The Future of Drug Development and Therapy in Myeloma

The treatment options and outcomes for patients with myeloma has dramatically improved over the past decade, due in large part to the availability of improved anti-myeloma treatments including high-dose therapy, thalidomide, bortezomib, and lenalidomide. Many of the currently active agents are effective because of their impact on normal plasma cell biology, suggesting that targeting the plasma cell, rather than malignant cell biology has led to more effective therapy. Additionally, the use of combination therapy, with agents that are synergistic when combined, has led to deeper responses, and these have likely also contributed to improvements not only in progression free but also overall survival. With the wealth of new agents coming into the myeloma space, it is incumbent upon us as investigators to utilize efficient study designs with novel statistical approaches in order to rapidly test and evaluate new drugs. These concepts as well as a few selective promising targets in early development will be reviewed in the current discussion.