Identification of Raptor and GLI1 as USP37 substrates highlight its context-specific function in medulloblastoma cells

Oncogene. 2026 Feb;45(4):521-533. doi: 10.1038/s41388-025-03651-2. Epub 2025 Dec 18.

Abstract

The USP37 gene encodes a deubiquitylase (DUB), which catalyzes the proteolytic removal of ubiquitin moieties from proteins to modulate their stability, cellular localization or activity. Its expression is downregulated in a subgroup of medulloblastomas driven by constitutive activation of sonic hedgehog (SHH) signaling. Patients with SHH-driven medulloblastomas with elevated expression of the RE1 silencing transcription factor (REST) and reduced expression of USP37 have poor outcomes. In previous studies, we showed sustained proliferation of SHH-medulloblastoma cells due to blockade of terminal cell cycle exit and neuronal differentiation stemming from a failure in USP37-dependent stabilization of its target, the cyclin-dependent kinase inhibitor (CDKI)-p27. This finding suggested a tumor suppressive function for USP37. Interestingly, the current study also uncovered Raptor, a component of the mTORC1 complex, as a novel target of USP37. Under conditions of low-USP37 expression, reduced Raptor stability and mTORC1 activity caused a decline in phosphorylation of 4E-binding protein 1 (4EBP1) and increased its interaction with eukaryotic elongation factor 4E (eIF4E), which is known to inhibit CAP-dependent translation initiation. Surprisingly, a subset of patients with SHH-driven medulloblastomas with elevated expression of USP37 and the Glioma-associated Oncogene 1 (GLI1), also exhibited poor outcomes. Using genetic and biochemical analyses, we showed that USP37-mediated stabilization of GLI1, a terminal effector of SHH signaling, increases pathway activity and upregulates expression of its target oncogene product, CCND1, to drive cell proliferation. These data indicate that USP37 elevation in SHH-driven medulloblastomas has the potential to promote non-canonical activation of SHH signaling. Overall, our findings suggest that USP37 may have context-specific oncogenic and tumor suppressive roles in medulloblastoma cells.

MeSH terms

  • Cell Line, Tumor
  • Cell Proliferation
  • Cerebellar Neoplasms* / genetics
  • Cerebellar Neoplasms* / metabolism
  • Cerebellar Neoplasms* / pathology
  • Endopeptidases* / genetics
  • Endopeptidases* / metabolism
  • Gene Expression Regulation, Neoplastic
  • Hedgehog Proteins / genetics
  • Hedgehog Proteins / metabolism
  • Humans
  • Mechanistic Target of Rapamycin Complex 1 / metabolism
  • Medulloblastoma* / genetics
  • Medulloblastoma* / metabolism
  • Medulloblastoma* / pathology
  • Regulatory-Associated Protein of mTOR* / genetics
  • Regulatory-Associated Protein of mTOR* / metabolism
  • Signal Transduction
  • Ubiquitin Thiolesterase* / genetics
  • Ubiquitin Thiolesterase* / metabolism
  • Zinc Finger Protein GLI1* / genetics
  • Zinc Finger Protein GLI1* / metabolism

Substances

  • Zinc Finger Protein GLI1
  • GLI1 protein, human
  • Ubiquitin Thiolesterase
  • USP37 protein, human
  • Regulatory-Associated Protein of mTOR
  • RPTOR protein, human
  • Hedgehog Proteins
  • Endopeptidases
  • SHH protein, human
  • Mechanistic Target of Rapamycin Complex 1