Sickle Cell Anemia
Sickle Cell Anemia is caused by an inherited hemoglobin disorder. Healthy red blood cells are discoid and can deform and move through small blood vessels to carry oxygen to all parts of the body. In sickle cell disease, as red blood cells circulate and oxygen is released in the circulatory system, the deoxygenated abnormal hemoglobin S polymerizes. When this occurs, the red blood cells can become sticky and elongated to look like a C-shaped farm tool called a 'sickle'. These sickled red blood cells do not deform. They obstruct small blood vessels and can block normal red blood cells from traveling through the circulatory system limiting oxygen delivery to tissues and organs. This is known as a "sickle crisis".
The number of crises a patient experiences varies. Most Sickle Cell Anemia patients experience at least one sickle crisis at some point during their lives; some may suffer 15 or more crises each year.
Sickle Cell Crisis
Patients suffering from a sickle crisis experience severe pain and are at risk of stroke, heart attack or even death. Preclinical studies have shown that carbon monoxide (CO) at therapeutic, non-toxic, levels stabilizes the hemoglobin of patients with sickle cell disease and prevents the sickling of red blood cells.
MP4CO has been designed to deliver targeted therapeutic levels of CO to patients suffering from sickle cell crisis. Once CO has been released, MP4 takes up oxygen, when circulating through the lungs, for delivery to ischemic tissues. It is believed that MP4CO will improve perfusion and limit progression of the vaso-occlusive crisis associated with sickle disease, through its anti-sickling, anti-inflammatory and anti-ischemic properties.
There are currently no approved medications to treat sickle cell crises. However, Phase 1 clinical trials of MP4CO in patients with sickle cell disease are underway.
This could mean less time in the hospital and an improved quality of life for patients with sickle cell anemia.