Levamisole (the trade name Ergamisol), an anthelminthic drug with immunological properties. It also has antitumor activity when administered with 5-fluorouracil in patients with Duke's C colorectal carcinoma; however, this use was discontinued. The mechanism of the antitumor effect is unknown but has been postulated to be related to levamisole's immunomodulatory properties. Levamisole can stimulate antibody formation to various antigens, enhance T-cell responses by stimulating T-cell activation and proliferation, potentiate monocyte and macrophage functions including phagocytosis, chemotaxis and increases motility, adherence, and chemotaxis. Levamisole inhibits alkaline phosphatase and possesses cholinergic activity. The mechanism of action of levamisole as an antiparasitic agent, for example, to treat ascariasis, relates to its agonistic activity to L-subtype nicotinic acetylcholine receptors in nematode muscles. In addition, levamisole was studied for preventing relapses of the steroid-sensitive idiopathic nephrotic syndrome (SSINS). It was shown, that alone or in combination with steroids, the drug can prolong the time to relapse and prevented recurrence during one year of treatment. However, these studies also were also discontinued.

Thioguanine is an antineoplastic anti-metabolite used in the treatment of several forms of leukemia including acute nonlymphocytic leukemia. Anti-metabolites masquerade as purine or pyrimidine - which become the building blocks of DNA. They prevent these substances becoming incorporated in to DNA during the "S" phase (of the cell cycle), stopping normal development and division. Thioguanine was first synthesized and entered into clinical trial more than 30 years ago. It is a 6-thiopurine analogue of the naturally occurring purine bases hypoxanthine and guanine. Intracellular activation results in incorporation into DNA as a false purine base. An additional cytotoxic effect is related to its incorporation into RNA. Thioguanine is cross-resistant with mercaptopurine. Cytotoxicity is cell cycle phase-specific (S-phase). Thioguanine competes with hypoxanthine and guanine for the enzyme hypoxanthine-guanine phosphoribosyltransferase (HGPRTase) and is itself converted to 6-thioguanilyic acid (TGMP), which reaches high intracellular concentrations at therapeutic doses. TGMP interferes with the synthesis of guanine nucleotides by its inhibition of purine biosynthesis by pseudofeedback inhibition of glutamine-5-phosphoribosylpyrophosphate amidotransferase, the first enzyme unique to the de novo pathway of purine ribonucleotide synthesis. TGMP also inhibits the conversion of inosinic acid (IMP) to xanthylic acid (XMP) by competition for the enzyme IMP dehydrogenase. Thioguanine nucleotides are incorporated into both the DNA and the RNA by phosphodiester linkages, and some studies have shown that incorporation of such false bases contributes to the cytotoxicity of thioguanine. Its tumor inhibitory properties may be due to one or more of its effects on feedback inhibition of de novo purine synthesis; inhibition of purine nucleotide interconversions; or incorporation into the DNA and RNA. The overall result of its action is a sequential blockade of the utilization and synthesis of the purine nucleotides. Thioguanine is used for remission induction and remission consolidation treatment of acute nonlymphocytic leukemias. It is marketed under the trade name Lanvis and Tabloid among others.

Phytic acid is a major phosphorus storage compound of most seeds and cereal grains. It has the strong ability to chelate multivalent metal ions, especially zinc, calcium, and iron. Phytic acid is also considered to be a natural antioxidant and is suggested to have potential functions of reducing lipid peroxidation and as a preservative in foods. Clathrin-associated adaprot complex AP-2 has it been suggested may act as one of the receptor sites for Phytic acid. Both in vivo and in vitro experiments have demonstrated striking anticancer (preventive as well as therapeutic) effects of Phytic acid.

Menadione bisulfite is a water-soluble analog of Vitamin K3. Pharmacologic studies on menadione bisulfate indicad that its toxicity is relatively low. In man, doses approximately ten times as great as those generally recommended for therapeutic use, given daily for a period of one week. Redox cycling compounds, such as menadione, have the potential to effectively mitigate the toxicity of organophosphorus pesticides including parathion. Menadione bisulfite behaved as a competitive inhibitor of chicken muscle aldose reductase.

Zuretinol (QLT091001, 9-cis-retinol) is a retinoid. Retinoids (vitamin A and its analogs) are essential dietary substances that are needed by mammals for reproduction, normal embryogenesis, growth, vision, and maintaining normal cellular differentiation and the integrity of the immune system. Within cells, retinoids regulate gene transcription acting through ligand-dependent transcription factors, the retinoic acid receptors (RARs), and the retinoid X receptors (RXRs). All-trans-retinoic acid binds only to RARs with high affinity, whereas its 9-cis isomer binds with high affinity to both RARs and RXRs. The actions of all-trans- and 9-cis-retinoic acid in regulating cellular responses are distinct and not interchangeable. Zuretinol is a retinal derivative for treatment of visual disorders. It is a synthetic retinoid replacement for 11-cis-retinal. It is an investigational product under development for the treatment of retinal diseases caused by gene mutations that interfere with the availability of 11-cis-retinal. The therapeutic strategy with Zuretinol is to facilitate recovery or restoration of visual function by acting as a replacement for missing 11-cis-retinal and restoring a key biochemical component of the visual (retinoid) cycle. Novelion Therapeutics is currently developing QLT091001 for the treatment of Inherited Retinal Disease caused by retinal pigment epithelium protein 65 (“RPE65”) and lecithin:retinol acyltransferase (“LRAT”) gene mutations, which include Leber Congenital Amaurosis (“LCA”) and Retinitis Pigmentosa (“RP”). QLT091001 has received orphan drug designations for the treatment of LCA (due to inherited mutations in the LRAT and RPE65 genes) and RP (all mutations) by the U.S. Food and Drug Administration (the “FDA”), and for the treatment of LCA and RP (all mutations) by the European Medicines Agency (the “EMA”).

