Machine learning-based predictive modeling maximizes the efficacy of mTOR/p53 co-targeting therapy against AML

While mTOR inhibitors show limited clinical efficacy against AML, this study demonstrates that combining mTOR inhibition with TP53 activation enhances anti-leukemic effects. We also developed a gene scoring system (Rapa-11) predicting rapamycin sensitivity and showed that monocytic AMLs with wild-type TP53 and low Rapa-11 score are highly responsive to mTOR/TP53 co-targeting therapy.