Abstract

Abstract This study aims to expand the application scope of boron neutron capture therapy (BNCT) for BPA-insensitive cancers. To apply BNCT in the treatment of brain tumors, convection-enhanced delivery (CED) is used as the drug administration method. CED is expected to enhance therapeutic efficacy by delivering drugs directly to tumor tissues. Therefore, the development of BNCT necessitates planning non-clinical trials from both pharmaceutical and medical device perspectives, including tests on standards, quality, and safety for clinical trial implementation. Previous investigations have demonstrated the high efficacy of BNCT using PBC-IP, a novel boron carrier targeting folate receptors, in a rat brain tumor model administered via CED. The group receiving PBC-IP CED irradiation alone showed 50% long-term survival (over 90 days), significantly outperforming the BNCT using intravenously administered boronophenylalanine (BPA) (MST: 37.0 days) used in clinical settings. Furthermore, studies on catheter insertion positions and neutron irradiation timing indicated that maintaining efficacy when administering drugs to the tumor margin and during the treatment window post-drug administration is feasible.Non-clinical trials, unlike routine basic investigations, are conducted following prior consultations with regulatory authorities, a process unfamiliar to many clinicians. Specific tests include general toxicity studies using rats and dogs. The results of these studies inform clinical trial planning and are essential for submission during regulatory consultations for clinical trial approval. As part of clinical trial preparation, drug efficacy evaluations and catheter performance data are considered to explore the clinical positioning of this drug in the treatment of malignant gliomas. Based on these findings, clinical trial implementation plans are formulated, aiming for the submission of a clinical trial application. This report examines the roadmap of drug development and current challenges in new drug development based on the current state of academic drug discovery.