While drugs have been the primary method of treatment for most diseases for many years, cell therapy may be the way of the future.

So says Edwin M. Horwitz, MD, PhD, Pediatric Hematologist/Oncologist at the Aflac Cancer and Blood Disorders Center of Children's and cellular therapy expert for 25 years. To help him on that journey of discovery, he has received a $31 million grant from the Marcus Foundation for a new Center for Pediatric Cellular Therapy, which will provide the infrastructure needed for clinical trials to make this a reality.

Cell therapy, in contrast to drugs, works by using natural or modified cells to treat disease. Methods include regenerative medicine; rebuilding diseased or damaged tissues with cells; and cellular immunotherapy, which modulates the immune system by boosting, calming or redirecting it, such as CAR-T therapy for cancer.

As Co-Director of the Center for Pediatric Cellular Therapy, Dr. Horwitz will promote the use of cells in all disciplines throughout pediatrics at Children’s and Emory. Already, seven clinical trials are in progress at the Center, studying such diseases as graft versus host disease (GVHD), asthma, neuroblastoma, dilated cardiomyopathy and osteogenesis imperfecta. The Center will include a GMP (good manufacturing practice) lab, known as the Marcus Cellular Production Facility, to be housed at Arthur M. Blank Hospital for generating cells to treat patients; a Cell Therapy Trials Office for managing clinical trials; and a Cell Therapies Correlative Studies Laboratory to analyze patient samples.

“We are in the midst of an evolutionary change in the way we take care of patients,” says Dr. Horwitz. “Drugs were the mainstay of therapy for many years, but more and more, cells will be used to do things that drugs cannot do.”

Researchers at Aflac Cancer and Blood Disorders Center of Children’s developed a new method for treating patients with severe hemophilia A in 2019. And that method – the “Atlanta Protocol” – is now being tested on a larger group of patients in an international, multisite, five-year trial.

Hemophilia A is an inherited bleeding disorder that causes abnormal blood clotting, resulting in more bleeding than normal due to low levels of factor VIII, a protein needed to form blood clots. About one-third of severe hemophilia A patients will develop even more bleeding due to inhibitors, antibodies generated by the patient’s body against their treatment.

The Atlanta Protocol involved combining an old approach, immune tolerance induction (ITI), with the drug, emicizumab. The treatment was effective in four out of seven patients who were able to rid their bodies of the disease’s stubborn antibody inhibitors. Given the successful results, Robert Sidonio Jr., MD, a Pediatric Hematologist/Oncologist at the Aflac Cancer and Blood Disorders Center of Children’s and Associate Professor of Pediatrics at Emory, is further investigating the approach as co-principal investigator of the 120-patient international trial. The protocol was originally developed by Dr. Sidonio, and Pediatric Hematologists/Oncologists Glaivy Batsuli, MD, and Shannon Meeks, MD.

The potentially life-saving results of bone and marrow transplant (BMT) procedures are sometimes compromised by a complication – one that the Aflac Cancer and Blood Disorders Center of Children’s is striving to overcome.

For hematologic cancer patients undergoing unrelated donor BMT, a condition known as acute graft-versus-host disease (GVHD) may occur after stem cell transplantation. This may result when the donated cells turn against the patient or host and begin attacking the patient’s organs. In the most severe forms, half of the transplant recipients die.

Providing new hope to patients, the drug abatacept was shown to reduce the risk of acute GVHD by 10 times among children and adults during a seven-year, multisite trial conducted by hematologists and oncologists at the Aflac Cancer and Blood Disorders Center of Children’s. Benjamin K. Watkins, MD, and Muna Qayed, MD, led the trial at Children’s, published their findings in the Journal of Clinical Oncology, and in December 2021 received FDA approval for the therapy.

“The incidence of severe disease dropped to only 3% in our abatacept treated group, compared to 30% for the control group, so it’s really a striking response,” says Dr. Watkins, an Assistant Professor of Pediatrics at Emory.

“Up until now, there has not been a groundbreaking drug to change the risk of getting GVHD in the first place,” says Dr. Qayed, Director of the BMT Program, an Associate Professor of Pediatrics at Emory and co-first author with Dr. Watkins. “Abatacept has the potential to save many lives by preventing this devastating disease.”