Research Horizons


Biological Agent Successfully Treats Eosinophilic Esophagitis 

Pathology | Top Scientific Achievement
2023 Research Discoveries Marc Rothenberg, MD, PhD, with EoE patient Aidan.

Benefits of weekly doses of dupilumab detailed in The New England Journal of Medicine

In May 2022, the FDA approved the first drug for eosinophilic esophagitis (EoE), a severe allergic disease in which the body treats the majority of foods as a harmful invader. This drug, dupilumab, blocks the signaling of key immune proteins, interleukins 4 and 13 (IL-4 and IL-13), which have essential roles in EoE.   

Scientists at Cincinnati Children’s took part in an international clinical trial of dupilumab in adolescents and adults with EoE. The findingspublished Dec. 21, 2022, in The New England Journal of Medicine—are the first to demonstrate that dupilumab is effective in treating EoE.   

Histology (examination of microanatomy) and symptoms improved when dupilumab was given once a week, which is now the FDA-approved dosing regimen for dupilumab for EoE.  


“These findings from this three-part clinical trial are already benefiting patients and clinical practice,” says co-first author Marc Rothenberg, MD, PhD, director of the Division of Allergy and Immunology and the Center for Eosinophilic Diseases at Cincinnati Children’s and principal investigator of the Consortium of Eosinophilic Gastrointestinal Disease Researchers (CEGIR). “I am pleased with the strongly positive data that this trial yielded, supporting the use of this FDA-approved therapeutic while we continue researching even better treatments and a cure for eosinophilic esophagitis.”  

Illustration tracks the cascading biological process that occurs when foods trigger an eosinophilic esophageal reaction and pinpoints how the recently approved drug dupilumab helps improve disease symptoms.

For the first part of this phase III clinical trial, adolescents and adults aged 12 years and older with EoE were randomized to 24 weeks of weekly subcutaneous dupilumab (300 mg) or placebo. Results from this first part were promising, as histologic remission was achieved in 59.5% for dupilumab versus 5.1% for placebo.  

For the second part of this trial, adolescents and adults with EoE were randomized to 24 weeks of weekly dupilumab (300 mg), biweekly dupilumab (300 mg), or placebo. Histologic remission was achieved in:  

  • 58.8% of patients who were administered dupilumab on a weekly basis
  • 60.5% of patients who were administered dupilumab biweekly
  • 6.3% of patients who were administered a placebo.   

However, patient symptoms did not improve by the end of the 24 weeks with biweekly dupilumab despite the histologic signs of the disease improving.   

For the third part of the study, patients who continued to receive weekly dosing of dupilumab for an additional 24 weeks showed sustained improvements. 

Chart illustrates that once-weekly dosing of dupilumab showed clear improvements in EoE symptoms, as measured by the Dysphagia Symptom Questionnaire (DSQ).

“This study leveraged two decades of translational science from my research laboratory, including findings supporting EoE being mediated by type 2 immune responses and the involvement of interleukins, including interleukin 13,” says Rothenberg. “We’re grateful to the academic researchers, industry partners, and patient and family collaborators, who have all made this work possible.”  

Looking ahead, the results of this trial will continue to improve how dupilumab is used in clinical practice, ultimately affecting better outcomes for children and adults with EoE. 

Dr. Rothenberg and colleagues at Cincinnati Children's have played central roles from the beginning of the EoE journey; from establishing the condition as its own disease to characterizing the genetic and molecular pathways involved to co-leading the clinical trials that led to the FDA approval of dupilumab.

In addition to Rothenberg, Cincinnati Children’s co-authors included Margaret Collins, MD.  

This clinical trial was funded by Sanofi and Regeneron Pharmaceuticals, Inc. ( number, NCT03633617)

Learn More  

Read more about EoE research at the Rothenberg CURED Lab   

Read more about the FDA approval of Dupilumab for EoE 

More 2023 Research Highlights

Chosen by the Division of Pathology

Puneet Agarwal, Avery Sampson, Kathleen Hueneman, Kwangmin Choi, Xueheng Zhao, Jessica Galloway-Pena, Kenneth Setchell, Daniel T. Starczynowski; A Circulating Microbial Metabolite Drives the Clonal Expansion of Pre-Leukemic Cells. Blood 2022; 140 (Supplement 1): 975–976. doi: 

Sumazin P, Peters TL, Sarabia SF, et al. Hepatoblastomas with carcinoma features represent a biological spectrum of aggressive neoplasms in children and young adults. J Hepatol. 2022;77(4):1026-1037. doi:10.1016/j.jhep.2022.04.035 

Bouffi C, Wikenheiser-Brokamp KA, Chaturvedi P, et al. In vivo development of immune tissue in human intestinal organoids transplanted into humanized mice. Nat Biotechnol. 2023;41(6):824-831. doi:10.1038/s41587-022-01558-x 

Wu LMN, Zhang F, Rao R, et al. Single-cell multiomics identifies clinically relevant mesenchymal stem-like cells and key regulators for MPNST malignancy. Sci Adv. 2022;8(44):eabo5442. doi:10.1126/sciadv.abo5442 


View more discoveries from 50 research divisions and areas

Return to the 2023 Research Annual Report main features

Publication Information
Original title: Dupilumab in Adult and Adolescent Patients With Eosinophilic Esophagitis
Published in: The New England Journal of Medicine
Publish date: Dec. 21, 2022
Read the Study

Research By

Marc Rothenberg, MD, PhD
Marc Rothenberg, MD, PhD
Director, Division of Allergy and Immunology

The Rothenberg CURED Research Laboratory, supported by the Campaign Urging Research for Eosinophilic Diseases (CURED), is focused on elucidating the mechanisms of allergic responses, especially in mucosal tissues such as the gastrointestinal tract and lung.