Biomarker & Intervention Discovery

ME/CFS (Myalgic Encephalomyelitis / Chronic Fatigue Syndrome)

This page lists 6 druggable gene targets, 38 approved or trial-tested drug candidates and 10 recent clinical trials for ME/CFS (Myalgic Encephalomyelitis / Chronic Fatigue Syndrome), with each agent ranked by the strength of its evidence (clinical, mechanistic or correlative). Candidates include existing drugs that could be repurposed. Sources are linked so you can check every claim.

Spontaneous remissionFull recovery without intervention: <5% of adult ME/CFS patients; partial improvement in ~40% over 5 years; post-COVID…
Best-intervention remissionNo disease-modifying treatment established; GET (graded exercise therapy) — former standard — shown harmful (PACE…
Gap sizeVery large — no effective treatment; historic misattribution to psychological causes delayed research by decades
Primary barrierbiological (no treatment) + framing (historic)
Full remission profile →

Remission & chronicity

Spontaneous remission
Full recovery without intervention: <5% of adult ME/CFS patients; partial improvement in ~40% over 5 years; post-COVID ME/CFS appears to have similarly low spontaneous recovery rate
Best-intervention remission
No disease-modifying treatment established; GET (graded exercise therapy) — former standard — shown harmful (PACE controversy, withdrawn from UK NICE guidance 2021); pacing (heart rate-based) prevents post-exertional malaise but not curative; low-dose naltrexone — ~30% improvement in small RCTs; rintatolimod (Ampligen) — Phase 3 failed FDA approval
Gap size
Very large — no effective treatment; historic misattribution to psychological causes delayed research by decades
Primary barrier
biological (no treatment) + framing (historic)
ME/CFS research has been systematically underfunded relative to disease burden (NIH funding $15M/year vs MS at $100M despite similar prevalence); PACE trial's GET/CBT approach adopted globally for years despite being harmful for many patients; NIH RECOVER initiative now represents largest-ever investment

Biomarker Targets & Therapeutic Agents

Gene targets queried against DGIdb, Open Targets, ChEMBL, PubMed, and Europe PMC. Agents ranked: Clinical > Mechanistic > Correlative.

ADRB2 368ATP5F1A 2CRH 1IL6 181TNF 287TRPV1 187
Therapeutic Agent Effect Direction Evidence Tier Potency Source
METAPROTERENOL ↑ activates Mechanistic
PROPRANOLOL HYDROCHLORIDE ↓ inhibits Mechanistic
OLODATEROL ↑ activates Mechanistic
TIMOLOL ↓ inhibits Mechanistic
CHEMBL:CHEMBL1465049 ? unclear Mechanistic
NOREPINEPHRINE ↑ activates Mechanistic
EPHEDRINE SULFATE ↑ activates Mechanistic
LEVALBUTEROL ? unclear Mechanistic
3-O-METHYLQUERCETIN ? unclear Mechanistic
RISPERIDONE ? unclear Mechanistic
CLENBUTEROL ↑ activates Mechanistic
ARFORMOTEROL ? unclear Mechanistic
CELIPROLOL ↑ activates Mechanistic
ISOPROTERENOL ↑ activates Mechanistic 0.1585 nM (EC50)
MEPHENTERMINE SULFATE ↑ activates Mechanistic
CARVEDILOL ↓ inhibits Mechanistic
PROCATEROL ↑ activates Mechanistic
CHEMBL:CHEMBL590927 ? unclear Mechanistic
TERTATOLOL ? unclear Mechanistic
FENOTEROL ↑ activates Mechanistic
MEPINDOLOL ? unclear Mechanistic
PROPAFENONE ↓ inhibits Mechanistic
CHEMBL:CHEMBL531322 ? unclear Mechanistic
CARMOTEROL ↑ activates Mechanistic
PINDOLOL ↓ inhibits Mechanistic
SALMETEROL ↑ activates Mechanistic
CGP12177 ↓ inhibits Mechanistic
CLENBUTEROL HYDROCHLORIDE ? unclear Mechanistic
NADOLOL ↓ inhibits Mechanistic
DESIPRAMINE ? unclear Mechanistic
CIMATEROL ↑ activates Mechanistic
FENOTEROL HBR ? unclear Mechanistic
RITODRINE ? unclear Mechanistic
PF-00610355 ? unclear Mechanistic
SOTALOL ↓ inhibits Mechanistic
TERBUTALINE ↑ activates Mechanistic
LAS190792 ↑ activates Mechanistic
NORADRENALINE ↑ activates Mechanistic
LEVOBUNOLOL HYDROCHLORIDE ↓ inhibits Mechanistic
PENBUTOLOL SULFATE ↓ inhibits Mechanistic

