Researchers Show Lorlatinib is Safe, Effective for Patients with High-Risk Neuroblastoma
Lorlatinib is safe and effective in treating high‑risk neuroblastoma with ALK alterations.
An international consortium led by Children’s Hospital of Philadelphia, Winship Cancer Institute, and the NANT Consortium reported that lorlatinib, a next‑generation ALK inhibitor, demonstrated safety, tolerability, and clinical activity in pediatric, adolescent, and adult patients with relapsed/refractory ALK‑driven high‑risk neuroblastoma. The study’s phase I results have prompted amendments to phase III COG and European trials to incorporate lorlatinib for newly diagnosed ALK‑altered patients.
High‑risk neuroblastoma remains a lethal pediatric cancer with poor survival, especially in ALK‑mutated patients where existing targeted therapies (e.g., crizotinib) show limited response rates. Demonstrating that lorlatinib is both safe and clinically active offers a potent, potentially curative option and may accelerate FDA approval, thereby improving outcomes for this vulnerable population.
Evidence level: Korai humán adat. Kis vagy feltáró emberi adat.
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