Drug discovery and human African trypanosomiasis: a disease less neglected?

Future Med Chem. 2013 Oct;5(15):1801-41. doi: 10.4155/fmc.13.162.

Abstract

Human African trypanosomiasis (HAT) has been neglected for a long time. The most recent drug to treat this disease, eflornithine, was approved by the US FDA in 2000. Current treatments exhibit numerous problematic side effects and are often ineffective against the debilitating CNS resident stage of the disease. Fortunately, several partnerships and initiatives have been formed over the last 20 years in an effort to eradicate HAT, along with a number of other neglected diseases. This has led to an increasing number of foundations and research institutions that are currently working on the development of new drugs for HAT and tools with which to diagnose and treat patients. New biochemical pathways as therapeutic targets are emerging, accompanied by increasing numbers of new antitrypanosomal compound classes. The future looks promising that this collaborative approach will facilitate eagerly awaited breakthroughs in the treatment of HAT.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Clinical Trials as Topic
  • Drug Evaluation, Preclinical
  • Eflornithine / therapeutic use
  • Humans
  • Neglected Diseases / drug therapy
  • Small Molecule Libraries / chemistry
  • Small Molecule Libraries / pharmacology
  • Trypanocidal Agents / chemistry*
  • Trypanocidal Agents / pharmacology
  • Trypanocidal Agents / therapeutic use
  • Trypanosoma / drug effects
  • Trypanosomiasis, African / drug therapy

Substances

  • Small Molecule Libraries
  • Trypanocidal Agents
  • Eflornithine