Published onChildren's View
By Lyn A.E. McCafferty
Three-year-old Briana is excitedly exploring her world. Six-year-old Parker loves riding his bike. Mya, 23, recently graduated college. These three young lives — and so many others with serious liver disease — were saved thanks to collaborative research, multidisciplinary care and innovative treatments at the Fred and Suzanne Biesecker Pediatric Liver Center at Children’s Hospital of Philadelphia.
Now celebrating the center’s 22nd year, clinical and research leaders at CHOP reflect on what they’ve learned about liver disease, how they’ve advanced liver treatments for children, and the philanthropic family who’s supported them every step of the way.
“No one was really talking about collaborative liver research until Fred Biesecker’s granddaughter, Mya, got sick in 1999,” says David A. Piccoli, MD, former Director of the Biesecker Center.
Mya was just weeks old when doctors at CHOP diagnosed her with biliary atresia, a serious liver disease with no known cause or cure. Because the condition results in a blockage or absence of bile ducts, Mya would need immediate surgery. Even if the procedure was successful, Mya would very likely need a liver transplant before adulthood.
“At the time,” Dr. Piccoli says, “nobody in the U.S. was even studying biliary atresia in a serious way, and no one was interested in funding the work.”
The Biesecker family changed that. They provided funding to CHOP to launch the Fred and Suzanne Biesecker Pediatric Liver Center with the goal of drawing doctors and researchers to CHOP to better understand what caused liver disorders and to develop cures and treatments.
The team included gastroenterologists, who treat the organs of the digestive system, including the GI tract, liver, pancreas and gallbladder; hepatologists, who are subspecialists in the liver, pancreas and gallbladder; nutritionists; immunologists; and geneticists. The CHOP team collaborates with adult medicine specialists from the University of Pennsylvania and other local institutions. Fred Biesecker and his daughter, Lissa Longacre, Mya’s mother [see spotlight, below], were on the leadership team, partnering with the clinicians every step of the way.
The group has met monthly since the center launched in Decemeber 2000 — sometimes with as many as 60 collaborators involved. They share details about current research, learnings about specific liver disorders and new scientific findings at CHOP and around the world.
“The open discussion provokes cross-pollination of ideas — from researchers and clinicians — and pushes ideas further,” says Kathleen M. Loomes, MD, current Director of the Biesecker Pediatric Liver Center. “It’s led to major advances in our understanding of liver disorders and improved models of care.”
Kindling for innovation
What the center has accomplished is nothing short of astounding. When it began, there were only four principal investigators doing liver research at CHOP; today there are nearly two dozen. Center investigators are making incredible progress in understanding and treating many of the most challenging liver diseases.
Today, the Biesecker Pediatric Liver Center supports clinicians and investigators engaged in basic, clinical and translational research — at CHOP and at centers around the world. This synergy is leading to sentinel discoveries that continue to advance the field of pediatric liver disease.
Recent breakthroughs launched or accelerated at CHOP
- The discovery of a plant toxin in Australia that causes biliary atresia in livestock; studies are underway to understand how the toxin causes bile duct injury and how these effects might be reversed.
- Studies to understand the differences in bile ducts of young infants that make them more susceptible to injury.
- Investigations to determine if personalization of immunosuppression medication is possible following liver transplant, rather than the current “one-size-fits-all” approach.
- Studies to determine if newer, noninvasive blood tests — rather than a liver biopsy — can be used to diagnose organ rejection.
- Studies to understand how the genetics of an organ donor contribute to the risk for diabetes or high cholesterol in a liver transplant recipient.
- Studies on new medications to treat biliary atresia and certain other liver diseases that could delay — or avoid — the need for a transplant.
- Studies of new treatments for hepatitis C in children.
“Over the past two decades, the monthly center meetings, along with enthusiastic engagement from the Biesecker/Longacre family, have led to an open exchange of ideas and a host of new collaborations that have enabled us to move the field forward much more quickly than would have otherwise been possible,” Dr. Loomes says.
Stoking the fire for years to come
After 22 years, the Biesecker Pediatric Liver Center continues to advance its mission. “We have a better understanding of the best care to provide to children with liver diseases, and more options in terms of medications to improve patients’ quality of life,” Dr. Loomes says. “In many cases, patients with liver disease can have an excellent quality of life without a transplant.”
“Our partnership with the Biesecker/Longacre family has helped make CHOP a destination for any child with pediatric liver disease,” Dr. Piccoli says.
And it has produced positive outcomes for children like Mya, Parker and Briana.
Inspiration for the Impossible
When Lissa and Jeffrey Longacre arrived at CHOP with their 5-week-old daughter, Mya, in 1999, it started a journey that would change not just their lives, but also those of many others.
Mya was diagnosed with biliary atresia, a rare disorder in infants that blocks the ducts that carry bile from the liver. Babies require surgery shortly after diagnosis, and about 80% of patients need a liver transplant by age 20. Biliary atresia is also an orphan disease, meaning it has not been adopted by the pharmaceutical industry for research development because there’s little financial incentive.
When Lissa Longacre learned these facts, she made it her mission to change the face of liver disorders research. “When someone you love needs help, you figure out how you can be part of the solution in whatever way possible,” she says.
In 2000, Lissa and her family partnered with CHOP to launch the Biesecker Pediatric Liver Center. Now in its 22nd year, the center is a global leader in breakthrough research and treatment.
Giving back is part of Lissa’s DNA. Her parents taught her from an early age that there were many ways to contribute — whether through gifts of time, talent or treasure — and after Mya’s diagnosis, the family committed all three to CHOP. Longacre’s father, Frederick Nielson Biesecker, joined CHOP’s Board of Trustees, and he was a fixture at the Biesecker Center’s monthly meetings until his death in 2012. Longacre now serves as a trustee herself and has upheld her father’s commitment.
“When you meet the amazing people at CHOP, you are compelled to want to be there more and to do more,” she says.
While she is proud of the center’s accomplishments, her dream is to “unlock the mystery of biliary atresia and truly transform the lives of children born with this disease.”
She adds: “Sometimes when you are an adult, you forget what it’s like to be a child. I believe in the magic of childhood, and I wish every child had the opportunity to live in this magical world and learn to dream. Dreams are the inspiration for doing the impossible.”
— Karen Hamilton