‘This new treatment brings hope’ – families on Huntington’s treatment

Families have expressed their excitement over the news of the first treatment for Huntington’s disease.

People affected by the debilitating disease said news of the first disease-modifying treatment offers “hope”.

It has also been described as “bittersweet” with some affected families knowing the drug will not be available until it is “too late” for them.

The condition, which affects almost all aspects of a person’s life including their movement, thinking and mood, gets worse over time.

There is currently no cure but scientists have just published a study on the first-ever treatment for the disease.

Experts tested a new gene therapy, AMT-130, which is delivered by brain surgery.

Those who were given a high dose of the treatment experienced 75% less disease progression after 36 months, according to gene therapy company uniQure and scientists from University College London.

Megan Ashmore’s father Simon died from Huntington’s disease in 2024 when he was 58 year old.

People with an affected parent have a 50% chance of inheriting the mutation which causes Huntington’s disease.

The 24-year-old from Nottingham does not know whether she has inherited the gene.

Asked about the results from the study, she told the PA news agency: “It is fantastic news for the community and for all individuals and families impacted by Huntington’s disease.

“I think for some it will be bittersweet as it may be too late for some individuals, but it brings a lot of hope for individuals living at risk, or those who are not yet symptomatic, and for future generations.

“It makes me emotional as it really has such positive results and will soon hopefully be implemented and accessible.”

She added: “The impact this has on individuals and families is huge, as it allows for people to be hopeful and to not be scared of their own future.

“For lots of individuals impacted by HD they have seen how the disease impacts family members and how this may be their own future can cause a lot of anxiety.

“It will mean that their quality of life will be able to continue, and further treatment will then also be developed.”

Ms Ashmore continued: “My dad was diagnosed with HD in 2012, he was already symptomatic but there was no other history of HD in my family.

“We lost my dad in December 2024, HD took so much away from his life in his final stages he was unable to talk, walk and required a lot of care due to a loss of independence.

“I am at a 50:50 risk of inheriting the faulty gene which causes Huntington’s disease and will go through testing before I have children of my own.

“I am also an ambassador for the Huntington’s Disease Association and also a part of the Huntington’s Disease Youth Voice. I also have an Instagram account where I share my story about being impacted by HD (@memydadandhd).”

Cath Staney, chief executive of the Huntington’s Disease Association, said: “This is a significant breakthrough, and I am sure it will bring hope to anyone affected by Huntington’s disease.

“This trial shows an astonishing 75% slowing of progression in the disease. That is remarkable and will bring much-needed optimism to the Huntington’s disease community.”