Progress in antileishmanial drugs: Mechanisms, challenges, and prospects

PLoS Negl Trop Dis. 2025 Jan 3;19(1):e0012735. doi: 10.1371/journal.pntd.0012735. eCollection 2025 Jan.

Abstract

Leishmaniasis, a neglected tropical disease caused by Leishmania parasites, continues to pose global health challenges. Current treatments face issues like resistance, safety, efficacy, and cost. This review covers the discovery, mechanisms of action, clinical applications, and limitations of key antileishmanial agents: pentavalent antimonials, amphotericin B, miltefosine, paromomycin, and pentamidine. Despite toxicity and resistance (antimonials), hospitalization needs and side effects (amphotericin B), regional efficacy variability (miltefosine), inconsistent outcomes (paromomycin), and severe side effects (pentamidine), these drugs are vital. Novel strategies to overcome the deficiencies of current therapies are highlighted, including combination regimens, advanced drug delivery systems, and immunomodulatory approaches. Comprehensive and cooperative efforts are crucial to fully realize the potential of advancements in antileishmanial pharmacotherapy and to reduce the unacceptable worldwide burden imposed by this neglected disease.

Publication types

  • Review

MeSH terms

  • Amphotericin B / pharmacology
  • Amphotericin B / therapeutic use
  • Animals
  • Antiprotozoal Agents* / pharmacology
  • Antiprotozoal Agents* / therapeutic use
  • Humans
  • Leishmania* / drug effects
  • Leishmaniasis* / drug therapy
  • Neglected Diseases / drug therapy
  • Paromomycin / pharmacology
  • Paromomycin / therapeutic use
  • Pentamidine / pharmacology
  • Pentamidine / therapeutic use
  • Phosphorylcholine / analogs & derivatives
  • Phosphorylcholine / pharmacology
  • Phosphorylcholine / therapeutic use

Substances

  • Antiprotozoal Agents
  • Amphotericin B
  • Pentamidine
  • miltefosine
  • Paromomycin
  • Phosphorylcholine

Grants and funding

This work was supported by the National Key Research and Development Program of China [2021YFC2600200 and 2022YFC2305500 to Y.L.]; the General Program of National Natural Science Foundation of China [82172299 to Y.L.]; the Hubei Natural Science Fund for Distinguished Young Scholars [2022CFA068 to Y.L.], and the Hubei Public Health Youth Talents Program [to Y.L.].The funders had no role in study design, data collection and analysis, decision to publish, or preparation of the manuscript.