Children’s Healthcare of Atlanta Opens First Clinical Trial For New Therapy Targeting Leukemia

Staff Report

Friday, April 14th, 2023

Pre-clinical findings show that the oral drug MRX-2843 inhibits the growth of leukemia cells in relapsed acute myeloid leukemia (AML) and acute lymphoblastic leukemia (ALL). Now, a phase 1 clinical trial will begin in adolescents at Children’s Healthcare of Atlanta to determine the maximum tolerated dose of MRX-2843 and establish its safety profile, including common side effects, in 45 patients. Melinda G. Pauly, MD, pediatric hematologist and oncologist, will lead the trial at the Aflac Cancer and Blood Disorders Center.

During three decades of research, Douglas K. Graham, MD, PhD, Chief of the Aflac Cancer and Blood Disorders Center, discovered the MerTK gene and has conducted research to validate it as a target for cancer therapy. His research team has shown that MerTK is turned on at high levels in leukemia cells, compared to normal blood cells, leading to the growth and proliferation of the cancer cells. In collaboration with Dr. Deborah DeRyckere at Emory University and scientists from the University of North Carolina (UNC) at Chapel Hill, Dr. Graham developed the new oral therapy, MRX-2843, to specifically target these leukemia cells and stop their growth. His preclinical work showed that blocking MerTK with MRX-2843 effectively kills leukemia cells, especially in combination with chemotherapy. Pre-clinical findings have also shown the drug can activate the immune system to combat the leukemia cells.

“From first discovery through all the different steps of drug development, every physician involved in research has an ultimate dream their work will have a positive impact on patients and improve outcomes,” said Dr. Graham. “When you work in team science, you pass the baton on to the next person. For this next step, we’re passing it over to Dr. Pauly who is extremely well qualified to run the clinical trial in adolescents with leukemia. Our next step will be to extend this therapy to all ages of children who have leukemia that is not responding to standard treatment protocols.”

This is the first cancer trial at Children’s and Emory to open for adults and children at the same time. William G. Blum, MD, Director of the Acute Leukemia Program at the Emory Winship Cancer Institute, will lead the trial for adults. The trial will also be conducted in adults and children at Memorial Sloan Kettering Cancer Center and UNC-Chapel Hill.

“Historically new therapies are tried in adults first, but this will give children with relapsed and resistant acute AML and ALL the opportunity to access sooner, hopefully improving their quality of life with an oral medicine that can be taken at home,” said Dr. Pauly, Medical Director at the Aflac Cancer and Blood Disorders Center.

Leukemia is a cancer of the white blood cells. When a child has leukemia, his or her bone marrow produces many abnormal white blood cells that can no longer fight disease causing poor immunity. They also interfere with the child’s production of red blood cells and platelets, which can cause anemia and bleeding problems. ALL is a fast-growing cancer of lymphocyte-forming cells called lymphoblasts, while AML is a fast-growing cancer that starts in immature bone marrow cells. The Aflac Cancer and Blood Disorders Center diagnoses about 150 new cases of leukemia each year.

Dr. Pauly hopes MRX-2843 will benefit kids like her former patient, Anna Charles Hollis, or “AC” to family and friends, the daughter of Peach Bowl, Inc. executive Benji Hollis. Anna Charles’ battle with leukemia became the Peach Bowl’s fight and inspired the $20 million commitment to the Aflac Cancer and Blood Disorders Center to establish the Peach Bowl LegACy Fund, named for Anna Charles. This fund, along with Swim Across America, will support the MRX-2843 trial.

The Peach Bowl LegACy Fund was created in 2019 with a $20 million gift from Peach Bowl, Inc. to be hyper focused on funding the most promising clinical drug trials for children fighting cancer. The goal of the Peach Bowl LegACy Fund is to ensure that high-priority novel agents, devices and treatment strategies can be tested in patients at an accelerated pace, eventually leading to additional treatment options for patients.

“This is a monumentally important milestone not just for the incredible doctors behind this great work, but for the hope of children and parents in the fight with leukemia,” said Gary Stokan, Peach Bowl, Inc. CEO and president. “This trial is the kind of progress we hoped we could impact when we created the Peach Bowl LegACy Fund.”

“That connection with the Peach Bowl has been really personally touching and their support of phase 1 medicine has been overwhelming,” said Dr. Pauly. “I am very honored to be leading a trial that is being sponsored by the Peach Bowl LegACy Fund.”