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  • New clinical trial gives hope to sickle cell patients
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New clinical trial gives hope to sickle cell patients

In honor of National Sickle Cell Awareness Month, experts at Children’s National Hospital share insight on the disease and new studies.

By Children's National Hospital Contributor September 25, 2019 at 5:08 pm

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Photo by Louis Reed

To help focus and raise awareness for the need for research, support and treatment of sickle cell disease, Congress has designated September as National Sickle Cell Awareness Month. The Comprehensive Sickle Cell Program at Children’s National Hospital is one of the largest pediatric programs in the United States and provides comprehensive care to more than 1,400 sickle cell patients in Washington, DC and the surrounding region.

Sickle cell disease is an inherited disorder of the red blood cells that affects approximately 100,000 people in the United States. One in every 13 African-Americans have sickle cell trait (SCT) and one in 375 have sickle cell disease. Sickle cell disease also occurs in one in every 16,300 Latino Americans.

To combat sickle cell disease, researchers within the Children’s National Sickle Cell Disease Program are implementing innovative clinical trials to help patients get better.

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“We recognize that there are limited disease-modifying treatments for sickle cell disease,” said Dr. Andrew Campbell, director of the Comprehensive Sickle Cell Program at Children’s National. “We need to continue pushing for resources to provide innovative approaches to improve the access of our patients to novel therapies and BMT treatments. “

As new research emerges in the fight against sickle cell disease, another clinical trial being co-led by Robert Nickel, M.D., hematology specialist at Children’s National and Allistair Abraham, M.D., blood and marrow transplantation specialist at Children’s National, are conducting sickle cell transplants using a Nonmyeloablative approach, also known as the “SUN” trial. The trial studies show how they can use sibling blood stem cell transplants to cure sickle cell without chemotherapy.

“Our hope is that the SUN trial moves us closer to a curative treatment for sickle cell disease without the need for hospitalization,” says Dr. Abraham. “Support for research like the SUN trial is critically important to advance cures for sickle cell disease.”

Using this method, the patient’s bone marrow will not be completely destroyed or wiped out. Currently, the SUN trial, supported by the Doris Duke Charitable Foundation, is a multiple-trial site with Children’s National being the lead.

For more information, visit childrensnational.org.

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