The California Institute for Regenerative Medicine (CIRM) supports every stage of clinical trials to accelerate cures for people who need them most.
Holding a curved device that looked a bit like a mariner’s sextant, neurosurgeon Warren Boling explained how he’ll use it to precisely deliver a new gene therapy deep into the brains of Parkinson's patients participating in a pivotal clinical trial. A colony of iPSCs from a Parkinson's patient (left) and dopaminergic neurons made from these … Continue reading Three CIRM-funded approaches advance potential Parkinson’s treatments
From the age of three, Tara Radcliffe Ghiglieri danced almost every day, leaping, pirouetting, tapping, and high-kicking her way into a Broadway career that included a multi-year stint as a Radio City Rockette. She trained hard, at times rehearsing eight hours a day, six days a week. "And performance days could be up to six … Continue reading Experimental treatment brings relief for a dancer with chronic knee pain
By Holly Alyssa MacCormick A single infusion of a modified CAR-T cell therapy could pave the way for a potential one-shot cure for HIV, according to preliminary findings of a small, early-stage clinical trial. Steven G. Deeks, MD, the principal investigator of this California Institute for Regenerative Medicine (CIRM)-funded clinical trial, discussed the findings at a recent CIRM board meeting. Steven Deeks, MD Photo: UCSF Roughly 40 million people worldwide and more … Continue reading Early trial shows promise for one‑shot cure for HIV
Knee joint pain rarely gets counted among major public‑health burdens. Yet it affects more than 3 million Californians and 34 million people across the U.S., with both massive economic and health consequences. Costly Impact In California alone, the cost of knee replacement surgeries exceeds $8 billion annually, according to Thomas Chalberg, PhD, founder and CEO … Continue reading Genascence discusses gene therapy for knee osteoarthritis
Only three years ago, the FDA approved the first gene therapies for sickle cell disease (Lyfgenia and Casvegy), historic breakthroughs decades in the making. Yet on World Sickle Cell Awareness Day this year, the reality is that high cost, limited availability, and complex logistics keep these treatments out of reach for most people who need them. Yet these breakthroughs offer tremendous hope. For many years, the … Continue reading Progress on Sickle Cell Disease Treatments
To speed promising new stem cell or gene therapies to patients who need them, the California Institute for Regenerative Medicine (CIRM) supports work at every phase of clinical trials, from early discovery research through clinical testing. CIRM currently supports more than 100 clinical trials in California, conditions that range from rare diseases to cancers and … Continue reading Understanding the Phases of Clinical Trials: How Success Builds at Every Step
Image Credit: Canva - studioroman Often, after decades of intensive scientific research, even the most promising potential new therapies come down to a critical final phase: courageous people stepping forward to participate in a clinical trial. For Clinical Trials Day this year, we're celebrating a few of the many patients who inspire us, while highlighting … Continue reading What We Wish People Knew About Clinical Trials
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
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
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 LAD-1 is extremely rare—about one in a million births—but because … Continue reading CIRM funded clinical trial gives siblings a second chance at life