The Hunt to End Huntington’s

On Dec. 11, possibly the biggest breakthrough in neurodegenerative diseases in the past 50 years was made when the defect that causes Huntington’s disease was corrected in patients for the first time. If this form of drug— called lonis-HTTRx— could be adapted to target harmful proteins in other incurable brain disorders, diseases such as Alzheimer’s could possibly be cured.  

An experimental drug, when injected into spinal fluid, successfully reduced the levels of the toxic protein that likely causes Huntington’s, further proving that there may actually be a cure for the inherited disorder. Mainly affecting motor and cognitive abilities, Huntington’s is similar to Alzheimer’s and Parkinson’s in that it frequently results in dementia and alters the mental processes of perception, memory, behavior and judgement. An estimated 30,000 Americans are currently living with the disease.

“You end up in almost a vegetative state, it’s a horrible end,” said Peter Allen, a 51-year-old Huntington’s sufferer and participant in the initial medical trial.

The trial consisted of 46 patients who had the drug injected into the fluid that connects their brain and spinal cord. Doctors then measured the levels of the toxic protein in the patients, finding a significant decline in levels of huntingtin after administering the drug lonis-HTTRx. Carried out by Leonard Wolfson Experimental Neurology Center in London, the experimental trial was highly uncertain and doctors genuinely didn’t know what would happen. Obviously, they were pleased with their findings.

“The results of the trial are of groundbreaking importance for Huntington’s disease patients and families,” said Sarah Tabrizi, the leader of the trial and director of University College London’s Huntington’s Disease Center. “For the first time we have the potential, we have the hope, of a therapy that one day may slow or prevent the disease.”