The HUMANOIDTM Center of Research Excellence (CoRE) is the translational arm for Institute for Network Medicine’s drug discovery pipeline, bringing a new level of personalization into disease modeling and clinical trials, providing the ability to test the efficacy of therapeutics in Phase ‘0’.
Current drug-discovery approaches have successfully eradicated all diseases in mice; however, the efficacy of drugs in genetically identical lab animals (e.g., mice), don’t reproduce in clinical trials on real-world patients. Failure is primarily due to lack of efficacy due to patient heterogeneity (i.e., diversity). With each failure, the society bears a tab of ~$2.5B, while patients continue to suffer and our appetite for cruelty against animals in-the-name-of-science only grows.
In recognition of these issues, the Food and Drug Administration (FDA) recently passed the FDA-modernization act which allows drug approvals without testing on mice, if they demonstrate efficacy in human pre-clinical models, such as stem-cell derived mini-organs, called ‘organoids’. Currently, such models have no established benchmarks and are yet to be objectively standardized. This leaves us dangerously exposed to another era of ‘reproducibility crisis’, one that could be potentially deadlier.
The UC San Diego HUMANOIDTM Center is uniquely poised to fulfil this urgent and unmet need. Established in 2019, HUMANOID’s scientists have relentlessly perfected the task of reverse-engineering human organoid-based models of ‘disease-in-a-dish’ that allow rapid testing for drug safety and efficacy in diverse human subjects. The introduction of these studies, as a Phase ‘0’ human trial, can solve many existing problems. Testing drugs in the setting of diversity much earlier than we do currently (i.e., Phase 3) allows the recognition and early termination of programs that are destined to fail.