New Treatments & Clinical Trials for Sickle Cell Disease

Sickle cell disease is an inherited blood disorder affecting about 100,000 Americans, primarily of African descent. Until recently, treatment focused on managing pain crises and preventing complications. That changed in 2023 when the FDA approved two gene therapies that can eliminate the disease for some patients.

What's actually going on in research

Trials are testing gene therapies that either edit patients' own blood stem cells or add a working gene, gene-editing approaches that reactivate fetal hemoglobin production, and newer drugs like voxelotor that prevent red blood cells from sickling. Researchers are also studying treatments for chronic pain, stroke prevention, and organ damage.

What to know before you search

Eligibility typically depends on disease severity (number of pain crises per year, organ complications), age, prior treatments including hydroxyurea, and whether someone is healthy enough for intensive therapies like gene therapy.

What types of trials are currently open

• Gene therapy trials — Testing approaches that modify a patient's blood stem cells to produce healthy red blood cells. These require collecting stem cells, editing them in a lab, then returning them after chemotherapy.
• Drug trials — Testing medications that reduce sickling, prevent crises, or manage complications. These often compare new pills or infusions to standard care.
• Pain management trials — Studies of treatments for acute pain crises and chronic pain, including new pain medications and non-drug approaches.
• Organ protection trials — Testing treatments to prevent or treat damage to kidneys, lungs, heart, and brain caused by repeated sickling episodes.
• Observational studies — Following patients over time to understand how the disease affects different organs and how well treatments work in real-world settings.