Sodium phenylbutyrate is a reliable treatment for urea cycle disorder and has emerged as a possible anti-cancer therapy option.
Sodium Phenylbutyrate Uses and Background
Sodium phenylbutyrate was originally developed for Johns Hopkins in the mid-80s as a treatment for inborn errors of urea synthesis metabolic disorders that result in severe mental and psychomotor retardation. In 1996, sodium phenylbutyrate became a viable treatment for urea cycle disorders and is available commercially worldwide for that indication.
Ongoing cancer research has revealed that sodium phenylbutyrate also targets the underlying molecular defects that cause cancer and switches on tumor suppressor genes. It has become a novel anti-cancer therapy that offers lower toxicity than traditional chemotherapy.
Today research continues into new and groundbreaking sodium phenylbutyrate uses including cancer, cystic fibrosis, sickle cell anemia, ALS, Parkinson’s disease, glaucoma, spinal muscular atrophy, tuberculosis, and more.
Chemical Structure and Metabolic Action
In terms of its chemical structure, sodium phenylbutyrate is a sodium salt of an aromatic fatty acid composed of an aromatic ring and butyric acid. The chemical name for sodium phenylbutyrate is 4-phenylbutyric acid sodium salt.
Sodium phenylbutyrate may be taken orally or intravenously. It has a naturally salty and slightly bitter taste which may or may not be apparent in its final tablet or powder form.
Although sodium phenylbutyrate is synthetically manufactured, once in the body it is quickly metabolized into a naturally occurring metabolite of phenylalanine. Because it is easily converted to a natural body substance, it has very low toxicity.