The biology behind mTOR inhibition in sarcoma

Xiaolin Wan, Lee J. Helman

Research output: Contribution to journalReview articlepeer-review

157 Scopus citations


Dysregulation of the mammalian target of rapamycin (mTOR) pathway has been found in many human tumors and implicated in the promotion of cancer cell growth and survival. Hence, the mTOR pathway is considered an important target for anticancer drug development. Currently, the mTOR inhibitor rapamycin and its derivatives CCI-779, RAD001, and AP23573 are being evaluated in cancer clinical trials. To date, clinical results have shown good tolerability of treatment with mTOR inhibitors in most reports and varying effectiveness of mTOR inhibitors in a variety of tumors in a subset of patients. For the targeted treatment of sarcomas, AP23573 has shown promising clinical efficacy and low toxicity profiles in patients. Further studies should define the optimal dose/schedule, patient selection, and combination strategies with other biological agents, especially those targeting signaling pathways crucial for cell survival.

Original languageEnglish (US)
Pages (from-to)1007-1018
Number of pages12
Issue number8
StatePublished - Aug 2007
Externally publishedYes


  • Clinical trial
  • Rapamycin
  • Sarcoma
  • mTOR

ASJC Scopus subject areas

  • Oncology
  • Cancer Research


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