Published human research · Every study cited

Muse cell clinical trials

Muse cells have been studied in humans, in registered clinical trials. Between 2018 and 2024, published human trials of Muse cell-based preparations were conducted across six indications: subacute ischemic stroke, acute myocardial infarction, cervical spinal cord injury, neonatal hypoxic-ischemic encephalopathy, amyotrophic lateral sclerosis, and dystrophic epidermolysis bullosa [16]. Across the published research, donor-derived (allogenic) cells were administered intravenously without HLA matching and without immunosuppressive drugs [17].

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/ 01 The Six Trials

The published trials

2020
First-in-human

The heart. The first Muse cell product administered to humans — patients with acute myocardial infarction (heart attack).

Circ J · PMID 32522904
2021
Phase 1/2 · open-label

The skin. Intravenous allogeneic Muse cells in adults with dystrophic epidermolysis bullosa.

J Eur Acad Dermatol Venereol · PMID 33656198
2023
Randomized · placebo-controlled

Stroke. The program’s most rigorous study — a single IV dose in subacute ischemic stroke, reporting a significant treatment effect.

J Cereb Blood Flow Metab · PMID 37756573
2023
Phase 2

ALS. Safety and clinical effects of a Muse cell-based product in patients with amyotrophic lateral sclerosis.

Cell Transplant · PMID 38014622
2024
Phase 1 · multicenter

The spinal cord. A single intravenous dose of allogenic Muse cells in cervical traumatic spinal cord injury.

Stem Cell Res Ther · PMID 39135172
2024
The SHIELD trial · Phase 1

Newborns. A Muse cell-based product in neonatal hypoxic-ischemic encephalopathy with therapeutic hypothermia.

Stem Cells Transl Med · PMID 39401019
  1. Subacute ischemic stroke

    Randomized · double-blind · placebo-controlled

    The most rigorous study in the published program: a randomized, double-blind, placebo-controlled trial of CL2020, an allogenic Muse cell-based product, administered as a single intravenous dose to patients with subacute ischemic stroke [1]. The published results report a significant treatment effect compared with placebo. No HLA matching and no immunosuppression were used. PMID 37756573

  2. Acute myocardial infarction

    First-in-human

    The first human study of a Muse cell-based product: a first-in-human trial in patients with ST-elevation acute myocardial infarction, reporting on the safety and efficacy of intravenous administration [2]. PMID 32522904

  3. Cervical spinal cord injury

    Phase 1 · multicenter

    A multicenter clinical trial of a single intravenous dose of allogenic Muse cells in patients with cervical traumatic spinal cord injury, reporting safety and feasibility [3]. PMID 39135172

  4. Neonatal hypoxic-ischemic encephalopathy

    The SHIELD trial · Phase 1

    A Phase 1 trial of a Muse cell-based product in newborns with hypoxic-ischemic encephalopathy receiving therapeutic hypothermia, reporting safety and tolerability [4]. PMID 39401019

  5. Amyotrophic lateral sclerosis

    Phase 2

    A Phase 2 clinical trial of a Muse cell-based product in patients with ALS, reporting safety and clinical effects over the study period [5]. PMID 38014622

  6. Dystrophic epidermolysis bullosa

    Phase 1/2 · open-label

    An open-label Phase 1/2 study of intravenous allogeneic Muse cells in adults with dystrophic epidermolysis bullosa [6]. PMID 33656198

/ 02 The Human Impact

What the results meant for patients

Stroke: independence, measured. In the placebo-controlled stroke trial, 40% of treated patients reached functional independence within 12 weeks — four times the placebo rate — and approximately 70% within one year [1]. For a person rebuilding a life after a stroke, independence is the outcome that matters: dressing, walking, living at home.

40%
of treated patients functionally independent within 12 weeks — four times the placebo rate [1]
and
~70%
within one year [1]

In the same controlled trial, 24% of treated patients had gray hair turn black. Zero in the placebo group [1]. The study was designed to measure stroke recovery; the scalp was never the target. Findings like this are reported here for what they are: an observed result in a published controlled trial, and a reminder that intravenously delivered Muse cells reach the whole body — not only the site the physicians were watching.

