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.

Our recent study about a hormone called FGF-21 in the blood can help explain why people with ME/CFS, FM, or both experience different symptoms and levels of brain fog. Instead of focusing only on diagnosis, we grouped patients by low, normal, or high FGF-21 levels and discovered clear patterns—like worse fatigue and thinking problems in those with low levels. This matters because it shows that FGF-21 could be used to personalize care, helping doctors better understand each patient’s condition and choose treatments that match their unique biology.

Multiple manuscripts on this work are in preparation. Two have been published:

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..