An investigational enzyme inhibitor may be able to cure malaria in a single dose when used in conjunction with another drug, and also be utilized as a preventative treatment, according to research findings in Science Translational Medicine.

Current first-line anti-malarial therapies include artemisinin-based combination therapies (ACTs), but there has been an increase in drug-resistant malaria parasites in Thailand, Cambodia, Vietnam, Myanmar, and Laos. The enzyme dihydroorotate dehydrogenase (DHODH) was first identified in 2008 as a requirement for the malaria to replicate; the compound DSM265 kills the malaria parasite Plasmodium in both liver and blood stages of infection.

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DSM265 is the first DHODH inhibitor to reach clinical development for treatment of malaria and will likely be paired with another new drug to combat drug resistance and used as a one-dose combination therapy. It could also be used as a once-weekly preventative medicine. In animal models it appears to be safely tolerated and the optimal dosing levels and length of drug effectiveness for humans has been estimated in preclinical models. Additional human studies are being planned for the drug as a treatment for malarial infection and prevention.

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