UNMC researchers reach milestone in effort to slow or halt progression of Parkinson's

UNMC researchers reach milestone in effort to slow or halt progression of Parkinson's | Health | omaha.com

I've been following this study for a few years and was wondering when they would finally release some results. This is great news! It's a shame that it will take the team over a year to begin the larger study.

The article is based on a paper written by Gendelman et al.. They write [1]:

"A potential therapeutic role for immune transformation in Parkinson’s disease evolves from more than a decade of animal investigations demonstrating regulatory T cell (Treg) nigrostriatal neuroprotection. To bridge these results to human disease, we conducted a randomized, placebo-controlled double-blind phase 1 trial with a well-studied immune modulator, sargramostim (granulocyte-macrophage colony-stimulating factor)."

The sample size is small: 10 active and 10 placebo. In Figure 4 there are two graphs, one showing the UPDRS III scores of the placebo group and the other the active group. To my eyes even before the treatment is started these groups have different properties.

Sargramostim has been used for, inter alia., fungal infections. I wonder if this is a confounding property.

Reference:

[1] "Evaluation of the safety and immunomodulatory effects of sargramostim in a randomized, double-blind phase 1 clinical Parkinson’s disease trial"
Howard E. Gendelman et al.
npj Parkinson's Disease 3, Article number: 10 (2017)
Evaluation of the safety and immunomodulatory effects of sargramostim in a randomized, double-blind phase 1 clinical Parkinson’s disease trial | npj Parkinson'''s Disease

John

Yes, the graphs certainly had some odd differences, and the sample size was small. Nevertheless, for a phase 1 safety and proof of concept study, this trial really was well designed and the results certainly have me interested in seeing a larger phase 2.