Researchers and organizations dedicated to the study of Huntington's disease are praising a drug that has been shown to significantly affect the nerve-killing protein that causes the disease during early phase clinical trials, The Washington Post reports.
Here are six things to know about the drug and the disease.
1. Huntington's is a hereditary disease in which certain cells in the brain, typically those responsible for an individual's motor and fine motor skills, begin to die, rendering a person unable to walk, talk or reason, but still able to understand language and recognize faces. Symptoms of the disease commonly begin to manifest between the ages of 30 and 50 until it proves fatal. Some patients and experts equate the disease to being diagnosed with Alzheimer's disease, amyotrophic lateral sclerosis and Parkinson's disease all at once, according to the report. Children who posses the mutated gene have a 50 percent chance of inheriting it.
2. The Huntington's gene uses RNA cells to create proteins that kill nerve cells and damage the brain. The drug, IONIS-HTTRx, targets those RNA cells to prevent them from building proteins.
3. The early phase clinical trial, supported by Ionis Pharmaceuticals, involved administering the drug to three dozen subjects diagnosed with the disease to evaluate the drug's safety and tolerability. However, the drug also proved to dramatically curtail the nerve-cell-killing protein in all subjects. No side effects were reported, the report states.
4. "This is the culmination of 20 years of work for all of us. To see that result and to know what it means — it means we've taken a really good promising step," said Blair Leavitt, MD, a professor in the department of medical genetics at the University of British Columbia in Vancouver and lead investigator for the North American group that conducted the study. "I think we're at the end of the beginning and the start of the end. This approach is going to make a difference in the very near future."
5. Researchers also said the method of targeting the central cause of Huntington's could be applied to the treatment of other neurodegenerative diseases, including some forms of Alzheimer's, ALS and Parkinson's, in future studies, according to the report.
6. The phase 3 clinical trial for the drug will begin next year. Researchers said they hope to be able to establish proof of the drug's effectiveness within the next three years.