UCLA stem cell scientists showcase potential autism, sickle cell, and cancer treatments and bright future

Three leading UCLA scientists from the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research presented new information Tuesday night to an overflow audience about potential clinical breakthroughs for autism, sickle cell disease, and cancer. 

The public event,Transforming Medicine: An Evening with UCLA Stem Cell Scientists, held in the Tamkin Auditorium of the UCLA Ronald Reagan Medical Center,highlighted the work of Drs. Donald Kohn, Antoni Ribas, and Daniel Geschwind who spoke about their contributions to the current revolution in stem cell science. 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. 

In his talk, Sickle Cell Disease: Gene Therapy, Dr. Donald Kohn, director of the UCLA Human Gene Medicine Program, outlined a new treatment strategy for sickle cell disease slated to begin clinical trial testing in patients next year. If proven safe and effective, Kohn’s strategy will essentially cure patients with a horrible genetic disease that affects 90,000 people in this country with a disproportionate impact on African-Americans (1 in 500) and Hispanics (1 in 36,000). 

Dr. Kohn’s method, growing from a cross-town collaboration with Children’s Hospital Los Angeles, and USC, uses blood stem cells, which grow into all the different types of blood cells, taken from the patient’s marrow. Kohn’s group, supported by a CIRM grant, modifies 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, where they replicate and grow into normal red blood cells without the dangerous sickling defect. This exciting therapy could be a 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.

Kohn’s method to treat sickle cell disease grew out of a treatment he successfully used to treat children with “Bubble Baby Disease” 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.

Dr. Antoni Ribas, director of the Tumor Immunology Program of UCLA’s Jonsson Comprehensive Cancer Center presented Cancer: Engineering Immunity, in which he explained how he and his colleagues, including Nobel laureate and former Caltech President Dr. David Baltimore, are using stem cell science to engineer the human immune system to recognize and eradicate cancer cells in patients with melanoma, the deadliest form of skin cancer. Ribas’ project is scheduled to initiate clinical testing in about 18 months.

Normally the immune system does not recognize cancer as a foreign entity. Dr. Ribas and his colleagues are using stem cells to engineer T cells, the foot soldiers of the immune system that will recognize and attack cancer cells as they would any other disease invader. Ribas played a film of the modified cells actually killing melanoma cells in the laboratory. Ribas indicated he demonstrated the proof of principle for this process by modifying regular blood cells. During a clinical trial, the modified blood cells showed great activity and effectiveness but only over a few months. He observed that by genetically modifying the blood-forming stem cells, Ribas hoped to generate a self-renewing population of immune cells that would kill the cancer.

A world renowned expert on autism, Dr. Geschwind, director of the UCLA Center for Autism Research and Treatment (CART) spoke on Autism: Disease in a Dish. He noted the challenge of trying to create treatments for a disease with possibly hundreds or thousands of implicated genes. Geschwind explained that his team is creating 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 dish, Geschwind’s team can study the causes and possible treatment targets, and identify potential effective drug therapies for this wide-spread and often devastating neurological disorder. 

To kick off the evening, Dr. Witte presented a brief history of the Broad Stem Cell Research Center, made possible in large part by a generous gift from the The Broad Foundations. Dr. Witte emphasized  the effectiveness of the Broad gift was due to sole use directed to supporting the acceleration of stem cell science. 

Dr. Witte highlighted the remarkable and diverse progress that has been made in this exciting and demanding field, and provided insight into the future of medicine through the development of cellular therapies as treatments and cures for disease.  He noted that there are many challenges ahead for stem cell scientists but the promise of this technology will indeed transform the way disease and injury are treated in the future as exemplified by Kohn, Ribas, and Geschwind’s presentations.

In his remarks, Dr. Thomas briefly reviewed the progress to date of CIRM, the state stem cell agency created by the passage of Proposition 71 by California voters.  He noted that CIRM has enjoyed enormous success funding leading-edge stem cell science, highlighting that UCLA is a leader in creating a pathway for future treatments. Dr. Thomas spoke of the future of CIRM and how its structure, with its funding protected from state and Federal government interference, has changed the paradigm of how to support medical research.

Dr. Thomas also mentioned that of all the CIRM awardees, UCLA was the first—with this conference—to offer a public meeting to explain transformative stem cell research to the people who voted for it. He congratulated the Broad Stem Cell Research Center faculty and staff for the success of the capacity crowd and said he viewed this conference as exemplary to the other CIRM awardees and would encourage them to hold similar meetings. 

Dr. Washington highlighted the integration of the UCLA David Geffen School of Medicine and the Broad Stem Cell Research Center, focusing on how the exciting stem cell technologies can be translated quickly from laboratory to the clinic bedside through collaborations of scientists from many different fields and clinicians working toward common goals. Dr. Washington summed up his remarks with three important points: 1) the audience and the citizens of California were thanked for their insight and prescience by voting for Proposition 71; 2) He reiterated that this is a revolutionary time in science, biology, and technology and that UCLA research with its collaborators, such as Caltech and USC, will make history; 3) He emphasized the bright future of stem cell science and the possible fundamental changes that will be seen in the practice of medicine through the development of cell-based therapies.