Clinical trial uses patients' own cells for treatment after bone marrow transplant

Posted: April 2, 2015 at 10:48 pm

Children's Healthcare of Atlanta and Winship Cancer Institute target graft-versus-host-disease through immune cell therapy

An innovative clinical trial using the science of "personalized" cellular therapy has begun enrolling children and adults suffering from graft-versus-host-disease (GVHD), a life-threatening complication of bone marrow transplantation in which donor immune lymphocytes attack the organs of the bone marrow transplant recipient.

Bone marrow transplantation is performed in some patients with cancers of the blood or bone marrow, including multiple myeloma and leukemia, as well as in some patients with sickle cell disease, thallesemia, aplastic anemia and inherited immune deficiency.

Physician-researchers at the Aflac Cancer and Blood Disorders Center at Children's Healthcare of Atlanta and Winship Cancer Institute of Emory University will harvest bone marrow cells from children and adults (12 to 65 years) with GVHD. Those cells will be used to manufacture large numbers of personalized autologous marrow mesenchymal stromal cells in the Emory Personalized Immunotherapy Center (EPIC), a dedicated pharmaceutical grade facility located within Emory University Hospital.

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, MD, MSc. a pediatric hematologist-oncologist at the Aflac Cancer Center at Children's and an assistant professor at Emory School of Medicine, will lead the clinical trial, which is offered only in Atlanta and is supported by CURE Childhood Cancer.

"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," says Dr. Qayed, "But we hope that through our clinical research, we will be able to significantly impact the course of this disease."

"This trial represents one of the most innovative clinical trials to arise from the growing partnership between the Hematology & Medical Oncology and Pediatrics departments at Emory School of Medicine, Emory Healthcare, and Children's Healthcare of Atlanta," says William (Bill) G. Woods, MD, director of the Aflac Cancer Center.

Blood and bone marrow cells have been used for more than a quarter century to treat life-threatening hematological conditions and are now used in established therapies worldwide. The current clinical trial will use 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.

"The beginning of this clinical trial is the culmination of two years' of collaborative effort by a terrific multidisciplinary team at Emory Healthcare, Children's Healthcare of Atlanta and the Aflac Cancer Center," says Edmund Waller, MD, director of Winship's Bone Marrow and Stem Cell Transplant Program and investigator on this trial.

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Clinical trial uses patients' own cells for treatment after bone marrow transplant

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