September 5 2002

Because of a $1-million research donation from Kaiser Permanente, two Amador County boys with a rare genetic disease may soon get experimental transplants that offer the only chance to save their lives.

Kaiser, the state's largest HMO, had refused for months to pay for umbilical cord-blood transplants for Hunter Bennett, 4, and his 2-year-old brother, Tommy. Their 6-year-old sister, Ciara, also has the disease, but is not eligible for the transplant because the illness has progressed too far.

The HMO argued that the transplants themselves could be life-threatening and had not been proved to work. But, after an onslaught of media coverage, Kaiser agreed to donate $1 million to Duke University in North Carolina for research into the children's condition, known as Sanfilippo syndrome. Duke can use the money to cover the boys' medical costs there.

Technically, the HMO's decision does not set a precedent or change its position on covering the experimental treatment--but it allows the family to pursue its only hope.

"As an organization made up of parents, grandparents, siblings and children, we know exactly how you feel," wrote George Halvorson, Kaiser's new chief executive, in a letter hand-delivered to the Bennett family Wednesday. "We realize and appreciate that the family's hope now rests beyond the realm of proven science and evidence-based medical judgment."

Sanfilippo patients lack a key enzyme needed to break down chains of sugar molecules. The sugar molecules build up over time, causing progressive organ damage and, ultimately, death. Most Sanfilippo patients don't live past their mid-teens.

A Duke researcher believes that the transplants using babies' umbilical cord blood may be able to stop the progression of Sanfilippo by replacing defective cells and providing the enzyme that its victims lack.

The boys' parents, John and Alicia Bennett, learned of Kaiser's $1-million gift several minutes before the start of a prearranged news conference Wednesday morning at Kaiser's national headquarters in Oakland.

Rather than criticizing the HMO, as they had planned, the Bennetts thanked them.

"I see a light at the end of our tunnel," said Alicia Bennett, who began crying when she learned of Kaiser's decision. "At least the boys have a future now."

Dr. Joanne Kurtzberg, director of the pediatric stem-cell transplant program at Duke, said she was elated to hear about the contribution. But she said she is still "a little bit gunshy," because she has not spoken to Kaiser officials directly.

"We're certainly grateful that they would do this, and we will use the money to treat these kids," she said. "If this gets worked out in the next day or so, we'd have them come next week or the week after to get the ball rolling."

The parents had been frantically trying to raise $1.5 million--through raffles, dinners and community fund-raisers--to pay for the experimental treatments. The parents had not even heard of Sanfilippo until after all three of their children were born.

The Times profiled their story last month, after which the family received about $75,000 in donations.

Although other insurers have covered cord-blood transplants for Sanfilippo patients in the past, ethicists said Kaiser should not necessarily be faulted for balking at an expensive, unproven procedure.

And a panel of independent medical experts hired by California's HMO regulators upheld Kaiser's refusal this summer.

But the Bennetts persisted. Because no other treatments are available for Sanfilippo patients, the children's alternative was death. Researchers have not devoted as much attention to Sanfilippo, which affects just one in 70,000 children, as they have to more common diseases.

In Wednesday's letter, Halvorson wrote that the HMO's action doesn't change its contention that the "proposed experimental treatment is both without any substantive evidence of value and too risky for the children."

But, he added, "We deeply hope that we are wrong ... and that our contribution to Duke University will both help your children and advance the medical community's understanding of how best to eventually treat this insidious disease."

Kurtzberg examined the boys in March, a month after the Bennetts learned that their sons had the disease. Before proceeding with the transplant, she needs to run a battery of expensive tests on them to determine if they are good candidates.

If they are, the boys will be admitted to the transplant unit, where they will receive nine days of high-dose chemotherapy. On the 10th day, they will get the cord-blood transplant, which is essentially a blood transfusion.

Because the chemotherapy and transplant obliterate the body's immune system, patients develop side effects including low blood counts, fevers, sores in the mouth, diarrhea and vomiting.

Patients are provided pain medicine, intravenous feeding and antibiotics. The children must stay in the hospital four to six weeks after the transplant, and then visit an outpatient clinic every day for 100 days.

The Bennetts, who have argued their case on television and in newspaper articles, have made some important allies, including California state Sen. Rico Oller (R-San Andreas), who has been pushing Kaiser to change its position.

"I'm absolutely tickled," Oller said of Kaiser's donation to Duke. "They really wanted to do the right thing.... This is what we hoped to accomplish."

The state's top HMO regulator also praised Kaiser's action.

"I thought it was a compassionate response given that their doctors and independent doctors weren't willing to endorse this as a medical procedure," said Daniel Zingale, director of the California Department of Managed Health Care. "It sounds to me like that contribution has the potential to accelerate the day when there is a real treatment."

John Bennett said he remembers how depressed he felt upon learning that all of his children had this incurable disease. On Wednesday, he said he felt "almost the opposite"--at least in the case of his two sons.

"It's starting to look a lot better to me," he said.

To cover remaining costs for their children's treatment, a Web site is soliciting donations: www.helpachild.net. Donations also may be sent to the Bennett Children's Fund, P.O. Box 1826, Jackson, CA 95642.  

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Copyright 2002 Los Angeles Times