Experimental Drug Shows Long-Term Health Benefits To Sickle Cell Warriors
affecting many organs throughout the body, leading to premature death,” she said.
Right now, patients with sickle cell, many of them children, have few treatment options. “So far, there are only two FDA-approved drugs for sickle cell disease: hydroxyurea and glutamine,” Aygun noted.
The new 17-month, phase 3 clinical trial was designed to see if a third treatment might be on the horizon. It was funded by Voxelotor’s maker, Global Blood Therapeutics, and included 274 patients, ages 12 to 65, in 12 countries.
Patients were divided into three groups that received either a 900-mg or 1,500-mg daily dose of the drug Voxelotor, or a “dummy” placebo pill.
The study found that 51% of patients who took the higher dose of Voxelotor had a significant increase in their hemoglobin levels after six months of treatment, compared with 7% of those who received the placebo.
Another finding was that 41% of patients who took the higher dose of the drug reached hemoglobin levels of more than 10g/dl at 24 weeks. A normal, non-anemic hemoglobin count ranges between 11.5 to 17.5 g/dl, depending on age and gender, the study authors noted.
“Chronic organ failure, which is predicted by the severity of anemia, is a leading cause of