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.

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.

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.

Stem Cell Agency Invests $46 Million in New Education Program

CIRM Bridges students 2022. The CIRM Board approved funding for a program to help even more students advance a career in science.

The governing Board of the California Institute for Regenerative Medicine (CIRM) has approved $46,076,430 to invest in its newest education pillar- the COMPASS (Creating Opportunities through Mentorship and Partnership Across Stem cell Science) training program.

Education is at the core of CIRM’s mission of accelerating world class science to deliver transformative regenerative medicine treatments in an equitable manner to a diverse California and world. And funding these additional programs is an important step in ensuring that California has a well-trained stem cell workforce.

The objective of COMPASS is to prepare a diverse cadre of undergraduate students for careers in regenerative medicine through combining hands-on research opportunities with strategic and structured mentorship experiences.

“Education and infrastructure are two funding pillars critical for creating the next generation of researchers and conducting stem cell based clinical trials,” says Jonathan Thomas, Ph.D., J.D., Chair of the CIRM Board. “The importance of these programs was acknowledged in Proposition 14 and we expect that they will continue to be important components of CIRM’s programs and strategic direction in the years to come.”

Most undergraduate research training programs, including those targeting students from underserved communities, target individuals with predefined academic credentials as well as a stated commitment towards graduate school, medical school, or faculty positions in academia. COMPASS will support the development and implementation of novel strategies to recognize and foster untapped talent that can lead to new and valuable perspectives that are specific to the challenges of regenerative medicine, and that will create new paths to a spectrum of careers that are not always apparent to students in the academic, undergraduate environment.

COMPASS will complement but not compete with CIRM’s Bridges program, a subset of which serve a different, but equally important population of undergraduate trainees; similarly, the program is unlikely to compete for the same pools of students that would be most likely to receive support through the major NIH Training Programs such as MARC and RISE.

Here are the 16 successful applicants.

Application numberTitlePrincipal InvestigatorAmount
EDUC5-13840  The COMPASS Scholars Program – Developing Today’s Untapped Talent into Tomorrow’s STEM Cell Researchers    John Matsui, University of California, Berkeley    $2,908,950
EDUC5-13634  COMPASS Undergraduate Program  Alice F Tarantal, University of California, Davis    $2,909,950  
EDUC5-13637  Research Mentorship Program in Regenerative Medicine Careers for a Diverse Undergraduate Student Body    Brian J. Cummings, University of California, Irvine    $2,729,900
EDUC5-13665  CIRM COMPASS Training Program (N-COMPASS)  Cindy S Malone, The University Corporation at California State University, Northridge    $2,909,700  
EDUC5-13817  COMPASS: Accelerating Stem Cell Research by Educating and Empowering New Stem Cell Researchers  Tracy L Johnson, University of California, Los Angeles    $2,910,000  
EDUC5-13744  Training and mentorship program in stem cell biology and engineering: A COMPASS for the future  Dennis Clegg, University of California, Santa Barbara    $2,746,000  
EDUC5-13636  Research Training and Mentorship Program to Inspire Diverse Undergraduates toward Regenerative Medicine
Careers (RAMP)
  Huinan Hannah Liu, The Regents of the University of California on behalf of its Riverside Campus    $2,910,000  
EDUC5-13679  Inclusive Pathways for a Stem Cell Scholar (iPSCs) Undergraduate Training Program    Lily Chen, San Francisco State University    $2,894,500
EDUC5-13733  A COMPASS to guide the growth of a diverse regenerative medicine workforce that represents California and benefits
the world
  Kristen OHalloran Cardinal, Cal Poly Corporation, an Auxiliary of California Polytechnic State University, San Luis Obispo    $2,887,939  
EDUC5-13619  Increase Diversity, Equity, and Advancement in Cell Based Manufacturing Sciences (IDEA-CBMS)  Michael Fino, MiraCosta College    $2,894,500  
EDUC5-13667  COMPASS Program for Southern California Hispanic Serving Institution  Bianca Romina Mothé, California State University San Marcos Corporation    $2,877,200  
EDUC5-13653  Student Pluripotency: Realizing Untapped Undergraduate Potential in Regenerative Medicine  Daniel Nickerson, California State University, San Bernardino    $2,909,853  
EDUC5-13647  COMPASS: an inclusive Pipeline for Research and Other Stem cell-based Professions in Regenerative medicine
(iPROSPR)  
  Alison Miyamoto, CSU Fullerton Auxiliary Services Corporation    $2,883,440
EDUC5-13686  Training Undergraduates in Stem Cell Engineering and Biology (TUSCEB)    Kara E McCloskey, University of California, Merced    $2,909,999
EDUC5-13853  COMPASS: Guiding Undergraduates to Careers in Regenerative Medicine    Senta Georgia, University of Southern California    $2,899,999
EDUC5-13910  IDEA-CBMS – Increase Diversity, Equity, and Advancement in Cell Based Manufacturing Sciences    James Dekloe, Solano Community College    $2,894,500

