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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 genetic disorders
Detecting and defeating cancer stem cells
Modeling disease
Discovering new drugs
These goals are both broad enough to encompass a variety of research initiatives yet focused upon the central mission of Boston Children’s Hospital. Three strategies have been developed to meet these goals. First, researchers seek to develop basic methods for creating customized stem cells that will permit these to be turned into any tissue in the body. Doing so provides the greatest degree of latitude to researchers and physicians working with children battling diseases or medical conditions. Second, researchers seek to apply those approaches to the diseases most likely to yield the first breakthrough treatments. Leukemia has been identified as a promising area, as research has been conducted in this area and the method for delivering the cells—blood transplantation—has already been established and is in use. Third and last, stem cells and other technologies developed at Boston Children’s Hospital are to be made available to colleagues worldwide in order to speed progress on a vast array of diseases. Doing so will permit rapid progress toward alleviating the suffering of the many children facing a variety of diseases and medical conditions.
To date, the progress achieved by Boston Children’s Hospital researchers has been impressive. George Q. Daley, for example, has worked to translate findings from stem cell biology into improved therapies for children facing genetic and malignant diseases. His laboratory has worked with human cell culture–based and murine models of human blood disease and has created customized stem cells to treat genetic immune deficiency in mice. Felix Engel and Mark Keating have also achieved breakthroughs for those children facing cardiovascular problems. Engel and Keating were able to have adult heart-muscle cells divide and multiply in mammals, which many view as the first step in regenerating heart tissue. In order to attack cardiovascular disease, Engel and Keating are now investigating whether this technique can improve heart function in animals suffering from cardiac problems.
Benowitz and members of his team have discovered a naturally occurring growth factor called oncomodulin. Oncomodulin stimulates regeneration in injured optic nerves. This research holds great promise for those hoping to treat blindness caused by optic-nerve damage and also has promise for achieving similar regeneration in the spinal cord and brain.
Boston Children’s Hospital has supported these and other research initiatives by providing first-rate facilities and resources to all involved in stem cell research. With nearly 700,000 square feet of research space, Boston Children’s Hospital has the largest research center located at a pediatric medical center. Employing over 1,100 scientists, Boston Children’s Hospital is the home to nine members of the National Academy of Sciences. Together with researchers at the Harvard University Medical School and the Howard Hughes Medical Institute, scientists at Boston Children’s Hospital continue to work on the cutting edge of stem cell research.
Stephen T. Schroth
Towson University
See Also: Clinical Trials, U.S.: Spinal Cord Injury; Harvard University; Howard Hughes Medical Institute; Rat Models to Study Stem Cells.
Further Readings
Blackburn, S. Ethics: A Very Short Introduction. New York: Oxford University Press, 2009.
Goodstein, J. R. Millikan’s School: A History of the California Institute of Technology. New York: W. W. Norton, 2006.
Park, A. The Stem Cell Hope: How Stem Cell Medicine Can Change Our Lives. New York: Plume, 2011.
Scott, C. T. Stem Cell Now: A Brief Introduction to the Coming Medical Revolution. New York: Plume, 2006.
Slack, J. Stem Cells: A Very Short Introduction. New York: Oxford University Press, 2012.