Publication date: May 10, 2019
Scientists are gearing up a major study to find out whether a drug can silence the gene that causes a devastating illness called Huntington’s disease.
The new study will determine whether that drug can also stop progression of the disease.
It is also another sign that drugs built with DNA, or its cellular collaborator RNA, can be powerful tools for tempering diseases that until now have seemed out of reach.
Huntington’s disease is an apt target because it’s caused by a single mutated gene.
Jeanette Garcia discovered through genetic testing that she is going to develop Huntington’s disease, eventually.
Courtesy of Jeanette Garcia hide caption toggle caption Courtesy of Jeanette Garcia Jeanette Garcia discovered through genetic testing that she is going to develop Huntington’s disease, eventually.
Last year, drugmaker Roche’s Genentech unit said that an experimental drug sharply reduced the amount of illness-inducing protein measured in people’s spinal fluid.
The researchers are now about to launch a trial involving 660 volunteers with early symptoms of the disease, to see if the drug, called RG6042, can slow or stop Huntington’s progression.
“The drug could actually transfer quite readily to the brain and then sink into the target brain tissue,” says Dr. Scott Schobel, who heads the research effort on this drug at Roche, which is co-developing the experimental drug with Ionis.
George Yohrling, a scientist at the Huntington’s Disease Society of America, says his main concern is whether the experimental drug will penetrate deeply enough into the brain to stop the disease.
As scientists gradually worked their way through these challenges, Huntington’s disease emerged as an appealing target, despite being a rare disease with a far smaller potential market than, for example, a drug for Alzheimer’s disease.
And there are now competitive products targeting that disease, thanks in part to the financial incentives drug companies get to develop drugs for “orphan” diseases.
Before the FDA even considers approving a treatment for Huntington’s, Roche will have to demonstrate that its experimental drug is safe and effective.
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