The invention consists of compounds for the treatment of parasitic diseases such as Trypanosomiasis and Leishmaniasis. Compounds are first-in-class, and selectively targeted towards pathogenic cells with nanomolar potency.
Trypanosoma brucei, which lead to the African sleeping sickness disease in humans, is widespread and infects over 2 million people worldwide. Therapeutic treatments for this disease are limited due to toxicity of the compounds, and the emergence of drug-resistant parasites. This invention overcomes limitations to current therapeutic options for the treatment of this disease. The compounds in this invention specifically target parasites that have a specific organelle that contain DNA material. These compounds were originally identified from a panel of compounds with activity in mammalian cells. Many analogs were generated that demonstrated low nanomolar potency against the parasites. The compounds are highly selective for parasites and useful for therapeutic development.
Therapeutic treatment for infectious diseases.
The therapeutic compounds in this invention activate an adaptive mechanism in mammalian cells that induces death of the parasitic cell.
The compounds are potent anti-parasitic drugs, with no effects on mammalian cells. The invention represents an advancement in the treatment of African sleeping sickness and Leishmaniasis, which affect millions of people around the world. The compounds are relatively easy and cheap to manufacture with broad spectrum activity in a number of infectious disease-related parasites.
A major challenge in the treatment of parasitic infections (e.g., African sleeping sickness, leishmaniasis, and malaria) is the development of resistance to the current treatments. Since the compounds presented here work by activating the 'normal' cell to fight off the infectious cell, this treatment avoids the processes that lead to drug resistance when using conventional treatments.
Data also showed that compounds can be used as therapeutics against several other human diseases.