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Research profile · Small molecule

AICAR

Also known as Acadesine, AICA riboside

Clinical — evidence level 4 of 4: Established clinical evidenceExperimental — UK regulatory status: Experimental — not authorised for human use in the UK

Overview

A nucleoside used as a laboratory activator of an energy-sensing enzyme. Its large phase 3 trial was stopped for futility.

Category
Small molecule
Highest evidence level identified
Established clinical evidence
Last scientifically reviewed
20 August 2026
Last regulatory review
20 August 2026

Mechanism under investigation

AICAR is a nucleoside taken up by cells and phosphorylated to an AMP analogue that can allosterically activate AMP-activated protein kinase, a central regulator of cellular energy metabolism. In its clinical development it was characterised differently, as an agent raising local adenosine concentrations in ischaemic tissue. A systematic review concludes that many effects historically attributed to kinase activation are in fact independent of that kinase, so the mechanism should be treated as proposed and incompletely resolved.

Research areas

  • Metabolic research
  • Cardiac research

These are fields in which the compound has been investigated. Listing a field is not a statement that anything was demonstrated within it.

Current evidence

In-vitro evidence

Cells, tissues or biochemical systems outside a living organism.

Extensive — thousands of records. This is one of the most widely used laboratory activators of its target enzyme, and is also reported to induce cell death in certain leukaemia cell lines. Almost all of this work uses concentrations chosen for cell culture rather than achievable systemic exposure.

Animal evidence

Studies in living non-human animals.

Extensive, covering cardiac ischaemia and reperfusion, diabetic neuropathy, lymphoma models and a wide metabolic literature.

Human observational evidence

Studies observing people without assigning an intervention.

No relevant observational cohorts were identified. Analytical studies detecting the compound in doping control are laboratory method work rather than clinical evidence.

Clinical-trial evidence

Studies assigning an intervention to human participants.

This is the one compound of its kind with large-scale definitive human data, and that data is negative. A phase 3 randomised double-blind placebo-controlled trial across 300 sites in seven countries was stopped early for futility after 3,080 of a planned 7,500 participants. The primary composite outcome occurred in 5.0 per cent on placebo against 5.1 per cent on the drug — an odds ratio of 1.01 with a confidence interval spanning unity — and there were no differences in key secondary endpoints. An earlier trial in 2,698 patients had reported improved survival; that positive signal was not reproduced when tested prospectively. A phase 1/2 study in relapsed leukaemia established a maximum tolerated dose with only variable efficacy trends, and a later haematology trial was terminated.

Study limitations

  • The evidence level records that definitive clinical evidence exists, not that the compound works. The large phase 3 trial was stopped early for futility: the finding is an absence of benefit, and it is firmly established rather than merely unproven.
  • The earlier positive cardiac findings came from meta-analysis and secondary analyses and were not reproduced prospectively — a textbook case of a promising signal failing confirmation.
  • Most mechanistic literature uses the compound as a laboratory tool at cell-culture concentrations, and a systematic review found many of its effects independent of the enzyme it is supposed to act through.
  • There is no trial evidence whatsoever for the metabolic or endurance uses for which it is popularly discussed. The human trials that exist are in cardiac surgery and blood cancers.

UK regulatory context

Experimental — not authorised for human use in the UK

A compound appearing in research literature that holds no UK authorisation for human use in any indication.

Source: Active ingredients beginning with A — electronic Medicines Compendium index (opens medicines.org.uk in a new tab) (electronic Medicines Compendium)

Regulatory classification is jurisdiction-specific. A compound authorised elsewhere is not thereby authorised in the UK, and a supplier’s description of a substance does not determine how it is classified in law. See safety and regulation for the framework and the authoritative sources.

Safety and interpretation

This compound is not inert in humans. The phase 1/2 leukaemia study reported frequent raised uric acid, transient anaemia and low platelets, renal impairment and clinically significant infusion-related low blood pressure, and defined a maximum tolerated dose. In the large cardiac surgery trial it produced neither benefit nor differences in key secondary endpoints. It has never been authorised as a medicine anywhere, so no post-marketing surveillance exists. It is also prohibited at all times in sport.

References