The word “niche” implies a specialized environment. But to Fiona Doetsch, the stem cell niche is anything but. For brain stem cells, “the whole organism is the niche,” Doetsch told the audience at the third plenary session of the International Society for Stem Cell Research (ISSCR) annual meeting in Hamburg this week. It’s a surprising idea at first, given the brain’s protection from many circulating substances via a series of barriers, including the blood-brain barrier and the blood-cerebrospinal fluid barrier.
Additional early-stage research and drug discovery news in brief, from: Lisata Therapeutics, Ovid Therapeutics, Recce Pharmaceuticals, Virpax Pharmaceuticals.
Gasherbrum Bio Inc. has disclosed GLP-1 receptor agonists reported to be useful for the treatment of type 2 diabetes, dyslipidemia, hyperglycemia, hypertension, obesity, stroke, myocardial infarction and gout, among others.
Hangzhou Zhongmei Huadong Pharmaceutical Co. Ltd. has described proteolysis targeting chimera (PROTACs) compounds comprising an E3 ubiquitin ligase-binding moiety covalently linked to a mitogen-activated protein kinase kinase kinase kinase 1 (MAP4K1; HPK1; MEKKK1)-targeting moiety. They are reported to be useful for the treatment of cancer.
Sironax Ltd. has discovered (+) hydrolase SARM1 (SAMD2; MyD88-5) inhibitors reported to be useful for the treatment of amyotrophic lateral sclerosis, diabetic neuropathy, multiple sclerosis, Parkinson's disease, chemotherapy-induced peripheral neuropathy and traumatic brain injury.
Haisco Pharmaceutical Group Co. Ltd. has identified phosphatidylinositol 3-kinase α (PI3Kα) (H1047R mutant) inhibitors reported to be useful for the treatment of breast cancer.
Boehringer Ingelheim Pharma GmbH & Co KG has synthesized D-3-phosphoglycerate dehydrogenase (3-PGDH; PHGDH) inhibitors reported to be useful for the treatment of cancer, autoimmune disease, infections and inflammation.
Ependymoma is a brain cancer that occurs during childhood and is usually resistant to cytotoxic therapy. Molecular profiling has helped in better understanding the mechanisms behind the disease and has unveiled EPH receptor B2 (EPHB2) as a critical player in driving disease.
The use of artificial intelligence (AI) in drug discovery has shown promise in recent years with a growing number of new compounds moving forward in the pipeline.
Rgenta Therapeutics Inc. has received IND clearance by the FDA for RGT-61159, which is being developed for adenoid cystic carcinoma, colorectal cancer and other solid tumors, as well as acute myeloid leukemia.
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