Stem-Cell Researcher Fights to Find Diabetes Cure
Stem-Cell Researcher Fights to Find Diabetes Cure
When Douglas Melton's son, Sam, was 6 months old, he suddenly became severely ill. Not initially knowing what was wrong, doctors feared he might not survive. (Pictured: Douglas Melton of the Harvard Stem Cell Institute)
Sam's blood and urine showed high levels of glucose, and he was diagnosed with type 1, or juvenile diabetes, a disease that can be treated with multiple daily insulin injections but is still without a cure. Melton's daughter, Emma, was later diagnosed with the same illness.
A personal and professional crusade was born. A molecular biologist and co-director of the Harvard Stem Cell Institute, Melton goes way beyond the scientific pursuit of a researcher in his work. He is on a moral mission - both as a scientist and a father.
"My dream, or my desire," he said, "is to find a cure for type I diabetes so that my children, and the millions of other children and adults with diabetes, will live healthy, normal lives."
Stem cells are the body's most basic cell type from which all other cells and tissues arise. It is believed that if one understands the transformation from stem cell to specialized cell, such as an insulin-producing cell, then one may eventually be able to fix the damaged cells and cure diseases.
But human embryonic stem cell research is no stranger to moral conflict. Its opponents believe the embryos from which stem cells arise are human lives being sacrificed for science. The Bush administration has placed strict limitations on their use in research.
Melton disagrees. "Because of political and religious disputes surrounding embryonic stem cell research," he said, "people tend to think of the field as being entirely focused in that area."
For scientists, the question of morality is most crucial when they are prevented from using the best tools available to help alleviate human suffering.
And for Melton, that suffering is very personal.
A Father's Quest
"Insulin is not a cure for diabetes," Melton said in testimony before the United States Senate. "It is merely life support."
Until his son's diagnosis, Melton had focused his career as a biologist on the cellular development of frogs. After his son's diagnosis, his life's work changed forever.
Melton's lab began studying the pancreas, the organ in the body that makes insulin and is the root of the cause of type 1 diabetes.
Melton and his Harvard colleagues want to know how the pancreas develops and how it makes insulin. By understanding the pancreas, they hope to discover what goes wrong in diabetes and how to fix it on the cellular level. To do so, they study human embryonic stem cells.
"Studying stem cells and how they replenish and repair our bodies will provide a deep understanding of the biology that keeps us healthy," Melton said. "On the clinical side, I hope what will emerge from this work is far greater insight into chronic, often lethal diseases that have stumped us up to now."
Because of the limitations the federal government has placed on publicly funded stem cell research, Melton developed 31 private stem cell lines, funded by the nonprofit Howard Hughes Medical Institute, that he and other scientists are able to use for their research without government restrictions. He makes these lines available for free to any legitimate researcher who wants to use them.
In addition to diabetes, it is hoped that stem cell research will be the key that unlocks the mysteries of other serious diseases, such as Alzheimer's, Parkinson's, amyotrophic lateral sclerosis (also known as ALS or Lou Gehrig's disease), sickle-cell anemia, leukemia and other types of neurodegenerative, blood and heart diseases and cancers.
Spearheading Cutting-Edge Research
In a new, privately funded effort, Harvard Stem Cell Institute researchers - from Harvard and its affiliated research hospitals - and the Boston IVF fertility provider and Columbia University, have joined forces to begin the next major set of stem cell experiments aimed at understanding and curing disease.
The process, known as somatic cell nuclear transfer or SCNT, produces disease-specific stem cell lines. This is done by replacing the nucleus of a donated egg with the nucleus from the cell of an individual with a particular disease, which potentially will produce a line of stem cells containing the genes for that disease.
Melton and Kevin Eggan, an assistant professor of molecular and cellular biology at Harvard, are using SCNT to study type 1 diabetes.
"I believe we'll make this progress by developing insights into how disease-specific stem cells differ from normal stem cells," Melton said. "And it goes without saying that I hope that once we can enhance our understanding of these processes, we will find ways to develop treatments and cures for a whole range of diseases."
Already, Melton's work has had an impact in the world of medicine. "He has been an important contributor to the field of brain differentiation, showing that an isolated stem cell will tend to become a nerve cell by default," said Dr. Curt Freed, director of the Neuroscience Center at University of Colorado Health Science Center.
Colleagues say Melton also has been instrumental in forming collaborations that bring together the most promising researchers in the field to push the limits of biomedical exploration and unlock the mysteries of health and disease.
"An absolutely critical need for the field of stem cell biology at this point is a group of leaders capable of finding consensus in a complex, divisive and politically charged environment," said Dr. Daniel Salomon, of the department of molecular and experimental medicine at the Scripps Research Institute in La Jolla, Calif.
"Around these leaders will coalesce centers for stem cell research. & These centers will be the focus of philanthropy, collaborations with emerging biotechnology companies and a critical mix of institutional and state government funding," Salomon said. "These will be the drivers that enable the next round of advances in stem cell research & a perfect example of such a leader is Doug Melton."
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