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). The odds of being born with LAD-1 are rare, literally one-in-a-million, but the disease is genetic, so the Langenhop’s unborn child had a roughly 25 … Continue reading CIRM-funded clinical trial gives siblings a second chance at life
Disease Areas
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, CEO and co-founder of Neurona Therapeutics, discusses progress in clinical trials for patients with epilepsy that does not respond to drugs.
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. It’s those patients who don’t respond to medications that Neurona Therapeutics is hoping … Continue reading Closer to Cures: Cory R. Nicholas, CEO and co-founder of Neurona Therapeutics, discusses progress in clinical trials for patients with epilepsy that does not respond to drugs.
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
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 could—one day—benefit many people living with genetic diseases
Written by Holly Alyssa MacCormick Image Credit: Audrey Davidow－Calvin 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 could—one day—benefit many people living with 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 Alzheimer’s Disease—she 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
Winner of Beast Games becomes champion for his son’s rare disease
Written by Holly Alyssa MacCormick You can do a lot with $10 million. Yet, when Jeffrey Randall Allen won the largest prize in game show history on Amazon Prime Video’s Beast Games there was only one thing he wanted to spend his $10 million on—research that might help find a cure for his son’s rare … Continue reading Winner of Beast Games becomes champion for his son’s rare disease
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
The Stem Cellar 