Development of New Diagnostic and Treatment Options for Helminthic Neglected Diseases

Our project aims at developing new therapeutic and diagnostic tools to contribute to the control of Neglected deseases caused by helminth parasites, suchasystic (CE) and alveolar (AE) echinococcosis. Due to the scarcity of available anthelmintic drugs and the possible emergence of resistance, the discovery of new anthelmintic drugs is mandatory. Our international and interdisciplinary team has characterized a number of molecules that may play important roles in nutrient acquisition, attenuation of host’s immune response and development of these parasites and has also developed in vitro and in vivo models as well as studied epidemiological and clinical aspects of these diseases. We propose a new approach based on distinct biological and metabolic aspects of parasitic helminths, considering in particular parasite specific a) lipi binding proteisn (LBPs) and b) micro RNAs (miRNAs). These molecules are unique to these pathogens or highly divergent from the host counterparts and may exert essential functions, thus fulfilling the main requirements for good selective therapeutic targets. We will also assess their cellular expression in order to prioritize targets that are widely expressed, including in the keay stemcell population.The uniqueness/divergence of several miRNAs and the ability to be detected in biological fluids also makes them potential new specific biomarkers. Ultrasound studies and sera collection of human population affected by CE will be performed in order to include the potentially new biomarkers in the stagespecific approach according to WHOIWGE (Informal Working Group on Echinococcosis).

In addition, US surveys will provide a rapid impact on the health system of the rural populations involved in the study. Sera from AE patients are already available. We will evaluate specific parasite molecules as new therapeutic/diagnosis targets using bioinformatics, molecular biology, biochemistry and biophysical methods integrated with relevant clinical and epidemiological information. The expected outcome of the project is the development of new compounds that bind and inhibit essential and unique molecules of these parasites, and to find new detection tools, to improve the status of both treatment and early diagnosis of these complex and neglected diseases.