Dayton Children’s becomes first in the world to trial new gene therapy for rare neurologic disorder

DAYTON — Dayton Children’s Hospital became the first hospital in the world to treat a child affected by Canavan Disease, a rare neurologic disorder, with gene therapy, the hospital announced Friday.

Inherited and fatal, Canavan Disease is characterized by the spongy degeneration of the white matter in the brain. Beginning in infancy, it destroys a child’s vision, speech and motor function. There is not a cure to Canavan Disease currently.

The National Institutes of Health reports that infants diagnosed with Canavan Disease appear “normal” at birth. Symptoms are found to develop between two and six months of age and typically include poor head control, an abnormally large head and severely diminished muscle tone.

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The clinical trial, led by Dr. Robert Lober and Dr. Christopher G. Janson, used a newly modified recombinant Adeno-Associated – Viral Vector (rAAV). The rAAV is customized to express a healthy gene in the myelin-producing cells to restore enzymatic function in the white matter and promote brain development.

“For the last year in our hospital windows, we’ve featured ‘hope lives in Dayton,’ and that couldn’t be truer than it is today,” Dr. Lober, pediatric neurosurgeon at Dayton Children’s, said in a statement. “This work is giving hope to so many families and will open the door for additional research at Dayton Children’s, allowing us to provide life-changing care for kids within our own community and around the world.”

Over the next two years, at least 16 families from around the world will travel to Dayton to participate in the early phase I/II trail, the hospital announced.

According to the National Institutes of Health, between 750 and 1,000 children are believed to be living with Canavan Disease. Most children with the disease do not live past 10.

Cox Media Group