‘Significant’ breakthrough in treating Huntington’s disease

HUNTINGTON’S disease patients have been offered hope after a trial showed a new genetic treatment appears to slow down the illness.

Current medications only treat symptoms rather than the underlying condition, which gradually eats away at the brain, robbing people of movement, speech and dignity.

But a trial of 46 adults showed that the new drug silences the genetic mutation that causes Huntington’s.

Prof Sarah Tabrizi, the director of University College London’s Hunting- ton’s Disease Centre, who led the phase one trial, said the results were “beyond what I’d ever hoped” and eventually it may be possible to stop the disease before irreversible damage to the brain had occurred.

“The results of this trial are of ground-breaking 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.”

The incurable degenerative disease is caused by a single defect in the huntingtin gene, which turns a usually helpful protein into a lethal brain cell killer. It affects around 10,000 people in Britain. 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, crucially, it reduced the levels of Huntingtin defects in the brain. “This is probably the most significant moment in the history of Huntington’s,” added Prof Tabrizi.

To deliver the drug to the brain, it has to be injected into the fluid around the spine once a month using a fourinch needle. Potentially, it could be adapted to target Alzheimer’s and Parkinson’s.

Roche, the Swiss pharmaceutical giant, has paid $45million (£33.7million) for the rights to the drug, and is expected to launch a major trial to test its clinical effectiveness.

Cath Stanley, the chief executive of the 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.”