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Finding Treatments for Sickle Cell Disease Is a Global Health Issue

Jacqueline Burrell

Communications Director, Sickle Cell Disease Association of America

Sickle cell disease (SCD) means pain. For siblings Elizabeth and Raymond, and over 100,000 people just like them in the United States and millions more around the world, SCD means staying indoors during cold weather. Cold can trigger debilitating pain episodes. It means strokes, multiple surgeries, blood transfusions, and numerous hospital visits.  It means organ damage, anemia, fatigue, infections, and, sooner than it should – death. Elizabeth was 22 years old when she died. Raymond was just four.

Impacts of SCD

SCD is an inherited blood disorder. It alters the blood’s hemoglobin, the red blood cell molecule that delivers life-giving oxygen to the body’s organs and tissues. SCD blood forms sticky, sickle-shaped cells that glob together, blocking the oxygen.

SCD affects people whose ancestors came from sub-Saharan Africa; Spanish-speaking regions in the Western Hemisphere (South America, the Caribbean, and Central America); Saudi Arabia; India; and Mediterranean countries such as Turkey, Greece, and Italy. Worse, 4 million people live with sickle cell trait.

Advancements in care

Underfunded and under-researched, SCD treatments could be counted on one hand with fingers to spare. But today, SCD is realized as a global health issue. Promising clinical trials are underway, some using gene therapy. 

Last November, the FDA approved two new treatments: Adakveo (Crizanlizumab), a monthly injection inhibiting the stickiness of blood cells, and Oxbryta (Voxelotor), daily pills blocking the abnormal binding of red blood cells. Cost, however, remains a serious factor.

Other options

There are two others treatments. Endari is an oral L-glutamine therapy, approved in 2017 for children as young as five and adults and the first new treatment in almost 20 years. Hydroxyurea, for years the sole treatment used to reduce the frequency of pain crises, was first tested in 1984 and FDA approved in 1998. 

None are cures. A complicated bone marrow transplant is the only known cure at this time.

Beverley Francis-Gibson, president and CEO of the Sickle Cell Disease Association of America, says, “Now more than ever, we need the sickle cell community to stand up and advocate for greater access to new treatments and to increase understanding of the impact of SCD on individuals and families.”

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