Dr. Wells and his team of researchers will be using a stem cell-based “cell village” platform to analyze factors underlying differences in viral susceptibility across 150 donors. These cell villages are composed of cells that capture the immense diversity of racial and ethnic groups across California, creating an environment for comprehensive analysis of genetic variants and cellular characteristics.
“As the personalized medicine revolution begins to take shape, it’s imperative that we ensure our findings are relevant to all people. Cell villages could play a significant role in addressing societal issues like the health disparities experienced by women and ethnic minorities in the United States,” said Dr. Wells.
This research has the potential to impact health disparities and advance future antiviral drug development. By studying the biological factors that influence differences in immune response to viruses, the project could provide valuable insights into how to develop more effective treatments.
“CIRM is dedicated to promoting diversity, equity, and inclusion in the research we fund. Projects such as this one, harnessing knowledge utilizing stem cell lines from a wide array of ancestral backgrounds, underlines our commitment to research that is reflective of our diverse population and is a cornerstone of our overarching objectives,” said Rosa Canet-Avilés, PhD, Vice President of Scientific Programs at CIRM.
Images of cell-villages above were provided by Dr. Michael Wells’ Lab and UCLA.
Regenerative medicine is a rapidly evolving field. As more therapies reach commercialization and clinical trial stages, there remains an immediate need to equip direct patient care nurses with skills to support patients on complex clinical trials as well as novel standard-of-care therapies.
The CIRM-funded Alpha Clinics are a network of top California medical centers specializing in delivering stem cell and gene therapy clinical trials to patients. Each Alpha Clinic site contributed educational materials for the symposium.
A Dynamic Learning Experience for California Nurses
The two-day program provided a dynamic learning experience to direct care registered nurses, clinical research nurses, advanced practice providers and others seeking to enhance their knowledge in regenerative medicine. The event aimed to empower nurses to disseminate accurate, evidence-based information to their students and colleagues, ultimately advancing the field of regenerative medicine nursing in California.
CIRM Board Chair Vito Imbasciani, PhD, MD, kicked off the event and provided an overview of CIRM’s history and the important role nurses play in advancing the state agency’s mission to “accelerate world class science to deliver transformative regenerative medicine treatments in an equitable manner to a diverse California and world.”
“You are pioneers,” said Imbasciani. “You are at the threshold of a dawning era where cell and gene therapies, as well as regenerative medicine as a whole, will become more and more commonplace and a part of routine clinical care.”
Throughout the event, attendees were immersed in engaging and practical sessions, emphasizing nurses’ pivotal roles in the realm of cell and gene therapy, particularly through CIRM’s Alpha Clinics. Attendees were eligible to receive up to 14 Continuing Education Units (CEUs) for both days of attendance.
Highlighting the Patient Experience
Numerous presenters described the patient journey in clinical trials, highlighting that patients often encounter financial and logistical challenges participating in cell and gene therapy trials because they require many visits to a medical center. CIRM’s Associate Director of Patient Access Geoff Lomax underscored the patient journey and the value of CIRM’s Patient Support Program and Community Care Centers of Excellence, emerging CIRM programs aimed at providing financial, logistical and social support for patients in clinical trials.
Symposium speakers also highlighted the critical importance of nurses in healthcare, especially in the administration of cutting-edge treatments like CAR-T Cell therapies. A nurse participant recognized that many nursing educational courses don’t include regenerative medicine-focused trainings and emphasized the need for training in such therapies to provide the best care to patients.
Other topics discussed at the event included apheresis—a technology that separates donated blood components to treat certain illnesses—and Good Manufacturing Practices (GMP). Because nurses play an integral role in these processes, several event sessions were dedicated to practical considerations.
Representatives from bluebird bio, a biomedical company and event sponsor, presented exciting developments in gene therapy, highlighting the recent FDA approval of LYFGENIA, a gene therapy treatment for sickle cell disease.
The symposium fostered networking opportunities among attendees, enabling them to exchange ideas, experiences, and best practices with peers and staff throughout the state.
“Symposiums like these are so important for nurses because we get to hear about all these different therapies that we don’t typically work with,” said Emma King, an attendee from Children’s Hospital Los Angeles. “You can see what’s coming in the future and what different hospitals around California do for regenerative medicine and hear about patient experiences. It makes you a better, well-rounded nurse.”
Kelly Tomlinson, DNP, RN, NEA-BC, is the administrative director of Immune Effector Cell Therapy at host hospital City of Hope and was both a moderator and speaker at the symposium. She said regenerative medicine “is the future.”
