Nationwide Children’s Hospital and Ohio State University are on the vanguard of U.S. research centers trying to get long-promised gene therapies approved and into patients.
“We clearly have done more gene therapy trials for muscle disease than anyplace in the world,” Dr. Jerry Mendell, director of the Center for Gene Therapy at Children’s, told me Monday. “Our movement ahead has had very rapid growth.”
We spoke by phone as a release was going out about the completion of a licensing deal for a therapy developed at the independent Columbus hospital, which also is the pediatric arm of Ohio State’s medical school. The company, Dallas-based AveXis Inc., had announced last week that the treatment for spinal muscular atrophy, the leading genetically caused killer of children under age 2, received fast-track status toward approval from the U.S. Food and Drug Administration.
This is the second gene-therapy licensing deal in the past few weeks for Children’s, and the hospital also has another treatment – not yet licensed to a company – for a form of muscular dystrophy showing promise in clinical trials.
The licensing brings private companies with the resources to take a treatment to commercialization, while Children’s and often the original inventing scientist reap eventual royalties. Mendell, who oversees most of the trials, never has a personal investment in the technologies.
If any of the therapies eventually are approved, he said, “It would be a big business win for Children’s.”
Gene therapy is most often attempted by hijacking nature’s own hijacker: viruses. Essentially a protein coat and DNA, viruses have no means to reproduce but insert their DNA into host cells. The research turns that power for good, removing the virus’ harmful DNA and filling the capsule with a human gene that is the correct copy of the patient’s faulty gene. Here, let the government explain.
A pall was cast over the field when a patient died in 1999. That research involved modifying the common cold virus, which triggered a massive immune response, but Children’s uses a different type of virus that the immune system doesn’t notice.
“There’s never been an adverse event for (the new type of virus),” Mendell said.
What sets Children’s apart is that it has all the components needed to do everything from the basic research to clinical trials, he said. It’s one of the few centers that has equipment to manufacture the modified viruses by the millions. Children’s invested about $2 million a decade ago in the facility. Within the next week, Mendell says he will be asking for further investment to increase production capacity.
The hospital also is seeking a federal grant to help it set up such centers at other hospitals.
The therapies being researched may not be all that far from approval. The U.S. Food and Drug Administration regulates gene therapy separately from pharmaceuticals, so the phase 2 clinical trials that Children’s is conducting have a chance of being the final research needed before the companies make the final formal application for the FDA nod, skipping a phase 3 trial in multiple centers.
“We’re paving the way, there’s no question,” Mendell said.
Carrie Ghose covers health care, startups and technology for Columbus Business First.