RAD001 in Relapsed or Refractory AML, ALL, CML in Blastic-Phase, Agnogenic Myeloid Metaplasia, CLL, T-Cell Leukemia, or Mantle Cell Lymphoma
Key claim: A phase I/II clinical trial is evaluating RAD001 (everolimus, an mTOR inhibitor) to determine the highest safe and effective dose in relapsed/refractory hematologic malignancies including myelofibrosis (agnogenic myeloid metaplasia).
Abstract
The goal of this clinical research study is to find the highest safe dose of RAD001 that can be given as a treatment for leukemia, mantle cell lymphoma, or myelofibrosis. Another goal is to learn how effective the dose that is found is as a treatment.
Why this matters
mTOR inhibition has been a relatively under-explored axis in MPN compared with JAK, BET, and XPO1 strategies, and this trial extends a first-in-class mTOR inhibitor into myelofibrosis (agnogenic myeloid metaplasia) and blast-phase contexts simultaneously. For PMF the finding adds another pathway-targeted backbone candidate for the JAKi-suboptimal subpopulation, and for blast-phase MPN it situates mTOR inhibition alongside HMA-, BCL-2-, and IDH-directed salvage approaches in a uniformly dismal-outcome setting.