Huntington’s disease is a genetic disorder that results in the breakdown of the brain’s nerve cells. This leads to a decline in mental and physical capabilities. Over 30,000 U.S. citizens currently are symptomatic for the disease with more than 200,000 being at risk of developing it. Right now, there is no cure for Huntington’s, but a recent study shows that a new drug might have the potential for future treatment.
The drug in question is called RG6042. It is an agent that targets the mRNA of the mutant protein believed to cause the disease. This protein is caused by huntingtin (HTT) protein. The study found that after administering the medication, levels of this protein decreased.
For patients who took 90mg doses every four weeks, the mutant protein levels dropped by 40% vs those who took a placebo. There was a 60% decrease in this protein for those who took 120mg. The study also tried to figure out what dosage and timing of dosages were optimal. Researchers also report that taking the medication was proven to be safe.
This is still very preliminary research on RG6042. The next step is to research if it can show clinical effect in larger sample sizes over a longer period. However, this is a promising first step towards potential treatment options.
Tabrizi, Sarah J., et. al., “Targeting Huntingtin Expression in Patients with Huntington’s Disease | NEJM.” New England Journal of Medicine, 6 May 2019, www.nejm.org/doi/10.1056/NEJMoa1900907.