First-in-children cancer clinical trial

Last year, Theodore S. Johnson, MD, PhD, was awarded Georgia Regents University/Medical College of Georgia’s first grant from Alex’s Lemonade Stand Foundation (ALSF), an organization devoted to promoting research on childhood cancers. This three-year study is picking apart the mechanisms that drive tumor destruction when drugs that block the protein indoleamine 2,3-dioxygenase (IDO) are combined with chemotherapy and radiation. IDO is a natural regulator of the immune system that is often hijacked by tumors to help them escape immune attack. Blocking IDO removes this protection from the tumor and allows the immune system to play a major role in battling the cancer. Preclinical data from this study forms the basis for novel pediatric clinical trials.
Now, those trials can begin – thanks again to ALSF.

Dr. Johnson, a pediatric oncologist at the GRU Cancer Center, is one of just three inaugural recipients of ALSF’s new Bio-therapeutics Impact Grants, designed to accelerate clinical trials for childhood cancers. The Foundation’s unprecedented award amount of up to $1.5 million will support a $840,000 first-in-children clinical trial using an IDO inhibitor, indoximod, in combination with chemotherapy to target pediatric brain tumors. To guide the treatment of children, Dr. Johnson is a co-investigator for a clinical trial in adults, using indoximod plus chemotherapy for adult and adolescent patients with brain tumors. According to Dr. Johnson, “This kind of cutting-edge combination immunotherapy/chemotherapy trial is now state-of-the-art in adults, but has not yet been made widely available to children. The goal of the proposed pediatric clinical trial is to bring IDO-based combination therapy into the clinic for children with brain tumors, who currently have few options and limited access to immunotherapy.”

A banner story for GRU, Dr. Johnson earned his MD and PhD degrees here while studying IDO when it was first discovered by GRU Cancer Center investigators Drs. Andrew Mellor and David Munn in 1998. Four years later, they published the role of IDO in cancer. Now Dr. Johnson is bringing this truly translational, bench-to-bedside research to pediatric patients, with the goal of establishing the GRU Cancer Center as a leader in developing clinical trials using immunotherapy-based strategies to combat childhood cancer.

The other ALSF Bio-therapeutics Impact Grants were awarded to Baylor College of Medicine (Houston, TX) and Fred Hutchinson Cancer Research Center (Seattle, WA).