What’s Ahead in Pediatric IBD Care? Cure-Based Therapies and Prevention-Based Interventions

Symposium highlights areas of hope and advancement within the field

While treatment options for pediatric inflammatory bowel disease (IBD) have improved significantly over the last several decades, disease manifestations have also become more complex and with earlier onset in children than has been noted in previous eras.

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Still, physicians say there is justified enthusiasm as targeted and personalized treatment, improved surgical techniques, and more informed epidemiological insights give way to a new paradigm in the delivery of pediatric IBD care. 

These were central themes during the inaugural Kaplan Foundation Pediatric Inflammatory Bowel Disease Symposium hosted at Cleveland Clinic in May 2022. Jacob Kurowski, MD, a pediatric gastroenterologist who chaired the event, says when former colleague Barbara Kaplan, MD, who practiced at Cleveland Clinic for more than 20 years before retiring in 2021, shared her idea to create the symposium, he was eager to help bring the event to fruition.  

“The words were barely out of her mouth and I burst open with 400 ideas about how we could do this,” laughs Dr. Kurowski. On their relationship, he says, “I worked with Dr. Kaplan very closely since joining Cleveland Clinic seven years ago, and even though we trained in different eras, we had a very similar passion and approach to patient care.”

Presentations at a glance

Together they designed a schedule of presentations that addressed three current topics in the pediatric IBD community: the role of regenerative medicine in the treatment of IBD, the use of artificial intelligence (AI) in predicting outcomes, and leveraging data to develop new epidemiological research models.  

“It’s undeniable that technology—in therapeutics and data application—is changing the landscape in IBD care. These presentations provided a snapshot of some of that exciting work,” says Dr. Kurowski.

Using mesenchymal stem cells to treat fistulizing perianal Crohn’s disease

Amy Lightner, MD, Director of Cleveland Clinic’s Center for Regenerative Medicine and Surgery, discussed the role of mesenchymal stem cell (MSC) therapies in the treatment IBD-related fistulae. The approach—injecting MSCs into the fistula tract—has been widely adopted in adult practice over the last two decades and even heralded as a “magic potion,” according to Dr. Lightner, “and for good reason.” She says, “There are tremendous benefits: they are safe, effective, can be done in an outpatient setting, incur little to no risk of incontinence, and have other applications.”

In the pediatric setting, this approach is being conducted in a phase 1 study at Cleveland Clinic to treat perianal disease. Six patients have undergone treatment so far, and they have all shown an excellent clinical response and complete clinical healing at six months postoperatively. After this phase is complete, the physicians plan to open a multicenter study.

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She also discussed the potential therapeutic role of MSCs in treating luminal disease but cautioned that many unanswered questions remain about how MSC therapy works. These answers may unlock new information about pathways in pathophysiology, variability in efficacy, and how to overcome limitations in cellular therapy.

Leveraging artificial intelligence to better understand and treat IBD

Dr. Kurowski presented on current uses and future potential of AI in endoscopic, histologic and radiologic imaging, as well as machine learning applications of electronic medical records. He says applications can help predict responses to therapy, support disease monitoring, and inform the selection of therapy.

“AI includes machine learning, deep learning, supervised and unsupervised learning—each slightly different but all adept in removing inherent human-based bias and variability,” he says.

For his part, Dr. Kurowski and his team are currently involved in radiomics studies, which involve a pixel-by-pixel analysis of MRIs to develop a clearer picture of disease characteristics and use as a potential biomarker. He has also published several studies involving natural language processing, a machine learning tool that detects key terms in electronic health records and can help compare treatment outcomes and identify early response and failure.

He is hopeful AI can be used to inform precision medicine and help sharpen the delivery of care for patients with IBD.

“If we know that 600 patients we’ve previously taken care of with a certain set of characteristics have all responded to this therapy versus that therapy, that can help inform a treatment plan and support clinical decision-making with patients and families.”

A closer look at the global epidemiology of IBD

Eric Benchimol, MD, PhD, a pediatric gastroenterologist at the Hospital for Sick Children in Toronto, delivered the keynote address at the symposium. His presentation examined the global epidemiology of IBD, including literature that shows the growing incidence of IBD in newly industrialized and developing countries.

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He stressed that access to health data has enabled investigations linking IBD to certain exposures, particularly early antibiotic use and environmental risk factors such as air pollution, water quality, neighborhood walkability, and other variables. He also explored associations related to race and ethnicity, patterns of human migration, and their subsequent effect on the human microbiome, relating to the incidence of IBD.

The new way forward, he argues, is continuing bench research to improve bedside care, but also an increased focus on extrapolating patient data to better understand IBD causes and manifestations at the population level, and then delivering broader interventions that may also inform personalized care.

Dr. Benchimol is hopeful this new model is achievable by leveraging reliable data mining methodology and fostering collaboration among clinicians, basic and translational scientists, epidemiologists, computer and data scientists, engineers, policymakers, and patients and families.

The takeaway message

Dr. Kurowski says the presentations address themes that loom large within the field.

“There is a lot of hope and advancement in this field as we move toward cure-based therapies and prevention-based interventions. We have identified several environmental risk factors, some more modifiable than others, and that work will continue to be important. These presentations represent the evolution of the field and how it continues to move forward, not just in the coming years but in the coming decades.”