Calcutt · The Journal of clinical investigation 2017 · preclinical in vitro and animal mechanistic study · n=?

Selective antagonism of muscarinic receptors is neuroprotective in peripheral neuropathy.

Cited 94 times in the scientific literature.

Level 5 - mechanism / opinion, no new human data

Preclinical in vitro and animal experimental research.

PubMed 28094765 · doi:10.1172/JCI88321 · record verified 2026-08-29

What was done

The authors investigated the role of muscarinic acetylcholine type 1 receptors (M1R) on neurite outgrowth and mitochondrial function via AMPK in adult sensory neurons. They evaluated sensory neurons from M1R-knockout mice and tested selective M1R antagonists (pirenzepine, VU0255035, and muscarinic toxin 7 [MT7]) in vitro and in vivo across multiple rodent models of peripheral neuropathy, including streptozotocin-induced diabetes, chemotherapy neurotoxicity (dichloroacetate, paclitaxel), and HIV envelope protein gp120 exposure.

What was found

The abstract reports no numerical values. In vitro, M1R deletion or selective pharmacological inhibition (pirenzepine, VU0255035, MT7) promoted neurite outgrowth, activated AMPK, and reversed diabetes-induced mitochondrial dysfunction. In vivo, M1R deficiency or antagonist administration prevented or reversed indices of peripheral neuropathy, including sensory nerve terminal depletion, thermal hypoalgesia, and reduced nerve conduction velocity across diabetic, chemotherapy-induced, and HIV-associated rodent models.

Why it matters

This identifies M1R-mediated cholinergic signaling as an endogenous brake on axonal plasticity and mitochondrial bioenergetics. Because some antimuscarinic agents like pirenzepine are already approved for human use, M1R antagonism represents a potentially repurposable therapeutic target for diverse forms of peripheral neuropathy.

Limits

The study is entirely preclinical, relying on rodent models and cell cultures; clinical efficacy and safety in human peripheral neuropathy remain unproven. The abstract does not provide sample sizes, effect sizes, statistical parameters, or details on dose regimens, durability of response, or potential off-target antimuscarinic side effects.

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