A pilot phase IIa randomized, placebo-controlled, double-blind trial of intravenous human pluripotent adipose-derived stem cells (PASCs) in Parkinson’s disease: The REPOSE study
10 affiliations
- 1ClusterXStem Inc., Los Angeles, CA, USA
- 2IBioBA-MPSP, Buenos Aires, Argentina
- 3ClusterXStem Costa Rica, San José, Costa Rica
- 4David Geffen School of Medicine at the University of California Los Angeles, Los Angeles, CA, USA
- 5NeuroFT, San José, Costa Rica
- 6Provida. Laboratorio de Células Madre, San José, Costa Rica
- 7Hospital Clínica Bíblica, San José, Costa Rica
- 8Research Unit, The Regenerative Medicine Institute, San José, Costa Rica
- 9Centro de Investigación en Hematología y Trastornos Afines (CIHATA) & Centro de Investigación en Enfermedades Tropicales (CIET), Universidad de Costa Rica, San José, Costa Rica
- 10Hospital Internacional La Católica, San José, Costa Rica
Abstract
Pluripotent adipose-derived stem cells (PASCs) are a non-tumorigenic, stress-enduring SSEA-3+ cell population sharing key biological markers with Muse cells while representing a phenotypically distinct population. This Phase IIa randomized, double-blind, placebo-controlled pilot trial evaluated the safety, feasibility, and exploratory clinical effects of intravenous PASCs in Parkinson’s disease (PD). Forty participants with Hoehn and Yahr stages I–III PD were randomized 1:1 to receive three intravenous infusions of PASCs (25 million cells) or placebo at baseline, month 3, and month 6, with 12-month follow-up. All participants underwent a structured neurological rehabilitation program. The primary endpoint was safety (CTCAE v5.0). No treatment-related adverse events of any grade were observed across 60 infusions. One thromboembolic event in the PASC group was independently adjudicated as of uncertain relationship to the study intervention, and one death in the placebo group was adjudicated as unrelated. For each of the seven exploratory functional and quality-of-life endpoints, the global treatment-by-time interaction was tested and adjusted for multiplicity using the Benjamini-Hochberg procedure; no endpoint reached significance after control of the false discovery rate (all q > 0.05). Repeated intravenous PASC administration is safe and feasible in PD. Efficacy was not demonstrated in this pilot trial, and the exploratory findings are hypothesis-generating, supporting further investigation in adequately powered Phase IIb/III trials.
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BibTeX
@article{gimeno2026pilot,
title = {{A pilot phase IIa randomized, placebo-controlled, double-blind trial of intravenous human pluripotent adipose-derived stem cells (PASCs) in Parkinson’s disease: The REPOSE study}},
author = {María Laura Gimeno and Gregorio Chazenbalk and Lisa Riggioni and Abby Ortega and Luis Miranda and Juan Antonio Valverde and José A Molina-Mora and Linette González Rojas and Pamela Vargas and Carlos Zuñiga and Luis Rosales and Freddy Henríquez},
journal = {Cell Transplantation},
year = {2026},
month = sep,
doi = {10.1177/09636897261492438},
url = {https://doi.org/10.1177/09636897261492438},
note = {CC BY-NC 4.0},
}
RIS
TY - JOUR
TI - A pilot phase IIa randomized, placebo-controlled, double-blind trial of intravenous human pluripotent adipose-derived stem cells (PASCs) in Parkinson’s disease: The REPOSE study
AU - María Laura Gimeno
AU - Gregorio Chazenbalk
AU - Lisa Riggioni
AU - Abby Ortega
AU - Luis Miranda
AU - Juan Antonio Valverde
AU - José A Molina-Mora
AU - Linette González Rojas
AU - Pamela Vargas
AU - Carlos Zuñiga
AU - Luis Rosales
AU - Freddy Henríquez
PY - 2026
DA - 2026/09/23
JO - Cell Transplantation
DO - 10.1177/09636897261492438
UR - https://doi.org/10.1177/09636897261492438
AB - Pluripotent adipose-derived stem cells (PASCs) are a non-tumorigenic, stress-enduring SSEA-3+ cell population sharing key biological markers with Muse cells while representing a phenotypically distinct population. This Phase IIa randomized, double-blind, placebo-controlled pilot trial evaluated the safety, feasibility, and exploratory clinical effects of intravenous PASCs in Parkinson’s disease (PD). Forty participants with Hoehn and Yahr stages I–III PD were randomized 1:1 to receive three intravenous infusions of PASCs (25 million cells) or placebo at baseline, month 3, and month 6, with 12-month follow-up. All participants underwent a structured neurological rehabilitation program. The primary endpoint was safety (CTCAE v5.0). No treatment-related adverse events of any grade were observed across 60 infusions. One thromboembolic event in the PASC group was independently adjudicated as of uncertain relationship to the study intervention, and one death in the placebo group was adjudicated as unrelated. For each of the seven exploratory functional and quality-of-life endpoints, the global treatment-by-time interaction was tested and adjusted for multiplicity using the Benjamini-Hochberg procedure; no endpoint reached significance after control of the false discovery rate (all q > 0.05). Repeated intravenous PASC administration is safe and feasible in PD. Efficacy was not demonstrated in this pilot trial, and the exploratory findings are hypothesis-generating, supporting further investigation in adequately powered Phase IIb/III trials.
SN - 0963-6897
SN - 1555-3892
ER -