Subscribe to RSS
‘Hope is contagious,’ Len Seymour discusses today’s results of the Moorfields gene therapy trial.
Len Seymour, professor of gene therapies at Oxford University and president of the British Society for Gene Therapy, discusses the impact of a gene therapy trial conducted on a young man with Leber’s congenital amaurosis (LCA), published as the leading article in the Independent today,
“Leber’s Congenital Amaurosis leads to progressive loss of sight. It is caused by a single fault in a single gene. By injecting a solution containing the normal gene into the retina at the back of a sufferer’s eye, researchers from Moorfields have managed to improve the patient’s sight.â€?
The paper, Effect of Gene Therapy on Visual Function in Leber’s Congenital Amaurosis, by James W.B. Bainbridge, et al, is published today, in the New England Journal of Medicine:
Summary
“Early-onset, severe retinal dystrophy caused by mutations in the gene encoding retinal pigment epithelium–specific 65-kDa protein (RPE65) is associated with poor vision at birth and complete loss of vision in early adulthood. We administered to three young adult patients subretinal injections of recombinant adeno-associated virus vector 2/2 expressing RPE65 complementary DNA (cDNA) under the control of a human RPE65 promoter. There were no serious adverse events. There was no clinically significant change in visual acuity or in peripheral visual fields on Goldmann perimetry in any of the three patients. We detected no change in retinal responses on electroretinography. One patient had significant improvement in visual function on microperimetry and on dark-adapted perimetry. This patient also showed improvement in a subjective test of visual mobility. These findings provide support for further clinical studies of this experimental approach in other patients with mutant RPE65. “
Whilst we all appreciate the impact that this news has upon the future of gene therapy , we must also be careful how this information is interpreted. As Professor Seymour states, “Hope is contagious” but poor interpretation by the media can spin out of control; I have already received a call from a patient this morning wanting to know if this procedure could be used for age related macular degeneration.
Expect a few calls over the coming weeks!
May 1st, 2008 ~ 
vaughan lewis // Apr 29, 2008 at 12:25 am
i’ve got Startgardt’s (gene ABCA4). the BBC inluded an interview where it was suggested that this trial could lead to a cure for AMD so the person who called you may be forgiven.
i suggest you put something up which is an antidote to inaccurave media coverage. better than leaving it to sufferers to scrabble around to find the full story and avoiding famiy members getting upset at another set of dashed hopes.
Jane Macnaughton // Apr 29, 2008 at 1:33 am
I agree with you. I think what would be more useful here is to have a well balanced article hightlighting the past, present and future of this research so that fellow pracitioners may offer the best advice to patients and family members. Thank you for this prompt.
Leave a Comment