Trial Testing Cough Medicine Ambroxol Starting Soon

The Phase 3 trial expects to enroll 330 Parkinson's patients in the UK

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by Steve Bryson, PhD |

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The world-first Phase 3 clinical trial testing the ability of ambroxol, a medicine used for decades to treat lung conditions, to slow Parkinson’s disease progression is expected to start in the next few months.

Called ASPro-PD, the end-stage trial is supported by results from the previous Phase 2 AIM-PD clinical trial (NCT02941822) that suggested the therapy may have neuroprotective effects by reducing the levels of toxic clumps of alpha-synuclein protein, a hallmark of Parkinson’s.

“I am delighted to be leading this exciting project,” Anthony Schapira, MD, a professor of neurological science at the University College London (UCL) Queen Square Institute of Neurology, in the U.K., said in a press release.

Conducted in partnership with Cure Parkinson’s in the U.K. and the Van Andel Institute, in Michigan, the trial expects to enroll 330 people with Parkinson’s across 10–12 sites in the U.K. Preparations for recruitment are underway, according to UCL.

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Often administered as the active ingredient in cough syrup, ambroxol (brand names Mucosolvan, Mucobrox, and Mucol, among others) has been widely used to treat respiratory diseases marked by sticky or excessive mucus.

The medicine also is known to boost the activity of beta-glucocerebrosidase (GCase), an enzyme encoded by the GBA gene. Mutations in this gene, resulting in a defective CGase enzyme, are a genetic risk factor for Parkinson’s.

GCase helps break down and recycle waste material from cells, including alpha-synuclein, whose toxic aggregates in Parkinson’s are thought to contribute to nerve cell loss and disease symptoms.

Boosting GCase in the brains of Parkinson’s patients has the potential to improve the body’s ability to clear away these harmful clumps, prevent neurodegeneration, and slow disease progression.

Previous trial results

The previous Phase 2 AIM-PD trial, also led by Schapira, evaluated the safety, tolerability, and pharmacodynamics of six months of treatment with oral tablets of ambroxol in 24 Parkinson’s patients with and without GBA mutations. Results confirmed that the therapy was able to safely access the brain and increase the GCase levels in Parkinson’s patients

These promising findings supported the launch of the upcoming Phase 3 ASPro-PD trial to confirm ambroxol’s therapeutic potential in a larger number of Parkinson’s patients.

“This will be the first time a drug specifically applied to a genetic cause of Parkinson’s disease has reached this level of trial, and represents ten years of extensive and detailed work in the laboratory and in a proof of principle clinical trial,” Schapira said.

ASPro-PD’s participants will be assigned randomly to receive ambroxol or a placebo for two years. The study’s main goal is to assess changes in disease progression through a scale including indicators of motor function and quality of life.

“The study design is the result of valuable input from people with Parkinson’s, leaders in the field of Parkinson’s, trial design and statistics from the UCL Comprehensive Clinical Trials Unit (CCTU), the MHRA [Medicines and Healthcare products Regulatory Agency] and a consortium of funders led by Cure Parkinson’s,” Schapira said. All are “operating as an effective team to ensure we have reached this stage.”

The MHRA is the U.K.’s regulatory body that oversees therapeutic development, similar to the U.S. Food and Drug Administration, and UCL’s CCTU helps design and coordinate multicenter clinical trials.

“We look forward to working with all these groups to ensure successful completion of the study,” Schapira added.

Ambroxol prioritized

Supported by Schapira’s pre-clinical studies identifying ambroxol as a potential Parkinson’s treatment and the positive Phase 2 data, the therapy’s development was prioritized by the international Linked Clinical Trials (iLCT) program, created and operated by the Van Andel Institute and Cure Parkinson’s.

iLCT’s mission is to evaluate potential therapeutics with promising safety data, or in some cases, approved medications for other indications, to slow, stop, or reverse Parkinson’s progression.

“This trial is a big step forward in the search to find new treatments for Parkinson’s,” said Will Cook, CEO of Cure Parkinson’s. “Once the ambroxol trial is underway, it will be one of only six Phase 3 trials on public record of potentially disease-modifying drugs in Parkinson’s, worldwide.”

“We at Cure Parkinson’s are working hard — through our efforts within the iLCT program and in our fundraising efforts — to increase this number significantly in the next few years, to accelerate our progress towards a cure for Parkinson’s,” Cook added.

ASPro-PD, costing more than $6.7 million (£5.5 million), will be funded by the Van Andel Institute, Cure Parkinson’s, and the John Black Charitable Foundation, along with the Parkinson’s Virtual Biotech, the medicinal discovery and development arm of Parkinson’s UK.