written by Holly Alyssa MacCormick The California Institute for Regenerative Medicine (CIRM) helped fund a clinical trial that involved an experimental stem cell treatment for a frequently paralyzing form of spina bifida. Today, several of those babies are now able to walk and run unassisted.Babies with severe spina bifida are often treated with surgery after … Continue reading Closer to Cures: Diana Farmer discusses clinical trials for spina bifida
Clinical Trials
First therapy supported by CIRM approved by the FDA
Image Credit: Canva stock images After 21 years (and a few months), the California Institute of Regenerative Medicine (CIRM) has hit a major milestone with its FDA approved therapy. The promise of stem cells In 2004, when California voters created CIRM to fund research with stem cells, which can divide to form many different tissues in the body. Stem cells held great potential, but scientists … Continue reading First therapy supported by CIRM approved by the FDA
CIRM funded clinical trial gives siblings a second chance at life
written by Holly Alyssa MacCormick Image Credit: The Langenhop Family Alicia Langenhop was seven months pregnant with her third child when she and her husband, Jon, learned that their two toddlers had a life-threatening immune disease called leukocyte adhesion deficiency-1 (LAD-1). One in a million The odds of being born with LAD-1 are rare, literally one-in-a-million, but the disease is … Continue reading CIRM funded clinical trial gives siblings a second chance at life
Stanford researcher Crystal Mackall discusses progress on rare cancers clinical trials
Written by Holly Alyssa MacCormick In 2023, the California Institute for Regenerative Medicine (CIRM) funded a clinical trial led by Stanford researcher Crystal Mackall, MD, to investigate a possible therapy for rare brain and spinal tumors. Devastating cancers These cancers, that arise from a specific genetic mutation, known as H3K27M, are devastating. Each year, about … Continue reading Stanford researcher Crystal Mackall discusses progress on rare cancers clinical trials
Cory R. Nicholas shares update on epilepsy clinical trials
Written by Holly Alyssa MacCormick Cory R. Nicholas presenting at the January 29 ICOC board meeting Roughly 3 million adults living in the United States have epilepsy, and about 400,000 of these people live in California, according to the CDC. Of these people, about one-third to half continue to have seizures that do not respond to medications on the market. Hope for those with epilepsy It’s those patients who don’t respond … Continue reading Cory R. Nicholas shares update on epilepsy clinical trials
CIRM board supports a rare disease funding plan, administrative changes
Written by Holly Alyssa MacCormick South San Francisco, CA, January 30, 2026 – As many as 1 in 10 people in the U.S. has a rare disease, many of which have no known cure. A new program by the California Institute for Regenerative Medicine (CIRM) could change this by funding novel ways to develop multiple … Continue reading CIRM board supports a rare disease funding plan, administrative changes
Neurona Doses First Patient in CIRM-Funded Bilateral Epilepsy Cell Therapy Trial
Image Credit: Canva Stock Photo Neurona Therapeutics has reached a major milestone in the development of its stem cell approach to treating epilepsy. The company recently announced that the first patient has been dosed in a Phase 1/2 clinical trial evaluating NRTX-1001, a cell therapy for adults whose epilepsy affects both sides of the brain … Continue reading Neurona Doses First Patient in CIRM-Funded Bilateral Epilepsy Cell Therapy Trial
Gene Therapy for Osteoarthritis Shows Positive Results in Early Trial
Image Credit: Shutterstock Here’s a shocking statistic: in 2020, 7.6% of the world’s population was living with the most common form of arthritis – osteoarthritis (OA). That amounts to 595 million people globally and 32.5 million adults in the U.S. That’s a lot of people with painful knees, hips, and other joints. Although pain medication … Continue reading Gene Therapy for Osteoarthritis Shows Positive Results in Early Trial
20 Years of CIRM: The evolution of a CIRM-funded therapy for macular degeneration
Image Credit: University of Southern California (USC) Eye Institute In celebration of CIRM’s 20th anniversary, we are reflecting on some of the early projects we supported that have since advanced to clinical trials. One such trial for a form of blindness originated with a 2007 SEED grant to David Hinton, MD, of the University of … Continue reading 20 Years of CIRM: The evolution of a CIRM-funded therapy for macular degeneration
New gene therapy aims to restore function in early-onset Parkinson’s
Image source: Shutterstock Nearly one million people in the U.S. are living with Parkinson’s disease, which causes progressive symptoms including tremors, stiffness, and gait and balance issues. Although there are medications to treat symptoms, the disease has no cure. To date, the California Institute for Regenerative Medicine (CIRM) has invested more than $109 million in … Continue reading New gene therapy aims to restore function in early-onset Parkinson’s