Showing top 40 of 368 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
CHEMBL5653589 ↓ inhibits Mechanistic 1741.11 nM (Kd)
CHEMBL3752910 ↓ inhibits Mechanistic 1478.97 nM (Kd)
Therapeutic Agent Effect Direction Evidence Tier Potency Source
CHEMBL5286255 ↓ inhibits Mechanistic 0.7 nM (Ki)
Therapeutic Agent Effect Direction Evidence Tier Potency Source
CLAZAKIZUMAB ↓ inhibits Mechanistic
MIDOSTAURIN ? unclear Mechanistic
ECHINACEA EXTRACT ? unclear Mechanistic
NELFINAVIR ? unclear Mechanistic
SELECTIVE ESTROGEN RECEPTOR MODULATOR ? unclear Mechanistic
SIGNAL TRANSDUCTION INHIBITOR ? unclear Mechanistic
OLOKIZUMAB ? unclear Mechanistic
IBUDILAST ? unclear Mechanistic
VITAMIN A PALMITATE ? unclear Mechanistic
IFOSFAMIDE ? unclear Mechanistic
SITAGLIPTIN ? unclear Mechanistic
METRONIDAZOLE ? unclear Mechanistic
PEGINTERFERON ALFA-2A ? unclear Mechanistic
PACIBEKITUG ↓ inhibits Mechanistic
PEGINTERFERON ALFA-2B ? unclear Mechanistic
SIRUKUMAB ↓ inhibits Mechanistic
HMG-COA REDUCTASE INHIBITOR ? unclear Mechanistic
SAFINGOL ? unclear Mechanistic
ENZYME INHIBITOR ? unclear Mechanistic
CISPLATIN ? unclear Mechanistic
GALLIUM NITRATE ? unclear Mechanistic
RITUXIMAB ? unclear Mechanistic
INFLIXIMAB ? unclear Mechanistic
C326 ? unclear Mechanistic
GEMFIBROZIL ? unclear Mechanistic
ZILTIVEKIMAB ? unclear Mechanistic
INSULIN, REGULAR, HUMAN ? unclear Mechanistic
OSIMERTINIB ? unclear Mechanistic
BINETRAKIN ? unclear Mechanistic
ALOGLIPTIN ? unclear Mechanistic
TENELIGLIPTIN ? unclear Mechanistic
CHINESE HERBS ? unclear Mechanistic
TOCILIZUMAB ? unclear Mechanistic
LINAGLIPTIN ? unclear Mechanistic
DOCETAXEL ANHYDROUS ? unclear Mechanistic
VILDAGLIPTIN ? unclear Mechanistic
LINEZOLID ? unclear Mechanistic
SELENIUM ? unclear Mechanistic
SAQUINAVIR ? unclear Mechanistic
PAREGORIC ? unclear Mechanistic

Showing top 40 of 181 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
DICLOFENAC SODIUM ? unclear Mechanistic
RDP58 ? unclear Mechanistic
METHYLENE BLUE ? unclear Mechanistic
ORTATAXEL ? unclear Mechanistic
CERTOLIZUMAB PEGOL ↓ inhibits Mechanistic
OZORALIZUMAB ↓ inhibits Mechanistic
THALIDOMIDE ? unclear Mechanistic
HUANG LIAN ? unclear Mechanistic
GOLIMUMAB ↓ inhibits Mechanistic
CYCLOSPORINE ? unclear Mechanistic
DOCETAXEL ANHYDROUS ? unclear Mechanistic
HALOFUGINONE ? unclear Mechanistic
ETANERCEPT ↓ inhibits Mechanistic
ISIS 104838 ? unclear Mechanistic
GEMCITABINE ? unclear Mechanistic
INDOMETHACIN ? unclear Mechanistic
INSULIN, REGULAR, HUMAN ? unclear Mechanistic
DIDANOSINE ? unclear Mechanistic
5,7-DIHYDROXY-4-METHYLCOUMARIN ? unclear Mechanistic
SOBLIDOTIN ? unclear Mechanistic
STAVUDINE ? unclear Mechanistic
CISPLATIN ? unclear Mechanistic
MEROPENEM ANHYDROUS ? unclear Mechanistic
AMPHOTERICIN B LIPOSOMAL ? unclear Mechanistic
MIDAZOLAM HYDROCHLORIDE ? unclear Mechanistic
RIFAMPIN ? unclear Mechanistic
AVX-470 ? unclear Mechanistic
KETOROLAC TROMETHAMINE ? unclear Mechanistic
PRAZOSIN HYDROCHLORIDE ? unclear Mechanistic
RISPERIDONE ? unclear Mechanistic
BENZO[E]PYRENE ? unclear Mechanistic
ETHAMBUTOL HYDROCHLORIDE ? unclear Mechanistic
REMTOLUMAB ↓ inhibits Mechanistic
SPIRONOLACTONE ? unclear Mechanistic
OMEPRAZOLE ? unclear Mechanistic
CARBOPLATIN ? unclear Mechanistic
MITOMYCIN C ANALOG KW-2149 ? unclear Mechanistic
NAFAMOSTAT ? unclear Mechanistic
ABBV-3373 ~ modulates Mechanistic
BACILLUS CALMETTE-GUERIN ANTIGEN ? unclear Mechanistic

