A new life-changing treatment for children and adults – voretigene neparvovec – is the first in a new generation of gene therapies that can be directly administered to patients, with many patients in the trials recovered their night time vision with the treatment.
Manchester Royal Eye Hospital’s first patient, Lee Morris, 39 from Lancashire, said “I feel like I’ve waited 39 years for this treatment and I am very grateful to be the first patient to undergo this treatment at MREH.”
“Early indications are that the treatment was a success, and I am hopeful this treatment will open so many more doors for me – not just at work in being able to go for promotions, but to increase my confidence in going out alone.”
“Winter is an especially difficult time for me as it’s so dark and I have no night-time vision at all.”
Robert MacLaren, Consultant Ophthalmologist at Oxford University Hospitals, said “I am delighted to see that gene therapy research has finally come to fruition and we now have our first approved treatment – in this case for a rare form of genetic blindness.”
“The NHS has been leading the world in gene therapy research with several successful clinical trials.”
“The recent decision by NICE to provide this novel treatment for our patients shows how the UK is maintaining its global position in delivering first class healthcare.”
Mr Assad Jalil, the Consultant Vitreoretinal Surgeon who undertook the procedure at Manchester Royal Eye Hospital, said “Treating inherited retinal diseases is extremely challenging but gene therapy offers hope to all such patients.”
“Subretinal Luxturna injection is probably the first of many such gene therapy-based treatments, which will become available in the future. We at Manchester Royal Eye Hospital are proud to be a part of this genomic revolution.”
Patients here will benefit from voretigene neparvovec, also known as Luxturna, after NHS England reached a NICE-endorsed deal with manufacturer Novartis to fund the drug.
It is expected that this will provide access to treatment for the first time to around 100 patients living with a retinal dystrophy, caused by a specific gene mutation, to have access to treatment for the first time. Until now no treatment has been available.
The condition blocks messages for making proteins in the eye that are essential for normal vision the drug works by recreating these processes like that in a healthy functioning eye.