Lopez, Juanita SuzanneJuanita SuzanneLopezHaefliger, SimonSimonHaefligerPlummer, RuthRuthPlummerClement, Paul MPaul MClementJeffry Evans, Thomas RThomas RJeffry EvansLäubli, HeinzHeinzLäubliRoth, PatrickPatrickRothKristeleit, RebeccaRebeccaKristeleitBrazil, LucyLucyBrazilTabatabai, GhazalehGhazalehTabatabaiWick, AntjeAntjeWickWunderlich, BenjaminBenjaminWunderlichBeebe, KirkKirkBeebeEisner, Joel RobertJoel RobertEisnerLane, HeidiHeidiLaneEngelhardt, MarcMarcEngelhardtKaindl, ThomasThomasKaindlHau, PeterPeterHauHundsberger, ThomasThomasHundsbergerSteinbach, JoachimJoachimSteinbach2025-06-202025-06-202025-06-17https://boris-portal.unibe.ch/handle/20.500.12422/212281Lisavanbulin is a prodrug of the microtubule-targeting agent avanbulin. Both avanbulin and lisavanbulin have demonstrated significant antitumor activity in several preclinical tumor models including glioblastoma. Previous human studies demonstrated that 48-h infusions of intravenous lisavanbulin were well tolerated with preliminary activity in recurrent glioblastoma. The current phase 1/2a study evaluates the safety and tolerability of once-daily oral lisavanbulin in patients with solid tumors or recurrent glioblastoma or high-grade glioma. Lisavanbulin is associated with profound, durable responses in a subset of patients with recurrent refractory grade 4 astrocytoma or glioblastoma. We present here the clinical and translational results from this trial, including a description of a response-predictive molecular signature that warrants further exploration in these tumor types of significant unmet need. The study is registered at ClinicalTrials.gov (NCT02490800).enastrocytomaglioblastomagliomalisavanbulinarticle10.48620/886374053265910.1016/j.xcrm.2025.102165