Severe Leukocyte Adhesion Deficiency-1 (LAD-1) is a rare condition that causes the immune system to malfunction and reduces its ability to fight off viruses and bacteria. Over time the repeated infections can take a heavy toll on the body and dramatically shorten a person’s life. But now a therapy, developed by Rocket Pharmaceuticals, is showing … Continue reading Promising results from CIRM-funded projects
Immune Disease
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
Two CIRM supported studies highlighted in Nature as promising approaches for blood disorders
Blood stem cells (blue) are cleared from the bone marrow (purple) before new stem cells can be transplanted.Credit: Dennis Kunkel Microscopy/SPL Problems with blood stem cells, a type of stem cell in your bone marrow that gives rise to various kinds of blood cells, can sometimes result in blood cancer as well as genetic and … Continue reading Two CIRM supported studies highlighted in Nature as promising approaches for blood disorders
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 challenges of living with IPEX
Last week the CIRM Board awarded $5.53 million to Dr. Rosa Bacchetta at Stanford to complete the work necessary to conduct a clinical trial for IPEX syndrome. This is a rare disease caused by mutations in the FOXP3 gene which leaves people with the condition vulnerable to immune system attacks on their organs and tissues. … Continue reading The challenges of living with IPEX
One family’s fight to save their son’s life, and how stem cells made it possible
CIRM's mission is very simple: to accelerate stem cell treatments to patients with unmet medical needs. Anne Klein's son, Everett, was a poster boy for that statement. Born with a fatal immune disorder Everett faced a bleak future. But Anne and husband Brian were not about to give up. The following story is one Anne … Continue reading One family’s fight to save their son’s life, and how stem cells made it possible
Video: Behind the scenes of a life-saving gene therapy stem cell treatment
“We were so desperate. When we heard about this treatment were willing to do anything to come here.” In the above quote from Zahraa El Kerdi, “here” refers to UCLA, a world away from her hometown in Lebanon. In September 2015, Zahree gave birth to a son, Hussein, who appeared perfectly healthy. But by six … Continue reading Video: Behind the scenes of a life-saving gene therapy stem cell treatment
The story behind the book about the Stem Cell Agency
WHY I WROTE “CALIFORNIA CURES” By Don C. Reed It was Wednesday, June 13th, 2018, the launch day for my new book, “CALIFORNIA CURES: How the California Stem Cell Research Program is Fighting Your Incurable Disease!” As I stood in front of the audience of scientists, CIRM staff members, patient advocates, I thought to myself, … Continue reading The story behind the book about the Stem Cell Agency
SCID kid scores big on TV
One of the stories I never tire of telling is about Evie Vaccaro. She’s the little girl who was born with a fatal immune condition called severe combined immunodeficiency or SCID. Children with this condition have no immune system, no protection against infections, and often die in the first two years of life. But thanks … Continue reading SCID kid scores big on TV