then given prior to transplantation.
As opposed to allogeneic transplantation, where the GVL effect is an active part of the treatment, the autologous transplantation is carried out only in order to be able to give such intensive cytostatic therapy that the bone marrow otherwise would not be able to restore its function. There is no risk for GVHD since the cells derive from the patient’s own immune system and the reestablishment of a new bone marrow function is more rapid than in allogeneic transplantation. The need for hospital care is then shortened and there is also a lower risk of severe infections. However, in acute leukemia, the risk of relapse is higher with autologous transplantation and therefore not considered an option today. At the moment, there is rapid development where patients with autoimmune diseases such as severe multiple sclerosis is being transplanted.
Donor Register
Bone Marrow Donors Worldwide (BMDW) is an organization based in Leiden, Netherlands, that coordinates registration of now 22.5 million donors from 72 hematopoietic cell donor registries in 52 countries. The donor’s personal HLA characteristics together with other relevant data are made searchable for today’s 850 transplanting physicians and search coordinators. The chance of finding a good match depends on the patients’ origin, because some minorities still have less representation. Another factor is the genetic diversity in a population. As an example, people of African descent are more genetically diverse than people of European descent and therefore need more potential donors to find a good match.
Prognosis
The prognosis after HSCT depends on disease type, cytostatic regimen, and how well the donor matches the patient. Allogeneic transplantation is considered the only curative treatment in some forms of acute leukemia, and if the patient survives the risks of transplantation, such as infections and GVHD, there is a good chance of long-term survival. Autologous transplantation is not considered as a curative treatment but leads to prolonged life expectancy of both myeloma and lymphoma patients. There are also promising results that autologous HSCT could help patients with severe multiple sclerosis and efficiently reduce the autoimmune reactions where the patient’s own immune system otherwise rapidly would cause progressive neurological disability.
Oscar Lindblad
Julhash U. Kazi
Lars Rönnstrand
Lund University
See Also: Blood Adult Stem Cell: Development and Regeneration Potential; Blood Adult Stem Cell: Existing or Potential Regenerative Potential; Cancer Stem Cells: Overview.
Further Readings
Bone Marrow Donors Worldwide (BMDW). http:/www.bmdw.org (Accessed May 2014).
European Group for Blood and Marrow Transplantation (EBMT). http:/www.ebmt.org (Accessed May 2014).
Worldwide Network for Blood and Marrow Transplantation (WBMT). http:/www.wbmt.org (Accessed May 2014).
Boston Children’s Hospital
Boston Children’s Hospital
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Boston Children’s Hospital
One of the preeminent research facilities in the United States, Boston Children’s Hospital has been a leader in stem cell research, especially as it relates to childhood diseases and medical conditions. The Stem Cell Program at Boston Children’s Hospital works to explore and understand developments in stem cell research and to translate these into treatment and care options for the children it serves. Researchers affiliated with Boston Children’s Hospital are especially interested in how stem cell research relates to children struggling with blood diseases, diabetes, Down syndrome, Parkinson’s disease, spinal cord injury, and a variety of other conditions. Boston Children’s Hospital’s scientists have collaborated with researchers at a variety of other institutions, including Harvard University, the Dana-Farber Cancer Institute, and the Howard Hughes Medical Institute. This work will in all probability continue to keep Boston Children’s Hospital at the forefront of stem cell research.
Background
Founded in 1869, Boston Children’s Hospital entered into an affiliation with the Harvard Medical School in 1903. Long on the forefront of medical research, Boston Children’s Hospital has maintained research laboratories that have sought solutions to a host of diseases and medical conditions that negatively impact children’s lives. As early as 1891, Boston Children’s Hospital established a lab that worked to produce bacteria-free milk for children’s consumption. During the 1920s, William Ladd established a variety of procedures and protocols that made it possible to correct a variety of congenital defects such as intestinal malformations, in effect, establishing pediatric surgery as a medical specialty. By the late 1930s, Robert E. Gross had performed the first surgery to correct a pediatric cardiovascular defect, beginning the era of pediatric cardiovascular surgery. During the 1940s, Sidney Farber, who later founded the Dana-Farber Cancer Institute, successfully used aminopterin and later amethopterin (methotrexate) to treat children suffering from acute leukemia, treatment that resulted in the first partial remission of acute leukemia. In 1954, John Enders and colleagues at Boston Children’s Hospital were awarded the Nobel Prize in Physiology or Medicine for their work in identifying the polio virus, making the development of the Salk and Sabin vaccines possible.
During the 1980s, doctors at Boston Children’s Hospital conducted the first successful children’s heart transplant, and Michael O’Reilly and Judah Folkman discovered endostatin, a powerful inhibitor of angiogenesis, which showed the ability to slow the growth of some cancers in mice. By the late 1990s, research at Boston Children’s Hospital had grown to include stem cell research. Specifically, Evan Snyder cloned the first neural stem cells from the human central nervous system. This work suggested the possibility that cell replacement or gene therapy could be used by patients facing neural injury, neurodegenerative disease, or paralysis. Larry Benowitz later was able to grow nerve cells in damaged spinal cords of rats, which offered great promise to those seeking to heal spinal cord injuries.
Spinal Cord Research
Boston Children’s Hospital was an early leader in some of the studies that first suggested that stem cell therapy could potentially provide treatment for children suffering from a variety of diseases and medical conditions. When interest in such research grew during the late 1990s, Boston Children’s Hospital was at the forefront of the debate regarding the ethical nature of such work. The leaders of Boston Children’s Hospital determined that the exploration of stem cell biology represented a key way to develop new and effective treatments for diseases affecting children. As a result, Boston Children’s Hospital and its considerable research facilities dedicated themselves to pursuing studies involving stem cell research as a means of discovering such treatments.
Significant stem cell research conducted at Boston Children’s Hospital has focused on five central goals set by the institution’s board of trustees. These goals are as follows:
Creating customized treatments
Reversing
