Cancer Drug (Alpelisib), a PIK3CA-Inhibitor, Shows Promise in Treating Infants with Rare Disease (PROS) That Causes Tissue Overgrowth

PIK3CA-related overgrowth spectrum (PROS) is a group of rare, incurable disorders caused by mutations in the PIK3CA gene that result in the malformation and overgrowth of various parts of the body. A new report published online on January 26, 2020 in the Journal of Experimental Medicine (JEM) describes the successful treatment of two young infants with PROS using the breast cancer drug alpelisib. The open-access article Is titled “Treatment of Two Infants with PIK3CA-Related Overgrowth Spectrum by Alpelisib.”