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Spontaneous Murine Model of Anaplastic Thyroid Cancer
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Targeted therapy with vemurafenib in BRAF(V600E)-mutated anaplastic thyroid cancer

Matthias Lang1, Thomas Longerich2, Chrysanthi Anamaterou3

  • 1Department of General-, Visceral- and Transplant Surgery, University Hospital Heidelberg, Heidelberg, Germany. matthias.lang@med.uni-heidelberg.de.

Thyroid Research
|March 1, 2023
PubMed
Abstract

Insights

BRAF inhibitor vemurafenib shows promise for anaplastic thyroid cancer (ATC) with BRAF V600E mutations, offering a survival benefit. However, acquired resistance to this targeted therapy remains a challenge in treating this aggressive malignancy.

Area of Science:

  • Oncology
  • Molecular Biology
  • Genetics

Background:

  • Anaplastic thyroid cancer (ATC) is a rare and aggressive malignancy with poor prognosis.
  • ATC exhibits resistance to conventional treatments like surgery, radioiodine, radiotherapy, and chemotherapy.
  • The BRAF V600E mutation occurs in approximately 25% of ATC cases.

Purpose of the Study:

  • To report the first case of significant response to vemurafenib in BRAF V600E-mutated ATC.
  • To review current treatment strategies for anaplastic thyroid cancer.
  • To evaluate the efficacy and limitations of BRAF inhibition in ATC.

Main Methods:

  • A case report of a 78-year-old male patient with BRAF V600E-mutated ATC treated with vemurafenib monotherapy.
  • Comprehensive literature review of current ATC treatment approaches and BRAF pathway targeted agents.
  • Analysis of treatment response and survival outcomes.

Main Results:

  • The patient achieved a marked partial response to adjuvant first-line vemurafenib monotherapy, sustained for 7 months.
  • Disease progression was observed after 7 months, with the patient dying 10 months post-diagnosis.
  • BRAF inhibition demonstrated a survival benefit in this BRAF-mutated ATC case.

Conclusions:

  • BRAF inhibition, exemplified by vemurafenib, offers a therapeutic benefit for patients with BRAF V600E-mutated anaplastic thyroid cancer.
  • Acquired resistance to BRAF-targeted therapy presents a significant limitation.
  • Further research into overcoming resistance mechanisms is crucial for improving outcomes in ATC.
Keywords:
Adjuvant targeted therapyAnaplastic thyroid carcinomaBRAF(V600E)-mutationBRAF-inhibitorReviewVemurafenib

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