Sickle Cell Disease
Emmaus Life Sciences is in clinical development of a patented experimental treatment approach of an orally administered pharmaceutical grade L-glutamine that may be determined to treat some of the symptoms of sickle cell disease (SCD).
SCD is an inherited blood disorder that affects red blood cells. Red blood cells contain hemoglobin which allows red blood cells to carry oxygen from the air in the lungs to all parts of the body.
Normal red blood cells contain hemoglobin A. In contrast, the bone marrow of people with SCD produces red blood cells with a different form of hemoglobin called hemoglobin S (S stands for sickle). When a person has SCD, rather than remaining round, smooth and flexible, the red blood cells become sickle (crescent) shaped, inflexible, and sticky as they release oxygen to other tissues in the body.
Normal red blood cells live for about 120 days before they are replaced with new ones. In sharp contrast, sickle-shaped red blood cells are destroyed faster, in about 16 days, and cannot always be replaced quickly enough. As a result, people with SCD are often anemic. The complications of sickle cell disease occur when sickle-shaped red blood cells block veins which then can cause pain in the arms, legs, back and stomach, bones, skin and other parts of the body. Tissue that does not receive normal blood flow eventually becomes damaged.
Currently, there is no universal cure for sickle cell disease.
Types of Sickle Cell Disease
There are several types of sickle cell disease. The most common are:
- Sickle Cell Anemia (SS)
- Sickle-Hemoglobin C Disease (SC)
- Sickle β+-thalassemia and Sickle β°-thalassemia
Emmaus Life Sciences is in clinical development of an experimental treatment approach covered under US Patent No. 5,693,671 to Niihara, et al, of which the company is the exclusive worldwide licensee. The Phase 3 clinical trial was completed in December 2013. Emmaus Medical’s treatment will consist of, if FDA approved, patients orally consuming pharmaceutical grade L-glutamine on a daily basis to treat some of the symptoms of SCD.