Trial of Deferiprone in Parkinson’s Disease (Iron Chelation) Complete: Looks like it made PD worse...

[Bolt_Upright]

So they completed the Deferiprone trial. I had not heard of the trial, but I had thought iron chelation could be a good avenue to pursue. Maybe iron chelation is still a good avenue, but it looks like this Deferiprone trial was worse than not effective. Sad to say but it looks like the placebo group lucked out this time.

Trial of Deferiprone in Parkinson’s Disease nejm.org/doi/full/10.1056/N...

"Results: A total of 372 participants were enrolled; 186 were assigned to receive deferiprone and 186 to receive placebo. Progression of symptoms led to the initiation of dopaminergic therapy in 22.0% of the participants in the deferiprone group and 2.7% of those in the placebo group. The mean MDS-UPDRS total score at baseline was 34.3 in the deferiprone group and 33.2 in the placebo group and increased (worsened) by 15.6 points and 6.3 points, respectively (difference, 9.3 points; 95% confidence interval, 6.3 to 12.2; P<0.001). Nigrostriatal iron content decreased more in the deferiprone group than in the placebo group. The main serious adverse events with deferiprone were agranulocytosis in 2 participants and neutropenia in 3 participants.

Conclusions: In participants with early Parkinson’s disease who had never received levodopa and in whom treatment with dopaminergic medications was not planned, deferiprone was associated with worse scores in measures of parkinsonism than those with placebo over a period of 36 weeks. (Funded by the European Union Horizon 2020 program; FAIRPARK-II ClinicalTrials.gov number, NCT02655315. opens in new tab.)"

This trial only included participants with newly diagnosed Parkinson’s disease who had never received levodopa. Maybe the results would be different for people already on levadopa?

For background, here is a video from the doctor when he was starting the trial:

youtu.be/DCRWKrNmPfw(Not working as expected?)

[Astra7]

Having had both a blood transfusion (giving birth) and 2 iron transfusions I have often wondered if this caused my PD.

Interesting that this trial was done, although a shame the results were not helpful.

[WinnieThePoo]

Just one in a long line of "alternative" possibilities - particularly of re-purposed drugs, which looked very promising at lab / in-vitro / rat / monkey / phase 1 stage , and then after a lengthy phase 3 bombed, including Isradipine and Simvastatin.

Exanatide (and other glp-1 receptor agonists) are the big hopes here, but also Ambroxyl. Folk read the early promise, try to get it off-label, and a few years down the line we find out it doesn't work. (I speak as one who changed his blood pressure meds to Isradipine, and whose father changed his statins to Simvastatin)

[Bolt_Upright in reply to WinnieThePoo]

I bought a bunch of Ambroxyl ($75 worth). I am set if I get a cough.

[WinnieThePoo in reply to Bolt_Upright]

Just to wipe the smile off my face, I have an annoying cough and head cold at the moment and wish I had some Ambroxyl!

[Solvang53]

I abstain from any big pharma drugs especially vaccines...