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.
Bizonyítékszint: Korai humán adat. Kis vagy feltáró emberi adat.
Kapcsolódó jelek
- Targeted immunotherapies for anaplastic lymphoma kinase-positive pediatric tumors: current advances and future perspectives
- Updates in Diagnosis, Management, and Treatment of Neuroblastoma
- Coexpression of MYCN and ALK Induces Neuroblastoma-Like Tumors From Human iPS Cell-Derived Cranial Neural Crest Cells.
- Emerging clinical and research approaches in targeted therapies for high-risk neuroblastoma
- Frequency and Clinical Significance of Clonal and Subclonal Driver Mutations in High‑Risk Neuroblastoma at Diagnosis: A Children's Oncology Group Study