New Treatments & Clinical Trials for Sickle Cell Disease

Sickle cell disease is an inherited blood disorder affecting about 100,000 Americans, mostly of African descent. Until recently, hydroxyurea and blood transfusions were the main treatments. Now three new therapies — two gene therapies and one CRISPR-based cure — have been FDA-approved, fundamentally changing what's possible.

What's actually going on in research

Trials are testing gene therapies that add a corrected gene or edit the patient's own cells to produce fetal hemoglobin, which doesn't sickle. Researchers are also studying drugs that prevent red blood cells from sickling, reduce inflammation and pain, and address organ damage from years of disease. Some trials focus on making gene therapy safer and more accessible.

What to know before you search

Eligibility typically depends on how severe the disease is, how many pain crises or complications a person has had, and whether previous treatments have worked.

What types of trials are currently open

• Gene therapy trials — Testing one-time treatments that modify a patient's stem cells to produce healthy hemoglobin. These require chemotherapy preparation and weeks in the hospital.
• Anti-sickling drug trials — Testing pills or infusions that prevent red blood cells from forming the sickle shape or reduce blockages when they do.
• Pain crisis prevention — Testing drugs to reduce the frequency and severity of pain crises, which are the main reason people with sickle cell go to the emergency room.
• Organ protection studies — Testing treatments to prevent or reverse damage to the kidneys, lungs, heart, and brain from repeated sickling episodes.
• Transplant trials — Studies of bone marrow transplant approaches, including using donors who are only half-matched, which could expand who can get this curative treatment.