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

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Showing 591 - 600 of 9908 results

Status:
Designated
Source:
FDA ORPHAN DRUG:312010
Source URL:

Class (Stereo):
CHEMICAL (ABSOLUTE)

Epitalon (amino acid sequence Ala-Glu-Asp-Gly) is a synthetic peptide that has been suggested to induce the activation of ribosomal genes, decondensation of pericentromeric structural heterochromatin and the release of genes repressed due to the age-related condensation of euchromatic chromosome regions. The researchers who generated Epitalon also reported an inhibitory effect on the development of spontaneous tumors in mice, and claimed that it increases neuronal activity and can be used in the treatment of Retinitis Pigmentosa. The peptide is still under pre‐clinical and clinical development and thus has not been approved for any therapeutic and/or prophylactic use by any government health authority in the USA or Europe.
Status:
Designated
Source:
FDA ORPHAN DRUG:517616
Source URL:

Class (Stereo):
CHEMICAL (ABSOLUTE)

Florilglutamic acid enters cells through a transport system known as xC- (cystine/glutamate antiporter) which is more abundant in cancer tissue. Florilglutamic acid (18F) is a radioactive compound for use with an imaging method known as positron emission tomography (PET). When injected into the patient, florilglutamic acid (18F) is more effectively taken up by the cancer cells in the liver from where it is expected to emit radiation that can be detected in a PET scan, thereby allowing the cancer to be diagnosed. Orphan designation (EU/3/16/1632) was granted by the European Commission to Piramal Imaging GmbH, Germany, for florilglutamic acid (18F) for the diagnosis of hepatocellular carcinoma.
Status:
Designated
Source:
FDA ORPHAN DRUG:321010
Source URL:

Class (Stereo):
CHEMICAL (ABSOLUTE)

GUANOSINE 5'-DIPHOSPHO-β-L-FUCOSE is classified as a member of the Purine nucleotide sugars. Guanosine 5'-diphospho-β-L-fucose sodium salt sodium salt is a substrate for fucosyltransferase.
Status:
Designated
Source:
FDA ORPHAN DRUG:643918
Source URL:

Class (Stereo):
CHEMICAL (ABSOLUTE)

Targets:

Conditions:

Monomethyl Auristatin E (MMAE) is an antimitotic agent which inhibits cell division by blocking the polymerization of tubulin. Monomethyl Auristatin E is the synthetic analog of the antineoplastic natural product Dolastatin 10, cannot be used as a drug itself. Monomethyl Auristatin E is commonly conjugated with monoclonal antibodies directed at antigens specific to cancer cells for tumor-directed cytotoxicity. MMAE is typically coupled to the antibody via a protease-cleavable linker, allowing separation of the drug from the antibody following intracellular localization. When coupled to cAC10, Monomethyl Auristatin E shows selective cytotoxicity in CD30+ cells and induces G2/M-phase growth arrest and cell death through the induction of apoptosis. When coupled to the anti-CD79b antibody, anti–CD79b-vcMMAE has very potent and broad activity across a large panel of NHL cell lines in vitro. When coupled to the anti-HER2 antibody, pertuzumab-vc-MMAE can also be effectively internalized and potently kill HER2 over-expressing tumor cells. In the Karpas 299 ALCL model, cAC10-vcMMAE induces complete, durable tumor regression, while free MMAE doesn’t produce detectable antitumor activity. In mouse xenograft models of NHL, anti–CD79b-vcMMAE strikingly results in sustained complete tumor remission.
Status:
Designated
Source:
FDA ORPHAN DRUG:96496
Source URL:

Class (Stereo):
CHEMICAL (ABSOLUTE)



DMP-777 (also termed L-694458) a cell permeant b-lactam inhibitor of a human leukocyte elastase was developed for treatment cystic fibrosis, juvenile rheumatoid arthritis.
Leupeptin is produced by various species of Actinomycetes. It strongly inhibits proteolysis by plasmin, trypsin and papin. Leupeptin is well absorbed through oral route. Leupeptin has been known to cause various neuropathological changes in vivo resembling those of aging or neurodegenerative processes in the human brain, including the accumulation of neuronal processes and neuronal cytoskeletal abnormalities leading to neurofibrillary tangle (NFT)-like formations. In in vitro experiments, leupeptin protects the heart from myocardial stunning. Leupeptin was found to inhibit tumorigenesis in mouse skin induced by a single, noncarcinogenic dose of 7,12- dimethylbenz(a)anthracene followed by repeated application of croton oil. Tumors that had already been induced were scarcely affected by leupeptin.
Geneticin (G-418) is produced by Micromonospora phodorangea. The antibiotic has brad spectrum antibacterial activity similar to gentamicins and other previously described aminoglycosides but of particular interest because of its high activity against protozoa, amoebae, tapeworm and pinworm infections in mice. G418 blocks polypeptide synthesis by inhibiting the elongation step in both prokaryotic and eukaryotic cells. G418 is commonly used in laboratory research to select genetically engineered cells.