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To save a child, family seeks therapy in China

Author Admin Views Posted at 2013/12/29

Jan 01,2008 ,by SARAH LARSON

Then they told you: some of your beautiful little boy´s enzymes are malfunctioning. Like garbage men on strike, they are not doing their jobs of clearing toxic buildup out of the cells in the brain, retina and central nervous system.

The cells are dying. Soon, your boy will be unable to walk. His chattering will stop as he loses the ability to talk.

As the disease continues its assault, he will go blind and become bedridden, unable to move on his own. In all likelihood, he will die within a few years, most certainly by the time his playmates head off to college.

Donna Kapper and Paul Anthony don´t have to imagine this hell. They are living it.

Their son Curtis Anthony, now 5, has been diagnosed with Batten disease, a rare, genetic disorder that leads to deterioration of neurological function and death.

But Donna and Paul, who live in Dublin, won´t give up their son without a fight. They soon will travel to a leading neurological hospital in China so Curtis can receive injections of stem cells that, it is hoped, may help slow the disease´s relentless assault.

"I can´t sit back and do nothing," Donna said. "Stem cell work in the United States, as everyone knows, is very controversial and strongly regulated by FDA, and Bush is very against it. But in China, they´re doing a lot of work with it."

A California company, StemCells Inc., is conducting a clinical trial using stem cells to treat Batten disease. The phase 1 trial is designed to test the safety of the therapy, in which stem cells are injected into the patients´brains by a pediatric neurosurgeon at Doernbecher Children´s Hospital in Portland, Ore. The trial began in November 2006 and will enroll up to six children, according to the Oregon Health and Science University, which runs the hospital.

Curtis´ friends and family had hoped he would be one of those six patients. But Donna and Paul found out in October that Curtis was not chosen for the trial.

Through other parents of children with Batten disease, Donna and Paul found out about research centers in China that are also administering stem cell therapies. And they have no rigorous controls such as those imposed here in the United States, Kapper said. Anyone with enough money can get the treatment.

Though hesitant at first, Donna looked into it.

"Several people had told us about it," she said. "I was wary of it, because it´s half a world away, but it seems to be helping some of the kids."

Only a few hundred children in the United States have Batten disease, according to the University of Rochester Medical Center, which was recently was awarded a $1.2 million federal grant for research and treatment of children with the disease.

Parents of two children, 6-year-old Sacha Skinner in England and 5-year-old Blake Dell´Aringa in California, sought stem cell treatment in China. They have written extensively about their experiences there on blogs and Web sites. Both families reported some positive results after the treatment, including better muscle control, hand coordination and communication.

Donna said Curtis´doctor at Alfred I. Du Pont Hospital for Children, in Wilmington, Del., has not tried to discourage the family from seeking out the experimental treatment, and has, in fact, been generally supportive.

So Donna and her mother and Curtis leave on Jan. 8 for China Stem Cells Medical Center, the largest neurosurgical medical center in Asia. There, Curtis will undergo four injections of stem cells into his brain.

"We hope it will slow down the progression of the disease, which is slowly taking away everything, starting with his speech," Kapper said. "I doubt that it can reverse the brain damage that´´s been done. What I hope and pray for is that it will stop any more damage from happening. And we don´´t know, a couple years down the road, they might find out a way to reverse brain damage."

The treatment and the stay in the medical centre? they will be there for eight weeks ? will cost $39,500, Kapper said. Airfare and other travel expenses are extra.

They set their sights on raising $50,000. Family friend Ellen Kozak had an idea: send an e-mail to 50,000 people, asking them each to send $1.

In the weeks the E-mail has been circulating, donations have poured in and have led to more efforts. Fundraisers at The Bay Pony Inn, in Lederach, and Donna´´s employer, the marketing research firm TNS in Horsham, have helped, as have donations from Land Tech Enterprises in Warrington. The fund now has more than $40,000 in it.

In the eight months since Curtis was diagnosed with the disease, life has changed dramatically.

"One day he was running around with his friends in a regular preschool, and now he´s in a wheelchair and doesn´t talk, can´t walk," Donna said.

Because the disease is passed on when both parents carry the defective gene, as Donna and Paul do, there was a chance that their daughter, Ava, also would inherit it. But tests have shown that she doesn´t have it, Donna said.

Now, a test can show whether prospective parents carry the defective gene for Batten. But there was no reason for Donna and Paul to think they needed to take the test, she said.

While the family prepares for the trip to China, Donna talks about Batten disease whenever she can. The more people who know about it, the better, she reasons. Maybe then there will be more support for research into a cure for the rare disease, which gets only a tiny amount of funding each year.

Her audience is growing, as more people learn about Curtis and reach out to help him.

"I´m just amazed at everybody who has come out of the woodwork to help us," Donna said. "It´s been just amazing."


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