"Transforming Medicine: An Evening with UCLA Stem Cell Scientists"

Crowd at the program

In recognition of “Stem Cell Day” the UCLA Broad Stem Cell Research Center hosted a unique evening sharing our developing therapeutic applications of stem cell therapies with the community. Drs. Antoni Ribas, Daniel Geschwind and Donald Kohn announced progress in the fight against a variety of serious medical conditions for cancer, autism spectrum disorder and sickle cell disease, respectively. They were joined by Center Director, Dr. Owen Witte who provided welcome remarks and an overview of the Center’s research activities and future directions; Dean of the David Geffen School of Medicine at UCLA, Dr. Eugene Washington who noted the importance of collaborative stem cell science as the basis for future treatments and cures; and Chairman of the California Institute for Regenerative Medicine (CIRM) Dr. Jonathan Thomas who applauded UCLA for being the first awardee institution to convene such a meeting to inform the public about stem cell research.

Engineering the Immune System
Dr. Ribas shared how his research team is using stem cells to engineer the human immune system to identify and destroy cancer cells in patients with melanoma. Theultimate goal of his work is to generate a self-renewing population of immune cells that destroy cancer in patients. To effectively demonstrate what these cells do, Dr. Ribas presented a video that showed the modified cells actually killing melanoma cells in a petri dish.

Creating a Model System for Autism
Dr. Geschwind, Director of the UCLA Center for Autism Research and Treatment (CART) discussed his efforts to observe the progression of Autism in a petri dish. Utilizing the most advanced technologies available; Dr. Geschwind’s team is reprogramming skin or blood samples from patients with autism into embryonic-like stem cells and then pushing them to become brain cells with the genetic defects that cause autism. This process enables his team to study the progression of disease in hopes of identifying a possible cause as well as potential treatments for this wide-spread neurological disorder.

Genetic Disorders of the Blood
Dr. Kohn shared with the audience a new treatment protocol for sickle cell disease scheduled to begin a Phase I clinical trial next year. If proven safe and effective, Kohn’s strategy will essentially cure patients with this devastating genetic disease that affects 90,000 Americans, primarily African-Americans and Hispanics. Through his work, Dr. Kohn repairs a patient’s stem cells to correct the genetic defect that causes their red blood cells to become misshapen and rigid. The modified blood stem cells are then infused back into the patient; multiplying and growing into normal red blood cells without the sickling defect. This new therapy has the potential to cure sickle cell disease and can be expanded to treat AIDS/HIV and potentially Hepatitis.

We look forward to future opportunities to share with the public the fascinating work underway at the UCLA Broad Stem Cell Research Center. Please feel free to suggest topics of interest and to share your thoughts on the event.