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Hope Renews For Sickle Cell Disease Patients

Advances in treating rare blood disorder grab media spotlight.

Vial over paper with words sickle cell disease

Talk of breakthrough treatments, an injection of research funding and powerful personal stories of high-profile patients are giving people living with sickle cell disease renewed attention from the medical community and beyond.

The potential for new ways to treat the disease have become a major focus of the medical revolution brought on by precision medicine and gene therapy. Recently, The New York Times and "60 Minutes" highlighted the burgeoning number of clinical trials of potential gene therapies for sickle cell disease. CSL Behring’s own gene therapy candidate is set to enter clinical trials later this year.

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The Times report came on the heels of the U.S. government’s renewal of substantial funding for research into the condition. The law signed late last year sets aside millions for sickle cell research. It follows the September announcement of the Cure Sickle Cell Initiative, a National Institutes of Health effort to take advantage of medical advances to push toward a cure for the disease.

This past season several National Football League players donned special cleats to draw attention to sickle cell as part of the NFL’s “My Cause, My Cleats” campaign. The campaign also featured players wearing cleats to recognize Uplifting Athletes, a player-run nonprofit which works to raise awareness and research funding for people living with all rare diseases, including sickle cell disease.

Read about the second annual Uplifting Athletes Young Investigator Draft presented by CSL Behring, held in Philadelphia last month.

Sickle cell disease is an inherited condition that affects some of a person’s red blood cells. Healthy cells are round, but sickle cells are crescent-shaped. The condition causes pain, swelling and fatigue and can lead to kidney disease and strokes. For more information on the disease, check out our infographic.