Cough syrup drug passes another trial as Parkinson’s treatment

Mr Peter Dockrill

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 an escalating dose of ambroxol to 1.26g per day.

In all participants, the drug crossed the blood-brain barrier and increased levels of GCase in cerebrospinal fluid by roughly 35 percent. No adverse effects were reported.

These results were seen in patients with and without the GBA1 mutation that impairs release of GCase, a surprise finding that will require further analysis in later studies.

There was also some improvement in patient’s motor control, an average of nearly nine points on total MDS-UPDRS score. The researchers stress that this is not a conclusive result however, given that it was a secondary outcome of a small trial with no control group.

If ambroxol is found to be an effective treatment for Parkinson’s disease it will be an incredibly significant development, given that all current medications only address the symptoms. This will hopefully be determined in the upcoming Phase III trials, the first step of which will be to establish the optimal dose.

>> Read the original article here

Source: ScienceAlert

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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Mr Peter Dockrill

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Mr Peter Dockrill

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