Stem Cell Agency funds clinical trial targeting scarred urethras

A urethral stricture is scarring of the tube that carries urine out of the body. If left untreated it can be intensely painful and lead to kidney stones and infections. That’s why the governing Board of the California Institute for Regenerative Medicine (CIRM) is investing more than $3.8 million in a Phase 1 clinical trial to create a stem cell-based therapy for the condition.

This is the 81st clinical trial that CIRM has funded.

When a scar, or stricture, forms along the urethra it impedes the flow of urine and causes other complications. James Yoo, M.D., Ph.D., and his team at Wake Forest University Health Sciences will use epithelial and smooth muscle cells, taken from the patient’s bladder, and layer them on to a synthetic tubular scaffold. The tube will then be surgically implanted inside the urethra.

The goal is for the progenitor cells to support self-renewal of the tissue and for the entire structure to become integrated into the surrounding tissue and become indistinguishable from it, restoring normal urinary function. Dr. Yoo and his team believe their approach has the potential to be effective for at least a decade.

“While not immediately life-threatening, urethral strictures lead to multiple health complications that impair quality of life and predispose to kidney dysfunction,” says Dr. Maria T. Millan, President and CEO of CIRM. “Developing an effective and durable treatment would significantly impact lives and has the potential to decrease the cumulative healthcare costs of treating recurrent kidney stones, infections and downstream kidney complications, especially of long-segment urethral strictures.”

Stem Cell Agency Board Invests in 19 Discovery Research Programs Targeting Cancers, Heart Disease and Other Disorders

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Dr. Judy Shizuru, Stanford University

While stem cell and gene therapy research has advanced dramatically in recent years, there are still many unknowns and many questions remaining about how best to use these approaches in developing therapies. That’s why the governing Board of the California Institute for Regenerative Medicine (CIRM) today approved investing almost $25 million in 19 projects in early stage or Discovery research.

The awards are from CIRM’s DISC2 Quest program, which supports  the discovery of promising new stem cell-based and gene therapy technologies that could be translated to enable broad use and ultimately, improve patient care.

“Every therapy that helps save lives or change lives begins with a researcher asking a simple question, “What if?”, says Dr. Maria T. Millan, the President and CEO of CIRM. “Our Quest awards reflect the need to keep supporting early stage research, to gain a deeper understanding of stem cells work and how we can best tap into that potential to advance the field.”

Dr. Judy Shizuru at Stanford University was awarded $1.34 million to develop a safer, less-toxic form of bone marrow or hematopoietic stem cell transplant (HCT). HCT is the only proven cure for many forms of blood disorders that affect people of all ages, sexes, and races worldwide. However, current methods involve the use of chemotherapy or radiation to destroy the patient’s own unhealthy blood stem cells and make room for the new, healthy ones. This approach is toxic and complex and can only be performed by specialized teams in major medical centers, making access particularly difficult for poor and underserved communities.

Dr. Shizuru proposes developing an antibody that can direct the patient’s own immune cells to kill diseased blood stem cells. This would make stem cell transplant safer and more effective for the treatment of many life-threatening blood disorders, and more accessible for people in rural or remote parts of the country.

