Bone marrow transplant
Bone marrow is a soft, fatty tissue inside the center of the bones, where blood cells are made. A bone marrow or stem cell transplant is a procedure that takes healthy cells from one person: the donor and puts them into someone whose bone marrow is not working properly. In SCD, the patient’s bone marrow is replaced with blood-forming stem cells from a donor who does not have SCD. The new bone marrow then produces red blood cells that are healthy since they do not contain a lot of hemoglobin S. For the transplant to work, the bone marrow must be a close match. Usually, the best donor is a brother or sister. Bone marrow or stem cell transplants are most common in cases of severe SCD for children who have minimal organ damage from the disease.
Gene therapies
In December 2023, the U.S. FDA approved two cell-based gene therapies for the treatment of SCD in patients 12 years and older: Exagamglogene autotemcel (also known as exa-cel), or CASGEVY™ and Lovotibeglogene autotemcel (also known as lovo-cel), or LYFGENIA™
Both products are made from the patients’ own blood stem cells, which are modified, and are given back as a one-time, single-dose infusion as part of a blood stem cell transplant. Prior to treatment, a patients’ own stem cells are collected, and then the patient must undergo high-dose chemotherapy, a process that removes cells from the patient’s bone marrow so they can be replaced with the modified stem cells.