Introduction
malignancies, tumour
MALIGNANCIES, TUMOUR, SIDE EFFECT
Our understanding of tumour biology has grown exponentially and a large number of novel molecularly targeted agents and immunotherapy have entered the clinic. Many of these agents have different side effect profiles compared to cytotoxic chemotherapy and their development plan may differ, with an emphasis on understanding target impact. Phase I studies include first-in-human studies as well as studies that combine two or more experimental drugs for the first time and may include patients who have exhausted standard therapies. Their objectives are to evaluate safety, pharmacokinetic and pharmacodynamic properties of these agents; to establish an optimal dose for Phase 2 efficacy trials; to describe initial antitumour response; and to gain information about the effect of a targeted agent on its target [A typical inclusion criterion for Phase 1 studies is ‘life expectancy >3 months’, however, the overall survival (OS) of patients with advanced solid malignancies is often difficult to predict, more so in the era of targeted therapy and immunotherapy. Prognostic scores have been developed to identify the prognosis of patients in Phase I studies. The Royal Marsden Hospital (RMH) prognostic score (which incorporates serum albumin, lactate dehydrogenase (LDH) levels, and the number of metastatic sites) is commonly used [