New Jersey’s Only Pediatric Bone Marrow and Stem Cell Transplant Program at Hackensack Meridian Health Joseph M. Sanzari Children’s Hospital Provides Patients with Access to Advanced Treatment Methods
Comprehensive Cellular Therapy Program for Malignant and Non-Malignant Diseases Now Incorporating Gene Therapy
Averaging 25 bone marrow transplants per year, the Pediatric Stem Cell Transplantation and Cellular Therapy Program provides patients access to therapies for a host of malignant and non-malignant conditions, from leukemia and sickle cell to bone marrow failure and immunodeficiencies.
One of the most recent and remarkable additions to the program is gene therapy.
Stacey Rifkin-Zenenberg, M.D. and Alfred Gillio, M.D. are participating in a multicenter Phase I/II clinical trial of an investigational gene therapy from bluebird bio, Inc. This trial is specifically for adolescents and adults with severe sickle cell disease (SCD) who cannot be effectively treated using standard therapies. 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. The Joseph M. Sanzari Children’s Hospital is the only participating pediatric site in New Jersey.
Learn more about advances in pediatric oncology happening at Joseph M. Sanzari Children’s Hospital and K. Hovnanian Children's Hospital by visiting:
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.