Endocrine/metabolic

Patient sample and cell culture studies suggest the BRAF V600E inhibitor Zelboraf vemurafenib could help treat Cushing disease, which involves excessive cortisol production triggered by ACTH-secreting pituitary adenomas. In 91 tumor samples from patients, 15 expressed the BRAF V600E mutation. In two human corticotroph adenoma cell lines expressing the mutation, Zelboraf decreased ACTH production compared with vehicle. Next steps could include testing Zelboraf in animal models of BRAF V600E-positive Cushing disease...