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Microphysiological systems: A promising new platform for Alzheimer’s drug development

Dr. Zane Martin
Program Director,
Division of Neuroscience (DN)
Dr. Nadezda Radoja
Nadezda RADOJA,
Senior Advisor for Regulatory Science,
Division of Neuroscience (DN)

One of the biggest and most persistent challenges in the quest for effective Alzheimer’s disease and related dementias therapies is finding better models for preclinical drug development. While animal models have been the gold standard for years, more than 95% of resulting investigational drugs have failed in clinical testing, emphasizing the difficulties in translating those models to human therapies.

Microphysiological systems (MPS, also called tissue chips or organs-on-chips) are a fast-growing technology with intriguing potential for improving and expediting the drug development process. These systems contain interconnected sets of different types of lab-grown cells, such as immortalized cell lines or induced pluripotent stem cells (iPSCs), usually packaged in a chip about the size of a standard microscope slide.

MPS platforms offer unprecedented physiological accuracy for the study of interactions among cells, drugs, and organs. Ultimately, MPS technology may advance the development of precision medicine strategies for effectively treating Alzheimer’s and related dementias, but it also presents several technical and regulatory challenges.

Growing MPS interest and resources

To explore these opportunities and challenges, last summer NIA convened a workshop (PDF, 588K) on MPS models and their potential for Alzheimer’s and related dementias therapy development. We assembled representatives from the broader NIH, the FDA, academia, and the biotech and pharmaceutical industries to learn about the latest in MPS technology, scientific rigor/reproducibility, and regulatory issues.

Around the same time, an exciting new resource in the field came online from the NIH Center for Alzheimer’s and Related Dementias (CARD) and its partners. A CARD research team developed an extensive catalog of human iPSCs for Alzheimer’s and related dementias, amyotrophic lateral sclerosis, and other neurodegenerative disorders. Be sure to check out the resources and protocols to see how this powerful research catalyst can boost your own MPS studies and translational efforts!

New MPS funding opportunity: Apply by March 27, 2023

If you’re eager to dive into the MPS world, apply for a new funding opportunity announcement (FOA) by March 27, 2023. Led by the NIH National Center for Advancing Translational Sciences with NIA participation, this FOA is designed to establish centers to support research to accelerate the translational use of MPS in drug development and support regulatory acceptance and adoption by industry. Learn more by reading the related NIA Notice of Special Interest.

NIA will host pre-application technical assistance webinars for this opportunity on Jan. 10 and 24, 2023, 3-4:30 p.m. ET. If you’re interested in attending a webinar, please email Zane. If you have questions, please email Zane or Nadezda. We look forward to the next big steps for this powerful technology in small packages!

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