Each year, more than 3,000 people in Canada and nearly 30,000 people in America are diagnosed with diseases for which a stem cell transplant can be a cure. The majority of these people suffer from some form of leukemia, the most common type of cancer in children. Other diseases that can be treated with a stem cell transplant include lymphomas and other cancers, immune disorders, and bone marrow failure.
In 2000, 6-year-old Molly Nash was thrust into the media spotlight when she was successfully treated with transplanted stem cells from her brother’s umbilical cord blood. Molly was born with a rare genetic disease called Fanconi anemia, which causes the body’s normal production of bone marrow to fail. When Molly’s parents learned her chance of survival would nearly double with a perfectly matched stem cell donor, they decided to use a new procedure that could identify a healthy, genetically matched embryo produced through in-vitro fertilization. The Nash’s baby was the first in the world conceived by this procedure to provide a stem cell donor for a sibling.
"Parents are coming to us from all over the world with many kinds of rare genetic diseases," says Oleg Verlinsky of the Reproductive Genetics Institute in Chicago, which did the immune system screening on Adam Nash before his birth. Since then, RGI has performed more than 250 such screenings, Verlinsky says, for children whose birth led to the treatment of an older sibling.
"Molly Nash is a wonderful story," Verlinsky says. "We worked so hard on her assay. The little girl was dying. Most patients come to us just for screening, but cases where parents come to you with an already-sick child are very hard."**
The Nash’s story is just one example of the long and successful history of stem cell transplantation at the University of Minnesota. In 1968, University physicians performed the world’s first successful stem cell transplant on a 4-month-old boy suffering from an immune disease. He is now a healthy adult, living in Connecticut, and the proud father of twins.
Stem cell transplants are also referred to as bone marrow transplants (using stem cells from bone marrow) or umbilical cord blood transplants (using stem cells from umbilical cord blood). Unlike an organ transplant that involves major surgery, a stem cell transplant is similar to a blood infusion. The stem cells that are transplanted produce all blood cells in the human body red blood cells, white blood cells, and platelets and are necessary for creating healthy bone marrow.
The highest concentration of stem cells is found in the bone marrow, a spongy tissue that fills the insides of the bones. Stem cells also are present in the bloodstream and in blood collected from a newborn’s umbilical cord. My colleague John Wagner, director of the University of Minnesota Umbilical Cord Blood Transplant Program, was part of the medical team that made medical history in 1991 by performing the first umbilical cord blood transplant in the world for leukemia. He also performed Molly Nash’s transplant.
Some bioethicists, such as former bioethics council chief Leon Kass of the American Enterprise Institute, raised worries those children born from such procedures would feel unloved, if they see themselves as exploited.**
"People are certainly entitled to their opinions. But we were doing what was best for our family," says Lisa Nash. She has become an advocate for cord-blood banking from newborns as a result of her experience. "I'd urge people to really think about it early in their pregnancy." **
Norma Ramsay is a professor and Head of the Division of Pediatric Hematology, Oncology, and Blood and Marrow Transplantation at the University of Minnesota. She is also Director of the Pediatric Blood and Marrow Transplant Program and a member of the University Cancer Center.
**excerpt from USA Today 1/10/2010 Dan Vergano
By Norma Ramsay, M.D.
