Thread by @ggottschalkPhD

We @RedefiningMECFS and @AvikRoy74969264 are excited to share some early results of our low dose rapamycin project in its pre-print version here. Thanks to all the patients for participating!

researchsquare.com/article/rs-659…

We found that a significant proportion of the patients who finished the three month trial period of rapamycin (an mTOR inhibitor) showed improvements in both clinical features of the illness (PEM) and saw improvement in blood based markers of autophagy function

Autophagy can be inhibited when mTOR activity is high. Rapamycin helps induce autophagy function. This is critical for the removal of metabolically inactive proteins and recycling mitochondria.

Our goal with this observational trial was to see if low dose rapamycin could change blood markers of autophagy function, specifically pATG13 based on our previous publication which found it to be elevated in patients with ME/CFS pubmed.ncbi.nlm.nih.gov/35487443/

We got NIH funding to further explore the mechanism of mTOR mediated autophagy disruption as it relates to key ME/CFS symptoms like PEM and published this work

pubmed.ncbi.nlm.nih.gov/39777574/

We were thankful to run into David Kaufman, MD who has been using rapamycin in his patients and together with this new hypothesis we initiated this trial back in Nov. 2023. Our clinical trial team has grown to include 8 clinical sites including @GrachStephanie and @BatemanHorne

This trial is not placebo controlled and we understand the limitations; however, our goal was to try and develop a predictive diagnostic to help objectively identify potential responders to therapy. This would be helpful in clinical practice and future double blinded placebo controlled trials.

In Feb 2025 we expanded our original trial and are happy to report that enrollment is soaring, thanks in large part to a partnership with @agelessrx_ who is providing clinical services, centralizing the drug supply and other critical elements.

Expect more updates soon! Our partnership with @agelessrx_ will help us lead to new therapies (and combinations) using FDA-approved agents in placebo controlled trials.