ambroxol trials any participants seen ben... - Cure Parkinson's

Cure Parkinson's

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ambroxol trials any participants seen benefits?

Coling•

17 days ago•13 Replies

i have been invited on a phase 2 trial of the above. I wonder if any conclusions can yet be drawn from this trial?

• Ambroxol,

• Doxycycline, or

• Ambroxol and Doxycycline combined.

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Coling

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13 Replies

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BlockRuys17 days ago

I am part of phase 3 Ambroxol study in the Netherlands. No benefits, pretty sure its placebo for me. The data is promising though especially for GBA1 carriers.

JakeMax in reply to BlockRuys16 days ago

Hi Block, what does the data say for GBA1 carriers?

Many thanks

BlockRuys in reply to JakeMax16 days ago

Here are some great references:

- Ambroxol - jamanetwork.com/journals/ja... && cureparkinsons.org.uk/2023/...

* The study results suggest that ambroxol therapy was safe and well tolerated; CSF penetration and target engagement of ambroxol were achieved, and CSF α-synuclein levels were increased. Placebo-controlled clinical trials are needed to examine whether ambroxol therapy is associated with changes in the natural progression of PD.

- Ambroxol reverses tau and α-synuclein accumulation in a cholinergic N370S GBA1 mutation model — ncbi.nlm.nih.gov/pmc/articl...

* Ambroxol increases glucocerebrosidase (GCase) activity and restores GCase translocation in primary patient-derived macrophages in Gaucher disease and Parkinsonism - pubmed.ncbi.nlm.nih.gov/336...

- Molecular mechanisms of the ambroxol action in Gaucher disease and GBA1 mutation-associated Parkinson disease - sciencedirect.com/science/a...

* One such a molecule that acts as a pharmacological chaperone for GCase was found to be ambroxol (2-amino-3,5-dibromo-N-[trans-4-hydroxycyclohexyl]benzylamine; Figure 1), found naturally in the Asian shrub Adhatoda vasica.

* However, some data have begun to question its activity as a chaperone against certain GCase variants. Then, a number of articles appeared pointing to other mechanisms of action of ambroxol, which may also contribute to the improvement of patients' condition.

JakeMax in reply to BlockRuys16 days ago

Thanks Block!!!

Coling in reply to BlockRuys15 days ago

Thankyou

Jessica-1 in reply to BlockRuys11 days ago

have phase 3 results been published? How many people participated phase 3 trials? Many thanks.

Coling in reply to BlockRuys10 days ago

Thanks BlockRuys when do you expect the results to be released?

BlockRuys in reply to Coling10 days ago

late 2025

Stillstandingstill16 days ago

Would individuals notice the benefit? It's my understanding that Ambroxol might slow progression, so without a control group an individual taking it wouldn't be able to gauge if it was helping or not. We will know in time as trials start to produce results.

Jessica-1 in reply to Stillstandingstill15 days ago

The results that you mentioned are from phase 3 or phase 2 trail? Thanks.

Stillstandingstill in reply to Jessica-115 days ago

A phase 3 trial funded by the Cure Parkinson's Trust is just about to start in the UK. Sounds like there are other trials elsewhere too.

Coling14 days ago

yes I am thinking has anyone had beneficial results from phase 2 trials ?

BlockRuys11 days ago

YES!

"Mean (SD) scores on part 3 of the Movement Disorders Society Unified Parkinson Disease Rating Scale decreased (ie, improved) by 6.8 (7.1) points (95% CI, –10.4 to –3.1; P = .001). These changes were observed in patients with and without GBA1 mutations."

jamanetwork.com/journals/ja...

7 points is a lot better in MDS-UPD III.