AAN 2021 | The latest clinical trials in Huntington’s disease: valbenazine, targeting behavior, and novel DMTs

Tiago Mestre, MD, MSC, The Ottawa Hospital Research Institute and University of Ottawa, Ottawa, Canada, shares updates on the latest clinical trials in Huntington’s disease. Several clinical trials of symptomatic treatments for Huntington’s are ongoing, looking to optimize treatment for chorea, as well as address unmet care needs such as irritability, cognition, and functional ability. In addition, the development of novel disease-modifying therapies (DMTs) that target the underlying biology of Huntington’s is a major focus. Antisense oligonucleotides (ASOs) that cause degradation of huntingtin mRNA have been looked into using non-allele specific and allele-specific approaches in the GENERATION HD1 (NCT03761849) and PRECISION-HD (NCT03225833; NCT03225846) trials, respectively. Recently reported data suggested these trials have not been successful. Other investigative approaches to intervene on the huntingtin pathway include RNA interference and splicing modification. This interview took place during the American Academy of Neurology (AAN) 2021 Annual Meeting.