Maximizing what's learned from clinical trials in children

TheoryLab

The St. Baldrick's Foundation, the largest charitable funder of childhood cancer research grants, and the American Cancer Society, a health organization dedicated to eliminating cancer, formed a partnership in 2019 to fund grants that will accelerate childhood cancer research with the goals of understanding and discovering new treatment options and improving care and survival in children with cancer. Kathleen Ruddy, St. Baldrick’s Foundation CEO, joined the podcast to talk through the goals of this unique partnership. “Why do some patients respond better than others to a particular treatment? Why does one treatment cause more late effects than another? What else can we learn to speed up progress, to cure more children, more effectively, and less harshly?” Then two of the grantees who have been funded through the partnership talked about what they hope to accomplish. Yael P. Mossé, MD, is Associate Professor of Pediatrics at the University of Pennsylvania and Director of the Neuroblastoma Developmental Therapeutics Program, as well as a pediatric oncologist at The Children's Hospital of Philadelphia. Dr. Mossé’s grant is focused on improving patient outcomes for ALK mutant neuroblastoma through precision molecular targeting. E. Anders Kolb, MD, is Vice Chairman for Research and Professor in the Department of Pediatrics at Sidney Kimmel Medical College at Thomas Jefferson University, as well as Director of the Nemours Center for Cancer and Blood Disorders at Nemours/Alfred I. duPont Hospital for Children. Dr. Kolb’s study aims to validate the detection of novel biomarkers for the Pediatric Acute Leukemia (PedAL) Initiative Sub-trials. 0:00 – Kathleen Ruddy, CEO of St. Baldrick’s Foundation 10:25 – Yael P. Mossé, MD, and E. Anders Kolb, MD 11:29 – Drs. Mossé and Kolb on why, “in pediatric cancer care, clinical trial participation is the standard of care” 13:31 – Dr. Mossé on how her team is “bringing the science to the patient” to learn how children with neuroblastoma respond (or don’t respond) to treatments 15:57 – Dr. Kolb on why it’s so important, and challenging, to bring precision medicine approaches to childhood cancer treatment 18:37 –Dr. Mossé explains the goal “to bring the science to the clinical trials in real time and not for there to be a lag” 21:38 – Dr. Kolb highlights how revolutionary it is for Dr. Mossé to change a clinical trial based on data emerging in the lab 23:26 – Dr. Kolb explains how “the AML that kids get is nothing like the AML that older adults get” and why this matters for drug development 27:43 – “We as pediatricians are taught early on,” notes Dr. Mossé, “that kids are not small adults, and it really is the same for pediatric cancer.” 30:17 – Dr. Kolb on the inspiration he drew from an initiative by St. Baldrick’s Foundation called Project:EveryChild, and describes his new study: “what we hope is that we will be as successful in relapse as we have been in newly diagnosed AML” 32:25 – Dr. Mossé on the value of collecting tissue over time, including at relapse, and how a major part of her new study is using liquid biopsies to collect samples in a less invasive way 34:43 – Dr. Kolb on the impact of this funding: “If we’re successful, we’re going to be able to rapidly screen for relevant biomarkers and we’re going to be able to enroll kids in the therapy that has the highest potential to provide benefit.” 37:46 – Dr. Mossé describes how this funding will support her research: “My hope and my expectation is to make a really big difference for a small subset of patients. I think that’s where cancer biology has turned now—one disease is not defined by its histology; it’s defined by its underlying molecular biology.” 40:29 – A message they’d like to share with children going through cancer treatment and with their parents and families