Huntington’s disease (HD) is within reach, a charity conference has heard.
Dr Ed Wild, one of the world’s leading researchers into the degenerative neurological condition said research into treatments for the condition was now on a “knife edge.”
Wild outlined how several particularly exciting drug programmes were now moving into advanced stages.
He told the delegates at the annual Scottish Huntington’s Association (SHA) conference: “In my career working with HD I’ve never been more excited,” he said.
“Not only is research into effective treatment making real progress, but the way the condition is perceived has changed beyond all recognition.
“When I first started working with HD patients it was very much a disease hidden away, people did not want to talk about it. Now people are signing up for worldwide clinical trials and openly sharing their stories to increase understanding.
“It really is beginning to step out of the shadows.”
The latest drugs in development aim to reduce production of the faulty protein created by the genetic change that causes HD. The treatment aims to allow the brain to repair damaged cells; it is hoped this will have the effect of slowing down the rate that symptoms develop.
The SHA is the only charity in the country that supports families living with the condition through a team of HD specialists; a dedicated youth support team and a financial wellbeing service.
HD is a complex neurological 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, make decisions or care for themselves requiring 24-hour care. It is also hereditary with each child of those diagnosed carrying a 50% risk of developing the disease. There is currently no cure.
It is estimated there are around 1,100 people living with HD in Scotland and between 4,000 to 6,000 potentially at risk.
“HD is often called the most curable incurable disease, because unlike other neurological conditions, such as Alzheimer’s, we know exactly what causes it, we just didn’t know how to treat it, I strongly believe we are now moving in the right direction,” added Dr Wild.