Collaborative Effort of Nonprofits Yields Big Results for Treating High-Risk Neuroblastoma

New York, NY, April 3, 2023 —The collaborative efforts of eight nonprofit organizations to impact survival rates of children with high-risk neuroblastoma by funding innovative clinical research has just resulted in very promising results. This collaboration, led by Solving Kids’ Cancer, supported the testing of lorlatinib as a targeted drug therapy for treating neuroblastoma tumors that have an alteration in the ALK gene. The clinical trial reported significant responses in relapsed and refractory patients who had disease that was resistant to other therapies.

The findings, recently published in Nature Medicine by senior study author Yael P. Mossé, MD, Professor of Pediatrics in the Cancer Center at Children’s Hospital of Philadelphia (CHOP) and colleagues [link], show that lorlatinib is safe and tolerable and resulted in far more responses than the first-generation ALK inhibitor crizotinib. This work has led to rapidly incorporating this drug in a phase 3 trial for newly diagnosed children with ALK-driven high-risk neuroblastoma, instead of crizotinib. The process took 4.5 years from testing the drug in phase 1 to phase 3, in contrast to 16 years for dinutuximab, the most recent drug approved for first line therapy for neuroblastoma

Neuroblastoma is an aggressive pediatric cancer that develops from early nerve cells, often appearing as a solid tumor in the chest or abdomen. The disease affects very young children, and accounts for up to 10% of childhood cancer deaths. Less than 50% of patients with high-risk disease survive, and researchers continue to identify new ways to treat the disease. With the discovery of subtypes that have mutations that can be targeted, drugs like lorlatinib can “turn off” the ALK mutated driver of the cancer cell and result in dramatic responses and even complete remission.

“We are so pleased and excited about these significant findings,” said Scott Kennedy, Executive Director of Solving Kids’ Cancer, “It validates moving promising therapies earlier in the treatment process before kids relapse. This is a big step in the right direction and illustrates the impact nonprofit collaboration can have with a shared common goal.”

Solving Kids’ Cancer, in partnership with Solving Kids’ Cancer UK, Band of Parents, The Catherine Elizabeth Blair Foundation, Children’s Neuroblastoma Cancer Foundation, The Evan Foundation, Ronan Thompson Foundation, and Wade’s Army also supported sites in London and Paris to give more children access to this important clinical trial.

CONTACT:  Kristi McKay

            Solving Kids’ Cancer

            kristi@solvingkidscancer.org

            212-588-6624

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About Solving Kids’ Cancer

Solving Kids’ Cancer (SKC) is not just our name, it’s our mission. SKC focuses on improving survival rates for the most aggressive childhood cancers by advancing clinical trials of next-generation treatments and using advocacy to improve uptake of currently available discoveries in a collaborative approach that addresses today’s unmet needs, because every kid deserves to grow up.