Research & Publications

The science behind MUSE cells

A curated selection of peer-reviewed studies documenting MUSE cell biology, mechanism of action, and therapeutic potential across multiple conditions.

178+

Peer-reviewed records

6

Published Phase 1/2 readouts

5

Active trials in preparation

This page presents published peer-reviewed research for educational purposes. Links open PubMed or the publisher's site. This site has no affiliation with any research institution, pharmaceutical company, or clinical provider.

Foundational Biology

Studies establishing the identity, isolation, and core properties of MUSE cells.

2010

Unique multipotent cells in adult human mesenchymal cell populations

Kuroda Y et al.·Proc Natl Acad Sci USA·107(19):8639–44

The landmark paper identifying MUSE cells as a distinct, naturally occurring pluripotent stem cell population in adult human mesenchymal tissues.

2011

Multilineage-differentiating stress-enduring (Muse) cells are a primary source of induced pluripotent stem cells in human fibroblasts

Wakao S, Kitada M, Kuroda Y et al.·Proceedings of the National Academy of Sciences·108(24):9875–80

Demonstrated that MUSE cells, not bulk fibroblasts, are the primary source of iPSC generation, revealing their innate pluripotency.

2014

Muse cells, newly found non-tumorigenic pluripotent stem cells, reside in human mesenchymal tissues

Wakao S, Akashi H, Kushida Y, Dezawa M·Pathology International·64(1):1–9

Characterized the tissue distribution of MUSE cells and confirmed their non-tumorigenic pluripotency across multiple human mesenchymal tissue sources.

2022

Endogenous reparative pluripotent Muse cells with a unique immune privilege system: Hint at a new strategy for controlling acute and chronic inflammation

Kuroda Y, Oguma Y, Hall K, Dezawa M·Frontiers in Pharmacology·13:1027961

Described the unique immune privilege properties of MUSE cells that allow allogeneic administration without immunosuppression, opening the door to off-the-shelf therapy.

Neurology & Stroke

Preclinical and clinical research on MUSE cells for stroke, ALS, spinal cord injury, and neonatal brain injury.

2016

Transplantation of Unique Subpopulation of Fibroblasts, Muse Cells, Ameliorates Experimental Stroke Possibly via Robust Neuronal Differentiation

Uchida H, Morita T, Niizuma K et al.·Stem Cells·34(1):160–73

Showed that transplanted MUSE cells differentiated robustly into neurons in a stroke model, reducing infarct size and improving functional recovery.

2017

Human Muse Cells Reconstruct Neuronal Circuitry in Subacute Lacunar Stroke Model

Uchida H, Niizuma K, Kushida Y et al.·Stroke·48(2):428–435

Demonstrated that intravenously delivered MUSE cells reconstructed neuronal circuitry and improved motor deficits in a lacunar stroke model.

2020

Therapeutic benefit of Muse cells in a mouse model of amyotrophic lateral sclerosis

Yamashita T, Kushida Y, Wakao S et al.·Scientific Reports·10(1):17102

Preclinical study showing MUSE cell therapy extended survival and preserved motor neuron populations in an ALS mouse model.

2023

Safety and Clinical Effects of a Muse Cell-Based Product in Patients With Amyotrophic Lateral Sclerosis: Results of a Phase 2 Clinical Trial

Yamashita T, Nakano Y, Sasaki R et al.·Cell Transplantation·32:9636897231214370

Phase 2 clinical trial reporting acceptable safety and signals of slowed disease progression in ALS patients treated with the MUSE cell product CL2020.

2023

Randomized placebo-controlled trial of CL2020, an allogenic muse cell-based product, in subacute ischemic stroke

Niizuma K, Osawa S, Endo H et al.·Journal of Cerebral Blood Flow & Metabolism·43(12):2029–2039

Phase 2 RCT demonstrating safety and neurological improvement in subacute ischemic stroke patients receiving intravenous allogeneic MUSE cells.

2024

Safety and feasibility of intravenous administration of a single dose of allogenic-Muse cells to treat human cervical traumatic spinal cord injury: a clinical trial

Koda M, Imagama S, Nakashima H et al.·Stem Cell Research & Therapy·15(1):259

Phase 1 clinical trial establishing safety and feasibility of a single intravenous dose of allogeneic MUSE cells in cervical spinal cord injury patients.

Cardiovascular

Research on MUSE cell therapy for heart attack, heart failure, and vascular repair.

2018

S1P-S1PR2 Axis Mediates Homing of Muse Cells Into Damaged Heart for Long-Lasting Tissue Repair and Functional Recovery After Acute Myocardial Infarction

Yamada Y, Wakao S, Kushida Y et al.·Circulation Research·122(8):1069–1083

Identified the S1P–S1PR2 signaling axis as the mechanism by which MUSE cells home to infarcted myocardium and achieve durable cardiac repair.

2020

Safety and Efficacy of Human Muse Cell-Based Product for Acute Myocardial Infarction in a First-in-Human Trial

Noda T, Nishigaki K, Minatoguchi S·Circulation Journal·84(7):1189–1192

First-in-human Phase 1 trial reporting safety and preliminary efficacy of MUSE cell therapy in acute myocardial infarction patients.

2022

Human Muse cells reduce myocardial infarct size and improve cardiac function without causing arrhythmias in a swine model of acute myocardial infarction

Yamada Y, Minatoguchi S, Baba S et al.·PLOS ONE·17(3):e0265347

Large-animal study confirming that MUSE cells reduce infarct size and improve ejection fraction without proarrhythmic effects in a swine MI model.

Neonatal & Pediatric

Clinical and preclinical research on MUSE cells for neonatal hypoxic-ischemic encephalopathy.

2021

Intravenously delivered multilineage-differentiating stress enduring cells dampen excessive glutamate metabolism and microglial activation in experimental perinatal hypoxic ischemic encephalopathy

Suzuki T, Sato Y, Kushida Y et al.·Journal of Cerebral Blood Flow & Metabolism·41(7):1707–1720

Demonstrated that IV MUSE cells reduced excitotoxic glutamate levels and neuroinflammation in a neonatal HIE model, supporting neuroprotective mechanisms.

2024

Safety and tolerability of a Muse cell-based product in neonatal hypoxic-ischemic encephalopathy with therapeutic hypothermia (SHIELD trial)

Sato Y, Shimizu S, Ueda K et al.·Stem Cells Translational Medicine·13(11):1053–1066

Phase 1 dose-escalation SHIELD trial establishing safety and tolerability of CL2020 MUSE cell therapy as an adjunct to hypothermia in neonatal HIE.

From bench to bedside

Six published Phase 1/2 readouts form the completed human-evidence base from the LSI/CL2020 era. Additional trials are advancing across the US, UAE, and Germany.

The studies listed on this page are published in peer-reviewed journals and are linked via PubMed and their DOI. Summaries are written for educational clarity and do not constitute medical advice. Always consult a qualified healthcare professional before making any medical decisions.

MUSE Cells

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For educational purposes only. This site does not provide medical advice, diagnosis, or treatment. Always consult a qualified healthcare professional.