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Researchers optimistic about sea change in cancer treatment.

SAN FRANCISCO—Successful trials of a drug designed to counter the deadly genetic glitches responsible for two forms of cancer portend a coming wave of novel therapies that will transform the way cancer is treated, said researchers gathered here for the annual meeting of the American Society of Clinical Oncology (ASCO). The new therapies, the result of decades of basic research aimed at understanding the molecular biology of cancer, herald “the beginning of a sea change in how we practice cancer medicine,” said ASCO President Larry Norton, MD, of Memorial Sloan-Kettering Cancer Center, New York. Such molecularly targeted therapies are expected to be the pharmaceutical equivalent of a smart bomb, homing in on a defined target and inflicting far less of the collateral damage to noncancerous cells than results from conventional chemotherapy and radiation. But because many malignancies may be caused by an accumulation of gene mutations, and cancer cells often develop resistance to anticancer drugs, some experts predict that few, if any, of the new drugs ultimately will be used as a single agent. Rather, an anticancer cocktail of several drugs, each of which targets a gene involved in a patient’s cancer, may prove to be a relatively nontoxic way of keeping the disease in check, suggested John Mendelsohn, MD, president of M. D. Anderson Cancer Center, Houston. A new oral drug, imatinib mesylate (formerly called STI-571), has proven so effective in clinical trials involving patients with chronic myelogenous leukemia (CML) that the US Food and Drug Administration approved it last month in record time. Now, in two studies presented at the ASCO meeting, American and European researchers have revealed that the drug (known by the trade names Gleevec [in the United States] and Glivec [in Europe]) also shows remarkable promise against a type of solid tumor, the gastrointestinal stromal tumor (GIST).