Repositioning Candidate Details
| Candidate ID: | R0464 |
| Source ID: | DB06819 |
| Source Type: | approved; investigational |
| Compound Type: | small molecule |
| Compound Name: | Phenylbutyric acid |
| Synonyms: | |
| Molecular Formula: | C10H12O2 |
| SMILES: | OC(=O)CCCC1=CC=CC=C1 |
| Structure: |
|
| DrugBank Description: | A monocarboxylic acid the structure of which is that of butyric acid substituted with a phenyl group at C-4. It is a histone deacetylase inhibitor that displays anticancer activity. It inhibits cell proliferation, invasion and migration and induces apoptosis in glioma cells. It also inhibits protein isoprenylation, depletes plasma glutamine, increases production of foetal haemoglobin through transcriptional activation of the γ-globin gene and affects hPPARγ activation. |
| CAS Number: | 1821-12-1 |
| Molecular Weight: | 164.2011 |
| DrugBank Indication: | Adjunctive therapy for the management of chronic urea cycle disorders due to deficiencies in carbamylphosphate (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase. it is indicated in all neonatal- onset efficiency presenting within the first 28 days of life. Also indicated in patients with late-onset, presenting after the first month of life with a history of hyperammonemic encephalopathy. |
| DrugBank Pharmacology: | Decreases elevated plasma ammonia glutamine levels |
| DrugBank MoA: | Sodium phenylbutyrate is a pro-drug that is metabolized to the active compound phenylacetate. Phenylacetate conjuages with glutamine via acetylation reaction to form the product phenylacetylglutamine, which is excreted by the kidneys. This provides an alternative mechanism for waste nitrogen excretion. |
| Targets: | Aromatic-amino-acid aminotransferase; Histone deacetylase |
| Inclusion Criteria: |
| Strategy ID | Strategy | Synonyms | Related Targets | Related Drugs |
|---|
| Target ID | Target Name | GENE | Action | Class | UniProtKB ID | Entry Name |
|---|
| Diseases ID | DO ID | Disease Name | Definition | Class |
|---|