A compound targeting the protein mTOR has potent anti-cancer effects on mouse and human leukaemic cells, according to research online in Nature Medicine.

mTOR is known to contribute to cancer development. Existing drugs targeting this protein have not been very effective at inhibiting cancer cell growth, and have the unwanted side effect of interfering with the immune system.

David Fruman and colleagues show that a new type of mTOR inhibitor that directly blocks the active site of the enzyme overcomes these drawbacks.

This inhibitor suppressed the growth of human acute lymphoblastic leukaemia cells in cell culture and in mice and enhanced the anti-cancer effects of another drug, dasatinib (Sprycel), that is commonly used to treat this type of leukaemia, with little immunosuppression. This new class of mTOR inhibitors for leukaemia therapy shows promise but will now need to be tested in human clinical trials.


Article DOI: 10.1038/nm.2091