The Personalization of Medicine

Targeted therapeutic regimes offer hope to those facing life-threatening diseases.

Amylynne Santiago Volker escorts her son, Nic Santiago Volker, into surgery at Children's Hospital of Wisconsin in Milwaukee on June 20, 2013. Her son developed a crippling disease as a toddler that ravaged his digestive tract.

Amylynne Santiago Volker escorts her son, Nic Santiago Volker, into surgery at Children's Hospital of Wisconsin in Milwaukee on June 20, 2013. Her son developed a crippling disease as a toddler that ravaged his digestive tract.

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Betty Lane didn't have a whole lot of options left. Diagnosed with stage 3b lung cancer in the summer of 2011, she underwent surgery and chemotherapy, which drove away her disease for about six months. But then it returned, this time in her liver. The Fayetteville, Tenn., psychotherapist enrolled in a drug trial, but her cancer didn't respond.

Then Lane's doctor at Vanderbilt-Ingram Cancer Center in Nashville suggested that they try a drug used to treat breast cancer. Lane's lung tumor had revealed a glitch in a gene more commonly abnormal in breast and gastric tumors, resulting in a mutant form of a protein targeted by the drug Herceptin. "I feel very fortunate," says Lane, 55, whose cancer is now shrinking, "to live in an age and a time when this is happening."

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Lane is a prime example of the promise of personalized medicine, which matches treatments to patients based on their genetic and other biological information. "There are now over 80 personalized treatments available," says Edward Abrahams, president of the Personalized Medicine Coalition, an education and advocacy organization. Herceptin and Gleevec, approved in 2001 to treat a certain type of leukemia, are two of the oldest. Just this past May, the Food and Drug Administration approved two drugs for advanced melanoma driven by certain mutations that join a crop of new therapies approved in the last few years. Also, in May, the Mayo Clinic launched an Individualized Medicine Clinic at its three locations in Minnesota, Arizona and Florida aimed at getting cutting-edge guidance to cancer patients who have failed standard treatments and to people with mysterious ailments that may have a genetic cause.

There are obstacles along this road, to be sure. While the National Institutes of Health says the cost of sequencing a genome is now about $5,700, compared to $95,000 a dozen years ago, all that information has to be analyzed and interpreted, and somehow made accessible to physicians and patients. And identifying the precise cause of a disease is no guarantee that there's a treatment. If there is, it's apt to be very expensive, since the cost of drug development must be spread out over a smaller number of patients.

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Still, the beachheads are being established, most notably in cancer. Vanderbilt-Ingram, Massachusetts General Hospital, Memorial Sloan-Kettering Cancer Center in New York and MD Anderson in Houston all now regularly test patients for certain mutations. At Vanderbilt-Ingram, all lung-cancer patients are tested for more than 40 glitches in 10 different genes, and melanoma patients are screened to see if they have one of 43 mutations in six genes, says William Pao, director of the hospital's division of hematology and oncology. Vanderbilt also tests colon and breast tumors to see if patients are eligible for trials of experimental drugs targeting particular mutations.

Researchers at Washington University School of Medicine in St. Louis took more drastic measures in 2011 to help their colleague Lukas Wartman. The Wash U oncologist and leukemia researcher was fighting leukemia himself, and his prognosis after a second relapse was grim. Rather than just screen his cancer for the small number of known genetic changes that occur with the disease, the researchers sequenced the entire genome of the cancer's DNA as well as that of Wartman's own normal cells, looking for any changes that could be driving the cancer. They also sequenced the RNA from his cancerous cells.

What they found, says Wartman, was that a certain gene called FLT3 was unexpectedly overproduced by his cancer cells. It so happens that a drug approved for kidney cancer inhibits that gene, and within days, Wartman started taking it. The drug sent his leukemia into remission, though he eventually had to call a halt because it also lowered his blood cell counts, a side effect. Now "I'm in uncharted waters," he says, adding that he remains in remission and is weighing the risks and benefits of taking the drug again.


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