Wyss Foundation Gift Elevates Thoracic Insufficiency Syndrome Research at CHOP

By Lauren Ingeno

After inventing a prototype for a new pediatric surgical device in the 1980s, the late Dr. Robert Campbell struggled to bring it to market. Dr. Campbell — who joined Children’s Hospital of Philadelphia in 2009 — created the vertical expandable prosthetic titanium rod (VEPTR) for children with thoracic insufficiency syndrome (TIS), an umbrella term for rare spine and chest-wall conditions that prevent normal lung development. The VEPTR helps to straighten the spine and separate ribs, allowing a child’s lungs to expand as they grow.

When Swiss businessman Hansjörg Wyss met Dr. Campbell, he saw the life-saving potential of VEPTR and agreed to manufacture the device through the medical device company he had founded. Thousands of pediatric patients have since received the world’s first expandable rib, and it remains the only FDA-approved device to treat TIS.

Today, the legacy of that partnership lives on, with the announcement of $5 million in additional funding to CHOP from the Wyss Foundation to support innovations that improve the lives of patients with TIS. The Wyss/Campbell Center for Thoracic Insufficiency Syndrome was established at CHOP in 2019 with an initial gift from Dr. Wyss, chairman of the Wyss Foundation.

The additional funding will ensure the center’s continued progress in the years ahead, and it will help to bring technologies developed by CHOP researchers to market, says Center Director Patrick Cahill, MD, a pediatric spine specialist.

“CHOP's approach is very multidisciplinary, where we partner with pulmonology, physical therapy, anesthesia and critical care, respiratory therapy, nutrition, neurology, and neurosurgery to take care of our patients,” Dr. Cahill said. “It’s extraordinarily exciting to be in a place where there is an almost instantaneous impact of research on clinical care, and, in turn, where patient problems inform the direction of research. The gift from the Wyss Foundation makes that possible.”

The Wyss/Campbell Center’s research efforts are dedicated to developing assessments of pulmonary function, improving clinical outcomes, and creating novel devices for patients with TIS.

One of the key innovations that has come out of the research center in the past five years is the development of the Dynamic Lung MRI. Having accurate assessments of a TIS patient’s lung function is critical for clinicians to provide a diagnosis, fine-tune a prognosis, and develop a personalized treatment plan. However, many patients are either too young or experience cognitive impairments, which prevents them from completing a standard lung function test (spirometry), according to Dr. Cahill.

To solve this problem, CHOP researchers collaborated with scientists at Penn Medicine’s Medical Imaging Processing Group to create imaging technology and software that provides a four-dimensional, real-time view of the lungs as they expand and contract. The team also developed a technique, quantitative dynamic lung MRI (QdMRI), which allows clinicians to measure lung volumes even more precisely.

“We can look at what’s happening in different regions of the chest — the right lung versus the left lung; we can look at how much air is being brought into the chest via the diaphragm versus the chest wall; and we can really isolate where there are problems in the respiratory cycle,” Dr. Cahill said. “We use it in our clinical decision-making meetings to decide which patients to operate on and when to do the surgery, and now we’re really trying to make it available outside of CHOP, so that anyone who is taking care of these kids can have access to this transformative tool.”

Wyss/Campbell Center researchers are also finding new ways to modify VEPTR, so that it can help an even greater population of children with rare spine conditions. One modification is the development of lateral supports that can expand very far and prevent the collapse of the rib cage. This device can be life-changing for patients with a condition called spinal muscular atrophy (SMA), which was until recently considered fatal.

“It’s a device that has a pretty small, niche application, so manufacturers haven’t jumped on board, so we’re trying to do as much as we can to develop clinical science to support its use,” Dr. Cahill said.

Dr. Cahill’s research team has also conducted an FDA-approved clinical trial to investigate vertebral body tethering, a method that uses a child’s own growth as the driving force to treat scoliosis. Surgeons implant screws and a flexible cord into a patient to guide the spine into an upright position as the child grows. The method can be used as an alternative to spinal fusion surgery, which can be invasive and may limit a child’s long-term flexibility.

The generous gift from Wyss Foundation will allow CHOP researchers to expand on this research, to train the next generation of TIS experts, and to create new innovations that will benefit TIS patients in Philadelphia and beyond.

“This support is providing our clinicians with the vital platform for innovation to make tangible improvements in the lives of the patient families we serve,” said Jack Flynn, MD, chief of the Division of Orthopaedics. “The progress we have made is possible because of this crucial backing, and we are excited to reach new milestones in the years to come.”