In two intracranial patient-derived xenograft tumor models in mice (glioblastoma multiforme and neural progenitor cell models), systemic administration of KHS101 reduced tumor growth and increased survival without discernible side effects. KHS101 crosses the blood-brain barrier (BBB) and selectively induces neuronal differentiation of hippocampal neural progenitor cells in vitro and in vivo. KHS101 treatment increases survival of glioblastoma multiforme xenograft tumor-bearing mice.
