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Evidence record

Thalidomide × Multiple Myeloma

Cereblon-dependent degradation of IKZF1/3 disrupts myeloma cell survival signaling.

Approved
PublishedLast updated June 20, 2026Download evidence report
Drug: ThalidomideDisease: Multiple MyelomaTarget: Cereblon

Why this drug?

After catastrophic teratogenicity halted its use as a sedative, thalidomide was repurposed for leprosy complications and later multiple myeloma based on anti-angiogenic and immunomodulatory mechanisms.

Known risks

  • Severe birth defects (absolute contraindication in pregnancy)
  • Peripheral neuropathy
  • Thromboembolism
  • Sedation and constipation

Evidence behind this

Confidence score96%
Signal strength: HighLevel 1 (RCT + regulatory approval)
Evidence grade
Approved

Improved response rates and survival in combination regimens; REMS mandated.

Mechanism

Cereblon-dependent degradation of IKZF1/3 disrupts myeloma cell survival signaling.

clinical trialfda labelPhase III

Authoritative sources

  • PubMed · 11752346

    Thalidomide for refractory myeloma

    Singhal S et al. (1999)

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  • PubMed · 19717844

    Lenalidomide and dexamethasone vs dexamethasone alone

    Dimopoulos MA et al. (2009)

    View
  • ClinicalTrials.gov · NCT00029429

    Thalidomide in multiple myeloma

    View
  • FDA · 021430

    Thalomid FDA approval

    View

AI-generated summary

Generated March 15, 2026Last verified June 20, 2026

Thalidomide plus dexamethasone demonstrated significant activity in relapsed myeloma, leading to FDA approval. REMS requirements reflect teratogenicity risk. Strong approved repurposing case.

Source papers
  • Singhal S et al., 1999
  • Dimopoulos MA et al., 2009

This summary is AI-assisted and human-reviewed. It does not constitute medical advice. Always consult authoritative sources and qualified professionals.