The Hunt For Cures: Cancer

Frank McCormick was flying home from a meeting on cancer genetics when a wild idea popped into his head. What if you could make a virus that would infect--and kill--cancer cells but leave healthy cells intact? The next day, McCormick excitedly explained his notion to colleagues at Onyx Pharmaceuticals in Richmond, Calif., a biotech company he had founded earlier that year. Some of them were as enthusiastic as he was. Others told him he was crazy; such a treatment couldn't possibly work.

Nine years later, McCormick, now director of the Comprehensive Cancer Center at the University of California, San Francisco, has proof of his sanity in the form of Onyx-015, a virus that works exactly as he envisioned. Last year the company reported results of a clinical trial in which Onyx-015 injections, in combination with chemotherapy, melted away tumors in 8 out of 30 patients with recurrent, late-stage head and neck cancer. In another study, involving 27 patients whose cancer had metastasized to the liver (a condition that usually kills in 6 months), 11 were still alive nearly two years after being treated with high doses of Onyx-015.

McCormick's story is one of hundreds of similar tales coming out of laboratories and cancer wards around the world, as treatments that were little more than half-baked ideas a decade ago now enter the final stages of testing. Of some 350 new compounds and molecules being tested on cancer patients, more than half are based on innovative, sometimes bizarre-sounding ways of homing in on tumors. Hundreds more are in earlier stages of development, putting clinicians and drug companies in the novel position of having more promising cancer treatments waiting to be evaluated than they can possibly handle.

The new therapies are emerging from two extraordinary decades of intense basic research, a fantastic voyage that scientists have taken into the heart of the cancer cell. "The life and death of cells is being worked out, and the dozens and dozens of molecules in the body that participate in those pathways are now becoming targets for therapy," says Alan Houghton, a medical oncologist and immunologist at Memorial Sloan-Kettering Cancer Center in New York City.

That's welcome news to clinicians and patients alike. Traditional cancer treatments--chemotherapy and radiation--are therapeutic blunderbusses; they blast indiscriminately at all fast-growing cells, often destroying healthy tissue along with the tumors. By comparison, the new drugs are smart bombs; they cause minimal collateral damage and trigger relatively few side effects.

Many of the new therapies also happen to be incredibly potent. Last month, for example, pharmaceutical giant Novartis reported spectacular results in a clinical trial of Glivec, a drug that disables a uniquely aberrant protein produced inside cells of chronic myelogenous leukemia, which afflicts 4,400 new patients in the U.S. each year. In the drug's very first test, every patient went into remission. In the most recent results, 30% showed no sign of the chromosomal damage that marks the disease and appeared to have been cured. "This drug is amazing," says Richard Stone, an oncologist at the Dana-Farber Cancer Institute who has been testing Glivec (also known as an STI, for signal transduction inhibitor). "Even patients who are near death, at the end stage of this disease, are going into remission."