UCLA researchers are making great strides in stem cell research. On the evening of October 22, they announced progress in the fight against a variety of serious medical conditions: cancer, autism, and sickle cell disease. They reported on the engineering of the immune system to recognize and attack deadly cancer cells, creating brain cells in the laboratory that model the genetic causes of autism to identify targeted treatment, modifying the genes in a patient’s blood cells to eliminate sickle cell disease., and creating brain cells in the laboratory.
Three leading UCLA scientists from the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research presented the new information to an overflow audience in the Tamkin Auditorium of the UCLA Ronald Reagan Medical Center. “Transforming Medicine: An Evening with UCLA Stem Cell Scientists” featured recent research conducted by Drs. Donald Kohn, Antoni Ribas, and Daniel Geschwind. They were joined with remarks by the Broad Stem Cell Research Center Director, Dr. Owen Witte; Dean of David Geffen School of Medicine, Dr. Eugene Washington; and Chairman of the California Institute for Regenerative Medicine (CIRM) Dr. Jonathan Thomas.
Dr. Ribas, director of the Tumor Immunology Program of UCLA’s Jonsson Comprehensive Cancer Center presented “Cancer: Engineering Immunity.” He discussed how his research team are using stem cell science to engineer the human immune system to identify and destroy cancer cells in patients with melanoma, which is the deadliest form of skin cancer. Clinical tests are scheduled to begin in about 18 months. The reason cancer cells multiply and invade structures is because the immune system does not recognize them as a foreign entity. Dr. Ribas and his colleagues are using stem cells to engineer T cells, which fight at the front lines of the immune, to recognize and attack cancer cells as they would any other disease invader. Dr. Ribas presented a video that showed the modified cells actually killing melanoma cells in the laboratory. He explained that his team had demonstrated the proof of principle for this process by modifying regular blood cells. During a clinical trial, the modified blood cells showed tremendous activity and effectiveness, but only over a few months. He observed that by genetically modifying the blood-forming stem cells, he hoped to generate a self-renewing population of immune cells that would destroy the cancer.
Autism expert, Dr. Geschwind, director of the UCLA Center for Autism Research and Treatment (CART) spoke on “Autism: Disease in a Dish.” He noted that the development of a treatment for a disease with possibly hundreds or thousands of implicated genes was a daunting task. His team is in the process of growing stem cells from skin or blood samples from patients with autism in order to generate brain cells (neurons) in the laboratory that have the genetic defects that cause autism. By duplicating the diseased cells in a laboratory dish, the team can study the causes and possible treatment targets, and identify potential effective drug therapies for this wide-spread and often devastating neurological disorder.
Dr. Kohn, director of the UCLA Human Gene Medicine Program, gave his presentation “Sickle Cell Disease: Gene Therapy.” In it, he outlined a new treatment strategy for sickle cell disease scheduled to begin clinical trial testing in patients next year. If proven safe and effective, Kohn’s strategy will essentially cure patients with a devastating genetic disease that affects 90,000 Americans, primarily African-Americans (1 in 500) and Hispanics (1 in 36,000). Dr. Kohn’s method was developed by a cross-town collaboration with Children’s Hospital Los Angeles and USC. It involves the use of blood stem cells taken from the bone marrow. These cells have the potential to develop into any type of blood cell. The researchers modify the stem cells in the laboratory to correct the genetic defect that causes the red blood cells to become misshapen and rigid like a sickle. The modified blood stem cells are then infused back into the patient; they then multiply and grow into normal red blood cells without the sickling defect. This new therapy has the potential to cure for sickle cell disease and eliminate the current treatment problems of finding matched sibling donors and the body’s possible rejection of donated bone marrow. The method to treat sickle cell disease grew out of a treatment Dr. Kohn successfully used to treat children with “Bubble Baby Disease”—a condition in which infants are born without a functioning immune system. He was able to correct the genetic defect so that the children can now live relatively normal lives.