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Annual Review 2011 - Wellcome Trust

Annual Review 2011 - Wellcome Trust

Understanding the brainA

Understanding the brainA new drug has been shown to help people withinherited blindness recover some of their vision.The most common form of inheritedirreversible blindness is Leber’shereditary optic neuropathy (LHON),affecting 2000 people in the UK. Thedisease strikes early in adulthood;people who can see normally until theonset of the condition generally losethe sight in one eye first and then,within three to six months, in theother eye.LHON causes a decline in levels ofthe antioxidant coenzyme Q10 inthe mitochondria of eye cells.Mitochondria are the cell’s ‘batteries’that power its function, and it isnotoriously difficult to treatmitochondrial disorders. In July 2011,however, Wellcome Trust-fundedresearchers published the resultsfrom a trial of a drug for LHON,which suggested they had identifiedthe first effective treatment for amitochondrial disease.The team, led by Professor PatrickChinnery, a Wellcome Trust SeniorResearch Fellow in Clinical Scienceat Newcastle University, conducteda multi-centre double-blindrandomised controlled trial in 85patients with LHON caused bymitochondrial DNA mutations.Participants recruited from hospitalsin Newcastle, Munich and Montrealwere given either a drug calledidebenone or a placebo for 24 weeks.Idebenone is a synthetic analogue ofcoenzyme Q10 and, although it hadnot been evaluated in a clinical trial,anecdotal reports had suggested thatit improved vision in people withLHON.Published in the journal Brain, thefindings indicated that idebenone wassafe and well tolerated, and that aftersix months, some patients who hadreceived it had improved vision andperception of colour. The greatestimprovement was seen in those at anearlier stage of the condition.In nine out of 36 severely affectedpatients, who could not see an eyechart on the wall at the start of thetrial, treatment with idebenoneresulted in a marked improvement:their vision improved to the extentthat they were able to read at least onerow of letters on the chart. None ofthe 26 patients given the placeboimproved to that extent.Restoring some vision to peoplelegally certified as blind can mean avast improvement in their quality oflife, enabling them to move aroundmore easily, use a computerisedviewer to read, get dressed and evensee family photos again. Moreover,patients report that they still haveimproved vision, even though they areno longer taking the drug. The teamnow aims to verify this and study theeffect further. They believe there maybe a case for offering idebenone fromthe moment that LHON is diagnosed– preferably before any symptomsdevelop.Artwork of the iris and pupil. Pasieka/SPLAnnual Review 2011 | 25

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