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出典(authority):フリー百科事典『ウィキペディア（Wikipedia）』「2012/10/14 11:30:49」(JST)

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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.

Phenylbutyrate has been used to treat urea cycle disorders.^[1]

Metabolism

Phenylbutyrate is a prodrug. In the human body it is metabolized by beta-oxidation to phenylacetate.

Phenylacetate conjugates with glutamine to phenylacetylglutamine, that is eliminated with the urine.

Nitrogen elimination

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 glioma and acute myeloid leukaemia.^{[citation needed]}

Phenylbutyrate has been associated with longer lifespans in Drosophila.^[2]

University of Colorado researchers Dr. Curt Freed and Wenbo Zhou demonstrated that phenylbutyrate stops the progression of Parkinson's disease in mice by turning on a gene called DJ-1 that can protect dopaminergic neurons in the midbrain from dying. As of July 2011^[update] they plan on testing phenylbutyrate for the treatment of Parkinson's disease in humans.^[3]

References

^ Batshaw, M. L.; MacArthur, R. B.; Tuchman, M. (2001). "Alternative pathway therapy for urea cycle disorders: twenty years later". J. Pediatr. 138 (1 Suppl): S46–S54; discussion S54–S55. doi:10.1067/mpd.2001.111836. PMID 11148549.
^ Kang, H. L.; Benzer, S.; Min, K. T. (2002). "Life extension in Drosophila by feeding a drug". Proc. Natl. Acad. Sci. U.S.A. 99 (2): 838–843. doi:10.1073/pnas.022631999. PMC 117392. PMID 11792861. //www.ncbi.nlm.nih.gov/pmc/articles/PMC117392/.
^ News Article

UpToDate Contents

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1. 尿素サイクル障害：管理 urea cycle disorders management
2. βサラセミアの治療 treatment of beta thalassemia
3. 高グレードの神経膠腫に対する実験的治療アプローチ experimental treatment approaches for high grade gliomas
4. バーター症候群およびギテルマン症候群 bartter and gitelman syndromes
5. ペルオキシソーム病 peroxisomal disorders

English Journal

Role of Epidermal Growth Factor Receptor and Endoplasmic Reticulum Stress in Vascular Remodeling Induced by Angiotensin II.

Takayanagi T1, Kawai T1, Forrester SJ1, Obama T1, Tsuji T1, Fukuda Y1, Elliott KJ1, Tilley DG1, Davisson RL1, Park JY1, Eguchi S2.
Hypertension.Hypertension.2015 Apr 27. pii: HYPERTENSIONAHA.115.05344. [Epub ahead of print]
The mechanisms by which angiotensin II (AngII) elevates blood pressure and enhances end-organ damage seem to be distinct. However, the signal transduction cascade by which AngII specifically mediates vascular remodeling such as medial hypertrophy and perivascular fibrosis remains incomplete. We have
PMID 25916723

Brain Endoplasmic Reticulum Stress Mechanistically Distinguishes the Saline-Intake and Hypertensive Response to Deoxycorticosterone Acetate-Salt.

Jo F1, Jo H1, Hilzendeger AM1, Thompson AP1, Cassell MD1, Rutkowski DT1, Davisson RL1, Grobe JL1, Sigmund CD2.
Hypertension.Hypertension.2015 Apr 20. pii: HYPERTENSIONAHA.115.05377. [Epub ahead of print]
Endoplasmic reticulum stress has become an important mechanism in hypertension. We examined the role of endoplasmic reticulum stress in mediating the increased saline-intake and hypertensive effects in response to deoxycorticosterone acetate (DOCA)-salt. Intracerebroventricular delivery of the endop
PMID 25895586

Recent advances in the treatment of hyperammonemia.

Matoori S1, Leroux JC2.
Advanced drug delivery reviews.Adv Drug Deliv Rev.2015 Apr 17. pii: S0169-409X(15)00065-4. doi: 10.1016/j.addr.2015.04.009. [Epub ahead of print]
Ammonia is a neurotoxic agent that is primarily generated in the intestine and detoxified in the liver. Toxic increases in systemic ammonia levels predominantly result from an inherited or acquired impairment in hepatic detoxification and lead to potentially life-threatening neuropsychiatric symptom
PMID 25895618

Japanese Journal

トランスポーター関連疾患に対する新規治療戦略の開発を指向した創薬研究

林久允
薬学雑誌 134(10), 1007-1011, 2014
NAID 40020237541

トランスポーター関連疾患に対する新規治療戦略の開発を指向した創薬研究

林久允
薬学雑誌. 乙号 134(10), 1007-1011, 2014
… We have previously shown that in many cases of PFIC2 patients, the dysfunction of BSEP is attributable to decreased BSEP expression on the hepatocanalicular membrane and that 4-phenylbutyrate (4PB), an approved drug for urea cycle disorder, may be a compound with potential to restore BSEP expression. …
NAID 130004693883

神経変性疾患における小胞体折りたたみ不全タンパク質処理応答に関する薬理学的研究

野村靖幸
薬学雑誌. 乙号 134(4), 537-543, 2014
The endoplasmic reticulum (ER) has physiological roles in the quality control of proteins. Various stresses (e.g., oxidation, aging) to the ER cause accumulation of unfolded/misfolded proteins in th …
NAID 130003391203

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リンク元	「フェニル酪酸」「フェニルブチレート」
拡張検索	「sodium phenylbutyrate」

「フェニル酪酸」

Sodium phenylbutyrate
Systematic (IUPAC) name
	Sodium 4-phenylbutanoate
Clinical data
AHFS/Drugs.com	Consumer Drug Information
Licence data	EMA:Link, US FDA:link
Pregnancy cat.	not to be used
Legal status	?
Pharmacokinetic data
Metabolism	to phenylacetic acid
Half-life	0.8 hours
Excretion	80% as phenylacetylglutamine
Identifiers
CAS number	1716-12-7
ATC code	A16AX03
PubChem	CID 9815454
ChemSpider	5068
ChEMBL	CHEMBL1746
Chemical data
Formula	C10H11NaO2
Mol. mass	186.2 g/mol
	SMILES [Na+].[O-]C(=O)C(c1ccccc1)CC;
	InChI InChI=1S/C10H12O2.Na/c1-2-9(10(11)12)8-6-4-3-5-7-8;/h3-7,9H,2H2,1H3,(H,11,12);/q;+1/p-1 ; Key:RHLFTMGPBSLHRS-UHFFFAOYSA-M ;
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