The first UK participant has been recruited onto the new Proof HD trial into medication for people with Huntington’s disease.
The PROOF-HD study will enrol up to 480 people with early-stage Huntington’s at around 60 sites across the US, Canada, and Europe.
Aberdeen University has now engaged its first patient, spearheading efforts in Scotland and the wider UK in the landmark study.
PROOF-HD, sponsored by Prilenia Therapeutics, is a phase three clinical trial. It will investigate the drug pridopidine as a treatment for the disease. In contrast to some other treatments being investigated, participants are required to take a capsule orally twice a day.
“Huntington’s disease is a serious condition with no known treatments that slow functional decline,” says Professor Zosia Miedzybrodzka, from the University of Aberdeen, who is leading the study.
“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.”
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 neuron function and survival. This may lead to beneficial effects on functional capacity in Huntington’s.
Pridopidine has been tested already in nearly 1,000 Huntington’s patients and has been shown to be safe and well tolerated.
Huntington’s disease is an inherited condition that causes neurons in the brain to degenerate, leading to parts of the brain being unable to function over time. It causes increasing memory difficulties, psychiatric problems and movements which the person cannot control.
Currently, there is no effective treatment to halt or slow its progression.
Alistair Haw, chief executive of Scottish Huntington’s Association, says: “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.”
