In her home on the island of Kauai with her husband Chris Constantino at her side and her two children playing nearby, Nichelle Obar breaks the illusion that nothing was ever amiss in her family's slice of paradise. "It all happened so fast," she says, explaining the cascade of news and life-and-death decisions she and … Continue reading Looking Back in Gratitude: One Family, One Trial, and Moving Science Forward
Clinical Trials
The California Institute for Regenerative Medicine (CIRM) supports every stage of clinical trials to accelerate cures for people who need them most.
How CIRM supports patients and their families in clinical trials
A year ago, Sam and his wife knew little about clinical trials, experimental therapies, or a rare, aggressive brain cancer known as diffuse midline glioma. Since their 6-year-old daughter was diagnosed a few months ago, they've become experts. "I wouldn't have known about any of it," said Sam. "It's so rare — maybe 300 cases … Continue reading How CIRM supports patients and their families in clinical trials
Featured
Three CIRM-funded approaches advance potential Parkinson’s treatments
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
Featured
Experimental treatment brings relief for a dancer with chronic knee pain
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
Early trial shows promise for one‑shot cure for HIV
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
Genascence discusses gene therapy for knee osteoarthritis
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
Progress on Sickle Cell Disease Treatments
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
Understanding the Phases of Clinical Trials: How Success Builds at Every Step
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
What We Wish People Knew About Clinical Trials
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
Closer to Cures: Diana Farmer discusses clinical trials for spina bifida
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