Overview of Neglected Tropical Diseases

Federico Gomez de las Heras

Abstract Neglected tropical diseases (NTDs), caused by a host of pathogens including helminthes, protozoa, bacteria, and viruses, remain a difficult and challenging area for drug discovery, being limited by meager resources and funding, challenged by gaps in knowledge of pathogen biology and drug targets, and faced with limitations specific to the developing world, including stringent cost of good constraints, the lack of robust infrastructure to effectively deliver and administer drug therapy, and socioeconomic considerations in disease endemic areas. This chapter will endeavor to provide a general overview of scientific approaches for NTDs, and review some of the current challenges and advancements in diseases of the developing world (DDW) drug discovery, as well as discuss the interplay between international networks of organizations, foundations, and public and private partners involved in neglected disease drug discovery. This chapter will also review general aspects and considerations common to many or all of these diseases, including burden of disease, coinfections, pathogenicity, drug resistance, drug combination therapy, and mass drug administration programs. This chapter will briefly address disease treatment on a general level, including the treatment of coinfections and care of special groups of patients, such as children and pregnant women.

Keywords Diseases of the developing world, Neglected tropical diseases, New drugs discovery and development, Product development partnership (PDP), Public-private partnership (PPP)


1 Introduction

2 The Diseases

2.1 Disease Burden

2.2 Pathogenicity .

F. Gomez de las Heras

Paseo de los Cerezos 5, 28016 Madrid, Spain e-mail: [email protected]

2.3 Epidemiologic Overlap of DDW: Coinfection

2.4 Unmet Needs

14 18

20 22

31 35 35 37

40 40 42

3 Current Status

3.1 General Socioeconomic Factors

3.2 New Commitments to Combat Neglected Diseases

3.3 Current Model for the R&D of New Drugs for DDW: PPPs

3.4 Incentives

4 Current Treatments

4.1 Nondrug Approaches

5 New DDW Drugs

5.1 Drug Discovery Strategies

5.2 R&D Drug Pipeline for DDWs

5.3 Issues and Opportunities

5.5 Potential Synergy Across Targets/Diseases

5.6 Criteria for Progression

6 Future Outlook/Conclusion


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