Newborns: the SHIELD babies. The neonatal HIE trial enrolled newborns whose brains had been deprived of oxygen at birth — infants facing the possibility of lifelong ventilator dependence. The trial’s published finding is safety and tolerability [4].

“Every baby who received Muse cells has avoided mechanical ventilation — many developed normally and are attending kindergarten.”
Mari DezawaDiscoverer of Muse cells · Tohoku University Attributed statement; reflects intravenous parent-cell clinical experience.

Spinal cord injury: function, not just safety. The cervical SCI trial’s published report describes improvements in neurological classification, activities of daily living, and quality-of-life measures across the study period, alongside its primary safety findings [3].

The heart: where human trials began. The first Muse cell product ever administered to humans was given to patients with acute myocardial infarction [2] — the program’s starting point, and an indication where follow-on work continues.

/ 03 The Authenticity Bridge

This evidence belongs to
a specific cell

Every trial on this page studied verified material: enriched, SSEA-3-characterized Muse cell preparations, produced within the licensed chain that runs from the discovery at Tohoku University to the vial [17]. The published record — the safety profile, the administration without immunosuppression, the outcomes above — belongs to the patented Dezawa Method™ for creation of authentic muse cells and cannot be applied simply because a product uses the word “muse.”

Material sold under the Muse name that did not come through the licensed process is a different material, and the research on this page is not about it. Vendors working around MCI’s intellectual property have been observed to cite these same trials, but citing a study performed on one specific kind of cellular material does not make its findings true of another different material.

If you are a provider or patient seeking real muse cells, verify first.

/ 04 The Research Map

Muse cell research

The six published human trials are part of a much wider research program. Across twelve areas — from neurology and cardiology to hepatic, renal, pulmonary and longevity research — Muse cells are the subject of published clinical results, active and in-preparation studies, and preclinical work. The grid below labels each study: CLIN published clinical · PREP active or in preparation · PRE preclinical · EXP exploratory.

Neurology

CNS · motor neuron · perinatal

  • Subacute stroke · Niizuma 2023 RCT 37756573 CLIN
  • ALS · Yamashita 2023 Phase 2 n=5 38014622 CLIN
  • Neonatal HIE · Sato 2024 SHIELD Phase 1 39401019 CLIN
  • HIE · Park & Borlongan + Dezawa review 34084971 EXP
  • Lacunar stroke preclinical · Uchida 2017 mouse 27999136 PRE

Cardiovascular

AMI · cardiometabolic

  • Acute MI · Noda 2020 STEMI FIH n=3 32522904 CLIN
  • Early-stage MI · PI being recruited PREP
  • Germany · cardiometabolic · Deutsches Zentrum für Zelltherapie · IRB in prep PREP
  • Swine AMI preclinical · Yamada 2022 · infarct 10.5% vs 21.0% 35324926 PRE
  • AMI biomarker · Tanaka 2018 · endogenous Muse mobilization predicts LV function 28931784 PRE

Skin & Wound Healing

EB · cornea · scarring

  • Epidermolysis bullosa · Fujita 2021 Phase 1/2 · adults with dystrophic EB 33656198 CLIN
  • EB preclinical · Fujita 2020 · IV Muse approach 32540249 PRE
  • Corneal scarring · Guo 2020 · mouse + tree shrew · prevents scar formation 32967971 PRE

Orthopedics

SCI · joint · cartilage

  • Cervical SCI · Koda 2024 Phase 1 multicenter n=10 · ISNCSCI + ADL + QoL improved 39135172 CLIN
  • Shoulder OA · Glashow · FL · IRB in prep PREP
  • Knee OA · early-stage PREP
  • Osteochondral repair preclinical · Mahmoud 2017 · rat model · Muse outperformed non-Muse + vehicle 29312455 PRE

