Idebenone is a drug that was initially developed by Takeda Pharmaceutical Company for the treatment of Alzheimer's disease and other cognitive defects. This has been met with limited success. The Swiss company Santhera Pharmaceuticals has started to investigate it for the treatment of neuromuscular diseases. In 2010, early clinical trials for the treatment of Friedreich's ataxia and Duchenne muscular dystrophy have been completed. As of December 2013 the drug is not approved for these indications in North America or Europe, and it was refused by the EMA for use in Leber's hereditary optic neuropathy (LHON) in 2013.
A neurofibroma is a benign nerve-sheath tumor in the peripheral nervous system. In 90% of cases, they are found as stand-alone tumors, while the remainder are found in persons with neurofibromatosis type I (NF1), an autosomal-dominant genetically inherited disease, they can result in a range of symptoms from physical disfiguration and pain to cognitive disability. Neurofibromas arise from nonmyelinating-type Schwann cells that exhibit biallelic inactivation of the NF1 gene that codes for the protein neurofibromin. This protein is responsible for regulating the RAS-mediated cell growth signaling pathway. In contrast to schwannomas, another type of tumor arising from Schwann cells, neurofibromas incorporate many additional types of cells and structural elements in addition to Schwann cells, making it difficult to identify and understand all the mechanisms through which they originate and develop.
Belimumab is a human monoclonal antibody that inhibits B-cell activating factor (BAFF), also known as B-lymphocyte stimulator (BLyS). It is approved in the United States, Canada, and Europe to treat systemic lupus erythematosus (SLE).
Lumiliximab is an IgG1k monoclonal antibody that targets CD23. It acts as an immunomodulator and was awarded orphan drug status and fast track designation by the FDA.
Lebrikizumab (INN) is a humanized monoclonal antibody and an experimental immunosuppressive drug for the treatment of asthma that cannot be adequately controlled with inhalable glucocorticoids. The drug was created by Tanox under the name TNX-650, and a phase I clinical trial for refractory Hodgkin’s lymphoma had been performed when Genentech acquired Tanox in 2007. It has successfully completed a Phase II clinical trial for the treatment of asthma.
Peter Jay Hotez is a scientist, pediatrician, and advocate in the fields of global health, vaccinology, and neglected tropical disease control. He serves as founding dean and chief of the Baylor College of Medicine National School of Tropical Medicine in the Department of pediatrics and holds the Texas Children's Hospital Endowed Chair in Tropical Pediatrics.
ClinicalTrials.gov is a registry of clinical trials. It is run by the United States National Library of Medicine (NLM) at the National Institutes of Health, and is the largest clinical trials database, currently holding registrations from over 230,000 trials from 195 countries in the world.
Lenvatinib is an anti-cancer drug for the treatment of certain kinds of thyroid cancer, and potentially for other cancers as well. It was developed by Eisai Co. and acts as a multiple kinase inhibitor against the VEGFR1, VEGFR2 and VEGFR3 kinases.
Briakinumab (ABT-874) is a human monoclonal antibody being developed by Abbott Laboratories for the treatment of rheumatoid arthritis, inflammatory bowel disease, and multiple sclerosis. As of 2011 drug development for psoriasis has been discontinued in the U.S. and Europe.
Mapracorat is an anti-inflammatory drug belonging to the experimental class of selective glucocorticoid receptor agonists (SEGRAs). It is in clinical trials for the topical treatment of atopic dermatitis, inflammation following cataract surgery, and allergic conjunctivitis. Preliminary investigation for the treatment of keratoconjunctivitis sicca has been conducted in cellular models.
Crenolanib besylate is an investigational inhibitor being developed by AROG Pharmaceuticals, LLC. The compound is currently being evaluated for safety and efficacy in clinical trials for various types of cancer, including acute myeloid leukemia (AML), gastrointestinal stromal tumor (GIST), and glioma. Crenolanib is an orally bioavailable benzamidazole that selectively and potently inhibits signaling of wild-type and mutant isoforms of class III receptor tyrosine kinases (RTK) FLT3, PDGFR α, and PDGFR β. Unlike most RTK inhibitors, crenolanib is a type I mutant-specific inhibitor that preferentially binds to phosphorylated active kinases with the ‘DFG in’ conformation motif.
Perfosfamide, or 4-hydroperoxycyclophosphamide was an experimental drug candidate for blood cancers that was rejected by the FDA in 1993 and never reached the market.
CimaVax-EGF is a vaccine used to treat cancer, specifically non-small-cell lung carcinoma (NSCLC). CIMAvax-EGF is composed of recombinant human epidermal growth factor (EGF) conjugated to a protein carrier.
Ezetimibe/atorvastatin is a cholesterol lowering combination drug. In the United States, it was approved in May 2013 by the Food and Drug Administration for the treatment of elevated low-density lipoprotein (LDL) in patients with primary or mixed hyperlipidemia as adjunctive therapy to diet. Liptruzet has also been approved to reduce elevated total cholesterol and elevated LDL in patients diagnosed with homozygous familial hypercholesterolemia as an adjunctive treatment to other hyperlipidemia treatments.
4-Hydroxycyclophosphamide is in the class of oxazaphosphorine compounds, and is the main, active metabolite of cyclophosphamide and of mafosfamide after they partially metabolized by cytochrome P450. It is then partially tautomerized into aldophosphamide, which, in turn, easily enters live cells and then is partially detoxified into inactive carboxycyclophosphamide by the enzyme ALDH, but partially is hydrolyzed by another cell's enzyme phosphatase to the two directly cytotoxic metabolites - phosphoramide mustard and acrolein.
Sonidegib is a Hedgehog signaling pathway inhibitor being developed as an anticancer agent by Novartis.
GL-ONC1 is an investigational therapeutic product consisting of the clinical grade formulation of the laboratory strain GLV-1h68, an oncolytic virus developed by Genelux Corporation. GL-ONC1 is currently under evaluation in Phase I/II human clinical trials in the United States and Europe.
Triciribine is a cancer drug which was first synthesized in the 1970s and studied clinically in the 1980s and 1990s without success. Following the discovery in the early 2000s that the drug would be effective against tumours with hyperactivated Akt, it is now again under consideration in a variety of cancers. As PTX-200, the drug is currently in two early stage clinical trials in breast cancer and ovarian cancer being conducted by the small molecule drug development company Prescient Therapeutics.
Ribociclib is an inhibitor of cyclin D1/CDK4 and CDK6, and is used for the treatment of certain kinds of breast cancer. It is also being studied as a treatment for other drug-resistant cancers. It was developed by Novartis and Astex Pharmaceuticals.