Lili Yang UCLA Broad Stem Cell Research Center: Photo courtesy Reed Hutchinson PhotoGraphics

Dr. Lili Yang at UCLA was awarded $1.4 million to develop an off-the-shelf cell therapy for ovarian cancer, which causes more deaths than any other cancer of the female reproductive system.

Dr. Yang is using immune system cells, called invariant natural killer T cells (iNKT) to attack cancer cells. However, these iNKT cells are only found in small numbers in the blood so current approaches involve taking those cells from the patient and, in the lab, modifying them to increase their numbers and strength before transplanting them back into the patient. This is both time consuming and expensive, and the patient’s own iNKT cells may have been damaged by the cancer, reducing the likelihood of success.

In this new study Dr. Yang will use healthy donor cord blood cells and, through genetic engineering, turn them into the specific form of iNKT cell therapy targeting ovarian cancer. This DISC2 award will support the development of these cells and do the necessary testing and studies to advance it to the translational stage.

Timothy Hoey and Tenaya Therapeutics Inc. have been awarded $1.2 million to test a gene therapy approach to replace heart cells damaged by a heart attack.

Heart disease is the leading cause of death in the U.S. with the highest incidence among African Americans. It’s caused by damage or death of functional heart muscle cells, usually due to heart attack. Because these heart muscle cells are unable to regenerate the damage is permanent. Dr. Hoey’s team is developing a gene therapy that can be injected into patients and turn their cardiac fibroblasts, cells that can contribute to scar tissue, into functioning heart muscle cells, replacing those damaged by the heart attack.

The full list of DISC2 Quest awards is:

APPLICATION NUMBERTITLE OF PROGRAMPRINCIPAL INVESTIGATORAMOUNT
  DISC2-13400  Targeted Immunotherapy-Based Blood Stem Cell Transplantation    Judy Shizuru, Stanford Universtiy  $1,341,910    
  DISC2-13505  Combating Ovarian Cancer Using Stem Cell-Engineered Off-The-Shelf CAR-iNKT Cells    Lili Yang, UCLA  $1,404,000
  DISC2-13515  A treatment for Rett syndrome using glial-restricted
neural progenitor cells  
  Alysson Muotri, UC San Diego  $1,402,240    
  DISC2-13454  Targeting pancreatic cancer stem cells with DDR1 antibodies.    Michael Karin, UC San Diego  $1,425,600  
  DISC2-13483  Enabling non-genetic activity-driven maturation of iPSC-derived neurons    Alex Savtchenko, Nanotools Bioscience  $675,000
  DISC2-13405  Hematopoietic Stem Cell Gene Therapy for Alpha
Thalassemia  
  Don Kohn, UCLA    $1,323,007  
    DISC2-13507  CAR T cells targeting abnormal N-glycans for the
treatment of refractory/metastatic solid cancers  
  Michael Demetriou, UC Irvine  $1,414,800  
  DISC2-13463  Drug Development of Inhibitors of Inflammation Using
Human iPSC-Derived Microglia (hiMG)  
  Stuart Lipton, Scripps Research Inst.  $1,658,123  
  DISC2-13390  Cardiac Reprogramming Gene Therapy for Post-Myocardial Infarction Heart Failure    Timothy Hoey, Tenaya Therapeutics  $1,215,000  
  DISC2-13417  AAV-dCas9 Epigenetic Editing for CDKL5 Deficiency Disorder    Kyle Fink, UC Davis  $1,429,378  
  DISC2-13415  Defining the Optimal Gene Therapy Approach of
Human Hematopoietic Stem Cells for the Treatment of
Dedicator of Cytokinesis 8 (DOCK8) Deficiency  
  Caroline Kuo, UCLA  $1,386,232  
  DISC2-13498  Bioengineering human stem cell-derived beta cell
organoids to monitor cell health in real time and improve therapeutic outcomes in patients  
  Katy Digovich, Minutia, Inc.  $1,198,550  
  DISC2-13469  Novel antisense therapy to treat genetic forms of
neurodevelopmental disease.  
  Joseph Gleeson, UC San Diego  $1,180,654  
  DISC2-13428  Therapeutics to overcome the differentiation roadblock in Myelodysplastic Syndrome (MDS)    Michael Bollong, Scripps Research Inst.  $1,244,160  
  DISC2-13456  Novel methods to eliminate cancer stem cells    Dinesh Rao, UCLA  $1,384,347  
  DISC2-13441  A new precision medicine based iPSC-derived model to study personalized intestinal fibrosis treatments in
pediatric patients with Crohn’s diseas  
  Robert Barrett Cedars-Sinai  $776,340
  DISC2-13512  Modified RNA-Based Gene Therapy for Cardiac
Regeneration Through Cardiomyocyte Proliferation
  Deepak Srivastava, Gladstone Institutes  $1,565,784
  DISC2-13510  An hematopoietic stem-cell-based approach to treat HIV employing CAR-T cells and anti-HIV broadly
neutralizing antibodies  
  Brian Lawson, The Scintillon Institute  $1,143,600  
  DISC2-13475  Developing gene therapy for dominant optic atrophy using human pluripotent stem cell-derived retinal organoid disease model    Xian-Jie Yang, UCLA  $1,345,691  

