Nearly 100,000 people in the US and millions worldwide suffer from the pain and shortened life-span associated with sickle cell disease. Today, scientists at Cincinnati Children's are on the brink of a gene transfer therapy that could end the suffering caused by this genetic disease.
What is Sickle Cell Disease?
Sickle Cell Disease (SCD) is a life-long, painful condition affecting more than 100,000 people in the United States and millions worldwide. The disease distorts normally round, red blood cells into rigid sickle or crescent shapes, which can clog small blood vessels, decreasing the oxygen and blood flow through the body. The result can be chronic episodes of extreme pain, breathing problems, infections, stroke, organ damage and many other complications, including a shortened lifespan. While people of any race can have SCD, it primarily affects those of African American, Mediterranean, Middle Eastern and Indian descent.
SCD Takes a Toll on Families
Sickle cell disease takes a heavy toll on children and families. Pain can strike at any time, so patients and families never know when they’ll need to rush to the nearest emergency department. Patients can spend large portions of their lives in the hospital being treated for pain and other conditions caused by the disease, which means they miss out on many day-to-day activities including school, work and time spent with loved ones. Currently, the only lasting treatment for SCD is bone marrow transplantation. However, there is only a 1 in 10 chance of finding a matching bone marrow donor.
We Are Working to End the Suffering
Researchers in the Comprehensive Sickle Cell Center at Cincinnati Children’s are on the brink of a discovery that may end the suffering caused by SCD.
Researchers at Cincinnati Children’s are working on a new gene therapy, the first of its kind in the nation, that appears to prevent the sickling of red blood cells altogether. Researchers are preparing for clinical trials for adults, the first step in making the treatment available to children.
As we enter a new era of hope and discovery, please join us in the battle to end suffering caused by SCD.