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Releases Packard
Children's News
Children's Fund
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The Promise of Regenerative MedicineBY MARK SHWARTZ WINTER 2007 -- Imagine a time when laboratories can grow healthy hearts for children in need of cardiac transplants. Or picture a future in which pediatricians treat severe skin diseases by grafting genetically engineered skin cells onto the patient’s body. And what if doctors had the ability to prevent the formation of cleft palates and other birth defects while the baby is still in the womb. These distant scenarios are coming closer to reality, thanks to pioneering research in stem cells and regenerative medicine now under way at Stanford University and Packard Children’s Hospital.
''Stem cell biology and regenerative medicine are fundamental to our understanding of human development,'' says Philip A. Pizzo, MD, the Carl and Elizabeth Naumann Professor and dean of the Stanford School of Medicine. ''How cells, organs, and systems are formed and regulated, how they are sustained and overcome injury with repair, and how they might go awry in the case of cancer are among the fundamental questions we are now addressing.'' This promising new field also offers many potential benefits for pediatric medicine. ''Congenital malformations of the heart, lungs, and nervous system are major causes of disease and death during the first years of life,'' Pizzo says. ''The knowledge gained from stem cell biology and regenerative medicine may help us better understand how some of these abnormalities arise, and could lead to new ways to repair damaged organs and systems--even during fetal development.'' To illustrate the university’s commitment to this emerging field, Pizzo points to the creation of the Stanford Institute for Stem Cell Biology and Regenerative Medicine in 2005. The Institute is led by Stanford’s Irving L. Weissman, MD, the Virginia and D.K. Ludwig Professor for Clinical Investigation in Cancer Research and professor of pathology and of developmental biology. ''Many genetic diseases of tissue and organ failure begin in children,'' Weissman says. ''Therefore, children are the most likely recipients of stem cell or regenerative medicine therapies.'' An internationally recognized leader in developmental medicine, Weissman was the first scientist to isolate animal stem cells, in the 1980s. Today, he and his Institute colleagues are expanding that groundbreaking work to include human embryonic stem cells, which have the potential to regenerate tissues and organs, and to cure cancers and genetic diseases such as Alzheimer’s and Parkinson’s. In November 2004, Stanford and other medical research institutions in California received a tremendous boost when voters passed Proposition 71, providing $3 billion over 10 years for statewide stem cell research. So far, the Stanford Institute for Stem Cell Biology and Regenerative Medicine has received more funding than any other institution--nearly $29 million, of which more than $1.8 million has been awarded to three faculty members in the Department of Pediatrics:
''One way to enhance adult and embryonic stem cells so that they are resistant to viral infections is through genetic modification,'' Weissman says. ''Mark Kay is one of the leading investigators in the world in this area. Ken Weinberg is a world leader in finding out how certain types of blood stem cells can regenerate the immune system rapidly and effectively. Julien Sage is a promising young investigator studying cancer gene pathways in cancer stem cells.'' A great deal of fundamental research will be needed to make the promise of stem cell and regenerative medicine therapies a reality for patients, Pizzo adds. ''This will require time, careful study, and, of course, resources,'' he says. ''But the extraordinary research at Stanford and the plethora of biotechnology in our surrounding communities will one day provide unique opportunities to translate laboratory discoveries into therapies for children who truly need them.'' |
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