ABSTRACT

The coming years, the early 21st century, promise to be an incredibly exciting era for bone marrow transplantation (BMT) and specifically pediatric BMT. The scientific advances in stem cell biology, transplantation immunology, and gene transfer technology will certainly lead to a wave of clinical trials that will boost our armamentarium to treat childhood disease. Of all physicians, these advances will be most relevant to specialists in pediatric BMT, physicians who focus on the processing and transplantation of stem cells, as well as the delivery of immune-mediated therapy. As biologists unlock the full potential of marrow stem cells, hematopoietic, mesenchymal, and other, as yet undescribed populations, as well understand the fundamental mechanisms of immune effector cells, the clinical transplant specialist will be called upon to develop therapy for a wide variety of disorders that will continue to expand the spectrum of diseases we treat beyond the traditional bounds of hematology-oncology. For example, we have cared for children with metabolic storage diseases as well as osteopetrosis over the last two decades, because we recognized that the defective cell in these diseases originates from the hematopoietic stem cell (HSC).