Breakthrough drug found to treat Huntington’s disease: report
In a major breakthrough, researchers have found a drug that could offer treatment for Huntington’s disease.
Huntington’s disease is a genetic disorder that affects the central nervous system and leads to involuntary movements, difficulty in talking and memory loss.
According to a report by BBC, inherited neurodegenerative Huntington’s disease progression could now be slowed or even halted by an experimental drug which has proved successful in early patient trials.
Researchers at the University of London have for the first time demonstrated that an experimental drug, injected into spinal fluid, can suppress the genetic defect by lowering the levels of toxic proteins in the brain. Initial results were announced Monday.
The results, hailed as the “biggest breakthrough in 50 years”, saw 46 patients of the incurable disease being given an experimental drug into their spinal fluid at the National Hospital for Neurology and Neurosurgery. Each patient received four doses of either the new drug or a placebo. The patients were enrolled across nine centres in the UK, Germany and Canada.
The new drug works by silencing the faulty Huntingtin gene—stopping the creation of damaged proteins in the brain, the BBC reported. BBC quoted Sarah Tabrizi, lead researcher, as saying, “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.”
Tabrizi, director of UCL’s Huntington’s Disease Centre, added: “This is of groundbreaking importance for patients and families.”
Currently treatments only exist for the symptoms of Huntington’s, which usually begin to appear between the age of 30 and 50 years, leading to patients’ gradual decline over the next 10 to 25 years.
The results of the new drug, Ionis-HTTRx, which is owned by Roche, created by Ionis Pharmaceuticals, are expected to published in a journal next year.
Neurologists in India say though the discovery of a new drug open up a whole new world of possibilities for treatment in India, there is still a long way to go. “That’s small data and still long way till it gets established as a drug for Huntington’s. But the preliminary results in first in human trial and concept of genetic therapy is encouraging and even if we will be able to half halt the progression and not cure it, it’s going to be a big boon and hope to all Huntington’s patients and families who otherwise have a dismal prognosis,” said Dr. Kaustubh Mahajan, neurologist, SL Raheja Hospital, a Fortis associate.
The number of Huntington’s disease patients in India is increasing, according to neurologists, though there is no registry that provides estimates for the incidence of the disease.
“This is a promising study in Huntington for which no definitive treatment is available till date and for which we could only treat patients symptomatically. We need a larger study to see whether genetically modifying production of Huntington protein translates into slowing down of the degenerative disease process. Any way it’s a ray of hope for thousands of patients worldwide suffering from this untreatable disease,” said Dr. Anshu Rohatgi, senior consultant neurologist at Sir Gangaram Hospital.