Blood Disorders and Stem Cells
Our bone marrow has the job of creating all of our blood cells: red blood cells to carry food and oxygen around the body, white blood cells to fight disease, and platelets to stop bleeding. Malfunctioning bone marrow, which can produce too few or too many cells, is the cause of many serious diseases of the blood.
While stem cells are currently used to treat a variety of blood disorders, researchers at Boston Children’s Hospital are committed to exploring their potential for development of new treatments. Stem Cell Program Director Leonard Zon, MD, has discovered a promising and previously unrecognized way to use the drug PGE2 to boost blood stem cell production in patients undergoing treatment for leukemia or lymphoma. The work started with studies in zebrafish and extended to mouse marrow transplants. Phase I clinical trials using PGE2 started in May of 2009.
Explore the world of zebrafish research by touring the Zon Lab in the following video:
Other significant blood disease research underway at Children’s:
Scott Armstrong, MD, PhD, an affiliate member of the Stem Cell Program at Boston Children’s Hospital, works with leukemia stem cells, which are the subset of cells in leukemia that are responsible for the development and continued growth of the disease. Leukemia stem cells are the critical cells that must be eradicated in order to cure leukemia. READ MORE.
George Q. Daley, MD, PhD, and other researchers at Children’s are dedicated to turning embryonic stem cells from mice into blood stem cells that could correct a blood disorder. This work is setting the stage for similar studies using human cells.
Children’s is already a leader in the area of bone marrow transplant, the first example of stem-cell-based therapy in which hematopoietic (blood-forming) stem cells from a donor are used to replace a patient’s diseased marrow. At Children’s, we currently use hematopoietic stem cell transplant to treat the following blood disorders, in close collaboration with the Dana Farber Cancer Institute:
- amegakaryocytic thrombocytopenia
- cyclic neutropenia
- dyskeratosis congenital
- Shwachman-Bodian-Diamond syndrome
- Pearson syndrome
- severe congenital neutropenia
- thrombocytopenia absent radii
- aplastic anemia
- Diamond-Blackfan anemia
- Fanconi anemia
- sickle cell disease