Sodium phenylbutyrate
Encyclopedia
Sodium phenylbutyrate is an orphan drug
, marketed by Ucyclyd Pharma (Hunt Valley, USA) under the trade name Buphenyl and by Swedish Orphan International (Sweden) as Ammonaps.
It has been used to treat urea cycle disorder
s.
On July 5, 2011, Colorado researchers, Dr. Curt Freed and Wenbo Zhou, have discovered a drug that stops the progression of Parkinson's disease in mice and is now being tested on humans. They have found that the drug phenylbutyrate turns on a gene, called DJ-1, that can protect dopamine neurons in Parkinson's disease. Parkinson's disease is caused by dying midbrain dopamine neurons.
. In the human body it is metabolized by beta-oxidation to phenylacetate.
Phenylacetate conjugates with glutamine to phenylacetylglutamine
, that is eliminated with the urine.
and acute myeloid leukaemia.
PBA has been associated with longer lifespans in Drosophila
.
Orphan drug
An orphan drug is a pharmaceutical agent that has been developed specifically to treat a rare medical condition, the condition itself being referred to as an orphan disease...
, marketed by Ucyclyd Pharma (Hunt Valley, USA) under the trade name Buphenyl and by Swedish Orphan International (Sweden) as Ammonaps.
It has been used to treat urea cycle disorder
Urea cycle disorder
An urea cycle disorder or urea cycle defect is a genetic disorder caused by a deficiency of one of the enzymes in the urea cycle which is responsible for removing ammonia from the blood stream. The urea cycle involves a series of biochemical steps in which nitrogen, a waste product of protein...
s.
On July 5, 2011, Colorado researchers, Dr. Curt Freed and Wenbo Zhou, have discovered a drug that stops the progression of Parkinson's disease in mice and is now being tested on humans. They have found that the drug phenylbutyrate turns on a gene, called DJ-1, that can protect dopamine neurons in Parkinson's disease. Parkinson's disease is caused by dying midbrain dopamine neurons.
Metabolism
Phenylbutyrate is a prodrugProdrug
A prodrug is a pharmacological substance administered in an inactive form. Once administered, the prodrug is metabolised in vivo into an active metabolite, a process termed bioactivation. The rationale behind the use of a prodrug is generally for absorption, distribution, metabolism, and...
. In the human body it is metabolized by beta-oxidation to phenylacetate.
Phenylacetate conjugates with glutamine to phenylacetylglutamine
Phenylacetylglutamine
Phenylacetylglutamine is a product formed by the conjugation of phenylacetate and glutamine. It is a common metabolite that can be found in human urine....
, that is eliminated with the urine.
Research
Sodium phenylbutyrate is also under investigation for the treatment of some sickle-cell disorders (Blood Products Plasma Expanders and Haemostatics) and for use as a potential differentiation-inducing agent in malignant gliomaGlioma
A glioma is a type of tumor that starts in the brain or spine. It is called a glioma because it arises from glial cells. The most common site of gliomas is the brain.-By type of cell:...
and acute myeloid leukaemia.
PBA has been associated with longer lifespans in Drosophila
Drosophila
Drosophila is a genus of small flies, belonging to the family Drosophilidae, whose members are often called "fruit flies" or more appropriately pomace flies, vinegar flies, or wine flies, a reference to the characteristic of many species to linger around overripe or rotting fruit...
.