Dichloroacetic Acid

Summary

Dichloroacetic Acid (CHEMBL13960) is a phase-3 clinical-stage small-molecule astringent; indicated across 6 conditions including melas syndrome and glioblastoma.

At a glance

• Status: Max clinical phase 3 (not approved)
• Modality: Small molecule
• Indications: 6 conditions
• Clinical trials: 23
• Chemistry: 128.94 Da · C2H2Cl2O2

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL13960
Name	Dichloroacetic Acid
Type	Small molecule
Max phase	3
FDA approved	no
PubChem CID	6597
ChEBI	CHEBI:36386
Molecular formula	C2H2Cl2O2
Molecular weight	128.94
InChIKey	JXTHNDFMNIQAHM-UHFFFAOYSA-N

SMILES: C(C(=O)O)(Cl)Cl

IUPAC name: 2,2-dichloroacetic acid

ChEBI definition: An organochlorine compound comprising acetic acid carrying two chloro substituents at the 2-position. It occurs in nature in seaweed, Asparagopsis taxiformis.

Pharmacological roles (ChEBI): astringent.

Other ChEBI roles (chemical / environmental): marine metabolite.

Also known as: Bichloracetic acid, Bichloroacetic acid, Dichloracetic acid, Dichlorethanoic acid, Dichloroacetate, Dichloroacetic acid, Dichloroethanoic acid, NSC-2654, Dichloro-acetic acid, Dichloro-acetic acid anion, SID17389935, SID26753010

Patent coverage: 17,090 distinct patent families (55,469 SureChEMBL compound mentions), from 1 matched compound structure(s). Mentions count patents naming the compound (not distinct inventions), so promiscuous / reference molecules inflate the mention figure — families are the dedup metric.

Targets

Targets

Broader ChEMBL bioactivity targets: 2 (assay-derived). Sample: Prelamin-A/C, Lethal factor.

Bioactivity

ChEMBL activities: 2 potent at pChembl ≥ 5 of 2 total. Top 100 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
LMNA	7.15	Potency	70.8	nM	CHEMBL_ACT_3650791
P15917	5.3	Potency	5012	nM	CHEMBL_ACT_4651773

Target pathways

No target-pathway data for this drug (no mapped target genes).

Indications & clinical

Indications

5 diseases in clinical trials (phase 1–3, investigational — not approved indications). Highest ChEMBL trial phase per disease; a non-cancer approved use is occasionally logged at phase 3 here.

Disease (in trials)	Phase	MONDO	EFO
MELAS syndrome	2	MONDO:0010789	Orphanet:550
glioblastoma	1	MONDO:0018177	EFO:0000519
neoplasm	1	MONDO:0005070	EFO:0000616
head and neck cancer	1	MONDO:0005627	EFO:0006859
brain neoplasm	1	MONDO:0021211	EFO:0003833

Clinical trials

Total trials: 23.

Phase distribution

Phase	Trials
PHASE1	8
Not specified	7
PHASE2	4
EARLY_PHASE1	2
PHASE4	1
PHASE3	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT02469337	PHASE4	UNKNOWN	Role of Preoperative Carbohydrates Drinks, Dichloroacetate and Exercise on Postoperative Muscle Insulin Resistance
NCT02616484	PHASE3	ACTIVE_NOT_RECRUITING	Trial of Dichloroacetate in Pyruvate Dehydrogenase Complex Deficiency:
NCT05120284	PHASE2	ACTIVE_NOT_RECRUITING	Trial of Dichloroacetate (DCA) in Glioblastoma Multiforme (GBM)
NCT00068913	PHASE2	UNKNOWN	Evaluating the Effectiveness of a Dichloroacetate in MELAS Syndrome
NCT00540176	PHASE2	COMPLETED	The Safety and Efficacy of DCA for the Treatment of Brain Cancer
NCT01029925	PHASE2	TERMINATED	Dichloroacetate (DCA) in Patients With Previously Treated Metastatic Breast or Non-Small Cell Lung Cancer (NSCL)
NCT00566410	PHASE1	COMPLETED	A Phase I, Open-Labeled, Single-Arm, Dose Escalation, Clinical and Pharmacology Study of Dichloroacetate (DCA) in Patients With Recurrent and/or Metastatic Solid Tumours
NCT00703859	PHASE1	WITHDRAWN	Combining Radiotherapy and Temozolomide With Dichloroacetate in Patients With Newly Diagnosed Glioblastoma
NCT01083524	PHASE1	COMPLETED	Dichloroacetate (DCA) for the Treatment of Pulmonary Arterial Hypertension
NCT01111097	PHASE1	COMPLETED	Study of the Safety and Efficacy of Dichloroacetate (DCA) in Glioblastoma and Other Recurrent Brain Tumors
NCT01123005	PHASE1	TERMINATED	Imaging and Biomarkers of Hypoxia in Solid Tumors
NCT01163487	PHASE1	COMPLETED	Metabolic Reprogramming Therapy for Treatment of Recurrent Head and Neck Cancers
NCT01807702	PHASE1	COMPLETED	13C-Pyruvate Breath Test
NCT02690285	PHASE1	COMPLETED	A Pilot Study of Rapid Haplotyping Procedure for Personalized Dosing of Dichloroacetate (DCA) in Healthy Volunteers
NCT05317455	EARLY_PHASE1	RECRUITING	Regulation of Brain Glucose Metabolism in Type 1 Diabetes
NCT03356457	EARLY_PHASE1	COMPLETED	Development of Agents to Diminish the Risk of Hypoglycemia-induced Brain Injury in Type 1 Diabetes
NCT06931262	Not specified	AVAILABLE	Expanded Access Treatment Protocol With DCA for Patients With PDCD
NCT00015015	Not specified	COMPLETED	Dichloroacetate Kinetics, Metabolism and Toxicology
NCT00031161	Not specified	COMPLETED	Prevention of Dichloroacetate Toxicity
NCT00865514	Not specified	TERMINATED	Pharmacotoxicology of Trichloroethylene Metabolites
NCT00874276	Not specified	COMPLETED	Pharmacotoxicology of Trichloroethylene Metabolites
NCT01797276	Not specified	NO_LONGER_AVAILABLE	Dichloroacetate Treatment of Congenital Lactic Acidosis Phase B: Expanded Access
NCT04046081	Not specified	COMPLETED	A Clinical Trial to Evaluate Dichloroacetate (DCA) as a Treatment for Endometriosis-associated Pain

Clinical evidence (CIViC)

No CIViC predictive evidence (expected for non-precision-medicine drugs).

Pharmacology

Pharmacogenomics

No PharmGKB pharmacogenomic data curated for this drug.

Related molecules

Related molecules

No competitor molecules sharing a primary target (ChEMBL phase ≥2 or PubChem drug-class).