Hematology

Mouse studies suggest inhibiting PI3K could help treat hereditary hemorrhagic telangiectasia (HTT) type 2. In two mouse models of HTT type 2, a pan-PI3K inhibitor tool compound decreased the number of arteriovenous shunts, vascular density and the number of vascular branch points in the retina - three vascular defects associated with the disease - compared with vehicle. In one of the models, the pan-PI3K inhibitor decreased the number of veins on the surface of the small intestine. Also in the model, the PI3K inhibitor pictilisib decreased the number of arteriovenous shunts, vascular density and the number of vascular branch points in the retina. Next steps could include testing PI3K inhibitors in additional models...