Advisors to FDA Recommend Approval of Gene Therapy for Blindness

Spark Therapeutics’s Luxturna would be the first approved therapy in the U.S. that replaces or repairs a defective gene inherited from one’s parents.

By Jef Akst | October 12, 2017

gene therapy for blindnessFLICKR, HELGA BIRNA JÓNASDÓTTIRUpdate (December 19): The US Food and Drug Administration (FDA) today approved Luxturna for the treatment of an inherited form of vision loss. It is the first gene therapy approved in the U.S. to correct a specific disease-linked gene. “We’re at a turning point when it comes to this novel form of therapy,” FDA Commissioner Scott Gottlieb said in a statement. “I believe gene therapy will become a mainstay in treating, and maybe curing, many of our most devastating and intractable illnesses.”

A panel of 16 independent advisers to the US Food and Drug Administration (FDA) voted unanimously today (October 12) to recommend the approval of Luxturna, a gene therapy meant to treat Leber congenital amaurosis—a rare, inherited form of childhood blindness—and other retinal disorders, STAT News reports. If the agency agrees with the panel’s recommendation, Luxturna would be the first gene therapy aimed at correcting a congenital defect approved in the U.S.

The therapy, developed by Philadelphia-based Spark Therapeutics, is injected directly into the eye in a one-time treatment. Billions of virus particles deliver a functional copy of the RPE65 gene—which encodes a protein necessary for normal vision and is mutated in patients with certain vision disorders—to the retina. So far, more than two dozen children and adults with RPE65 mutations have been treated with Luxturna in the context of clinical trials, and some 93 percent of them have shown improved light sensitivity and functional vision. The effects are long lasting, with improvements continuing for at least four years in some patients that have been tracked that long.

“If my child or myself had this condition I would not hesitate for a moment getting treatment with [Luxturna],” Albert Maguire, a retinal disease specialist at Children’s Hospital of Philadelphia who led the Luxturna clinical trials, said at the advisory panel hearing today, according to STAT News.

If approved, Luxturna would be the first gene therapy that fixes an inherited genetic disorder in the U.S. (The official title of first approved gene therapy goes to Novartis’s CAR T-cell therapy, greenlighted this August.) The FDA must make its decision by January 12, 2018.

“This is what I believe medicine is going to be like for the next 20, 30, if not 50 years,” Spark CEO Jeff Marrazzo tells MIT Technology Review of these types of gene therapies. “I think this is the beginning of an age that is going to fundamentally change medicine.”

See “Targeting DNA

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Comments

Avatar of: dhojnowski

dhojnowski

Posts: 1

October 15, 2017

Quick question: Isn't one of the issues with gene therapy the rogue behavior of the viral vectors used that unfortunately cause cancerous cells? Why is this situation different?

Avatar of: skinny

skinny

Posts: 7

Replied to a comment from dhojnowski made on October 15, 2017

October 18, 2017

Viruses are not inherently carcinogenic. Some do carry oncogenes, but if they are used as vectors those are removed. There is, however, the risk that a gene will insert into the middle of a gene that perhaps keeps cancer in check. If this gene is inactivated, it is then possible that cancer would result. Usually, this is prevented by carefully selecting the vector and controling the target for gene insertion. Generally, the risk is rather low, but it is still in the research stage.

Avatar of: skinny

skinny

Posts: 7

October 18, 2017

 

 

Viruses are not inherently carcinogenic. Some do carry oncogenes, but if they are used as vectors those are removed. There is, however, the risk that a gene will insert into the middle of a gene that perhaps keeps cancer in check. If this gene is inactivated, the cancer risk would increase. Usually, this is prevented by carefully selecting the vector and controling the target for gene insertion. Generally, the risk is rather low, but it is still in the research stage.

 

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