Great new blog by Alan Hoffman

[rrunk7]

Alan is one of the 12 participants in the Nilotinib study at Georgetown. He is a great guy and has begun a really good blog with lots of info and commentary that anyone with PD will find very interesting.

[jeffreyn]

rrunk7,

I did a quick Google search but didn't find the website. Can you give us the link?

Thanks,

Jeff

[rrunk7 in reply to jeffreyn]

Sorry I forgot to include!

advenventurouslifewithparki...

[p-oui]

Thank you for posting this. This is the first clinical study to examine the effects of the cancer drug Nilotinib in the PD population. It was a small group, 11, and seemed to show some pretty amazing results to those tested and the study concluded may be safe and tolerated in subjects with PDD or DLB. All participants were recruited from the movement disorders clinic at MedStar Georgetown University Hospital.

PD: Parkinson’s disease; DLB: dementia with Lewy body; MCI: mild cognitive impairment; PDD: Parkinson’s disease with dementia; UPDRS: Unified Parkinson’s Disease Rating Score.

The primary objective was to determine the safety and tolerability of daily Nilotinib for 6 months at low oral daily doses of 150 mg or 300 mg Nilotinib (compared to 600–800 mg in cancer). The secondary objectives were to determine whether Nilotinib crosses the BBB and whether it affects phosphorylation of Abl in CSF samples. Motor and cognitive functions were also measured as exploratory clinical outcomes. Other exploratory outcomes are that Nilotinib may alter PD-related CSF biomarkers DJ-1 and α-synuclein. Nilotinib effects on progression of dementia were evaluated using the Mini Mental State Examination (MMSE) and Scales for Outcomes in Parkinson’s disease-Cognition (SCOPA-Cog). The Columbia Suicide Severity Rating Scale (CSSRS) was performed at every visit.

"In this study we show that Nilotinib may enter the CSF and that it may inhibit CNS Abl. These findings are consistent with our preclinical studies, which showed that Nilotinib enters the brain and inhibits Abl, leading to protection of DA neurons"

"Participants in the Nilotinib trial experienced incremental improvement in motor and non-motor UPDRS I-IV scores"

Read more at content.iospress.com/articl...

For those better at reading through these details I welcome your thoughts. Additional trials are being planned at Georgetown Clinic and also through MJFF to kick off asap. My guess is that while the initial study to determine safety was focused on advanced PD patients, subsequent studies will include those early in their progression. Stay tuned!

[p-oui]

Try also this for those who are not familiar with this drug's initial trial for PD:

gumc.georgetown.edu/news/Re...

Among the biomarker findings were that:

The level of the dopamine metabolite homovanillic acid — an indicator that dopamine is being produced — steadily doubled, even with the loss of most dopamine neurons. Most study participants were able to stop using, or reduce their use of, dopamine replacement therapies;

The level of the Parkinson's related oxidative stress marker DJ-1 — an indicator that dopamine-producing neurons are dying — was reduced more than 50 percent after niltonib treatment; and

The levels of cell death markers (NSE, S100B and tau) were significantly reduced in cerebrospinal fluid (CSF) suggesting reduced neuronal cell death.