An emerging toolkit for targeted cancer therapies.

The investment in the “War on Cancer” initiated by the National Cancer Act of 1971 is paying off in terms of improved patient outcomes, with more than 65% of all cancer patients now surviving more than five years in developed countries (Howlader et al. 2011). The number of cancer survivors now exceeds 11 million in the United States alone, with one in 30 adults being a cancer survivor (Howlader et al. 2011). Due to the intensive research programs focused on cancer, our understanding of the processes leading to the initiation and progression of cancer has improved markedly, and this, in turn, has fueled the transition to rational approaches to therapy by targeting the effects of the underlying genomic events driving the pathophysiology of cancer rather than previously relatively non-specific and more toxic chemotherapy approaches. This progress has been hard won in terms of the number of patients who have contributed by participating in clinical trials and certainly has been much slower than hoped for. However, a rapidly emerging toolkit is creating a “perfect storm” of deep mechanistic understanding, relevant model systems, an array of targeted therapeutics, and technological advances allowing the characterization of the patient and tumor genome that are the topics of this special issue of Genome Research and that should herald an unprecedented rate of improvement in patient outcomes.