Abstract B001: The BAIAP2 pathway regulates proliferation and migration in medulloblastoma

Abstract Medulloblastoma (MB) is the most common malignant pediatric brain tumor, with some forms resistant to standard-of-care therapy. Therefore, novel drugs are needed that target different aspects of MB biology, including the proliferative and migratory properties of tumor cells. Using bioinformatic approaches, we demonstrate that the cytoskeletal scaffolding protein BAIAP2 is differentially expressed in MB compared to non-tumor brain cells, and overexpressed in MB and metastatic MB tissue compared to normal cerebellum. BAIAP2 interacts with the Rho GTPases CDC42 and RAC1, which have known roles in migration and invasion in multiple cell types. We demonstrate that BAIAP2 knockdown affects migration and invasion of MB cells. To characterize the BAIAP2 pathway further, we developed a small molecule screen using surface plasmon resonance (SPR) for compounds that bind BAIAP2. We discovered that the small molecule NSC678917 binds to BAIAP2 and inhibits MB cell viability and migration. Collectively, our studies suggest that the BAIAP2 pathway is a novel point of therapeutic intervention in MB. Citation Format: Luz C. Levanda-Sousa, Nitish Jangde, Robert Suter, Anna Jermakowicz, Jonathan Chu, Matthew D'Antuono, Mi-Hye Lee, Nagi Ayad. The BAIAP2 pathway regulates proliferation and migration in medulloblastoma [abstract]. In: Proceedings of the AACR Special Conference in Cancer Research: Brain Cancer; 2026 Mar 23-25; Philadelphia, PA. Philadelphia (PA): AACR; Cancer Res 2026;86(6_Suppl):Abstract nr B001.