Regenerative medicine is the practice of replacing or “regenerating” human cells, tissues and organs to establish or restore their normal function. The field includes a wide range of approaches to replacing damaged tissue or stimulating the body’s own repair mechanisms to heal organs or tissues. Examples of regenerative treatments include cellular therapies, immunomodulation therapy and tissue engineering. Some of these treatments include the use of stem cells, which can be harvested from cord blood, bone marrow and peripheral blood.
Cellular therapy is the transplantation of human cells to repair or replace damaged cells and/or tissues.
Cellular therapies use many different types of cells, including hematopoietic (blood-forming) stem cells (HSCs), skeletal muscle stem cells, mesenchymal stem cells, lymphocytes, dendritic cells and pancreatic islet cells. They are used to treat patients with many types of diseases and conditions.
The most common cell therapy is hematopoietic stem cell transplantation (HSCT) from bone marrow. It is used to treat patients with a variety of blood cancers and hematological conditions.
HSCs are present in blood and bone marrow. Individuals may donate them for their own treatment (autologous donation) or for someone else (allogeneic donation). Allogeneic transplants require the donor and patient to share matching human leukocyte antigen types.
Bone marrow, which is liquid and resembles blood, is collected through a surgical procedure that takes approximately an hour. The donor usually receives general anesthesia and a physician uses a hollow needle to remove bone marrow from the donor’s hip bones.
Peripheral blood progenitor cells are collected from the blood using an apheresis device, which separates blood into components, keeping the progenitor cells and returning the rest of the blood to the donor. Donors commonly receive a growth factor for several days prior to cell collection.
Cord blood is the blood found in the umbilical cord and placenta. It can only be collected after a baby is born and the umbilical cord has been cut. The baby’s parents can choose to donate it to a public cord blood bank, where it will be available to anyone who needs it, or store it at a private blood bank to potentially be used for personal medical needs later.
Cord blood is a biological product regulated by the Food and Drug Administration. Like bone marrow and peripheral blood, cord blood contains HSCs. At present, cord blood is approved for HSCT, which is used to treat patients with disorders of the hematopoietic system. These include malignant diseases, such as blood cancers, and non-malignant conditions, including inherited and acquired blood and immune system disorders. When cord blood is transplanted, it can serve as a graft source to restore the body’s ability to produce blood cells, typically following chemotherapy.
Stem cell transplants have been approved to treat the following diseases. This is not meant to be a comprehensive list, and new therapies are currently under investigation.
Leukemias
Lymphomas
Neuroblastoma
Myelodysplastic Syndrome
Myelofibrosis
Multiple Myeloma
Solid Tumors
Anemias
Sickle Cell Disease
Thalassemia
Diamond-Blackfan Anemia
Severe Combined Immunodeficiency
Osteoporosis
Tay-Sachs Disease
Researchers are also studying biotherapies to treat injuries, non-blood related diseases and immune system disorders, such as autoimmune diseases - including arthritis, damaged cartilage in joints, neurological disorders, spinal cord injuries and urinary complications.