Renal

CKD · FSGS · glomerular

  • FSGS preclinical · Uchida 2017 JASN · glomerular reconstruction · creatinine clearance recovered 28674043 PRE
  • UAE multi-indication · Cleveland Clinic PIs · renal included · IRB submitted PREP

Hepatic

regeneration · fibrosis

  • Liver regeneration · Katagiri 2015 · partial hepatectomy · hepatocyte 74% / cholangiocyte 18% 26663569 PRE
  • Liver fibrosis · Iseki 2017 · CCl4 mouse · HepPar-1 71% + albumin 54% · CYP1A2 active 27938474 PRE

Pulmonary

IR injury · ARDS

  • Lung IR injury · Yabuki 2018 · rat preclinical · KGF/HGF/Ang-1/PGE2 pleiotropic 29707971 PRE

Aesthetics

facial · split-face · skin

  • Facial aesthetic · Layke · DMC injection · recruiting PREP
  • Split-face V1 DMA · Neinstein · Dezawa MuseExosomes® + HA · recruiting PREP

Longevity & Healthspan

aging · frailty · performance

  • Healthspan thesis · Dezawa 2025 · Biogerontology · “possible use for healthspan optimization” 40601066 PRE
  • Frailty / longevity · early-stage clinical discussions PREP
  • Multi-system age-reversal case study · Mathews 2025 · J Case Rep DOI 10.30654/MJCR.10210 EXP

Autoimmune & Immune Privilege

mechanism · inflammation

  • HLA-mismatch tolerance · Minatoguchi 2024 · donor Muse without HLA-matching or immunosuppressant 38560897 PRE
  • S1P + immune privilege · Kuroda 2022 · endogenous reparative pluripotent-like 36339573 PRE
  • Neuroinflammation · Yin 2023 · M1→M2 microglia shift · TLR4/MyD88/NF-κB 35799545 PRE

Translational & Multi-indication

cross-border · multi-program

  • UAE multi-indication · Cleveland Clinic PIs · IRB submitted PREP
  • Early-stage · multi-indication portfolio · knee OA · MI · frailty / longevity · PI being recruited PREP

Future Research Directions

additional indications

  • Autism spectrum disorder
  • Cerebral palsy
  • Military medicine
  • Geroscience & frailty
/ 05 The Sources

Read the research yourself

/ 06 The Boundaries

Muse cell-based products are not approved by the FDA for any indication. Where treatment is offered, it is provided by licensed clinicians under the laws and regulations that apply in their state or jurisdiction.

The field’s larger trials are its current work. As new results publish, this page and the Research Hub are updated — with citations, or not at all.

/ 07 Questions

Frequently asked questions

Have Muse cells been studied in humans?

Yes. Published human clinical trials of Muse cell-based preparations exist across six indications — subacute ischemic stroke, acute myocardial infarction, cervical spinal cord injury, neonatal hypoxic-ischemic encephalopathy, ALS and dystrophic epidermolysis bullosa [16] — including a randomized, placebo-controlled trial in stroke [1].

What conditions have been studied in Muse cell trials?

Subacute ischemic stroke, acute myocardial infarction, cervical spinal cord injury, neonatal hypoxic-ischemic encephalopathy, amyotrophic lateral sclerosis, and dystrophic epidermolysis bullosa [16]. Preclinical and in-preparation work spans additional areas, labeled by stage on this page. Findings are specific to each study’s protocol and population.

Do these trial results apply to any product sold as “Muse cells”?

No. The published trials studied verified, SSEA-3-characterized preparations produced within the licensed chain [17]. Material that did not come through that chain is a different material, and the published findings are not evidence about it. Whether material is authentic Dezawa MuseCells® is verifiable directly: the provider appears on MuseCell Innovations’ authorized lists, or the supply is confirmed by MCI itself — see /how-to-verify-muse-cells.

What did the Muse cell stroke trial show?

The randomized, double-blind, placebo-controlled trial of muse cells in subacute ischemic stroke reported a significant treatment effect compared with placebo — 40% of treated patients functionally independent within 12 weeks, four times the placebo rate — from a single intravenous dose without HLA matching or immunosuppression [1].

