Federal Grant Award Summary

The National Cancer Institute awarded a $680,600 Project Grant to The Regents of the University of California, San Francisco, effective August 1, 2026, through July 31, 2031, under the Cancer Treatment Research program (CFDA 93.395). This award supports research to overcome medulloblastoma resistance to treatment through development and validation of innovative functional genomic approaches. The primary deliverable involves applying in vivo Perturb-Seq methodology—which combines CRISPR Interference (CRISPRi) screens with single-cell transcriptomics—to identify and characterize druggable genetic targets in pediatric medulloblastoma. By utilizing convection-enhanced delivery and epigenetic editing tools, the research aims to link genetic perturbations to transcriptional phenotypes and cellular behaviors, thereby enabling discovery of new therapeutic targets with reduced toxicity profiles.

The project addresses a critical clinical need in pediatric oncology, as current medulloblastoma treatments, while curative in 70% of patients, result in severe long-term neurocognitive, endocrine, and growth impairments for survivors, along with elevated rates of treatment-induced secondary malignancies. The research will deliver validated therapeutic candidates and preclinical data to support the development of improved treatment approaches that maintain or improve survival rates while minimizing treatment-related morbidity. Deliverables will include transcriptional maps, functional genomic datasets, and preclinical therapeutic candidates suitable for advancement toward clinical evaluation.