Dare to Dream: A Mission to Advance Pediatric Cancer Research

Gwen Nichols, MD, chief medical officer of Blood Cancer United (formerly the Leukemia and Lymphoma Society), discusses efforts to transform pediatric cancer care by addressing long-standing challenges in drug development for children, particularly in rare diseases such as pediatric acute myeloid leukemia. She explains that clinical trials for children have historically been delayed due to cost and low patient numbers, meaning therapies are often tested in adults first and reach pediatric patients too late.

To overcome this, she helped launch the global initiative “Dare to Dream,” which unites researchers, institutions, and countries to run collaborative pediatric trials. This international approach increases patient numbers and makes studies more feasible. The program also provides tumor sequencing for children—even when insurance does not cover it—to identify genetic targets and support future therapy development.

Nichols highlights that traditional clinical trial models are too slow for rare pediatric diseases, prompting a shift toward using real-world data and large-scale patient databases. By partnering with data platforms such as Tempus, the initiative aims to streamline research, reduce trial sizes, and accelerate drug approvals.

She also emphasizes improving access to care, ensuring children in smaller or underserved settings can receive appropriate treatments, and supporting families through financial assistance and centralized medical records. Overall, her work focuses on building a faster, more collaborative, and more equitable system for developing pediatric cancer therapies.