Formal Trial Name
A Gene Transfer Study Inducing Fetal Hemoglobin in Sickle Cell Disease
This study is funded in part by the National Institutes of Health (NIH)/National Heart, Lung, and Blood Institute (NHLBI) and the California Institute for Regenerative Medicine (CIRM).
To test whether the gene therapy approach (lentiviral vector shmiR), which decreases expression of the BCL11A gene, can improve or eliminate painful episodes.
This phase 2 trial is based on a pilot/phase 1 study also funded by the NIH being completed at Boston Children’s Hospital, which showed early safety and efficacy in the initial 10 patients enrolled in the trial.
The study is designed to test whether a gene therapy approach targeting the BCL11A (B cell lymphoma/leukemia 11A) gene can eliminate painful episodes. In this study, a patients’ blood-forming cells (blood stem cells) are collected and modified in the laboratory (gene transfer) in order to reduce the expression of the form of hemoglobin that causes the red blood cells to sickle, and to increase a naturally occurring form of hemoglobin that does not sickle called fetal hemoglobin (HbF). The goal of this gene therapy is to maintain high levels of HbF production. In a pilot study, decreasing the expression of this gene in sickle cell patients increased the amount of fetal hemoglobin while also reducing the amount of sickle hemoglobin in their blood, which reduced the severity of the disease.