The goal of the meeting is to provide a place for people in the cell and gene therapy field to learn from the latest scientific research, stay up to date on new technologies and make career-advancing connections with peers. CIRM was a gold sponsor of the event.
In addition to setting up a booth in the exhibit hall, CIRM participated in two sessions including a workshop that highlights the work being done in cell and gene therapy to advance neurological disorders. Speakers highlighted the impact of CIRM funding on research and the use of technologies such as digital biomarkers, optogenetics, and imaging to advance understanding of such disorders.
The BGTC is a public-private partnership, managed by Foundation for the National Institutes of Health (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 like CIRM to streamline the development and delivery of gene therapies for rare diseases.
The workshop at ASGCT was the BGTC’s first annual meeting, and highlighted progress so far, engaged with the community on next steps, and provided member and patient perspectives on overcoming the challenges faced in the gene therapy field. Read more about CIRM’s involvement in the BGTC here.
Throughout the week-long conference, CIRM had many great conversations with presenters, grantees, researchers and partnering organizations about its funding opportunities, programs and mission.
Sponsoring and attending conferences like ASGCT not only supports the advancement of cell and gene therapies in California but also provides opportunities for the public to share ideas and feedback directly with CIRM that help shape the future of its programs.
Imbasciani (pictured above at left) was elected to the six-year term at CIRM’s January Board meeting. He will replace outgoing chair Jonathan Thomas, who has served in the position since 2011.
“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.
CIRM also bid farewell to Sen. Art Torres (ret) and Jonathan Thomas (also known as JT) for their service on our Board.
JT was elected as Chair of the Board in 2011 after he was nominated by then-Governor Jerry Brown, Treasurer Bill Lockyer, and Lieutenant Governor Gavin Newsom.
He served as chair of the Governing Board for more than 12 years. In that time, he led CIRM in granting $2.5 billion in grants to support groundbreaking research and discovery to advance stem cell research and accelerate stem cell treatments to patients with unmet medical needs.
He led the agency as it expanded its work with industry, revamped its award processes, prepared for the expiration of bond funding, supported the drafting of Proposition 14, and planned for the next phase of CIRM’s programs after the voters approved $5.5 billion in additional funding.
Senator Torres was nominated in 2009 as CIRM Vice Chair by then-Lieutenant Governor Gavin Newsom and Treasurer Bill Lockyer, and shortly after was elected by the CIRM Governing Board.
Senator Torres served on the Grants and Standards working groups, and served as the inaugural Chair of the Accessibility and Affordability Working Group. He served on numerous subcommittees, led CIRM’s government relations efforts, and played a pivotal role in launching CIRM’s SPARK high school internship program.
CIRM expresses its deepest gratitude to Senator Torres and JT for their service on its Governing Board and for their dedication to the advancement of stem cell research and our mission to accelerate world class science to deliver transformative regenerative medicine treatments in an equitable manner to a diverse California and world.
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.
“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.”
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.”
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.”
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 email@example.com.
Earlier this year, CIRM welcomed many energetic and enthusiastic high school students at the 2022 SPARK Program annual conference in Oakland. The SPARK program is one of the California Institute for Regenerative Medicine’s (CIRM) many programs dedicated to building a diverse and highly-skilled workforce to support the growing regenerative medicine economy right here in California.
At the SPARK conference, a handful of students presented the stem cell research they did over the summer. It was a great opportunity to share their experiences as well as findings to their high school peers.
Just recently, Simran Ovalekar—a 2022 SPARK program intern—had the unique opportunity to share her research and findings with a wider audience, including undergraduate and PhD students at STEM Shadow Day in San Diego. The event aims to provide college prep students from San Diego and Imperial Valley counties with a unique experience to witness the “real world” of work in an engineering or scientific environment.
“At first I was nervous because I understood that I would be presenting not only in front of high school students, but also undergraduates and PhD candidates,” Simran says. “After reviewing my research, I felt solid and excited to present. I absolutely loved working in the lab so I knew all I had to do was be myself and show my enthusiasm.”
During the SPARK summer internship, Simran joined the Sacco Lab to study Duchenne Muscular Dystrophy (DMD) and how stem cells can be used to provide treatment. DMD is a progressive muscle wasting disorder with life expectancy of approximately age 20. There are around 17,000 people, the vast majority of them boys, diagnosed with DMD in the US.
