Ambroxol - new hope for Parkinson’s

On to Phase 3

Breaking news this morning of what sounds like an important milestone on the road to a cure for the world,’s fastest growing neurological condition. The charity Cure Parkinson’s - which is focused on doing what it says on the tin  - is announcing that a large scala phase 3 clinical trial of Ambroxol, a drug used to treat respiratory diseases, is about to get underway.

The trial will involve 330 people across the UK and will aim to discover whether the drug can slow the onward march of Parkinson’s. Why is this exciting for people who follow the rather tortuous progress of research into a condition which is still not well understood? Slowing the disease may not sound like a cure but right now no drug does that - ever since Levdopa was approved more than fifty years ago the medication has been all about masking the symptoms rather than changing what is going on in the brain.

Now Ambroxol joins five other potentially disease modifying drugs in the final stage of clinical trials with hopes rising that at least one will conclusively prove that it does put the brakes on Parkinson’s. We always have to be cautious in using words such as breakthrough but that would genuinely be a historic day in the battle against this cruel disease.

Exenatide, a repurposed diabetes drug, which has already entered its Phase 3 trial, has also been supported by Cure Parkinson’s. This small charity really is punching above its weight (full disclosure - I am doing some work with its staff, helping them to hone their messaging). The £17 million it has spent so far on research is small change compared to the budgets of big pharma companies but by working with bigger partners such as the Michael J. Fox Foundation and building coalitions behind promising drugs. As a result it reckons it has been able to mobilise something like ten times its own funds behind vital research and trials.

But the road io succes is still long and tricky to navigate.. You might think that using repurposed drugs which are already on the market and have been proved to be safe would speed things up. It does - but not much. It was three years ago that the results of the phase 2 trial of Ambroxol were published and only now is it moving to phase 3 which will probably take another three years. But people with Parkinson’s have learned to be patient and this morning feels like an occasion to be just a little more hopeful.