Disease modeling has been one of the bigger focal points of this past year, and 2019 will be closing on a high note in terms of developments in this realm, as three different collaborative effects are underway to develop new options for disease modeling. The Johns Hopkins School of Medicine is partnering with Bloomberg Philanthropies and the New York Stem Cell Foundation Research Institute to further precision medicine by using stem cells to generate better disease models; Hesperos Inc. and the University of Central Florida are applying funding to further develop human-on-a-chip technology to model opiate overdose and treatment impact on multiple organs; and MIMETAS and Hubrecht Organoid Technologies are working to develop organoids-on-a-chip.
Better precision with stem cells
NEW YORK & BALTIMORE—Three organizations—the Johns Hopkins School of Medicine, the New York Stem Cell Foundation (NYSCF) Research Institute and Bloomberg Philanthropies—are looking to combine their respective strengths in a new initiative. The collaboration will explore new ways of elucidating disease states through the use of pluripotent stem cells and advance Johns Hopkins’ Precision Medicine Initiative, which is focused on identifying biomarkers to provide better information on diseases and enable more personalized treatments. The partners also plan to make a large number of human disease models globally available.
“Johns Hopkins is working intensively to realize the great promise of precision medicine for all those in our care, locally and globally,” said Johns Hopkins President Ronald J. Daniels. “This significant new collaboration with Bloomberg Philanthropies and NYSCF moves us ever closer to that aim as we join together our far-reaching research capacities to advance knowledge and deliver better health outcomes for populations and people around the world.”
Induced pluripotent stem cells generated from patients will enable researchers to study differences in disease markers and clarify how diseases manifest differently in different patients. Beyond making it possible to offer more tailored treatments, it’s also hoped that this will help researchers pinpoint all possible genetic mechanisms and pathways implicated in different diseases.
Samples will be taken from participating patients at the Precision Medicine Centers of Excellence at Johns Hopkins and used to create stem cell models of disease through the application of the NYSCF Global Stem Cell Array. This technology can reprogram skin or blood cells into stem cells, differentiate them into disease-relevant cell types, and then perform genome editing to explore the genetics behind the condition. The plan is to examine a variety of diseases, including multiple sclerosis, Alzheimer’s disease, chronic renal failure and cancers of the lung, breast, prostate, pancreas and bladder.
“The NYSCF Research Institute has invented and scaled the most advanced methods of human cell manipulation, which is critical for studying disease at the level of the individual patient,” commented NYSCF CEO Susan L. Solomon. “By combining our capabilities with Johns Hopkins’ extensive clinical data and expertise, we will be able to develop effective, personalized therapies for patients suffering from diseases with a high unmet need.”
Opiates and organs
ORLANDO, Fla.—On a different front, another effort is looking to better model the effects of drugs—both harmful and benign—on different organs. Thanks to the first phase of a $3.8-million milestone-based grant from the National Institutes of Health, Hesperos Inc. and the University of Central Florida will look into how overdosing on opiates affects the body’s organs, as well as how drugs meant to save individuals from overdose can have their own deleterious effects on various organs. Dr. James Hickman, chief scientist at Hesperos and a professor at UCF, is leading this work as principal investigator.
The work will be centered on Hesperos’ human-on-a-chip technology. Hesperos will develop overdose models in a multi-organ system to study opiate use and the immediate and long-term effects of overdose treatments on recovery. The work will look primarily at the kidneys, heart, liver and muscles, as well as how the parts of the brain that control autonomic processes such as breathing are impacted.
“We are grateful to have funding to support research in an area that represents such a large and growing need,” Hickman remarked. “Our interconnected human-on-a-chip system provides a non-invasive way to emulate the response of compounds among all ‘organ’ compartments, and to concurrently predict potential toxicity and efficacy of drugs, including opioids and opioid antagonists such as Narcan.”
The human-on-a-chip system was developed at UCF by Hickman and Michael Shuler, president and CEO of Hesperos and professor emeritus, Cornell University. The technology was licensed to Hesperos.
Mimicking organs with MIMETAS
LEIDEN & UTRECHT, The Netherlands—Last but not least, MIMETAS and Hubrecht Organoid Technologies (HUB) shared the news of a strategic collaboration under which they would market organoids-on-a-chip models. This agreement provides MIMETAS with a license to market HUB Organoids in an assay-ready format, and the companies will work together on the development of disease-specific models. Specific financial details were not disclosed.
The companies are both pursuing organ-on-a-chip technology. MIMETAS’ OrganoPlate supports 3D cell culture under continuous perfusion, with membrane-free co-culture and epithelial and endothelial tubules, according to the company. MIMETAS develops customized disease, toxicology and transport models, including models for the kidney, liver, brain and gut. HUB has made a name for itself in the development of stem cell-derived ‘mini organs,’ or organoids, and HUB’s Organoid Technology makes it possible to generate in-vitro models of any epithelial disease.
“We are excited to work with MIMETAS and add a range of new assay systems to the HUB Organoid platform,” said Dr. Rob Vries, CEO at HUB. “The OrganoPlate platform is unrivaled in its field. It is a perfect fit with our biology and enhances ease of use, imageability and screenability of complex Organoid models. Together we will develop diseased and healthy patient-relevant models that serve the needs of the drug development industry.”