A drug to reverse the effects of Huntington’s disease could be available within the decade, as a trial begins to test its safety in humans.
Huntington’s is an inherited degenerative brain disorder that causes psychiatric problems and difficulties with behaviour, feeding and communication, eventually leaving the sufferer dependent on nursing care.
About 12 in every 100,000 people in the UK have the condition, which usually manifests itself between the ages of 35 and 55. There are no treatments to prevent, slow or cure the disease.
Sarah Tabrizi, director of the Huntington’s Disease Centre at University College London, said: “It is a devastating disease. Children often nurse an affected parent, knowing they are at risk themselves.”
The experimental drug ISIS-HTTRx, developed by Isis Pharmaceuticals, targets a protein known to cause the disease. In tests on mice the drug appeared to be safe and efficient. The first patients in the trial have had it injected it into their spinal fluid.
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