Researchers Receive $1 Million to Study Effects of Cancer Drug on Rare Lung Disorder

CINCINNATI—Researchers at the University of Cincinnati have received a $1 million award from the U.S. Department of Defense to study the effects of a cancer drug on certain patients with lymphangioleiomyomatosis (LAM).

LAM is a rare but serious lung disease that affects women, causing shortness of breath and recurrent lung collapses, or pneumothoraces. The disease is caused by atypical smooth muscle cells invading the lung, causing tissue destruction by creating holes or cysts. It can be fatal.

Frank McCormack, MD, a UC Health pulmonologist and principal investigator of the study, says although there is no cure for LAM, there are a number of promising candidate drugs available, many of which are approved by the Food and Drug Administration (FDA) for other indications.

"Letrozole, trade name Femara, is an oral non-steroidal aromatase inhibitor used for the treatment of hormonally responsive breast cancer after surgery,” says McCormack. "Aromatase inhibitors block the synthesis of estrogen, lowering the serum estrogen level and slowing the growth of cells that are driven by estrogen.

"Like breast cancer, LAM appears to be hormonally modulated. We want to see if letrozole can slow the growth rate of LAM cells in the lungs of patients, reducing the rate of decline in lung function.”

The national, multi-center study, known as the Trial of Aromatase Inhibition in Lymphangioleiomyomatosis (TRAIL), will take place over a two-year period.

Sixty participants, all of whom must be post-menopausal women who have been diagnosed with LAM, will be randomized to either receive letrozole treatment—2.5 milligrams/per day for 12 months—or a placebo. 

McCormack says researchers will be using the LAM Foundation Clinical Research Network (LFCRN) sites across the country to implement this study and gather data. Additional members of the team at UC include Lisa Young, MD, Brent Kinder, MD, and Tammy Roads, project manager.

"The primary objective of this trial is to find an effective treatment for LAM, but the benefits of TRAIL will extend beyond this rare disease,” says McCormack. "Studying LAM, a very simple and slow-moving form of cancer caused by a single gene mutation, can teach us a lot about diagnosis and treatment of more aggressive malignancies."

Volunteers, staff and resources provided by the Tuberous Sclerosis Alliance were also instrumental in the acquisition of this award. For more information on the mission of the alliance, visit www.tsalliance.org/.