Cultivating a World-class Academic Medical Research Community

A transformational $50 million donation from Mitchell L. Morgan, Dr. Hilarie L. Morgan, and their family supports the new Morgan Center for Research and Innovation, designed as a hub for the acceleration of team science to advance Children's Hospital of Philadelphia's bench-to-bedside strategies.

Madeline Bell, CHOP President and CEO

The 17-story, 350,000 square foot facility will provide state-of-the-art laboratory and learning spaces. The building's flexible layout and co-location of both wet and dry labs will cultivate increased collaboration between researchers, graduate students, and postdoctoral fellows while accommodating the ever-evolving needs of scientific research.

CHOP leaders and Philadelphia representatives gathered in June with the Morgan family at the site of the new facility, scheduled to open in 2025, for a beam signing ceremony to commemorate the gift.

"The Morgan Center will be the epicenter for advances in immunology, infectious disease, neuroscience, and much more," said President and CEO of CHOP Madeline Bell. "I'm grateful to the Morgan family for sharing our mission and our vision with this gift."

Cutting-edge research in child health today requires biobanking of pediatric biological samples, such as blood and tissue along with associated health information. Diseases that affect children are often more diverse and more rare than adult diseases. And childhood-presenting conditions more often have a genetic contribution to them than diseases that affect adults.

Enter the Children's Hospital of Philadelphia Biobank, a unique and innovative institutional resource launched this year, which gathers genomic and phenotypic data from CHOP patients and relatives. It's a reservoir of knowledge, helping to unravel the mysteries of childhood health and disease.

A central part of CHOP's Omics and Big Data Initiative, the CHOP Biobank provides a robust biorepository and data set for the entire CHOP community to access. CHOP is in the unique position to have biobanking represent its diverse patient community, including those not typically contacted for research and apparently healthy neuro-typical children without chronic disease.

"What the CHOP Biobank allows us to begin to pivot toward is not only the deep dive into understanding disease, not only assisting us along the pathway of increasingly providing precision medicine, but the ultimate goal in pediatrics of providing predictive medicine," said CHOP Biobank principle investigator Lisa Guay-Woodford, MD, Senior Advisor for Clinical and Translational Research Initiatives.

As the sole manufacturer of cell and gene therapies at Children's Hospital of Philadelphia, the Cell Based Therapy Laboratory (CBTL, formerly the Cell and Gene Therapy Lab) is active in more than 50 clinical trials and played a crucial role in the clinical trials that supported the approval of two gene therapies to treat sickle cell disease. In striving to keep up the pace of breakthroughs in research and advancements in the pharmaceutical industry, the CBTL has embarked on a phased expansion project scheduled for completion in the next three to four years.

Yongping Wang, MD, PhD

Therapeutic products must be manufactured under strict regulations upheld by the Food and Drug Administration in sterile environments called cleanrooms. Each cleanroom can house only one product at a time, and at the end of expansion, Lab Director Yongping Wang, MD, PhD, and Associate Director Stephan Kadauke, MD, PhD, envision eight to 10 cleanrooms under one roof on the first floor of the Ruth and Tristan Colket Jr. Translational Research Building.

Also to be built is a new Process Development Lab, a dedicated space where new investigational cell and gene therapies will be developed at clinical scale. By offering in-house process development, the CBTL hopes to streamline the path from bench to bedside while ensuring retention of intellectual property and keeping logistical control inside CHOP.

"What I'm most excited about is just the opportunity to make all these new therapies," Dr. Kadauke said. "We're expanding in terms of what we can do by such an order of magnitude that CHOP will be at the forefront of making these new products."

With the designation of the Kidney Stone Center at Children's Hospital of Philadelphia as a Frontier Program, researchers are hard at work studying the emergence of early-onset kidney stone disease to improve the diagnostic and therapeutic options available for treating pediatric patients.

Frontier Programs differentiate CHOP because of their unique combination of translational research and exceptional clinical care of children.

Through multi-omics research, data science, and machine learning, researchers will expand on patient-oriented investigation of kidney stone disease. The Center's primary focus is developing new kidney stone diagnostic tests using a single urine sample collected at home or in the clinic. Patient samples also will be collected in a biorepository to help build the data infrastructure needed for comparative effectiveness studies, developing diagnostic testing, and eventually organizing clinical trials.

"This is a disease that will often exist for many patients over their lifespan, so we need to develop new solutions when individuals are affected early in life," said attending urologist Gregory Tasian, MD, MSc, MSCE, a Co-leader of the Center. "As we encounter the boundaries of our knowledge in clinical care, our goal is to produce new knowledge that can be incorporated back into clinical care."

