Stritch alum develops new drug to treat Duchenne Muscular Dystrophy

By Elizabeth Czapski

The field of pediatric neurology is changing, and Stritch alum Dr. Edward Kaye (BS ’71, MD ’75) is leading the charge. The child neurologist and CEO/CMO of Sarepta Therapeutics has played a key role in the development of a new drug to combat Duchenne Muscular Dystrophy (DMD), continuing his longstanding passions of caring for children and unlocking the mysteries of neurogenetic diseases.

Five years ago, Kaye was recruited by Sarepta Therapeutics, a biopharmaceutical company currently based in Cambridge, Massachusetts, to be the company’s chief medical officer. Later on he accepted an offer from the company’s board of directors to take on the additional role of chief executive officer, but along the way his primary focus has been the development of Eteplirsen, a drug that Kaye hopes will slow the progression of DMD.

DMD is an isolate genetic disorder in which muscles gradually deteriorate due to an absence of the protein dystrophin in the body. It is most common in boys, and most people with the disorder die by their mid- 20s, says Kaye. Eteplirsen manipulates the patient’s RNA so that the body can produce functional proteins. “And what we saw with this drug is by replacing the protein,” says Kaye, “we slow down the deterioration of the muscles and we allow the boys to walk for a longer period of time.”

Studies began with boys ranging from age 7 to 13, but Kaye says the goal is now to start earlier by treating younger boys. “The hope is that we’ll be able to prevent some of the muscle damage that occurs and basically just try to slow down the progression of the disease,” he says.

After earning his bachelor’s degree from Loyola in 1971, Kaye made the decision to enroll as a med student at Stritch. He credits the clinical training he received and the exposure to several different areas of medicine with helping him make the decision to go into pediatrics. “I’ve always found it much more enjoyable to take care of children,” says Kaye, noting that he found it easier to make a substantial difference in the health of younger patients. “With adults, there are a lot of lifestyle issues you can’t change. But in children, you can.”

Kaye didn’t always envision himself as a neurologist, assuming instead that he’d become a general pediatrician. But during his residency he found himself drawn to the complexities of the nervous system, which led him down the path to his current work in treating children with DMD.

“My focus really has been in neurogenetic diseases because those are the least understood,” says Kaye. “And so I was really interested in trying to understand the nervous system to see if there was anything that I would be able to do to help these children.”