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3rd Experimental Procedure May Be Boy's Last Hope

The 4-year-old, one of three Amador County siblings with a fatal genetic condition, will have a stem-cell infusion today.

September 19, 2003|Charles Ornstein | Times Staff Writer

Nearly out of cash but clinging to hope, John and Alicia Bennett will watch today as their youngest son undergoes -- for the third, and likely last, time -- an experimental procedure that is his only chance at survival.

"Half of me is hopeful. Half of me is not," said Alicia Bennett, whose family has traveled from their home in Ione, Calif., to Duke University Medical Center for Tommy's treatment. "Part of me doesn't want to get hopeful, just in case ...."

Tommy spent his fourth birthday Thursday at the Durham, N.C., hospital receiving radiation therapy. Today, he will receive an infusion of stem cells aimed at reversing the progression of Sanfilippo syndrome, a rare genetic condition that also afflicts his two older siblings.

Tommy's first two stem-cell transplants, in November and January, failed.

Children with Sanfilippo typically die by their early teens, and doctors have determined that Tommy's siblings -- Ciara, 7, and Hunter, 5 -- are too disabled to undergo the experimental procedure.

Sanfilippo patients lack an enzyme needed to break down sugar molecules in the blood. Over time, the molecules build up, causing progressive brain and organ damage and ultimately death.

The stem cells, which are derived from the blood of a newborn baby's umbilical cord and placenta, contain the enzyme that Tommy's body lacks. The transplant is not a surgical procedure, but more like a blood transfusion.

Even if the transplant is successful, however, doctors are not sure that it will keep Tommy alive.

Ten other children have undergone the procedure in the past two years. Four have died of complications and it's too early to tell whether the procedure has stopped the disease's progression in the remaining cases.

The first two children are beginning to show improvements, said Dr. Joanne Kurtzberg, director of Duke's pediatric stem-cell transplant program.

"It's really going to take longer to know" whether this treatment works, Kurtzberg said.

The Bennetts' struggle, chronicled by The Times, has been one of hope and disappointment.

Tommy and his mother have spent the past year away from family and friends in Amador County, near Sacramento. His father, a trucker, and the other two children have shuttled back and forth. John Bennett is on leave from his job to stay at Duke for several weeks.

Although the family has raised more than $100,000 from friends, neighbors and strangers, Alicia Bennett said they have only $1,000 left after paying for leases in North Carolina and California, airfare, medication, food and clothing for their children.

In her online diary documenting Tommy's progress, Alicia Bennett has conveyed the minutiae of their lives -- what they eat, what movies they watch, and when Tommy falls asleep. She has also talked about the difficulty of being away from family and friends for so long.

"We have been here at Duke for one whole year and there is still no end in sight," she wrote Aug. 11.

Now, however, there is an end in sight. Even if the third procedure does not succeed, doctors have said there's little more they can do. "One way or another, in six months, we'll be home," Alicia Bennett said in an interview.

She said there have been more high points than low ones because "he's still here .... He could be gone."

Just getting to Duke was a challenge. The Bennetts fought for months to persuade 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.

Last September, Kaiser agreed to donate $1 million to Duke, and the hospital is now picking up the rest.

Despite the failure of the past two procedures, Kurtzberg said, Tommy has actually made a lot of developmental progress in the past year, which normally would not be expected of Sanfilippo children at his age. She attributes his advances to a regimen of immune-suppressing drugs that slowed the disease's progress.

"He's a strong kid," the doctor said. "What I can say right now is that he's improved over this year and I think that we haven't lost any ground because of that.

While Tommy has been in North Carolina, his sister Ciara has become progressively worse. She cries for hours on end and she takes strong pain-killers that cause her to sleep nearly all day.

"She's not the same little girl she was before," Alicia Bennett says, choking up. "I know Tommy's going to be fine because he's a trooper. I'm more worried about Ciara."

Donations to help cover the Bennetts' costs can be sent to Bank of America, attention Alyssa Boreiko, 851 Willow Drive, Chapel Hill, N.C. 27514.

Previous stories about Tommy can be found at Alicia Bennett's journal can be found at www.caring

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