Discovery of Treatment for Rare Lung Disease Earns UC Researcher National Award

Michele Leatherbury, 66, says that a year ago, she needed eight to 10 liters of oxygen to exercise or clean her house.

“I was diagnosed with lymphangioleiomyomatosis (LAM) in 2002, after my pulmonary doctor in Dayton sent a lung biopsy to the Mayo Clinic,” she says. "A year ago, when I began needing more and more oxygen to complete normal tasks, I called Dr. (Frank) McCormack in Cincinnati. Though I had seen him off and on since my diagnosis, I had never needed so much oxygen.”

LAM is a rare lung disease mostly affecting women in their mid-30s and 40s, in which abnormal muscle-like cells invade the lungs, lymph nodes and kidneys. Over time, this process destroys the normal lung tissue, resulting in emphysema-like cystic changes and progressive respiratory failure.

Leatherbury scheduled an appointment with McCormack, MD, pulmonologist and researcher at the University of Cincinnati who specializes in interstitial lung diseases. He prescribed her an FDA-approved drug called sirolimus, used primarily to prevent rejection in organ transplant patients.

Now, Leatherbury says she only needs two to three liters of oxygen when exercising or cleaning house and needs none when at rest.

“I can do all of the things I couldn’t do before,” she says. “I can work out three times a week and walk the dog. It’s amazing what a difference this made in the quality of my life.”

But McCormack didn’t just prescribe Leatherbury sirolimus on a whim. It came after years of research and clinical trials now gaining McCormack and the entire LAM team at UC national recognition.

McCormack, B. Gordon & Helen Hughes Taylor Chair and professor in the division of pulmonary, critical care and sleep medicine at UC, was honored for one of the “Top 10” clinical research projects in the country and received a Clinical Research Forum Clinical Research Achievement Award at the forum’s annual meeting and awards dinner April 18, 2012, in Washington, D.C.

The award is given to projects that exemplify scientific innovation that results from U.S. investment in clinical research that can benefit human health and welfare.

The Clinical Research Forum is a nonprofit organization dedicated to providing national leadership in clinical research, with a mission to generate support for clinical research and promote understanding of its impact on health and health care delivery. Members are among the nation’s most prestigious academic medical centers and health systems.

McCormack, who has studied interstitial lung diseases for over 20 years, was nominated for his team’s landmark paper, “Efficacy and Safety of Sirolimus in Lymphangioleiomyomatosis,” published in the March 16, 2011, advance online issue of the New England Journal of Medicine.

The study, known as the MILES trial, found that the sirolimus stabilized lung function in women with LAM. This was the first randomized, controlled study designed to develop a therapy for this life-threatening disease.

"I am honored to accept this prestigious award on behalf of the entire MILES team and everyone at the UC Academic Health Center who made this trial possible,” says McCormack. “MILES translated the efforts of so many collaborators here into patient benefit; forty years of groundbreaking basic research in the LAM cellular pathway by George Thomas, the first discovery of LAM genetic defects with Teresa Smolarek and Anil Menon, the promising lung responses in an earlier sirolimus trial with John Bissler and David Franz and the critical infrastructure provided by the Rare Lung Disease Consortium created with Bruce Trapnell. 
 
“Sincere thanks to the 24 co-investigators and 175 clinical trial team members at 13 sites in Japan, Canada and the U.S. who participated in MILES, and to the National Institutes of Health (NIH) and the Food and Drug Administration (FDA) for their support of the trial.”

“It is truly an accomplishment for Dr. McCormack and for the Academic Health Center as a whole,” says Thomas Boat, MD, dean of UC’s College of Medicine and vice president for health affairs. “This work is an example of how academic medicine makes a difference. I’m proud to have such talent at this institution.”

Aside from McCormack, awardees of the honor are coming from Baylor College of Medicine, Duke University, The Ragon Institute of MGH, MIT and Harvard, among others.

The award winners’ studies, all published within the past two years, are the latest in notable health advances that were propelled by combined investment in basic science and clinical research. Collectively, the work was funded by a range of agencies including the NIH, the FDA and the Centers for Disease Control and Prevention.

In addition, McCormack’s study was funded in part by UC’s Center for Clinical and Translational Science and Training (CCTST), providing support for translational research—basic science that progresses to treatments for patients—and by the LAM Foundation, where he serves as scientific director. CCTST is the academic home of the university’s institutional Clinical and Translational Science Award (CTSA)—a $23 million NIH grant awarded to UC and its health care affiliates 2009.

McCormack is one of four Clinical Research Forum award winners who have received support through CTSA funding.