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Updated: Oct 28, 2025

Studying Cryptosporidium Infection in 3D Tissue-derived Human Organoid Culture Systems by Microinjection
Published on: September 14, 2019
Emerging treatment options for cryptosporidiosis
Melissa S Love1, Robert K M Choy2
1Calibr, a division of The Scripps Research Institute, La Jolla, California, USA.
Purpose Of Review:
Substantial progress has been made recently on the development of new therapeutics for cryptosporidiosis, an infection by the protozoan parasite Cryptosporidium that is associated with diarrhea, malnutrition, growth stunting, cognitive deficits, and oral vaccine failure in children living in low-resource settings.
Recent Findings:
Various drug discovery approaches have generated promising lead candidates. The repurposed antimycobacterial drug clofazimine was tested in Malawian HIV patients with cryptosporidiosis but was ineffective. Target-based screens identified inhibitors of lysyl-tRNA synthetase, phenylalanyl-tRNA synthetase, methionyl-tRNA synthetase, and calcium-dependent protein kinase 1. Phenotypic screens led to discovery of a phosphatidylinositol 4-kinase inhibitor, the piperazine MMV665917, and the benzoxaborole AN7973. The relationship between pharmacokinetic properties and in-vivo efficacy is gradually emerging. A pathway to clinical trials, regulatory approval, and introduction has been proposed but additional work is needed to strengthen the route.
Summary:
Several lead compounds with potent activity in animal models and a favorable safety profile have been identified. A sustained effort will be required to advance at least one to clinical proof-of-concept studies. The demonstrated risk of resistance indicates multiple candidates should be advanced as potential components of a combination therapy.
Insights
New therapeutics for cryptosporidiosis are advancing, with promising drug candidates identified through various discovery approaches. Further research is needed to move these treatments to clinical trials and combat drug resistance.
Area of Science:
- * Parasitology and Tropical Medicine
- * Drug Discovery and Development
Background:
- * Cryptosporidiosis, caused by the parasite Cryptosporidium, leads to severe health issues including diarrhea, malnutrition, and developmental problems in children.
- * It disproportionately affects children in low-resource settings, contributing to long-term health deficits and vaccine failure.
Purpose of the Study:
- * To review recent advancements in the development of novel therapeutics for cryptosporidiosis.
- * To identify promising drug candidates and outline potential pathways for clinical development and regulatory approval.
Main Methods:
- * Exploration of various drug discovery strategies, including target-based and phenotypic screening.
- * Evaluation of repurposed drugs and novel chemical entities against Cryptosporidium.
- * Assessment of lead compound activity in preclinical models and emerging pharmacokinetic data.
Main Results:
- * Identification of several promising lead compounds, including inhibitors of specific synthetases and kinases.
- * Testing of clofazimine in HIV patients with cryptosporidiosis showed ineffectiveness.
- * Emerging understanding of the link between pharmacokinetic properties and in vivo efficacy.
Conclusions:
- * Several compounds show potent activity and favorable safety profiles in preclinical studies.
- * Advancing at least one candidate to clinical proof-of-concept is crucial.
- * Developing combination therapies is recommended due to the risk of drug resistance.
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