Did trial patients need immunosuppression?

No. Across the published program, donor-derived Muse cell preparations were administered intravenously without HLA matching and without immunosuppressive drugs [17] — a distinctive feature of the cells’ published immune-privilege profile.

Are Muse cells FDA-approved?

No. muse cell products are not FDA-approved, and nothing on this page is a claim to diagnose, treat, cure or prevent any disease. The trials above are published clinical research; whether treatment is appropriate for any individual is a decision for a treating physician.

These products have not been evaluated or approved by the United States Food and Drug Administration (FDA) and are not intended to diagnose, treat, cure, or prevent any disease or medical condition. Any clinical use is at the sole discretion of a licensed healthcare provider and must comply with all applicable federal and state laws and regulations. Individual results may vary. No claims are made regarding safety or effectiveness for any specific clinical application.

Does stem cell therapy actually work?

The honest answer is: it depends on which cells, for what condition, in whose hands — and whether the material administered is the material that was studied. For Muse cell-based preparations specifically, published human trials exist across six indications, including a randomized placebo-controlled stroke trial reporting a significant treatment effect [16]. The boundaries matter as much as the results: findings belong to the conditions and protocols studied. This page lists every published trial with its citation.

Can stem cells regenerate cartilage?

For Muse cells, the published cartilage and osteochondral-repair work is preclinical — animal-model research, labeled PRE in the research map above. No published human Muse cell trial has studied cartilage regeneration. A field that tells you which evidence is preclinical is a field you can trust with your knee.

What is a Phase 1 clinical trial?

The first stage of human testing, designed primarily to establish safety, dosing and feasibility — not to prove efficacy. Most of the published Muse cell program is Phase 1 and 2, which is why this page reports safety findings prominently and treats efficacy findings as study-specific [16].

/ 08 References

References

  1. Niizuma K, Osawa SI, Endo H, et al. Randomized placebo-controlled trial of CL2020, an allogenic muse cell-based product, in subacute ischemic stroke. J Cereb Blood Flow Metab. 2023;43(12):2029-2039. PMID 37756573 · DOI 10.1177/0271678X231202594
  2. Noda T, Nishigaki K, Minatoguchi S. Safety and Efficacy of Human Muse Cell-Based Product for Acute Myocardial Infarction in a First-in-Human Trial. Circ J. 2020;84(7):1189-1192. PMID 32522904 · DOI 10.1253/circj.CJ-20-0307
  3. Koda M, Imagama S, Nakashima H, et al. 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. Stem Cell Res Ther. 2024;15(1):259. PMID 39135172 · DOI 10.1186/s13287-024-03842-w
  4. Sato Y, Shimizu S, Ueda K, et al. Safety and tolerability of a Muse cell-based product in neonatal hypoxic-ischemic encephalopathy with therapeutic hypothermia (SHIELD trial). Stem Cells Transl Med. 2024;13(11):1053-1066. PMID 39401019 · DOI 10.1093/stcltm/szae071
  5. Yamashita T, Nakano Y, Sasaki R, et al. Safety and Clinical Effects of a Muse Cell-Based Product in Patients With Amyotrophic Lateral Sclerosis: Results of a Phase 2 Clinical Trial. Cell Transplant. 2023;32:9636897231214370. PMID 38014622 · DOI 10.1177/09636897231214370
  6. Fujita Y, Nohara T, Takashima S, et al. Intravenous allogeneic multilineage-differentiating stress-enduring cells in adults with dystrophic epidermolysis bullosa: a phase 1/2 open-label study. J Eur Acad Dermatol Venereol. 2021;35(8):e528-e531. PMID 33656198 · DOI 10.1111/jdv.17201
  7. Kuroda Y, Oguma Y, Hall K, Dezawa M. Endogenous reparative pluripotent Muse cells with a unique immune privilege system. Front Pharmacol. 2022;13:1027961. PMID 36339573 · DOI 10.3389/fphar.2022.1027961
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