A Patient-Derived hiPSC Neuronal Platform for Drug Discovery in Parkinson’s Disease and Amyotrophic Lateral Sclerosis

Project Overview

Parkinson’s Disease (PD) and Amyotrophic Lateral Sclerosis (ALS) are two of the most common and devastating neurodegenerative diseases, affecting more than 2% of the population over the age of 65. However, a major hindrance in developing therapies for disorders of the brain has been limited access to human neurons from affected patients. A breakthrough came from the field of stem cell biology, in which a group of factors were identified that could transform skin or blood cells from any individual, into stem cells, termed an induced pluripotent stem cell (iPSC). These cells possessed all the normal features of a stem cell, and more importantly; could be induced to form many different cell types, including neurons. Thus, with easy access to human neurons, diseases such as PD and ALS could now be modelled and interrogated in a dish. The interdisciplinary team led by Dr Edward Fon at the Montreal Neurological Institute and Hospital (MNI) in partnership with the Centre for Drug Research and Discovery (CDRD) proposes to develop a “hiPSC-based drug discovery platform in Canada focused on PD and ALS”. Taking advantage of these advances, the team will provide curated PD and ALS patient samples along with expertise in the development of exquisitely disease relevant cell-based assays. They believe it will be the access to hiPSC lines from patients and the team’s broad and cutting-edge expertise in these disorders that uniquely positions them to succeed where earlier attempts by pharma, going at it alone, have failed. The team will generate and comprehensively test a panel of PD and ALS patient-derived hiPSC neurons in a suite of state-of-the-art, industry standard, cell-based assays to enable the discovery of novel therapies.

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