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

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Showing 1 - 10 of 12 results

Omaveloxolone (RTA-408) is a synthetic triterpenoid exerting antioxidant inflammation modulator properties. It activates the transcription factor Nrf2 and inhibits NF-κB signaling. Omaveloxolone demonstrated antioxidant, anti-inflammatory, and anticancer activities. Reata Pharmaceuticals is developing omaveloxolone for the treatment of cancers, Friedreich's ataxia and mitochondrial disorders.
Pioglitazone (brand name Actos) is a prescription drug of the thiazolidinedione class with hypoglycemic action used in the treatment of type 2 diabetes. Pioglitazone selectively stimulates the nuclear receptor peroxisome proliferator-activated receptor gamma (PPAR-γ) and to a lesser extent PPAR-α. It modulates the transcription of the genes involved in the control of glucose and lipid metabolism in the muscle, adipose tissue, and the liver. As a result, pioglitazone reduces insulin resistance in the liver and peripheral tissues, decreases gluconeogenesis in the liver, and reduces the quantity of glucose and glycated hemoglobin in the bloodstream. Pioglitazone is used to lower blood glucose levels in the treatment of diabetes mellitus type 2 (T2DM) either alone or in combination with a sulfonylurea, metformin, or insulin. Pioglitazone cannot be used in patients with a known hypersensitivity to pioglitazone, other thiazolidinediones or any of components of its pharmaceutical forms. It is ineffective and possibly harmful to diabetes mellitus type 1 and diabetic ketoacidosis. Pioglitazone can cause fluid retention and peripheral edema. As a result, it may precipitate congestive heart failure (which worsens with fluid overload in those at risk). It may cause anemia. Mild weight gain is common due to increase in subcutaneous adipose tissue. In studies, patients on pioglitazone had an increased proportion of upper respiratory tract infection, sinusitis, headache, myalgia and tooth problems.
Dyclonine is an local anesthetic used to provide topical anesthesia to mucous membranes through sodium channel inhibition. It is the active ingredient in Sucrets, an over-the-counter throat lozenge. It has been used as a local anesthetic agent prior to laryngoscopy, bronchoscopy, esophagoscopy, or endotracheal intubation. However, oral solutions no longer are commercially available in the US. Recently, additional activities of dyclonine have been discovered. Dyclonine represents a novel therapeutic strategy that can potentially be repurposed for the treatment of Friedreich's ataxia. Dyclonine enhances the cytotoxic effect of proteasome inhibitors on cancer and multiple myeloma cells.
Status:
Investigational
Source:
NCT01898884: Phase 1 Interventional Completed Friedreich's Ataxia
(2013)
Source URL:

Class (Stereo):
CHEMICAL (ACHIRAL)



3-Indolepropionic acid (IN-OX1; Indole-3-propionic acid; OX-1; Oxigon; SHP 22; SHP-622; VP-20629), an endogenous substance produced by bacteria in the intestine, is a deamination product of Tryptophan (T947200) that protects the hippocampus (studied in gerbils) from ischemic damage and oxidative stress. It’s ability to protect the neurons in this way is attributed to its potent antioxidative effects. 3-Indolepropionic acid is also hypothesized to have protective effects on the thyroid gland. 3-Indolepropionic acid is being studied for therapeutic use in Alzheimer's disease. 3-Indolepropionic acid (IPA) completely protected primary neurons and neuroblastoma cells against oxidative damage and death caused by exposure to Abeta, by inhibition of superoxide dismutase, or by treatment with hydrogen peroxide. In kinetic competition experiments using free radical-trapping agents, the capacity of IPA to scavenge hydroxyl radicals exceeded that of melatonin, an indoleamine considered to be the most potent naturally occurring scavenger of free radicals. In contrast with other antioxidants, IPA was not converted to reactive intermediates with pro-oxidant activity. In 2011, Intellect redirected the focus of the OX1 program from Alzheimer's disease to FA (Friedreich's Ataxia). Research suggests that the symptoms associated with FA are the result of oxidative stress caused by the abnormal accumulation of iron. OX1's ability to neutralize ROS could be an effective agent to reduce oxidative stress in FA, thereby eliminating the symptoms of FA and increasing both quality of life and longevity in affected individuals.
Status:
Designated
Source:
EU-Orphan Drug:EU/3/10/793
Source URL:

Class (Stereo):
CHEMICAL (ACHIRAL)



