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
Disease Areas
First therapy supported by CIRM approved by the FDAย
Image Credit: Canva stock images After 21 years (and a few months), the California Institute of Regenerative Medicineย (CIRM)ย has hit a major milestone: the firstย therapyย supported by CIRMย has been approved by the Food and Drug Administrationย for use in patients. In 2004, whenย California voters created CIRMย to fundย research withย stem cellsย โ cells that can divide to form many different tissues … Continue reading First therapy supported by CIRM approved by the FDAย
CIRM-fundedย clinicalย trialย givesย siblingsย 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).ย Theย odds of being born with LAD-1ย areย rare,ย literally one-in-a-million,ย but the disease is genetic, soย theย Langenhopโsย unborn child had aย roughlyย 25 … Continue reading CIRM-fundedย clinicalย trialย givesย siblingsย a second chance at life
Closer to Cures: Stanford researcher Crystal Mackall discusses progress on clinical trials for rare cancers of the brain and spine with no known cure.
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 that arise from a specific genetic mutation, known as H3K27M. These cancers are devastating. Each year, about 1,000 people … Continue reading Closer to Cures: Stanford researcher Crystal Mackall discusses progress on clinical trials for rare cancers of the brain and spine with no known cure.
Closer to Cures: 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 adultsย living in theย United States have epilepsy, and about 400,000 of these peopleย liveย inย California, according to the CDC.ย Of these people,ย aboutย one-third toย halfย continue to have seizures that do notย respondย to medications on theย market. Itโsย those patients whoย donโtย respond to medications thatย Neuronaย Therapeutics is hoping … Continue reading Closer to Cures: 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 โ 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
New program to help patients with rare disease
Recently, the California Institute for Regenerative Medicine (CIRM) announced a new approach for how its funding can advance treatments for people living with rare diseases. Individually, few people have any single rare disease, but in aggregate, about 30 million people in the U.S. have one of the roughly 10,000 rare diseases, and 95% have no … Continue reading New program to help patients with rare disease
Early-stage research couldโone dayโbenefit many people living with ย genetic diseases
Written by Holly Alyssa MacCormick Image Credit: Audrey Davidow๏ผCalvin outside Wanting the best for your child is a universal experience, and Audrey Davidow wanted her baby to have a bright future full of possibility. But in March of 2012, her 13-month-old son Calvin was diagnosed with a rare disease called Pitt Hopkins Syndrome, and she … Continue reading Early-stage research couldโone dayโbenefit many people living with ย genetic diseases
A conversation with CIRM board member Yael Wyte
Written by Holly Alyssa MacCormick Yael Wyte knows all too well how difficult it can be to have or care for someone with Alzheimerโs Diseaseโshe has been working with and advocating for people with Alzheimer's Disease for more than 20 years. Earlier this year, Wyte joined the board of the California Institute for Regenerative Medicine … Continue reading A conversation with CIRM board member Yael Wyte
Fueling the Brain: Exploring the connection between metabolism and neurodevelopment
Top left to right: Aparna Bhaduri, PhD; Heather Christofk, PhD; Harley Kornblum, PhD; William Zeiger, MD, PhDBottom Left to right: Elizabeth Crouch, PhD; Mohammed Mostajo-Radji, PhD; Carrie Beardon, PhD; Michael Zito, MD As part of our goal of accelerating discoveries and therapies for central nervous system disorders, in 2023, the California Institute for Regenerative Medicine … Continue reading Fueling the Brain: Exploring the connection between metabolism and neurodevelopment