Center for Stem Cell & Organoid Medicine (CuSTOM)

Center for Stem Cell & Organoid Medicine (CuSTOM)

Jim Wells, PhD, CuSTOM's Chief Scientific Officer, presented Dorcas Cummings lecture "Growing human organs in the lab: Using organoids to understand human disease and discover new therapeutics" at the Cold Spring Harbor Laboratories. Watch the lecture on YouTube.

Cincinnati Children’s scientists open doors to new wave of disease origin research: "Organoids Validated as Tool for Studying Fetal Intestine Development"

RIKEN-BDR and CuSTOM held their annual international Symposium "Integrated organoid science: Stem cells, Engineering, Medicine" in Kobe, Japan, on February 15-17, 2023. Stay tuned for details on the next Symposium in Spring 2024 in Cincinnati!

Scientists Grew Mini Human Guts Inside Mice 

Michael Helmrath, MD and his colleagues announced in the journal Nature Biotechnology that they had transplanted tiny, three-dimensional balls of human intestinal tissue into mice. After several weeks, these spheres known as organoids developed key features of the human immune system. The model could be used to mimic the human intestinal system without having to experiment on sick patients. These tiny organoids with working immune systems mimic the function of the GI tract and could be used to study intestinal diseases and drugs to treat them.

Dr. Masaki Kimura, member of the Takebe Lab, developed new technology to create liver organoids from human stem cells at a population scale. Leveraging a population organoid panel, the team conducted organoid level genotype-phenotype association study and resolved the puzzling role of the genetics for fatty liver disease, aka NASH, associated with Type 2 diabetes. This study is one of the earliest applications of the new population organoid creation technique to study the causes of human disease at the individual and population levels, paving a way for precision medicine application.

Learn more >

Center for Stem Cell & Organoid Medicine (CuSTOM) team developed intestine organoids that include an immune system, which can open the door for making better treatments for IBD, Crohn's, Celiac Disease and more. View the study here.  Read the Research Horizons blog about the study.

Mingxia Gu, MD, PhD, FAHA, has being selected as one of 10 distinguished early career scientists to serve on the first Stem Cell Reports Early Career Editorial Board (ECEB). Stem Cell Reports is the peer-reviewed, open access, online journal of the International Society for Stem Cell Research (ISSCR). Throughout the next two years, the members of the ECEB will provide strategic advice, participate in the editorial review process, and receive mentorship from current editors.

Heather McCauley has been recruited as an assistant professor in the Department of Cell Biology and Physiology in the School of Medicine at the University of North Carolina at Chapel Hill. Her lab will build on her postdoctoral work in Jim Wells’ lab finding new roles for enteroendocrine cells in the regulation of gastrointestinal physiology and metabolism. Email Heather for more information.

Congratulations to newly minted PhD, Jacob Enriquez, from the Wells lab! Dr. Enriquez’s work was published in November 2022 in Cell Reports and featured in Nature Research Highlights. His next phase will be working as a postdoctoral researcher at the University of Chicago in the Mirmira lab. His new research will be addressing questions about cell-specific targeting of the insulin-secreting beta-cells in the context of Type 1 Diabetes. The Mirmira lab is a world-leader in Type-1 Diabetes research and frequently publishes high impact work with a focus on translational therapies. We wish him the best of luck on his future work!

2023 RIKEN BDR-CuSTOM Joint Organoid Symposium “Integrated Organoid Science: Stem Cells, Engineering, Medicine”

February 15-17, 2023 in Kobe, Japan

Registration is now open for 2023 RIKEN BDR-CuSTOM Joint Organoid Symposium, co-sponsored by RIKEN Center for Biosystems Dynamics Research (BDR) and CuSTOM.

2023 RIKEN BDR-CuSTOM Joint Organoid Symposium flier

2023 RIKEN BDR-CuSTOM Joint Organoid Symposium “Integrated Organoid Science: Stem Cells, Engineering, Medicine”

Takanori Takebe, MD, PhD, is the JASGC Annual Gala Dinner Featured Keynote Speaker.

Takanori Takebe, MD, PhD, CuSTOM Director of Commercial Innovation, is the JASGC Annual Gala Dinner Featured Keynote Speaker. Dr. Takebe is a leader in the field of organoid research and was appointed a Professor at Tokyo Medical and Dental University in 2018 at the age of 31 - recorded as the youngest person ever to have achieved that status in Japan's university history.

Takanori Takebe, MD, PhD, is the JASGC Annual Gala Dinner Featured Keynote Speaker.

