Memorial Sloan-Kettering Cancer Center will begin evaluating a new stem-cell-based gene therapy for patients with the inherited blood disorder β-thalassemia. The clinical trial is the first to receive US Food and Drug Administration approval to treat this disease with genetically engineered cells and is a culmination of more than two decades of research led by Memorial Sloan-Kettering investigators.

This study is being done to see if the investigators can make the stem cells produce normal red blood cells and hemoglobin. The investigators do this by collecting the stem cells. The genes with mistakes are removed from the cells. These cells are then treated so they have the corrected gene for making normal hemoglobin. These treated cells are given back to the patient through an injection (shot) in the vein. This is also known as gene transfer. In order for the body to accept these cells, the patient will need to receive a low dose of a drug called busulfan. It is a drug that will prepare the body to receive the new stem cells.

This study will let the investigators know:

• If it is safe to give the patient the treated stem cells
• If the treated stem cells will go into the bone marrow without causing side effects.

More Information: clinicaltrials.gov, Cooley's Anemia Foundation