“This symposium helped to educate and engage our nursing workforce by giving them new tools and knowledge within this emerging field,” Tomlinson said. “The expertise at this event was astounding. I was honored to not only be a participant but a moderator and speaker. I left with a wealth of knowledge and a renewed excitement for what the future holds for our patients and communities.”
Thank you to the City of Hope Alpha Clinic for organizing the event and to each CIRM Alpha Clinic for contributing speakers and materials for the program. The event was sponsored by Johnson & Johnson, bluebird bio, Kite Pharma, and AbelZeta. Senior Advisor, Community Outreach and Advocacy Jacqueline Hantgan contributed to this article.
The California Institute for Regenerative Medicine (CIRM) recently awarded $1.5 million to Denise Al Alam, PhD, of the Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center to support research that aims to understand lung disease in individuals with Trisomy 21, also known as Down Syndrome.
Although Trisomy 21 affects multiple organ systems, respiratory complications are a major cause of death in both children and adults with this genetic condition. Dr. Al Alam’s project will use patient-derived pluripotent stem cells from ethnically diverse backgrounds to model alveolar defects inherent to Trisomy 21.
The study is poised to generate new Trisomy 21 cell lines to study defects specific to this condition. Researchers hope to uncover the genes and pathways associated with these defects, paving the way for targeted therapeutic approaches.
In California, about 667 babies are born with Down Syndrome every year, with the highest rate among Latinx infants.
“Respiratory complications are a significant cause of mortality in both children and adults with Down Syndrome. We are thrilled to support this research that aims to deepen our understanding of lung disease in individuals with Down Syndrome. This knowledge holds immense potential to intervene early and improve outcomes for those with this condition,” added Dr. Canet-Avilés.
Understanding lung disease in individuals with Trisomy 21 is crucial to improving outcomes for those living with this genetic condition. This innovative research project has the potential to lead to targeted therapeutic approaches and improve the quality of life for those affected by Trisomy 21.
This funding will support 11 projects under CIRM’s Foundation Awards Program, which aims to drive rigorous studies addressing critical basic knowledge gaps in the biology of stem cells and regenerative medicine approaches and advance stem cell-based tools.
Additionally, CIRM awarded $6 million to Eugenio Cingolani, PhD, of Cedars Sinai, to support a preclinical project in the Agency’s clinical awards (CLIN 1). The project is designed to advance a noninvasive cell-derived therapy for ventricular tachycardia, a condition in which the heart rhythm is irregular.
“CIRM is dedicated to the advancement of early-stage transformative therapies for prevalent health conditions such as heart disease,” said Dr. Abla Creasey, PhD, Vice President of Therapeutics Development at CIRM.
CIRM also awarded $2.5 million to support establishing a Patient Support Program (PSP) to enhance patient access to CIRM-funded clinical trials, an essential component of CIRM’s mission and Infrastructure Program.
The discovery awards approved at CIRM’s March Independent Citizens’ Oversight Committee (ICOC) meeting include:
Application #
Program Title
Principal Investigator/Institution
Amount
DISC0-15949
Neuroimmune interactions in the developing human brain
Nowakowski, Tomasz – UCSF
$1,626,000
DISC0-15737
Village-based identification of human risk factors for viral neuropathogenesis
Wells, Michael F. – UCLA
$1,577,448
DISC0-15921
Interrogating Satellite Cell and Myofiber Defects and Repair in Human DMD using Single Nuclei/Single Cell RNA Sequencing of Muscle Resident Cells
Miceli, M. Carrie – UCLA
$1,578,000
DISC0-16039
39 Lewy body dementia α-synuclein, and cell-specific mechanisms of neurodegeneration
Finkbeiner, Steven M. – Gladstone
$1,739,760
DISC0-16122
Mapping and modeling endothelial cell fate decisions for pulmonary arterial hypertension
Qiu, Xiaojie – Stanford
$1,540,798
DISC0-15654
4 Modeling and understanding alveolar hypoplasia in Down syndrome using iPSCs-derived alveolar type II cells
Al Alam, Denise – Lundquist Institute
$1,524,196
DISC0-15816
Investigating the SGF29/SAGA complex in regulation of normal and cancer stem cells
Deshpande, Aniruddha – Sanford-Burnham
$1,647,600
DISC0-15774
Modeling of GATAD2B-associated neurodevelopmental disorder and NuRDopathies: Investigation of cellular & molecular anomalies altering neurodevelopment
Pierson, Tyler Mark – Cedars-Sinai
$1,318,441
DISC0-15972
Immune cloaking of human stem cell-derived insulin producing cells for curative cell therapy without immunosuppression
Digovich, Katy – Minutia, Inc.
