Sickle cell anemia is a genetic disorder that reduces the capacity of red blood cells to deliver oxygen to organs across the body. The disease is estimated to affect more than 100,000 Americans total and has a market worth billions globally. Up to 31% of patients with the condition may develop episodes of vaso-occlusive crisis (VOC), which is a life-threatening condition caused by the accumulation of deformed, or sickle-shaped, red blood cells that block major blood vessels.
For over 60 years, the number of treatment options available were either limited or posed severe risk to patients. For starters, sickle cell patients could receive blood transfusions, but such procedures require regular visits to the clinic and take up to four hours. Next, while bone marrow transfusions can cure the disease, the wait-line can be excruciatingly long and success depends on finding a match. Finally, although patients developing VOCs can take a generic drug known as hydroxyurea to reduce their frequencies, the drug is a carcinogen (substance causing cancer) and can lead to leukemia.
But last year, the Food and Drug Administration approved two innovative therapies for the treatment of sickle cell disease. In addition, there is significant progress in using gene therapy to treat the condition. Without further ado, let's see if the companies behind these new treatments are worth the buck.