First-in-human clinical study of an embryonic stem cell product for urea cycle disorders.

ABSTRACT:

Background

This study assesses the safety and efficacy of hepatocyte-like cell (HLC) infusion therapy derived from human embryonic stem cells as bridging therapy for neonatal-onset urea cycle disorders (UCD). The research includes both preclinical and clinical evaluations to determine the feasibility of HLC infusion as a therapeutic option for safer pediatric liver transplantation.

Methods

Preclinical studies were conducted to validate the safety, biodistribution, and ammonia metabolism capabilities of HLCs using SCID mice models of UCD and extensive animal studies. In the clinical trial, five neonates with UCD received HLC infusions, intending to maintain metabolic stability and exceed a target weight of over 6 kg, which is considered necessary for safer liver transplantati

SUBMITTER: Umezawa A 

PROVIDER: S-EPMC11887382 | biostudies-literature | 2025 Mar

REPOSITORIES: biostudies-literature