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Professor Robert MacLaren performs gene therapy treatment on a patient with choroideremia. Image courtesy of John Hopkins University
News, Research

Gene therapy for incurable disease improves vision after 5 years

12/12/2018By Myles Hume
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Choroideremia patients have gained or maintained vision five years after participating in the first gene therapy trial for the previously untreatable blinding condition.

The Oxford Eye Hospital trial – led by UK ophthalmologist Professor Robert MacLaren since 2011 – involved 14 patients with the genetic mutation, each having a virus containing the missing gene injected into the back of their eye.

According to the latest trial results, researchers have identified a significant gain in vision across the patient group. Of the 12 patients who received treatment without any complications, all either gained or maintained vision in their treated eyes, which was sustained for up to five years.

During that time only 25% of untreated eyes, which acted as controls, maintained vision.

“These early results support the notion that a single gene correction can have long-lasting beneficial effects on nerve cells of the retina to prevent blindness,” a University of Oxford statement said.

The study’s success has led to an expansive gene therapy trial involving more than 100 patients across nine countries in the European Union and in North America. Nightstar Therapeutics, a gene therapy spin-out company established by the University of Oxford and Syncona, is overseeing the trial to further develop the treatment.

Professor Robert MacLaren
Professor Robert MacLaren

Dr Chris Hollowood, chief investment officer at Syncona, said Nightstar had advanced the therapy to a pivotal Phase 3 trial. If successful, it could result in the gene therapy treatment being formally approved by the relevant regulatory bodies worldwide.

“We look forward to continuing our support of Robert and Nightstar as we seek to bring transformational treatments to patients,” he said.

An inherited condition that predominantly affects males, choroideremia is among a spectrum of inherited eye diseases occasionally referred to as retinitis pigmentosa. It is the most common cause of untreatable blindness in young people.

In September, the European Medicines Agency formally recommended its first gene therapy treatment for a different eye disease – inherited retinal dystrophy – leading to expectations that other incurable diseases will follow.

The director of research, policy and innovation at UK eye research charity Fight for Sight, Dr Neil Ebenezer, said the benefits of the trial could be far reaching.

“All research breakthroughs are made by standing on the shoulders of others. Our mission is to fund research that will stop sight loss and we’re delighted to have been part of this breakthrough which will have huge benefits for the future,” he said.

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“This technique could transform how we treat diseases and could have broad applicability to a range of other conditions.”

The study was published in Nature Medicine.

“This technique could transform how we treat diseases and could have broad applicability to a range of other conditions.” Neil Ebenezer, Fight For Sight.

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