Completed Infection & Immunity Digestion, Kidneys & Other Organs

Development of novel, boron-containing small molecules for the treatment of Chagas disease

In plain English

AI plain-English summary

A single oral pill could replace the painful, weeks-long injections currently used to treat Chagas disease. This parasitic infection, spread by blood-sucking insects, affects millions of people across Latin America and beyond. The two existing drugs require prolonged treatment courses, cause severe side effects, and often fail to cure the chronic stage of the disease. Many patients abandon therapy as a result. This project aims to deliver a new compound, ready for Phase 2 clinical trials, that overcomes these shortcomings. The researchers are designing small molecules containing boron—a chemical element rarely used in antiparasitic drugs—to kill the *Trypanosoma cruzi* parasite more effectively and safely. They will use Target Product Profiles, a set of formal criteria based on patient needs, to guide development toward a once-daily oral medication with minimal toxicity. If successful, this compound could become the first modern treatment for Chagas disease that is both highly effective and practical to administer in resource-limited settings. It would replace a failing standard of care with a therapy patients can actually complete, potentially preventing the heart and digestive complications that kill tens of thousands of people each year.

View original technical description
Chagas disease (CD) is among the most neglected global diseases. Despite millions of affected people, treatments are limited with significant safety, efficacy, ease-of-use and cost deficiencies. Research and development has evolved in recent years; however, there is still a critical need for new therapeutic options. The goal of this project is to deliver a Phase 2-ready compound for the treatment of Chagas disease that addresses the significant deficiencies of current standard of care medications. We will use Target Product Profiles (TPPs) to ensure we deliver a compound that will meet patient needs (see Appendix 3 for TPP). The targeted clinical candidate will combine anti-protozoal efficacy with an acceptable safety profile and pharmacokinetics/physicochemical properties commensurate with oral delivery for testing against Chagas disease.

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Researchers

Robert Jacobs (EPMC Awardee)

Related Research

Grants with similar aims, by meaning.

Development of a preclinical candidate for Chagas disease that acts through inhibition of Trypanosoma cruzi squalene synthase
Optimising pharmacometric assessment in phase 2 studies of Chagas disease
Development of a new preclinical candidate for Chagas disease
Elucidating molecular determinants of Trypanosoma cruzi persisters.
Development of E-1224 for the Treatment of Chagas Disease.

Original classification

Seeding Drug Discovery Award

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