PLB-1001 is a selective RET kinase inhibitor primarily used in preclinical and early clinical research for targeted cancer therapy. It specifically targets RET gene fusions or mutations, which are found in certain thyroid cancers (e.g., medullary thyroid carcinoma) and non-small cell lung cancers (NSCLC). By inhibiting abnormal RET signaling, it reduces tumor cell proliferation, induces apoptosis, and slows cancer progression in genetically defined models. Its ability to cross the blood-brain barrier makes it promising for central nervous system metastases. As a research tool, PLB-1001 supports pharmacokinetic, pharmacodynamic, and efficacy studies in precision oncology, with ongoing clinical trials evaluating its safety and effectiveness in humans.