Sancilio recently completed enrolling children with sickle cell anemia/disease in a Phase 2 clinical trial to evaluate a new therapy called Altemia (docosahexaenoic acid).
The SCOT trial, as it will be known, is aimed at determining the optimal dose of Altemia for patients, as well as determining its general safety and effectiveness. To do this, the participants, aged 5 to 17, of the trial will be randomized to either receive one of three dosages of the drug or a placebo, orally, once per day.
The study is expected to be finished in August, with the hope that Altemia will prove to lower certain red blood cell fatty acids in patients.
But how will this help in the treatment of sickle cell anemia?
Sickle cell anemia is a rare and chronic blood disease, and there is currently a lack of suitable treatments available for its management. To learn more about these treatments, and causes and symptoms of the disease, click here.
Studies have shown that sickle cell patients have fatty acid levels in their blood that are abnormally high, and in 2001, one even proved that certain fatty acids can actually reduce episodes of pain in sickle cell patients, as well as increase hemoglobin, reducing anemia and organ damage.
With all of this in mind, Sancilio engineered Altemia to be able to replenish these fatty acids that have been destroyed by beta thalassemia gene mutations, which are responsible for sickle cell disease.
Restoring the function of them would in turn restore a balance to levels of hemoglobin in the blood cells. The cascading effect of Altemia would be the minimization of damage to the fluidity of blood cells, ultimately, reducing sickle cell crises and death overall.
