Safety and Outcomes of MUSE Stem Cell Therapy in Individuals With Traumatic Brain Injury
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: MUSE Stem Cell Therapy.
- Who it may be relevant to
- Registry conditions: Traumatic Brain Injury, Traumatic Brain Injury (TBI) Patients, Traumatic Brain Injury (TBI); Concussion, Initial Encounter, Traumatic Brain Injury (TBI); Concussion, Subsequent Encounter. Basic parameters: 6 years — 75 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States, Mexico
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
An Observational Study on the Safety and Outcomes of MUSE Stem Cell Therapy in Participants With Traumatic Brain Injury Receiving International Clinical Treatment
Overview
This prospective observational study evaluates the safety profile and patient-reported outcomes associated with MUSE (Multilineage-differentiating Stress-Enduring) stem cell therapy in individuals aged 6 to 75 with chronic traumatic brain injury (TBI). Participants independently elect to receive MUSE cell treatment through international clinical programs, and this study aims to capture real-world evidence on the potential therapeutic effects and risks of this emerging regenerative approach. The study does not administer any intervention. Instead, it follows participants who have received, or plan to receive, MUSE cell infusions outside the United States. Over a 12-month follow-up period, data will be collected on neurological functioning, quality of life, activities of daily living, and any reported adverse events or complications. Information will be gathered through remote interviews, structured digital surveys, and review of medical documentation when available. This research is sponsored by Healing Hope International and is intended to contribute to the ethical and responsible advancement of novel cell-based therapies by generating real-world evidence that may guide future clinical trial development and inform patient care practices.
Detailed description
This observational study is designed to systematically evaluate the safety, tolerability, and potential neurological outcomes associated with MUSE (Multilineage-differentiating Stress-Enduring) stem cell therapy in individuals with chronic traumatic brain injury (TBI) who independently obtain treatment through international clinical programs.
MUSE cells are a distinct subpopulation of mesenchymal stem cells characterized by stress resilience, spontaneous triploblastic differentiation, and the capacity to home to sites of tissue injury. Preclinical research suggests that MUSE cells may contribute to neuroregeneration through differentiation into neural and glial lineages, modulation of inflammatory pathways, and repair of damaged central nervous system structures. While early findings are promising, clinical evidence remains limited, highlighting the need for structured real-world data.
This study does not randomize participants or administer any treatment. Instead, it functions as a registry-style, real-world evidence platform following individuals who have elected to receive MUSE cell therapy at licensed facilities outside the United States. Collected data will include baseline demographics, TBI history, details of the stem cell procedure (such as cell source, administered dose, and route of delivery), and longitudinal follow-up over 12 months.
Primary domains of interest include:
Neurological function, assessed with validated clinical instruments (e.g., Glasgow Outcome Scale-Extended).
Quality of life, measured using standardized patient-reported outcome tools (e.g., PROMIS-29, EQ-5D).
Functional abilities and activities of daily living, to assess practical day-to-day impact.
Safety and tolerability, including documentation of adverse events, patient-reported symptoms, and any medical complications.
Data will be obtained through remote telemedicine visits, structured digital surveys, and review of available medical records. This study does not collect biospecimens and does not involve the administration of any investigational product.
The overarching objective is to generate high-quality real-world evidence regarding the use of MUSE stem cells in chronic TBI, supporting the scientific foundation needed for future controlled clinical trials and potential compassionate use pathways. The study is conducted by Healing Hope International, a nonprofit organization dedicated to advancing ethical access and research in regenerative medicine.
