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.

Physicians and patient advocates on the front lines of the fight for a more equitable health system

Over the last year there has been increasing awareness of the inequalities in the American healthcare system. At every level there is evidence of bias, discrimination and unequal access to the best care. Sometimes unequal access to any care. That is, hopefully, changing but only if the new awareness is matched with action.

At the recent World Stem Cell Summit CIRM helped pull together a panel of physicians and patient advocates who have been leading the charge for change for years. The panel was called ‘Addressing Disparities, Promoting Equity and Inclusion in Clinical Research.’

The panelists include:

This image has an empty alt attribute; its file name is ysabel-duron.jpeg
Ysabel Duron – Founder of The Latino Cancer Institute & CIRM Board member
Adrienne Shapiro – sickle cell disease patient advocate, Founder of Axis Advocacy – Sickle Cell Disease support and advocacy group
Dr. Leah Ke‘ala‘aumoe Dowsett – Clinical geneticist, serves on hospital DEI committee, Board member Association of Native Hawaiian Physicians
Dr. Nathan Chomilo – Co-Founder, Minnesota Doctors for Health Equity and head of the Minnesota COVID Vaccine Equity Program

The conversation they had was informative, illuminating and fascinating. But it didn’t sugar coat where we are, and the hard work ahead of us to get to where we need to be.

Enjoy the event, with apologies for the inept cameo appearance by me at the beginning of the video. Technology clearly isn’t my forte.

Driving Innovation While Addressing Health Disparities Among People of Color

Image courtesy of Science Photo Library

One of the wonders of regenerative medicine is its broad applicability, which provides us with the opportunity to build upon existing knowledge and concepts.  In the midst of a global pandemic, researchers have responded to the needs of patients severely afflicted with COVID-19 by repurposing existing therapies being developed to treat patients.  The California Institute for Regenerative Medicine (CIRM) responded immediately to the pandemic and to researchers wanting to help by providing $5 million in emergency funding for COVID-19 related projects.  In a short time span, this funding has driven innovation in the form of 17 new projects targeting COVID-19, many of which are based on previously developed concepts being repurposed to deal with the novel coronavirus.

One such example is a clinical trial funded by CIRM that uses natural killer (NK) cells, a type of white blood cell that is a vital part of the immune system, which are administered to patients with COVID-19. NK cells play an important role in defense against cancer and in fighting off viral infections.  In fact, this exact same therapy was previously used in a clinical trial for patients with Acute Myeloid Leukemia, a type of blood cancer.

Another clinical trial funded by CIRM uses mesenchymal stromal cells (MSCs), a type of stem cell, to treat acute respiratory distress syndrome (ARDS), a life-threatening lung injury that occurs when fluid leaks into the lungs.  As a result of ARDS, oxygen cannot get into the body and patients have difficulty breathing.  ARDS is one of the most serious and lethal consequences of COVID-19, which is why this trial was expanded after the coronavirus pandemic to include COVID-19 positive patients.   

Despite these great strides in driving innovation of therapies, one challenge that still needs to be tackled is providing patients access to these therapies, particularly people from underrepresented and underserved communities.  In California alone, there have been over 621,000 positive cases as of August 2020, with more cases every day.  However, the impact of the pandemic is disproportionately affecting the Latinx and African American communities more than others. An analysis by the Los Angeles Times found that the Latinx and African American communities have double the mortality rate from the coronavirus in Los Angeles County.  Additionally, a surge in cases is being seen in poorer communities in comparison to wealthier ones.

Until a vaccine can be successfully developed and implemented to obtain herd immunity, the number of cases will continue to climb.  There is also the challenge of the long term health effects of COVID-19, which can consist of neurological, breathing, and heart problems according to an article in Science.  Unfortunately, a study published in the New England Journal of Medicine found that despite disproportionately higher rates of COVID-19 infection, hospitalization and death among people of color, they are significantly underrepresented in COVID-19 clinical trials.

