Children’s Hospital of Philadephia has launched the most ambitious fundraising effort in its 162-year history, with the goal of raising a record-setting $1 billion by 2020.

The campaign, “For Tomorrow’s Breakthroughs: The Campaign for Children’s Hospital of Philadelphia” will fund the development of comprehensive, wide-ranging advances to save more children’s lives. It’s off to a great start — the campaign has already reached the $800 million mark.

Donations will support five critical areas. Funds will go toward research to support the development of new treatments for the most complex conditions in order to set the standard for pediatric healthcare worldwide. Creating an exceptional patient-family experience by providing the most advanced treatments at the best facilities, with the most comprehensive support programs, will also be a funding priority.

Focusing on training pediatric care leaders of the future, including healthcare providers, specialists, and researchers, will give staff opportunities to advance their education so they can offer the best care possible for children and families. Funds raised will further support outreach to children and families in need in order to improve the health and well-being of children and families in the region, nation, and world.

Finally, the increased funding will give CHOP the resources to act quickly on new opportunities and meet unexpected needs. The Children’s Fund provides financial resources to take a broad range of actions that can make a difference, such as purchasing revolutionary new equipment, moving a promising discovery forward, or hiring a leading expert.

Children’s Hospital of Philadelphia awarded two centers Frontier Program status in 2018, providing the Immune Dysregulation and Food Allergy Programs with the support and resources needed to drive world-changing discoveries, deliver life-saving therapies, and strengthen the bridge between research breakthroughs and exemplary clinical care.

Unraveling the Architecture of Immune System Disorders

With a team of clinical and scientific leaders across multiple disciplines, the Immune Dysregulation Program will enable the diagnoses of patients with rare, complex diseases of the immune system and develop novel precision drugs for treatment.

“If we can discover a mutation in a patient’s gene, then understand why and how that mutation causes a problem with the immune system, we can think of a medicine that can attack that particular problem,” said Edward Behrens, MD, chief of the Division of Rheumatology and co-leader of the Immune Dysregulation Program alongside Kathleen Sullivan, MD, PhD; Michele Lambert, MD, MSTR; and David Teachey, MD.

In 2015, Dr. Behrens and his team diagnosed a patient with an autoinflammatory disease caused by a mutation of the NLRC-4 protein and successfully developed exactly that type of precision treatment approach. According to Dr. Behrens, Frontier support empowers him and fellow scientists to conduct the same in-depth investigation for even more patients, armed with resources they have dreamed of obtaining for years. These include novel laboratory testing, rapid genetic testing, and an expanded multidisciplinary clinical staff.

Addressing the Unknowns in Food Allergy Research

The Food Allergy Center at CHOP, already a nationally recognized program for its expertise in all four types of food allergy, will utilize Frontier support to raise their clinical care standards by developing new diagnostic assays and testing novel therapies.

“The Frontier Program allows us to really bring new clinical and research endeavors to patients,” said Jonathan Spergel, MD, PhD, chief of the Allergy Section at CHOP and co-director of the Food Allergy Frontier Program alongside co-directors Terri Brown-Whitehorn, MD, and Megan O. Lewis, MSN, CRNP. “There are so many important unanswered questions needed to improve care, and we will begin to answer those questions and move things forward to the ultimate goal: Treat, prevent, and cure food allergies.”

In partnership with Oxford University researchers, Dr. Spergel and his team are currently studying how to predict which patients will develop a severe reaction to a food allergen versus a mild one. By looking at cell function on a genome-wide array before and after a food challenge, Dr. Spergel hopes to elucidate not just who is at risk for a severe reaction, but what is different at the cellular level when such a reaction occurs.

The program also has a robust research pipeline for eosinophilic esophagitis (EoE), an inflammatory disease of the esophagus triggered by food allergens. Dr. Spergel’s previous research showed that T-cells become activated in response to milk allergens in patients with EoE, and the next step involves studying T-cell response to other foods and developing a clinically useful test to measure what foods might be causing EoE.

A team of Research Institute experts in library science, computational biology, informatics, and patient privacy are creating an integrated data science platform to make entirely new kinds of breakthroughs in pediatric research.

Aptly named Arcus, which means “arch” in Latin, the new platform will give investigators across campus access to a robust data platform that bridges the wealth of clinical and research data at Children’s Hospital of Philadelphia.

