Esophageal carcinoma (EC) is a tumor type of the digestive tract that accounts for about 300,000 deaths worldwide annually, with high aggressiveness and poor prognosis. Chinese researchers have investigated the implication of the enzyme aldehyde dehydrogenase 18 family member A1 (ALDH18A1) in EC.
Sorafenib is a widely used treatment for hepatocellular carcinoma that has advanced beyond surgical resection, but only 30% of patients respond and many develop resistance within half a year. The resistance has been linked to showing greater ‘stemness’ characteristics of tumor cells, such as activation of signaling pathways mediated by Wnt/b-catenin, Hedgehog and PI3K-Akt.
Researchers at Wenzhou Medical University reported the synthesis and preclinical evaluation of a novel series of betulin-succinate derivatives developed as potential anticancer agents. Betulin is a naturally occurring triterpene that shows significant potential as a lead compound for the development of novel antitumor agents, owing to its selective cytotoxicity against cancer cells and low toxicity toward normal cells.
Mutations in the GTPase KRAS drive proliferation of many types of tumors, and daraxonrasib (RMC-6236), which inhibits various KRAS mutants, can slow growth of pancreatic and lung cancers. Researchers at City University of New York and Weill Cornell Medical College have demonstrated that it also showed effects in osteosarcoma models expressing mutated KRAS.
Opus Genetics Inc. has obtained IND clearance from the U.S. FDA application for OPGx-BEST1, a gene therapy for the treatment of bestrophin-1 (BEST1)-related inherited retinal disease (IRD).
Researchers from China Pharmaceutical University and collaborators have identified various naphthalene and indane derivatives as selective USP7 inhibitors.
Hangzhou Qihan Biotech Co. Ltd. has gained IND approval by the FDA for QT-019B, a universal, dual-target CAR T-cell therapy for refractory systemic lupus erythematosus (SLE). A phase I/II trial will be conducted in the U.S.
One of the major obstacles to treating neurological disorders such as Alzheimer’s disease is the blood-brain barrier: drugs that are injected into the circulation usually do not enter the brain effectively. Researchers at Denali Therapeutics Inc., Biogen Inc. and the University of Minnesota have devised a vehicle for transporting antibodies against amyloid-β that can bypass the blood-brain barrier by binding to the transferrin receptor, which is expressed much more abundantly in capillaries than in arteries.
German researchers have cracked the decades-long mystery of why males are more susceptible to acute kidney injury than females, demonstrating that estrogen has a protective effect in females.
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