Tears of joy as scientists close to Huntington’s cure

Doctor patient

​Breakthrough as new drug offers huge hope for patients with life shortening disease

12th December 2017 by Robert Armour 3 Comments

A groundbeaking drug could offer a cure for Huntington’s disease.

Results of a new trial have for the first time corrected the defect that is responsible for the devastating neurodegenerative disease.

The results, hailed as the biggest breakthrough in 50 years, saw 46 patients of the incurable disease given an experimental drug.

University College London (UCL) researchers discovered the pill lowered their levels of toxic proteins in the brain.

Researchers called the the drug a "tremendous step forward". It has also been found to be safe despite fears it could lead to meningitis. 

Progression of the inherited disease could be slowed or even halted by the new treatment.

Prof Sarah Tabrizi, lead researcher and director of the Huntington's Disease Centre at UCL, said: "I've been seeing patients in clinic for nearly 20 years, I've seen many of my patients over that time die.

"For the first time we have the potential, we have the hope, of a therapy that one day may slow or prevent Huntington's disease.

"This is of groundbreaking importance for patients and families."

Huntington's disease is a genetic disorder that affects the central nervous system and leads to involuntary movements, difficulty talking and memory loss. 

The condition, which affects 8,500 adults in the UK and around 30,000 in the US, occurs as a result of a fault in the huntington gene.

On average, patients live for between 10 and 20 years after their diagnosis. 

Neuroscientist Marie Short, 46, has the disease and is a trustee of the Scottish Huntington’s Association (SHA).

She says she is excited by the findings.

"I am a neuroscientist. I know how specific it is and how important it is and I am absolutely thrilled. It's not a total cure but it's the next best thing at the moment," she said.

"I have not felt like this way about any other study that has come out ever. I am just ecstatic.

"I phoned my mum and we had a little cry. It's just phenomenal.”

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