On December 2, 2024, University of Iowa Health Care medical researchers discovered a possible drug that could slow the progression of Huntington’s disease. They used clinical information from a database of over 21,000 Huntington’s disease patients. They tentatively concluded that the use of beta-blocker drugs (used for patients with heart and blood pressure issues)

Researchers analyzing large HD registries have found that people with HD mutation who were taking beta-blockers for other reasons (for example to treat high blood pressure, heart disease, or anxiety-related symptoms) tended to develop motor symptoms later than matched people who were not taking beta blockers and show a slower rate of clinical worsening once symptomatic.

The studies so far are purely observational not randomized controlled trials. Observational studies show associations but cannot prove cause and effect due to possible confounding and bias. The authors of this research are calling for prospective trials to test causality. Multiple independent write ups corroborate that the finding came from registry analyses and that more research is needed before any change in clinical care is recommended.

The next step for researchers is to run randomized controlled trials to test whether beta blockers slow biomarker or clinical progression in premanifest and early manifest HD. Several groups and commentators have urged this as the logical next step. Recent registry studies report an association between beta blocker use and delayed motor onset plus slower progression of Huntington’s disease. Rigorous randomized trials and mechanistic research are needed before beta blockers can be recommended as disease modifying treatment.