One Son Offered Fighting Chance
John and Alicia Bennett learned Wednesday that only one of their two sons--both ill with a rare genetic disease--is a good candidate for an experimental transplant that could save his life.
The Northern California couple accepted the news with the same resilience with which they have handled the devastating toll of the disease on all three of their youngsters, who range in age from 3 to 6. Children with the condition usually die by their teens, after severe brain and organ damage.
Experts at Duke University Medical Center told the Bennetts that their 4-year-old son, Hunter, shows too much irreversible damage caused by Sanfilippo syndrome, in which the body lacks an enzyme needed to break down sugar molecules. As a result, he stands to gain little from an experimental stem-cell transplant using umbilical-cord blood in an effort to replace the enzyme.
However, prospects for improvement are much better if Hunter’s younger brother, Tommy, receives a transplant, doctors said. Tommy, who recently turned 3, most likely will be scheduled for the procedure next month after minor surgery to remove his tonsils and adenoids, and a nine day regimen of chemotherapy.
Their older sister, Ciara, 6, also has Sanfilippo, but her condition is very advanced, and she was not considered for a transplant.
The parents, who live in Amador County near Sacramento, did not protest the medical decision for just one transplant. But that doesn’t mean it was easy to digest.
“I still emotionally want to get the procedure done on Hunter, but you have to do the right thing intellectually,” said John Bennett, a truck driver. “Nobody wants to say I’m going to let my kid die.”
Tommy and Hunter have been undergoing tests at Duke for the last two weeks, measuring their brain activity, development skills and other bodily functions.
On Wednesday, the Bennetts met with Dr. Joanne Kurtzberg, director of Duke’s pediatric stem-cell transplant program. Tommy’s transplant, the doctor told them, may reverse some of his symptoms, but he will never be normal--or even close. He should be able to communicate, and his life expectancy may be extended.
“I know these are really hard decisions,” Kurtzberg told the Bennetts. “My best guess is that he would still need somebody to take care of him.”
John Bennett said he believes Tommy can still learn new things, unlike Hunter and Ciara. “If five years down the road, he’s playing piano and designing rocket trajectories, then we’re having a good time, but we’re not expecting that,” the father told Kurtzberg during a meeting attended by a Times reporter.
Bennett continued, “We’re going into this realistically. We don’t expect ... “
“Miracles,” his wife said, finishing his sentence.
As for Hunter, Kurtzberg said, the magnetic resonance imaging scan showed major damage and extensive loss of brain tissue. The boy’s developmental tests showed that he was functioning on a 12-month-old’s level, with little comprehension or communication skills. By comparison, Tommy has the skills of a 20- to 22-month-old, except for his inability to speak.
“I worry that the transplant is not going to help [Hunter], and it would be very difficult for him to get through it,” Kurtzberg said.
About 10% of children with similar diseases die as a result of complications from the transplant, but the risks are higher for children whose illnesses are more advanced.
During the last two weeks of testing, the Bennetts have gradually accepted Hunter’s fate, although they had been hopeful for months that he would be eligible. Short of a transplant, there is no other medical treatment for Sanfilippo, which affects about one in 70,000 children.
“Hunter has a good year left, give or take, of being aware of his surroundings, and I just don’t want to take it away,” his father said.
Ciara has accompanied her brothers to Duke, although she generally sits in a stroller, sleeping or staring into space--having lost nearly all her ability to comprehend what’s going on around her. Ciara was diagnosed with the disease in February 2001, and the boys’ cases were confirmed a year later.
Sanfilippo children develop somewhat normally during their first couple of years, learning to walk, talk and eat on their own. But as the disease progresses, the children lose those skills.
Earlier this summer, the Bennetts’ insurer, Kaiser Permanente, refused to cover the transplants at Duke, saying they haven’t been proven effective and citing the risk of death during the procedure. An independent panel of medical experts hired by state regulators concurred with Kaiser’s decision.
But the Bennetts kept the pressure on, contacting the media and attempting to raise $1.5 million through fund-raising dinners and raffles. For a time, the family worried that they would have to make a Sophie’s choice--deciding which son should get the transplant--if they were not able to raise enough money for both.
In early September, Kaiser agreed to donate $1 million to Duke for research on Sanfilippo with the understanding that it could be used to care for the boys. That sum is more than enough to care for Tommy, doctors say.
Though she is hopeful, Kurtzberg said there isn’t enough real-life experience to know for sure whether the transplants will reverse or even slow the disease. Her team has performed the procedure on only four other children with Sanfilippo, although it has a longer track record of success with children who have related diseases.
On Tuesday, a team of doctors, psychologists, physical therapists, social workers, speech and hearing experts at the University of North Carolina examined Tommy and Hunter in the final stage of determining their eligibility for transplants.
Dr. Maria Luisa Escolar, a developmental pediatrician at UNC’s Center for the Study of Development and Learning, said doctors should assist families in deciding about transplants, but not make the decision for them.
“I don’t think I have a right to do that,” said Escolar, who works with Kurtzberg. “It is more of a mutual decision. I believe that every family is different, every child is different, and you can’t be too opinionated about doing one thing or another.”
The decision to proceed with an experimental transplant comes with plenty of risks, said medical ethicist Arthur Caplan of the University of Pennsylvania. Even when parents are told of the drawbacks, they tend to focus only on the prospects for success.
“It’s more like signing up for a trip into the unknown, like an astronaut, than it is like signing up for a therapy that you would normally expect to get from a doctor,” Caplan said.
Although the Bennetts maintained their composure during nearly all of Wednesday’s meeting about their children’s fate, their emotions broke through once. Both parents cried when John Bennett asked Kurtzberg whether the couple should consider donating the bodies of Ciara and Hunter for research after they die. The doctor said yes, but then Alicia Bennett cut off the discussion. “I don’t want to think about it,” she said wiping away a tear.
To maintain their strength, the couple said they try to enjoy the time they have with the children and to separate emotion from reason.
“There’s still only one right thing to do, and that’s what’s best for them,” John Bennett said.
The Bennetts have created an online journal describing daily developments and posting photographs. The Web address is
www .caringbridge.org/ca/bennettboys.
For past stories in The Times on the family’s struggle with Sanfilippo, go to latimes.com/bennett.
