CIRM invests $26 million in clinical-stage research, including Phase 2b trial for bipolar depression
FOR IMMEDIATE RELEASE
Contact:
Koren Temple-Perry
Sr. Director, Marketing & Communications
press@cirm.ca.gov
South San Francisco, CA, Jan. 29, 2024 – The California Institute for Regenerative Medicine (CIRM), the world’s largest institution dedicated to regenerative medicine, awarded almost $26 million to fund various clinical research — from treating bipolar depression to advancing a therapy for spinal cord injury.
The awards will support four 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 a concept proposal for the Community Care Centers of Excellence (CCCE), a new model for increasing clinical trial access to diverse communities throughout California.
The clinical awards approved at the CIRM January Independent Citizens’ Oversight Committee (ICOC) meeting include:
| Application #
|
Program Title
|
Principal Investigator/Institution | Amount
|
| CLIN1-14840 | Prevention of GvHD in patients receiving HLA mismatched related or unrelated allogeneic HSCT for the treatment of hematologic malignancies | de Vries, David – Tr1X Inc | $4,000,000 |
| CLIN2-15085 | Personalized antisense oligonucleotide therapy for rare pediatric genetic disease: SCN2A |
Kim-McManus, Olivia – UCSD | $985,713 |
| CLIN2-15395 | A Phase 2b Study of the Efficacy of a Novel Pro-Neurogenesis/Pro-Plasticity Drug for Bipolar Depression Using a Precision Psychiatry Approach | Etkin, Amit – Alto Neuroscience | $15,000,000 |
| CLIN1-15450 | Human Embryonic Stem Cell-Derived Neural Stem Cells for Severe Spinal Cord Injury (SCI) | Tuszynski, Mark – UCSD | $6,000,000 |
Improving the Treatment Landscape for Bipolar Depression
A $15 million award to Alto Neuroscience and Amit Etkin, MD, PhD, President and CEO of Alto Neuroscience will advance a Phase 2b trial to test a novel therapeutic treatment for bipolar depression (I or II), a condition that impacts 4.4% of U.S. adults at some point in their lives.
The drug is designed to act on neural progenitor cells by enhancing the brain’s ability to create new neurons and improve connections in the hippocampus, a specific region of the brain implicated in bipolar depression.
Bipolar depression is a severe, life-long psychiatric condition associated with a significant burden of illness and risk of suicide.
“Given the prevalence of bipolar depression and the high unmet medical needs of those facing this challenging disorder, this potential new therapy could expand treatment options to the hundreds of thousands of Californians impacted by this chronic neuropsychiatric disease,” said Abla Creasey, PhD, Vice President of Therapeutic Development at CIRM.
Unlike current treatment options which consist of a regimen of antipsychotics and mood-stabilizing medications, this small molecule drug has the potential to offer better tolerability, and includes a diagnostic approach designed to identify patients who are most likely to benefit.
Using Stem Cells to Restore Function after Spinal Cord Injury
Another project added to CIRM’s clinical program includes a $6 million award to Mark Tuszynski, MD, PhD, from the University of California San Diego (UCSD) to explore the use of human embryonic stem cells to treat severe spinal cord injury (SCI).
The proposed therapy works by implanting neural stem cells at the injury site to regenerate and repair damaged axons associated with SCI.
SCI impacts more than half a million Americans and is associated with significant morbidity, including long-term and severe disability, chronic neuropathic pain as well as high healthcare costs.
“The potential therapy represents a unique and forward-looking approach in the treatment landscape of severe spinal cord injury and underscores CIRM’s commitment to advancing groundbreaking solutions to transform patients’ lives,” said Dr. Creasey.
Improving Access to Treatments Across California
Additionally, CIRM approved a plan for the Community Care Centers of Excellence (CCCEs). The program is projected to award up to $60 million in CCCE awards.
The California Stem Cell Research, Treatments, and Cures Initiative of 2020 (Proposition 14) mandates the establishment of the CCCE Program to promote access across the state to clinical trials, treatments, and therapies arising from CIRM-funded research.
The CCCEs are designed to serve as a hub for expanding access to clinical trials, regenerative medicine treatments, and CIRM education and training programs. The program will seek to enlist community-based partnerships to engage populations that can benefit from these treatments.
A strategic aim is to provide equitable access to communities and populations that would otherwise have more limited opportunities in the absence of these centers.
About the California Institute for Regenerative Medicine (CIRM)
At CIRM, we never forget that we were created by the people of California to accelerate stem cell treatments to patients with unmet medical needs, and act with a sense of urgency to succeed in that mission. To meet this challenge, our team of highly trained and experienced professionals actively partners with both academia and industry in a hands-on, entrepreneurial environment to fast track the development of today’s most promising stem cell technologies. With $5.5 billion in funding and more than 150 active stem cell programs in our portfolio, CIRM is one of the world’s largest institutions dedicated to helping people by bringing the future of cellular medicine closer to reality.
For more information go to www.cirm.ca.gov