Blood and Marrow Transplant and Cellular Therapy Program
Overview
UConn Health is at the forefront of healthcare and medical advancement, and we have access to many leading-edge treatments for some of the most prevalent and debilitating diseases. For decades, bone marrow transplants have been used to treat aggressive cancers, but after 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 and Marrow Transplant
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To schedule an appointment or for more information, call: Nurse Navigator: 860-308-4483 Email: BoneMarrowTransplant@uchc.edu
A blood and marrow transplant (also called hematopoietic stem cell transplant) is a medical procedure used to treat a variety of diseases. Blood and marrow transplants can be used to 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 a donor (allogeneic) or your own body (autologous).
Autologous Stem Cell Transplant
In an autologous stem cell transplant, the recipient’s stem cells are flushed out of their bone marrow using injections. These stem cells are taken from the bloodstream and then 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. Autologous stem cell transplant is also used to treat aggressive lymphoma that has relapsed after initial treatment, especially late relapse. In each of these conditions, transplant is the standard of care and is not considered experimental. Autologous transplants can benefit certain severe cases of autoimmune diseases, 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 in 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 blood of a placenta 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. Allogeneic stem cell transplant 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 (through genetic engineering in special labs) outside the body as cancer killers. The patient is given lymphodepleting chemotherapy to prevent the body from rejecting the CAR-T cells, and the genetically activated, powerful 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 the treatment of 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 using genetic material to treat or potentially cure disease. 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. For this, a patient’s blood, bone marrow, or stem cells are collected. Then, in a lab, these cells are modified 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.