Strategies and Challenges in Clinical Trials Targeting Human Aging

John C. Newman, Sofiya Milman, Shahrukh K. Hashmi, Steve N. Austad, James L. Kirkland, Jeffrey B. Halter, Nir Barzilai

Research output: Contribution to journalReview articlepeer-review

73 Scopus citations


Interventions that target fundamental aging processes have the potential to transform human health and health care. A variety of candidate drugs have emerged from basic and translational research that may target aging processes. Some of these drugs are already in clinical use for other purposes, such as metformin and rapamycin. However, designing clinical trials to test interventions that target the aging process poses a unique set of challenges. This paper summarizes the outcomes of an international meeting co-ordinated by the NIH-funded Geroscience Network to further the goal of developing a translational pipeline to move candidate compounds through clinical trials and ultimately into use. We review the evidence that some drugs already in clinical use may target fundamental aging processes. We discuss the design principles of clinical trials to test such interventions in humans, including study populations, interventions, and outcomes. As examples, we offer several scenarios for potential clinical trials centered on the concepts of health span (delayed multimorbidity and functional decline) and resilience (response to or recovery from an acute health stress). Finally, we describe how this discussion helped inform the design of the proposed Targeting Aging with Metformin study.

Original languageEnglish (US)
Pages (from-to)1424-1434
Number of pages11
JournalJournals of Gerontology - Series A Biological Sciences and Medical Sciences
Issue number11
StatePublished - Nov 1 2016


  • Acarbose
  • Aging
  • Clinical trials
  • Metformin
  • Rapamycin

ASJC Scopus subject areas

  • Aging
  • Geriatrics and Gerontology


Dive into the research topics of 'Strategies and Challenges in Clinical Trials Targeting Human Aging'. Together they form a unique fingerprint.

Cite this