Sickle cell disease agents
Newer drugs that reduce pain crises in sickle cell disease by protecting red cells or stopping them sticking to vessel walls.
What they are
In sickle cell disease, haemoglobin S polymerises when it gives up oxygen, so red cells become rigid and stick to the endothelium, blocking small vessels (vaso-occlusive crises). Hydroxycarbamide remains the base treatment (own page). L-glutamine (oral) and crizanlizumab (an antibody against P-selectin) are added to reduce crises. Gene therapies are now authorised in some countries.
Mechanism of action
L-glutamine is thought to improve the redox balance (NAD) of sickle red cells and raise their reduced glutathione, protecting them from oxidative damage. Crizanlizumab binds P-selectin on activated endothelium and platelets and blocks the contacts between vessel wall, platelets, red cells and leucocytes that start a vaso-occlusive crisis.
Target: Red cell oxidative stress and adhesion to the endothelium (P-selectin)
Drugs in this class
- L-glutamineAn amino acid given as an oral powder to reduce pain crises and other acute complications of sickle cell disease from…Redox balance of sickle red cells (NAD and reduced glutathione)
- CrizanlizumabA humanised antibody against P-selectin, given by infusion to reduce pain crises in sickle cell disease from age 16.P-selectin on activated endothelium and platelets
Others in the class
- hydroxycarbamide (hydroxyurea), on its own page
- exagamglogene autotemcel (gene therapy)
- lovotibeglogene autotemcel (gene therapy)
Test yourself
Think of the answer, then tap the question to see it.
Which adhesion molecule does crizanlizumab block?
P-selectin.
What is the usual base drug for sickle cell disease?
Hydroxycarbamide (hydroxyurea).
How is L-glutamine given?
As an oral powder mixed with a drink or soft food.
Educational content on how drugs work; not a prescribing guide and no substitute for a health professional's advice. No doses are given.
Sources: ATCDDD - ATC/DDD Index (WHO Collaborating Centre for Drug Statistics Methodology, 2026); DailyMed - L-GLUTAMINE powder, for solution (DailyMed (U.S. National Library of Medicine), 2026).
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Representative model, for educational purposes only.