Closing the Treatment Divide micr scope THE under
Bridging the Gap Between Pediatric and Adult IBD Care
Critical Studies That Push Pediatrics
Exploring Food-Based Treatment For Kids
Spotlight: Dr. Neera Gupta
Closing the Treatment Divide: Bridging the Gap Between Pediatric
and Adult IBD
Care
Since 2009 pediatric Crohn’s and colitis diagnoses have risen by 22% and 29% respectively
A2024 study published in Gastroenterology by the Crohn’s & Colitis Foundation and the Universities of North Carolina and Pennsylvania revealed a sharp rise in U.S. pediatric IBD rates since 2009 (the prevalence of Crohn’s has risen 22 percent and ulcerative colitis, 29 percent). Yet a recent open letter from leading pediatric gastroenterologists to the FDA highlights a bigger issue: Despite the availability of new biologics and small-molecule drugs for adults, pediatric patients face significant treatment delays due to restrictive regulatory processes and limited industry incentives, leaving them “therapeutic orphans” in the shadow of two decades of IBD treatment advancements for adults.
“The letter underscores major approval gaps,” says Alan Moss, chief scientific officer, Crohn’s & Colitis Foundation. “For example, infliximab (Remicade®) and adalimumab (Humira®) were approved for pediatric Crohn’s disease 6-8 years after adults, and no new advanced therapies have been approved for children in over a decade.” And unlike European and Japanese regulators, Moss says the U.S. system “forces clinicians
to rely on off-label treatments without proper pediatric dosing and safety data, leaving the FDA lagging and failing thousands of children annually.”
While the letter urges stronger post-market safety monitoring and “full extrapolation” (i.e., applying adult trial effectiveness data to pediatric patients more quickly), the Foundation is doing its part in full force to advance pediatric IBD research. Here is a snapshot of several innovative studies aimed at closing the treatment gap for the country’s youngest IBD patients.
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...no new advanced therapies have been approved for children in over a decade.
The for Pediatrics Push
Here’s how Foundation studies are advancing pediatric IBD treatment to match adult care.
PIANO: Study Transforms Pregnancy Care for Women with IBD
The PIANO Study (Pregnancy in Inflammatory Bowel Disease and Neonatal Outcomes), launched in 2007, is the largest prospective study of biologic safety in pregnancy for IBD patients. Tracking over 2,200 pregnant women, PIANO has shown that biologic and thiopurine therapies (that reduce inflammation and slow the immune system) do not increase risks of congenital malformations, preterm birth, low birth weight, or neonatal infections. These findings have reshaped treatment guidelines, reassuring expectant mothers with IBD about medication safety during pregnancy.
Natalie Hayden, a Crohn’s & Colitis Foundation Patient Advocate (pictured with her family at right), IBD mom, blogger, and 2024 Lead Patient Ambassador for the Global Consensus Conference on IBD and Pregnancy, has actively contributed to this research. During her third pregnancy, she provided blood and cord blood samples to PIANO, which will monitor her son Connor’s development until age 18. “The timeline for clinical research is long, but the impact is lasting,” says Hayden, who has advocated for IBD research both at the FDA and during the Advances in IBD conference alongside Dr. Uma Mahadevan. “It’s a win-win to participate in this life-changing research that provides hope and clarity for future families while giving me comfort about my own family’s health.”
CAPTURE IBD: Shaping Pediatric Care
CAPTURE IBD (the Cohort for Pediatric Translational and Clinical Research in IBD) is a longitudinal study designed to advance precision medicine for children and young adults with IBD.
CAPTURE IBD participants will receive care from their IBD physician and provide health information, blood, stool, and tissue samples during routine medical visits. This data is integrated into the Foundation’s IBD Plexus® research accelerator, the most comprehensive collection of IBD patient data and biosamples in the world. Researchers can use this information to better understand IBD and treatment effectiveness in children and young adults.
“Much of what informs pediatric IBD care is derived from adult studies,” says Alandra Weaver, vice president, Clinical Quality and Research Innovation. “We need to address this gap to meaningfully advance pediatric research and care.”
PIANO continues to evaluate newer IBD medications and their effects during pregnancy and breastfeeding.
Launched in April 2024 with 12 diverse sites across the U.S., the study aims to enroll 2,000 children and young adults, tracking clinical, molecular, and patient-reported data over time. This study is generously supported by the David R. Clare & Margaret C. Clare Foundation.
RISK Stratification Study: A Breakthrough for Kids with Crohn’s
The Pediatric RISK Stratification Study is the largest new-onset pediatric Crohn’s disease study ever conducted, tracking 1,800 newly diagnosed children across 28 clinics across the U.S. and Canada over a 13-year, $10+ million effort by the Foundation. The study focused on 913 children who remained complication-free within 90 days of diagnosis, and followed for 8 years collecting biosamples (blood, stool, biopsies) and clinical, demographic, immunological, and genetic data to identify predictive markers for severe disease progression.