Octenidine dihydrochloride is a cationic surfactant, with antimicrobial activity against Gram-positive and Gram-negative bacteria. Octenidine approved as a medicinal substance in several European countries and used for skin antisepsis in combination with aliphatic alcohols, e.g. propan-1-ol and propan-2-ol, or with detergents such as antiseptic soap. Octenidine is also used for antisepsis on wounds and mucosa either as a single substance, as an approved combination of Octenidine and phenoxyethanol. Octenidine is virtually not absorbed via the skin or mucous membranes. Because Octenidine is only approved and used topically and is virtually not absorbed, no systemic effects are to be expected. Therefore, no further pharmacokinetic studies or studies on behalf of metabolism have been conducted. Octenidine is easy and safe to handle, chemically stable, not inflammable, without resistance development and low toxicity to man and the environment alike. Its popularity among therapists and wound care specialists is based on good clinical results, easy and pain-free application and local tolerance. Beside readily available combinations with phenoxyethanol, mouth rinses, and vaginal applications, semi-fluid preparations and dressings are described.

Butyric acid (butanoic acid) belongs to a group of short-chain fatty acids and is thought to play several beneficial roles in the gastrointestinal tract. The butyric anion is easily absorbed by enteric cells and used as a main source of energy. Moreover, butyric acid is an important regulator of colonocyte proliferation and apoptosis, gastrointestinal tract motility and bacterial microflora composition in addition to its involvement in many other processes including immunoregulation and anti-inflammatory activity. Butyric acid shows a protective effect in inflammatory response secondary to inflammatory bowel diseases. A beneficial effect of butyric acid as one constituent of a multifaceted mechanism modulating gastrointestinal function has also been stressed in patients with the stoma and coexisting constipation. Butyric acid supplementation combined with the use of probiotics should be adopted as one of the basic therapeutic strategies in this patient group, preceding treatment with laxatives. Sodium butyrate in the form of enemas (combined in a mixture with A-300 silicon dioxide) may be a successful method of therapeutic management in patients with radiation proctitis. Sodium butyrate may also prevent diarrhea through an increased passive absorption of water in the colon and its effects on the gut microflora.

Dichloroacetic acid, often abbreviated DCA (dichloroacetate), is an acid analog of acetic acid in which two of the three hydrogen atoms of the methyl group have been replaced by chlorine atoms. The salts and esters of dichloroacetic acid are called dichloroacetates. Salts of DCA are used as drugs since they inhibit the enzyme pyruvate dehydrogenase kinase. Early reports of its activity against brain cancer cells led patients to treat themselves with DCA, which is commercially available in non-pharmaceutical grade. A phase 1 study in 5 patients concluded that DCA was safe, but wasn't designed to establish effectiveness. DCA was approved for use in Canada in 1989 (as a topical formulation for the treatment of warts and for cauterization and removal of a wide variety of skin and tissue lesions), but was cancelled post market. DCA is a noncompetitive inhibitor of the endoplasmic reticulum enzyme HMG CoA reductase, which catalyzes the rate limiting step in cholesterol biosynthesis. DCA has been researched in adults, children, animals, and cells as a monotherapy as well as in combination with other therapies for the treatment of severe metabolic disorders including diabetes and hypercholesterolemia, lactic acidosis, certain heart conditions, and cancer. DCA has been prescribed to reduce tumour size and tumour markers, prevent angiogenesis, reduce cancer related symptoms, manage pain, and aid in palliation.

Ornithine is an amino acid produced in the urea cycle by the splitting off of urea from arginine. It is a central part of the urea cycle, which allows for the disposal of excess nitrogen. Ornithine is also a precursor of citrulline and arginine. Arginine stimulates the pituitary release of growth hormone. Burns or other injuries affect the state of arginine in tissues throughout the body. As de novo synthesis of arginine during these conditions is usually not sufficient for normal immune function, nor for normal protein synthesis, ornithine may have immunomodulatory and wound-healing activities under these conditions (by virtue of its metabolism to arginine).

Doconexent (Docosahexaenoic acid, DHA) is an omega-3 fatty acid that is a primary structural component of the human brain, cerebral cortex, skin, and retina. DHA is widely used as a food supplement, and is beleived to support healthy brain development in young childred, prevent cardiovascular disease and cognitive decline during Alzheimer's disease. Most of these claims, however, were not supported by clinical trials. DHA spray is used as a tanner.