U.S. Department of Health & Human Services Divider Arrow National Institutes of Health Divider Arrow NCATS

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Status:

Class (Stereo):
CHEMICAL (ACHIRAL)


Triheptanoin (also known as C7 oil) is an investigational medical food or supplement. Triheptanoin is thought to have an anaplerotic role, meaning that it can replenish substances involved in the tricarbolic acid cycle, a pathway used by cells to produce energy, providing an alternative source of energy to the brain. It supplies the body with heptanoate which can either be oxidized to propionyl-CoA directly or is metabolized by the liver to the“C5 ketones”, β-ketopentanoate and/or β-hydroxypentanoate, which are released into the blood. After one month of triheptanoin use, the level of energy production in the brain during visual stimulation had become normal in Huntington’s patients. Triheptanoin was anticonvulsant in two chronic mouse models and increased levels of anaplerotic precursor metabolites in epileptic mouse brains. Despite the unknown mechanism of triheptanoin’s anticonvulsant action, the fact that triheptanoin has been used safely in several animals and for various metabolic diseases in children and adults should expedite the ethical and regulatory approval processes for a clinical trial in medically refractory patients with epilepsy. Triheptanoin is phase II clinical trial for the treatment of glycogen storage disease type V, Huntington's disease, Rett syndrome and amyotrophic lateral sclerosis.
Sodium phenylbutyrate is a salt of an aromatic fatty acid. The compound is used to treat urea cycle disorders, because its metabolites offer an alternative pathway to the urea cycle to allow excretion of excess nitrogen. Sodium phenylbutyrate is also a histone deacetylase inhibitor and chemical chaperone, leading respectively to research into its use as an anti-cancer agent and in protein misfolding diseases such as cystic fibrosis. It is used as 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. Sodium phenylbutyrate is a pro-drug and is rapidly metabolized to phenylacetate. Phenylacetate is a metabolically active compound that conjugates with glutamine via acetylation to form phenylacetylglutamine. The kidneys then excrete Phenylacetylglutamine. PBA (phenylbutyric acid) is absorbed from the intestine and converted by way of β-oxidation to the active moiety, phenylacetic acid (PAA). PAA is conjugated with glutamine in the liver and kidney by way of N-acyl coenzyme A-l-glutamine N-acyltransferase to form phenylacetylglutamine (PAGN). Like urea, PAGN incorporates two waste nitrogens and is excreted in the urine. On a molar basis, it is comparable to urea (each containing two moles of nitrogen). Therefore, phenylacetylglutamine provides an alternate vehicle for waste nitrogen excretion.
Status:
First marketed in 1921
Source:
Sodium Glycerophosphate U.S.P.
Source URL:

Class (Stereo):
CHEMICAL (RACEMIC)



Synthetic glycerophosphates have been known for many years and have been prepared in several ways. The acid may exist in two isomeric forms, alpha and beta. The L-a-acid is the naturally occurring form; the b-acid, present in hydrolyzates of lecithins from natural sources, arises from migration of the phosphoryl group from the a-carbon atom. Dehydrogenation of L-glycerol 3-phosphate produces Dihydroxyacetone phosphate and is part of the entry of glycerol (sourced from triglycerides) into the glycolytic pathway.
Status:
US Approved OTC
Source:
21 CFR 349.12(d)(5) ophthalmic:demulcents propylene glycol
Source URL:
First approved in 1961
Source:
VOSOL PROPYLENE GLYCOL by WAMPOLE LABS
Source URL:

Class (Stereo):
CHEMICAL (RACEMIC)


PROPYLENE GLYCOL is a component of SYSTANE® Lubricant. It is used for the temporary relief of burning and irritation due to dryness of the eye.
Status:
US Approved OTC
Source:
21 CFR 347.10(h) skin protectant glycerin
Source URL:
First marketed in 1921
Source:
Glycerin U.S.P.
Source URL:

Class (Stereo):
CHEMICAL (ACHIRAL)


Glycerin (glycerol) is 3-carbon alcohol naturally occurring in the human body. It is the structural backbone triacylglycerol molecules, and can also be converted to a glycolytic substrate for subsequent metabolism. Glycerin is a colorless, odorless, viscous, sweet-tasting liquid. The FDA classifies glycerol as "generally recognized as safe". Glycerin is used in the pharmaceutical industry as a sweetener in syrups, lozenges, and as an excipient in eyewash solutions. As an individual prescription product, glycerin has uses as a hyperosmotic, osmotic diuretic, and ophthalmic agent. It may be used as an eye drop in the treatment of glaucoma to reduce intraocular pressure, as a solution or suppository for short-term treatment of constipation, to evacuate the bowel prior to a colonoscopy, and in some ocular surgeries. It may be given intravenously to reduce pressure inside the brain and used externally on the skin as a moisturizer. Glycerin has many other uses in the agricultural, food and pharmaceutical industry.
Status:
Investigational
Source:
Am J Dent. Feb 2002;15(1):8-10.: Phase 2 Human clinical trial Completed N/A
Source URL:

Class (Stereo):
CHEMICAL (MIXED)

Status:
Investigational
Source:
INN:mosedipimod [INN]
Source URL:

Class (Stereo):
CHEMICAL (RACEMIC)

EC-18 (now known as mosedipimod), a synthetic copy of 1-palmitoyl-2-linoleoyl-3-acetyl-rac-glycerol (PLAG) that was developed by Enzychem Lifescience for oral administration for the treatment of immune and inflammatory related diseases, including psoriasis, rheumatoid arthritis, asthma, atopic dermatitis, and sepsis. The US Food and Drug Administration (FDA) has granted Orphan Drug Designation to EC-18 for the treatment of Acute Radiation Syndrome (ARS) and for the for the neutropenia treatment. Besides, the EC-18 is participating in phase II clinical trials to investigate the efficacy and safety of the agent for chemoradiation-induced oral mucositis and chemotherapy-induced neutropenia. This drug has a multimodal mechanism of action. It stimulates calcium influx into T lymphocytes and increases the production of various cytokines. In addition, EC-18 enhances the cytolytic activity of natural killer (NK) cells and suppresses the expression of the transmembrane protein tumor cell toll-like receptor 4 (TLR-4) on cancer cells, thus suppresses tumor cell proliferation.
Status:
Investigational
Source:
INN:glucalox
Source URL:

Class (Stereo):
CHEMICAL (RACEMIC)

Glucalox an antacid that was used as a digestive agent. Information about the current use of this compound is not available.
Status:
Investigational
Source:
NCT00004450: Not Applicable Interventional Completed Adrenoleukodystrophy
(1998)
Source URL:

Class (Stereo):
CHEMICAL (ACHIRAL)

Glyceryl trierucate (trierucin) is a trierucic acid triglyceride that has been investigated for the treatment of adrenoleukodystrophy, a rare genetic disorder characterized by the breakdown or loss of myelin in the brain. To investigate treatment options for adrenoleukodystrophy, most studies have used “Lorenzo's oil" (LO), a 4:1 mixture of glyceryl trioleate and glyceryltrierucate. Lorenzo’s oil was found to reduce the levels of saturated very long chain fatty acids (ELOVL) in the plasma, adipose tissue and liver, but to a lesser extent in the brain. Inhibition of ELOVL 1 may be an underlying mechanism by which Lorenzo's oil exerts its action. Several clinical trials have been conducted to study the potential of glyceryl trierucate in treatment of adrenoleukodystrophy.
Status:
Investigational
Source:
INN:febuverine
Source URL:

Class (Stereo):
CHEMICAL (MIXED)

Febuverine is a spasmolytic and local anesthetic.