The Queen Elizabeth University Hospital in Glasgow has been chosen as a UK research centre to trial a new drug treatment for the degenerative, neurological condition, Huntington’s disease (HD).
Wave Life Sciences’ PRECISION-HD trials are being carried out at multiple locations around the world with two drugs in development specifically targeting the faulty gene that causes HD.
A number of patients with early onset HD symptoms have been recruited to take part in the Glasgow trial.
Lead researcher at the University Hospital, Dr Stuart Ritchie said: ‘I am very excited Glasgow has been chosen to take part in this early stage drug trial. The Huntington’s Disease community in the West of Scotland now have the opportunity to be involved in these ground-breaking research programmes.’
Wave Life Sciences, headquartered in the United States, focuses on delivering transformational therapies for patients with serious, genetically-defined diseases such as HD.
‘Wave’s PRECISION-HD programme is the first to target the underlying cause of Huntington’s disease,’ said Michael Panzara, MD, MPH, Neurology Franchise Lead at Wave Life Sciences. ‘These investigational compounds have the potential to address a critical unmet need for the HD patient community where no disease-modifying treatments are currently approved.’
HD is a complex condition with symptoms that typically begin to develop between the ages of 30 and 50. It causes three main groups of symptoms: changes to thinking processes – a type of early onset dementia, loss of muscle control and involuntary movements which lead to loss of speech and swallow along with mental illness. Those impacted by HD may eventually lose the ability to walk, talk, eat, drink or make decisions and will eventually need 24 hour care. It is also hereditary with each child of those diagnosed at 50% risk of developing the disease. There is currently no cure.
It is estimated there are around 1100 people living with HD in Scotland and up to 6000 potentially at risk. The Scottish Huntington’s Association is the only charity in the country supporting families impacted by HD through a national network of specialists, a world leading youth support team and a financial wellbeing service.
‘Although every step closer to an effective treatment is fantastic news, it is important to remember that many thousands of people will continue to live with this condition. Their families will continue to need support, and we will need to continue to help improve their quality of life any way we can,’ said SHA chief executive, John Eden.
The phase 1b/2a trials are likely to last for approximately a year with the company expecting to release top line results in the first half of 2019. ends