From the Department of Molecular Pharmacology, St. Jude Children's Research Hospital, Memphis, TN
Authors disclosures of potential conflicts of interest are found at the end of this article.
Address reprint requests to Peter J. Houghton, PhD, Department of Molecular Pharmacology, St. Jude Children's Research Hospital, 332 N. Lauderdale St., Memphis, TN 2794; e-mail: peter.houghton{at}stjude.org
Overview: Although conventional multimodality therapy is curative for most patients diagnosed with nonmetastatic rhabdomyosarcoma (RMS), the survival rate in children with metastatic disease has not improved significantly with the use of intensified therapy. With an increased understanding of the molecular pathogenesis of RMS, it is now possible to consider rational ideas to augmenting current therapies with molecularly targeted approaches. Here we consider the rationale for combining inhibitors of the serine/threonine kinase mTOR (mammalian target of rapamycin) with antibodies that inhibit the type 1 insulin-like growth factor receptor (IGF-1R).