Butler County boy's cupcake dream motivates doctor

| Monday, May 21, 2012, 11:47 p.m.

Austin Rath is almost 6 years old and never has had a drink of water. He hasn't eaten a cupcake, either.

The kindergartener from Renfrew in Butler County lost about 90 percent of his intestines shortly after birth because of an illness known as short gut, or short bowel, syndrome. For nutrition, he gets four liters of intravenous fluid every day.

Austin's parents, Jennifer and Jason Rath, believe his best shot at living a normal life is an artificial intestine that scientists at Children's Hospital of Pittsburgh are developing.

"My biggest wish ever is to get a new tummy," Austin told Dr. David Hackam, the research project's leader, in a video he sent him on Christmas Eve. "Work very hard."

The boy's plea captivated Hackam, a pediatric surgeon who has spent two years creating an intestine. Although Hackam's team mastered a way to culture stem cells removed from the gut, they were unable to create a scaffold structure where cells can grow.

About 18 months ago, Hackam met with a Cornell University scientist, John March, who developed a scaffold similar to the intestine, an intricate three-dimensional structure. The teams partnered to push the project forward, in part with the help of a $543,571 grant from The Hartwell Foundation in Memphis.

In the Children's Hospital model, stem cells are rolled into a tube that doctors can place in tissue within the belly, Hackam said. The tissue gives the tube a blood supply that can absorb feedings. Hackam said he expects to test the artificial intestine in mice within a year and then would experiment on pigs.

Human trials might begin in about three years, Hackam said. Austin's case, he noted, pushed him to work harder.

"I was so touched," said Hackam, a professor of surgery, cell biology and physiology at the University of Pittsburgh School of Medicine. "When I saw his video, I literally started to get teary eyes. I told Austin I will not rest until I can generate a new tummy for him."

The artificial intestine is of particular interest to people with short gut syndrome, a condition in which the body cannot absorb food. As a result, those people receive nutrients through a vein, which can cause infections and liver toxicity.

Dr. Tracy Grikscheit, whose team at Children's Hospital of Los Angeles successfully created a tissue-engineered small intestine in mice, said scientists are addressing roadblocks such as enabling the cells to grow with adequate blood supply.

"It is much more difficult to try to make a full-thickness part of the intestine. It's like trying to build a skyscraper -- you have to make the supporting structure before furnishing the rooms and then have enough supply lines, or blood vessels and nerves," she said.

The possibility of a solution to his condition motivates Austin. He's sad and frustrated because he can't eat what other kids eat at school, especially when classmates bring treats to celebrate a birthday or holiday, said his mother.

The Raths, who have three other children, initially believed Austin could be cured only with an organ transplant. That surgery, however, comes with potential complications and would force Austin to take immunosuppresive drugs for life. Little more than half of children ages 1 to 5 survive 10 years after the transplant.

Austin devised a clever idea for his birthday on Wednesday. Instead of getting gifts, he told his parents he wants people to give money toward Hackam's research.

Austin started what he calls "Austin's Cupcake Fund" because his biggest wish is to be able to eat as many cupcakes as he wants. He has collected $5,000 toward Hackam's research.

"I just want to eat everything," he said.

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