Aminoglutethimide, marketing as Cytadren has been used in the treatment of advanced breast and prostate cancer. It was formerly used for its weak anticonvulsant properties. Cytadren is indicated for the suppression of adrenal function in selected patients with Cushing’s syndrome. Morning levels of plasma cortisol in patients with adrenal carcinoma and ectopic ACTH producing tumors were reduced on the average to about one half of the pretreatment levels, and in patients with adrenal hyperplasia to about two thirds of the pretreatment levels, during 1-3 months of therapy with Cytadren. Data available from the few patients with adrenal adenoma suggest similar reductions in plasma cortisol levels. Measurements of plasma cortisol showed reductions to at least 50% of baseline or to normal levels in one third or more of the patients studied, depending on diagnostic groups and time of measurement. Because Cytadren does not affect the underlying disease process, it is used primarily as an interim measure until more definitive therapy such as surgery can be undertaken or in cases where such therapy is not appropriate. Only small numbers of patients have been treated for longer than 3 months. A decreased effect or “escape phenomenon” seems to occur more frequently in patients with pituitary dependent Cushing’s syndrome, probably because of increasing ACTH levels in response to decreasing glucocorticoid levels. Cytadren blocks several other steps in steroid synthesis, including the C-11, C-18, and C-21 hydroxylations and the hydroxylations required for the aromatization of androgens to estrogens, mediated through the binding of Cytadren to cytochrome P-450 complexes. A decrease in adrenal secretion of cortisol is followed by an increased secretion of pituitary adrenocorticotropic hormone (ACTH), which will overcome the blockade of adrenocortical steroid synthesis by Cytadren. The compensatory increase in ACTH secretion can be suppressed by the simultaneous administration of hydrocortisone. Since Cytadren increases the rate of metabolism of dexamethasone but not that of hydrocortisone, the latter is preferred as the adrenal glucocorticoid replacement. Although Cytadren inhibits the synthesis of thyroxine by the thyroid gland, the compensatory increase in thyroid-stimulating hormone (TSH) is frequently of sufficient magnitude to overcome the inhibition of thyroid synthesis due to Cytadren. In spite of an increase in TSH, Cytadren has not been associated with increased prolactin secretion. At low doses, aminogluthethimide is only an effective inhibitor of aromatase (Cytochrome P450 11A1), but at higher doses, it effectively blocks Cytochrome P450 11A1 (P450scc) as well. Citadel was marketed previously as an anticonvulsant but was withdrawn from marketing for that indication in 1966 because of the effects on the adrenal gland.

Fumagillin, an antimicrobial compound first isolated in 1949 from the fungus Aspergillus fumigatusa, naturally occurring water-insoluble antibacterial agent developed by sanofi-aventis, is approved in France for the treatment of microsporidiosis. Fumagillin (Flisint, Sanofi-Aventis, Paris, France) has been approved in France since 2002 for the treatment of intestinal microsporidiosis due to E. bieneusi in patients with AIDS, and is also available through an expanded access program for patients without AIDS. It has not been approved, however, by the US Food and Drug Administration. The discovery of fumagillin, a MetAP-2 inhibitor, with potent antiangiogenic and antiproliferative activities promoted the development of fumagillin analogues as a novel class of anticancer agents. It has been the subject of research in cancer treatments by employing its angiogenesis inhibitory properties.

TRIETHYLENEMELAMINE, an aziridine derivative, is an alkylating agent with antineoplastic properties. Formerly it was used in the palliative treatment of the lymphomas and leukemias. Now it is used as a research tool to produce chromosome aberrations and cancers.

Aminopterin is a synthetic derivative of pterins with antineoplastic and immunosuppressive properties. As a folate analog, aminopterin competes for the folate binding site of the enzyme dihydrofolate reductase, thereby blocking tetrahydrofolate synthesis, and resulting in depletion of nucleotide precursors and inhibition of DNA, RNA and protein synthesis. Aminopterin was marketed by Lederle Laboratories (Pearl River, New York) in the United States from 1953 to 1964 for the indication of pediatric leukemia. The closely related antifolate methotrexate was simultaneously marketed by the company during the same period. Aminopterin was discontinued by Lederle Laboratories in favor of methotrexate due to manufacturing difficulties of the former. During the period Aminopterin was marketed, the agent was used off-label to safely treat over 4,000 patients with psoriasis in the United States, producing dramatic clearing of lesions. The use of aminopterin in cancer treatment was supplanted in the 1950s by methotrexate due to the latter's better therapeutic index in a rodent tumor model. Now in a more pure preparation and supported by laboratory evidence of superior tumor cell uptake in vitro, aminopterin is being investigated in clinical trials in leukemia as a potentially superior antifolate to methotrexate.

