Monday, March 2, 2020

First patients begin gene therapy treatment for blindness as part of NHS Long Term Plan

The 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, in this case through an injection. Many patients in the trials recovered their night time vision with this treatment.

Jake Ternent, patient at Moorfields Eye Hospital was the first in the UK to receive the treatment. Jake had no night time vision and his daylight vision has been deteriorating since childhood and he is now blind.

Jake said: “After participating in 15 years of research I now have the golden opportunity to be the first person in the UK to have this treatment on the NHS. I am hopeful that this procedure will not only improve my own quality of life but that it will be able to help others with my condition too.”

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.”

Matthew Wood, a patient in Oxford, speaking ahead of the operation, said: “There’s a mix of nervousness and excitement. I’ve known I’ve had retinitis pigmentosa since I was a child, but the specific gene was identified just a few years ago, and that’s what’s made this treatment possible.

“I have had many eye tests over the years and I am registered blind, but actually what I realise is I value the sight that I have, and if this treatment can prevent further deterioration, then that’s a big thing for me. The prognosis is that my sight will deteriorate further, so maintaining the sight I have is really important.”

Through the NHS Long Term Plan, the NHS is committed to freeing up the funds needed to invest in world-class, cutting edge technologies and treatments such as the fast-tracking of CAR-T therapy which uses the body’s own immune system to tackle previously untreatable cancers.

Professor Stephen Powis, NHS medical director, said: “Loss of vision can have devastating effects, particularly for children and young people, but this truly life changing treatment offers hope to people with this rare and distressing condition.

“Once again, the NHS is at the forefront of the genomic revolution with patients in England among the first to benefit from this new form of treatment – a modern day miracle – as part of the Long Term Plan.”

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.”

Professor James Bainbridge, retinal surgeon at Moorfields Eye Hospital and Great Ormond Street Hospital, said:  “The ability to provide gene therapy is a major landmark in NHS care.

“This is the first of a whole new generation of treatments for sight impairment-  made available by the NHS with the expectation of changing lives.”

Tina Houlihan, Chief Executive, Retina UK, said: “The progressive and debilitating nature of this rare genetic condition has a life-long physical, emotional and financial impact on those living with inherited sight loss and their families. We, and our community, will be eager to hear about progress after treatment.

“This pivotal moment demonstrates what can be achieved when industry, the NHS and charities like Retina UK, who represent the patient voice, work together to ensure the best outcomes for families.

“We very much hope that this is just the start and that other therapies will soon be discovered and made available on the NHS for people in the UK who live with a wide range of inherited retinal dystrophies.”

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 for patients living with a retinal dystrophy, caused by a specific gene mutation. Until now no treatment has been available and it is estimated that as many as 100 patients could benefit from the new gene therapy.

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.

The treatment for adults will initially be available from three national specialist centres in Manchester, London, and Oxford, with the option to roll-out the treatment to other hospitals.

Great Ormond Street Hospital in London, which played a key role in the research behind the treatment, has also already begun the treatment on two children – sisters with LCA.

Robert Henderson, consultant ophthalmologist at Moorfields Eye Hospital and Great Ormond Street Hospital, said: “After many years of research it is incredibly exciting to now be treating the first NHS-funded patient with this pioneering gene therapy.

“We are now able to provide a treatment for this rare disease where previously there wasn’t anything available – this could potentially be life changing for Jake and other patients in his position.”

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