Simmaron recently published findings from Phase II of its rapamycin treatment trial. Those findings describe 3 pathways that are disrupted in ME patients: mTOR activation increasing cellular stress, mitochondrial dysfunction leading to oxidative stress, and purine dysregulation signaling inflammation.

In the next 3 emails, we are going to take a closer look at these broken pathways and how rapamycin might improve them in the subset that responded. We’ll start with mitochondrial dysfunction.

Impaired mitochondria are well known in ME. What’s new are findings suggesting that rapamycin might improve their stress response and reserve capacity. 

Our study found that people with ME/CFS showed impaired mitochondrial function, particularly a reduced ability to increase energy production during periods of cellular stress. 

After 90 days of low-dose rapamycin, mitochondrial energy production and reserve capacity - the ability to meet increased energy demands - significantly improved. Greater reserve capacity was also associated with improvements in fatigue, post-exertional malaise (PEM), sleep problems, and orthostatic intolerance.

The researchers also found that rapamycin reduced mitochondrial oxidative stress and altered purine metabolism, which may help explain how treatment improves mitochondrial function and reduces inflammation. We’ll dig deeper into purine’s role in the next email.

Overall, the findings suggest that rapamycin may help restore the cells’ ability to produce energy when under stress. 

We know how important it is to test these findings in a placebo-controlled trial, and we designed a randomized trial to do just that in collaboration with Medical College of Wisconsin.  As we start the randomized control trial, we continue to raise funds so we can enroll statistically significant numbers.

Our small non-profit is delivering big findings, thanks to your support.

Watch our webinar describing these findings here.

Journal of Translational Medicine publication.