First-of-its-Kind Phase 2 Study Shows Oral Therapy is Viable Treatment Option

This is the first study to look at using the oral agent lenalidomide in elderly AML patients with a specific genetic abnormality, and the results show promise for using a new combination of treatments for the disease in the future.

Lenalidomide is a targeted agent used to treat lower risk MDS patients with a deletion on the fifth chromosome [del(5q)].  Researchers hypothesized that the drug would also work in the subset of older AML patients with the same genetic abnormality. Older AML patients with a chromosome 5q deletion have a response rate of less than 25 percent with conventional chemotherapy.

A nationwide, multi-institutional research team, led by Mikkael Sekeres, MD, Director of the Leukemia Program at Cleveland Clinic’s Taussig Cancer Institute, tested the safety and effectiveness of using lenalidomide in 37 previously untreated older AML patients who declined standard chemotherapy. Patients were treated with 50 milligrams of lenalidomide daily for 28 days followed by 10 milligrams daily for 21 days until their disease progressed or they experienced toxicity. Five patients (14%) had a partial or complete response to the drug, and relapse-free survival was an average of five months.

“We feel like we’ve connected the dots in many ways with this study, realizing that, even though MDS and AML are quite different in disease severity, certain MDS and AML cases involve the same genetic abnormality, and therefore, they may respond similarly to medication that targets that abnormality,” said Dr. Sekeres. “Our research also demonstrates that studies with oral medications can be successfully executed in high-risk populations. We designed this study specifically for the elderly with AML to find a personalized approach to treating their leukemia.”

Researchers also point out that lenalidomide, which is taken orally, achieved more than half of the response rate that is typically seen with the current regimen for AML of IV chemotherapy, which can only be delivered to hospitalized patients.

As a result of this study, research is now underway to look at the effectiveness of a combination therapy, involving IV chemotherapy with a targeted oral agent, for AML patients. Cleveland Clinic is collaborating with H. Lee Moffitt Cancer Center in Tampa, Fla., on this study, and both sites are actively enrolling patients. Visit the Taussig Cancer Institute to find out more information about this trial.

The study was supported in part by funding from the National Cancer Institute, as part of the U.S. cancer cooperative groups consortium.

About Cleveland Clinic

Celebrating its 90th anniversary, Cleveland Clinic is a nonprofit multispecialty academic medical center that integrates clinical and hospital care with research and education. It was founded in 1921 by four renowned physicians with a vision of providing outstanding patient care based upon the principles of cooperation, compassion and innovation. Cleveland Clinic has pioneered many medical breakthroughs, including coronary artery bypass surgery and the first face transplant in the United States. U.S.News & World Report consistently names Cleveland Clinic as one of the nation’s best hospitals in its annual “America’s Best Hospitals” survey. About 2,800 full-time salaried physicians and researchers and 11,000 nurses represent 120 medical specialties and subspecialties. Cleveland Clinic Health System includes a main campus near downtown Cleveland, nine community hospitals and 15 Family Health Centers in Northeast Ohio, Cleveland Clinic Florida, the Lou Ruvo Center for Brain Health in Las Vegas, Cleveland Clinic Canada, and opening in 2013, Cleveland Clinic Abu Dhabi. In 2010, there were 4 million visits throughout the Cleveland Clinic health system and 155,000 hospital admissions. Patients came for treatment from every state and from more than 100 countries. Visit us at Follow us at

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Categories Cancers