Showing top 40 of 287 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
RHTX ↑ activates Mechanistic
ABT-102 ↓ inhibits Mechanistic
ASIVATREP ↓ inhibits Mechanistic
BCTC ↓ inhibits Mechanistic
MAVATREP ↓ inhibits Mechanistic
POLYGODIAL ? unclear Mechanistic
ALLICIN ↓ inhibits Mechanistic
JTS-653 ↓ inhibits Mechanistic
SB452533 ↓ inhibits Mechanistic
MR-1817 ↓ inhibits Mechanistic
GRC-6211 ↓ inhibits Mechanistic
ACETAMINOPHEN ↑ activates Mechanistic
DWP05195 ↓ inhibits Mechanistic
STREPTOZOCIN ? unclear Mechanistic
CAPSAICIN ↑ activates Mechanistic 19.95 nM (EC50)
AMG517 ↓ inhibits Mechanistic
5S-HETE ↑ activates Mechanistic
BENZYL ISOTHIOCYANATE ? unclear Mechanistic
MASOPROCOL ? unclear Mechanistic
[3H]RESINIFERATOXIN ↑ activates Mechanistic
[3H]A778317 ↓ inhibits Mechanistic
CAPSAZEPINE ↓ inhibits Mechanistic 120.0 nM (Ki)
NABIXIMOLS ? unclear Mechanistic
H+ ↑ activates Mechanistic
RECOMBINANT TUMOR NECROSIS FACTOR FAMILY PROTEIN ? unclear Mechanistic
NADA ↓ inhibits Mechanistic
ZUCAPSAICIN ↑ activates Mechanistic
SB366791 ↓ inhibits Mechanistic
GRC-15300 ? unclear Mechanistic
MRS-1477 ~ modulates Mechanistic
ANANDAMIDE ↑ activates Mechanistic
JNJ17203212 ↓ inhibits Mechanistic
LEVODOPA ? unclear Mechanistic
INDOMETHACIN ? unclear Mechanistic
AZD1386 ↓ inhibits Mechanistic
CAMPHOR OIL ↑ activates Mechanistic
ISOPROPYL ISOTHIOCYANATE ? unclear Mechanistic
VOACANGINE ? unclear Mechanistic
FERRUGINENE B ? unclear Mechanistic
PHENYLACETYLRINVANIL ↑ activates Mechanistic

Showing top 40 of 187 agents ranked by evidence tier.


Clinical Trials

Drug, biologic and supplement trials (treatment or prevention) from ClinicalTrials.gov whose main condition is ME/CFS (Myalgic Encephalomyelitis / Chronic Fatigue Syndrome): recruiting, active or completed, most recently updated first. Data retrieved 2026-10-28.

NCT06739720 Phase: 2/3 Started: 2025-01-02

The goal of this clinical trial is to test if a Lingzhi supplement (CP003) helps people with chronic fatigue or post-COVID fatigue feel better. It will also check if the supplement is safe to use. The main questions it aims to answer are: Does the Lingzhi supplement reduce fatigue symptoms? Is it sa...

NCT07753122 Phase: N/A Started: 2026-08-15

This is a 16-week controlled treatment trial of an over-the -counter supplement, hydrogen water, for individuals with ME/CFS patients. An active treatment condition will be compared to a placebo-control condition. The study will also seek confirmation of heart rate variability (HRV) as a biomarker, ...

NCT05697640 Phase: 2 Started: 2023-06-22

The goal of this clinical trial is to evaluate the therapeutic value of an approved drug (Vericiguat) in patients with post-COVID-19 syndrome, who suffer from profound tiredness or fatigue, regardless of bed rest.The main questions it aims to answer are: • Does Vericiguat relieve fatigue and/or othe...

NCT07009691 Phase: N/A Started: 2025-06-09

The goal of this clinical trial is to learn if the OTC supplement, hydrogen water, works to treat the fatigue-related symptoms and functional limitations in adults with myalgic encephalomyelitis/chronic fatigue syndrome (ME/CFS). It will also examine if heart rate variability (HRV) can be used to pr...

