Duchenne muscular dystrophy is the most common muscular dystrophy in children, affecting boys. While this devastating, progressive neuromuscular disorder has no cure, several gene therapy programs are making strides to improve patient outcomes.
Aravindhan Veerapandiyan, M.D., known as "Dr. Panda," a pediatric neurologist at Arkansas Children's Hospital (ACH) and an associate professor of pediatrics at the University of Arkansas for Medical Sciences (UAMS), is the principal investigator on four current DMD gene therapy trials. He is a researcher with Arkansas Children's Research Institute.
He serves as director of the comprehensive neuromuscular program and the Parent Project Muscular Dystrophy Certified Duchenne Care Center and co-director of the Pediatric Muscular Dystrophy Association Care Center, all at ACH. Dr. Panda, who earned his medical degree from the K.A.P. Vishwanatham Government Medical College in India, is passionate about clinical research and education, publishing over 60 peer-reviewed articles and presenting at international and national meetings.
Since joining the health system in 2018, Dr. Panda has been the principal investigator for over 15 Duchenne clinical trials, treating over 100 patients living with Duchenne and Becker muscular dystrophy.
Parmaceutical companies reached out to Dr. Panda to be a principal investigator on several studies for new therapies to combat DMD.
"I want to make sure that children from Arkansas and the neighboring rural states have equal opportunity to access these clinical trials," Dr. Panda said. "Historically, if you look at clinical trial participation, representation from the underserved rural states such as Arkansas is minimal."
An estimated 15,000 boys in the U.S. live with DMD, and about 300,000 worldwide.
"Though available treatments such as corticosteroids and newer treatments, such as exon-skipping therapies, can slow down the disease process, no available therapy can halt the condition's progression or, more optimistically, reverse it," Dr. Panda said. "These cutting-edge therapies have the potential to impact the trajectory of the disease."
Dr. Panda’s trials are:
These gene therapy trials and treatments are changing outlooks for DMD patients like Arkansas Children’s patient Wesley Benham, 7. Just before turning 6, Benham received his single Elevidys infusion Aug. 11, 2023. At the time, he was one of about 25 boys in the United States to receive the FDA-approved gene therapy Elevidys. Now, several more patients have received the treatment.
Wesley and his family traveled roughly five hours round-trip weekly for blood tests to check his organ function.
"There are side effects with this drug that need close follow-up and monitoring. I know they're a reliable family. I can have them follow-up with me as much as I want to ensure he's doing OK and safe after receiving the therapy," Dr. Panda said.
While DMD is a deadly disease with no cure and, in the past, “no open doors,” Dr. Panda said these novel gene therapies reach beyond what was formerly possible.
"All these newer gene transfer therapies are opening doors for these boys to possibly live longer and lead a good quality of life. It's changing the trajectory of the disease, changing the trajectory of their lives, improving the quality of life for the boys and their families," Dr. Panda said. "It also serves as a prototype for several other rare neuromuscular diseases where we can integrate these gene transfer therapies."
“All these newer gene transfer therapies are opening doors for these boys to possibly live longer and lead a good quality of life.”