Noscapine (also known as Narcotine, Nectodon, Nospen, Anarcotine and (archaic) Opiane) is a benzylisoquinoline alkaloid from plants of the poppy family, without painkilling properties. This agent is primarily used for its antitussive (cough-suppressing) effects. Noscapine is often used as an antitussive medication. A 2012 Dutch guideline, however, does not recommend its use for coughing. Noscapine can increase the effects of centrally sedating substances such as alcohol and hypnotics. Noscapine should not be taken in conjunction with warfarin as the anticoagulant effects of warfarin may be increased. Noscapine, and its synthetic derivatives called noscapinoids, are known to interact with microtubules and inhibit cancer cell proliferation. Mechanisms for its antitussive action are unknown, although animal studies have suggested central nervous system as a site of action. Furthermore, noscapine causes apoptosis in many cell types and has potent antitumor activity against solid murine lymphoid tumors (even when the drug was administered orally) and against human breast and bladder tumors implanted in nude mice. Because noscapine is water-soluble and absorbed after oral administration, its chemotherapeutic potential in human cancer merits thorough evaluation. Antifibrotic effect of noscapine based on novel mechanism, which it shows through EP2 prostaglandin E2 receptor-mediated activation of protein kinase A.

2′-Deoxycytidine (deoxyC) is one of the deoxy nucleosides, which after phosphorylation to dCTP is used to synthesize DNA via various DNA polymerases or reverse transcriptases. Deoxycytidine is phosphorylated by deoxycytidine kinase (dCK). This enzyme catalyzes the initial conversion of the nucleosides deoxyadenosine (dA), deoxyguanosine (dG), and deoxycytidine (dC) into their monophosphate forms, with subsequent phosphorylation to the triphosphate forms performed by additional enzymes.

Perflenapent (EchoGen, Sonus Pharmaceuticals, Bothel, WA, USA) is a transpulmonary echocardiographic contrast agent for use in patients with suspected or established cardiovascular disease to provide opacification of cardiac chambers, enhance left ventricular border delineation with resulting improvement in wall motion visualisation. EchoGen comprises liquid droplets of dodecafluoropentane stabilised by polyfluoroalkyl(polyoxyethylene) ethanol and dispersed in an aqueous formulation containing sucrose. Dodecafluoropentane is the chemical name used to reflect the perflenapent/perflisopent mixture. Following activation and administration of EchoGen, dodecafluoropentane microbubbles are formed. EchoGen should only be used in patients where the study without contrast enhancement is inconclusive. The European Commission has approved SONUS Pharmaceuticals' EchoGen (perflenapent emulsion) in all 15 countries of the European Union. EchoGen is a fluorocarbon-based ultrasound contrast agent which has been approved as a transpulmonary echocardiographic contrast agent for use in patients with suspected or established cardiovascular disease who have had previous inconclusive non-contrast studies. Additional approved labeling in the EU states that the use of EchoGen in spectral and color Doppler studies was shown to enhance the visualization of blood flow across mitral, aortic and tricuspid valves in a subset of patients. In 2000 SONUS Pharmaceuticals withdrew the NDA for EchoGen in the USA.

Psoralen, the active ingredient of Fructus Psoraleae (FP) the dried ripe fruit of Psoralea corylifolia L., which is used in Chinese herbal medicine. Psoralen promotes chondrocyte proliferation by activating the Wnt/β-catenin signaling pathway and can play an important role in the treatment of osteoarthritis. Besides, this compound stimulates osteoblast proliferation through the activation of NF-κB and MAPK signaling and may be a viable therapeutic agent in the treatment of osteoporosis.

Cordycepin, or 3'-deoxyadenosine, is a derivative of the nucleoside adenosine, differing from the latter by the absence of the hydroxy group in the 3' position of its ribose part. Cytostatic effect of cordycepin is due to incorporation of phospho-cordycepin into mRNA and inhibition of mRNA synthesis. Cordycepin exhibit rapid antidepressant effect due to potentiation of AMPA receptors