Drug halts the progression of Lewy body dementia (in mice)

[Xenos]

medicalnewstoday.com/articl...

[Hidden]

It’s a start! Better in mice than in nothing and no one likes it’s been until now. My chronically negative self is feeling glimmers of substantiated optimism about our future.

[Xenos in reply to Hidden]

I've a good feeling about this one.

Too bad to have to wait a year.

[parkylot]

Rather than wait. I’ll ask for one of their brilliant mice and he’ll do the thinking for me. 🐭

[park_bear]

This is an excellent study because they used a reasonable facsimile of Parkinson's disease instead of the sloppy MPTP or rotenone models that merely mimic the symptoms of Parkinson's disease. This treatment has a real possibiilty to become a game changer. Of course these things take years. Actual study here:

mdpi.com/1422-0067/22/12/61...

[Bolt_Upright]

Great news. And the links to hypothyroidism keep coming

Acute Treatment with T-Type Calcium Channel Enhancer SAK3 Reduces Cognitive Impairments Caused by Methimazole-Induced Hypothyroidism Via Activation of Cholinergic Signaling

pubmed.ncbi.nlm.nih.gov/295...

[Despe]

"Prof. Dexter cautioned that there remains much more work to do."

From the trials to the market, 10-15 years??

[gl2238]

If they spent as much time and money as they did on Covid, seems help would be just around the corner, but no such luck.

[Hidden]

“We discovered the novel, disease-modifying therapeutic candidate SAK3, which, in our studies, rescued neurons in most protein-misfolding, neurodegenerative diseases,” says senior author Kohji Fukunaga, Professor Emeritus.

Powerful summary on SAK3, how about this one on a similar "misfolded protein" acting med:

"This proposal outlines a completely novel pharmacological target for PDD, namely the enzyme GCase. It also proposes a completely novel therapy using the drug Ambroxol, an agent considered safe enough to give to pregnant women, which has improved GCase function in pilot studies in humans. This strategy could stop or reverse the underlying pathology of PD; it might allow patients to get better." centerwatch.com/clinical-tr.... Also pretty powerful, particularly since Ambroxol's available now, and not perhaps in 5-10 years, giving us a chance to act now on something that can potentially help PwPD (whether or not SAK3 is ultimately found to clean up better or worse than ambroxol).

So how does Ambroxol do it? Like SAK3 which has been found to clean-up "clumps of a misfolded protein called alpha-synuclein", so does Ambroxol, which "is a GCase small molecule (protein) chaperone...reported to increase GBA protein and activity in dopaminergic neurons...and its activity enhances the clearance of monomericα-synuclein in human dopaminergic neuronal cells..."reader.elsevier.com/reader/...

Since it is thought that the aggregation of α-synuclein (clumping) in the brain is linked to PD, a reduction in clumping should slow the progression of PD (perhaps even at some point "reverse the underlying pathology of PD"). Interestingly, I just discovered a new research article published on 'Enhancing the Activity of Glucocerebrosidase as a Treatment for Parkinson Disease' in July 2020 (which I rented) with this Ambroxol comment in it: "Among the class of small chaperones, ambroxol is one of the most promising candidates as a disease-modifying treatment in PD." It also references the Ambroxol for PD trial completed last year: "The study confirmed that ambroxol is able to penetrate CSF and is well tolerated in patients with PD (even at 10 times higher dose and longer duration of administration than specified in its license)... with a significant improvement in motor symptoms severity". link.springer.com/article/1... As I have been on Ambroxol for going on 17 months now, I continue to experience firsthand its improvements, and have no intention of stopping now and potentially passing the point of no return "beyond which the damage to the neural systems affected by synucleinopathy can no longer be protected or revived, even with the most effective therapies". (movementdisorders.onlinelib... Looking forward to publication of the 2nd trial findings to be published next year on "Ambroxol as a novel disease-modifying treatment for Parkinson’s disease dementia", and getting a clearer picture of its potential improvements and dosage required to achieve them. One way of the other, we will know soon enough in Ambroxol's case.

[TiredNRetired in reply to Hidden]

A lot to be encouraged by. Do you have a neuro prescribing the Ambroxol or are you going it alone? I procured some but have yet to try it.

[Hidden in reply to TiredNRetired]

My neuro is aware and is fine with me taking it in addition to my current PD meds. No prescription is possible for Ambroxol, you have to procure it yourself just as you have.

[TiredNRetired in reply to Hidden]

Thanks!

[Farooqji]

I don't even read the mice and monkeys experiments for PD