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Summary: An international panel of investigators has published a study of erlotinib (Tarceva) that shows the drug helps lung cancer patients live longer. These results were first presented at the 2004 meeting of the American Society of Clinical Oncology and led to the drug being approved for treatment of patients with lung cancer who were no longer being helped by chemotherapy. The study appears in the New England Journal of Medicine.

Why it's important: Lung cancer is the leading cause of cancer-related deaths in the US and is expected to kill around 172,000 Americans in 2005. Chemotherapy is the main treatment for patients with advanced disease. But once patients no longer respond to their first chemotherapy regimen they are less likely to respond to a second and rarely respond to a third. Erlotinib helps patients in this situation. Before this drug was developed, they had no other options.

What's already known: Erlotinib is a special form of treatment called targeted therapy. It targets and blocks a molecule, called a growth factor, in the cancer cells that may stimulate tumor growth. Another drug, gefitinib (Iressa), works in the same way and was introduced into practice before erlotinib. But it hasn't proved as effective as erlotinib and the FDA has limited its use.

How this study was done: Between 2001 and 2003, patients with advanced lung cancer who had failed chemotherapy were divided randomly into 2 groups. One group received erlotinib, which is given as a pill, and the other was given an inactive (placebo) pill. The patients were followed until death. By the close of the study in April 2004, 587 of the 731 original patients had died.

What was found: Patients given erlotinib lived, on average, 2 months longer than those on placebo. At the end of 1 year, 31% of the treated patients were alive compared with 22% of the placebo group. About 10% of treated patients experienced shrinkage of their tumors. Patients receiving erlotinib also felt better, reporting less pain, cough, and shortness of breath.

The drug seemed to work best in women, patients who had never smoked, Asians, and patients with a certain type of lung cancer called adenocarcinoma. Some patients who had none of these characteristics were also helped, but there were fewer of them. Although some patients developed diarrhea and rash from the drug, these did not seem to be major problems and only 5% of the patients stopped the drug because of toxic side effects.

The bottom line: Erlotinib is an effective drug for patients with advanced lung cancer who are not responding to chemotherapy. It can prolong their lives and reduce their symptoms. It causes some side effects, but these are usually tolerable. One problem not discussed in the article is that the drug is expensive and will cost at least $2,000 per month. As long as it is FDA approved, insurance plans that pay for drugs may cover it. Medicare does not yet pay for the drug, but patients may be able to get it at a reduced cost through the Medicare Replacement Drug Demonstration Project, which runs until December 31, 2005.

Citation: "Erlotinib in Previously Treated Non-Small-Cell Lung Cancer." Published in the New England Journal of Medicine (Vol. 353, No. 2:123-132). First author: Frances A. Shepherd of Princess Margaret Hospital and the University of Toronto.