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.
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.
Study Type
OBSERVATIONAL
Enrollment
10
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 observational study, participants independently obtain MUSE stem cell therapy at licensed treatment facilities outside the United States as part of their personal medical care. The study team does not provide, administer, manufacture, or direct the therapy.
Healing Hope International
Houston, Texas, United States
Stem Solutions
Monterrey, Nuevo León, Mexico
Change in global functional outcome (Glasgow Outcome Scale-Extended)
The Glasgow Outcome Scale-Extended (GOSE) is an 8-point ordinal scale (1 = Death, 8 = Upper Good Recovery) used to evaluate global functional status following traumatic brain injury. Higher scores reflect better functional outcome. This observational study will measure the change in GOSE score from baseline to 12 months after the participant's first independently obtained MUSE cell therapy session. Assessments will be conducted through structured interviews via in-person visits or telehealth, following standardized GOSE scoring procedures.
Time frame: Baseline (pre-MUSE cell treatment) and 12 months after first MUSE cell treatment
Change in post-concussive symptoms (Rivermead Post-Concussion Symptoms Questionnaire)
The Rivermead Post-Concussion Symptoms Questionnaire (RPQ) is a patient-reported outcome measure used to assess the severity of common post-concussive symptoms. The RPQ includes 16 items, each scored from 0 to 4, producing a total score range of 0-64, with higher scores indicating greater symptom severity. This study will evaluate the change in RPQ total score from baseline to each follow-up time point. Assessments will be conducted remotely using standardized RPQ administration procedures.
Time frame: Baseline; 3, 6, and 12 months after first MUSE cell treatment
Change in cognitive function (Montreal Cognitive Assessment)
The Montreal Cognitive Assessment (MoCA) is a 30-point screening instrument used to evaluate global cognitive function across multiple domains, including attention, memory, executive functioning, language, and visuospatial abilities. Higher scores indicate better cognitive performance. This study will assess change in total MoCA score from baseline to 6- and 12-month follow-up visits. Assessments will be conducted using standardized MoCA administration procedures via in-person or telehealth formats.
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
Health related quality of life will be assessed using the EuroQol 5-Dimension 5-Level Questionnaire (EQ-5D-5L). The EQ-5D-5L measures health status across five domains: mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. Responses are converted into a single summary health utility index score using country-specific value sets. Scale range: Typically less than 0 (health states worse than death) to 1.0 (perfect health) Interpretation: Higher EQ-5D-5L index scores indicate better overall health-related quality of life Changes in EQ-5D-5L index scores will be evaluated from baseline to follow-up assessments.
Time frame: Baseline; 6 and 12 months after first MUSE cell treatment
Change in functional independence (Functional Independence Measure)
Functional independence will be assessed using the Functional Independence Measure (FIM), an 18-item scale with total scores ranging from 18 to 126, higher scores indicating greater independence in activities of daily living. The outcome will be change in FIM total score from baseline to 6 and 12 months.
Time frame: Baseline; 6 and 12 months after first MUSE cell treatment
Change in mood and anxiety symptoms (HADS)
The Hospital Anxiety and Depression Scale (HADS) is a 14-item self-report questionnaire with two subscales-HADS-Anxiety and HADS-Depression-each scored from 0 to 21, with higher scores indicating more severe symptoms. This study will evaluate changes in HADS-Anxiety and HADS-Depression subscale scores from baseline to 6- and 12-month follow-up assessments. The HADS will be administered using standardized procedures through remote or in-person data collection.
Time frame: Baseline; 6 and 12 months after first MUSE cell treatment
Change in Interleukin-6 (IL-6) Serum Concentration
Serum concentrations of Interleukin-6 (IL-6), a pro-inflammatory cytokine associated with chronic traumatic brain injury, will be obtained when available from participants' independently ordered clinical laboratory tests. IL-6 values will be abstracted from medical records or participant-provided laboratory reports. The study team does not order laboratory testing, collect biospecimens, or perform assays. Unit of measure: picograms per milliliter (pg/mL) Interpretation: Higher IL-6 concentrations indicate greater systemic inflammation Changes in IL-6 concentrations will be evaluated between baseline and follow-up time points.
Time frame: Baseline; 3 and 6 months after first MUSE cell treatment
Incidence of treatment-emergent serious adverse events
Serious adverse events (SAEs) will be documented using participant reports and available medical records. SAEs will be classified according to ICH and FDA definitions, including events resulting in death, life-threatening conditions, hospitalization or its prolongation, persistent or significant disability/incapacity, or other medically significant events. This study will record the number and proportion of participants experiencing at least one SAE, as well as the total number of SAEs reported during the observation period.
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)
Self-perceived overall health status will be assessed using the EuroQol Visual Analogue Scale (EQ-VAS). The EQ-VAS is a validated vertical visual analogue scale on which participants rate their current health status. Scale range: 0 to 100 0 = Worst imaginable health state 100 = Best imaginable health state Interpretation: Higher EQ-VAS scores indicate better self-rated health Changes in EQ-VAS scores will be evaluated from baseline to follow-up assessments. Proxy-reported responses will be permitted when participants are unable to self-report due to neurological impairment.
Time frame: Baseline, 6 months, and 12 months after first MUSE cell treatment
Change in Tumor Necrosis Factor Alpha (TNF-α) Serum Concentration
Serum concentrations of Tumor Necrosis Factor Alpha (TNF-α), a pro-inflammatory cytokine implicated in neuroinflammation and secondary injury following traumatic brain injury, will be obtained when available from participants' independently ordered clinical laboratory tests. TNF-α values will be abstracted from medical records or participant-provided laboratory reports. No laboratory testing or biospecimen collection is performed by the study team. Unit of measure: picograms per milliliter (pg/mL) Interpretation: Higher TNF-α concentrations indicate greater inflammatory activity Changes in TNF-α concentrations will be evaluated between baseline and follow-up time points.
Time frame: Baseline, 3 months, and 6 months after first MUSE cell treatment
Change in C-Reactive Protein (CRP) Serum Concentration
Serum concentrations of C-Reactive Protein (CRP), a systemic inflammatory biomarker commonly associated with chronic inflammation and injury response, will be obtained when available from participants' independently ordered clinical laboratory tests. CRP values will be abstracted from medical records or participant-provided laboratory reports. The study does not order laboratory testing or collect biospecimens. Unit of measure: milligrams per liter (mg/L) Interpretation: Higher CRP concentrations indicate greater systemic inflammation Changes in CRP concentrations will be evaluated between baseline and follow-up time points.
Time frame: Time Frame: Baseline, 3 months, and 6 months after first MUSE cell treatment
Tamara C Tamas Director of Clinical Research, MS. Biopharmaceutical RA
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