Evie Vaccaro: Photo courtesy Nancy Ramos Three families battling a life-threatening immune disorder got some great news last week. A clinical trial that could save the life of their child has once again been given the go-ahead by the US Food and Drug Administration (FDA). The clinical trial is the work of UCLA’s Dr. Don … Continue reading Bubble baby treatment cleared to restart clinical trial
Orchard Therapeutics Ltd.
New Study Shows CIRM-Supported Therapy Cures More than 95% of Children Born with a Fatal Immune Disorder
Dr. Donald B. Kohn; Photo courtesy UCLA A study published in the New England Journal of Medicine shows that an experimental form of stem cell and gene therapy has cured 48 of 50 children born with a deadly condition called ADA-SCID. Children with ADA-SCID, (severe combined immunodeficiency due to adenosine deaminase deficiency) lack a key … Continue reading New Study Shows CIRM-Supported Therapy Cures More than 95% of Children Born with a Fatal Immune Disorder
Building a progressive pipeline
Dr. Kelly Shepard By Dr. Kelly Shepard One of our favorite things to do at CIRM is deliver exciting news about CIRM projects. This usually entails discussion of recent discoveries that made headlines, or announcing the launch of a new CIRM-funded clinical trial …. tangible signs of progress towards addressing unmet medical needs through advances … Continue reading Building a progressive pipeline
Meet the people who are changing the future
Kristin MacDonald Every so often you hear a story and your first reaction is “oh, I have to share this with someone, anyone, everyone.” That’s what happened to me the other day. I was talking with Kristin MacDonald, an amazing woman, a fierce patient advocate and someone who took part in a CIRM-funded clinical trial … Continue reading Meet the people who are changing the future
Perseverance: from theory to therapy. Our story over the last year – and a half
Some of the stars of our Annual Report It’s been a long time coming. Eighteen months to be precise. Which is a peculiarly long time for an Annual Report. The world is certainly a very different place today than when we started, and yet our core mission hasn’t changed at all, except to spring into … Continue reading Perseverance: from theory to therapy. Our story over the last year – and a half
Celebrating a life that almost didn’t happen
Evie Vaccaro You can’t look at this photo and not smile. This is Evie Vaccaro, and it’s clear she is just bursting with energy and vitality. Sometimes it feels like I have known Evie all her life. In a way I have. And I feel so fortunate to have done so, and that’s why this … Continue reading Celebrating a life that almost didn’t happen
CIRM Board Meeting Highlights Important Updates to Clinical Trials
Dr. Maria T. Millan, President and CEO of CIRM, presenting the President's Report This past Thursday the governing Board of the California Institute for Regenerative Medicine (CIRM) were presented with an update on CIRM’s clinical portfolio, which to date includes 60 clinical trials in various areas including kidney failure, cancer, and other rare diseases. The … Continue reading CIRM Board Meeting Highlights Important Updates to Clinical Trials
Stem Cell/Gene Therapy combo heals patients battling rare disorder
Brenden Whittaker and his dog: Photo by Colin McGuire A few years ago, Brenden Whittaker was running out of time. Brenden was born with a rare condition called x-linked chronic granulomatous disease or XCGD. It meant he lacked a critical part of his immune system that protects against bacterial or fungal infections. Over 22 years … Continue reading Stem Cell/Gene Therapy combo heals patients battling rare disorder
Facebook Live: Ask the Stem Cell Team
On December 12th we hosted our latest 'Facebook Live: Ask the Stem Cell Team' event. This time around we really did mean team. We had a host of our Science Officers answering questions from friends and supporters of CIRM. We got a lot of questions and didn't have enough time to address them all. So … Continue reading Facebook Live: Ask the Stem Cell Team
Good news for two CIRM-supported therapies
Jake Javier, a patient in the spinal cord injury stem cell therapy clinical trial It’s always satisfying to see two projects you have supported for a long time do well. That’s particularly true when the projects in question are targeting conditions that have no other effective therapies. This week we learned that a clinical trial … Continue reading Good news for two CIRM-supported therapies
The Stem Cellar 