Novel Compounds for Treatment of Parasitic Infections

African Trypanosomiasis, also known as “sleeping sickness,” is caused by microscopic parasites transmitted to humans by the Tsetse fly, found in rural Africa. It is a serious public health problem in sub-Saharan Africa, with around 10,000 new cases each year. This serious disease is fatal if left untreated, however, the currently available treatments are incredibly difficult to administer, and can be extremely toxic to the patient. Melarsoprol, one of the primary drugs used to treat late stage disease, is an arsenic derivative that is lethal to around 5% of patients who receive it. The global market for treatment of neglected tropical diseases was valued at $1.2 billion in 2018 and is expected to rise to $2.6 billion by 2021. The patient need, combined with global warming increasing the spread of tropical diseases north and southward, has created a strong demand for new solutions. Clemson researchers have developed novel compounds that are active against this devastating pathogen, offering an avenue for dramatically improved treatment.

Application                                                                   Stage of Development

Drug discovery, Neglected tropical                            Preliminary Data

disease treatments

Advantages

• Novel synthesis pathway is simple and high yielding, resulting in cheaper production costs and high volumes produced
• Novel compounds have never been used for treatment of any prior disease, reducing acquired pathogen resistance to practically zero
• Compounds are active against Trypanosoma brucei, but non toxic to mammalian cells, providing improved targeting and less patient toxicity than previously available treatments

Technical Summary

These compounds are diazocyclobutenes, synthesized via a novel and simple method, and could potentially be active against many different diseases. The compounds are currently demonstrated to be efficacious against Trypanosoma brucei, and have a superior safety profile to some of the currently available drugs. They are active in the nano- to micro-molar range, which is crucial for the development of a pharmaceutical, and although the structure-function relationship is not well understood, they are non-toxic to mammalian cells, a drastic improvement over the arsenic-derived compounds currently used to treat the disease. 

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Inventor:                       Daniel Whitehead, Jim Morris

Patent Type:                  Provisional

Serial Number:             62/817,150

CURF Ref No:              2019-019