Antroquinonol is isolated from Antrodia camphorata, a camphor tree mushroom, and is a valuable traditional Chinese herbal medicine that exhibits pharmacological activities against several diseases, including cancer. Antroquinonol displayed anticancer activity against hepatocellular carcinoma cell lines through activation of 5′ adenosine-monophosphate-activated protein kinase and inhibition of the mammalian target of rapamycin (mTOR) pathway. Antroquinonol also exhibits anticancer activity in human pancreatic cancers through inhibition of the phosphoinositide-3 kinase (PI3K)/Akt/mTOR pathway, which in turn downregulates the expression of cell cycle regulators. The translational inhibition causes a G1 arrest of the cell cycle and ultimately mitochondria-dependent apoptosis. A study on the A549 pulmonary adenocarcinoma cell line demonstrated that antroquinonol-induced apoptosis was associated with disrupted mitochondrial membrane potential and activation of caspase-3 and poly ADP ribose polymerase cleavage. Moreover, antroquinonol treatment downregulated the expression of B-cell lymphoma 2 proteins, which was correlated with decreased PI3K and mTOR protein levels, without altering the levels of pro- or antiapoptotic proteins. Antroquinonol is currently in phase II trials (USA and Taiwan) for the treatment of non-small-cell lung carcinoma (NSCLC), atopic dermatitis; colorectal cancer; hepatitis B; hyperlipidaemia; pancreatic cancer. Antroquinonol was also approved for drug clinical trials by the Russian Ministry of Health (MoH). The MoH gave permission to test the efficacy and safety of Phase II clinical trials in patients with acute myeloid leukemia in Russia. Antroquinonol received the Orphan Drug Designation by the FDA in treatment of pancreatic cancer, liver cancer and acute myeloid leukaemia.

2-[18F]Fludarabine is a Positron Emission Tomography (PET) tracer for imaging lymphoma.

Tomivosertib (eFT-508) is a dual inhibitor of MAP kinase-interacting kinases (MNK1 and MNK2) thereby controlling translation. It has potent in vivo antitumor activity in models of diffuse large cell B-cell lymphoma and solid tumors. Tomivosertib acts on multiple points in the cancer immunity cycle simultaneously, underscoring its therapeutic potential as both a monotherapy and in combination with existing checkpoint inhibitor treatments. Preclinical studies suggest combining tomivosertib with a checkpoint inhibitor can overcome mechanisms of resistance to checkpoint inhibitors, resulting in enhanced sensitivity to checkpoint inhibitors and a higher response rate. In addition, preclinical data demonstrate that tomivosertib as a single agent promotes antitumor immunity that persists after stopping drug treatment. Tomivosertib is currently being evaluated in Phase 2 clinical trials in solid tumors and lymphoma. Additionally, tomivosertib may be a new therapeutic for neuropathic pain.

BL-1021 was developed by BioLineRx Ltd. for the treatment of neuropathic pain or pain that results from damage to nerve fibers. The drug has completed phase I clinical trials, however further information is not available.

SCY-635 is a nonimmunosuppressive cyclosporine-based analog that exhibits potent suppression of hepatitis C virus (HCV) replication. SCY-635 has reached the phase II clinical trial for the treatment of patients with hepatitis C Infection. In addition, in the preclinical model, the SCY-635 was studied for the treatment of hepatitis-B in USA.

CE-326597 is a potent and selective agonist of type 1 cholecystokinin receptor, developed by Pfizer Inc for the treatment of obesity. The physical properties of CE-326597 provided the desired low systemic exposure which was driven by poor absorption of the drug into the intestinal wall. Unfortunately, CE-326597 demonstrated insufficient efficacy for the treatment of either diabetes or obesity after 12 weeks of dosing in Phase 2 clinical trial and was discontinued.

CX-5461 represents an innovative-targeted agent with numerous differentiating features when compared to current options for treatment of hematologic cancers. CX-5461 is a first-in-class small molecule inhibitor of RNA polymerase I (Pol I) that triggers the nucleolar stress surveillance pathways to activate p53, without causing direct DNA damage. Molecular studies demonstrate that CX-5461 inhibits the initiation stage of rRNA synthesis and induces both senescence and autophagy, but not apoptosis, through a p53-independent process in solid tumor cell lines. Currently, CX-5461 is in clinical trial for patients with advanced hematological malignancies.

Dizocilpine (MK-801) is an antagonist of the N-methyl-D-aspartate (NMDA) receptor in the glutamate category involved with the central nervous system (CNS). The drug displays a variety of physiological actions, many of which involve the CNS, such as anesthetic and anticonvulsant properties. It penetrates readily into CNS and was described as the agent with central sympathomimetic properties. Co-administration of dizocilpine with psychostimulants, such as cocaine, amphetamine and nicotine, has been reported to prevent the development of behavioural sensitization to these drugs as well as associated neuroadaptations in rodents. However, studies with bromocriptine have suggested that co-administration of dizocilpine might merely cause sensitization to become state-dependent. A single injection of MK-801 to rats models both positive and negative symptoms of schizophrenia. Treatment of mice with dizocilpine induced learning impairment.