The asthma drug salbutamol, a β2-adrenoceptor agonist, may reduce the risk of Parkinson disease (PD), suggests epidemiological and basic research published in Science .

PD is a neurodegenerative condition that is characterized by the accumulation of α-synuclein in the brain. When researchers at Harvard Medical School in Boston set out to find drugs that could lower α-synuclein expression, they discovered that salbutamol offered promise in both in vivo and in vitro models. Collaborating with researchers in Norway, they performed a large-scale epidemiological study of 4 million Norwegians to show that people who used this drug for the treatment of asthma were approximately one-third less likely to develop PD than people who did not use the drug. Conversely, Norwegians who used the β2-adrenoceptor antagonist propranolol to control high blood pressure were twice as likely to develop the neurodegenerative disease.

These results do not prove causation, but the real-world and mechanistic evidence suggest that salbutamol or another β2-adrenoceptor agonist could provide a path forward for PD treatment. “Evaluation in additional populations and in clinical trials will be required to determine whether the insights gained in this work can be translated to patients with PD,” the authors write.

“Our study presents a path to drug development that is distinct from traditional approaches. Targeting the endogenous expression of a human disease gene may be a useful strategy for other diseases attributed to copy number variation or regulatory variants. The drug development pipeline tested in this study could be more generally applicable to rapid discovery and translation of therapeutics for other brain diseases,” they add.

Earlier this year Roche advanced the first anti-α-synuclein antibody into phase II trials (Nat. Rev. Drug Discov. 16, 371–373; 2017).