Photo Courtesy of Andrea Fernandez de Soto Imagine a life where every time you step outside, everything in your environment poses a life-threatening risk. For children born with ADA-SCID (adenosine deaminase severe combined immunodeficiency), this is a reality. Their immune systems are so compromised that a common cold or a simple scrape on the knee … Continue reading Gene Therapy Brings Hope to Families Affected by Rare Immune Disorder
Blood stem cells
Stem Cell Awareness: What are stem cells and why research is important
Have you heard of stem cells but don't know what they are or why they are essential in medical research? In celebration of Stem Cell Awareness Day, the second Wednesday in October that is dedicated to educating about the importance of stem cells, the California Institute for Regenerative Medicine (CIRM) is providing this guide on … Continue reading Stem Cell Awareness: What are stem cells and why research is important
CIRM awards $8 million to Support Groundbreaking Gene Therapy for AML and MDS
Image source: Canva Around 20,000 Americans are affected by acute myeloid leukemia (AML) each year. AML is a type of cancer that originates in the bone marrow and rapidly enters the bloodstream. Additionally, many individuals are impacted by myelodysplastic syndromes (MDS), which are blood disorders that result in the improper formation or functioning of blood … Continue reading CIRM awards $8 million to Support Groundbreaking Gene Therapy for AML and MDS
Tackling sickle cell disease from within
Photo Credit: Jaquell Chandler Evie Junior was born with sickle cell disease, a life-threatening condition that affects around 100,000 Americans, most of them Black and Latino. People with sickle cell disease have blood cells that are shaped like a hook (or sickle) rather than smooth and round, which can create clogs, causing intense pain, organ … Continue reading Tackling sickle cell disease from within
Funding development of a vaccine for acute myelogenous leukemia (AML)
Dr. Karin Gaensler. Photo credit: Steve Babuljak/UCSF Adult acute myelogenous leukemia—also known as acute myeloid leukemia (AML)—is a blood cancer in which the bone marrow makes a large number of abnormal blood cells. About 20,000 new cases of AML are diagnosed each year in the US with a 5-year survival rate of around 29%. In … Continue reading Funding development of a vaccine for acute myelogenous leukemia (AML)
Bubble baby treatment cleared to restart clinical trial
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
Making transplants easier for kids, and charting a new approach to fighting solid tumors.
Every year California performs around 100 kidney transplants in children but, on average, around 50 of these patients will have their body reject the transplant. These children then have to undergo regular dialysis while waiting for a new organ. Even the successful transplants require a lifetime of immunosuppression medications. These medications can prevent rejection but … Continue reading Making transplants easier for kids, and charting a new approach to fighting solid tumors.
Sweet 16 and counting for stem cell clinical trial
Dr. Judy Shizuru: Photo courtesy Jasper Therapeutics Over the years the California Institute for Regenerative Medicine (CIRM) has invested a lot in helping children born with severe combined immunodeficiency (SCID), a fatal immune disorder. And we have seen great results with some researchers reporting a 95 percent success rate in curing these children. Now there’s … Continue reading Sweet 16 and counting for stem cell clinical trial
Developing a natural killer for cancer
Lili Yang UCLA Broad Stem Cell Research Center: Photo courtesy Reed Hutchinson PhotoGraphics When Lili Yang was studying for her PhD she approached her mentor, the Nobel Laureate Dr. David Baltimore, and told him she was thinking about writing her thesis on a combination of gene therapy, immunotherapy and stem cell therapy. She says he … Continue reading Developing a natural killer for cancer
Pioneering a new approach to HIV/AIDS
Dr. Steven Deeks. Photo courtesy UCSF I’ve always been impressed by the willingness of individuals to step forward and volunteer for a clinical trial. Even more so when they are the first person ever to test a first-in-human therapy. They really are pioneers in helping advance a whole new approach to treating disease. That’s certainly … Continue reading Pioneering a new approach to HIV/AIDS
The Stem Cellar 