Maintaining rigorous drug development standards in personalized cancer treatment

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New advances in genomic testing have the potential to radically change the way cancer drugs are developed and prescribed, offering personalized therapy with treatment tailored to the patient’s tumor genome. With the advent of this “personalized medicine,” scientists are hopeful that drugs shown to work in a cancer caused by a specific genetic mutation that appears in one anatomical site – the breast, for example – may prove to be successful in treating a cancer caused by the same type of mutation that occurs in another anatomical site – the liver, for example, or the colon.

In a new Perspective piece in the New England Journal of Medicine, Harvard T.H. Chan School of Public Health Acting Dean David Hunter looks at the challenges of maintaining the rigorous current model of drug design — through randomized, controlled trials (RCTs) testing safety and efficacy — in this new paradigm.

The article appeared online August 19, 2015.

Hunter, who also is Vincent L. Gregory Professor in Cancer Prevention, notes that genomic sequence-informed drug design has been successful in several cases involving common cancer types and target tumor mutations. But each patient’s tumor has a spectrum of mutations, he writes, making it difficult to aggregate information from the hundreds or thousands of patients usually needed to get the kind of assessment of effectiveness and safety yielded by randomized controlled trials.