Interventions
- Biological MUSE Stem Cell Therapy
MUSE (Multilineage-differentiating Stress-Enduring) stem cell therapy refers to the use of a naturally occurring subpopulation of mesenchymal lineage cells characterized by stress tolerance, expression of SSEA-3, and the capacity to differentiate into multiple cell types. Preclinical studies have shown that MUSE cells can migrate to sites of tissue injury, including the central nervous system, and may contribute to tissue repair through paracrine and regenerative mechanisms. In this observation
Primary outcome measures
- Change in global functional outcome (Glasgow Outcome Scale-Extended) [Time frame: Baseline (pre-MUSE cell treatment) and 12 months after first MUSE cell treatment]
Secondary outcome measures (10)
- Change in post-concussive symptoms (Rivermead Post-Concussion Symptoms Questionnaire) [Time frame: Baseline; 3, 6, and 12 months after first MUSE cell treatment]
- Change in cognitive function (Montreal Cognitive Assessment) [Time frame: Baseline; 6 and 12 months after first MUSE cell treatment]
- Change in Health-Related Quality of Life Measured by EQ-5D-5L Index Score [Time frame: Baseline; 6 and 12 months after first MUSE cell treatment]
- Change in functional independence (Functional Independence Measure) [Time frame: Baseline; 6 and 12 months after first MUSE cell treatment]
- Change in mood and anxiety symptoms (HADS) [Time frame: Baseline; 6 and 12 months after first MUSE cell treatment]
- Change in Interleukin-6 (IL-6) Serum Concentration [Time frame: Baseline; 3 and 6 months after first MUSE cell treatment]
- Incidence of treatment-emergent serious adverse events [Time frame: From first MUSE cell treatment through 12 months of follow-up]
- Change in Self-Rated Health Measured by Visual Analogue Scale (EQ-VAS) [Time frame: Baseline, 6 months, and 12 months after first MUSE cell treatment]
- Change in Tumor Necrosis Factor Alpha (TNF-α) Serum Concentration [Time frame: Baseline, 3 months, and 6 months after first MUSE cell treatment]
- Change in C-Reactive Protein (CRP) Serum Concentration [Time frame: Time Frame: Baseline, 3 months, and 6 months after first MUSE cell treatment]
Eligibility criteria
Inclusion criteria
Individuals aged 6 to 75 years at the time of enrollment.
Documented history of traumatic brain injury (TBI) occurring at least 6 months prior to enrollment (chronic phase).
Participant has independently elected to receive MUSE (Multilineage-differentiating Stress-Enduring) stem cell therapy at a licensed treatment facility outside the United States.
Ability of the participant or legally authorized representative to provide informed consent for participation in an observational study.
Willingness to participate in remote or in-person follow-up assessments for up to 12 months.
Ability to provide medical records, laboratory reports, or treatment documentation when available.
Exclusion criteria
Individuals who have not received, and do not plan to receive, MUSE cell therapy as part of their independent medical care.
Inability or unwillingness to complete study assessments (e.g., severe communication barriers not manageable through caregiver assistance or technology).
Any condition that, in the opinion of the study team, would make participation in an observational registry unsafe or infeasible (e.g., inability to provide minimal required data).
Planned participation in another research study that would prevent collection of observational outcomes for this registry.
Individuals currently incarcerated or in institutional settings where research participation is restricted.
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Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Ecologic or community
Study locations
United States · 1 center
- Healing Hope International — Houston
Mexico · 1 center
- Stem Solutions — Monterrey
Publications
- Uchida H, Niizuma K, Kushida Y, Wakao S, Tominaga T, Borlongan CV, Dezawa M. Human Muse Cells Reconstruct Neuronal Circuitry in Subacute Lacunar Stroke Model. Stroke. 2017 Feb;48(2):428-435. doi: 10.1161/STROKEAHA.116.014950. Epub 2016 Dec 20. PMID 27999136
- Abe T, Aburakawa D, Niizuma K, Iwabuchi N, Kajitani T, Wakao S, Kushida Y, Dezawa M, Borlongan CV, Tominaga T. Intravenously Transplanted Human Multilineage-Differentiating Stress-Enduring Cells Afford Brain Repair in a Mouse Lacunar Stroke Model. Stroke. 2020 Feb;51(2):601-611. doi: 10.1161/STROKEAHA.119.026589. Epub 2019 Dec 12. PMID 31826733
- Yamauchi T, Kuroda Y, Morita T, Shichinohe H, Houkin K, Dezawa M, Kuroda S. Therapeutic effects of human multilineage-differentiating stress enduring (MUSE) cell transplantation into infarct brain of mice. PLoS One. 2015 Mar 6;10(3):e0116009. doi: 10.1371/journal.pone.0116009. eCollection 2015. PMID 25747577
Identifiers
NCT: NCT07326059 · HHI-TBI-MUSE-001