Aberdeen has recruited the first UK patient for a new trial investigating pridopidine, an oral drug for the treatment of Huntington's disease.
The PROOF-HD study, sponsored by Prilenia Therapeutics, will enrol up to 480 people with early-stage Huntington’s at approximately 60 sites across the US, Canada, and Europe.
Huntington's disease – often known as HD – is a fatal inherited condition that causes neurons in the brain to degenerate. This in turn stops parts of the brain working properly over time.
It causes increasing memory difficulties, psychiatric problems and twitchy movements which the person cannot control.
It is usually fatal around 20 years after it becomes obvious. There is currently no effective treatment to halt or slow its progression.
HD is caused by a faulty gene. Each child of a parent with HD has a 50% chance of developing the condition.
Around one in 7,000 people in Scotland have symptoms of Huntington's and many more live at risk of developing symptoms from the faulty gene.
The PROOF-HD study is a phase 3 clinical trial. It will investigate a drug called pridopidine as a treatment for the disease. In contrast to some other treatments being investigated, the PROOF-HD study only requires participants to take a capsule orally twice a day.
The drug is designed to activate a certain receptor which is highly expressed in the brain called the Sigma-1 receptor (S1R). Activation of the S1R triggers mechanisms that are crucial for maintaining neurons function and survival. This may lead to beneficial effects on functional capacity in HD.
Pridopidine has been tested already in nearly 1000 HD patients and is shown to be safe and well tolerated.
Patients who are eligible for the study will be contacted by their consultant.
Professor Zosia Miedzybrodzka, from the University of Aberdeen, who is leading the study, said: “Huntington’s disease is a serious condition with no known treatments that slow functional decline.
“With no effective treatment currently trials such as PROOF-HD are of huge importance and the promise of effective treatments is really important to families living with the disease.”
Alistair Haw, Chief Executive Officer of Scottish Huntington’s Association, said: “Whilst there are no current treatments that slow the advance of HD, families impacted by this appalling condition can be encouraged by the volume of trials currently underway to find the breakthrough we all long for.
“When this breakthrough comes, as we firmly believe it will, it will be thanks to the courage of family members who selflessly volunteer to take part in such trials combined with the expertise of the world’s top researchers, scientists and clinicians. We are hugely fortunate so have such a dedicated community in Scotland, and we look forward to working with Professor Miedzybrodzka, her team and our HD families as they take this exciting new research project forward.”