Phenylacetic acid (abr. PAA and synonyms are: α-toluic acid, benzeneacetic acid, alpha tolylic acid, 2-phenylacetic acid, β-phenylacetic acid) is an organic compound containing a phenyl functional group and acarboxylic acid functional group. Because it is used in the illicit production of phenylacetone (used in the manufacture of substituted amphetamines), it is subject to controls in countries including the United States and China Phenylacetic acid is used in some perfumes, possessing a honey-like odor in low concentrations, and is also used in penicillin G production. It is also employed to treat type II hyperammonemia to help reduce the amounts of ammonia in a patient's bloodstream by forming phenylacetyl-CoA, which then reacts with nitrogen-rich glutamine to form phenylacetylglutamine. This compound is then secreted by the patient's body. In Phase 2 of clinical research it investigated in the treatment of Brain and Central Nervous System Tumors.

Cytarabine is a pyrimidine nucleoside analog. Cytarabine or cytosine arabinoside (Cytosar-U or Depocyt) is a chemotherapy agent used mainly in the treatment of cancers of white blood cells such as acute myeloid leukemia (AML) and non-Hodgkin lymphoma. It also has antiviral and immunosuppressant properties. Cytarabine is an antimetabolite antineoplastic agent that inhibits the synthesis of DNA. It is a cell cycle phase-specific, affecting cells only during the S phase of cell division. Intracellularly, cytarabine is converted into cytarabine-5-triphosphate (ara-CTP), which is the active metabolite. The mechanism of action is not completely understood, but it appears that ara-CTP acts primarily through inhibition of DNA polymerase. Incorporation into DNA and RNA may also contribute to cytarabine cytotoxicity. Cytarabine is cytotoxic to a wide variety of proliferating mammalian cells in culture.The drug has a short plasma half-life, low stability and limited bioavailability. Overdosing of patients with continuous infusions may lead to side effects. Thus, various prodrug strategies and delivery systems have been explored extensively to enhance the half-life, stability and delivery of cytarabine. Alternative, delivery systems of cytarabine have emerged for the treatment of different cancers. The liposomal-cytarabine formulation has been approved for the treatment of lymphomatous meningitis.

TPI-287 is a microtubule stabilizer. TPI-287 readily crossed the normal BBB. While paclitaxel and TPI-287 showed similar cytotoxicity to breast cancer cells in vitro, and in a model of primary breast tumors, only TPI-287 was able to significantly reduce metastatic colonization of breast cancer in the brain. TPI-287 is a poor substrate for the P-gp efflux pump. Potential limitation to the clinical use of TPI-287 is the weight loss observed upon treatment. This loss recovered after cessation of treatment in the early treatment model, and may be ameliorated by hydration. TPI 287 was well tolerated by pediatric patients with refractory and relapsed neuroblastoma and medulloblastoma. The combination of TPI 287 and temozolomide is well tolerated in patients with metastatic melanoma, with the exception of neuropathy. TPI 287 is in phase II clinical trial for the treatment of breast cancer, glioblastoma and malignant melanoma.

Antineoplaston A10 is a piperidinedione antineoplaston with potential antineoplastic activity. Antineoplaston A10 was originally isolated from human urine but is now synthetically derived. This agent intercalates into DNA, resulting in cell cycle arrest in G1 phase, reduction of mitosis, and decreased protein synthesis. Antineoplaston A10 may also inhibit ras-oncogene expression and activate tumor suppressor gene p53, leading to cell differentiation and apoptosis. Antineoplaston A10 has been used in trials studying the treatment of glioma, sarcoma, lymphoma, lung cancer, liver cancer, and kidney cancer, among others.

7β-hydroxycholesteryl-3-oleate has been shown to inhibit astrogliosis and intracranial glioblastoma growth. Local administration of 7β-hydroxycholesteryl-3-oleate inhibits growth of experimental rat C6 glioblastoma. These data suggest that 7β-hydroxycholesteryl-3-oleate might be useful for local glioblastoma chemotherapy. 7β-hydroxycholesteryl-3-oleate is a potent inhibitor of the endogenous cholesterol biosynthesis in brain showing a correlation between cholesterogenesis and reactive astrocyte proliferation. 7β-hydroxycholesteryl-3-oleate can reduce the astrocytic reaction following spinal cord injury, promoting the serotonergic reinnervation of a denervated territory. On 17 December 2010, orphan designation (EU/3/10/816) was granted by the European Commission to Intsel Chimos SA, France, for 7-beta-hydroxy cholesteryl-3-beta-oleate for the treatment of glioma. The substance is going to be injected directly into the brain tumour contained within liposomes, which are expected to carry the medicine into the glioma cells.

