Ticagrelor is an approved blood thinner, or compound that prevents blood platelet clumping. Its maker, AstraZeneca, is also looking at it as a potential treatment for sickle cell anemia.

AstraZeneca markets ticagrelor in the United States under the brand name Brilinta. People who have had a heart attack use it to reduce their risk of developing blood clots and having a stroke or another heart attack.

AstraZeneca is investigating whether ticagrelor could be an effective way to reduce blood vessel blockages in sickle cell anemia patients. Blood vessel blockades, or vaso-occlusive crises, are a common cause of pain in these patients.

How ticagrelor works

Platelets are a type of blood cell. Their role is to form clumps, or clots, to stop bleeding when a blood vessel is damaged. Platelets do not form clots until they are activated, typically by contact with a damaged blood vessel wall.

Sickle cell anemia patients have more activated platelets than normal. This makes it more likely that sickle cells will stick to blood vessel walls or that other clumps of blood cells will form, increasing the frequency of blood vessel blockages.

It interacts with a protein receptor called P2Y12 that is found on the surface of platelets and plays an important role in regulating clotting. Ticagrelor is an antagonist to the receptor, which means that it blocks it. Blocking P2Y12 reduces clotting or platelet clumping. The hope is that it will reduce the frequency of blood vessel blockages in sickle cell anemia patients.

Ticagrelor in clinical trials

AstraZeneca has conducted three clinical trials of ticagrelor in adults and children with sickle cell anemia.

The objective of one Phase 2 trial (NCT02482298) was to see if ticagrelor could reduce the frequency and intensity of adult patients’ pain events. Raw results from the study have been posted on the clinical trial’s website but they have yet to be published.

Because children sometimes respond to medication differently than adults, ticagrelor also studied ticagrelor in children. This Phase 2 trial (NCT02214121) looked the effect of different doses of ticagrelor, as well as how the body absorbed and metabolized them. Results of the study are expected soon.

A Phase 1 trial (NCT03126695) in healthy volunteers examined how well the body absorbed ticagrelor from different types of tablets. Its results are also expected soon.

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