An unintended result of a small study into muscular dystrophy has opened up a new frontier for national gene therapy research.
As part of the first clinical trial addressing gene therapy, researchers studying muscular dystrophy were attempting to use a virus to safely provide patients with the disease a genetic protein that could treat them.
To their surprise, researchers found some patients already had a version of the protein, said R. Jude Samulski, director of the Gene Therapy Center and an author of the study.
He said researchers were puzzled when patients’ bodies mounted an autoimmune response that attacked the good protein as if it were bad.
They hypothesized that the existing protein caused the patients’ bodies to create an immunity to it , similar to immunity to the flu after a shot, he said.
“The body has already seen these fibers, and the immune response is to attack them,” Samulski said.
The discovery means patients in future research trials will need to be screened and treated based on their results, providing researchers with a clearer idea of which course of treatment might be better.
“People hypothesize that this may be seen in all genetic disorders,” Samulski said. “That’s where all the hoopla is coming from.”
Tara Britt, director of research at the Gene Therapy Center, said more projects — even some unrelated to muscular dystrophy — will see increased funding as a result of the new findings.