The challenge of underrepresentation in clinical trials and research needs to be addressed by creating a more diverse population of study participants, so as to better generalize results to the U.S. population as a whole.  CIRM Board Member Ysabel Duron, a leading figure in cancer education in the Latinx community, has advocated for more inclusion and outreach efforts directed towards underserved and underrepresented communities.  By communicating with patients in underserved and underrepresented communities, building relationships established on a foundation of trust, and connecting patients with potential trial matches, underrepresentation can be alleviated.

To help in addressing these disparities, CIRM has taken action by changing the requirements for its discovery stage research projects, which promote promising new technologies that could be translated to enable broad use and improve patient care, and clinical trial stage projects.

For clinical trials, all proposals must include a written plan in the application for outreach and study participation by underserved and disproportionately affected populations. Priority will be given to projects with the highest quality plans in this regard. For discovery projects, all proposals must provide a statement describing how their overall study plan and design has considered the influence of race, ethnicity, sex and gender diversity.  Additionally, all proposals should discuss the limitations, advantages, and/or challenges in developing a product or tools that addresses the unmet medical needs of California’s diverse population, including underserved communities.  There is still much more work that needs to be done to address health disparities, but steps such as these can help steer progress in the right direction.

Driving innovation while addressing health disparities among people of color is just one of many opportunities and challenges of regenerative medicine in a post pandemic world.  This blog post is part of Signal’s fifth annual blog carnival. Please click here to read what other bloggers think about this topic.

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

Some of the stars of our Annual Report

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

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

Dr. Catriona Jamieson, UC San Diego physician and researcher

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

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

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

Jordan Janz and Dr. Stephanie Cherqui

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

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

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

We hope you enjoy it.

Journalism Pioneer and Founder of Latino Cancer Institute Joins Stem Cell Agency Board

Ysabel Duron

Ysabel Duron, a pioneering award-winning Latina journalist, and a leading figure in cancer education in the Latino community in the US, has been appointed to the governing Board of the California Institute for Regenerative Medicine.  

State Controller Betty Yee made the appointment saying: “Ms. Duron’s personal perspective as a (cancer) survivor and her commitment to equity will serve the institute’s mission well.”

Ms. Duron was a journalist and TV news anchor for more than 43 years winning numerous awards, including two EMMYS. She has been inducted into the Hall of Fame of the National Association of Hispanic Journalists and given the Living Legacy Award by the Chicana/Latina Foundation.

As a journalist she covered her own battle against Hodgkin’s Lymphoma, using her reporting to help raise awareness about the disease and the health disparities involved in treating it in communities of color.

In 2003, as a result of her own experience, she founded Latinas Contra Cancer, a non-profit organization that advocates for and serves the Latino community. She is now the President of the Latino Cancer Institute, a national network of Latino cancer service agencies addressing the community’s cancer disparities. 

“As a veteran journalist, I like to think I am as curious as a scientist, I just frame the question differently,” says Ms. Duron. “Usually I am looking for the best return for the public health! This appointment gives me a new learning opportunity to understand a very complex issue, and, make it bite size so the public, patients and advocates will understand how these scientific revelations will impact lives in the short term and the long run. As a steward of taxpayer dollars, I also want to make sure there is equity for communities across California, and that the research serves all of us”

We are delighted to welcome Ysabel to the Board,” says Jonathan Thomas, CIRM Board Chair. “She has a well-deserved reputation as a champion for patients and an activist committed to breaking down barriers that prevent people in the Latino community accessing quality care. She will add a powerful voice to our Board.”

Ms. Duron replaces Sherry Lansing as the CIRM Board patient advocate for cancer.

“It is impossible to overstate Sherry’s importance and contributions to CIRM over her long tenure on the Board,” says Thomas. “Sherry was one of the original Board Members and a towering presence who played a central role in the formulative years of the Agency, including co-chairing the Standards Working Group, which set the ethical guidelines for the future research CIRM would enable.  Since that time, she has been a commanding voice of reason and an unrelenting positive force on behalf of all patients. She will be sorely missed.”