$1,192,586
DISC0-15920
Harnessing the rejuvenating capacity of pregnancy-associated factors to restore aged stem cell function
Alperin, Marianna – UCSD
$1,539,520
DISC0-15689
Utilizing Age-Specific Adipocyte Progenitor Cells for Cell Therapy in Older Patients
The California Institute for Regenerative Medicine (CIRM), one of the world’s largest institutions dedicated to regenerative medicine, is proud to announce the launch of “Championing Change for Sickle Cell in California,” a pioneering campaign aimed at increasing awareness of Sickle Cell Disease (SCD) and research aimed at improving the treatment landscape for the condition.
With more than 100,000 Americans and 20 million people worldwide affected by this debilitating condition, CIRM is committed to funding cutting-edge cell and gene therapy programs that have the potential to transform the lives of SCD patients.
Sickle cell disease is a genetic disorder that causes red blood cells to assume a sickle shape under stress, leading to clogged blood vessels, excruciating pain crises, and organ damage.
This disease disproportionately affects people of African American and Latinx heritage. It is estimated that SCD occurs among about 1 out of every 365 Black or African-American births. Latinx Californians make up 7.3 percent of sickle cell disease cases—and Latinx newborns make up 11 percent of new cases.
In collaboration with key organizations such as sickle cell patient advocacy group Axis Advocacy, the National Heart, Lung, and Blood Institute’s (NHLBI) Cure Sickle Cell Initiative, and the UCSF Sickle Cell Center of Excellence, CIRM is working to champion change and drive progress for sickle cell treatments.
Flyers and social media graphics are available for download at CIRM’s website.
Community partners and patient advocates play a crucial role in the development and success of these treatments, and CIRM recognizes their importance. Among the key calls to action of the campaign include becoming a sickle cell disease advocate, spreading awareness of clinical trial options for sickle cell, downloading a fact sheet and learning more about the NHLBI Cure Sickle Cell Initiative.
In addition to spreading awareness of this condition, the campaign highlights how CIRM is funding research into stem cell, gene therapy, and other regenerative medicine procedures for sickle cell disease. To date, CIRM has invested over $65 million towards research for sickle cell, ranging from basic discovery to clinical trials.
Evie Junior participated in a CIRM-funded clinical trial for sickle cell disease at UCLA. Photo: Jaquell Chandler
CIRM’s new campaign comes at the heels of two recently FDA-approved gene therapy treatments for sickle cell disease. Vertex and CRISPR Therapeutics’ Casgevy and bluebird bio’s Lyfgenia have brought forth a new era of treatment options for people living with the condition.
CIRM introduced the “Championing Change for Sickle Cell in California” campaign at Oakland’s Black Joy Parade in February 2023. Pictured is CIRM’s Community Outreach Manager Aditi Desai.
“As the first regulatory authorization for a CRISPR-based gene-editing therapy in the world, this approval not only marks a milestone in the advancement of cell and gene therapies, but it also marks a transformative step in the treatment landscape for the millions of people impacted by sickle cell disease,” said CIRM Vice President of Therapeutics Development, Abla Creasey, PhD.
For more information on CIRM’s Sickle Cell Disease campaign, ongoing research projects, and ways to champion change, please visit cirm.ca.gov/sickle-cell-disease. To get involved in the campaign, please contact CIRM’s Community Outreach Manager Aditi Desai at adesai@cirm.ca.gov.
CIRM has produced videos highlighting the experiences of participants from our educational and training programs.
At the California Institute for Regenerative Medicine (CIRM), we are passionate about catalyzing growth in regenerative medicine while championing inclusivity. One of CIRM’s strategic goals is to build a diverse and skilled workforce that represents the rich tapestry of California’s population.
That’s why the CIRM—spearheaded by the Scientific Programs & Education team—is launching a set of videos to showcase the unique perspectives of CIRM’s educational and training program participants from across the state, including students, trainees, program directors, and CIRM leadership. Watch both videos on CIRM’s YouTube channel here and here.
Funding Training Programs for a Diverse California
CIRM’s education and training programs support the next generation of regenerative medicine scientists, ranging from high school and undergrad students to doctoral candidates and clinical fellows.
