Officials from a California medical institute unanimously voted Thursday to award $7.4 million to a UCLA center to fund therapy clinical trials for a cure to X-linked chronic granulomatous disease.
The California Institute for Regenerative Medicine voted unanimously for the UCLA Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research to receive the award.
UCLA scientists will lead Phases I and II trials for X-linked chronic granulomatous disease (CGD), which is an immunodeficiency disorder. Chronic granulomatous disease is a life-threatening genetic disorder that inhibits white blood cells from effectively killing bacteria and other microorganisms in the body. The disorder affects one in every 200,000 people in the United States.
Donald Kohn, a professor in the David Geffen School of Medicine and lead researcher in the trials, said the $7.4 million will cover patient care costs for the stem cell transplant, research staff wages and medical supplies. The patient pool consists of toddlers, children and young adults, Kohn said.
The Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research conducts research to understand human embryonic stem cells, supporting research for future treatments.
The therapy will remove and modify patient’s blood-forming stem cells to correct the genetic defect that causes the X-linked CGD. Once treated, the blood stem cells will be transplanted back into the patient, where they should continue to produce white blood cells that allows the immune system to fight infection.
According to the press release, UCLA researchers will oversee stem cell gene therapy of patients at three research sites in the U.S, including Mattel Children’s Hospital UCLA, the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center and the National Institute of Allergy and Infectious Diseases.
Though the gene therapy has not yet been approved by the U.S. Food and Drug Administration, clinical trials will treat 10 patients over the next two to three years, Kohn said. It will take up to four years to acquire results from the tests.
Compiled by Alejandra Reyes-Velarde, Bruin senior staff.