The Open Medicine Foundation’s Collaborative Center at Montreal, directed by Dr. Alain Moreau, recently launched a new study called Myalgic Encephalomyelitis Adaptive Therapeutic Solution Platform (MEDUSA). The overarching goal of MEDUSA is to explore therapeutic targets and promising treatments for ME/CFS using a precision medicine approach. 

MEDUSA consists of a couple of phases. The first phase will involve deep phenotyping of people with ME/CFS to identify therapeutic targets. In the context of MEDUSA, deep phenotyping includes health questionnaires, testing on blood and urine samples, microRNA profiling, and cognitive testing. Then, the study team will conduct pilot trials of FDA-approved drugs that can be repurposed as interventions for those therapeutic targets.

In initial work on MEDUSA phase 1, started as part of the center’s REMEDIAL study, the team found that elevated sphingomyelin phosphodiesterase acid like 3B (SMPDL3B) was correlated with symptom severity and is associated with immune dysregulation. They are exploring therapeutic approaches for a pilot trial based on these preliminary findings.

The results of phase 1 ultimately contribute to the study design, the first stage of the research process, of MEDUSA pilot trials.