CIRM funds four unique internship programs, each with its own set of requirements: SPARK (high school), Bridges (Undergraduate and Masters), COMPASS (Undergraduate), and Scholars (Pre-Doctoral, Post-Doctoral, and Clinical Fellows).
Each program offers hands-on experience to foster a workforce adept in stem cell science and the technical skills that are crucial for present and future scientific and medical advancements.
Partnering with community colleges and universities, as well as research institutions and biotechnology companies, each program aims to attract diverse talent to regenerative medicine by offering varied career paths and accessibility across a broader educational spectrum.
Aileen Nava is doctoral student at UCLA and a CSU Fullerton Bridges alumna from 2017
To date, CIRM’s educational and training programs have supported 3,800 interns and fellows, the majority of whom have achieved employment or chose to pursue further education in regenerative medicine or related fields.
Alumni from these programs participate in a broad spectrum of rewarding careers. These range from traditional scientific disciplines to in-demand, complementary and/or ancillary pressions within education, community and patient advocacy, business development, and regulatory affairs. Over 70 institutions have joined CIRM in its mission to advance world class science, deliver real world solutions, and provide opportunities to all.
Opportunities for People with Various Educational Backgrounds
In addition to highlighting experiences of program participants, the videos emphasize that careers in regenerative medicine aren’t limited to those with advanced degrees like PhDs or MDs. There are diverse roles and job opportunities accessible to individuals with various educational backgrounds and skill levels.
David Mendoza Bautista is a masters student intern at Novo Nordisk and a Cal Poly Bridges Trainee.
“Often [students] are first gen, [from] underrepresented groups, these are an amazing group of students who are getting their first opportunity both at the higher education and also the first in their families to do research in labs,” said Bridges Program mentor Samantha Butler, PhD.
At CIRM, diversity fosters innovation. In addition to academic and industry research opportunities, CIRM aims to grow the entire regenerative medicine workforce pipeline, including opportunities in therapeutics manufacturing and clinical trials.
To learn more about CIRM’s educational programs, including information on how to apply, visit our website at cirm.ca.gov/education.
CIRM thanks all program participants, program directors, and CIRM leadership who made these videos possible. Video produced by CIRM’s Scientific Programs & Education team and Hartlove-Goodyear.
Hataalii Tiisyatonii Begay (HT) participated in a CIRM-funded clinical trial at UCSF for Artemis-SCID, a condition that can be life-threatening or fatal. Photo courtesy Barbara Ries / UCSF.
The California Institute for Regenerative Medicine (CIRM) funds innovative cell and gene therapy research and educational programs to advance regenerative medicine in the Golden State. Just as important is CIRM’s commitment to ensuring that Californians from diverse backgrounds have access to groundbreaking clinical trials for a variety of diseases and condition resulting from the research.
That’s why CIRM has approved awarding $2.5 million to EVERSANA—a leading provider of global commercial services to the life sciences industry—to establish a Patient Support Program (PSP) to assist patients enrolled in CIRM-funded clinical trials.
Addressing Barriers to Clinical Trials
For many patients battling diseases and chronic health conditions, getting access to a clinical trial can be lifesaving, but it can also be very challenging. Clinical trial patients often face financial challenges, long-distance travels, and require family commitments that can make it difficult to maintain participation.
Through this award, CIRM and EVERSANA will address informational, financial and logistical bottlenecks experienced by clinical trial patients and their family members. The Patient Support Program will be particularly important for providing equal access to California clinical trial participants.
Evie Junior (left) participated in a CIRM-funded clinical trial at UCLA for sickle cell disease. Photo courtesy UCLA Broad Stem Cell Research Center.
Evidence shows that support programs positively impact clinical trial accrual rates, patient diversity, trial adherence, humanistic outcomes, reduced healthcare utilization costs, and quality of life.
Encouraging Trial Participation in Underserved Communities
Services offered by the PSP will include maintaining a Patient Support Center to refer patients to clinical trials, verifying participation and financial support eligibility, as well as administering Patient Assistance Fund (PAF) reimbursements to cover travel expenses, meals, accommodations, childcare, and other out of pocket expenses.
Funds for the PSP are set aside under Proposition 14, the voter-approved initiative that renewed CIRM’s funding in 2020.
Under Prop 14, royalty revenues that CIRM grantees earn from licensing, inventions or technologies are 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, CIRM has been appropriated $15.6 million from the Patient Assistance Fund to support patients.
