Omacetaxine mepesuccinate (trade name Synribo) formerly named as homoharringtonine or HHT, is a pharmaceutical drug substance that is indicated for treatment of chronic myeloid leukemia (CML). It is a natural ester of the alkaloid cephalotaxine from Cephalotaxus harringtonia, now manufactured by hemi-synthesis. It was approved by the US FDA in October 2012 for the treatment of adult patients with CML with resistance and/or intolerance to two or more tyrosine kinase inhibitors (TKIs). The mechanism of action of omacetaxine mepesuccinate has not been fully elucidated but includes inhibition of protein synthesis and is independent of direct Bcr-Abl binding. Omacetaxine mepesuccinate binds to the A-site cleft in the peptidyl-transferase center of the large ribosomal subunit from a strain of archaeabacteria. In vitro, omacetaxine mepesuccinate reduced protein levels of the Bcr Abl oncoprotein and Mcl-1, an anti-apoptotic Bcl-2 family member. Omacetaxine mepesuccinate showed activity in mouse models of wild-type and T315I mutated Bcr-Abl CML.

Lorcaserin, currently marketed under the trade name Belviq and previously Lorqess during development, is a weight-loss drug developed by Arena Pharmaceuticals. Lorcaserin is a selective 5-HT2C receptor agonist, and in vitro testing of the drug showed reasonable selectivity for 5-HT2C over other related targets. 5-HT2C receptors are located almost exclusively in the brain, and can be found in the choroid plexus, cortex, hippocampus, cerebellum, amygdala, thalamus, and hypothalamus. The activation of 5-HT2C receptors in the hypothalamus is supposed to activate proopiomelanocortin (POMC) production and consequently promote weight loss through satiety. This hypothesis is supported by clinical trials and other studies. While it is generally thought that 5-HT2C receptors help to regulate appetite as well as mood, and endocrine secretion, the exact mechanism of appetite regulation is not yet known. Lorcaserin has shown 100x selectivity for 5-HT2C versus the closely related 5-HT2B receptor, and 17x selectivity over the 5-HT2A receptor

L-Malic acid is a tart-tasting organic dicarboxylic acid that plays a role in many sour or tart foods. L-Malic acid is the naturally occurring form, whereas a mixture of L- and D-malic acid is produced synthetically. In humans, L-malic acid is both derived from food sources and synthesized in the body through the citric acid cycle or Krebs cycle which takes place in the mitochondria. L-Malate's importance to the production of energy in the body during both aerobic and anaerobic conditions is well established. Under aerobic conditions, the oxidation of L-malate to oxaloacetate provides reducing equivalents to the mitochondria through the malate-aspartate redox shuttle. During anaerobic conditions, where a buildup of excess of reducing equivalents inhibits glycolysis, L-malic acid's simultaneous reduction to succinate and oxidation to oxaloacetate is capable of removing the accumulating reducing equivalents. This allows L-malic acid to reverse hypoxia's inhibition of glycolysis and energy production. In studies on rats it has been found that only tissue malate is depleted following exhaustive physical activity. Notably, the administration of malic acid to rats has been shown to elevate mitochondrial malate and increase mitochondrial respiration and energy production. L-Malic acid is the source of extreme tartness in United States-produced confectionery, the so-called extreme candy. It is also used with or in place of the less sour citric acid in sour sweets. These sweets are sometimes labeled with a warning stating that excessive consumption can cause irritation of the mouth. The quantitative determination of L-malic acid is especially important in the manufacture of wine, beer, bread, fruit and vegetable products, as well as in cosmetics and pharmaceuticals. It is one of the most important fruit acids, and has the highest concentration of all acids in wine. In the wine industry, the level of L-malic acid is monitored, along with L-lactic acid, during malolactic fermentation. Malic acid is approved for use as a food additive in the EU, US and Australia and New Zealand. Malic acid, when added to food products, is denoted by E number E296.

Sodium 2-naphthalenesulfonate is a sodium salt of naphthalene sulfonic acid. Sodium 2-naphthalenesulfonate is a surfactant-hydrotrope used in cosmetics. Use concentrations would be typically below 2%. In clinical studies, Sodium 2-naphthalenesulfonate was neither an irritant (tested up to 2%), cumulative irritant (tested up to 1%), nor a sensitizer (tested up to 1%). Sodium 2-naphthalenesulfonate is considered safe as used in cosmetic formulations intended to be applied to the skin.

Citrulline (name derived from citrullus, the Latin word for watermelon, from which it was first isolated) is an amino acid. It is made from ornithine and carbamoyl phosphate in one of the central reactions in the urea cycle. It is also produced from arginine as a by-product of the reaction catalyzed by nitric oxide synthase (NOS) family. Citrulline supplements have been claimed to promote energy levels, stimulate the immune system and help detoxify ammonia (a cell toxin). Citrulline is not involved in protein synthesis. Several pharmacokinetic studies have confirmed that citrulline is efficiently absorbed when administered orally. Oral citrulline could be used to deliver arginine to the systemic circulation or as a protein anabolic agent in specific clinical situations (for example in case of malnourishment), because recent data have suggested that citrulline, although not a component of proteins, stimulates protein synthesis in skeletal muscle through the mammalian target of rapamycin signaling pathway. Citrulline is converted to L-arginine by argininosuccinate synthase. L-arginine is in turn responsible for citrulline's therapeutic effects. Many of L-arginine's activities, including its possible anti-atherogenic actions, may be accounted for by its role as the precursor to nitric oxide (NO).

Methylenetetrahydrofolate reductase (MTHFR) is an enzyme required for the formation of 5-methyltetrahydrofolate (5-MTHF), a form of folate able to cross the blood-brain barrier and which is necessary as a substrate for the remethylation of homocysteine to methionine by methionine synthase. Patients with severe MTHFR deficiency cannot make 5-MTHF and have extremely low levels in the CSF. Only treatment with oral 5-MTHF given as calcium mefolinate resulted in an increase in CSF 5-MTHF.