Clinical trial examines whether Ambroxol can slow dementia in people with Parkinson's

[Sly Saint]

Dementia poses a major health challenge with no safe, affordable treatments to slow its progression.

Researchers at Lawson Research Institute (Lawson), the research arm of St. Joseph's Health Care London, are investigating whether Ambroxol - a cough medicine used safely for decades in Europe - can slow dementia in people with Parkinson's disease.

Published today in the prestigious JAMA Neurology, this 12-month clinical trial involving 55 participants with Parkinson's disease dementia (PDD) monitored memory, psychiatric symptoms and GFAP, a blood marker linked to brain damage. Parkinson's disease dementia causes memory loss, confusion, hallucinations and mood changes. About half of those diagnosed with Parkinson's develop dementia within 10 years, profoundly affecting patients, families and the health care system.

Led by Cognitive Neurologist Dr. Stephen Pasternak, the study gave one group daily Ambroxol while the other group received a placebo. "Our goal was to change the course of Parkinson's dementia," says Pasternak. "This early trial offers hope and provides a strong foundation for larger studies."

Key findings from the clinical trial include:

• Ambroxol was safe, well-tolerated and reached therapeutic levels in the brain

• Psychiatric symptoms worsened in the placebo group but remained stable in those taking Ambroxol.

• Participants with high-risk GBA1 gene variants showed improved cognitive performance on Ambroxol

• A marker of brain cell damage (GFAP) increased in the placebo group but stayed stable with Ambroxol, suggesting potential brain protection.

Ambroxol supports a key enzyme called glucocerebrosidase (GCase), which is produced by the GBA1 gene. In people with Parkinson's disease, GCase levels are often low. When this enzyme doesn't work properly, waste builds up in brain cells, leading to damage. Pasternak learned about Ambroxol during a fellowship at The Hospital for Sick Children (SickKids) in Toronto, where it was identified as a treatment for Gaucher disease - a rare genetic disorder in children caused by a deficiency of GCase.

Clinical trial examines whether Ambroxol can slow dementia in people with Parkinson's

Dementia poses a major health challenge with no safe, affordable treatments to slow its progression.

www.news-medical.net

research paper:

https://jamanetwork.com/journals/jamaneurology/article-abstract/2835760

 

[Utsikt]

That’s a weird way of putting it. This is from the paper:

Statistical analyses compared ambroxol high dose vs placebo. There was no evidence to suggest differences between groups on primary or secondary outcomes.

Conclusions and Relevance Results of this randomized clinical trial reveal that ambroxol was safe, well-tolerated, and demonstrated target engagement. However, the effect of ambroxol on cognition was not confirmed.

https://jamanetwork.com/journals/jamaneurology/fullarticle/2835760

 

[hotblack]

But also from the same paragraph in the paper

Individuals receiving placebo showed a clinically meaningful worsening in neuropsychiatric symptoms, whereas those receiving ambroxol remained stable;

I see what you mean though @Utsikt the wording may be a bit weird and almost feels contradictory in places. They seem to have confirmed it’s safe and well tolerated, but be more vague on the second part of it is a disease modifying treatment.

Seems this idea has been around for a while in some form or another

Ambroxol improves lysosomal biochemistry in glucocerebrosidase mutation-linked Parkinson disease cells - PMC

Heterozygous GBA gene mutations are the most frequent Parkinson’s disease risk factor. Using Parkinson’s disease patient derived fibroblasts McNeill et al. show that heterozygous GBA mutations reduce glucosylceramidase activity, and are associated ...

pmc.ncbi.nlm.nih.gov

Edit: I suppose if the outcomes were ‘is it safe tolerated and does it end up at levels we expect’ then the wording makes sense? My brain is too fuzzy to make sense I think

 

[Utsikt]

But also from the same paragraph in the paper
I see what you mean though @Utsikt the wording may be a bit weird and almost feels contradictory in places. They seem to have confirmed it’s safe and well tolerated, but be more vague on the second part of it is a disease modifying treatment.

I don’t understand how both of those paragraphs can be true simultaneously.

According to the clinicaltrials.gov registration, the primary and secondary outcomes were measurements of various aspects of the disease. If there were no differences between the groups for the primary and secondary outcomes, one can’t have fared better than the other.

There is no mention of safety or tolerability as primary or secondary outcomes as far as I can see.

This is confusing to me.

 

[Kitty]

As an aside, I found it to be a good painkiller when I didn't have access to prescription meds. I was told about it by someone who used it to help with FMS pain.

I don't think it was available over the counter in the UK, but it was in Germany. I buy my injectable B12 from a German pharmacy, so I got it from them. It was very cheap—the sort of amount you'd pay for supermarket own-brand hay fever meds—and I got no side effects from the standard dose given on the pack. I found it much more effective on muscle pain than paracetamol, though not as effective as tramadol or DHC.

Haven't taken it for donkey's years, as I did finally get help with my nighttime pain issues.