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Lung cancer patients with a specific genetic abnormality may benefit from a new treatment drug that targets tumors, according to a study published in the New England Journal of Medicine (2010;363:1727).

Based on findings of past studies that genetic abnormalities that trigger uncontrolled cell growth underlie several types of cancer, a multi-institutional research team conducted a study to investigate the effect of crizotinib in patients whose tumors were driven by alterations in the anaplastic lymphoma kinase (ALK) gene.

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For the first part of the study, patients with any solid tumor that had not responded to standard therapies received increasing daily oral doses of crizotinib to establish the highest dose that would not cause intolerable side effects. With the data obtained, the research team was able to establish the maximum tolerated dose. When researchers found that two of the participants that had ALK-altered non-small cell lung cancer (NSCLC) showed symptom improvement, the study was expanded to include NSCLC with ALK-rearranged tumors.

During the second phase of the study, researchers reported that crizotinib shrank the tumors of more than half of those patients whose tumors were driven by alterations in the ALK gene, and in one-third of the study participants, crizotinib treatment suppressed tumor growth. Specifically, of the 82 patients with ALK-altered tumors who eventually enrolled in the trial, crizotinib treatment reduced tumor size in at least 47 patients and halted tumor growth in 27. And among those participants, 63 have continued receiving the drug. “This therapy is allowing patients to function without pain or a constant cough,” Eunice Kwak, MD, PhD, of the Massachusetts General Hospital Cancer Center and corresponding author of the study, explained. “The most rewarding thing about treating patients with this drug is watching them change from being completely controlled by their cancer to resuming a very normal life.” ■