New treatments are in the pipeline
Sickle cell disease, the most common inherited blood disorder in the United States, affects about 100,000 children and adults. New treatments are needed to prevent and treat its serious complications. That’s why the Food and Drug Administration (FDA) is working to help develop new treatments for SCD.
Though sickle cell disease most commonly affects people of African descent, it also can affect Hispanics, Asians and people of Mediterranean and Middle Eastern descent. More than 3 million Americans, including one in 12 African Americans, carry the sickle cell trait, the gene that can allow the disease to be passed on to children. In the U.S., screening for sickle cell disease and trait is typically done at birth.
Sickle cell disease is chronic and debilitating, with severity of the disease varying from person to person. People with the disease have “sickled” or abnormally shaped red blood cells that get stuck in small blood vessels and block the flow of blood and oxygen to major organs in the body. These blockages can cause severe pain, organ damage or even stroke. Other complications include susceptibility to infection, fatigue and delayed growth. Most people with the disease will have a shortened lifespan.
Current treatments for sickle cell disease are limited and include a medication called hydroxyurea. Bone marrow or stem cell transplants may be an option for younger patients, but serious and potentially life-threatening side effects can accompany these procedures. The transplants are expensive and require a donor with suitable bone marrow or stem cells.
“The majority of patients with sickle cell disease are treated with hydroxyurea, pain medication and chronic transfusion, and are hospitalized when crises occur,” explains Ann T. Farrell, M.D., director of FDA’s Division of Hematology Products. Transfusions are given regularly to prevent complications.
“Unfortunately, although a number of patients will experience a reduction in their crises and hospitalizations with current treatments, some patients will have no reduction of their symptoms and the disease will continue to progress,” Dr. Farrell says. “Better therapies are desperately needed.”
A growing number of these new therapies are in the pipeline, and researchers are exploring new approaches to treating the disease. “These potential treatments are in different stages of development, including early and late clinical trials,” Dr. Farrell says.
The FDA also has met with patients to learn more about their experiences with sickle cell disease and their views on existing treatments.
“Learning the patient perspective—what symptoms bother them the most and where they would like to see treatments to focus—is important in the development of new therapies,” Dr. Farrell says. “During the meeting, patients stated very clearly that the symptoms that matter most to them in terms of their disease are acute and chronic pain, stroke, fatigue and sleep disturbances. Patients also expressed a willingness to participate in clinical trials to identify new treatments for their disease.”
Patients also confirmed that the health effects of sickle cell disease are wide-ranging, and that the challenges of adequately managing their disease are much greater than the availability of medical treatments.
“FDA’s Division of Hematology Products considers the development of new treatments a top priority. We will continue to facilitate problem-solving with sponsors as much as possible in order to help remove roadblocks to new product development,” Dr. Farrell says. “We feel for these patients because they have to live with the devastating consequences of this chronic condition.”