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). Evie Vaccaro's Story The clinical trial is the work of … Continue reading Bubble baby treatment cleared to restart clinical trial
Dr. Don Kohn
CIRM invests in therapies that use killer cells to target cancers
While there have been some encouraging advances in treating cancer in recent decades, there are still many cancers that either resist treatment or recur after treatment. Today the governing Board of the California Institute for Regenerative Medicine (CIRM) approved investing in a therapy targeting some of these hard-to-treat tumors. BioEclipse Therapeutics Inc. was awarded nearly … Continue reading CIRM invests in therapies that use killer cells to target cancers
UCLA gene therapy for LAD-1
Photo courtesy of Tamara Hogue/UCLA Broad Stem Cell Research Center Leukocyte adhesion deficiency type 1 (LAD‑1) is a rare pediatric disorder that cripples the immune system. Children experience recurrent, severe bacterial and fungal infections and struggle to heal wounds. The disease results from a missing protein that normally allows white blood cells to leave blood … Continue reading UCLA gene therapy for LAD-1
Good news on two CIRM-funded research on blood disorders
Dr. Rosa Bacchetta on the right with Brian Lookofsky (left) and Taylor Lookofsky after CIRM funded Dr. Bacchetta's work in October 2019. Taylor has IPEX syndrome It’s always lovely to end the week on a bright note and that’s certainly the case this week, thanks to some encouraging news about two CIRM-funded research on blood … Continue reading Good news on two CIRM-funded research on blood disorders
First patient in CIRM funded X-CGD trial gives back by working in patient care
Brenden Whittaker Brenden Whittaker was born with a rare genetic disorder called X-linked chronic granulomatous disease (X-CGD). This condition affects the immune system's ability to fight off common germs, specifically bacteria and fungi, and can result in infections that would only be mild for healthy people. Unfortunately for Brenden, he has suffered life-threatening infections that … Continue reading First patient in CIRM funded X-CGD trial gives back by working in patient care
The treatment that saved Evie Vaccaro
Evie Vaccaro You can see in this photo that Evie Vaccaro is full of energy and life. It sometimes feels as if I have known her forever. In a way, I have. I feel lucky for that, and this photo is powerful because her life nearly ended before it began. Evie was born with severe … Continue reading The treatment that saved Evie Vaccaro
Progress to a cure for Bubble Baby Disease
Evie, a former "bubble baby" enjoying life by playing inside a giant plastic bubble 'Bubble baby disease' will one day be a thing of the past. That’s a bold statement, but I say it with confidence because of the recent advancements in stem cell gene therapies that are curing infants of this life-threatening immune disease. … Continue reading Progress to a cure for Bubble Baby Disease
Stem cell agency funds clinical trials in three life-threatening conditions
A year ago the CIRM Board unanimously approved a new Strategic Plan for the stem cell agency. In the plan are some rather ambitious goals, including funding ten new clinical trials in 2016. For much of the last year that has looked very ambitious indeed. But today the Board took a big step towards reaching … Continue reading Stem cell agency funds clinical trials in three life-threatening conditions
A look at 2014: some of the lowlights of stem cell research this past year
It’s been quite a year in stem cell research. Here at the stem cell agency eight projects that we are funding have been approved for clinical trials and several more hope to get approval in early 2015. And Dr. Don Kohn and his team at UCLA announced that they have effectively cured Severe Combined Immunodeficiency … Continue reading A look at 2014: some of the lowlights of stem cell research this past year