Four prominent researchers from UCLA’s Eli & Edythe Broad Center of Regenerative Medicine and Stem Cell Research have received Early Translational research awards totaling approximately $13 million from the California Institute for Regenerative Medicine (CIRM) the state stem cell agency. The UCLA researchers received four of the 12 total awards; no other applicant institution received more than one award.

Dr. Jerome Zack professor of medicine and microbiology, immunology and molecular genetics; Dr. Robert Reiter, Bing professor of urologic research; Dr. Donald Kohn professor of pediatrics and microbiology, immunology and molecular genetics in the life sciences; and Dr. Gerald Lipshutz, associate professor-in-residence of surgery, urology and medicine each received grants that were announced at the meeting of the Independent Citizens Oversight Committee, CIRM’s governing body, in La Jolla, CA on August 28, 2013.

The grants are part of CIRM’s Early Translational Research Initiative, which aims to fund and advance promising, innovative discoveries using stem cells.  In this “early translation” phase, scientists are expected to do research that will result in the development of drugs or cellular therapies to be used in FDA-approved clinical trials; translating discoveries from the laboratory to the clinic as quickly as possible.

“Our CIRM grants highlight the excellence of the UCLA bench-to-bedside research program,” said Dr. Owen Witte, director of the Broad Stem Cell Research Center.

Dr. Zack, who has dedicated his distinguished career to finding a cure for HIV/AIDS,  received approximately $5.3 million for his team’s project to engineer blood-producing stem cells that will create T-cells, the foot soldiers of the immune system, that recognize and attack HIV. The engineered T-cells are to be given to the patient through a bone marrow transplant, a one-time treatment that will give the patient an inexhaustible source of immune system cells capable of eliminating HIV-infected cells. This treatment would serve as a functional HIV cure with minimal adverse effects, a great improvement over the current standard of care with expensive, regularly given drug cocktails.

Dr. Reiter, a prominent prostate cancer researcher, received approximately $4 million dollars for his research in developing a type of drug called a monoclonal antibody to target castration-resistant prostate cancer stem cells. Castration-resistant prostate cancer is an aggressive, recurrent form of the disease. This potentially transformative treatment for cancer patients could possibly eliminate the cancer stem cells responsible for recurrent disease and lead to long-term remissions.

Dr. Kohn, whom CIRM President Alan Trounson acknowledged as a world leader in gene therapy, received approximately $1.8 million for his project to treat sickle cell disease (SCD), a genetic disorder in which red blood cells “sickle,” causing pain crises and eventually organ failure. Currently, the only effective treatment for SCD is a bone marrow transplant from a rare matched sibling donor. Dr. Kohn’s team developed a gene editing technology to correct the sickle gene defect in the blood-forming stem cells.  After collecting the patient’s stem cells from the bone marrow, Dr. Kohn and his team will genetically modify the  cells using the gene editing technology and transplant the corrected cells back into the patient. It is hoped that the new blood forming stem cells will create healthy red blood cells that do not “sickle,” effectively curing the disease.

Dr. Lipshutz, a leading transplant surgeon, received approximately $1.8 million for his project to develop a treatment for a condition called arginase deficiency. This genetic disorder of the liver causes the amino acid arginine (a building block of proteins) and ammonia to accumulate gradually in the blood, which has a harmful effect on the central nervous system, causing stiffness and muscle spasticity, slower than normal growth, developmental delay, and eventually intellectual disability, tremor and seizures. Dr. Lipshutz and his team seek to develop a source of gene-corrected liver-like cells for treating patients with this disease. They will attempt to correct the genetic defect by using induced pluripotent stem cells (iPS cells) that they develop from the skin cells of patients.  They then drive the iPS cells to become liver cells with the corrected gene and give the modified cells back to the patient. This treatment would eliminate the organ rejection problems of liver transplant, the current standard treatment, and could be used for other diseases besides arginase deficiency that are treatable with liver transplants.

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