Cough medicine to be trialled for MND treatment
by Professor Brad Turner
Researchers at the Florey Institute will lead a world-first clinical trial of Ambroxol as a potential treatment for MND. Ambroxol is a generic drug found in cough syrup and has been used to treat respiratory conditions for over 30 years with an excellent safety profile.
Ambroxol is a mucolytic agent (drug that helps to clear mucus from the respiratory tract) which helps break down phlegm and acts as a cough suppressant.
Now, research led by Professor Brad Turner at the Florey Institute, University of Melbourne, has discovered a novel action of Ambroxol for MND. His group has shown that Ambroxol works by reprogramming metabolism inside motor neurons, leading to stronger physical connections between motor neurons and muscles. These connectionsare thought to be disrupted early in MND, leading to characteristic muscle weakness and wasting. Accordingly, Ambroxol treatment improved disease in models of MND across different laboratories, supporting its potential for slowing down MND progression.
This Phase 2 trial called AMBALS will launch in March 2023. This clinical, multi-centre trial will recruit 50 MND patients across Australia for treatment with escalating oral doses of Ambroxol for 6-months with an option for a 6-month open label extension. Participants will receive either Ambroxol or a placebo treatment. The effectiveness of Ambroxol will be measured using clinical rating scores, questionnaires, electrodiagnostic tests and blood biomarkers of disease progression.
Professor Turner said, “We are excited to lead this trial which represents five years of extensive research across an international team of collaborators. Ambroxol is an ideal drug candidate for MND. It is a safe drug, taken orally as a syrup and readily enters the brain. We will repurpose Ambroxol for MND for the first time. We look forward to working with the MND community in this trial.”
Professor Brad Turner is leading the trial.
The AMBALS trial is funded by FightMND and the Australian Government. Patient recruitment for this trial opens February to September 2023 at 5 clinical sites, including Calvary Health Care Bethlehem, VIC; Concord Hospital, NSW; Brain and Mind Centre, NSW; Flinders University Medical Centre, SA; and Launceston General Hospital, TAS. The expected trial completion will be late 2024. Please speak with your neurologist if you are interested in participating in this trial.