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

The first patients have received a revolutionary new gene therapy that can restore eyesight as part of the NHS Long Term Plan.

Babies born with an inherited retinal disorder, known as Leber’s Congenital Amaurosis (LCA), have poor sight which swiftly deteriorates, with many ultimately losing their vision completely in childhood.

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 yesterday 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, yesterday 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, yesterday 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, yesterday 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, yesterday 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, yesterday 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.”

Robert Henderson, consultant ophthalmologist at Moorfields Eye Hospital and Great Ormond Street Hospital, yesterday 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.”

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.

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.


Channel website: https://www.england.nhs.uk/

Original article link: https://www.england.nhs.uk/2020/02/first-patients-begin-gene-therapy-treatment-for-blindness/

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