What you need to know
Joseph M. Sanzari Children’s Hospital and John Theurer Cancer Center recently launched a clinical trial evaluating gene therapy for severe sickle cell disease in adolescents and adults.
This is a multicenter Phase I/II clinical trial of an investigational gene therapy from bluebird bio, Inc, specifically for adolescents and adults with severe sickle cell disease (SCD) who cannot be effectively treated using standard therapies such as antibiotics, vitamins, blood transfusions or any pain relieving medications.
Details on the study
The study is evaluating the safety and effectiveness of LentiGlobin® for sickle cell disease, a gene therapy produced using the patient’s own modified stem cells to treat their sickle cell disease. By using the patient’s own cells to produce functional hemoglobin that can prevent sickling of their red blood cells, LentiGlobin for SCD offers patients the opportunity to treat their disease without the need to have a matched bone marrow donor.
In the current study:
- A patient’s stem cells will be collected from his or her blood and sent to a lab. LentiGlobin for SCD is used to add functional copies of a modified form of the β-globin gene (βA-T87Q-globin gene) into a patient’s own hematopoietic (blood) stem cells (HSCs). Once patients have the βA-T87Q-globin gene, they have the potential to make functional red blood cells.
- The patient will receive chemotherapy to prepare the bone marrow for the modified hematopoietic (blood) stem cells (HSCs) that now carry the βA-T87Q-globin gene.
- The modified stem cells are then returned to the patient, so that they will repopulate the bone marrow and produce red blood cells that are healthy.
Participants will be followed for two years after treatment with LentiGlobin for SCD to assess the treatment’s safety and effectiveness, based on blood tests and frequency of sickle cell disease symptoms and complications.
To date, the only cure for sickle cell disease is receiving a stem cell transplant from a matched donor, but this is not a therapeutic option for many patients. Without a marrow donor, there has been no alternate curative therapy. Life expectancy of a person with sickle cell disease is 20 to 40 years of age. In some cases, patients using disease modifying medications can live to 50 or 60.
The John Theurer Cancer Center is one of a limited number of centers internationally, and the Joseph M. Sanzari Children’s Hospital is the only pediatric site in New Jersey, where the study, which is enrolling patients age 12-50, is taking place.
For more information
To learn more about this study, please contact the study research staff email@example.com, or call 551-996-5600.