ABSTRACT
This chapter highlights the strengths and limitations of the clinical trial strategies used to develop targeted cancer therapies to date and to provide alternatives strategies for future research endeavors. For cytotoxic drugs, the recommended dose corresponds to the highest dose associated with an acceptable level of toxicity and is derived from clinical data and preclinical dose–toxicity and dose–activity studies. Traditional cytotoxics induce tumor regression, which is not typically seen in the absence of drug-induced antitumor effects. Assessments of functional measures of tumor metabolism, blood flow, and other parameters are now possible with positron emission tomography (PET), magnetic resonance imaging, and computer tomography. Traditionally, cancer agents have been evaluated for their activity in patients populations that have been selected based on histology and tumor stage. Basic, translational, and clinical research efforts have all played significant roles in the development of targeted agents for the treatment of cancer patients.