Generating Gastrointestinal Organoids

Cincinnati Children’s scientists report significant improvements to the methods for generating gastrointestinal organoids. This study demonstrates that organoids can be formed by the aggregation of precursor cells, completely eliminating the variability and unpredictable yields of organoid formation using standard methods. This method enables reliable organoid formation from any human pluripotent stem cell line and facilitates significant scale-up of organoid production. The study also demonstrates that precursor cells can be cryopreserved and subsequently thawed and aggregated for organoid production. This breakthrough permits large scale production of quality-controlled frozen organoid precursors, enabling both expanded dissemination of organoid technologies, as well as permitting researchers to produce organoids without the need to grow stem cells.

Generating Gastrointestinal Organoids

2022 Virtual Frontiers in Stem Cell & Organoid Medicine Symposium, March 24-25, 2022

Registration is now open for 2022 Virtual Frontiers in Stem Cell & Organoid Medicine Symposium. Co-sponsored by the Center for Stem Cell & Organoid Medicine (CuSTOM) and RIKEN Center for Biosystems Dynamics Research (BDR), as well as a number of leading biotechnology companies, this international Symposium will focus on recent breakthroughs and acceleration of clinical translation, and will feature emerging leaders in the field, including top academic researchers, industry scientists, and junior investigators.

Topics to be covered range from basic research to clinical and commercial translation of iPS cell and organoid technology. Discussion panel sessions will focus on major hurdles and opportunities in organoid medicine.

Theresa Alenghat Gives Microbiome TedTalk

CuSTOM Scientist Theresa Alenghat, VMD, PhD, leads a research lab that investigates microorganisms that normally live in our gut (our microbiome). Her research has revealed new pathways for how the microbiome impacts infection, obesity, and inflammation. In her TedTalk, Theresa shares how bacteria in the gut communicate with cells and how this relationship can be affected by diet. This type of research illustrates that bodies are quite sensitive to an ongoing dialogue with microorganisms in the gut and opens doors to a new range of ways to improve health by harnessing the power of the microbiome.

View the TedTalk >

Renewed Whitney Family Philanthropic Gift Supporting ALS Research  

The Ziyuan Guo Lab has received a renewal of philanthropy from the Whitney Family to support its Amyotrophic Lateral Sclerosis (ALS) research. The Lab's research aims to not only understand fundamental questions on how disease mutation leads to illness, but also to identify natural de novo mutations, which can prevent illness development. These novel mechanisms can be used to improve patient treatment.

New Assembly Approach Generates Most Complex Stomach Organoids to Date

Cincinnati Children’s report success at developing a stomach organoid so sophisticated that it has distinct glands and nerve cells that can control smooth muscle contractions. The achievement demonstrates that separate layers and portions of complex organs can be grown from separate lines of human pluripotent stem cells (PSCs) and be combined for continued development. And the approach used to produce these multi-layered stomach organoids also can be used to make more-complex versions of other lab-grown organs.

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Dr. James Wells Named Academic Editor for Development

James Wells, PhD, Chief Scientific Officer for CuSTOM, has been named an Academic Editor for the journal Development. He is honored to take on this role. He has great respect for the journal and hopes to bring new model systems that CuSTOM teams develop to other developmental biologists.

Read the Journal's Interview with Dr. Wells >

From 4,500 Possibilities, One Compound Emerges as Promising Treatment for PAH

A team of scientists at Stanford University and Cincinnati Children’s report finding a potentially effective needle in a haystack for treatment of pulmonary arterial hypertension (PAH), a potentially dangerous condition occurring among premature newborns. Lead author of the study published in Science Translational Medicine, Mingxia Gu, MD, PhD and team screened 4,500 compounds to identify the tyrosine kinase inhibitor AG1296 as the lead compound for further investigation.

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Scientists Regenerate 3D Skin in Lab with Stem Cells to Find Out How Inherited DNA Repair Defects in Kids Cause Deadly Cancer

Physicians and scientists at Cincinnati Children’s Hospital Medical Center used new stem cell technology to regenerate and study living patient-specific skin in the lab, giving them a precise close up view of how inherited DNA defects cause skin damage and deadly squamous cell carcinoma in children and young adults with Fanconi anemia (FA). Susanne Wells, PhD reported their findings in the the journal Cell Stem Cell. They are now using the complex 3D laboratory models of FA patient epidermis – and the enhanced biological detail they provide – to screen for drugs that could slow or stop the disease progression.

