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Updated: Jun 11, 2026

Chemical Inactivation of the E3 Ubiquitin Ligase Cereblon by Pomalidomide-based Homo-PROTACs
Published on: May 15, 2019
[Thalidomide in oncological and hematological diseases]
Sanna-Maria Kivivuori1, Pekka Anttila
1HYKS, lasten veri- ja syöpätautien sekä kantasolusiirtoyksikkö, Lasten ja nuorten sairaala, PL 281, 0029 HUS.
Thalidomide, once known for birth defects, is now valued for its antiangiogenic and immunomodulatory effects. It is cautiously used in myeloma therapy and pediatric brainstem tumor studies despite potential side effects.
Area of Science:
- Pharmacology
- Immunology
- Oncology
Context:
- Thalidomide's historical teratogenicity contrasted with its later discovered therapeutic properties.
- Recent decades have seen a resurgence in thalidomide research and clinical application.
- Understanding thalidomide's complex pharmacological profile is crucial for its safe use.
Purpose:
- To review the evolving understanding of thalidomide's therapeutic applications.
- To highlight its antiangiogenic and immunomodulatory mechanisms.
- To discuss current clinical uses and safety considerations.
Summary:
- Thalidomide, initially recognized for teratogenic effects, exhibits significant antiangiogenic and immunomodulatory activities.
- Current applications include multiple myeloma treatment and investigational use in pediatric brainstem tumors.
- Cautious administration is essential, particularly for fertile patients, due to teratogenicity and other adverse effects like thrombosis and polyneuropathy.
Impact:
- Re-evaluation of thalidomide demonstrates its value in specific oncological and potentially other therapeutic areas.
- Emphasizes the importance of risk-benefit assessment in drug repurposing.
- Informs clinical practice regarding the safe and effective use of thalidomide.
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