Big gains in treating fatal disease
BRITAIN: Huntington’s disease patients have been offered new hope after a trial showed a new genetic treatment appears to slow down the illness.
Current medications only treat symptoms rather than treating the underlying condition, which gradually eats away at the brain, robbing people of movement, speech and dignity.
But a trial of 46 adults showed the new drug silences the genetic mutation that causes Huntington’s.
Professor Sarah Tabrizi, director of University College London’s Huntington’s Disease Centre, who led the phase one trial, said the results were ‘‘beyond what I’d ever hoped’’ and said it eventually may be possible to stop the disease before irreversible damage to the brain had occurred.
‘‘The results of this trial are of groundbreaking importance for patients and families,’’ she said. ‘‘For the first time, we have the potential, we have the hope, of a therapy that one day may slow or prevent Huntington’s.’’
It is an incurable degenerative disease caused by a single defect in the huntingtin gene, which turns a usually helpful protein into a lethal brain cell killer.
The drug, called Ionis-httrx, works by intercepting a messenger molecule and destroying it before the harmful protein can be made, effectively silencing the effects of the mutant gene. It effectively stops Huntington’s in its tracks.
Although the trial was too small, and not long enough, to show whether patients’ clinical symptoms improved, it showed the drug was safe, well tolerated by patients and reduced the levels of huntingtin in the brain.
‘‘This is probably the most significant moment in the history of Huntington’s,’’ added Tabrizi.
To deliver the drug to the brain, it has to be injected into the fluid around the spine once a month, using a 10cm needle. It could potentially be adapted to target Alzheimer’s and Parkinson’s.
Roche, the Swiss pharmaceutical giant, has paid US$45 million for the rights to the drug, and is expected to launch a major trial to test its clinical effectiveness, while trial data is sue to be published shortly.
Cath Stanley, chief executive of Britain’s Huntington’s Disease Association, said: ‘‘This is a great day for the Huntington’s disease community. Although there is still some way to go before the overall results are known, this is a big step forward.’’
– Telegraph Group