To overcome possible limitations and drawbacks of allosteric mTOR inhibitors, such as rapamycin and RAD001, novel molecules acting as competitive inhibitors of the mTOR ATP active site have been developed; one of these, PP242 strongly suppresses both mTORC1 and mTORC2-mediated activities [36] and exerted potent cytotoxicity against leukemia cells [37]. The gene discussed is MTOR; the disease is leukemia.