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Precision Medicine
for Myalgic Encephalomyelitis
Drug Discovery and ClinicaI Trials (REMEDIAL)

We conducted a comprehensive analysis and found 27 circulating microRNAs that were significantly different in people with ME/CFS and POTS (postural orthostatic tachycardia syndrome) compared to those with ME/CFS alone or POTS alone.

  • We identified two microRNAs that were only elevated in patients with both ME and POTS, and two others that were elevated only in patients with POTS but not ME.
  • By examining the targets of these microRNAs, we discovered new genes and proteins that could lead to new treatment options.
  • We also found a potential shared mechanism between people with both ME and POTS and those with POTS alone, which involves dopamine receptors.


Cognitive assessments using BrainCheck enabled the stratification of ME/CFS patients into three distinct clusters based on cognitive response.

  • These clusters led us to discover specific microRNAs associated with particular cognitive domains, which may provide insight into mechanisms underlying cognitive impairment in ME/CFS.
  • Our studies have identified three key microRNAs—miR-29a-3p, miR-150-5p, and the miR-181 family—that show promising connections to cognitive function and decline in the context of ME/CFS.


Multiple manuscripts on this work are in preparation..

STUDY HYPOTHESIS AND DESCRIPTION

Our study will couple a deep phenotyping
characterization of ME/CFS patients to the
development of cellular and animal models
for repurposing current drugs to accelerate
the proof-of-concept of different therapeutic
interventions for ME/CFS. Indeed, precision
medicine is essential to address the clinical
complexity of ME/CFS and to identify the
best therapeutic options to cure ME/CFS.

Three researchers, two men and a woman in a lab. The man in the foreground is recording findings.

OBJECTIVES

  • Deep phenotyping of ME patients through precision medicine.
  • Development of cellular and mouse models for the validation of targets.
  • Preparation and launch of pilot phase 2b clinical trials..