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The pipeline for drugs for control and elimination of neglected tropical diseases: 2. Oral anti-infective drugs and drug combinations for off-label use.

Parasit Vectors

October 2023

UNICEF/UNDP/World Bank/WHO Special Programme for Research and Training in Tropical Diseases (WHO/TDR), World Health Organization, Geneva, Switzerland.

Article Synopsis
  • The World Health Organization (WHO) laid out its objectives for controlling and eliminating neglected tropical diseases (NTDs) in a 2021-2030 roadmap, emphasizing the need for new treatment options and research.
  • A review of current anti-infective drug developments reveals that the existing pipeline cannot meet all treatment needs for NTDs, and WHO allows for off-label drug use based on safety and efficacy.
  • The article summarizes the lack of clinical research over the past decade on oral small-molecule drugs for numerous NTDs that are neither approved by regulatory authorities nor included in WHO strategies, identifying specific diseases and drugs that have not been thoroughly evaluated.
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Schistosomiasis is a disease caused by parasitic trematode worms (schistosomes) that currently affects 200 million people living in tropical and subtropical environments. It is a chronic disease and the latest estimates for sub-Saharan Africa are that it kills > 200000 people every year. Soil-transmitted helminthiasis (STH) is caused by intestinal nematodes.

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Chemotherapy-induced, age-related changes in antischistosome antibody responses.

Parasite Immunol

February 2003

Comparative Epidemiology and Informatics, Department of Veterinary Clinical Studies, University of Glasgow Veterinary School, Bearsden Road, UK.

Humoral responses directed against Schistosoma mansoni soluble egg antigen were studied in Zimbabwean children before and after treatment with either praziquantel (PZQ) or oxamniquine (OXAM). Treated children showed a significant increase in the proportion producing IgE and IgG3 and in mean levels of IgE, IgM, IgG3 six weeks post-treatment. At 18 weeks post-treatment, the proportion of treated children producing IgA, IgE, and IgG3 increased while the proportion producing IgG1 and IgG4 decreased.

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Human schistosomiasis, a chronic and debilitating parasitic disease of the tropics, is ranked second after malaria in terms of public health importance. At present, there is no vaccine available, and chemotherapy is the cornerstone of schistosomiasis control. Praziquantel is the drug of choice.

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Oxamniquine, at 40 mg/kg body weight, was used in a pilot schistosomiasis control programme in an endemic community in Ethiopia. After mass screening of the population using Kato's thick smear method, 1556 positive patients were treated in divided doses over two consecutive days. However, only 1183 (76%) persons completed the total dose.

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