Targeted Therapies: The Rare Cancer Paradigm

Marco A. Pierotti, Tiziana Negri, Elena Tamborini, Federica Perrone, Sabrina Pricl, Silvana Pilotti

Research output: Contribution to journalArticlepeer-review


This review analyzes the state of the art of targeted therapies for several tumors, starting from the paradigmatic example of Imatinib treatment in chronic myelogenous leukemia (CML). We discuss how rare tumors can be models for various mechanisms of receptor tyrosine kinase (RTK) activation, and provide the opportunity to develop new therapies also for more common cancer types. We discuss the activation of the downstream RTK effectors as further targets for therapies in colorectal cancer. Finally, we highlight how a novel multidimensional approach which adds an in silico dimension to the in vitro and in vivo approach, can predict clinical results.

Original languageEnglish
Pages (from-to)19-37
Number of pages19
JournalMolecular Oncology
Issue number1
Publication statusPublished - Feb 2010


  • Activation of downstream RTK effectors
  • Chronic myelogenous leukemia
  • Imatinib treatment
  • Targeted therapies

ASJC Scopus subject areas

  • Cancer Research
  • Genetics
  • Molecular Medicine


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