MacDonald leads first-in-pediatrics brain tumor trial
Tobey MacDonald, M.D., Director of the Aflac Cancer and Blood Disorders Center’s Neuro-Oncology Program, helped to initiate the “first in child” clinical investigation of Indoximod (immunotherapy targeting the IDO immune checkpoint pathway) for the treatment of children with all types of refractory brain tumors. He is also one of 8 members on the NIH Brain Malignancies Steering Committee and a member of the FDA Pediatric Oncologic Drug Advisory Committee, which work together to help to shape the future direction of national clinical trials for pediatric brain tumors. Dr. MacDonald was recognized by U.S. News & World Report in 2016 as a top pediatric oncologist in the country.
Learn more about Tobey MacDonald, M.D.
Qayed leads trial using mesenchymal stromal cells from bone marrow
Bone marrow transplantation is performed in some patients with cancers of the blood or bone marrow, as well as in some patients with sickle cell disease, thalassemia, aplastic anemia and other disorders of the immune system. Graft-versus-host-disease (GVHD) is life-threatening complication of bone marrow transplantation in which donor immune lymphocytes attack the organs of the bone marrow transplant recipient. Symptoms of GVHD disease include severe abdominal pain, diarrhea, fever, weight loss, skin rash and liver damage. Additionally, chronic GVHD can affect the joints and lungs, among other organs. Available therapies designed to suppress GVHD do not work in everyone. For patients with GVHD who do not respond to first-line therapy, there is no reliable cure, and GVHD can be life-threatening or a life-long disabling condition. The frequency of GVHD after bone marrow transplantation is high, highlighting the need for new therapies.
The current clinical trial uses mesenchymal stromal cells from the bone marrow. These cells have been studied more recently for treatment of a wide array of diseases, including autoimmune diseases. They have appealing properties that help with modulating the immune system and promoting wound healing. In most other studies, the cells are obtained from a donor (not the patient), they are frozen after preparation and are infused immediately after thawing. The personalized cells in this trial are derived from the patient's own bone marrow rather than using another person's, making the product more likely to be effective. Physician-researchers at the Aflac Cancer and Blood Disorders Center at Children's Healthcare of Atlanta and Winship Cancer Institute of Emory University harvest bone marrow cells from children and adults (12 to 65 years) with GVHD.
The team manufactures these cells without using animal products, in the Emory Personalized Immunotherapy Center (EPIC), a dedicated pharmaceutical grade facility located within Emory University Hospital, and the cells are delivered fresh and living to the patient. By infusing large doses of these personalized bone marrow cells into bone marrow transplant recipients, the physician-researchers aim to target sites of inflammation, potentially reducing GVHD in the intestine, liver and skin and limiting long-term organ damage.
Muna Qayed, M.D., MSc. is a Pediatric Hematologist/Oncologist at the Aflac Cancer and Blood Disorders Center and an assistant professor at Emory School of Medicine. Dr. Qayed leads the clinical trial, which is offered only in Atlanta and is supported by CURE Childhood Cancer.
Learn more about Emory Personalized Immunotherapy Center (EPIC)
Learn more about CURE Childhood Cancer