Validation trials to explore promise for organoids

Organoids will be used in experimental drug research, with an original focus on cystic fibrosis

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UTRECHT, The Netherlands—Hubrecht Organoid Technology (HUB) and the health insurance companies CZ, Zilveren Kruis and Menzis of the Netherlands have recently launched a €3-million validation trial for use of HUB Organoid Technology to test if it can be used to determine the response of cystic fibrosis (CF) patients to new drug therapies. The exploration is seen as good news for researchers looking to predict likely human response to experimental drugs in preclinical development stages.
Hans Clevers, chief scientific officer of HUB, said: “The new project is a major next step for the implementation of the HUB Organoid Technology. We are very happy that this very recently developed technology might already benefit patients.”
An organoid is a miniaturized and simplified 3D version of an organ that shows realistic micro-anatomy. They are derived from one or a few cells from a tissue, embryonic stem cells or induced pluripotent stem cells, which can self-organize in three-dimensional culture owing to their self-renewal and differentiation capacities. Rooted in Clevers’ discovery that the identity of adult epithelial stem cells is conserved among organs, HUB Organoid Technology developed a method for in-vitro culturing of not only the stem cells, but the differentiated offspring of these organ specific stem cells. Their in-vitro cultures proved to be not only genetically stable but also to functionally mimic the in-vivo function of the epithelium.
According to Robert Vries, chief operating officer and managing director of HUB, the current focus explores the potential for organoids in experimental drug development. “The major hurdle in drug development has been the lack of relevant models. The organoids fundamentally change the possibilities … They proved to be a truly personalized medicine tool in cystic fibrosis, where we demonstrated that the organoids predict the patient’s response,” says Vries. “We have now started a validation trial to demonstrate that indeed the organoids can be used as a companion diagnostic and/or as a technology to stratify patients for clinical trials either prospectively or retrospectively.”
While their initial aim seeks a treatment for cystic fibrosis, they plan similar validation trials for colon cancer, lung cancer and other diseases. “We are now working on cystic fibrosis, several cancers, inflammatory bowel disease, some liver and kidney diseases, as well as virus and bacterial diseases that involve the epithelium. In principle, all disease of the epithelium can benefit,” asserts Vries.
The new project will demonstrate if the HUB Organoid Technology can be used as a predictive test for treatment, which would greatly help to treat cystic fibrosis patients effectively. CF is a life-threatening disease with newly developed breakthrough drug treatments that have proven to be effective in some CF patients. However, the costs of these therapies and the limited benefit in certain patients have limited insurance coverage for such therapies. Being able to systematically identify the responding patients may ensure ongoing access of the therapy.
“It is important that also expensive medication is available for patients, and therefore treatment needs to be carefully considered so that we treat only patients who respond. Because we do not want to exclude anyone who requires new medication, this validation trial is very important to develop a test that will show who does and who does not benefit from a specific therapy,” said Joep de Groot, part of the board of directors of CbusineZ, an innovation company of health insurer CZ.
Should the validation tests prove to serve as a predictor for efficacy, this organoid technology offers broad potential for widespread experimental drug discovery. HUB maintains a biobank of organoids, characterized by genome sequencing, expression profiling and sensitivity to known and experimental drugs to establish a database linking genetic and transcriptional information to drug responsiveness. HUB offers licenses to its patented HUB Organoid Technology for drug-screening and access to organoids in the HUB biobanks for preclinical drug discovery and validation.
“Organoids are the first truly functional personalized medicine tool, and we are continuously increasing the models and their application of the organoids. We have generated numerous commercial applications which are increasingly used by pharma companies, and clinical applications currently implemented in collaboration with Dutch national regulatory authorities, but HUB is discussing this with FDA and EMA as well,” concludes Vries.

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