The defect that causes the neurodegenerative disease Huntington’s has been corrected in patients for the first time, the BBC has learned. An experimental drug, injected into spinal fluid, safely lowered levels of toxic proteins in the brain. The research team, say there is now hope the deadly disease can be stopped.
A drug that suppresses the rogue gene behind Huntington’s disease has been successfully tested in humans, scientists have said, raising the hopes of a treatment for about 8,000 people in the UK who have the condition.
By targeting and eliminating toxic proteins it also represents a new approach that some believe could help to treat other neurological diseases, such as Alzheimer’s and Parkinson’s.
Huntington’s is a terminal disease in which a mutant gene causes brain damage that gets gradually worse from middle age. Before people die they suffer from hand tremors, personality changes and problems communicating. They also know there is a 50 per cent chance that they will have passed on the condition to their children.