LCA Family Conference: The Road To Treatment

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Sofia Sees Hope is holding its first LCA Family Conference on Saturday in Groton, CT. This event is the culmination of our first five years of work and fulfills our mission to connect LCA patients and families
to researchers, industry experts and others who are keenly interested in LCA and and rare inherited retinal diseases.

The goal of this conference is to provide updates about advances in research, deepen understanding of the roles various organizations play in developing treatments, and provide insight into how an active patient community can support and accelerate treatment.

We are pleased to welcome Jeffrey Finman, Jupiter Point Pharma Consulting, LLC, Board Member and

Sofia Sees Hope; Dr. Wiley Chambers II, Supervisory Medical Officer in the Office of New Drugs at the FDA; Jennifer Hunt, Vice President of Clinical Operations, Editas Medicine; Tami Morehouse, Phase 1 RPE65 Trial Subject RPE65 genetic therapy trial, to talk about the The Road to Treatment: Understanding How Therapies Are Developed.

Dr. Chambers took a few minutes recently to chat with us.

Dr. Wiley Chambers II, MD

Wiley Chambers planned to join his ophthalmologist father in Connecticut after his residency at George Washington University Medical Center. But word got around the Washington, D.C., medical community that the Food and Drug Administration had no ophthalmologists; he thought he’d give the job a try for a year or two. He could always go to Connecticut.

Thirty-one years later, and having married a lobbyist along the way, Chambers remains in D.C. at the FDA where he has held multiple positions that have all included reviewing ophthalmology products. He has received numerous Public Health Service, FDA and Center for Drug Evaluation and Research awards for his work at the agency. Since 2011, he has been Deputy Director of the Division of Transplant and Ophthalmology Products, Office of New Drugs, in the FDA’s Center for Drug Evaluation and Research. As a Supervisory Medical Officer, he is usually the final person to review and sign off on new proposals before the beginning of clinical trials.

The FDA is involved anytime anyone wants to use an unapproved drug in humans or an approved drug for a different use in humans.

Chambers has clinically reviewed more than 100 ophthalmology drugs that have received FDA approval, including the first gene therapy “done inside a person,” as he said, in the form of LUXTURNA™, approved in December. The drug is a human-engineered virus, injected under the retina in the back of the eye, which contains a healthy version of the RPE65 gene that causes blindness in patients with Leber congenital amaurosis (LCA).

You won’t hear Chambers describe LUXTURNA as groundbreaking or extraordinary. He does not care for the superlatives, because every product that helps an individual is important to the person that it helps. Instead: “If it’s safe and effective, that’s what I care about most.”

In the study process, he said, reviewers and the company that makes LUXTURNA, Spark Therapeutics, ultimately came up with a maze for patients to navigate. “The issue was not how quickly you got through it. The issue was, could you navigate the maze in a lower level of light?”

He called the review and approval process of the medication “routine,” except for the endpoint.
“The endpoint was different than one we used for any other drug product – the ability to navigate in low light. We had never approved something for that before.”

Ultimately, Chambers says, his goal is to have cures, saying “Treatments are OK, but I’d much prefer cures.

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