Cincinnati Children’s New Center Furthers Innovative Research in Uptown

Cincinnati Children’s Hospital Medical Center recently launched the Center for Stem Cell and Organoid Medicine (CuSTOM), a multidisciplinary center dedicated to the advancement of stem cell-organoid technology. It is believed to be the first facility at a pediatric medical center focused on this type of research. 

Scientists in the center use pluripotent stems cells to bioengineer human tissue that mimics natural human development, known as organoids. The tissue can be used to study how diseases are caused and progress, to test drugs before clinical trials, and ultimately to address the shortage of organs available for transplant. 

“Today in the lab, we’re using organoids from patients that are actually in the hospital to discover unknown disease pathologies. And then we’re working with the clinicians to try to improve the patient care,” said James Wells, PhD, CuSTOM Chief Scientific Officer and Director of Research in the Division of Endocrinology at Cincinnati Children’s. 

The use of patients’ own stem cells to generate the organoid tissue creates a new platform for personalized medicine. For example, according to Cincinnati Children’s, liver organoids hold the nearest-term clinical potential as a personalized platform to test toxicity and efficacy for new drugs.   

Bioengineered human intestine with working nerves.

Bioengineered human intestine with working nerves.

Currently, most of the center focuses on gastrointestinal tissue like the small intestine, colon, liver and stomach, but Cincinnati Children’s researchers are working on developing esophageal, kidney and lung tissue as well. 

While this research has been happening at Cincinnati Children’s for years, forming the center will help accelerate the technology from bench to bedside. 

“We have this convergence of transformative breakthroughs in organ-generation technology at Cincinnati Children’s, and it’s critical that we speed up the translation cycle so patients can benefit more quickly,” said Aaron Zorn, PhD, CuSTOM Director and Associate Director of the Digestive Health Center at Cincinnati Children’s. 

The overall effort to study translatable applications for the center’s technology is led by Michael Helmrath, MD, CuSTOM associate director of clinical translation and surgical director of intestinal research and rehabilitation. Helmrath and Takanori Takebe, MD, CuSTOM Associate Director of Commercial Innovation, help identify technology within the center with potential to transition to clinical practice and coordinate with Cincinnati Children’s Center for Technology Commercialization to find industry collaborators. 

Many of the organoid technologies at CuSTOM are already close to being useable in clinical settings, but the timeline for translating the technology to patient care hinges on final technological developments and industry partnerships. 

“The key to success is going to be getting help from industry, philanthropy and the medical center’s technology transfer team, so that we can transfer this organoid technology to the patient care pipeline as quickly as possible,” said Zorn. 

Researchers at Cincinnati Children’s hope to see more potential industry partners coming to the Uptown area in the Uptown Innovation Corridor to increase opportunities for collaboration, investment and development. 

For more information on Cincinnati Children’s Center for Stem Cell and Organoid Medicine, visit