Bone Marrow Transplantation for Non-Malignant Disorders
Although conventional bone marrow transplantation has been shown to cure hemoglobinopathies and enzyme deficiencies, there is still debate as to whether the morbidity and mortality associated with lethal conditioning can be justified. Therefore, the preferred approach to treating non-malignant diseases is mixed chimerism. A couple of advantages of mixed chimerism are that individuals with mixed chimerism are relatively resistant to GVHD and that conditioning individuals only partially so that endogenous hematopoiesis can occur if graft failure results.A problem with the conventional bone marrow transplantation is that there are few individuals who HLA identical bone marrow donor. Using a matched unrelated donor, the incidence and severity of GVHD and the risk of graft failure is significantly increased. Up to know, BMT using unrelated donors has been limited to fatal diseases for which no adequate treatment is currently available.
Since most individuals will not have an HLA identical donor, we will use partially matched donors either related or unrelated. Partially ablative conditioning will minimize the risks of graft failure in individuals without HLA identical sibling donors. Also, mixed chimerism will be an additional protective measure against GVHD.
Recently, we have identified a bone marrow cell that facilitates engraftment of allogeneic bone marrow. When we purify and transplant this "facilitating"cell (FC) and hematopoietic stem cells (HSC), we optimize the conditions for engraftment yet attempt to minimize morbidity and mortality of BMT by using nonlethal conditioning and decreasing the incidence and severity of GVHD.
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