Dr. Sacco’s lab—which has also received CIRM funding—is researching ways to generate healthy adult muscle stem cells using the patient’s own cells to generate healthy skeletal muscle.
For Simran, conducting research for DMD was personal, as her sister was born with a defect affecting the heart.
“When I began this program, I had a superficial understanding of what a stem cell was. Now, however, I am amazed at the possibilities stem cells provide, and with certainty, can say stem cells are the future of medicine.”
After her presentation at STEM Shadow Day, Simran says she received a positive response from attendees and was reminded why she loves science and of her passion for pursuing a career in stem cell research.
“I am looking forward to continue skeletal stem cell research and am even open to experimenting with other avenues of molecular medicine,” Simran says. “I am eager to have the opportunity to pursue the hands-on research I enjoyed this past summer.”
CIRM has also funded a clinical trial for people with DMD. We blogged about that work and how the impact it is having on some people’s lives.
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.
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:
The Stanford Alpha Stem Cell Clinic
Stanford University – Matthew Porteus
UCSF Alpha Stem Cell Clinic
U.C. San Francisco – Mark Walters
A comprehensive stem cell and gene therapy clinic to advance new therapies for a diverse patient population in California
Cedars-Sinai Medical Center – Michael Lewis
The City of Hope Alpha Clinic: A roadmap for equitable and inclusive access to regenerative medicine therapies for all Californians
City of Hope – Leo Wang
Alpha Stem Cell Clinic for Northern and Central California
U.C. Davis – Mehrdad Abedi
Expansion of the Alpha Stem Cell and Gene Therapy Clinic at UCLA
U.C. Los Angeles – Noah Federman
Alpha Clinic Network Expansion for Cell and Gene Therapies
University of Southern California – Thomas Buchanan
A hub and spoke community model to equitably deliver regenerative medicine therapies to diverse populations across four California counties
U.C. Irvine – Daniela Bota
UC San Diego Health CIRM Alpha Stem Cell Clinic
U.C. San Diego – Catriona Jamieson
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.
Aaliyah Staples-West didn’t originally envision becoming a stem cell researcher. As a student at San Diego State University, she admits that she sometimes struggled with reading protocols or finishing experiments on time. She also was originally studying chemistry, a very distinct scientific field from regenerative medicine.
“Everything I was looking for aligned with what I wanted to do,” she says. “I applied and I was greeted with open arms to an acceptance about a week later.” She even stayed in college for an extra semester so she could enroll in the CIRM internship program.
In the lab, Aaliyah worked with stem cells to derive brain organoids, which are three-dimensional, organ-like clusters of cells. She also researched vascular endothelial cells, which form a single cell layer that lines all blood vessels. She tested and observed these to further understand the causes of CSB.
Aaliyah also had opportunities to do work outside of the lab, traveling to various scientific conferences across the state to explain her work to other scientists.
She enjoyed sharing her findings, but Aaliyah says it was a challenge at first to learn all the complex science and terminology relating to stem cells. She overcame that obstacle by asking lots of questions and putting in extra effort to understanding the biology and reasoning behind her work.
“I would write down all the terms my mentor would say that I didn’t understand and look them up,” she says. “I would even practice using them in a sentence. I made it very intentional that if I wanted to continue researching in this field I needed to be on the same page.”
Now that her internship is over, Aaliyah is much more confident and has learned various techniques to successfully complete research projects. She now works for biotechnology company Resilience as a research associate working with induced pluripotent stem cells (iPSCs) and hematopoietic stem cells. Though she originally intended to go to medical school, she is now looking into MD/PhD programs where she can apply all that she’s learned in her training and education.
“I never thought I would have a love for stem cell research until participating in this program,” she says. “Stem cell research and regenerative medicine provide infinite opportunities for developing, understanding and potentially curing diseases. It’s important to continue this type of research to ensure science is quickly evolving and to make an impact on overall health.”
To date, there are 1,663 Bridges alumni, and another 109 Bridges trainees are completing their internships in 2022. Learn more about CIRM’s internship programs here.
All photos courtesy of Sarah White/SDSU and Aaliyah Staples-West.
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.
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.