What could the latest gene therapy project mean for you?Posted: Wednesday 20 February 2019 at 09:33
This week saw the announcement that a lady from Oxford has become the first person in the world to have gene therapy to try to halt the progression of dry age-related macular degeneration (AMD).
Many people have been asking what this means for them. We've answered some of your questions:
What is this latest trial?
The early stage trial, at Oxford Eye Hospital, is primarily designed to check the safety of the procedure and is being carried out in patients who have already lost some vision.
When was this carried out and what are the results?
The treatment was carried out under local anaesthetic last month at Oxford Eye Hospital by Robert MacLaren, professor of ophthalmology at the University of Oxford.
It is too early to tell the results from this trial, but if it is successful, the aim would be to treat patients before they have lost any sight, in a bid to halt AMD in its tracks.
Who is the treatment for?
The patients currently being treated on this trial have dry AMD.
What does this mean for macular dystrophies and other macular conditions?
Gene therapies are very specific to the disease they are treating, because the gene mutation is always different.
However, we know gene therapy is a technique which holds a great deal of promise for many diseases. There is already an approved gene therapy treatment called Luxturna available for a rare eye disease (not related to the macula), so the technique can be successful.
Would this help treat a macular hole?
No, surgery is still the best option for a macular hole, as it is not a genetic condition.
Can this gene therapy restore vision?
This gene therapy cannot restore vision already lost. It will only halt the progression of the disease. If this became a successful treatement, would be to treat patients before they have lost any sight.
How many patients are on the trial?
This is the first of 10 patients with dry AMD who are taking part in the trial of the gene therapy treatment.
Can we get on the trial?
You cannot register your interest to take part in future trials for this particular study. The Macular Society is often approached to help find people to participate in clinical trials and research studies. Our research participant database is for anyone with a macular condition, as well as healthy family members and volunteers. You can sign up whether or not you are a member of the Macular Society by completing the form.
Can you get this as a treatment?
It is not currently available as a treatment as it is still experimental at this stage. The news is exciting