and modify them so that they can identify and target HIV cells to control the virus without medication. 

CIRM also funded a clinical trial at UCSF to develop a functional cure for HIV/AIDS. In the trial, the team takes a patient’s blood and extracts T cells, a type of immune cell. The T cells are then genetically modified to express two different chimeric antigen receptors (CAR), which enable the newly-created duoCAR-T cells to recognize and destroy HIV infected cells. The modified T cells are then reintroduced back into the patient. 

The goal of this one-time therapy is to act as a long-term control of HIV with patients no longer needing to take ART, in effect a form of HIV cure.  This approach would also address the needs of those who are not able to respond to current approaches, which is estimated to be 50% of those affected by HIV globally. 

Last year, researchers in the UCSF trial shared that they had dosed the first patient in the trial testing their anti-HIV duoCAR-T cell therapy. You can read the announcement here.  

There are approximately 38 million people worldwide living with HIV/AIDS. And each year there are an estimated 1.5 million new cases. The vast majority of those living with HIV do not have access to the life-saving antiretroviral medications that can keep the virus under control. People who do have access to the medications face long-term complications from them including heart disease, bone, liver and kidney problems, and changes in metabolism. 

To learn more about CIRM’s commitment and investments in finding treatments for HIV, visit our website

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.

California agency invests $4 million in stem cell treatment for Parkinson’s Disease

The California Institute for Regenerative Medicine (CIRM) is investing $4 million in a late-stage preclinical project by Ryne Bio aiming to improve treatment for Idiopathic Parkinson’s disease (PD).

PD is characterized by a loss of dopamine producing neurons that result in motor symptoms, such as dyskinesias (involuntary, erratic, writhing movements of the face, arms, legs or trunk) and non-motor effects such as dementia, depression and sleep disorders.

PD is the second-most common neurodegenerative disease after Alzheimer’s disease affecting approximately 1 million people in the U.S. In California, it is estimated that 116,900 people live with PD, representing the highest number of people with the disease in the country.

At its early stages, PD can be treated with medication such as Levodopa to treat symptoms but these become less effective as the disease progresses.

The proposed stem cell therapy in this project offers the potential to restore dopamine neurons, which play a role in many important body functions, including movement and memory.

Investigators at Ryne Bio are aiming to deliver dopamine producing cells to replace the lost neurons to the brain of Parkinson’s disease patients to restore/improve motor function.

The current grant is being funded to conduct Investigational New Drug (IND) enabling, nonclinical safety studies per the US Food and Drug Administration (FDA) Guidance. The IND is the authorization needed to begin a clinical trial in Parkinson’s patients.

CIRM has a vested interest in seeing this therapy succeed. To date, CIRM has invested more than $59 million in helping research for Parkinson’s disease progress from a basic or Discovery level through clinical trials.

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.

Investing in a stem cell treatment for Hurler syndrome

The California Institute for Regenerative Medicine (CIRM) awarded $5,444,353 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 are no signs or symptoms of the condition at birth, although some have a soft out-pouching around the belly-button or lower abdomen. Those with severe MPS I generally begin to show other signs and symptoms of the disorder within the first year of life. 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.

“The funding will pave the way for trials in people to realize a more effective therapy for this devastating disease,” Gomez-Ospina said. “We will also generate safety and toxicity data that could facilitate the application of our genome editing platform to other genetic disorders for which a significant unmet need still exists.”

Advancing cutting-edge treatment to improve kidney transplantation in children

Stanford physician-scientist Alice Bertaina, MD, PhD, associate professor of pediatrics has received about $18 million from the California Institute for Regenerative Medicine (CIRM) for a clinical trial to allow kidney transplantation without the need for long-term immunosuppression.

Dr. 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 which have significant long-term risks.

Investing in CAR T-cell therapy to treat cancer

Photo credit: UC Regents 

The California Institute for Regenerative Medicine (CIRM) is investing $4 million to support Dr. William Murphy and UC Davis researchers to develop and test a chimeric antigen receptor (CAR) T-cell therapy to treat various B-cell malignancies, ranging from lymphomas to leukemias. 

In this Q&A—courtesy of UC Davis Health—Dr. Murphy discusses the importance of T-cell therapy and its implications for developing cancer treatments. His work is a collaboration between CIRM, the nonprofit organization Caring Cross, and UC Davis Health. 


What are B-cell malignancies? 

B-cells are a type of white blood cells that make antibodies. They are key to the body’s immune system. When healthy B-cells change into fast-growing cancer cells that don’t die, they cause B-cell malignancies. 

This can affect people at different ages. They may show up in children as B-cell acute lymphoblastic leukemia (B-ALL), an aggressive blood and bone marrow cancer. In adults, they make up about 85% of non-Hodgkin lymphoma (NHL), a cancer that starts in B lymphocytes. In the elderly, B-cell malignancies may come as multiple myeloma, a cancer of the plasma cells. 

There are different lines of treatments for B-cell lymphoma and leukemia, including immunotherapy using chimeric antigen receptor (CAR) T cells. These cells have revolutionized cancer treatment since they have been shown to work, and cure, when nothing else can. 

What is chimeric antigen receptor (CAR) T-cell therapy? 

Chimeric antigen receptor (CAR) T-cell therapy uses the body’s own defenses to fight disease. It is a new and exciting form of immunotherapy that works by modifying the receptors of immune cells (T cells) involving antibodies to target specific cancers, such as leukemias and lymphomas. 

CAR T cells are being used to treat some blood cancers with long-term success. The U.S. Food and Drug Administration (FDA) first approved CAR T-cell therapy in 2017. Their use is growing rapidly and being applied to other tumor types. Yet, this therapy is extremely expensive, even with insurance. It’s also a very intensive procedure and it takes time to generate the CAR T cells from the patient. 

While it could be considered a game changer, one of the issues with this therapy is the case relapse rate. The big holy grail in cancer therapy is how to prevent tumors from evading or escaping the immune attack. Around 60% of patients who get CAR therapy see their cancer return. If we can get the relapse rate down to negligible, that would be a tremendous advance. 

How do you intend to use CAR products to reduce cancer relapse? 

In CAR therapy, we take the immune T cells from a patient and use gene therapy to give a new receptor to signal and direct the T cell. The receptor usually has an antibody that recognizes a particular tumor antigen. Current FDA-approved CAR T therapies only target one tumor antigen. 

CARs have had tremendous success. However, there is significant patient relapse because the tumor adapts and may lose that one antigen that we are targeting, allowing it to escape the treatment. Our strategy is to target multiple antigens to reduce the potential for relapse since the tumor cannot adapt that quickly. 

We are also proposing a novel vector that will carry a CAR product, known as DuoCAR, that targets three antigens at the same time. As long as the tumor has one of the three antigens, then there’s little chance for the tumor to escape all three antibodies. This is similar to when you think about HIV treatment with the triple-drug therapy, where one alone is not sufficient. 

The hope is that the 60 to 70% of the population who would have relapsed if they had the original CAR T cell treatment, would have a home run with our kind of treatment or product. 

So, is this treatment for cancer patients who have relapsed? 

We see this product as a new frontline therapy and not just for patients who relapse. What the patient has to go through in order for CAR T therapy to work is very strenuous. So, yes, if there are relapsed patients, they can be given DuoCAR, but we’re also hoping this will become the new standard of care, replacing the other CARs in the future for everyone. 


To read the full Q&A, click here