By looking at a database of individuals with declared Huntington’s disease (HD) or in the pre-symptomatic stage (pre-H), a team from the University of Iowa established an association between the use of beta-blockers (BB) and the slowing of disease progression. For pre-symptomatic individuals, illness onset was delayed in those who took BBs for heart conditions compared to those who did not. In patients with a more advanced form, the worsening of symptoms was slowed under BB.
For researchers, this effectiveness could be explained by the blocking action of norepinephrine exerted by BB, norepinephrine being a molecule involved in the “fight or flight” response (response to stress). “Individuals with HD have a more active sympathetic nervous system, and theoretically more norepinephrine, which increases the fight or flight response, and could contribute to neurodegeneration,” they detail. The results of this study funded by the National Institutes of Health (NIH) are published in the journal Jama Neurology.
Analysis of nearly 650 individuals
The University of Iowa team exploited the largest observational HD database: Enroll-HD. This cohort of more than 21,000 people includes patients with HD and individuals at risk of developing HD in their lifetime. For this study, the researchers carried out the analysis on 646 matched individuals and divided into two groups: a group of carriers of the mutation which causes HD, but whose clinical symptoms are not declared (pre-HD) and a group having already received a clinical diagnosis (MH). In both groups, researchers identified those who took BBs for a heart condition and those who did not.
They thus noted that BB users in the pre-H group (n = 174) had a significantly lower annual risk of clinical diagnosis of HD (HR = 0.66) compared to those who did not take it (n = 174). In the HD group, BB users (n = 149) showed a significant slowdown in the worsening of their motor (mean difference = -0.45), cognitive (MD = 0.10) and functional (MD = 0) symptoms. .10) compared to those who did not take it (n = 149).
A potential disease-modifying treatment
According to the authors, these results show the potential of BB as a modifying treatment for Huntington’s disease at different stages of the disease. “Given that there are no disease-modifying agents, the possibility that beta-blockers, which are inexpensive and have a known safety profile, could benefit patients at different stages of the disease is very exciting,” explain the authors.
However, they temper their conclusions explaining that the data do not demonstrate a cause and effect relationship, but only an association. They nevertheless affirm that their work points to the autonomic nervous system as a potential therapeutic target in this pathology.