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. Read More
A single low-dose injection with anti-DLL4 in a nonhuman primate model of acute graft-vs.-host disease (aGVHD) dramatically improved post-transplant survival, providing durable protection from otherwise lethal gastrointestinal GVHD, researchers reported in the June 28, 2023, issue of Science Translational Medicine. Blocking DLL4 specifically increased the migration of beneficial regulatory T cells into the intestines, with concomitant reduction in effector T cells, which are the main culprits in aGVHD. Ultimately, these activities effectively provided protection against T-cell-mediated damage in a nonhuman macaque primate model. Read More
Researchers from Shenyang Pharmaceutical University and affiliated organizations presented the discovery of novel tubulin polymerization inhibitors as potential anticancer drug candidates. Read More
Blue Water Biotech Inc. has released preliminary preclinical data supporting the use of its norovirus shell and protrusion (S&P) virus-like particle (VLP) platform to develop a novel mpox vaccine candidate. Read More
Researchers at Jiangsu Hansoh Pharmaceutical Group Co. Ltd. and Shanghai Hansoh Biomedical Co. Ltd. have disclosed phosphatidylinositol 3-kinase (PI3K) inhibitors reported to be useful for the treatment of cancer and more. Read More
CD122 signaling plays a key role in the function of CD4, CD8 T cells and natural killer (NK) cells, as well as in the development and survival of tissue-resident memory T cells (TRMs). Read More
Congenital hyperinsulinism is an inherited condition affecting the pancreatic β cells, leading to overproduction of insulin and persistent low blood sugar levels. The most severe type of hyperinsulinism arises from mutations in ABCC8 or KCNJ11 genes, encoding the subunits of two ATP-sensitive potassium channels found in β cells. Diazoxide is the only FDA-approved treatment for hyperinsulinism, but patients with these mutations are unresponsive to this drug and need a pancreatectomy. Therefore, novel therapies for severe hyperinsulinism are urgently needed. Read More
Boehringer Ingelheim Pharma GmbH & Co. KG and Vanderbilt University have synthesized annulated 2-amino-3-cyano thiophenes and derivatives acting as KRAS GTPase and its mutant inhibitors reported to be useful for the treatment of cancer. Read More
Centro Nacional de Investigaciones Oncológicas Carlos III (CNIO) has identified Pim kinase inhibitors reported to be useful for the treatment of cancer. Read More
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. Read More
Sortina Pharma AB has divulged sortilin (NT3; Gp95) antagonists reported to be useful for the treatment of cancer, Alzheimer's disease and Parkinson's disease. Read More
Exevir Bio BV has been awarded a 2-year R&D grant of €1.6 million by Flanders Innovation & Entrepreneurship (VLAIO) for preclinical research towards prophylactic and therapeutic solutions for dengue. Read More
The FDA has awarded orphan drug designation to HL-001, a novel lysophosphatidic acid receptor-1 (LPA1) antagonist being developed for idiopathic pulmonary fibrosis (IPF) by Ube Corp. and Hilung Inc. Read More
Scripps Research Institute has described nucleoside analogues acting as prodrugs reported to be useful for the treatment of SARS-CoV-2 infection (COVID-19). Read More
Additional early-stage research and drug discovery news in brief, from: Aim Immunotech, Blue Earth Therapeutics, Chemomab Therapeutics, Edesa Biotech. Read More
