# Development of a Microphysiological Organ-on-Chip System to Model Amyotrophic

> **NIH NIH UH3** · CEDARS-SINAI MEDICAL CENTER · 2022 · $1,174,019

## Abstract

Project Abstract
There are no animal models for sporadic forms of amyotrophic lateral sclerosis (ALS)
and Parkinson’s disease (PD) that account for over 90% of all patients. As the genetics
of these diseases are not understood there are no drug targets to go for either. This
proposal aims to use MPS devices to produce some of the first sporadic ALS and PD
models of disease. Using patient specific induced pluripotent stem cells from rapidly
progressing patients with early onset for maximal chance of seeing phenotypes, we will
differentiate the cells into motor neurons or dopamine neurons – the two major cell types
affected in the disease. Using cutting edge electrophysiology techniques and
metabalomics combined with RNAseq and proteomic analysis we will establish solid
biomarkers that define these two diseases when compared to healthy controls (UG3
phase). We will then develop novel MPS screens using the NCATS library of
compounds based on the biomarkers discovered in the UH3 phase (UG3 phase).
Together these studies will produce some of the first models of sporadic neurological
disease using iPSC technology combined with MPS technology. Cedars-Sinai and the
company Emulate have a 2 year relationship that forms the foundation of this proposal
and an integrated institutional platform on which to collaborate to achieve the goals of
the study.

## Key facts

- **NIH application ID:** 10438517
- **Project number:** 5UH3NS105703-05
- **Recipient organization:** CEDARS-SINAI MEDICAL CENTER
- **Principal Investigator:** CLIVE Niels SVENDSEN
- **Activity code:** UH3 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2022
- **Award amount:** $1,174,019
- **Award type:** 5
- **Project period:** 2017-09-15 → 2024-02-28

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10438517

## Citation

> US National Institutes of Health, RePORTER application 10438517, Development of a Microphysiological Organ-on-Chip System to Model Amyotrophic (5UH3NS105703-05). Retrieved via AI Analytics 2026-05-22 from https://api.ai-analytics.org/grant/nih/10438517. Licensed CC0.

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