Medical management of soft tissue sarcomas (STS) has been restricted by the limited availability of active drugs. A plethora of new oncologic agents are now available, many of which have specific therapeutic targets. Gemcitabine and docetaxel is a combination of drugs that have limited single-agent activity. Yondelis, a novel chemotherapeutic that binds DNA and functions partially by inhibiting transcription, is being tested alone and in combination with doxorubicin. Inhibitors of mTOR, a serine/threonine kinase that regulates cell cycle activation and cell growth, are also being tested. Growth factor receptor inhibitors are being evaluated in a variety of sarcomas that have been found to express the targets. In addition, a variety of agents are being assessed in gastrointestinal stromal tumors (GIST). Single agents and agents combined with imatinib are being tested in imatinib-refractory and in metastatic GIST. The increased use of targeted agents underscores the need for understanding sarcoma biology.
Margaret von Mehren
%. Overall survival at 12 months was 79%. Because of these findings, in 2007 the FDA approved nilotinib use in imatinib-refractory or -intolerant CML in chronic or accelerated phase. Nilotinib has also shown promise in blastic phase disease, 51 although this
George D. Demetri, Margaret von Mehren, Cristina R. Antonescu, Ronald P. DeMatteo, Kristen N. Ganjoo, Robert G. Maki, Peter W.T. Pisters, Chandrajit P. Raut, Richard F. Riedel, Scott Schuetze, Hema M. Sundar, Jonathan C. Trent and Jeffrey D. Wayne
supported by single case studies and unpublished data. Sunitinib has shown substantial antitumor activity and acceptable tolerability in a small series of imatinib-refractory pediatric patients. 193 TTP was longer on sunitinib than on prior imatinib