Patients living with Leber’s congenital amaurosis (LCA) have started to receive a new gene therapy on the NHS that has the potential to restore sight.
The treatment, voretigene neparvovec, is directly administered through an injection, with many patients in trials recovering their night-time vision.
There has previously been no available treatment for LCA in the UK and it is estimated that 100 patients could benefit from the new gene therapy.
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.’
Voretigene neparvovec is marketed as Luxturna by pharmaceutical company Novartis and was recommended by the National Institute for Health and Care Excellence (Nice) in September 2019.
The drug has been made available after NHS England reached a Nice-endorsed deal with Novartis to fund it.
It is recommended as an option for treating RPE65-mediated inherited retinal dystrophies in people living with vision loss.
A blind patient at Moorfields Eye Hospital was the first in the UK to receive the treatment. Jake Ternent had no night-time vision and his daylight vision has been deteriorating since childhood.
‘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,’ Ternent said.
The treatment will initially be made available from three specialist centres in Manchester, London and Oxford with the option to roll it out to other hospitals.
Great Ormond Street Hospital in London, which played a role in the research behind the treatment, has also started the treatment on two children living with LCA.