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Boy Receives Stem-Cell Transplant

'It's his rebirth day,' mother says as the 3-year-old undergoes procedure to fight Sanfilippo syndrome, a rare genetic disease.

November 23, 2002|Charles Ornstein | Times Staff Writer

DURHAM, N.C. -- Three-year-old Tommy Bennett's only hope for long-term survival arrived Friday morning -- after days of chemotherapy, weeks of medical tests and months of fighting between his parents and their HMO.

Tommy, of Ione, Calif., received a stem-cell transplant, derived from a newborn baby's umbilical cord blood, at 11:25 a.m. at Duke University Medical Center here. The hope is that the stem cells will replace enzymes he is missing because of Sanfilippo syndrome, a rare but fatal genetic disease that afflicts one in 70,000 children. Those afflicted usually die by their early teens.

Though the transplant is Tommy's only possibility for a reprieve, the 30-minute procedure was somewhat anticlimactic. There were no incisions. No anesthesia. No bandages. Unlike a liver or heart transplant, this one was more like a simple blood transfusion than invasive surgery.

"It's his rebirth day," said Alicia Bennett, Tommy's mother. "There's no turning back now."

Tommy remained awake during the transplant, watching a "Barney" video and playing with a toy hammer-and-nails set and a Chicken Dance Elmo doll. He wore a red T-shirt emblazoned with the words "Kids rule" and a red hat with the transplant unit's slogan, "Grow cells grow."

The toughest challenges are ahead. In the coming days, he will lose his hair and develop painful sores in his mouth and throat from the chemotherapy.

Tommy will also be at high risk of deadly infections and organ failure because the chemotherapy drugs have wiped out his immune system. About 10% of children who undergo similar procedures die from complications.

The alternative was certain death. Sanfilippo children lack an enzyme needed to break down chains of sugar molecules. As they age, the molecules are stored in their cells, causing progressive organ damage that is ultimately lethal.

During the transplant, 1 billion healthy stem cells (in 55 milliliters of fluid) were infused into Tommy's body through a chest tube.

In a process that still baffles researchers, those cells travel to the bone marrow and begin producing new cells with the missing enzyme. They are then carried throughout the body.

"We don't know how they do it, but they do," said Dr. Joanne Kurtzberg, director of the pediatric stem-cell transplant program at Duke. "Nature is doing most of the work. We're just facilitating it."

The cells were drawn from a healthy boy born May 4, 2000, with the consent of his parents. The cells had been kept in a liquid-nitrogen freezer at minus 196 degrees Celsius. It will take up to four weeks to know whether the transplanted stem cells have engrafted and successfully given Tommy a new immune system.

One of the five other Sanfilippo children who have undergone the transplant needed a second one because his first didn't take.

"The doctor came in last night and said, 'It's been easy so far,' " said Alicia Bennett, who will sleep at the hospital during Tommy's stay. "Now is when it's going to get hard."

Even if Tommy survives the transplant, there are no published studies showing that it will stop the progression of his disease. The first Sanfilippo child received her cord-blood transplant in February 2001, and a second took place three months later.

Magnetic resonance imaging tests on the brains of those two children show significant improvement, and their developmental skills have remained stable, or improved slightly, since the transplants.

But because the damage caused by Sanfilippo occurs slowly, research on whether children with transplants avoid that decline will take years.

The Bennetts noted that luck so far has not been on their side. Their two other children -- Hunter, 6, and Ciara, 4 -- also have Sanfilippo, and testing showed that the illness had progressed too far in both, stranding them on the path to early death.

The couple fought for months to convince their insurer, Kaiser Permanente, to pay for the treatment. After an independent panel of medical experts upheld Kaiser's denial, the couple took their case to the media.

In September, Kaiser agreed to donate $1 million to Duke for Sanfilippo research, more than enough to cover the cost of Tommy's transplant.

John Bennett said the fight with Kaiser needlessly delayed treatment for Tommy.

Kurtzberg said the Bennetts have held up well during the transplant process. "I don't think [Tommy] really understands what's going on," she said. "With a child this age, the parents are more the patients than the child.... And they've been great."

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Previous stories about the Bennetts can be found at www.latimes.com/bennett.

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