Avoiding Clinical Trial Failures in Neglected Tropical Diseases: The Example of Chagas Disease

Area of Science:

• Drug discovery and development
• Neglected tropical diseases
• Clinical trial design

Background:

• Human clinical trials are expensive and failures can deter future research, especially for neglected tropical diseases.
• Chagas disease drug discovery has faced numerous human clinical trial failures.
• Effective strategies are needed to improve the success rate of clinical trials for neglected diseases.

Purpose of the Study:

• To provide guidelines for de-risking drug candidates in Chagas disease before human clinical trials.
• To address challenges and leverage advantages in Chagas disease research to accelerate patient access to new treatments.
• To offer insights applicable to other neglected tropical diseases.

Main Methods:

• Exploiting multi-species natural infection systems to pre-clinically evaluate drug compounds.
• Analyzing major challenges in Chagas disease, such as the lack of a definitive cure test.
• Acknowledging and addressing specific hurdles in drug development for neglected tropical diseases.

Main Results:

• Identification of Chagas disease's unique advantages, particularly its multi-species infection models for pre-clinical de-risking.
• Recognition of significant challenges, including the absence of a reliable test for cure.
• Development of guidelines to improve the efficiency and success of drug discovery for Chagas disease.

Conclusions:

• Fully utilizing pre-clinical advantages like multi-species models is crucial for Chagas disease drug discovery.
• Addressing inherent challenges proactively can lead to better therapeutic options for patients.
• The proposed guidelines can potentially benefit other neglected tropical diseases.