thalidomide
Meaning of thalidomide: A sedative withdrawn after causing catastrophic birth defects, later brought back under strict control for myeloma and leprosy reactions.
Definition of thalidomide
Thalidomide is a drug that suppresses inflammation, blocks the growth of the new blood vessels a tumor depends on, and alters immune cell signaling. It is used against multiple myeloma, a cancer of plasma cells in the bone marrow, and against a painful inflammatory skin reaction that occurs in leprosy. Taken during pregnancy it causes severe malformations of the limbs and organs.
Why is thalidomide so tightly controlled?
It was first sold as a sedative and a remedy for morning sickness. Thousands of children were born with shortened or absent limbs, and with damage to the eyes, ears, heart and internal organs, before the connection was recognized and the drug withdrawn. The harm depends on exact timing: exposure during the short window when limbs are forming produces the characteristic pattern, and a single dose in that window is enough. Every country that now permits the drug requires a strict risk management program, with pregnancy testing, two forms of contraception and controlled dispensing.
How does it work against myeloma?
Thalidomide binds a protein called cereblon, part of the machinery a cell uses to tag unwanted proteins for destruction. Binding redirects that machinery onto two control proteins that myeloma cells depend on for survival. It also cuts the supply line, because a tumor cannot grow beyond a few millimeters without recruiting new vessels, and the drug interferes with angiogenesis, the formation of those vessels. A third effect falls on the immune system, stimulating T cells and natural killer cells while reducing the inflammatory messenger TNF. Success in multiple myeloma prompted the development of related drugs that share the mechanism at greater potency.
What is it used for now?
| Use | Basis |
|---|---|
| Multiple myeloma | Given in combination with other drugs |
| Erythema nodosum leprosum | Damps the inflammatory reaction of leprosy |
| Selected inflammatory conditions | Used where standard options have failed |
It is sold under the brand name Thalomid and as a generic in several strengths.
What side effects matter most?
Nerve damage in the hands and feet causes numbness, tingling and pain, and it can persist after the drug is stopped. The risk of clots in veins is raised, particularly in combination with steroids or chemotherapy, so measures to prevent clotting accompany treatment. Drowsiness, constipation and dizziness on standing are common, and blood counts can fall.
Also known as
Used in a sentence
Thalidomide was prescribed with an anticoagulant because of the recognized increase in venous clot risk.
Good to know
A single dose taken while limbs are forming can cause severe birth defects, which is why supply is governed by mandatory risk management programs wherever the drug is permitted.