Bringing Novel Therapies to Patients With Pediatric Cancers

New research initiative aims to increase the number of clinical trials and therapies


The treatment of childhood cancers has been a medical success story, taking what were universally fatal diseases to now curing approximately 85% of children with cancer. However, cancer remains the leading medical cause of death among children and the cure rates for many childhood cancers have plateaued for decades. The treatments also carry high toxicity and most survivors have serious long-term side effects.

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In order to bring novel, more effective therapies to the youngest patients, Cleveland Clinic launched a new, early phase clinical trial program called Cleveland Clinic Children’s Cancer Innovative Therapy Program, led by Matteo Trucco, MD. The program takes a multipronged approach to improving treatments for children with cancer. In addition to trials though the Children’s Oncology Group, Cleveland Clinic Children’s is an active member of smaller, more agile consortiums including the National Pediatric Cancer Foundation’s Sunshine Project, and have developed several trials. Applying evolutionary biology and mathematical modeling principles to guide therapy to “outsmart” cancers prone to relapse, improving immunotherapy by including natural killer cell-based therapies, adding epigenetic regulators to immune checkpoint inhibitors to better unmask and destroy cancer cells, and repurposing old drugs such as disulfiram to overcome resistance to chemotherapy are only some of the innovative strategies we are developing. These trials hold the potential to revolutionize how we treat childhood cancer, leading to better and less toxic therapies.