NCT06952413 Phase: 2 Started: 2025-04-09

The efficacy and safety of rituximab on ME/CFS symptoms after administration to patients with myalgic encephalomyelitis/chronic fatigue syndrome will be compared in an exploratory, placebo-controlled, double-blind fashion. In the subsequent secondary evaluation period, subjects who received rituxima...

NCT06211062 Phase: 2 Started: 2022-12-20

This clinical study aims to evaluate the use of i3.1 probiotic in participants who meet the Institute of Medicine (Canadian Consensus Criteria) case definition for ME/CFS and who may or may not be diagnosed with irritable bowel syndrome (IBS). The main questions it aims to answer are: * how effectiv...

NCT03186027 Phase: N/A Started: 2017-06-28

The main aim of the study is to examine the effect of oral CoQ10 plus NADH (Reconnect®) supplementation twice daily for 8-weeks on the changes in fatigue perception, sleep disturbances, autonomic dysfunction and HRQoL assessed by patient-reported outcome measures in CFS/ME.

NCT05128292 Phase: N/A Started: 2018-01-01

In recent years, it has been suggested that nutritional deficiencies may be of causal relevance in individuals with ME/CFS. These include deficiencies of vitamins and trace elements. It is likely that the observed nutritional deficiencies contribute to the core symptoms of the disease. Coenzyme Q10 ...

NCT04741841 Phase: N/A Started: 2020-03-30

Myalgic Encephalomyelitis/Chronic Fatigue Syndrome (ME/CFS) is a neurological disease. Currently there is no effective treatment for ME/CFS due to unclear etiology of the disease. The aim of this randomized double-blind placebo-control clinical trial is to study the efficacy of the probiotic food su...

NCT06366724 Phase: 2 Started: 2024-09-10

The LIFT will be conducted at Brigham and Women's Hospital (BWH) of Harvard Medical School, focusing on the effect of Pyridostigmine (Mestinon) and Low-Dose Naltrexone (LDN) in subjects aged 18-70 meeting the Canadian consensus criteria (CCC) for Myalgic Encephalomyelitis/Chronic Fatigue Syndrome (M...


Agents Found By Disease-Level Search

Searched directly from “ME/CFS (Myalgic Encephalomyelitis / Chronic Fatigue Syndrome)” via ClinicalTrials.gov interventions and Open Targets' disease→drug data — independent of the 6-gene target panel above. This surfaces agents whose mechanism doesn't route through a curated gene (combination therapies, standard-of-care drugs, targets outside the panel).

38 agents shown (approved or with trial evidence) — 37 not found by the gene-target search above.

CBTNOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT00920777 NCT00920777

NAC 900mg/dayNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04542161

NAC 3600mg/dayNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04542161

Hyperbaric oxygen therapyNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07621068

LingZhi capsuleNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06739720

CoQ10 plus SeleniumNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05128292

IA with TheraSorb ® column (Miltenyi)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07529197

Floradapt Intensive GINOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06211062

ImmunoadsorptionNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05710770

Rituximab

· 1 trial · source: ClinicalTrials.gov
NCT02229942

CyclophosphamideNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02444091

Hydrogen water which is prepared from an OTC supplement.NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07009691

AmpligenNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00215800

LumbrokinaseNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06511050

Vericiguat Oral TabletNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05697640

ASARMNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02082730

Molecular hydrogen produced by a metallic magnesium tablet, consumed 3x a day dissolved in water. Treatment duration is 16 weeks.NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07753122

Transfer PackageNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07523113

Follow Up Phone CallsNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07523113

Rituximab(Genetical Recombination)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06952413

Prednisolone 20 mg/ 5 mgNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05638633

Vitamin B compound (100mg B1, 50 mg B6, 500 µg B12)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05638633

melatonin plus zincNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03000777

Coenzyme Q10 plus NADHNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03186027

phosphoserine plus vitamin CNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03186027

Acceptance and Commitment Therapy (ACT)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03562325

Control groupNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00920777

Bupivacaine InjectionNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05664711

Hyperbaric oxygen therapy (HBOT)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06118138

A 3-day courseNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05236465

Waiting listNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05236465

Intervention GroupNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07363655

IA with TheraSorb ® columnNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05629988

GutMagnific™ H.NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04741841

GutMagnific™ L.NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04741841

PyridostigmineNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06366724

Low-Dose NaltrexoneNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06366724

ProbioticsNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03773003

Generated by the Open Source Medicine Foundation Biomarker Pipeline — 2026-10-08. Data sources: DGIdb, Open Targets, ChEMBL, PubMed, ClinicalTrials.gov. Not medical advice.