The power of the patient advocate: how a quick visit led to an $11M grant to fund a clinical trial

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Members of NFOSD visiting UC Davis in 2013

At the California Institute for Regenerative Medicine (CIRM) we are fortunate in having enough money to fund the most promising research to be tested in a clinical trial. Those are expensive projects, often costing tens of millions of dollars. But sometimes the projects that come to our Board start out years before in much more humble circumstances, raising money through patient advocates, tapping into the commitment and ingenuity of those affected by a disease, to help advance the search for a treatment.

That was definitely the case with a program the CIRM Board voted to approve yesterday, investing more than $11 million dollars to fund a Phase 2 clinical trial testing a cell therapy for dysphagia. That’s a debilitating condition that affects many people treated for head and neck cancer.

Patients with head and neck cancer often undergo surgery and/or radiation to remove the tumors. As a result, they may develop problems swallowing and this can lead to serious complications such as malnutrition, dehydration, social isolation, or a dependence on using a feeding tube. Patients may also inhale food or liquids into their lungs causing infections, pneumonia and death. The only effective therapy is a total laryngectomy where the larynx or voice box is removed, leaving the person unable to speak.

Dr. Peter Belafsky and his team at the University of California at Davis are developing a therapeutic approach using Autologous Muscle Derived Progenitor Cells (AMDC), cells derived from a biopsy of the patient’s own muscle, elsewhere in the body. Those AMDCs are injected into the tongue of the patient, where they fuse with existing muscle fibers to increase tongue strength and ability to swallow.

The $11,015,936 that Dr. Belafsky is getting from CIRM will enable them to test this approach in patients. But without grass roots support the program might never have made it this far.

Ed Steger is a long-term survivor of head and neck cancer, he’s also the President of the National Foundation of Swallowing Disorders (NFOSD). In 2007, after being treated for his cancer, Ed developed a severe swallowing disorder. It helped motivate him to push for better treatment options.

In 2013, a dozen swallowing disorder patients visited UC Davis to learn how stem cells might help people with dysphagia. (You can read about that visit here). Ed says: “We were beyond thrilled with the possibilities and drawing on patients and other UCD contacts our foundation raised enough funds to support a small UCD clinical trial under the guidance of Dr. Belafsky in mouse models that demonstrated these possibilities.”

A few years later that small funding by patients and their family members grew into a well-funded Phase I/II human clinical trial. Ed says the data that trial produced is helping advance the search for treatments.

“Skipping forward to the present, this has now blossomed into an additional $11 million grant, from CIRM, to continue the work that could be a game changer for millions of Americans who suffer annually from oral phase dysphagia. My hat is off to all those that have made this possible… the donors, patient advocates, and the dedicated committed researchers and physicians who are performing this promising and innovative research.”

Our hats are off to them too. Their efforts are making what once might have seemed impossible, a real possibility.