Doctors announce girl’s recovery after innovative transplant

by Tess Langfus

A 6-year-old girl’s future was determined Wednesday by doctors at a press conference in the University Cancer Center.
Her parents sat with hands folded and cautious smiles as University specialist John Wagner revealed whether or not the surgery had been successful. On Sept. 26, University surgeons performed a life-altering cord-blood transplant on Molly Nash, using her newborn brother’s umbilical blood.
“We have a success,” said Wagner, who performed the delicate surgery. “This was the big moment today of the culmination of many years of research.”
Fanconi anemia — a progressive genetic disease resulting in bone marrow failure in all patients — has plagued Molly from birth. She has overcome operations to restore her hip sockets, manages without thumbs, and lives with two holes in her heart and portions of her brain missing.
With eyes full of tears, elated parents Jack and Lisa Nash described their wonderment at their daughter’s seemingly instant recovery.
Expecting a slow recovery with frequent bouts of nausea from her chemotherapy treatments, Lisa said she was amazed that Molly was ready to dance and play like a normal 6-year-old when weeks before she had little energy for any activity.
And as Molly cradled Adam on her lap, she insisted to her parents she was well enough to take care of her little brother.
“That was when we knew that was the right thing to have done,” Lisa said. “We are breathing a sigh of relief.”
The Nashs plan to tell Adam when he is older that he helped to save his older sister’s life.
Wednesday’s test results revealed that Adam’s bone marrow cells are surviving within Molly’s body, a good indication that the disease could be combatted.
Wagner noted that, for the first time in years, Molly is producing white blood cells that will help her fight bacteria.
Normally, the disease causes marrow to fail and blood no longer contains enough red blood cells to carry oxygen, white blood cells to fight infection or platelets to help blood clot.
While few deny the favorable outcome of the umbilical cord blood transplant, there are ethical issues behind the means of acquiring a disease-free donor for Molly.
Rather than search for a successful bone marrow match for Molly, her parents opted to create one.
Through in vitro fertilization, 12 embryos were produced from Lisa’s eggs and Jack’s sperm. After the embryos were allowed to develop to a certain stage, they were genetically tested to determine if they were affected by the disease. A healthy one was then implanted into Lisa’s uterus for natural development.
It is the genetic testing that is causing ripples of concern in the public arena.
In an ethics conference to be held Nov. 17, University professor and program director Jeffrey Kahn will consider the implications of such research.
“We have to be careful that children do not get used as an end of the means for their parents,” Kahn said. “Do you want to do this for the sake of the children, or for the benefits of the parent?”
Molly’s parents defend their decision to use embryo testing to save their daughter’s life.
“We have a sick child,” Lisa said. “We chose to use this technique and it was ethically correct for us.”
They went public with their family’s trauma in order to increase public acceptance of embryo research.
Embryo testing could benefit people with diseases such as sickle cell anemia and other genetic disorders by identifying acceptable donors from either relatives or nonrelatives, Wagner said.
Contrary to common misconceptions, Wagner said, there was no DNA manipulation and the embryos were tested only to identify the presence of the debilitating disease.
However, he added genetic research could have the potential to decrease the number of abortions performed because of genetic disease.
For the Nashs this research offers them the chance to have more children in the future. The remaining disease-free embryos have been frozen and will be stored indefinitely until they are chosen to be developed into another child.
While doctors continue to monitor Molly’s progress, Wagner said there is a strong probability the little girl can be cured of the disease.
“The first hurdle has been hit head-on,” Wagner said.
Yet Molly’s long-term future is not free of potential turmoil.
While few people with Fanconi anemia survive beyond childhood, some of those who do survive are stricken by other types of cancer when they reach their 20s, Wagner said.
Although Molly may be freed from any immediate threat, she might have to fight other diseases such as head, neck or cervical cancer when she reaches adulthood.
But for now, the primary concern is for Molly’s health and her acceptance as a normal, active 6-year-old girl.
Because the disease causes growth hormone deficiency, Molly is now the height of a 4-year-old. Wagner said she will soon undergo hormone injections to promote her growth.
Molly is expected to be released from the hospital within a week, but she will continue outpatient visits for the next several months.
The family could return to their hometown of Englewood, Colo., as early as December.
Jack and Lisa said as they watched Adam’s blood rhythmically drip through to Molly’s veins, their decision to utilize genetic research to benefit their suffering daughter was confirmed.
“It bonded them together,” Lisa said.
Tess Langfus welcomes comments at [email protected]