Related Experiment Video
Updated: Nov 10, 2025
Studying Cryptosporidium Infection in 3D Tissue-derived Human Organoid Culture Systems by Microinjection
Published on: September 14, 2019
Opportunities and Challenges in Developing a Cryptosporidium Controlled Human Infection Model for Testing
Rajiv S Jumani1, Johanne Blais1, Hanns-Christian Tillmann2
1Novartis Institute for Tropical Diseases, Novartis Institutes for BioMedical Research, Inc., Emeryville, California 94608-2916, United States.
Insights
A controlled human infection model for cryptosporidiosis (CHIM) can accelerate the development of new treatments for children. This approach evaluates drug safety and efficacy in adults before pediatric trials.
Area of Science:
- * Infectious Diseases
- * Pediatric Gastroenterology
- * Parasitology
Background:
- * Cryptosporidiosis is a major cause of childhood diarrhea and mortality in low- and middle-income countries, linked to malnutrition and stunting.
- * Current treatments for cryptosporidiosis are inadequate, especially for young children, and no vaccine is available.
- * Novel anti-Cryptosporidium agents show promise in preclinical studies, but clinical validation is needed.
Purpose of the Study:
- * To explore the potential of a Cryptosporidium controlled human infection model (CHIM) for evaluating new therapeutic agents.
- * To assess the feasibility of using CHIM to establish safety and preliminary efficacy of novel treatments in adults before pediatric studies.
Main Methods:
- * Discussion of the opportunities and challenges associated with establishing and utilizing a Cryptosporidium CHIM.
- * Focus on the model's utility for proof-of-concept studies in healthy adult volunteers.
Main Results:
- * A CHIM model offers a potential pathway to accelerate the development of new cryptosporidiosis therapies.
- * Establishing safety and preliminary efficacy in adults via CHIM can inform pediatric trial design.
Conclusions:
- * The Cryptosporidium CHIM presents a valuable strategy for advancing novel therapeutics towards pediatric clinical studies.
- * This model can de-risk drug development by providing early clinical data on safety and efficacy.
- * Further development and validation of the CHIM are crucial for its successful implementation.
Abstract:
Cryptosporidiosis is a leading cause of moderate-to-severe diarrhea in low- and middle-income countries, responsible for high mortality in children younger than two years of age, and it is also strongly associated with childhood malnutrition and growth stunting. There is no vaccine for cryptosporidiosis and existing therapeutic options are suboptimal to prevent morbidity and mortality in young children. Recently, novel therapeutic agents have been discovered through high-throughput phenotypic and target-based screening strategies, repurposing malaria hits, etc., and these agents have a promising preclinical in vitro and in vivo anti-Cryptosporidium efficacy. One key step in bringing safe and effective new therapies to young vulnerable children is the establishment of some prospect of direct benefit before initiating pediatric clinical studies. A Cryptosporidium controlled human infection model (CHIM) in healthy adult volunteers can be a robust clinical proof of concept model for evaluating novel therapeutics. CHIM could potentially accelerate the development path to pediatric studies by establishing the safety of a proposed pediatric dosing regimen and documenting preliminary efficacy in adults. We present, here, perspectives regarding the opportunities and perceived challenges with the Cryptosporidium human challenge model.
