While most people at the University have ambitious career goals and aspirations, one six-year-old Colorado girl has come to the University with one small goal — to be able to ride a school bus.
Molly Nash was born with two holes in her heart and without thumbs, hip sockets and part of her brain. Several reconstructive surgeries later, the effects of an often-fatal genetic disorder — Fanconi anemia — still linger.
Later this month, University doctors will use umbilical cord blood from her newborn brother, Adam, for a transplant physicians say is Molly’s best chance for survival.
Although cord blood transplants are nothing new, the high-tech genetic testing researchers are using to help Molly reach her dream is a first.
Researchers from Chicago’s Reproductive Genetics Institute conducted a pre-implantation genetic diagnosis on embryos from Molly’s mother, Lisa. The tests determined whether the embryos were free of the genetic disease and if the embryo would be a donor match for Molly.
Lisa, a neo-natal intensive care nurse, heard about the institute’s research and contacted them about helping her family.
“I was handed this beautiful girl with a potentially fatal disease,” she said. “We decided she was going to beat the odds. One way or another Molly is going to outlive us.”
The genetic testing is believed to be the first used to determine whether or not a child will have a disease and be a donor match.
Fanconi anemia is a progressive disease, resulting in bone marrow failure in all patients and leukemia in a significant portion. When the marrow fails, the blood no longer contains enough red blood cells to carry oxygen, white blood cells to fight infection or platelets to help the blood clot.
Both Molly’s parents are carriers of the disease. Although the parents remain completely healthy, they have a 25 percent chance of passing the disease on to their children.
Since the odds of an embryo matching tissue perfectly and being disease-free are slim, researchers created 15 test-tube embryos using the parents’ sperm and eggs.
“It’s easier for couples to conceive multiple children in a test-tube,” said Charles Strom of the genetics institute. “One cell was taken from each embryo, tested, and one healthy embryo that matched as a donor was transferred to the mother.”
That embryo developed into Adam, arriving Aug. 29 to save his big sister. His umbilical cord was saved and shipped to the University for future use.
Without the test-tube procedure, the Nashs would have needed to conceive a child on their own, wait 18 weeks to test for the disease and then possibly make a decision to abort.
“That was not an option for us,” Lisa said.
Doctors say having a related donor match is paramount. The transplant success rate increases to 85 percent with related donors compared to the 40 to 50 percent success rate of non-related donors.
Using umbilical cord blood rather than bone marrow also saves three-week-old Adam from enduring surgery to save his sister. Instead, all he had to do was be born.
But now comes the hard part for Molly.
Fairview-University Medical Center admitted her yesterday to begin treatment with five days of chemotherapy and radiation. Hair loss and flu-like systems are only a few of the side effects Molly will endure.
University cord blood specialist John Wagner said the treatment will destroy Molly’s bone marrow to make way for the injection of its replacement — Adam’s cord blood.
Surgery is set for Sept. 26.
“Molly will be closely monitored for side effects of the transplant. If all goes well, she will be back home within three months,” Wagner said.
Although some are calling the high-tech genetic testing a breakthrough, Molly’s father Jack said after watching his daughter go through eight surgeries in six years he’s not trying to make history.
“We really don’t look at it as being pioneers,” he said. “We just want a healthy baby.”
The procedure should prevent Molly from contracting leukemia, but Wagner is quick to point out that it is not a cure-all. Even if the surgery is successful, Molly is still susceptible to several cancers because of her condition.
Lisa said at least the transplant gives Molly a fighting chance.
“Six years ago there was no chance for her,” she said. “But now there are new procedures. Maybe six years from now there will be a cure for cancer.”

Craig Gustafson welcomes comments at (612) 627-4070 x3222. He can also be reached at [email protected].