Breaking Cancer's Gene Code

Understanding the genetic underpinnings of cancer is a giant step toward personalized medicine.

Video: Cancer Treatments

Comprehensive genomic information of this kind will also be critical to evaluating new therapies. And here we have to humbly admit a gaping deficit: The gold standard of today, the randomized controlled clinical trial, assumes that randomly chosen test subjects have identical tumors, based on the old thinking. That just doesn't work for cancer anymore. If in fact those tumors are markedly different from one another, little wonder that—as Gould reminds us—a drug that extends life a mere two months on average fails many people but gives precious years of life to some. Better answers would come from smaller patient studies comparing genetically matched cancers. Such trials would be faster, cheaper, and vastly improved for all patients—both for those who should, and should not, partake in the new therapy.

Next steps. The path to cancer 2040 demands coordinated and cooperative efforts by researchers spurred on by goals and timetables not unlike the ones that made the mapping of the human genome so famously successful. Among the next big challenges: Study and harness the cell's natural instincts to self-destruct when it senses deranged DNA. Learn the wily ways of cancer stem cells, which in small but powerful numbers seem to initiate tumors and just may be the brains behind cancer's perpetual growth. Find the way to consistently manipulate the immune system to attack and destroy even disseminated cancers, as it has done on rare occasions to bring nearly miraculous cures. And finally, enlist engineers and computer scientists to embrace the grand challenge of personalized medicine, as recently proposed by the National Academy of Engineering. What's needed, for example, are tools to readily store, analyze, and use massive amounts of genomic data, and gene chips and sensors to find and track cancers early, anywhere in the body.

As the following story makes clear, the possibilities of discovery are rich and at hand. And the urgency is evident in the faces of patients who yearn for better. It will take flexibility, organization, and heart to get there, but it can happen—perhaps long before 2040.