UCLA Health researchers have been awarded a $1.75 million federal grant to investigate why some Duchenne muscular dystrophy patients develop liver toxicity after receiving a gene therapy treatment meant to prevent muscle deterioration.
Funded by a U.S. Department of Defense grant through 2029, the research will investigate liver toxicity caused by treatment with Elvidys, a one-time gene therapy treatment to help Duchenne muscular dystrophy patients produce a functional version of a missing muscle-protecting protein called micro-dystrophin. One potential side effect of the treatment is liver injury and acute liver failure among some individuals.
Duchenne muscular dystrophy is one of the most common genetic diseases of childhood for boys and causes rapid muscle deterioration. The disease causes loss of ambulatory function by age 12, loss of upper arm use in teen years and eventually heart and respiratory failure that leads to death in their 20s. There is no known cure.
The new study will be led by UCLA neurologist Dr. Melissa Spencer in collaboration with neurologist Dr. Perry Shieh, Assistant Professor Tayo Ikotun of the Department of Molecular & Medical Pharmacology and Professor Alex Huk of the Department of Psychiatry and Biobehavioral Sciences. They will use biological samples collected from patients before and after treatment to investigate the immune, molecular and physiological responses associated with liver injury and identify factors that may place certain patients at great risk of liver toxicity.
The long-term goal of the study is to develop ways to predict, prevent and effectively manage liver toxicity and thereby improve safety of the treatment and other similar gene therapies.
