Lorlatinib monotherapy or combination therapy in anaplastic lymphoma kinase-driven high-risk neuroblastoma
A targeted drug for ALK-driven neuroblastoma worked in nearly two-thirds of children, with almost perfect response in those with specific mutations.
In 25 patients with ALK-driven neuroblastoma treated with lorlatinib, the overall ORR was 64.7% with 100% ORR in ALK hotspot mutation-only patients and significantly reduced efficacy in those with concurrent MYCN amplification (ORR 25%). These findings from NPJ Precision Oncology establish molecular determinants of lorlatinib sensitivity in pediatric neuroblastoma and reveal resistance mechanisms including BRAF fusions and MET amplification.
What the study was
- Study design
- Single-arm retrospective clinical series
- Population
- ALK-driven neuroblastoma patients (n=25, 17 evaluable), primarily high-risk; pediatric oncology
- Sample size
- 25
- Category
- Treatment Innovation
- Maturity
- Exploratory
- Journal
- NPJ Precision Oncology
Why it surfaced
NPJ Precision Oncology. Pediatric high-risk neuroblastoma is a high-unmet-need area. Lorlatinib 100% ORR in hotspot ALK mutations is a strong efficacy signal, though limited by n=25 and single-arm design.
A plain-language summary of published research — not medical advice. Talk to a clinician about your own care.