| Field | Value |
|---|---|
| NCT Number | NCT06775964 |
| Title | Stem Cell Therapy for Early Alzheimer's Disease |
| Phase | Phase 1b/2a |
| Status | RECRUITING |
| Sponsor | Paul E Schulz, MD (UTHealth Houston) |
| Collaborator | Weston Brain Institute |
| Location | UT Health Science Center at Houston, TX |
| Enrollment | 12 subjects (estimated) |
| Start Date | February 2026 |
| Primary Completion | December 2026 |
| Study Completion | January 2027 |
This is a Phase 1b/2a open-label, single-arm study evaluating the safety, tolerability, and efficacy of autologous, adipose-derived mesenchymal stem cells (adMSCs) in adults with late pre-symptomatic or prodromal Alzheimer's disease.
Most currently approved AD drugs primarily treat symptoms. While anti-amyloid antibodies have successfully reduced amyloid burden, they have only modestly affected cognitive progression. This suggests that other pathological pathways are equally important for AD progression.
Neuroinflammation has emerged as a critical player in AD pathogenesis. Evidence includes:
This study targets neuroinflammation directly using mesenchymal stem cells, which exhibit multi-therapeutic effects:
Biological: adMSC — IV-infusion of autologous, adipose-derived mesenchymal stem cells (adMSCs), approximately 2×10⁸ cells in 250mL saline.
Dosing Schedule:
| Measure | Description | Timeframe |
|---|---|---|
| Change in TSPO levels | Measured by PET scan from baseline to midpoint and end of study | Baseline, Day 169 |
| Inflammatory cytokines in CSF | Cytokine panel measured via ELISA | Baseline, Day 169 |
TSPO (Translocator Protein) is a marker for activated microglia. TSPO PET imaging correlates with neuroinflammation levels in the brain.
| Measure | Description | Timeframe |
|---|---|---|
| Treatment-related adverse events | Safety monitoring throughout study | Baseline - Day 337 |
| Neurofilament light chain (Nf-L) in CSF | Neurodegeneration biomarker | Baseline, Day 169 |
| GFAP in CSF | Astrocyte activation marker | Baseline, Day 169 |
| Total Tau/phospho-Tau ratios in CSF | AD progression markers | Baseline, Day 169 |
| FDG PET imaging | Cerebral metabolism activity | Baseline, Day 169 |
| Amyloid-β 42/40 ratio in CSF | Amyloid pathology marker | Baseline, Day 169 |
| MMSE score | Cognitive function | Baseline, Day 337 |
| RBANS scores | Neuropsychological assessment | Baseline, Day 337 |
| Lawton IADL Scale | Functional independence | Baseline, Day 337 |
| Immune pathway markers in blood | Systemic inflammation | Baseline, Day 169, Day 337 |
| Immune pathway markers in CSF | CNS inflammation | Baseline, Day 169 |
MMSE (Mini-Mental State Examination): Scoring ranges from 0-30
RBANS (Repeatable Battery for the Assessment of Neuropsychological Status)
Lawton IADL Scale: Measures instrumental activities of daily living
| Biomarker | Tissue | Significance |
|---|---|---|
| TSPO | PET | Microglial activation / neuroinflammation |
| Nf-L | CSF | Axonal neurodegeneration |
| GFAP | CSF | Astrocyte activation |
| Total Tau | CSF | Neurodegeneration |
| Phospho-Tau | CSF | Tau pathology |
| Aβ42/40 | CSF | Amyloid pathology |
| FDG PET | Brain | Cerebral glucose metabolism |
The brain's immune system plays a dual role in Alzheimer's disease. While initially protective, chronic neuroinflammation drives disease progression through:
Translocator protein (TSPO, 18kDa) is expressed primarily on activated microglia and peripheral immune cells. TSPO PET imaging provides:
Note: TSPO SNP rs6971 is an exclusion criterion because it affects tracer binding affinity, potentially confounding PET results.
Adipose-derived MSCs offer several advantages:
| Role | Name | Phone | |
|---|---|---|---|
| Principal Investigator | Paul E Schulz, MD | — | — |
| Study Contact | Harshali Patel | 713-486-0531 | Harshali.Patel@uth.tmc.edu |
| Study Contact | Javier Ortiz, PhD | 713-486-0505 | Guadalupe.J.Ortiz@uth.tmc.edu |
| Study Contact | Christine Farrell, PhD | 713-486-0527 | Christine.M.Farrell@uth.tmc.edu |