Precision medicine directed therapy enabling long-term survival in medulloblastoma: a case report - Summary - MDSpire

Precision medicine directed therapy enabling long-term survival in medulloblastoma: a case report

  • By

  • Laura Vazquez

  • Jonathan Lerch

  • Divya Gandra

  • Abigail Moore

  • Jessica Lincoln

  • Abhinav B. Nagulapally

  • Suzanne Treadway

  • Krishnamoorthy Thamburaj

  • Valerie Brown

  • Giselle Saulnier Sholler

  • July 14, 2026

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Objective:

To present a case of a 6-year-old male with subgroup-SHH medulloblastoma treated with targeted therapy after relapse.

Approach:
  • Case Presentation: A 6-year-old male with SHH medulloblastoma underwent surgery, chemotherapy, and ASCT, but relapsed nine months later.
  • Genomic Analysis: The tumor was sequenced for DNA and RNA, leading to a precision medicine approach.
  • Targeted Therapy: The molecular tumor board recommended vorinostat, vismodegib, curcumin, and palbociclib, resulting in a complete tumor response.
Key Findings:
  • The patient had a complete tumor response to targeted therapy.
  • Long-term survival was achieved post-treatment.
  • Combined molecular therapies demonstrated synergistic efficacy compared with single-agent treatment.
Interpretation:

Limitations:
  • The findings are based on a single case report.
  • Generalizability to broader patient populations is uncertain.
Conclusion:

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