Senicapoc could benefit SCD patients

In a 12-week, multicenter, Phase II, randomized, double-blind, dose-finding study, senicapoc was evaluated for its effect on hemoglobin level and markers of red blood cell destruction in sickle cell disease patients.

Those treated with the Gardos channel blocker, which works by limiting solute and water loss thereby preserves RBC hydration, achieved measurable improvements in hemoglobin levels. "An understanding of the pathophysiology of sickle cell disease is extremely important in order to identify new therapeutic targets," said Kenneth Ataga, of the Division of Hematology/Oncology at the University of North Carolina at Chapel Hill, USA, and lead author of the study.

Those in the high-dose senicapoc arm achieved a hemoglobin increase of 0.68g/dL versus 0.01g/dL for those on placebo. The team also found corresponding increases in hematocrit and RBC counts in those on senicapoc when compared to placebo. Notably, the sub-groups of patients taking hydroxyurea achieved similar results. In the low-dose group, the increase in hemoglobin was not statistically significant, noted Dr Ataga.