What Is Huntington's Disease? Experimental Drug Promises To Curb Brain Condition

A new experimental drug developed by a team of researchers from the University College London promised to curb the deadly Huntington’s Disease. The drug, which is to be injected into the spinal fluid, gave a ray of hope after it was tested on some patients during a trial.

The clinical trial conducted at the Leonard Wolfson Experimental Neurology Centre at the National Hospital for Neurology and Neurosurgery in London consisted of 46 patients. During the trial, the researchers injected a synthetic molecule (experimental drug) into the fluid that bathes the brain and spinal cord of the subjects. The experiment appeared to prevent the mutated Huntington gene from producing the faulty protein.

With this experiment, the researchers derived if the drug inserted into the patients succeeds in preventing the Huntington gene from producing the faulty protein, it could even effectively cure the deadly disease.

Peter Allan, 51, from Essex, a county in southeast England, who took part in the trial, said of the disease: “You end up in almost a vegetative state, it's a horrible end. It's so difficult to have that degenerative thing in you. You know the last day was better than the next one's going to be.”

“Huntington’s Disease (HD) is a fatal genetic disorder that causes the progressive breakdown of nerve cells in the brain. It deteriorates a person’s physical and mental abilities during their prime working years and has no cure. HD is known as the quintessential family disease because every child of a parent with HD has a 50/50 chance of carrying the faulty gene,” the Huntington’s Disease Society of America (HDSA) said on its website.

HDSA, on its website, also stated “approximately 30,000 Americans have HD, but the devastating effects of the disease touch many more. Within a family, multiple generations may have inherited the disease. Those at-risk may experience tremendous stress from the uncertainty and sense of responsibility.”

According to Mayo Clinic, the symptoms of Huntington’s Disease include movement, cognitive and psychiatric disorders along with behavioral and physical changes.

The website also stated the common causes of death due to the disease include pneumonia or other infections, injuries related to falls, and complications related to the inability to swallow.

According to a BBC report, experts said the experimental drug could be the biggest breakthrough in neurodegenerative diseases in almost 50 years.

The new drug could possibly reverse the effect of Huntington’s Disease or at least slow down its effect to a large extent.

Speaking to BBC, Prof Sarah Tabrizi, the 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 ground-breaking importance for patients and families,” she added.

“I really think this is, potentially, the biggest breakthrough in neurodegenerative disease in the past 50 years. That sounds like hyperbole - in a year I might be embarrassed by saying that - but that's how I feel at the moment,” John Hardy, a neuroscientist studying Alzheimer’s disease at the University College London told the BBC.