Marrow and Blood Cell Transplantation
Our Approach to Bone Marrow Transplantation
Bone marrow transplants are a vital weapon in our fight against a number of complex diseases, including leukemia, lymphoma, brain tumors, genetic disorders, immune system disease and sickle cell disease. In an effort to provide the best possible care, we have assembled a team of physicians, specialists, and support staff, including some of the nation's most formidable experts in pediatric bone marrow transplantation.
Bone marrow transplantation is an arduous experience. Patients require lengthy hospitalization, which is physically and emotionally challenging for patients and their families. Our comprehensive and family-focused brand of care eases stress and anxiety as much as possible. Our nurses, Child Life workers and support staff are specially trained to help our patients and their loved ones successfully manage this difficult experience.
Excellence in Stem Cell Transplantation
Particularly troublesome or recurring cancers may respond best to high doses of chemotherapy, which damage patients' normal blood cells. An emerging technology, stem cell transplantation is one of the most valuable lines of defense against this damage.
CHAM's bone marrow transplantation team excels at performing autologous stem cell transplantation: a treatment that uses the patient's own stem cells to fight diseases like advanced neuroblastomas, brain tumors and sarcomas. We've equipped ourselves with the highly specialized expertise and resources necessary to perform autologous transplants, including a unit specializing in the protective isolation necessary for optimum treatment.
Innovating New Treatments
As a research hospital, we pursue new agents that improve the efficacy and safety of bone marrow transplantation. We collaborate with other institutions to evaluate new medicines and bring them to clinical trials as part of our ongoing efforts to win the battle against childhood cancer.
Gene Therapy for Sickle Cell Disease and Thalassemia
Every person’s body has thousands of genes that act like an instruction manual, telling our cells how to work. In sickle cell disease or thalassemia, one of those genes doesn’t work the way it should. This causes red blood cells to be shaped differently or not carry enough oxygen, leading to pain, anemia, or the need for frequent transfusions.
Gene therapy uses a patient’s own blood stem cells, which are genetically modified in a lab to bypass the genetic problem that causes their disease.
After this process, the cells are returned through an infusion—similar to a blood transfusion. These new stem cells can make healthy red blood cell variants that reduce or even eliminate pain episodes, anemia, and the need for ongoing transfusions.
Recently, the FDA approved two gene therapies for sickle cell disease:
- Casgevy – uses CRISPR-CAS9 gene editing to turn on fetal hemoglobin, preventing sickling
- Lyfgenia – uses a lentiviral vector to add a healthy hemoglobin A variant
For thalassemia, the FDA-approved Zynteglo therapy allows many patients to stop the requirement of lifelong transfusions. Zynteglo uses a lentiviral vector to add a healthy hemoglobin A variant.
Every child’s journey is unique — and we work closely with families to ensure safety, understanding, and support throughout the process.
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