Skip to main content
ISSCR 2025 Annual Meeting Main banner

Clinical Applications
Session Type: Concurrent Session

CLINICAL TRIAL UPDATES

Wednesday, June 11, 2025
8:30 AM – 10:00 AM HK Time
Location: Theater 1, L1

    Presenting Author(s)

  • Andres Bratt-Leal, PhD

    Research
    Aspen Neuroscience Inc., United States

  • Michael Davis, PhD

    Biomedical Engineering
    Emory University, United States

  • Clive Svendsen, PhD

    Regenerative Medicine
    Cedars-Sinai Board of Governors Regenerative Medicine Institute
    Cedars-Sinai, United States

  • Jun Takahashi, MD, PhD

    Center for iPS Cell Research and Application (CiRA), Kyoto University
    Center for iPS Cell Research and Application, Kyoto University, Japan

We pursue a cell replacement therapy for Parkinson's disease (PD) using induced pluripotent stem cells (iPSCs). To this goal, we have developed a method for 1) scalable dopaminergic (DA) neuron induction on human laminin fragments and 2) sorting DA progenitor cells using a floor plate marker, CORIN. The grafted CORIN+ cells survived well, functioned as midbrain DA neurons in the PD model rats and monkeys, and showed a minimal risk of tumor formation. Based on these results, we started a clinical trial to treat PD patients at Kyoto University Hospital in 2018. The trial evaluated the safety and efficacy of transplanting human iPSC-derived DA progenitors into PD patients' putamen. Using a stereotaxic surgical technique, we implanted approximately 5 or 10 million cells into the bilateral putamen of the patients. The target was seven patients, and we finished the two-year observation of all patients in 2023. In this presentation, I will summarize this project.

Presentations:

  • A CLINICAL TRIAL TRANSPLANTING HUMAN NEURAL PROGENITORS SECRETING GDNF INTO THE MOTOR CORTEX OF ALS PATIENTS: SURVIVAL AND MIGRATION INTO REGIONS OF DYING MOTOR NEURONS TO ENGAGE TARGET

    Presenting Author: Clive N. Svendsen, PhD – Cedars-Sinai Board of Governors Regenerative Medicine Institute

  • PHASE 1 RANDOMIZED STUDY OF CARDIAC STEM CELLS IN PATIENTS WITH HYPOPLASTIC LEFT HEART SYNDROME: CHILD TRIAL

    Presenting Author: Michael Davis, PhD – Emory University

  • SAFETY, TOLERABILITY, AND EFFICACY OF TRANSPLANTED AUTOLOGOUS IPSC-DERIVED DOPAMINERGIC PRECURSORS IN MODERATE TO ADVANCED PARKINSON’S DISEASE

    Presenting Author: Andres Bratt-Leal, PhD – Aspen Neuroscience Inc.

  • IPSC-BASED THERAPY FOR PARKINSON’S DISEASE

    Presenting Author: Jun Takahashi, MD, PhD – Center for iPS Cell Research and Application (CiRA), Kyoto University