“ARCUS provides the bricks in the foundation we need in order to be in the right place at the right time for the arrival of big data in pediatric research,” said Jeff Pennington, associate vice president and chief research informatics officer at the Research Institute, who is leading the Arcus program. “This is a window in time when we can build our own in-house expertise at relatively low cost to use rapidly advancing machine learning and artificial intelligence methods to have an impact on child health.”

With about 2.6 million patients on record at CHOP, Arcus is an ambitious project that uses a library science approach to catalog, cross reference, and enrich the data produced over the course of patients’ clinical encounters and research study visits throughout childhood and adolesence. Hundreds of terabytes of data — from MRIs, CTs, bedside monitors, laboratory results and more — have been collected through CHOP’s single electronic medical record since about 2010. In addition, the Research Institute currently has about six petabytes of research data, mainly genomic and molecular biology data.

Arcus will link the vast quantities of pediatric data CHOP generates as a clinical enterprise with the data it generates as a research enterprise to produce a more holistic picture of pediatric health and disease. Importantly, the Arcus team also includes patient families who represent the patient perspective in the development of such a significant new data resource.

A growing cohort of pilot studies conducted by scientists who are investigating a wide range of scientific questions along the research continuum are driving this one-of-its-kind data library. As data sets are continually added, Arcus will become a powerful key to unlocking chronic and complex pediatric diseases and opening new viewpoints into how healthy children grow and develop.

A new Center for Computational and Genomic Medicine at Children’s Hospital of Philadelphia will drive biological discoveries and medical innovations by leveraging recent advancements in sequencing technology and computational biology. The Center will contribute to CHOP’s entire ecosystem of research, including collaborations with the Center for Cellular and Molecular Therapeutics and the Department of Biomedical and Health Informatics, among others.

Yi Xing, PhD, moved his lab and team of about 20 investigators from University of California Los Angeles to CHOP’s research campus and officially opened the Center Sept. 1. It will provide an intellectual home for recruiting tenure track faculty who are not only adept at using existing technology, but also can develop new genomic technologies or computational tools that can be offered to a broader community of scientists. The Center will have a hybrid character — computational biology research will pave the way for new projects in wet laboratories.

“Our goal is to make the center an engine for technological and biomedical innovation,” said Dr. Xing, who also is a professor of Pathology and Laboratory Medicine in the Perelman School of Medicine at the University of Pennsylvania. “There will be tight interaction from both the basic science and technology side, as well as the translational and clinical side of genomics and computational biology.”

Dr. Xing and his team have addressed stimulating concepts in the field, such as defining the fundamental data structure and algorithms for looking at the complexity of RNA. Because so many human mutations disrupt RNA and contribute to disease, Dr. Xing’s work in mastering how to manage and interpret big data offers opportunities for improved diagnoses and treatments.

“A fun part about working in the area of genomics and computational biology is the technologies are constantly changing, and they generate a lot of interesting problems to solve,” Dr. Xing said. “In turn, those technologies generate a huge amount of data. A big part of our lab is to develop innovative computational methods for integrating those larger scale data sets in order to translate big data into knowledge.”

Dr. Xing holds the new Francis West Lewis Chair in Computational and Genomic Medicine at CHOP.

We are always striving for excellence at Children’s Hospital of Philadelphia Research Institute — from our first days of orientation to when we become leaders and mentors at the top of our fields. A new Office of Academic Training and Outreach Programs (ATOP) launched in 2018 to create engaging and comprehensive educational experiences for researchers and their staffs at all levels of the career ladder.

The new office led by Wendy Reed Williams, PhD, senior director, reflects three tiers of service: academic training, outreach programs, and specialty programs and diversity. The academic training arm of the office includes expanded and formalized support with an emphasis on academic success skill training programs, in partnership with key faculty across the Research Institute. Outreach programs creates internships, events, and career exposure opportunities for students and community partners to develop future generations of leaders in child health. The newest service tier focuses on developing targeted research programs and pilot grant opportunities, while also building and supporting a diverse workforce at the Research Institute.

ATOP will add and expand upon its core services for research staff and trainees to meet the educational needs of the research community while promoting a culture of best practices and integrity in research. Some of the initiatives that will be rolled out over the course of FY2019 and beyond include reaching a wider audience of early career researchers, offering new research opportunities for science-minded undergrads and high school students, developing a formalized diversity support infrastructure at the Research Institute, and creating discipline-specific research programs and pilot grant opportunities for scientists-in-training, among others.

This transition and expansion of this critical support office will be conducted under the purview of Chief Scientific Strategy Officer, Beverly Davidson, PhD, to align its work with key strategic initiatives at the Research Institute.