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Aberdeen University is the first in UK to recruit for a new Huntington’s disease trial


By David Porter

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Aberdeen University has recruited the first UK patient for a new trial investigating pridopidine, an oral drug for the treatment for Huntington’s disease.

Professor Zosia Miedzybrodzka is leading the study
Professor Zosia Miedzybrodzka is leading the study

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 and each child of a parent with HD has a 50 per cent or one in two chance of developing the condition.

Around one in 7000 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.”


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