Olympian says shadow has lifted after Huntington’s breakthrough

AN Olympic medallist from Henley who carries the mutated gene that causes Huntington’s disease said the announcement of the first successful treatment of the disease felt “life-changing”.

Sarah Winckless, a former Team GB rower and patron of Scottish Huntington’s Association, said the breakthrough had come after “a number of false dawns” in the race to treat it over the years.

Huntington’s is a rare and devastating inherited disease. It affects the nervous system, killing brain cells and impacting on movement, thinking and mental health, and until last week was thought to be incurable.

However, a new gene therapy trialled by uniQure has been heralded as a breakthrough after a three-year trial found the drug slowed progression of symptoms by 75 per cent.

Winckless, who lost her mother, Valerie, to the disease in 2019, said the breakthrough was “huge”. She said: “Hearing the news just felt life-changing.

“In the past we’ve had some positive ideas in mouse models and we think when we put it into a human model it will work and then that hasn’t been the case, so we’ve had a number of false dawns over the years. This is the first time that scientists have been able to slow the disease in this amount.”

Winckless, who won a bronze medal in the double sculls at the 2004 Athens Olympic games, said it was “incredibly tough” watching the symptoms alter her mother’s life.

She said that by the 2004 games, her mother had lost her ability to walk and was confined to a wheelchair. Four years later at the Beijing Games, she was then unable to travel and had lost most of her verbal communication.

A person with the disease has a 50 per cent chance of passing the mutated gene down to their child, with symptoms typically appearing in your thirties and forties. It is normally fatal within 10 to 20 years.

Following her mother’s diagnosis, Winckless decided to be tested and found she had inherited her mother’s gene.

She said: “Over six months I got my genetic testing and I learned that I had inherited mum’s genetics. I was young and I was a sportswoman so I took a lot of solace that if I was still doing my sport at a high level I was probably doing good things for my body. It has been a shadow over my existence I think and that shadow feels a lot lighter right now.”

UniQure hopes to get regulatory approval in the US next year. It would then be processed by the UK regulator, the Medicines and Healthcare Regulatory Agency, to determine it is safe and effective. It would then require approval from National Institute for Health and Care Excellence to decide if it is affordable for the NHS.

The drug is given directly to the brain during a 12- to 18-hour surgery, bypassing the blood-brain barrier.