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Takebe Receives NIH New Innovator Award

Organoid development expert Takanori Takebe, MD, is among 53 scientists nationwide to receive a “New Innovator” award from the National Institutes of Health High-Risk, High-Reward Research Program. The award provides a five-year, $1.5 million grant to support Takebe’s Engineering Multi-Organs in a Dish project.

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Cincinnati Children's Scientists Identify Hormone That Might Help Treat Malabsorption

James Wells, PhD, Heather McCauley, PhD and a team of scientists at Cincinnati Children's used human intestinal organoids grown from stem cells to discover how our bodies control the absorption of nutrients from the food we eat. They further found that one hormone might be able to reverse a congenital disorder in babies who cannot adequately absorb nutrients and need intravenous feeding to survive.

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Two Discoveries Boost Next-Generation Organoid Development

In back-to-back reports published in Nature Communications, a team of scientists from Cincinnati Children’s and Japan report discoveries that will be vital to a new wave of more-complex organoid development. Their findings advance efforts to use human stem cells to grow organs from the fetal foregut including the trachea, esophagus, stomach, liver, gallbladder, bile ducts and pancreas. 

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Patient Stem Cells Reveal New Therapeutic Target for Congenital Heart Disease

Children born with hypoplastic left heart syndrome require major surgery and even with a repaired heart, one in four children experience life threatening complications. In a recent study, Dr. Mingxia Gu and colleagues used advanced single-cell transcriptomics and cardiac tissue derived from patient stem cells (IPSCs) to model the disease progression in a dish and discover a potential new therapeutic strategy for this devastating condition.

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CuSTOM Annual Fact Sheet

Our annual CuSTOM Fact Sheet is now available. Read more to learn about our progress across technology, breakthrough discoveries, and expanding expertise, resources and partnerships. You will also see number of collaborating research labs and divisions, publications and funding.

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CuSTOM Makes 2019’s Top 7 Breakthrough Discoveries List

Infants born with esophageal atresia, children who develop eosinophilic esophagitis, and others coping with throat diseases stand to benefit from the latest breakthrough in organoid development from Cincinnati Children’s. For now, these tiny-but-functional organs in a dish will help researchers explore how organ formation goes wrong, and in testing the safety and effectiveness of experimental medications. Eventually, experts hope to grow personalized tissues in large enough volume to use in transplants that would not require anti-rejection medications. This breakthrough, led by graduate student Stephen Trisno, and senior author James Wells, PhD, was published in the journal Cell Stem Cell.

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Cincinnati Lemonade Stand Goes Viral, Raises $205,000 for CuSTOM

Two Cincinnati families held a lemonade stand over Labor Day weekend, 2019. What started as a $148 donation to organoid research at Cincinnati Children's grew to $205,000 when one of the families posted to social media, asking for matching donations.

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James Wells, PhD receives a $1.5 million, three-year grant from the foundation of Microsoft co-founder Paul G. Allen

Jim Wells will lead a team studying a rare type of intestinal cell known as enteroendocrine cells that sense nutrients from the food we eat and then control how nutrients are absorbed and stored. Wells’ team will use stem cells from patients with genetic disorders affecting their ability to absorb nutrients to study these cells in the lab. Understanding more about enteroendocrine cells, which produce hormones that regulate our ability to process food, could pave the way for new therapies for malnutrition and diseases like Type 2 diabetes. 

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New International Collaboration

A new international collaboration between CuSTOM and the RIKEN Center for Biosystems Dynamics Research (BDR) in Japan is set to accelerate the discovery and clinical translation of innovative organoid technology. The CuSTOM – RIKEN (BDR) collaboration will support the exchange of junior scientists between institutions, establish a joint laboratory for collaborative research projects, and hold an annual international symposium, bringing together top academic researchers and industry scientists from around the world.

World’s First Three-Organoid System Opens Doors for Medical Research and Diagnosis

Instead of growing mini human organs independently in separate lab dishes, a team led by Takanori Takebe, MD, succeeded at growing a connected set of three organs: the liver, pancreas and biliary ducts. By themselves, human organoids already provide a sophisticated tool for research. But this advance allows scientists to study how human tissues work in concert. This major step forward could begin reducing the need for animal-based medication studies, sharply accelerate the concept of precision medicine, and someday lead to transplantable tissues grown in labs.

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Scientists Bioengineer Human Liver Disease in the Lab to Find New Treatments

Dr. Takanori Takebe, MD and fellow scientists developed a reproducible method to bioengineer multicellular human liver organoids using pluripotent stem cells from healthy human donors and those with liver disease. Their study overcomes major hurdles to unraveling the molecular mysteries of liver diseases and finding desperately needed new, personalized therapies.

