Explore Cancer & Blood DisordersBlood and Marrow Transplant and Cellular Therapy Program
Overview
For decades, bone marrow transplants have been used to treat aggressive cancers. Today, through scientific research and innovation, this widely used therapy can now treat many more diseases through our Blood and Marrow Transplant and Cellular Therapy Program.
Blood & Marrow Transplant
Blood and marrow transplants
(also called hematopoietic stem cell transplants) can treat aggressive leukemias, bone marrow failure, inherited immunodeficiency, and other autoimmune and genetic diseases, such as multiple sclerosis. The blood and marrow used in the transplant can come from your own body (autologous) or
a donor (allogeneic).
Autologous Stem Cell Transplant
In an autologous stem cell transplant, the recipient’s stem cells are flushed out of their bone marrow using injections, then taken from the bloodstream and frozen until needed. When enough stem cells have been collected and frozen, the patient receives very high doses of anticancer chemotherapy for several days, followed by an intravenous infusion of the thawed stem cells.
Diseases Treated by Autologous Stem Cell Transplant
Autologous stem cell transplant is most often used to treat multiple myeloma and aggressive lymphoma that has relapsed after initial treatment. In each condition, a transplant is the standard of care and is not considered experimental. Certain severe cases of autoimmune diseases can also benefit from
Autologous transplants, particularly in multiple sclerosis and in some non-hematologic cancers, such as neuroblastoma and poor-prognosis testicular cancer. Autologous transplants are also used for CAR-T therapy (explained below) for many blood cancers or gene therapy for diseases like sickle cell anemia.
Allogeneic Stem Cell Transplantation
In an allogeneic stem cell transplant, high doses of anti-cancer chemotherapy are given to the patient before receiving an intravenous infusion of hematopoietic stem cells from a donor. In this case, the donor must closely match the patient’s genes on human chromosome 6, called the HLA gene cluster. Sometimes, half-matched donors (called haplo-donors) can also be considered. The donor can be a brother, sister, parent (in some cases), or an unrelated donor. Patients can access more than 41 million potential donors worldwide through the National Marrow Donor Program, or the donor cells can be taken from the placenta blood that has been stored for this purpose (a cord blood transplant).
Diseases Treated by Allogeneic Transplant
Allogeneic stem cell transplant is used primarily to treat acute myeloid leukemia, a related disorder called myelodysplasia, and acute lymphoblastic leukemia. It is also used for bone marrow failure syndromes, more commonly aplastic anemia. Less often, allogeneic hematopoietic transplantation is used to treat other leukemias and sometimes aggressive lymphomas.
CAR-T Therapy
Chimeric-antigen receptor T cell therapy (CAR-T) is a groundbreaking treatment where a patient’s T-cells are extracted from the bloodstream, similar to an autologous transplant. Unlike autologous stem cell transplants, no injections are required since these cells normally circulate in the bloodstream. These immune cells are then activated outside the body as cancer killers. The patient is given lymphodepleting chemotherapy to prevent the body from rejecting the CAR-T cells, and the cancer-killing T-cells are re-infused back into the body through a vein.
Diseases Treated by CAR-T
Currently, CAR-T therapy is FDA-approved for treating certain leukemias, relapsed or refractory acute lymphoblastic leukemia, lymphomas like large B cell lymphoma, follicular lymphoma, mantle cell lymphoma, and relapsed multiple myeloma.
Gene Therapy
Gene therapy is an innovative technique that uses genetic material to treat or potentially cure diseases. These therapies can work by replacing a disease-causing gene with a healthy copy of the gene, inactivating a disease-causing gene that is not functioning properly, or introducing a new or modified gene into the body to help treat a disease. With gene therapy, a patient’s blood, bone marrow, or stem cells are collected, then these cells are modified in a lab to contain the desired genetic material using viral vectors or advanced gene editing technologies like CRISPR before injection back into the patient’s body.
Diseases Treated by Gene Therapy
Gene therapy is currently FDA-approved for sickle cell disease, thalassemia, and rare inherited and congenital disorders like muscular dystrophy and spinal atrophy.