Tailored Cancer Therapy

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Science  31 Aug 2001:
Vol. 293, Issue 5535, pp. 1559-1561
DOI: 10.1126/science.293.5535.1559f

Prompted by the discovery that tumors derived from a given tissue type can have distinct molecular features, it may now be possible to develop “tailored” cancer therapy. The basic idea is to classify tumors according to the specific growth signaling pathways that are aberrantly activated in the tumor cells and to apply therapies that selectively target those pathways.

The potential benefit of tailored cancer therapy is illustrated by the preclinical work of Neshat et al. and Podsypanina et al., who studied tumors genetically deficient in PTEN phosphatase, the product of a tumor suppressor gene that is commonly mutated in human endometrial, prostate, and brain cancers. Noting that PTEN-deficient tumors show elevated activity of the mTOR/S6 kinase signaling pathway, the authors tested the anticancer activity of a drug (CCI-779) known to inhibit this pathway. Treatment with the drug retarded the growth of PTEN-deficient tumor cells in culture and PTEN-deficient tumors in mice, but was much less effective against tumors showing normal expression of PTEN. Thus, PTEN-deficient human tumors may be especially sensitive to the antiproliferative effects of CCI-779 and the related drug rapamycin, which is already in clinical use as an immunosuppressant. — PAK

Proc. Natl. Acad. Sci. U.S.A.10.1073/pnas171076798; 10.1073/pnas.171060098.

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