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Boys Put to the Test in Lifesaving Effort

Medicine: Results will show whether two brothers with rare Sanfilippo syndrome are transplant candidates.

September 22, 2002|CHARLES ORNSTEIN | TIMES STAFF WRITER

DURHAM, N.C. — Tommy Bennett spent much of his third birthday last week sedated on a hospital bed with six electrodes strapped to his head and loud noises clicking in his ears.

In a nearby building, his 4-year-old brother, Hunter, was held still by strips of medical tape so X-ray technicians could photograph every part of his body.

The boys' parents, John and Alicia Bennett, stood by as doctors administered a battery of tests to determine whether they are good candidates for experimental umbilical-cord blood transplants.

It is the last hope for saving their lives.

"We hope to celebrate his 30th and 40th birthday someday," said John Bennett, a trucker in Northern California, lying next to Tommy on the hospital bed Wednesday at Duke University Medical Center.

The Amador County brothers and their older sister, Ciara, 6, have Sanfilippo syndrome, a rare genetic condition in which their bodies fail to produce an enzyme needed to metabolize sugar molecules. As the children age, sugar builds up and causes progressive damage in their organs, joints and brains. Most Sanfilippo patients die during their teenage years.

The disease has no proven treatment. John and Alicia Bennett have staked their hopes on experimental transplants intended to provide the children with the enzyme they lack.

The parents know Ciara is not eligible because the disease has progressed too far. They fear that Hunter may also be deemed a bad candidate because he turns 5 in March, an age at which major deterioration usually has begun.

They will receive the results of the tests for their sons in the next few days.

At Duke last week, the boys were shuffled from blood work-ups to magnetic resonance imaging, eye exams, X-rays and tests to measure brain activity.

Ciara, meanwhile, sat near her parents in a stroller, unable to talk and seemingly unaware of her surroundings, a constant reminder of Sanfilippo's destructive path.

The Bennetts' visit to Duke was something they had dreamed of--and dreaded--for months.

"We're trying to squeak [Hunter] in, if possible," Alicia Bennett told Dr. Joanne Kurtzberg, director of the pediatric stem-cell transplant program at Duke, who agreed that Hunter was a borderline case.

Kurtzberg said she wants to treat youngsters who have the potential to develop basic skills such as speaking and dressing themselves. That is why she is testing their brain function and developmental status. As it stands, the Bennett boys can't speak coherently.

"I don't consider prolonging life enough," Kurtzberg said, even if parents do.

The parents did not find out that Ciara had Sanfilippo until February 2001, after all three children had been born. The boys' diagnosis was confirmed last February.

Even if the boys receive the transplants, there's no guarantee that the procedures will work. Some scientists are skeptical, based on previous results involving Sanfilippo children who received bone-marrow transplants.

But Kurtzberg, one of the few doctors to explore treatments for the disease, said she is hopeful based on the success of cord-blood transplants in children with diseases related to Sanfilippo.

In addition, two Sanfilippo children who underwent the procedure at Duke more than a year ago are showing signs of progress, although it's too soon to call the transplants a success.

The alternative is death. Even if their boys won't lead normal, or even semi-normal, lives, the Bennetts are not ready to give up.

"I'm Mom. I have to root for them," Alicia Bennett said.

On his birthday, Tommy was too sedated to celebrate. The day after, his parents found a Chuck E. Cheese's restaurant in Durham and held a muted celebration. As his father held him, Tommy tossed miniature basketballs, grinning and giggling the whole time.

The Bennetts had planned an all-out party for 30 relatives and friends on Saturday at Chuck E. Cheese's in Sacramento, but it had to be canceled because of the last-minute trip to Duke.

Just getting to Duke was a challenge for the Bennetts. Their insurance company, Kaiser Permanente, refused to pay for the procedures because its doctors said there was no evidence showing that they work. Kaiser also said that the children risked serious complications, even death, from the treatment itself.

Kaiser's position was upheld by an independent panel of medical experts hired by the state to review the case this summer.

Earlier this month, after the Bennetts' story drew attention from the media and state lawmakers, Kaiser gave a $1-million research grant to Duke, with the understanding that it could be used to cover the boys' testing and treatment.

Before Kaiser's donation, the Bennetts had raised more than $140,000, which will now be used for any additional care their children need. Leftover money will be used to help other children.

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