A strategic framework for novel drug development in multiple myeloma.

Although multiple myeloma remains incurable, recent advances in research have created a new paradigm for the development of novel agents for myeloma treatment. Biological insights are being translated rapidly from bench to bedside, with an increasing number of agents available at all stages of disease. Concomitantly, the drug development and approval process has become more challenging. Defining optimal clinical dosing schedules and therapeutic regimens is increasingly dependent on insights derived from genomic, proteomic and cell signaling studies. Because the maximum tolerated dose of a novel agent may not be the same as the optimal biological dose, designing phase I trials is more complex. Evaluation of new agents in patients who have relapsed is more challenging because the number of effective therapies has increased. Finally, choosing a ‘standard therapy’ control arm for randomised trials is more difficult because of the increased number of potential comparator regimens. In June 2005, the Multiple Myeloma Research Foundation (MMRF) sponsored a roundtable symposium in Washington, DC, to define the strategies and guidelines for phases I, II and III development of new myeloma agents, with the ultimate goal of speeding delivery of new therapies to myeloma patients. [ABSTRACT FROM AUTHOR]