Researcher hopes to cure babies before birth
Nam D. Trần studies ways to diagnose, and fix, defects while still inside the mother’s womb.
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 Nam D. Tr?n envisions a world in which life-threatening or life-shortening illnesses such as cystic fibrosis and thalassemia are cured in babies before they leave the safety of their mothers’ wombs. That’s why Tr?n, a researcher at the University of California, San Francisco’s Department of Reproductive Services, conducts animal research that focuses on helping cure diseases when babies are still in the womb. This work, which he tests using mainly mice, definitely has human applications, he said. “You try to diagnose the needs of a baby still inside a woman. It’s not so much that there is anything wrong at the moment, but after birth, the baby will develop problems like thalassemia, which is common in Asians and African Americans, and cystic fibrosis, which usually happens in Caucasians,” said Tr?n, who left Vi?t Nam with his parents and sister in 1984, arriving in the United States in 1987. “It’s all based on family history. If people have a disease on both sides of the family,” a large possibility exists that the as-yet unborn baby might develop the disease later in life, he said. Cystic fibrosis is a chronic and progressive disease that causes mucus to build up and clog passages in many of the body’s organs, primarily the lungs and the pancreas. It can lead to serious breathing problems, malnutrition, growth and development disorders. Thalassemia is an inherited blood disorder that causes the body to produce less hemoglobin. Low hemoglobin can result in anemia, and severe anemia can damage organs and eventually lead to death. Doctors use prenatal tests to conduct genetic analyses that help them determine which babies are candidates for the suspect diseases, said Tr?n, who also is an obstetrics and gynecology resident at the UCSF Medical Center. If tests indicate that the disease is likely, the theory is that the baby’s genes could be manipulated in such a way as to eliminate any chance that the disease could take hold after the child is born, he said. “Usually, we can diagnose them pretty early, at 13 weeks (of gestation) on to about 20 weeks or so. It is a new way of treating disease. We want to treat early,” but all work is experimental. Traàn’s “ability to construct questions in ways that not only produce results but produce results that have the possibility of practical application is tremendous,” said Emily Norland, an ob/gyn resident at the University of Washington Medical Center in Seattle, who said she considers Tr?n both a colleague and mentor. “His work is complex in ways that belie the duration of his career.” In part because of his research and also because of his interest and work in the ob/gyn field, Tr?n “brings to women’s health an incredible background of basic science knowledge with really sharp clinical skills and experience,” said Peter Klatsky, an ob/gyn resident at the UCSF Medical Center. While Tr?n uses mice in his studies, many of his colleagues experiment on larger animals such as dogs, cats, sheep, ferrets and monkeys. Even though he is testing for diseases, such as thalassemia, which are found in humans, “we can do that (testing) in mice, sheep, monkeys. Mice are the easiest and the cheapest and genetically are similar to humans,” said the researcher, whose work includes using hematopoietic stem cells that are found in bone marrow. “You can knock out certain genes in the baby (mouse while it is still in the womb). It’s all about stem-cell research that we use in animals. My part is that I do a correcting gene or I can isolate disease and have stem cells that are good injected in the baby,” which then is born healthy. “Like everyone has heard of the Bubble Baby, with a genetic defect in the X gene for ADH (a normal body hormone). He’s healthy inside the womb, but when born, he has no immune cells, so has to live in a germ-free bubble environment. If not, he would get sick and would die eventually,” he added. Sometimes, he and others in the field work with humans, but only on a case-by-case basis. They diagnose a potential future problem for an unborn baby and they transfer stem cells, thus allowing a healthy birth. Mothers, he said, “don’t have to do all of this. They can have an abortion. But some people don’t believe in abortion. Treating the disease inside is more effective and cheaper for society. And, it’s safer for the patient, mentally,” he said. “If at one point you can prevent the disease from happening, people would be better off mentally and in productivity. That is the bottom line.”
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