Thalidomide is a medication used to treat cancers, particularly newly diagnosed multiple myeloma, and erythema nodosum leprosum. A piperidinyl isoindole originally introduced as a non-barbiturate hypnotic, thalidomide was withdrawn from the market due to teratogenic effects.
- Mechanism
- Curator reviewed · 3 references
The mechanism of action of thalidomide is not fully understood. Previous research indicate that thalidomide binds to cerebron, a component of the E3 ubiquitin ligase complex, to selectively degrade the transcription factor IKZF3 and IKZF1. These 2 transcription factors are vital for the proliferation and survival of malignant myeloma cells.
Regarding TNF-alpha, thalidomide seems to block this mediator via a variety of mechanism. Thalidomide can inhibit the expression myeloid differentiating factor 88 (MyD88), an adaptor protein that is involved in the TNF-alpha production signalling pathway, at the protein and RNA level. Additionally, thalidomide prevents the activation of Nuclear Factor Kappa B (NF-kB), another upstream effector of the TNF-alpha production pathway. Finally, some evidences suggest that thalidomide can block alpha-1 acid glycoprotein (AGP), a known inducer of the NF-kB/MyD88 pathway, thus inhibiting the expression of TNF-alpha. The down-regulation of NF-kB and MyD88 can also affect the cross talk between the NF-kB/MyD88 and VEGF pathway, resulting in thalidomide's anti-angiogenic effect.
- Primary indication
- Thalidomide is primarily used for the acute treatment and maintenance therapy to prevent and suppress the cutaneous manifestations of moderate to severe erythema nodosum leprosum (ENL).Curator reviewed · 12 structured indications
- Formula / weight
- C13H10N2O4 · 258.2295 g/mol (avg)
- First approval
- Canada, 2010 · United States, 2003 · European Union, 2016
- Also known as
- (+)-Thalidomide
- Code names
- NSC-527179 · NSC-66847
- Brand names
Resolves to
UEJJHQNACJXSKW-UHFFFAOYSA-NSMILESO=C1N(C2CCC(=O)NC2=O)C(=O)C2=CC=CC=C12What you can answer from here — as of September 20, 2026
Which drugs share a target with Thalidomide, and which of those have an active Phase 3 trial?
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