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sodium phenylacetate
- Development stage
- Phase 2
- Lead developer
- Valeant Pharmaceuticals
- Modality
- Small Molecules
- Administration
- Intravenous
01
Overview
Sodium phenylacetate is a small molecule salt of phenylacetic acid that serves as a nitrogen-scavenging agent. It is primarily used to treat hyperammonemia in patients with urea cycle disorders, typically in combination with sodium benzoate (marketed as Ammonul). Its mechanism involves conjugating with glutamine to form phenylacetylglutamine, which is then excreted by the kidneys, providing an alternative pathway for nitrogen removal when the urea cycle is deficient. Beyond its metabolic applications, sodium phenylacetate has been investigated as a differentiation-inducing antineoplastic agent. In oncology, it has been shown to suppress tumor growth and promote differentiation in experimental models of glioblastoma, prostate cancer, and melanoma, potentially by modulating the expression of growth factors like transforming growth factor-alpha (TGF-alpha) and inducing cytostasis.
- Brand names
- Ammonul
- Other names
- sodium benzeneacetatephenylacetic acid sodium saltNaPA
02
Targets
PRKCA (Protein kinase C alpha)HDAC1 (Histone Deacetylase 1)DAO (D-amino-acid oxidase)
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