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The targeted cancer therapies, also known as “molecular targeted therapies,” take advantage of the molecular and genetic differences between the cancer cells and the normal cells. It needs a thorough understanding of the cancer cells to develop drugs that can target specific molecular aspects that drive the growth, progression, and spread of cancer cells without affecting the growth and survival of other normal cells in the body.
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Related Experiment Video

Updated: Jun 28, 2025

An Orthotopic Mouse Model of Anaplastic Thyroid Carcinoma
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Teprotumumab in thyroid eye disease.

Hila Goldberg1,2, Amina I Malik1

  • 1Department of Ophthalmology, Blanton Eye institute, Houston Methodist Hospital, Houston, Texas, USA.

Saudi Journal of Ophthalmology : Official Journal of the Saudi Ophthalmological Society
|April 17, 2024
PubMed
Summary

Teprotumumab (Tepezza) is a novel therapy for thyroid eye disease (TED). This targeted treatment effectively reduces inflammation and symptoms, offering a new option for patients with this autoimmune condition.

Keywords:
Autoimmunegravesorbitopathyproptosistargetedteprotumumabthyroid

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Area of Science:

  • Ophthalmology
  • Endocrinology
  • Immunology

Background:

  • Thyroid eye disease (TED) is an inflammatory orbital condition often associated with hyperthyroidism.
  • Current treatments for TED range from conservative measures to systemic immunomodulators.
  • There was a need for targeted therapies to address the underlying inflammatory processes in TED.

Purpose of the Study:

  • To review the clinical features, pathophysiology, and traditional management of TED.
  • To detail the development and efficacy of teprotumumab, a targeted therapy for TED.
  • To summarize clinical trial data, safety profiles, and future research directions for teprotumumab.

Main Methods:

  • Review of clinical features and pathophysiology of TED.
  • Analysis of traditional management strategies for active TED.
  • Examination of pivotal clinical trials supporting teprotumumab's FDA approval.

Main Results:

  • Teprotumumab, a monoclonal antibody targeting IGF-1, is the first FDA-approved targeted therapy for TED.
  • Clinical trials demonstrate teprotumumab's efficacy and safety in active, moderate-to-severe TED.
  • Emerging data suggests teprotumumab's effectiveness in a broader patient population.

Conclusions:

  • Teprotumumab represents a significant advancement in TED treatment by targeting underlying inflammation.
  • The drug has shown proven efficacy and a favorable safety profile in clinical studies.
  • Ongoing and future investigations aim to further define teprotumumab's role in managing TED.