Innovators 2001 - CATHERINE VERFAILLIE
By Rachel K. Sobel
Three zipperlike scars frame Catherine Verfaillie's knee, and that's where the story of her scientific career begins. As a 6-foot, 1-inch-tall high school student in Belgium, Verfaillie hurdled and high-jumped to stardom, winning the junior national pentathlon. She went to college to be a gym teacher, with hopes of also training for the Olympics. But a few months after she enrolled, Verfaillie's knee buckled, rupturing tendons, ligaments–and her athletic dreams. Crestfallen, she nonetheless swiftly moved on–"I decided overnight"–and switched her studies to another field that intrigued her: medicine.
Now she's hurdling to a new form of stardom, as a leader in the hot new field of stem-cell research. The 43-year-old Verfaillie, a hematologist-oncologist at the University of Minnesota, is one of the few people in the world to have coaxed adult stem cells to differentiate and grow into a host of different tissues, a feat many thought impossible. Her work could lead to a revolution in the way diseases are treated. Verfaillie says: "What I envision is that you could go to the doctor and take a vial of new heart muscle cells off the shelf to repair diseased heart tissue."
Her work with adult cells is particularly significant because it bypasses the thorny ethical issues surrounding the use of stem cells derived from human embryos. Just two years ago, scientists announced that they had isolated these master cells, which are capable of developing into any one of the body's 210 different tissues. Since then, many researchers have started nudging the cells toward a variety of fates, with hopes of one day growing tissues or even replacement organs for ailments such as heart disease and Parkinson's. But antiabortion activists and conservative members of Congress have condemned the use of embryonic tissue in research and have threatened to ban it.
Enter Verfaillie, who along with a few other leading investigators around the world has illuminated an entirely new avenue of thought–using stem cells derived from adults. Three years ago, Verfaillie received a phone call from a pediatrician who wanted a supply of bone and cartilage to treat a rare genetic disease in children. Verfaillie advised her graduate student Morayma Reyes to grow the tissues from stem cells found in adult bone marrow, as usual. But she said to make sure to leave out one ingredient, cow's serum, because its proteins could react with children's immune systems. The result they found in the dish was bizarre: There were not only bone and cartilage cells but also endothelial cells, which line blood vessels.
Favorite recipes. After seeing that, Verfaillie had a hunch that such mother cells could be made to differentiate into even more kinds of cells, given the proper stimulation. The trick was figuring out how to mimic the body's own environment for growing particular tissues. To that end, Verfaillie–an "extraordinary" cook, according to one student who has tasted her homemade quiche lorraine and fish stew–has successfully concocted several recipes. By last summer, after months of trial and error, she had figured out how to transform stem cells derived from the bone marrow into nerve, liver, and heart cells. Her eyes light up: "You could actually see the heart cells beating in a dish."
The intense and careful Verfaillie, who usually rises at 3:45 a.m. to head to work, is now retesting the cells she has cooked up to confirm their identities. "She will check and recheck," says John Wagner, a director of clinical research at the University of Minnesota. "Some scientists will test two or three times; Catherine will come back and say she's done it 26 times." Soon Verfaillie will transplant the cells into mice with illnesses such as multiple sclerosis and Alzheimer's disease to see if the cells will travel to damaged areas and repair them.
hasn't gone unnoticed, even though it has yet to be published in a major
scientific journal. Last year, after the University of Chicago tried to
woo her away, Minnesota countered with leadership of a brand-new Stem Cell
Institute, which includes lab space for 13 new scientists. That will make
it possible for her to hire an embryonic-stem-cell researcher, so that
she can compare the differences between embryonic and adult cells. Raised
in a devout Catholic family, Verfaillie objects to efforts to ban research
with embryonic or fetal tissue, because it's impossible to tell yet which
will be more useful to medicine: "It's way too early."