Phase 2 Trial Shows Nilotinib Potential to Modulate Dopamine in Parkinson’s

Phase 2 Trial Shows Nilotinib Potential to Modulate Dopamine in Parkinson’s

I can only find the Abstract describing this research:
https://www.iaprd-world-congress.com..._Abstracts.pdf

The conclusions:

"a single time oral administration of Nilotinib may increase brain dopamine levels and metabolism. These results suggest Nilotinib, in a dose dependent manner, may have a symptomatic effect through modulation of brain dopamine levels. Additionally, the significant reduction of oligomeric alpha-synuclein, which is expected to increase in the CSF of PD patients as the disease progresses, suggests that Nilotinib may reduce misfolded alpha-synuclein accumulation and have a longterm disease modifying effect. Importantly, the dose response of oligomeric alpha-synuclein and HVA changes to nilotinib suggests that the dose administered may depend on the stage of disease to potentially halt PD progression."

John

Great Find!

I think an "abstract" is all we are likely to get for quite a while yet. As it says in the PNT article, the Georgetown Phase 2 trial is still ongoing.

If the present trials show continued safety and success will this drug get fast track status as a breakthrough therapy? Indications so far sure suggest that it qualifies!

https://www.fda.gov/forpatients/appr...st/default.htm

From the conclusion:
"Importantly, the dose response of oligomeric alpha-synuclein and HVA changes to nilotinib suggests that the dose administered may depend on the stage of disease to potentially halt PD progression."

I had a lot of trouble understanding this sentence, and I found the PNT article to be of no help in that regard.

Assuming I've understood it correctly, here is an alternative wording that might (or might not!) be clearer:
"The dose-dependent decrease in oligomeric alpha-synuclein levels (and increase in HVA/DOPAC levels) suggests that (to potentially halt PD progression) the stage-of-disease may dictate the appropriate dose of nilotinib to administer."

Anyway, it seems to me that there is still a lot of research to be done to establish the correct dose for each stage of the disease. This might well affect the prospects for a fast-track approval.

Hopefully, another write-up will appear (from AlzForum, SoPD blog, ...) and shed some more light.

Although maybe it’s technically possible it would be highly unlikely for this to happen. Fast track is used for new molecules that are ground breaking and greatly needed for a particular disease. There is no need for that in this case as this is already an approved drug. It’s available for any doctor to legally prescribe, if they believe it would be an effective treatment. If data comes out to compel the fda, which hasn’t happened yet, it would just make it easier for doctors to prescribe. So, I don’t think the fda would feel compelling pressure to fast track.

Yes, as Jeff replied, this was only presented as an Abstract at the Oral Poster Presentation of the 2018 iaprd. This is a common occurrence to present abstracts and posters of ongoing clinical resesrch to provide updates as data is analysed.