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Experimental cancer drug effective against certain types of lung cancer [Updated]

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An experimental cancer drug called crizotinib is effective in reversing the course of certain types of lung cancer in early-stage trials, researchers reported Wednesday. Crizotinib blocks the action of a gene called anaplastic lymphoma kinase or ALK, that is present in an estimated 3% to 5% of lung cancer tumors, primarily those that occur among patients who do not smoke. Although that seems like a small percentage, lung cancer is the most common form of cancer in the United States, striking an estimated 222,000 Americans each year. That means that about 10,000 have the ALK mutatoin and could potentially benefit from the new drug.

“These are really exciting findings, and I don’t say that about many studies,” Dr. Len Lichtenfeld, chief medical officer of the American Cancer Society, told WebMD. “This really has the potential to be a game changer for a select group of patients with a disease that is very difficult to treat.”

The trial involved non-small-cell lung cancer (NSCLC), which accounts for about 85% of lung tumors. Nearly three-quarters of such tumors progress to metastatic disease before they are detected and the response to chemotherapy is very low, generally about 15%. The new drug thus promises to be a valuable addition to the oncologist’s armamentarium.

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A team headed by Dr. Eunice Kwak of the Massachusetts General Hospital Cancer Center in Boston administered the drug to 82 NSCLC patients who had been confirmed to have the ALK mutation. The drug reduced the size of the tumor in at least 47 of the patients and halted progression in an additional 27, the team reported in the New England Journal of Medicine. Among those patients, 63 have continued receiving the drug, some for as long as two years. “This therapy is allowing patients to function without pain or a constant cough,” Kwak said in a statement. “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.” The team has seen few side effects from the treatment, but some of the patients have developed resistance to the drug.

A Phase 3 trial of the drug has already begun in an effort to demonstrate its effectiveness against lung cancer. Information about enrollment in the trials is available at (877) 369-9753.


[Updated Friday, 10:45 a.m.: An earlier version of this article gave an incorrect telephone number regarding information about clinical trial enrollment. The correct number, (877) 369-9753, is now listed.


In Los Angeles, the trial is being conducted at UC Irvine and the phone number is (714) 456-6241. Pfizer Inc., which manufactures the drug and sponsored the trial, said it plans to submit data to the Food and Drug Administration next year to seek marketing approval. Meanwhile, the drug might prove useful in treating other forms of cancer as well. In an editorial accompanying the report in the New England Journal, researchers noted that some other forms of cancer involve the same mutation, including about 10% of neuroblastomas, a severe childhood tumor.

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