Cough syrup drug passes another trial as Parkinson’s treatment

Cough syrup drug passes another trial as Parkinson’s treatment

The search for a treatment to slow, stop or reverse Parkinson’s disease has found a promising, if unlikely, candidate; a cough syrup drug in use since the 1970s.

A research team led by University College London reported the results of a Phase II clinical trial on the use of ambroxol for the treatment of Parkinson’s disease in January. The results indicate that the drug is safe for patients with the disease, provided ‘proof of concept’ for a mechanism by which it may slow progression, as well as hinting at positive effects on motor control.

Based on these encouraging results, the team’s funding is being continued to conduct Phase III trials with a much larger cohort.

The focus of the study was on whether the drug would penetrate the blood/brain barrier and increase levels of glucocerebrosidase proteins (GCase), which clean the bran and prevent build-up of harmful alpha-synuclein proteins, the most likely cause of Parkinson’s disease.

In the open-label, non-controlled trial 17 patients with Parkinson’s disease of moderate severity were assessed over 6 months while taking ...


Mullin S, Smith L, Lee K, D’Souza G, Woodgate P, Elflein J. Ambroxol for the Treatment of Patients With Parkinson Disease With and Without Glucocerebrosidase Gene Mutations: A Nonrandomized, Noncontrolled Trial. JAMA Neurol. 2020 Jan 13. DOI: 10.1001/jamaneurol.2019.4611 [Epub ahead of print]

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