In April, we highlighted a pioneering Massachusetts Eye and Ear (Mass. Eye and Ear) gene therapy treatment offering hope to a young patient with an inherited retinal disorder. Though the surgical-first, newly therapeutic Luxturna had yielded encouraging clinical trial results, there was no way to know for sure how it would impact the 13-year-old patient’s eyesight—until now.

Young Jack Hogan’s recent return to Mass. Eye and Ear to assess his progress began with anecdotal reports of changes he’d noticed since making history as the first person to receive the $850,000 treatment. There was plenty of encouraging evidence that Luxturna had worked according to plan: basketball matches in low light, clear views of classroom whiteboards without visual aids, evening bike rides with friends. Everyday activities most 13-year-olds take for granted, suddenly made possible—and changing the game—for Jack. 

Then, a series of tests performed by the medical team of Jack’s surgeon, Jason Comander, MD, confirmed the anecdotal with the scientific: Jack’s eye chemistry had responded to the medication and was now making accommodations for his missing gene. And that translates to better visual acuity and the ability to see more with less light. The innovative treatment had worked.

Spark Therapeutics, the maker of Luxturna, expects lifelong benefits from the therapy, with four years of stable improvement demonstrated during clinical trials. For Jack, each day of improved vision is cause for celebration.

“Nothing was guaranteed,” says Dr. Comander. “To see it coming through for him is great.”

Read more in STAT and The Boston Herald.

Photo credit: The Boston Herald.

Topics: Technology, Clinical Trials, Innovation

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