A pioneering trial led by Oxford University has delivered a potential new treatment for a type of blindness which was previously thought to be incurable. The trial was to cure the genetic disease choroideremia, which affects up to 1,500 people in Britain and 100,000 people worldwide.
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If the trial is successful, it may have significant consequences for neuroscience and for many other eye diseases as the virus employed could be used in future to create cures for genetic brain disorders.
If the trial is successful, it may have significant consequences for neuroscience and for many other eye diseases as the virus employed could be used in future to create cures for genetic brain disorders.
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Choroideremia is commonly detected in childhood, and leads to blindness, usually by the late forties. It is caused by a faulty gene REP1, which triggers the death of light-sensing cells in the retina called photoreceptors. The disease only affects males.
Choroideremia is commonly detected in childhood, and leads to blindness, usually by the late forties. It is caused by a faulty gene REP1, which triggers the death of light-sensing cells in the retina called photoreceptors. The disease only affects males.
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Jonathan Wyatt, a former barrister from Bristol, is the only patient to have undergone the trial so far. He will not recover his sight, but the procedure should stop his vision deteriorating any further- he would otherwise become totally blind in a few years. If the surgery is effective, the doctors aim to perform the same treatment on his right eye. They also plan to do surgery on twelve other patients.
Jonathan Wyatt, a former barrister from Bristol, is the only patient to have undergone the trial so far. He will not recover his sight, but the procedure should stop his vision deteriorating any further- he would otherwise become totally blind in a few years. If the surgery is effective, the doctors aim to perform the same treatment on his right eye. They also plan to do surgery on twelve other patients.
The treatment was developed by Professor Robert MacLaren at Oxford University, and Professor Miguel Seabra of Imperial College.
Professor Seabra remarked that “the ability to offer a gene replacement treatment for these patients was the final objective of 20 years of intense research in my laboratory. This is a moment of fulfilment for us and a dream come true for all choroideremia patients.’”
Professor MacLaren, who also undertook the surgery, admitted that the outcome was “unpredictable” considering the possibility of removing all the remaining sight in Mr Wyatt’s eye. Mr Wyatt commented, “I assessed the risks and decided they were worth taking, both for me and for all the other people who have the problem.”
Professor MacLaren informed Cherwell “To say I’m very excited would be an understatement. I am also relieved. Relieved because we delivered the treatment without causing any harm, and relieved because we have proved that viral vectors can work.” MacLaren also told us of the pleasure of being able to convert his lab results into a working treatment.
The blindness charity Fight for Sight responded to news of trial that they were “absolutely delighted with the news that a clinical trial for choroideremia is underway.”
One medic, Imogen Welding, a first year at Corpus commended the research saying, “I think it’s really exciting that this kind of advance in technology can help people who otherwise can’t be treated.”
However, Jen Todd, a first year Balliol medic, had some tempering advice commenting, “It is really important, but we shouldn’t get ahead of ourselves. Restoring someone’s sight will be much harder than just stopping the vision deteriorating.”
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