A clinical study evaluating Isis Pharmaceuticals' investigational gene therapy ISIS-HTT in the treatment of Huntington disease has been initiated, Vancouver Sun reported.
The early-stage study of 36 patients is designed to confirm the safety of the treatment in patients with very early stages of the disease.
ISIS-HTT includes strands of artificial DNA that bind selectively with messenger RNA generated when the huntingtin gene is activated, preventing production of the lethal protein.
"You can target pretty much any gene in the body with a specific set of codes and that’s what makes this therapy so powerful," explained Blair Leavitt, director of the Centre for Huntington Disease at the University of British Columbia, adding "you can build a sequence of nucleotides that are present in one gene and nothing else, so they only bind to that RNA and they only degrade that RNA."
"This is the first gene-silencing drug that we have; it's now in safety trials and it is incredibly promising," commented Bev Heim-Myers, executive director of the Huntington Society of Canada, continuing "this is the first drug that will address the root cause of Huntington Disease."
"[Gene-silencing] drugs have great potential for many neurodegenerative diseases because they can be tailored to modify the production of any target protein," noted Frank Bennett, senior vice-president of research at Isis Pharmaceuticals, adding "Huntington is ideally suited to this innovative therapeutic technology because it comes with genetic certainty: everyone with the mutant gene will get the disease at some point."