Oxaloacetate (OAA), a salt of oxaloacetic acid, is a metabolic intermediate in many processes, e.g., urea cycle, gluconeogenesis, etc. that occur in animals. Experiments on animal have revealed that OAA was able to protect hepatocytes from hypoxia and liver ischemia/reperfusion injury. OAA also possesses a neuroprotective effect against ischemic injury, which strengthens the likelihood of its future applicability as a novel neuroprotective agent for the treatment of ischemic stroke patients. In addition, experiments on adipose stromal cells have shown that OAA directly protected cerebellar granule neurons from apoptosis induced by serum and potassium deprivation.

Topotecan, a semi-synthetic derivative of camptothecin (a plant alkaloid obtained from the Camptotheca acuminata tree), is an anti-tumor drug with topoisomerase I-inhibitory activity similar to irinotecan. DNA topoisomerases are enzymes in the cell nucleus that regulate DNA topology (3-dimensional conformation) and facilitate nuclear processes such as DNA replication, recombination, and repair. During these processes, DNA topoisomerase I creates reversible single-stranded breaks in double-stranded DNA, allowing intact single DNA strands to pass through the break and relieve the topologic constraints inherent in supercoiled DNA. The 3'-DNA terminus of the broken DNA strand binds covalently with the topoisomerase enzyme to form a catalytic intermediate called a cleavable complex. After DNA is sufficiently relaxed and the strand passage reaction is complete, DNA topoisomerase reattaches the broken DNA strands to form the unaltered topoisomers that allow transcription to proceed. Topotecan interferes with the growth of cancer cells, which are eventually destroyed. Since the growth of normal cells can be affected by the medicine, other effects may also occur. Unlike irinotecan, topotecan is found predominantly in the inactive carboxylate form at neutral pH and it is not a prodrug. Topotecan has the same mechanism of action as irinotecan and is believed to exert its cytotoxic effects during the S-phase of DNA synthesis. Topoisomerase I relieves torsional strain in DNA by inducing reversible single strand breaks. Topotecan binds to the topoisomerase I-DNA complex and prevents religation of these single strand breaks. This ternary complex interferes with the moving replication fork, which leads to the induction of replication arrest and lethal double-stranded breaks in DNA. As mammalian cells cannot efficiently repair these double strand breaks, the formation of this ternary complex eventually leads to apoptosis (programmed cell death). Topotecan mimics a DNA base pair and binds at the site of DNA cleavage by intercalating between the upstream (−1) and downstream (+1) base pairs. Intercalation displaces the downstream DNA, thus preventing religation of the cleaved strand. By specifically binding to the enzyme–substrate complex, Topotecan acts as an uncompetitive inhibitor. Topotecan is used for the treatment of advanced ovarian cancer in patients with disease that has recurred or progressed following therapy with platinum-based regimens. Also used as a second-line therapy for treatment-sensitive small cell lung cancer, as well as in combination with cisplatin for the treatment of stage IV-B, recurrent, or persistent cervical cancer not amenable to curative treatment with surgery and/or radiation therapy. Topotecan is sold under the trade name Hycamtin.

KX-02 is a compound demonstrating dual inhibitory activity against Src kinase and tubulin polymerization. It readily crosses the blood-brain-barrier in mice. It is under development for the treatment of solid tumors.

Antineoplaston (Phenylacetylglutamine) is the amino acid acetylation product of phenylacetate (or phenylbutyrate after beta-oxidation). The chemical structure of Antineoplaston AS2-5 corresponds to phenylacetylglutamine. Two synthetic derivatives of Antineoplaston A10 were named Antineoplaston AS2-1 and AS2-5. All antineoplaston formulations were submitted for Phase I clinical studies in advanced cancer patients. The treatment was free from significant side-effects and resulted in objective response in a number of advanced cancer cases. Antineoplastons are an experimental cancer therapy developed by S.R. Burzynski, MD, PhD. Chemically, antineoplastons are a mixture of amino acid derivatives, peptides, and amino acids found in human blood and urine. The developer originally isolated antineoplastons from human blood and later found the same peptides in urine. Urine was subsequently used because it was less expensive and easier to obtain. Since 1980, antineoplastons have been synthesized from commercially available chemicals at the Burzynski Research Institute. According to the developer, antineoplastons are part of a biochemical surveillance system in the body and work as "molecular switches." For the developer, cell differentiation is the key to cancer therapy. At the molecular level, abnormal cells that are potential cancer cells need to be "switched" to normal mode. Antineoplastons are the surveillance system that directs cancer cells into normal channels of differentiation. According to statements published by the developer, people with cancer lack this surveillance system because they do not have an adequate supply of antineoplastons.