Ken nearly died one night in 1999. He was forty-eight years old and had been treated repeatedly with blood transfusions for unexplained anemia, but never for a minute did he think he was close to death. He was working every day and enjoying life. Then came the night all hell broke loose. He awoke suddenly with terrible abdominal pain and went into shock. If fast-moving EMTs and a savvy surgeon at his community hospital had not realized that Ken required immediate abdominal surgery, he would not have survived the night.
The operation revealed a grapefruit-size tumor attached to his small intestine. Some of the tumor cells had grown into his bowel, leaving a huge hole in his intestine. That night, the contents of Ken’s gut had poured into his abdomen and caused terrible inflammation and shock. Worse, the tumor cells had scattered all over his belly. He was doomed to have multiple cancers grow in his abdomen.
A few weeks later, Ken and his wife, Peggy, learned he had a form of cancer that would result in an utterly unmanageable situation when the distributed tumor cells began to grow. He had no traditional treatment options, since the cance —gastrointestinal stromal tumor, most often called GIST—stubbornly resists radiation and chemotherapy. And its complete surgical removal was impossible. But Peggy did not give up hope. She went on the Internet and found that George Demetri, an oncologist at the Dana-Farber Cancer Institute in Boston, was just a few months away from starting a clinical trial with the first so-called smart drug treatment for tumors like Ken’s.
One of the leading physicians interested in GIST, Demetri had learned that a mutation in a single gene in one of the billions of cells in an otherwise healthy body causes the disease. The altered gene, called an oncogene, creates proteins that figuratively shout at cells to divide constantly— that is, to become cancerous. The oncologist also found out that the pharmaceutical company Novartis had developed a drug that should be able to halt the mutated gene’s actions by blocking the shouting protein’s function, thus killing the cancer. What’s more, the drug wasn’t supposed to seriously damage any other system in the body. When hundreds of GIST cells that had spread in Ken’s abdomen began to grow, Demetri started treating him with the drug. The tumors vanished.
Ken’s story illustrates the radically new era on the horizon of cancer therapy—using drugs that are “smart” in that they precisely block the defective genetic pathways that cause and promote cancer. These new compounds destroy cancer cells yet cause minimal damage to normal cells.
The revolution in cancer genetics is giving new hope to everyone, from clinicians and medical researchers to patients and their loved ones. Pharmaceutical companies are quickly developing smart drugs. A few medicines have already passed required testing phases and have entered general oncology clinics for broad use. Within two decades—perhaps even one—a high proportion of cancer patients will receive such treatment. We are already beating back many kinds of aggressive cancers with smart drugs, and we will defeat even more in the years to come.