Childhood CancerCURE Childhood Cancer

CURE Funds Clinical Trial to Improve Survival Rate for AML Subtype

By February 17, 2015 No Comments

CURE-sponsored researcher, Dr. Melinda Pauly, a member of the pediatric oncology team at the Aflac Cancer and Blood Disorders Center of Children’s Healthcare of Atlanta and Emory University, is focused on better treatments for patients with Acute Myeloid Leukemia (AML). Approximately one in five leukemia cases are classified as AML. Survival rates for this type of leukemia are significantly lower than other types of leukemia at only 60 to 70 percent.

“Sixty to 70 percent survival rates are unacceptable,” said Dr. Pauly.

“We need to do better,” she continued, which is why she is using CURE funds to research a novel drug, dasatinib, which has proven successful in fighting other types of cancer and has shown promise fighting AML.

Dr. Pauly and the Aflac Cancer Center will be partnering with nearly ten top-tier cancer research institutions across the United States, including Seattle’s Children’s, for a Phase I clinical trial using dasatinib and chemotherapy to treat children with a particular subset of AML called core binding factor (CBF). CBF accounts for nearly 20% of all AML cases and is frequently resistant to chemotherapy.

“Twenty to 25 percent of patients with core binding factor AML will experience relapse,” said Dr. Pauly.

“Dasatinib specifically targets the gene mutation found in those core binding AML patients who relapse. If this trial proves successful, we can move to large-scale use of dasatinib in patients during the initial round of chemotherapy–preventing relapse before it occurs, which will save countless lives,” said Dr. Pauly.

While Pauly and her team are testing dasatinib in patients in the United States, she will also be communicating and sharing data with a team of European researchers who will be conducting a similar study abroad.

“This parallel study will make for the best possible testing ground to prove dasatinib’s effectiveness in treating CBF AML patients” said Dr. Pauly.

Both trials are set to take place over the next two to three years, testing the drug in small sample populations, and then moving to larger Phase II trials and treatment of even more children.

Dr. Pauly, who wanted to be a doctor “as long as she could remember,” received her medical degree from the Medical College of Georgia, followed by a residency at Children’s Health System in Birmingham and a fellowship at Emory.

“I like the unique relationships you form with patients and families in pediatrics, as well as the complex biology of leukemia and lymphoma patients,” said Dr. Pauly.

“We have very high cure rates for leukemia,” she said. “But we’re not at 100 percent. And for every individual child, it HAS to be 100 percent.”