Source: Radio New Zealand
Huntington’s disease causes progressive breakdown of nerve cells in the brain. 123RF
A treatment for Huntington’s disease being trialled in New Zealand and Australia is showing promising early results, the US biotech company behind the drug says.
Skyhawk Therapeutics began trialling the novel drug, SKY-0515, on New Zealand and Australian patients a year ago and plans to expand globally.
Local disease advocates have described the treatment as a real hope.
Huntington’s disease is a rare degenerative brain disorder with no cure at present, affecting about one in 10,000 people.
Each child of a parent with the disease has a 50 percent chance of inheriting the faulty gene and, if they do, will develop the disease – with symptoms typically emerging between the ages of 35 and 45.
Skyhawk Therapeutics said patients taking its daily pill saw a significant drop in mutant huntingtin protein (mHTT) in their blood, which causes symptoms to worsen.
The drug improved their scores on health tests compared to the typical physical decline observed in untreated patients over the same period.
Huntington’s Disease Association Auckland chief executive Jo Dysart said she was lost for words after reading the trial results.
“For our Huntington’s population in New Zealand, it’s amazing, groundbreaking. This is real hope,” she said.
“Our families are very cautious about hope… and we don’t like to use the word cure – I’m not saying this will be a cure by any means – but the fact that we’ve got a tablet that gets over the barrier to the brain in human beings in New Zealand is amazing.”
Dr Greg Finucan, a neuropsychiatrist and chief medical adviser for the Huntington’s Association, said the drug was akin to a stop sign in RNA that prevented the body from reproducing mHTT.
Last year, researchers at University College London’s Huntington’s Disease Centre published results showing an experimental gene therapy slowed disease progression.
That was a positive result but the latest drug from Skyhawk was unbelievable, Finucan said.
“I don’t think we were expecting anything quite as good as this,” he said.
“The [gene therapy trial] showed that a very invasive treatment, involving injections into the brain, slowed down progression to about 25 percent of what it would have been. These preliminary results, from this trial, make it look as though there’s no progression at all, which is quite amazing for a medication.
“It’s just so much more practical to have people at home taking medication than going into a hospital at a huge expense. We calculated the cost of those injections and you could build a new hospital for what it took for that other treatment.”
Finucan said it was important the phase-two trial – now underway – proved there were no serious adverse effects.
“It’s looking very good, but we just need to get the numbers,” he said.
Dysart said families affected by Huntington’s would be keen to know – if the drug was successful – that it would be affordable.
The Huntington’s Disease Association supported 1500 people affected by the disease in Auckland and the North Shore, including people living with Huntington’s, those at risk, and those who were gene-positive, she said.
Skyhawk Therapeutics was working toward testing 520 people at over 52 total sites around the world throughout phase-two and -three of the trial.
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– Published by EveningReport.nz and AsiaPacificReport.nz, see: MIL OSI in partnership with Radio New Zealand
