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Updated: Jun 25, 2026

Quantitative Analysis of Autophagy using Advanced 3D Fluorescence Microscopy
Published on: May 3, 2013
Autophagy Agents in Clinical Trials for Cancer Therapy: A Brief Review
Samiha Mohsen1, Philip T Sobash2, Ghada Fahad Algwaiz3
1Faculty of Medicine, University of Toronto, Toronto, ON M5S 1A8, Canada.
Abstract:
Autophagy has been of novel interest since it was first demonstrated to have effect in Burkitt's lymphoma. Since that time, the autophagy agents chloroquine and hydroxychloroquine have become the only FDA (Food and Drug Administration)-approved autophagy inhibitors. While not approved for cancer therapy, there are ongoing clinical trials to evaluate their safety and efficacy. Pevonedistat has emerged as a novel inhibitor through the neddylation pathway and is an autophagy activator. This paper summarizes and presents current clinical trials for hydroxychloroquine (HCQ), chloroquine (CQ), and Pevonedistat for the clinician.
Insights
Autophagy research is advancing, with chloroquine and hydroxychloroquine as approved inhibitors and Pevonedistat as an activator. Current clinical trials are evaluating these agents for potential therapeutic applications.
Area of Science:
- Oncology
- Cell Biology
- Pharmacology
Background:
- Autophagy, a cellular process, gained interest following its effects in Burkitt's lymphoma.
- Chloroquine (CQ) and hydroxychloroquine (HCQ) are FDA-approved autophagy inhibitors, with ongoing trials for cancer therapy.
- Pevonedistat is a novel autophagy activator targeting the neddylation pathway.
Purpose of the Study:
- To summarize and present current clinical trials for autophagy modulators.
- To provide an overview of hydroxychloroquine (HCQ), chloroquine (CQ), and Pevonedistat in clinical development.
- To inform clinicians about the status of these agents in cancer research.
Main Methods:
- Review of ongoing and recently completed clinical trials.
- Analysis of data on safety and efficacy of HCQ, CQ, and Pevonedistat.
- Literature search for relevant studies on autophagy inhibitors and activators.
Main Results:
- Ongoing trials are assessing the safety and efficacy of HCQ and CQ in various cancer types.
- Pevonedistat is being investigated for its role as an autophagy activator in clinical settings.
- The clinical utility of these autophagy modulators is under active investigation.
Conclusions:
- Hydroxychloroquine, chloroquine, and Pevonedistat represent key agents in the ongoing exploration of autophagy modulation for cancer treatment.
- Clinicians should be aware of the current clinical trial landscape for these compounds.
- Further research is necessary to establish the definitive role of these agents in oncological therapies.
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