Synthetic compound prevents pain crises in sickle cell anemia

Aptamer against cell adhesion preventing RBCs from sickle accumulate in small blood vessels

Foto: Phil Jones/GHSU
From left to right: Dr. Diana R. Gutsaeva, C. Alvin Head, Tohru Ikuta and James B. Parkerson
From left to right: Dr. Diana R. Gutsaeva, C. Alvin Head, Tohru Ikuta and James B. Parkerson

Synthetic compound prevents congestion of cells that causes episodes of debilitating pain and mortality associated with sickle cell anemia, according to researchers at the Medical College of Georgia, USA.

The aptamer developed by Archemix Corporation in the United States, works occupying the receptors sticky lining the walls of small blood vessels where red blood cells sickle-shaped and white cells can accumulate. Bottling cell blocks the flow of blood and oxygen, causing pain and organ damage, and possibly death.


In mice with the disease, the administration of the aptamer before a painful crisis reduced the adhesion of sickle erythrocytes in 90% and white blood cells by 80%. The animals also had increased blood flow velocity and reduction in mortality.

Researchers argue that the compound can now proceed to clinical trials with the potential to treat an acute painful crisis, dealt with drugs today. The current form of the aptamer is liquid and it was administered intravenously or injected under the skin.

The new aptamer targets the receptor P-selectin, which are highly expressed in patients with sickle cell anemia.

"The aptamer blocks the receptors so they do not work," said study co-author, Tohru Ikuta. "There is almost no good compound that inhibits cell adhesion and many are toxic. At least in animal studies, the new compound is not toxic and does not provoke an immune response."

"Many people are focusing on developing new therapies for sickle cell disease, because today there is only one choice approved by the FDA (hydroxyurea, a chemotherapeutic agent)," explained study author, Diana R. Gutsaeva. "We believe that this aptamer has the potential to be one of the new therapies."