COLUMN: Nationwide Children's Hospital leads effort for gene therapy treatment for Duchenne muscular dystrophy

Nationwide Children's Hospital

Sarepta Therapeutics announced Sept. 19 that the FDA has granted accelerated approval for eteplirsen for the treatment of Duchenne muscular dystrophy (DMD). Nationwide Children's Hospital was the sole original site for the clinical study that resulted in this approval, led by Dr. Jerry Mendell, director of the Center for Gene Therapy in The Research Institute at Nationwide Children’s.

“FDA approval of eteplirsen is incredibly gratifying to my team and me,” said Dr. Mendell, also a faculty member of The Ohio State University College of Medicine.  “I've conducted many, many clinical trials and never encountered one that was so clean, effective and very well tolerated. Our first-of-its-kind Phase I/II trial of eteplirsen began in 2011 so it has been a long haul for the patients, their families and our research team.”

DMD is the most common life-threatening childhood form of muscular dystrophy. It is caused by genetic defects that lead to missing or non-functional proteins. Eteplirsen is the first FDA-approved treatment for this disease.

“The FDA approval represents a significant milestone and defining moment of progress and hope for patients with Duchenne muscular dystrophy, as well as our team at Nationwide Children’s, who has been pursuing treatments for this devastating disease for decades,” said Dr. Mendell. “I believe this is the best and safest approach for these patients.”

The Center for Gene Therapy at The Research Institute  at Nationwide Children’s is where some of the only early phase gene therapy trials in the world are now in progress at Nationwide Children's Hospital. The mission of the Center is to investigate and employ the use of gene and cell based therapeutics for prevention and treatment of human diseases including: neuromuscular and neurodegenerative diseases, lysosomal storage disorders, ischemia and re-perfusion injury, neonatal hypertension, cancer and infectious diseases. In 2014, Nationwide Children’s was named the first Certified Duchenne Care Center by Parent Project Muscular Dystrophy (PPMD), the leading advocacy organization working to end Duchenne muscular dystrophy.