Accelerating safe drug development: an ideal approach to approval

Although enormous progress in therapeutic research has improved the lives of patients with hematologic malignancies, these earlier achievements resulted from strategic combinations of agents with unique mechanisms of action and nonoverlapping toxicities. Continued investment in the modern era of drug discovery and development will focus on targeted therapies. Targeting of specific molecular pathways is expected to achieve effective tumor cell reduction with less overall toxicity. The translational processes involved in moving novel therapeutic strategies from the laboratory toward the clinic require close monitoring. The efforts in both cancer drug discovery and development will require extensive collaboration among basic scientists, clinical investigators, and regulatory scientists. The transition from older methods of therapeutic research will require laboratory support to define eligible patients based upon their pretreatment profile. The principles of preclinical drug development based upon decades of experience in predicting toxicity and designing therapeutic strategies are still needed to insure that safety is a high priority. The opportunities for developing novel targeted combination therapies in uniquely profiled patients will hopefully enable successful breakthroughs. Several concrete examples of exciting new agents are discussed here. Defining the predicted mechanism of resistance to these new targeted agents will enable investigators to subsequently design strategies to circumvent resistance with effective combinations. Drug discovery and development are complex and expensive, so efficiency and cooperation in task completion must be tracked.