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ME/CFS Adaptive Therapeutic Solution Platform (MEDUSA) – Phase 1

Study Aim

The purpose of phase 1 of this study is to identify promising treatments for specific subgroups of people with ME/CFS, which will be evaluated in later phases.

Investigators

  • Alain Moreau, PhD

Updates and Potential

  • Elevated SMPDL3B was found to be correlated with symptom severity and is associated with immune dysregulation.
  • The team is exploring novel therapeutic approaches based on these preliminary findings.
  • A manuscript is in preparation.
STUDY HYPOTHESIS AND DESCRIPTION

Building on our previous studies, MEDUSA will allow us to explore therapeutic targets and promising treatments for ME/CFS, thereby offering hope for patients who have long been without meaningful options.

This first phase represents foundational investigations focusing on systematically investigating promising therapeutic targets and assessing FDA-approved drugs for repurposing as targeted interventions. This phase employs state-of-the-art molecular profiling, including transcriptomics, proteomics, and functional assays in model cell lines and patient-derived cells, to identify key pathways and molecules implicated in disease mechanisms. These findings will form the critical foundation for subsequent phases.

OBJECTIVES

a scientist is testing various pills in order to improve their quality an effectiveness in the laboratory, a chemist is testing the chemistry of various pill capsules in a medicine laboratory

  • Deep phenotyping of ME/CFS patients through precision medicine. This step is essential (a) to predict ME/CFS patients who could benefit from a specific treatment, and (b) to finalize the validation of new monitoring clinical and molecular tools necessary to evaluate the therapeutic efficacy of proposed treatments.
  • Exploration of past clinical trials. Exploring biological samples from previous clinical trials presents a unique opportunity to extract additional value from these original clinical studies and to deepen our understanding of the underlying mechanisms of disease or treatment response. By reanalyzing these samples with advanced methodologies or newly developed biomarkers, we can potentially uncover novel insights that were not part of the initial analysis.
  • In vitro validation of FDA-approved drugs for repurposing as targeted therapeutics in ME/CFS. The repurposing of FDA-approved drugs offers a promising strategy to accelerate the identification of effective therapeutic interventions for ME/CFS. This approach aligns with the principles of precision medicine, enabling us to address the clinical heterogeneity of ME/CFS and tailor interventions to specific patient subgroups.

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Myalgic Encephalomyelitis / Chronic Fatigue Syndrome (ME / CFS) Post Treatment Lyme Disease Syndrome (PTLDS), Fibromyalgia Leading Research. Delivering Hope.Open Medicine Foundation®

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