RG2833 is a compound originally developed by the Scripps Insitute for the treatment of Friedrick's Ataxia. RG2833 is a potent and selective inhibitor of neuronal histone deacetylase. RG2833 has been granted orphan drug status has been investigated in phase one clinical trials. There has also been proof of concept studies conducted for the treatment of Parkinson's disease.
Cianidanol is an antioxidant flavonoid, occurring especially in woody plants as both (+)-catechin and (-)-epicatechin (cis) forms. One of the polyphenols present in green tea, (+)-catechin (Cianidanol), has been studied for its effects on animal models of hepatitis, as well as in human clinical studies. Pure (+)-catechin (also known as (+)- cyanidanol-3 – trade name Catergen) has been used to treat hepatitis since 1976. This compound has been shown to be an efficient immune stimulator, promoting activation of macrophages, cytotoxic-T-lymphocytes, and natural killer cells in mice. Several clinical studies demonstrate the effectiveness of (+)-catechin in the treatment of viral hepatitis. Pure (+)-catechin has been found to cause hemolysis in some patients, possibly by the promotion of antibody formation against (+)-catechin, which might cross-react with red blood cells. However, there are no reports in the literature of green tea, green tea extracts, or green tea polyphenols causing this side-effect.
Idebenone is a synthetic short-chain benzoquinone and a substrate for the enzyme NAD(P)H:quinone oxidoreductase (NQO1) capable of stimulating mitochondrial electron transport and supplementing cellular energy levels. Idebenone was initially developed by Takeda Pharmaceutical Company for the treatment of Alzheimer’s disease and other cognitive defects. This has been met with limited success. The Swiss company Santhera Pharmaceuticals has started to investigate it for the treatment of neuromuscular diseases. In 2010, early clinical trials for the treatment of Friedreich’s ataxia and Duchenne muscular dystrophy have been completed. In clinical trials, Idebenone (Raxone/Catena) had a positive impact on a measurement of respiratory function (Peak Expiratory Flow, or PEF) in non-ambulatory Duchenne muscular dystrophy patients who were not taking steroids. As of December 2013 the drug is not approved for these indications in North America or Europe. It is approved for the treatment of Leber's hereditary optic neuropathy (LHON) in Europe. Idebenone (Raxone) is indicated for the treatment of visual impairment in adolescent and adult patients with Leber’s Hereditary Optic Neuropathy (LHON). Because the number of patients with Leber's hereditary optic neuropathy is low, the disease is considered ‘rare’, and Raxone was designated an ‘orphan medicine’ on 15 February 2007. Idebenone is thought to help improve production of energy by restoring mitochondrial function, thereby preventing the cellular damage and the loss of sight seen in LHON. Idebenone is a rapidly absorbed, safe and well-tolerated drug and is currently the only clinically proven treatment option for Leber's hereditary optic neuropathy (LHON) patients.
Ethyl linoleate (ELA) is an essential fatty acid used in many cosmetics for its anti-inflammatory properties. It inhibits the action of the reactive species of oxygen released by neutrophils due to an excess of bacteria, and prevents the hyperkeratinization induced by a lack of linoleic acid. Though there are few reports stating the antiinflammatory activity of ELA, the mechanism by which ELA exhibits anti-inflammatory activity remains unclear. An aqueous emulsion of ethyl linoleate can be used as parenteral injection for curring ailments where a high plasma-cholesterol level of the blood is indicated. Improvements in liver function tests during the administration of ELA were noticed in almost all cases with a few aggravated exceptions. The administration of ELA can expect the satisfactory response to the patients both with liver cirrhosis and with chronic hepatitis.
Pioglitazone (brand name Actos) is a prescription drug of the thiazolidinedione class with hypoglycemic action used in the treatment of type 2 diabetes. Pioglitazone selectively stimulates the nuclear receptor peroxisome proliferator-activated receptor gamma (PPAR-γ) and to a lesser extent PPAR-α. It modulates the transcription of the genes involved in the control of glucose and lipid metabolism in the muscle, adipose tissue, and the liver. As a result, pioglitazone reduces insulin resistance in the liver and peripheral tissues, decreases gluconeogenesis in the liver, and reduces the quantity of glucose and glycated hemoglobin in the bloodstream. Pioglitazone is used to lower blood glucose levels in the treatment of diabetes mellitus type 2 (T2DM) either alone or in combination with a sulfonylurea, metformin, or insulin. Pioglitazone cannot be used in patients with a known hypersensitivity to pioglitazone, other thiazolidinediones or any of components of its pharmaceutical forms. It is ineffective and possibly harmful to diabetes mellitus type 1 and diabetic ketoacidosis. Pioglitazone can cause fluid retention and peripheral edema. As a result, it may precipitate congestive heart failure (which worsens with fluid overload in those at risk). It may cause anemia. Mild weight gain is common due to increase in subcutaneous adipose tissue. In studies, patients on pioglitazone had an increased proportion of upper respiratory tract infection, sinusitis, headache, myalgia and tooth problems.
Pioglitazone (brand name Actos) is a prescription drug of the thiazolidinedione class with hypoglycemic action used in the treatment of type 2 diabetes. Pioglitazone selectively stimulates the nuclear receptor peroxisome proliferator-activated receptor gamma (PPAR-γ) and to a lesser extent PPAR-α. It modulates the transcription of the genes involved in the control of glucose and lipid metabolism in the muscle, adipose tissue, and the liver. As a result, pioglitazone reduces insulin resistance in the liver and peripheral tissues, decreases gluconeogenesis in the liver, and reduces the quantity of glucose and glycated hemoglobin in the bloodstream. Pioglitazone is used to lower blood glucose levels in the treatment of diabetes mellitus type 2 (T2DM) either alone or in combination with a sulfonylurea, metformin, or insulin. Pioglitazone cannot be used in patients with a known hypersensitivity to pioglitazone, other thiazolidinediones or any of components of its pharmaceutical forms. It is ineffective and possibly harmful to diabetes mellitus type 1 and diabetic ketoacidosis. Pioglitazone can cause fluid retention and peripheral edema. As a result, it may precipitate congestive heart failure (which worsens with fluid overload in those at risk). It may cause anemia. Mild weight gain is common due to increase in subcutaneous adipose tissue. In studies, patients on pioglitazone had an increased proportion of upper respiratory tract infection, sinusitis, headache, myalgia and tooth problems.

Showing 1 - 10 of 12 results