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Dr. Takanori Takebe Receives Prestigious Award for Excellence in Scientific Research

Dr. Takanori Takebe was selected as one of six young Japanese researchers to receive the JSPS PRIZE. The Japan Society for the Promotion of Science (JSPS) offers this annual award to recognize and support young researchers with superlative research abilities in early stages of their careers. Recipients have the potential to become world leaders in their fields. Dr. Takebe received the award from the Imperial Prince of Japan in February 2019.

Learn more about PRIZE >

Dr. Jim Wells Records Podcast for Stem Cell Podcast

Jim Wells discusses "The Power of Organoids" in a podcast offered by The Stem Cell Podcast. The group offers a series of podcasts to create a resource for scientists to keep current on the latest developments in stem cell research, as well as to interview key influencers in the stem cell field about their work and perspectives on the field.

Listen to the Podcast >

CuSTOM Scientists Grow Human Esophagus Tissue

Dr. Jim Wells and other CuSTOM scientists work to bioengineer the entire human gastrointestinal system in a laboratory. They now report using pluripotent stem cells to grow human esophageal organoids. These tiny organoids enable personalized disease diagnosis and regenerative therapies.

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Dr. Jim Wells Speaks at TEDx Cincinnati

At an independently-organized TEDx event in Cincinnati, Jim Wells discussed how the CuSTOM team is using stem cells to grow miniature versions of human organs in the laboratory to help diagnose patients and improve care. The team is working to generate lab-grown organs for future transplantation into patients.

Watch the TEDx Talk >

Cell Reports Highlights Recent Complex Organoid Engineering Method

Clinical transplantation of tissue fragments, including islets, faces a critical challenge because of a lack of effective strategies that ensure efficient engraftment through the timely integration of vascular networks. Takanori Takebe and his team recently developed a complex organoid engineering method by ‘‘self-condensation’’ culture based on mesenchymal cell-dependent contraction, thereby enabling dissociated heterotypic lineages, including endothelial cells, to self-organize in a spatiotemporal manner. The successful adaptation of this method for generating complex tissues from diverse tissue fragments derived from various organs, including pancreatic islets, was reported in the cover article of the May 8, 2018 issue of Cell Reports.

$5 Million Challenge Grant

The Farmer Family Foundation pledges a $5 million gift to advance pioneering research being conducted at Cincinnati Children’s Center for Stem Cell and Organoid Medicine. This generous challenge grant, will encourage other donations and accelerate the clinical translation of organoid technology to one day transform the health care of children.  Read more.

Organoids: the body builders

Stem-cell-derived organoids offer increasingly sophisticated models for studying human development and disease, as well as powerful emerging tools for precision medicine. Researchers at Cincinnati Children's are converting stem cells into organized three-dimensional organoids that mimic the structure and function of organs in order to create a higher-order structure. Featured in Nature.

Mass Production of human Liver Buds in the lab

A team led by Takanori Takebe reports methods for efficient clinical-scale production of millions of human liver bud organoids from induced pluripotent stem cells (IPSC). The research published in Cell Reports (Takebe et al) provides basis for mass-production of transplantable (clinical grade) liver organoids particularly for treating pediatric liver disease. Transplantation of these liver bud organoids rescued mice with acute liver failure. Featured in our press release and in Healthline.

Team grows mini-colon in a dish

A team led by the Wells lab report the generation of human colonic organoids from pluripotent stem cells (Munera et al, Cell Stem Cell 2017 ). This world’s first opens the door to study and develop therapies for disease of the larger intestine such as ulcerative colitis and colorectal cancer. Editor's Choice, Science Magazine.

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Uncovering the origins of trachea and esophagus birth defects

A collaborative team of clinicians, embryologists and stem biologists at CuSTOM have been award a large consortium grant (P01 HD093363) from the National Institutes of Health to uncover the developmental basis of life-threatening trachea and esophageal birth defects. The team led by the Zorn, Kingman and Wells labs will use genetic screening in patients, animal models and human esophageal organoids to advance our understanding and treatment of these devastating pediatric conditions.

Bioengineered human livers mimic natural development

An international team of researchers led by the Takebe lab bioengineering human liver tissues uncovered previously unknown molecular networks that control the organ’s developmental processes – greatly advancing efforts to generate healthy and usable human liver tissue from human pluripotent stem cells (Camp et al, Nature 2017). Featured in Science News.

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