Huntington's Disease Treatments Evolving

New medication to treat Huntington's disease tested in mice and monkeys, now in human trials.


Huntington's disease has always been a genetic condition with no treatment available, but new treatment options are possible.

Researchers from the University of British Colombia have developed a new medication. The new drug, IONIS-HTTRx, may prevent the mutated gene from destroying nerve cells and causing the symptoms associated with Huntington's. The medication was tested in mice and monkeys, and found to be effective.

Huntington's disease is hereditary. Each child of a parent with Huntington's disease has a 50 percent chance of developing the disease.

The disease causes damage to nerve cells. The damaged nerve cells bring about symptoms such as uncontrolled muscle movements, however symptoms don't begin until patients are in their 30s or older.

As the disease progresses, patients lose intellectual abilities and develop emotional problems. Those who inherit the mutated gene that causes Huntington's disease will eventually develop symptoms.

Principal investigator Blair R. Leavitt, M.D., said in a press release, “It is very exciting to have the possibility of a treatment that could alter the course of this devastating disease. Right now we only have treatments that work on the symptoms of the disease.”

Dr. Leavitt is a neurologist and professor in the Department of Medical Genetics at the University of British Columbia.

IONIS-HTTRx is a “gene silencer” medication, according to Leavitt. This means it inhibits the production of the protein that actually causes disease symptoms.

The medication must be administered into the spinal fluid so that it will travel to the brain and nervous system. The current human trials use four doses of the medication administered once a month. The animal trials saw long-lasting effects.

However, Leavitt said it will probably be years before the medication is available for patients outside of clinical testing.

Data from the most recent clinical trials was presented at the American Academy of Neurology’s 68th Annual Meeting in February.

The clinical study is supported by Ionis Pharmaceuticals, which helped develop IONIS-HTTRx.

Information on conflict of interest was not available.