A key finding indicates that initiating early biologic therapy, within 90 days after diagnosis of Crohn’s disease, prevented the development of fistulas (abnormal connections between the intestine and other organs) a severe complication that requires surgery, supporting the notion that early biologic treatment can enhance the quality of life for young patients. The study also uncovered mRNA- based biomarkers that help predict which children are most likely to develop these complications.
The Foundation is developing an AI-based prognostic blood test to predict disease progression and guide treatment decisions for pediatric Crohn’s disease at diagnosis. Using RISK biosamples, Foundation scientists in collaboration with Genedata and Olink have discovered novel prognostic biomarkers and developed a prototype prognostic test, now undergoing patenting with the aim of licensing this technology to test developers for clinical use.
VEOIBD: Cracking the Code of Childhood Diagnoses
Dr. Lauren Collen is a pediatric gastroenterologist at Boston Children’s Hospital who is leading groundbreaking research on very early onset inflammatory bowel disease (VEOIBD), a rare and often treatment-resistant form of IBD diagnosed in children under 6. Her study funded by a Foundation’s research fellowship award (RFA) focuses on five patients with defective STAT3 activation, a key molecule in immune regulation. Using advanced techniques like RNA sequencing and CyTOF, Dr. Collen is identifying unique genetic and protein signatures that may help define new subgroups of IBD patients and guide individualized treatment strategies.
Now in its second year of funding, Dr. Collen’s work has already yielded promising insights. Early findings suggest that some patients with STAT3 defects respond well to IL-23 blockade therapy — an observation that could revolutionize targeted treatments for both pediatric and adult IBD cases. Her research also explores whether STAT3 signaling patterns can serve as biomarkers to predict treatment responses before patients endure severe disease progression. With five research awards, seven publications, and a provisional patent on a transcriptional signature linked to ustekinumab (Stelara®) response, Dr. Collen’s work represents a major step toward understanding and treating the most complex cases of pediatric IBD. This work is generously supported by The Palmer Foundation.
Beyond Liquid Diets: Exploring New Food-Based IBD Treatment for Kids
Funded by the Foundation’s Precision Nutrition Initiative, researchers at Glasgow University, led by Konstantinos Gerasimidis, Ph.D., are studying how diet may help children with Crohn’s disease stay in remission after completing enteral nutrition (EEN) — a liquid-only diet often used to induce remission.
Traditionally, achieving remission involves eight weeks on the regimen, and while effective, EEN is difficult to maintain, especially for children. To address this challenge, the team is investigating CDTREAT, a whole-food-based diet designed to mimic EEN’s positive effects on the gut microbiome. By limiting specific components like gluten, fiber, and food additives, CD-TREAT aims to reduce inflammation and keep children in remission without requiring a feeding tube.
In addition to testing whether CD-TREAT can sustain remission, researchers are working to identify microbial and immunological markers that could predict how children will respond to dietary triggers or this diet plan. Their findings could lead to personalized nutrition strategies, giving pediatric patients a sustainable, food-based approach to managing Crohn’s disease and improving long-term quality of life.
Dr. Neera Gupta
Advancing Sex-Specific Approaches to Pediatric IBD Therapy
Pediatric Crohn’s disease treatment hasn’t historically accounted for a patient’s sex, but Neera Gupta, MD, MAS, is changing that. As Director of the Growth Program for Inflammatory Bowel Diseases at Children’s National Hospital, Dr. Gupta is investigating why boys experience growth impairment more frequently than girls. Her research study “Sex Differences in Statural Growth Impairment in Pediatric Crohn’s Disease” (aka: The Growth Study) has uncovered sex-specific molecular pathways leading to statural growth impairment that appear to influence response to therapy. Mapping these pathways will help develop sex-specific treatment approaches that could transform care.
“In 10 years, we will be saying, ‘Of course we should be managing males and females differently for many different chronic inflammatory diseases. Why would we be treating them the same?’”
Dr. Gupta’s passion for pediatric IBD began during residency when she was captivated by both the children she treated and IBD’s complexities. Her message to the IBD community? “It’s important for families to ask their providers about their child’s growth status at every visit.”
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Foundation Resources for Pediatric IBD Patients
Visit crohnscolitisfoundation.org for these helpful tools (plus, insurance information, school accommodations templates, pediatric appeal letters, and more).
IBDKIDZ - Support groups for kids and parents
IBD & Me - Resources for young people
Just Like Me - Just for teens
Campus Connection - For college students
Camp Oasis - Sleepaway summer camp for kids with IBD
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The Crohn’s & Colitis Foundation is the leading nonprofit organization focused on research, education, and support for the millions of Americans affected by inflammatory bowel disease (IBD). We have played a role in every major research breakthrough in IBD and are the trusted source for IBD patients and caregivers, educating, guiding, and supporting them every step of the way.