Trial reportCell transplantation
Safety and Clinical Effects of a Muse Cell-Based Product in Patients With Amyotrophic Lateral Sclerosis: Results of a Phase 2 Clinical Trial.
Trial report in Cell transplantation. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 21 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
21 citing papers in PubMed, 29 citations in OpenAlex.
- Intravenously delivered multilineage-differentiating stress-enduring cells dampen hyperoxia-induced lung injury in a rat model with features of bronchopulmonary dysplasia.Stem cell research & therapy · 2026Article
- Therapeutic Window for Intravenous Human Muse Cell Administration in Mouse Spinal Cord Injury.International journal of molecular sciences · 2026Article
- Stem Cell Therapy for Parkinson's Disease: A Mechanistically Distinct Role for Muse Cells.Journal of clinical medicine · 2026Review
- Intravenous vs intrathecal transplantation of allogeneic GMP/GCP compliant Wharton's jelly mesenchymal stromal cells in ALS patients: a phase I study.Neurodegenerative disease management · 2026Article
- Therapeutic Challenges and Future Breakthroughs in Amyotrophic Lateral Sclerosis: From Precision Medicine to Innovative Trial Design.Drug design, development and therapy · 2026Review
- Hierarchical therapeutic potential in the mesenchymal stem cell landscape.Journal of translational medicine · 2025Review
- Cutting-edge treatments in amyotrophic lateral sclerosis: the role of molecular pathogenesis in targeted therapies.Stem cell research & therapy · 2025Review
- Updates on neonatal cell and novel therapeutics: Proceedings of the Second Neonatal Cell Therapies Symposium (2024).Pediatric research · 2025Article
- Comparison of MSCs and Muse cells: the possible use for healthspan optimization.Biogerontology · 2025Article
- Nose-to-brain delivery of human muse cells enhances structural and functional recovery in the murine ischemic stroke model.Scientific reports · 2025Article
- Sphingosine-1-Phosphate Receptor 2 Agonist Mobilises Endogenous Muse Cells to Repair Damaged Myocardial Tissue in Male Rabbits.Journal of cellular and molecular medicine · 2025Article
- 3D dynamic culture of muse cells on a porous gelatin microsphere after magnetic sorting: Achieving high purity proliferation.Regenerative therapy · 2025Article
- Macrophage- and pluripotent-like reparative Muse cells are unique endogenous stem cells distinct from other somatic stem cells.Frontiers in bioengineering and biotechnology · 2025Review
- Mesenchymal stromal cell therapy in epidermolysis bullosa: current perspectives and future directions.Frontiers in cell and developmental biology · 2025Review
- Systemic treatment of recessive dystrophic epidermolysis bullosa with mesenchymal stromal cells: a scoping review of the literature and conclusions for future clinical research.The Journal of dermatological treatment · 2024Article
- Safety and tolerability of a Muse cell-based product in neonatal hypoxic-ischemic encephalopathy with therapeutic hypothermia (SHIELD trial).Stem cells translational medicine · 2024Article
- Human post-implantation blastocyst-like characteristics of Muse cells isolated from human umbilical cord.Cellular and molecular life sciences : CMLS · 2024Article
- Donor Muse Cell Treatment Without HLA-Matching Tests and Immunosuppressant Treatment.Stem cells translational medicine · 2024Review
- Human Muse cells isolated from preterm- and term-umbilical cord delivered therapeutic effects in rat bleomycin-induced lung injury model without immunosuppressant.Stem cell research & therapy · 2024Article
- The Phoenix of stem cells: pluripotent cells in adult tissues and peripheral blood.Frontiers in bioengineering and biotechnology · 2024Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
12 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Amyotrophic lateral sclerosis (ALS) is characterized by progressive loss of motor neurons. Multilineage-differentiating stress-enduring (Muse) cells are unique endogenous stem cells that show therapeutic effects on motor function in ALS mouse models. We conducted a single-center open phase II clinical trial to evaluate the safety and clinical effects of repeated intravenous injections of an allogenic Muse cell-based product, CL2020, in patients with ALS. Five patients with ALS received CL2020 intravenously once a month for a total of six doses. The primary endpoints were safety and tolerability, and the secondary endpoint was the rate of change in the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) score. In addition, serum tumor necrosis factor-α (TNF-α), interleukin-6 (IL-6), sphingosine-1-phosphate (S1P), cerebrospinal fluid chitotriosidase-1 (CHIT-1), and neurofilament light chain (NfL) levels were evaluated. The CL2020 treatment was highly tolerated without serious side effects. The ALSFRS-R score change trended upward at 12 months post-CL2020 treatment compared with that at 3 months pre-administration, but the difference was not statistically significant. Among five patients diagnosed with ALS, three exhibited a decrease in the rate of ALSFRS-R score change, one demonstrated an increase, and another showed no change. In addition, the patients' serum IL-6 and TNF-α levels and cerebrospinal fluid CHIT-1 and NfL levels increased for up to 6 months post-treatment; however, their serum S1P levels continuously decreased over 12 months. These findings indicate a favorable safety profile of CL2020 therapy. In the near future, a double-blind study of a larger number of ALS patients should be conducted to confirm the efficacy of ALS treatment with CL2020.
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What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.