Co-leaders of the Frontier program include Dr. Tasian, Michelle Denburg, MD, MSCE, Director of Research in the Division of Nephrology; and Stephen Master, MD, PhD, Division Chief and Director of Metabolic and Advanced Diagnostics in the Department of Pathology and Laboratory Medicine and Leader of the Center for Diagnostic Innovation.

Improving clinical care and growing translational research for craniofacial anomalies is at the heart of the newly designated Craniofacial Frontier Program.

Frontier Programs at Children's Hospital of Philadelphia provide extraordinary clinical care and are dedicated to innovative translational research that advances the clinical programs.

Craniofacial anomalies are among the most common human birth conditions, causing facial disfigurements, as well as dysfunctions in speech, feeding, and breathing. The Craniofacial Program at CHOP is one of the largest of its kind — with more than 1,200 admissions for surgical procedures each year. Treatment for children with complex craniofacial conditions often requires multiple procedures over many years; however, that could change if researchers could harness genomic technologies to improve diagnostics, predict treatment outcomes, and develop new therapies for these children, according to Eric Liao, MD, PhD, a pediatric plastic surgeon-scientist and the Founding Director of the Center for Craniofacial Innovation at CHOP Research Institute.

"We'll now be able to take phenotypic, omics, and patient-reported data, and have that inform our research so that we know what to study, and we can link that data with outcomes so that we can better care for patients," Dr. Liao said.

The Craniofacial Program team is generating the world's largest craniofacial biobank, collecting patient biospecimens and phenotypic data. Machine learning is then used to integrate genetic, imaging, and clinical data to identify the human gene variants associated with craniofacial malformations in various disorders. This information will be input into a "clinical-support dashboard" to reveal important data for medical teams when making treatment decisions.

The team also will use the information to develop and test a gene therapy to treat fibrous dysplasia, which occurs when abnormal scar-like tissue replaces healthy bone, leading to fractures and facial asymmetry. Their goal is to bring a new gene therapy to clinical trial within three years.

Dr. Liao leads this Frontier Program alongside Jesse Taylor, MD, Chief of the Division of Plastic and Reconstructive Surgery, and William Peranteau, MD, who holds the Adzick-McCausland Distinguished Chair in Fetal and Pediatric Surgery.

Typically, outcomes research follows a linear model that begins with a question asked by a clinician, followed by years of research before the results impact clinical care. Children's Hospital of Philadelphia Center for Outcomes REsearch in Surgery (CORES) launched this year with a new model in mind — one that is patient-centric aimed at delivering results directly back into clinical care to improve outcomes for children with surgical conditions.

CORES welcomes collaboration among the many divisions in the Department of Surgery at CHOP, from otolaryngology and urology, neurosurgery, plastic surgery, and orthopedics. Each group is or will be represented by a CORES patient and family partner — who are either patients living with a surgical condition, or the caregivers of these patients to reflect a broad spectrum of patient priorities and inform patient-centered practices.

"The more we can conduct research that reflects real-world care, the greater impact we will have," said Gregory Tasian, MD, MSc, MSCE, leader of the Center.

Developing integrated Learning Health Systems (LHS) that improve child health requires a new generation of pediatric scientists with diverse backgrounds. Children's Hospital of Philadelphia researchers addressed this need by establishing a new National Pediatric LHS Embedded Scientist Training and Research Center to prepare faculty with skill sets to conduct, apply, and implement patient-centered outcomes research to improve quality of care and patient outcomes in an LHS.

With a five-year $5 million grant from the Agency for Healthcare Research and Quality and the Patient-Centered Outcomes Research Institute (PCORI), the Center is able to support highly qualified, diverse cohorts of LHS scientists — PEDSnet Scholars — who are committed to pursuing intensive training and mentoring in LHS science. The goal of LHS science is to accelerate how scientists learn, enhance decision-making, and improve outcomes.

The Center maintains a strong diversity focus through initiatives that address outreach, the application process, mentorship, and professional development of scientists from groups underrepresented in science. It invites projects that delve into comparative effectiveness research, improvement and implementation science, health equity, disparities, and health informatics.

"We anticipate the future PEDSnet Scholars working within our Center will become institutional and national leaders in not only advancing outcomes but also in forging the national pediatric LHS system," said Christopher Forrest, MD, PhD, Co-leader of the establishment of the National Pediatric LHS Embedded Scientist Training and Research Center and Director of PEDSnet.