# Thalidomide

Source: https://onco.cc/drugs/thalidomide/  
OnCo record `thalidomide` (Treatment). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

Thalidomide is the drug behind the 1960s birth-defect tragedy, rehabilitated as the first immunomodulatory myeloma drug and the parent of lenalidomide and pomalidomide.

## Summary

Singhal (NEJM 1999) showed activity in refractory myeloma; approved 2006 with dexamethasone for newly diagnosed disease under the strict REMS-type distribution programme first built for its leprosy indication (1998). Still used in low-cost regimens worldwide (thalidomide-based induction/maintenance, e.g. in Myeloma XI and LMIC settings). Neuropathy, thrombosis and sedation limit it.

## Fields

- Kind: Treatment
- Status: approved
- Last checked: 2026-09-08
- Tags: gap-fill; historic
- Brand: Thalomid
- Modality: Oral cereblon modulator (first IMiD)
- Mechanism: Binds cereblon, altering E3 ligase substrate specificity (IKZF1/3 degradation), with anti-angiogenic and immunomodulatory effects; the mechanism explained decades after its teratogenic disaster.
- Approvals: US 1998: Erythema nodosum leprosum; US 2006: Newly diagnosed multiple myeloma with dexamethasone; EU 2008: Untreated multiple myeloma ≥65 or transplant-ineligible, with melphalan-prednisone

## Sources

- Wikipedia: https://en.wikipedia.org/wiki/Thalidomide
- Wikipedia: https://en.wikipedia.org/wiki/Thalidomide

## Connected records

- cancers: [Multiple myeloma](https://onco.cc/cancers/multiple-myeloma/), [Newly diagnosed multiple myeloma, transplant-ineligible](https://onco.cc/cancers/myeloma-transplant-ineligible/)
- technologies: [Anti-angiogenic therapy](https://onco.cc/technologies/antiangiogenic/), [Nerve damage from chemotherapy: what recovers, and what helps](https://onco.cc/technologies/cipn-recovery-and-treatment/), [PROTACs & molecular glues (targeted protein degradation)](https://onco.cc/technologies/protac-degrader/)
- targets: [IKZF1 (Ikaros)](https://onco.cc/targets/ikzf1/)
- companies: [Bristol Myers Squibb](https://onco.cc/companies/bms/)
- trials: [CASSIOPEIA](https://onco.cc/trials/cassiopeia/), [Combination Bisphosphonate and Anti-Angiogenesis Therapy With Pamidronate and Thalidomide](https://onco.cc/trials/nct00083382/), [Dexamethasone With or Without Thalidomide in Treating Patients With Newly Diagnosed Multiple Myeloma](https://onco.cc/trials/nct00033332/), [Thalidomide to Chemotherapy Related Nausea and Vomiting in Pancreatic Cancer](https://onco.cc/trials/nct06017284/)
- key papers: [CASSIOPEIA: daratumumab added to bortezomib, thalidomide and dexamethasone before and after transplant in newly diagnosed myeloma](https://onco.cc/key-papers/paper-cassiopeia-lancet-2019/)
- terms: [Immunomodulatory drugs (IMiDs) and CELMoDs](https://onco.cc/terms/imid/), [Venous thromboembolism (VTE)](https://onco.cc/terms/vte/)

---
JSON: https://onco.cc/api/v1/entities/thalidomide.json