Introduction: Huntington's disease is a hereditary neurodegenerative disease. It is designated as a rare disease in the US, which means there are < 200,000 patients in the country who suffer from it. The drugs that are currently used to treat this disease were not designed specifically for it but developed for other diseases. Presently, two classes of drugs are being developed; those that provide symptomatic relief and those that may modify course of the disease.
Areas covered: This review is focused on seven selected drugs currently in clinical testing and describes their progress. Five of the seven drugs that are reviewed here, can be categorized as 'symptomatic' drugs, and, selisistat and PBT-2 are amongst the ones that would qualify as 'disease modifying' drugs.
Expert opinion: The authors believe that the future treatment paradigm for this disease is best met by using a disease-modifying drug that can be administered together with symptomatic drugs. Towards that end, it is important for the industry to focus on disease-modifying drugs by targeting unique pathways and targets. Furthermore, they propose that neuroprotective drugs, that is, drugs that directly work by preserving neuronal health and function is an opportunity for such disease-modifying drugs.
Keywords: Huntington’s disease; chorea; cognition; huntingtin; motor impairment; neurodegeneration; rare diseases.