Authors: Alice Bough (Future Science Group)
A recent clinical trial has demonstrated that the drug, IONIS-HTTRx, is safe for use in people with Huntington’s disease.
An international team of researchers has conducted a randomized, double blind, Phase I-IIA clinical trial for the use of IONIS-HTTRx in the treatment of Huntington’s disease. The main aim of the paper, published in the New England Journal of Medicine, was to determine whether the drug was safe for use in people with Huntington’s disease.
Utilization of the antisense oligonucleotide IONIS-HTTRx in preclinical models has demonstrated that the drug inhibits synthesis of the mutant huntingtin protein, which causes Huntington’s disease.
From study centers across Canada, Germany and the United Kingdom, 46 individuals with Huntington’s disease were recruited. Each participant received four intrathecal doses of the drug or placebo at 4 week intervals, subsequently to be monitored for adverse effects.
While 98% of all participants experienced adverse effects, none experienced any serious adverse effects. This suggests that the drug is safe for use in people with Huntingdon’s disease.
“This is a tremendously exciting and promising result for patients and families affected by this devastating genetic brain disorder,” commented Blair Leavitt (University of British Columbia; BC Canada).
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The researchers also performed pharmacokinetic studies on the cerebrospinal fluid of the participants. In comparison with the placebo group, the team observed a dose-dependent decrease in the concentration of mutant huntingtin protein.
“For the first time, we have evidence that a treatment can not only decrease levels of the toxic disease-causing protein in patients, but that it is also safe and very well tolerated,” concluded Leavitt.
While is known that IONIS-HTTRx inhibits production of the mutant huntingtin protein, how this can affect the progression of Huntington’s disease is yet to be elucidated. Hopefully, the answer will be clarified following Phase III trials currently being performed at Huntington’s disease centers around the world.
Sources: Tabrizi SJ, Leavitt BR, Landwehrmeyer GB et al. Targeting huntingtin expression in patients with Huntington’s disease. N. Engl. J. Med. doi: 10.1056/NEJMoa1900907 (2019) (Epub ahead of print); https://news.ubc.ca/2019/05/06/huntington-drug-successfully-lowers-levels-of-disease-causing-protein/