The FDA Has Approved A New Sickle Cell Treatment For The 1st Time In 20 Years
Prodigy, the late rapper who suffered from sickle-cell disease, played an important role in opening up dialogues about health in communities of color. The illness – which primarily affects African-Americans, Latinos, and other racial minority groups – caused him tremendous pain and suffering, a struggle about which he spoke frankly and openly throughout his career. And, while the details surrounding his specific cause of death have yet to be released, Prodigy’s stint at the Las Vegas hospital in which he died was directly related to complications stemming from his life-long disease.
Affecting roughly 100,000 Americans (about 1 out of every 5,000), it sounds like a relatively rare illness, but when further statistics are considered, its prevalence is astounding. According to the National Heart, Lung, and Blood Institute, one out of every 365 African-American babies is born with sickle-cell disease. Even more troubling is the fact that one in 13 is a genetic carrier of the disease at birth. The National Institute of Health places the number at 1 in 36,000 Hispanic-American children. All 50 states and Washington, D.C. require babies to be screened for sickle cell, but those living with the illness face a lifelong term of pain management and multiple forms of treatment at any given time. Furthermore, the life expectancy of someone living with the disease is 40 to 60 years – in the developed world.
There is no singular cure for sickle-cell disease, a hereditary blood disorder in which red blood cells become deformed and can cause blockages preventing blood flow to organs and tissue. Though treatable to some degree, the illness becomes potentially fatal when triggered by stressors including the cold, dehydration, and pregnancy, as blood cells become agitated and form into “sickle” shapes, sticking to the walls of vessels because they are unable to pass through the body normally. Gene therapy and bone-marrow transplants have in the past been used to varying degrees of efficacy to rid the patient of the illness, but for the average sufferer, these forms of treatment are hard to access and impossibly expensive.
Compounding the lack of viable, disease-ridding treatments is the fact that the Food & Drug Administration has not approved a new sickle-cell treatment drug in 20 years. That is, until this month. As reported by NBC, the FDA approved Endari, a drug that “reduces the complications associated with sickle-cell disease.” Dr. Alexis A. Thompson, head of the Hematology Section and Director of the Comprehensive Thalassemia Program at the Ann and Robert H. Lurie Children’s Hospital of Chicago, told reporters that the new drug is desperately needed. “We are able to provide vaccinations, penicillin, close medical management. But, when the complications arise, we have very few tools specific to sickle cell to offer patients,” he explains.
What makes Endari so promising as a panacea for sickle cell is that it consists of L-glutamine, an amino acid lacking in red blood cells of those suffering from sickle cell. The new drug elevates the levels of the missing compound in the blood, effectively ridding them of the fragility that allows them to morph into the deadly sickle shapes. As explained in the report, “Endari was studied in patients ages five to 58 years old, and researchers found that the patients who received the medication had fewer hospitalizations and shorter hospitalizations than those who took the placebo.”
Endari’s importance is also being expressed by the FDA, who quoted Richard Pazdur, M.D., acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research and director of the FDA’s Oncology Center of Excellence in its official announcement of the news. “Endari is the first treatment approved for patients with sickle cell disease in almost 20 years. Until now, only one other drug was approved for patients living with this serious, debilitating condition.”