BACKGROUND:  Parasitic diseases continue to take an enormous toll on human and animal health worldwide. One of the most prominent groups of parasites are the apicomplexans, a large group of obligate intracellular parasites that cause a wide array of diseases of medical and veterinary importance. There are over 5000 different species of apicomplexans, but the most notable of these is Plasmodium falciparum, the causative agent of malaria that kills 1-2 million people each year. Toxoplasma gondii is another important apicomplexan which infects approximately one-quarter of the world's human population and causes severe central nervous system disorders of immunocompromised (AIDS/transplant/lymphoma) individuals, birth defects in congenitally infected neonates, and ocular disease in immunocompetent persons. Other Apicomplexans that cause disease in humans include the opportunistic pathogens Cryptosporidia spp. and Isospora belli. Important veterinary pathogens include Neospora caninum (a pathogen of dogs/cattle), Theileria spp (cattle), and Eimeria spp (poultry). Currently there are no effective vaccines and few effective treatments available for most diseases caused by these parasites. In addition, current anti-parasite therapies target biosynthetic pathways in the parasite and are prone to mutations leading to rapidly developing resistance in this widespread group of pathogens.

INNOVATION:  Researchers at UCLA have discovered a small molecule that is highly effective in inhibiting attachment, invasion and motility of apicomplexan parasites. This molecule functions by blocking lipid fusion events, indicating that this small molecule inhibitor blocks the secretion of parasite proteins required for attachment, invasion and motility, thereby opening an entirely new approach for controlling Toxoplasma and related parasites.

POTENTIAL APPLICATIONS 

• Therapeutic treatment in human and animals of infections of a wide array of apicomplexan parasites.
• Microbicide to combat infections from numerous insect hosts (e.g. mosquitoes, ticks) of apicomplexan parasite.

ADVANTAGES

• Targets membrane fusion events thereby effective against a wide array of apicomplexan parasites.
• Parasites are less likely to develop resistance to this drug because disruption of membrane fusion events is lethal.
• Prevent parasite infestations that exert a major impact on levels of productivity and profitability.
• Tap into the single most valuable sector of animal health products, a $5 billion multibillion dollar market, offering tremendous profit potential.
• No apparent toxicity in vitro or in vivo.

DEVELOPMENT-TO-DATE:  The compound's effects on Toxoplasma motility, attachment and invasion have been studied in vitro.

Related Papers (Selected)

• Targeting purine and pyrimidine metabolism in human apicomplexan parasites. Hyde JE. Curr Drug Targets. 2007 Jan;8(1):31-47. Review. [more]
• The plastid-derived organelle of protozoan human parasites as a target of established and emerging drugs. Wiesner J, Seeber F. Expert Opin Ther Targets. 2005 Feb;9(1):23-44. [more]

Reference: UCLA Case No. 2009-769