Glioblastoma is a particularly aggressive form of brain cancer characterized by rapid infiltration into surrounding brain tissue, especially in areas of increased neuronal activity. Neurons and nerve fibers have recently been identified as vital components of the tumor microenvironment that favor the initiation and progression of a variety of solid tumors, including gliomas. Researchers have gained new insights into the molecular mechanisms driving glioblastoma infiltration and identified subtypes of neurons that serve as the substrate for driving tumor progression.
Duchenne muscular dystrophy (DMD) is an X-linked genetic disorder affecting roughly 1 in 3,500 males. DMD is due to mutations in the dystrophin gene, which encodes for an exceptionally large 427 kD protein. DMD is characterized by repeated degeneration and regeneration of muscle fibers, but ultimately replacement of muscle with fibrotic and adipose tissue. Despite advances in gene therapy and improvements in quality of life, most patients still die by 30 years of age due to cardiopulmonary failure.
Multiple sclerosis (MS) is and remains “a problem in the immune system” Stephen Sawcer told BioWorld. As in other autoimmune diseases, a faulty immune system attacks otherwise healthy tissues that it should be leaving alone. In the case of MS, the tissue in question is oligodendrocytes. And a study published online in Nature on June 28, 2023, suggests that while MS’ beginnings are autoimmune, the path it takes in an individual patient is determined in part by how well the brain can cope with the autoimmune attack.
Astrazeneca AB has synthesized pyraolo-and triazolo-azinone compounds acting as receptor-interacting serine/threonine-protein kinase 1 (RIPK1; RIP-1) inhibitors reported to be useful for the treatment of neurological disorders.
Benitec Biopharma Inc. has received FDA clearance of its IND application for BB-301, its silence and replace gene therapy for the treatment of oculopharyngeal muscular dystrophy (OPMD)-related dysphagia.
Convelo Therapeutics Inc. and Genentech Inc. have disclosed 3-β-hydroxysteroid-δ(8),δ(7)-isomerase (EBP) inhibitors reported to be useful for the treatment of multiple sclerosis, encephalomyelitis, optic neuritis, schizophrenia, amyotrophic lateral sclerosis, Alzheimer’s disease, Parkinson’s disease and Huntington’s disease, among others.
Researchers at Centre National de la Recherche Scientifique, Université De Picardie Jules Verne and Université Paris-Est Créteil Val de Marne (UPEC) have identified lipid anionic oligosaccharides acting as microtubule-associated protein tau (PHF-tau; MAPT) aggregation inhibitors reported to be useful for the treatment of Alzheimer’s disease.
University of Western Australia has disclosed 3,4-methylenedioxymethamphetamine (MDMA) analogues acting as dopamine transporter (DAT) and/or norepinephrine transporter (NET) and/or serotonin transporter (SERT) modulators reported to be useful for the treatment of neurological and psychiatric disorders.