The Phase I clinical trial investigates using a child's own immune system — CAR T-cells — to treat acute myeloid leukemia (AML)
No More Kids with Cancer is pleased to announce a $220,000 award to Children's Hospital of Philadelphia (CHOP). The $220,000 award will be directed to a Phase I clinical trial that will investigate the effectiveness of immune system cells — called CAR T cells — to treat relapsed or refractory acute myeloid leukemia (AML) in children and young adults. The trial will test the safety and potential efficacy of a new CAR T-cell immunotherapy in children and young adults with AML — the second most common form of leukemia in children.
Based at the Philadelphia campus, the trial will be led by Richard Aplenc, MD, PhD, MSCE, a physician-scientist and Assistant Vice President and Chief Clinical Research Officer at the Children's Hospital of Philadelphia Research Institute, and Professor of Pediatrics at the University of Pennsylvania School of Medicine. He will be working with Dr. Sarah Tasian at CHOP as well as Dr. Nirali Shah at the Pediatric Oncology Branch in the Center for Cancer Research at the National Institutes for Health (NIH) and Dr. Terry Fry at the University of Colorado, Denver.
There have been successes in using CAR T-cell therapies for children with other types of leukemia, and pre-clinical studies have demonstrated that CAR T-cell therapies could be used in AML treatments. During the Phase I clinical trial, the team will test appropriate dosage levels to be effective, determine the percentage of children who achieve remission, and identify side effects of the treatment. This trial is modeled upon current, successful Phase 1 trials for children with leukemia ongoing at CHOP and the National Institutes of Health.
"AML is the second most common form of leukemia in children, and there has been very little research into AML treatments specifically around relapsed/refractory AML," said Dr. Aplenc. "New strategies for treatment are needed in order to treat relapsed and refractory AML in a safer, more effective way. With advances in research, science and technology, we are aiming to find treatments that will give children not only a cancer-free future, but a future without lasting effects from debilitating treatments."
"Children with cancer deserve better treatments than drugs developed 50 years ago," said Amy Summy, Co-founder and President of No More Kids with Cancer. "AML is one of the most challenging childhood cancers to treat. This innovative trial brings the latest advances in cancer research to kids. We want to thank CHOP and the NIH team for their continued research innovation and dedication to children with cancer. We would also like to thank our donors for making this grant possible and believing in the power of research to cure kids. We hope this trial moves the ball forward for children and their families battling AML."
Children’s Hospital of Philadelphia