Improving Drug Safety and Translation Using Human Organoids and AI
Investigators at Cincinnati Children’s are contributing to national efforts to modernize preclinical drug development by replacing or augmenting animal testing with human relevant, data driven models. A major focus is the development of functional human liver organoids capable of modeling drug metabolism, response and toxicity in clinically meaningful ways.
This work is part of DATAMAP, a national initiative within the ARPA H-funded CATALYST program, which integrates patient derived organoids, organ on chip systems, and machine learning to generate predictive and mechanistic toxicity data. Cincinnati Children’s contributes human organoid platforms and translational expertise through the Center for Stem Cell & Organoid Research and Medicine (CuSTOM).
Among the investigators are Takanori Takebe, MD, PhD, a TIME100 Health list honoree, Magdalena Kasendra, PhD, Bhagwat Prasad, MS (Pharm), PhD and Tomoyuki Mizuno, PhD, FCP. This research reflects a broader institutional strategy to improve how therapies are evaluated before they reach pediatric patients—especially those with rare, complex or high risk liver disease.
Why This Matters for Referring Physicians
- Supports safer, more efficient clinical trials
- Improves risk stratification and therapeutic matching
- Creates opportunities for collaboration in translational research and early phase studies
Learn More / Collaborate
To learn more or discuss research collaboration or translational trial opportunities, visit the Center for Stem Cell & Organoid Medicine (CuSTOM) online.
(Published April 2026)
