Breakthrough hailed as miraculous but services in Scotland could suffer
A Scottish charity has hailed a breakthrough in treatment for Huntington's disease.
Researchers have revealed gene therapy has slowed the illness’s progression by 75% - effectively meaning people with the disease can live nearly normal lives.
The disease develops in families, kills brain cells and appears as a combination of dementia, Parkinson's and motor neurone disease.
CEO of Scottish Huntington’s Association, Alistair Haw, said: “These are the best results to date from any clinical trial aiming to slow the progression of Huntington’s disease. After many years of hope and false dawns, this is a day for rejoicing for the global Huntington’s community. The first treatment for this horrendous condition appears to finally be appearing at the end of this long dark tunnel.”
The new treatment is a type of gene therapy given during 12 to 18 hours of delicate brain surgery.
The first symptoms of Huntington's disease tend to appear in your 30s or 40s and is normally fatal within two decades – opening the possibility that earlier treatment could prevent symptoms from ever emerging.
Prof Tabrizi, director of the University College London Huntington's Disease Centre, described the results as "spectacular".
"We never in our wildest dreams would have expected a 75% slowing of clinical progression," she said.
However, Alistair Haw warned of the need to ensure that Scotland’s health and social care system is ready for the treatment.
“At present, people in a Huntington’s family who know they are at risk of inheriting the disease from a parent overwhelmingly choose not to be tested to find out if they have the faulty gene. With a treatment on the horizon, this will all change. More people will come forward requiring specialist help and support with this process. This will require more capacity in the system to cope with the demand, not less.
“Sadly, Glasgow City HSCP is planning to become the first area in history to defund our Huntington’s Disease Specialist Service. This was always an ill-advised false economy, but in light of today’s news this plan becomes an ill-advised false economy on steroids.
“After decades of progress Scotland is a world leader in providing specialist Huntington’s care and support thanks to services provided by Scottish Huntington’s Association backed by a Scottish Government supported National Care Framework. At this time of all times, we need to grow these services, not reduce them.
“Unless health and social care providers grasp this reality, and now, Scotland risks snatching defeat from the jaws of victory on this otherwise unprecedented day of celebration for the Huntington’s community. And that would be unforgiveable. We call on the Scottish Government to unite with us to prevent this from happening.”
