CIRM awards more than $56 Million to advance clinical research, including a novel therapy designed to treat Parkinson’s
FOR IMMEDIATE RELEASE
Contact:
Koren Temple-Perry
Sr. Director, Marketing & Communications
press@cirm.ca.gov
South San Francisco, CA, Feb. 23, 2024 – The California Institute for Regenerative Medicine (CIRM), the world’s largest institution dedicated to regenerative medicine, awarded $56 million to fund various clinical research aimed at treating Parkinson’s disease, autoimmune conditions and a variety of cancers.
The awards will support seven projects in the Agency’s clinical program which provides funding for eligible stem cell and gene therapy-based projects through all stages of clinical trial development.
In addition, CIRM approved awarding $21.3 million to create a network of Shared Resources Laboratories (SRLs) for Stem Cell-Based Modeling, an important component of CIRM’s Infrastructure Program.
The clinical awards approved at the CIRM January Independent Citizens’ Oversight Committee (ICOC) meeting include:
| Application #
|
Program Title
|
Principal Investigator/Institution | Amount
|
| CLIN1-14845 | Neural stem cell delivered oncolytic viro-immunotherapy for ovarian cancer | Aboody, Karen – City of Hope | $5,314,547 |
| CLIN1-15337 | Chimeric TGFB Signaling Receptor (CTSR) Enabled Anti-B7H3 CAR T-cell Therapy in Children and Adolescents and Young Adults (AYA) with Recurrent Solid Tumors | Asgharzadeh, Shahab – Children’s Hospital Los Angeles | $6,000,000 |
| CLIN2-14801 | Stem-Derived IL13Ra2 Chimeric Antigen Receptor T cells for Patients with Melanoma and Advanced Solid Tumors | Kalbasi, Anusha – Stanford | $10,211,085 |
| CLIN2-15547 | Phase 1/2a Dose Escalation Study of Autologous Neuron Replacement in Sporadic Parkinson’s Disease (PD) | Wirth, Edward – Aspen Neuroscience | $8,000,000 |
| CLIN2-15562 | Phase 1 Study of Autologous E-SYNC T Cells in Adult Participants with EGFRvIII+ Glioblastoma | Okada, Hideho – UCSF | $10,927,618 |
| CLIN2-16063 | A phase 1/2 study to evaluate a bi-specific CD19/CD20-directed CAR T-cell, in refractory lupus nephritis (LN) and systemic lupus erythematosus (SLE) | Benjamin, Jonathan – ImmPACT-Bio, Inc. | $8,000,000 |
| CLIN2-16303 | A Phase 1 Study in Participants with Moderate to Severe Active Systemic Lupus Erythematosus | Hickingbottom, Barbara – Fate Therapeutics | $7,934,448 |
An Individualized Treatment for Parkinson’s
An $8 million award to Aspen Neuroscience will 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.
Off-the-Shelf CD19 CAR T for Patients with Autoimmune Disease
Through a $7.9 million award to Barbara Hickingbottom, MD, of Fate Therapeutics, CIRM and Fate will 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.”
Identifying Potential New Treatment Options for Ovarian Cancer
Another project added to CIRM’s clinical program includes a $5.3 million award to Karen Aboody, MD of City of Hope for late-stage preclinical research to develop a neural stem cell mediated treatment for a chemo-resistant, metastatic ovarian cancer.
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 outcome 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.
Fostering Collaboration Through Shared Labs
The CIRM Board also awarded $21.3 million to create a network of Shared Resources Laboratories (SRLs) for Stem Cell-Based Modeling. The SRLs are an important component of CIRM’s Infrastructure Program, which is designed to address the challenges that hinder progress in the regenerative medicine field.
The approved awards include:
| Application | Program Title | Program Director – Institution | Amount |
| INFR6.1-15357 | Stem Cell-based Disease Modeling Shared Resource Laboratory | Carlesso, Nadia – City of Hope | $5,400,000 |
| INFR6.2-15383 | A modular automation approach to stem cell modeling to increase throughput, reproducibility and access | Plath, Kathrin – UCLA | $3,999,999 |
| INFR6.2-15368 | Shared Resources Laboratories to Enhance In Vitro Stem Cell Modeling and Training | Walsh, Craig – UC Irvine | $4,000,000 |
| INFR6.2-15527 | A Center for Stem Cell Disease Modeling and Therapeutics | Conklin, Bruce – Gladstone Institutes | $4,000,000 |
| INFR6.2-15400 | 0 CIRM ASCEND Center – Advancing Stem Cell Education and Novel Discoveries | Lindstrom, Nils – USC | $3,946,795 |
The CIRM Board also recommended that 11 applicants may revise and resubmit their applications for funding approval.
The goal of the SRLs is to foster collaboration among California researchers, break down research silos, and provide students and researchers access to top-notch resources and training in using stem cell-based models to accelerate world class science. These stem cell-based models include various cell types of the brain, cardiovascular system, and other organ systems, and will help advance knowledge of human diseases to identify potential therapies, biomarkers, and drug candidates.
The SRL network will also support use of state-of-the-art technologies including CRISPR, omics analyses, and automation workflows.
In a previous Infrastructure Program in 2007, CIRM awarded more than $50 million to finance construction of shared research laboratories at 17 academic and non-profit institutions, which dedicated lab space for research using human embryonic stem cells (hESCs). At the time, hESC research was still in its infancy and federal policy prohibited research involving hESCs from being conducted in laboratories constructed with any federal funding.
“By investing in shared resources laboratories, we are not only providing essential infrastructure for stem cell research but also positioning California at the forefront of this transformative research. These new SRL awards showcase the remarkable progress in stem cell research and highlight CIRM’s pivotal role in propelling stem cell research forward,” said Dr. Rosa Canet-Aviles, VP of Scientific Programs and Education at CIRM. “Through these awards, CIRM will continue to drive progress, now focusing on cutting-edge disease modeling using human stem cells.”