Manjunatha, Ujjini, Jumani, Rajiv Satish, Blais, Johanne, Tillmann, Hanns-Christian, Segal, Florencia, Wetty, Dean, Lakshman, Jay, Aziz, Natasha, Chandra, Richa, Ostermeier, Christian, Nuber, Natko, Chen, Wilbur, Chappell, Cynthia and Diagana, Thierry (2021) Opportunities and challenges in developing a Cryptosporidium controlled human infection model for testing novel anti-parasitic agents. ACS infectious diseases, 7. pp. 959-968. ISSN 2373-8227

Abstract

Cryptosporidiosis is a leading cause of moderate-to-severe diarrhea in the developing countries, responsible for high mortality in children younger than two years of age, and also strongly associated with childhood malnutrition and growth stunting. There is no vaccine for cryptosporidiosis and no effective chemotherapeutic options are available to prevent morbidity and mortality in young children. Recently, novel therapeutic agents have been discovered through high-throughput through 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. A challenge to bringing safe and effective new therapies to young vulnerable children in the clinic is the ethical obligation to establish some prospect of benefit before initiating pediatric studies, especially in low- and middle-income populations. 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 some perspectives on the opportunities and perceived challenges with Cryptosporidium CHIM.

Item Type:	Article
Date Deposited:	10 Jul 2021 00:45
Last Modified:	10 Jul 2021 00:45
URI:	https://oak.novartis.com/id/eprint/44247