Miracle cure for blindness
Astonishing US gene therapy restores the sight of Scottish law graduate in medical trial that will bring hope to thousands
A BLIND man has been given back his sight by an experimental treatment that could give hope to thousands of people.
Rory Dewar was only 22 when he lost his vision after developing a rare condition. But 18 months after signing up for a ground-breaking gene therapy trial in the US, the keen rugby player has fully recovered his sight – and is even playing the sport he loves again.
‘It’s been an incredible journey. I can hardly believe I was legally blind and now I can see again,’ he told The Scottish Mail on Sunday.
‘My family and friends are astonished. The clinical team are astonished. I’ve been their most successful candidate so far.’
The 90 volunteers from around the world suffer from Leber Hereditary Optic Neuropathy (LHON), a condition caused by a gene mutation which kills off healthy cells in the retina, leading to the optic nerve dying.
It affects about one in every 30,000 people – usually men aged 15 to 25 or women going through the menopause – but most are unaware they have it until their sight begins to deteriorate.
Rory was diagnosed with
LHON after suffering a loss of vision thought to be linked to a series of concussions he suffered playing amateur rugby in early 2017
Officially registered blind, he was offered a white stick, but declined.
His treatment in a medical facility in Colorado began in
May 2018 with a one-off injection into his eyeballs of a lab-engineered virus. He and his partner, Lauren, have made regular trips back to the US for check-ups paid for by GenSight Biologics, the French-based company which has developed the gene therapy.
He said: ‘It seems like a long jouralready ney at times but it’s been necessary. The idea is your cells replicate and the team needs to test you continuously while that happens. Your genetic make-up changes to get rid of the mutation and rejuvenate the optic nerve. It’s all been an incredible experience seeing it from the inside.’
The firm are now in talks with the US Drug and Food Administration and plan to meet with the European Medicines Agency soon before filing for market approval for their treatment, Lumevoq.
One LHON sufferer in France is already being treated with Lumevoq at the National Eye Hospital in Paris, after the French authorities applied for a temporary authorisation last month in response to an appeal from a consultant ophthalmologist. Scotland has a pathway for introducing new drugs for rare diseases, allowing sufferers to access treatment through the NHS up to three years before a decision is taken about its wider use.
Rory, who lives near Glasgow and will travel to Colorado in May for his final check-up, now hopes the Scottish and Westminster governments will consider gene therapy as an alternative to the current, he says, ‘less effective methods’, which failed to work for him and include vitamin therapy or steroids,
Now 25, he said: ‘I’ll always be grateful for the way this trial helped change my life around. Now I want to see everyone with LHON getting a second chance like me.’
Last night, a spokeswoman for GenSight Biologics said that compliance rules meant it was unable to comment as the trials are still ongoing.
‘A one-off injection into his eyeballs’