BIOLOGY | MEDICINE How “exceptionalresponders ” are revolutionizing treatment for the deadly desease BY KAT MCGOWAN UST LIKE EVERY NEW drugthe oncologistsat environmental mputs. So sometimes a patient will be Memorial Sloan-Kettering Cancer Center test- cured by a drug that is useless for everyone else. In ed against bladder cancer in the last 20 years, the past, these spectacular reactions were written off this one didn’t seem to be doing any good. For- _ as outlier responses that defied explanation—medical ty-four people in the study were given everolimusina mysteries. Doctors just shrugged their shoulders and last-ditch attempt to slow down or stop their advanced thanked their lucky stars that even though the study cancer. When the researchers analyzed the data, they tanked, they did manage to help one person. could see that the drug wasn’t slowing or stopping But this time was different. Clinical oncologist tumor growth. Everolimus seemed to be another bust. David Solit, director of developmental therapeutics Then there was patient number 45. She joined the at Sloan-Kettering, saw a new opportunity to explain trial with advanced metastatic cancer. Tumors had what happened by sequencing the whole genome of invaded deep into her abdomen, clouding her CT scan the woman’s cancer. Just five years ago, decoding and with solid grey blotches. She was 73 yearsold. None of analyzing all 3 billion bases of the DNA from a tumor the standard bladder cancer drugs were working for would’ve been absurdly time-consuming and expen- her anymore; she had “failed treatment,” in the dismal sive. Now the sequencing takes as little as a few days. lingo of oncologists. She enrolled in the study only Poring over the outlier patient’s genetic code, Solit because she happened to be a patient at Sloan-Ketter- pinpointed two mutations that made her tumor sensi- ingin January 2010. In April 2010, her cancer was gone. tive to this drug. He found that one of her mutations This sort of happy sur