“CIRM is committed to supporting patients through the clinical trial process to continue advancing transformative regenerative medicine therapies to the benefit of all Californians,” said Jonathan Thomas, PhD, JD, CIRM’s Interim President and CEO. “The Patient Support Program is just one initiative designed to get us a step closer to that vision.”
EVERSANA is expected to initiate work on the approved project plan within 120 days of the final contract.
Approximately 22,000 women are diagnosed with ovarian cancer—the most lethal gynecologic malignancy—each year in the US. At diagnosis, more than 70 percent of patients are already late stage with abdominal metastases, leading to a dismal 34 percent 5-year survival rate.
The standard of care includes aggressive chemotherapy, which often results in toxic side effects and the development of chemoresistance, underlining the need for safer, more effective treatment options to improve clinical outcomes for these patients.
This proposed treatment uses neural stem cells to target an oncolytic virus directly to abdominal ovarian tumor sites. The virus infects and kills the tumor cells, even if they are chemo resistant, which then stimulates the patient’s immune system to recognize, and fight the cancer.
If successful, this stem cell-delivered therapy can potentially lead to a more effective, less toxic treatment for Stage III ovarian cancer patients, improving survival and quality of life.
“The funding from CIRM enables us to complete the preclinical studies, product manufacturing, and clinical trial design needed to receive FDA approval to move this novel treatment to patients within 2-3 years,” said Dr. Aboody.
CIRM has previously supported Aboody and the City of Hope research team with an award for earlier-stage translational research.
Symptoms of lupus may include pain or swelling in the joints, extreme fatigue, a butterfly rash on the cheeks and nose, and more.
The California Institute for Regenerative Medicine (CIRM) has awarded $7.9 million to Barbara Hickingbottom, MD, of Fate Therapeutics to advance clinical research for FT819, an induced pluripotent stem cell (iPSC)-derived CD19 CAR T-cell therapy for Systemic Lupus Erythematosus (SLE).
SLE is a debilitating autoimmune disease and affects more than 200,000 Americans, particularly women of color.
FT819 targets B cells with the aim to reset the immune system and provide drug-free remission for patients with autoimmune diseases. Fate manufactures FT819 using a clonal master iPSC line as a renewal cell source, providing a uniform cell therapy product that is mass produced and delivered off-the-shelf to patients.
As a result, FT819 is designed to bring the curative potential of cell therapy to large numbers of patients with SLE and other autoimmune diseases.
“This innovative approach shows great promise in transforming clinical practice for Systemic Lupus Erythematosus, providing a new potential treatment option for individuals and families affected by this challenging disease,” added Dr. Creasey.
“CD19 CAR T cell therapy has demonstrated tremendous potential for patients with autoimmune diseases,” said Dr. Hickingbottom. “We look forward to partnering with CIRM to broadly realize this potential with FT819, the industry’s first CAR T-cell therapy manufactured from a clonal master iPSC line to reach clinical investigation.”
The California Institute for Regenerative Medicine (CIRM) has approved an $8 million award to Aspen Neuroscience to advance an investigational stem cell-derived dopaminergic neuron replacement therapy for Parkinson’s disease (PD).
This individualized potential therapy is being explored in a First in Human Phase 1/2a clinical trial for patients with moderate to advanced PD.
Affecting more than one million Americans, PD is a devastating neurodegenerative disorder that causes walking and motor problems, as well as impaired balance and coordination. Existing therapies alleviate symptoms but do not treat the disease, leading to a significant unmet medical need for those suffering from this chronic condition.
“We would like to thank CIRM for their support of this program to investigate an autologous cell therapy for Parkinson’s disease, which is a very personalized condition,” said Damien McDevitt, PhD, President and CEO of Aspen Neuroscience, Inc.
“This first-in-human trial is the culmination of many years of work by a remarkable team of researchers and clinicians. Our approach to provide patients with their own dopamine neuron cells has the potential to impact the entire field of neurodegenerative disorders.”
This clinical trial aims to evaluate the safety, tolerability, and preliminary efficacy of this one-time therapy that, if successful, would eliminate the need for daily medications.
“This clinical award represents a significant step forward in the treatment landscape of Parkinson’s disease by advancing individualized therapy that has the potential to restore motor function in those impacted by this devastating condition,” said Dr. Abla Creasey, PhD, Vice President of Therapeutics Development at CIRM.
The Stem Cellar 