NIH Funds Investigation of Inhaled Lung Cancer Treatment

April 11, 2011 – (BRONX, NY) – Just as inhaling cigarette smoke can cause lung cancer, inhaling medication may treat it. Two researchers at Albert Einstein College of Medicine of Yeshiva University have received a five-year, $2.4 million grant from the National Institutes of Health to clinically develop an inhaled therapy for lung cancer – to be used before the cancer becomes malignant and spreads.

Roman Perez-Soler, M.D.The grant recipients are Roman Perez-Soler, M.D., professor of medicine and associate director of clinical research at Albert Einstein Cancer Center; and Yiyu Zou, Ph.D., associate professor of medicine. Dr. Perez-Soler is also chair of oncology at Montefiore Medical Center.

Lung cancer is the leading cause of cancer mortality among men and women in the U.S. Each year, more than 220,000 are diagnosed and about 150,000 people die from the disease. The average five-year survival rate is only 15 percent, largely because nearly 80 percent of cases are diagnosed after the cancer has spread from the lungs to other organs in the body. Drs. Perez-Soler and Zou will take advantage of the fact that, for many years, the disease is confined to the bronchial epithelium (surface cells of the airways), where it lingers and develops in a premalignant state.

The research project involves inhaling a drug called 5-azacytidine to target the bronchial epithelium. 5-azacytidine is a demethylating agent, meaning it strips off methyl groups that have bound to genes and rendered them inactive. Since removing methyl groups can reactivate genes that suppress tumors, drugs such as 5-azacytidine can potentially treat a number of different types of cancers.

The first two years of the federally-funded study will focus on a mouse model of premalignant lung cancer, to determine what dose is likely to be safe and effective in humans. In years three through five, researchers will conduct a Phase I “feasibility and proof of principle” study in which patients with advanced non-small cell lung cancer (the most common type), and who no longer respond to standard therapy, will inhale 5-azacytidine. Albert Einstein College of Medicine has filed a patent application related to this research, which is available for licensing to partners interested in further testing and developing this treatment.

The ultimate aim of the research is to intervene with inhaled 5-azacytidine at an earlier, precancerous stage, when vital genes have been silenced but cancer has not yet developed. Using the therapy in this way it might prevent lung cancer from developing in people with a history of cigarette smoking. According to the Centers for Disease Control and Prevention, there are about 46 million adult smokers in the U.S.

This research is funded by the National Institutes of Health and the National Cancer Institute.