Investigational Drug Details
Drug ID: | D032 |
Drug Name: | Betaine |
Synonyms: | 2-(Trimethylammonio)Acetate; 2-N,N,N-trimethylammonio acetate; 2-trimethylammonioacetate; Abromine; Acidol; Betaine; Betaine, anhydrous; Glycine betaine; Glycinebetaine; N,N,N-trimethylammonioacetate; N,N,N-Trimethylglycine; TMG; Trimethylaminoacetate; Trimethylammonioacetate; Trimethylglycine; Trimethylglycocoll |
Type: | Chemical drug |
DrugBank ID: | DB06756 |
DrugBank Description: | Betaine is a methyl group donor that functions in the normal metabolic cycle of methionine and reduces homocystinuria in patients with inborn errors of methionine metabolism. In the United States, betaine is distributed under the brand name Cystadane® by Rare Disease Therapeutics. Many reports have shown that betaine's therapeutic effectiveness is limited, and does not lower tHcy levels or prevent clinical symptoms . |
PubChem ID: | 247 |
CasNo: | 107-43-7 |
Repositioning for NAFLD: | Yes |
SMILES: | [O-]C(=O)C[N+](C)(C)C |
Structure: |
|
InChiKey: | KWIUHFFTVRNATP-UHFFFAOYSA-N |
Molecular Weight: | 117.148 |
DrugBank Targets: | Apoptosis regulator Bcl-2 |
DrugBank MoA: | Betaine acts as a methyl group donor in the remethylation of homocysteine to methionine in patients with homocystinuria. |
DrugBank Pharmacology: | -- |
DrugBank Indication: | Betaine is indicated for the treatment of homocystinuria to decrease elevated homocysteine blood levels. Included within the category of homocystinuria are deficiencies or defects in: 1. cystathionine beta-synthase (CBS), 2. 5,10-methylenetetrahydrofolate reductase (MTHFR), 3. cobalamin cofactor metabolism (cbl). |
Targets: | DNMTs stimulant |
Therapeutic Category: | Improve insulin resistance |
Clinical Trial Progress: | Phase 2 completed (NCT00586911) |
Latest Progress: | Under clinical trials |

Trial ID | Source ID | Phases | Status | Study Results | Start Date | Last Update Posted | |
---|---|---|---|---|---|---|---|
L0215 | NCT00586911 | Phase 2 | Completed | No Results Available | January 2003 | April 8, 2015 | Details |
L0829 | NCT03073343 | Not applicable | Recruiting | No Results Available | 20/02/2017 | 12 December 2020 | Details |
Target ID | Target Name | GENE | Action | Class | UniProtKB ID | Entry Name |
---|
Strategy ID | Strategy | Synonyms | Related Targets | Related Drugs | |
---|---|---|---|---|---|
S01 | Improve insulin resistance | insulin sensitizer; insulin resistance; glucose tolerance | Biguanide: increases 5-AMP activated protein kinase signaling; SGLT-2 inhibitor; Thiazalidinedione: selective PPAR-γ agonists; GLP-1 agonist | Metformin; Empagliflozin; Canagliflozin; Rosiglitazone; Pioglitazone; Liraglutide | Details |
S13 | Anti-apoptosis | hepatocyte apoptosis; hepatic autophagy; apoptosis | Pan-caspase inhibitor | Emricasan | Details |
Article ID | PMID | Source | Title | |
---|---|---|---|---|
A00271 | 35159219 | Cells | S-Adenosylmethionine: From the Discovery of Its Inhibition of Tumorigenesis to Its Use as a Therapeutic Agent. | Details |
A01227 | 34822395 | Metabolites | Aqueous Metabolite Trends for the Progression of Nonalcoholic Fatty Liver Disease in Female Bariatric Surgery Patients by Targeted 1H-NMR Metabolomics. | Details |
A01443 | 34743980 | Eur J Pharmacol | Preventive and therapeutic role of betaine in liver disease: A review on molecular mechanisms. | Details |
A01525 | 34708066 | Front Nutr | Integrated Transcriptomic and Translatomic Inquiry of the Role of Betaine on Lipid Metabolic Dysregulation Induced by a High-Fat Diet. | Details |
A01587 | 34684533 | Nutrients | Supplementation with a Specific Combination of Metabolic Cofactors Ameliorates Non-Alcoholic Fatty Liver Disease, Hepatic Fibrosis, and Insulin Resistance in Mice. | Details |
A01608 | 34680557 | Biomedicines | Combined Metabolic Activators Decrease Liver Steatosis by Activating Mitochondrial Metabolism in Hamsters Fed with a High-Fat Diet. | Details |
A02223 | 34454176 | Biochem Biophys Res Commun | Translatome analysis reveals the regulatory role of betaine in high fat diet (HFD)-induced hepatic steatosis. | Details |
A03391 | 34019353 | Vopr Pitan | [Specialized products for dietary correction of the diet of patients with non-alcoholic steatohepatitis]. | Details |
A03918 | 33818349 | Nutr Res Rev | Effects of betaine on non-alcoholic liver disease. | Details |
A05753 | 33132631 | World J Gastroenterol | Role of betaine in liver disease-worth revisiting or has the die been cast? | Details |
A06375 | 32908189 | Sci Rep | 2,3,7,8-Tetrachlorodibenzo-p-dioxin (TCDD) dysregulates hepatic one carbon metabolism during the progression of steatosis to steatohepatitis with fibrosis in mice. | Details |
A06614 | 32808060 | Eur J Nutr | Betaine prevented high-fat diet-induced NAFLD by regulating the FGF10/AMPK signaling pathway in ApoE-/- mice. | Details |
A06660 | 32791310 | Diabetes Metab | Trimethylamine N-oxide levels are associated with NASH in obese subjects with type 2 diabetes. | Details |
A06681 | 32782033 | Microsc Microanal | Effect of Betaine Supplementation on Liver Tissue and Ultrastructural Changes in Methionine-Choline-Deficient Diet-Induced NAFLD. | Details |
A06792 | 32744096 | Am J Physiol Endocrinol Metab | Neurodegeneration in juvenile Iberian pigs with diet-induced nonalcoholic fatty liver disease. | Details |
A08075 | 32257815 | Mol Genet Metab Rep | Mtrr hypomorphic mutation alters liver morphology, metabolism and fuel storage in mice. | Details |
A08417 | 32119738 | J Nutr | One-Carbon Metabolism in Fatty Liver Disease and Fibrosis: One-Carbon to Rule Them All. | Details |
A08451 | 32105727 | Gastroenterology | TMAVA, a Metabolite of Intestinal Microbes, Is Increased in Plasma From Patients With Liver Steatosis, Inhibits γ-Butyrobetaine Hydroxylase, and Exacerbates Fatty Liver in Mice. | Details |
A09179 | 31846961 | Lifestyle Genom | One-Carbon Metabolism and Nonalcoholic Fatty Liver Disease: The Crosstalk between Nutrients, Microbiota, and Genetics. | Details |
A09448 | 31744629 | Environ Int | Perfluoroalkyl substances and severity of nonalcoholic fatty liver in Children: An untargeted metabolomics approach. | Details |