In the past 40 years, the model of drug discovery used by the pharmaceutical industry, while proving successful for some applications, has produced very little for neurodegenerative diseases.
“Take Parkinson’s disease, for instance: there have been zero new disease modifying medications developed in the last 40 years, despite hundreds of millions of dollars being invested in research,” says Dr. Edward Fon, whose lab focuses on molecular and cellular research into Parkinson’s disease. “We have to ask if there is a better way to find therapies that will modify the course of diseases, slowing or stopping them from progressing. While Open Science is not going to solve every problem, it will be a big part of the solution.”
The Neuro's drug discovery initiative aims to support research by developing assays that investigate different aspects of target cells and sharing these assays with colleagues around the world. The main vehicle for sharing assays is The Neuro’s Induced Pluripotent Stem Cell (iPSC) platform, developed recently with seed money generously donated by Sebastian van Berkom and Ghislaine Saucier.
“We can provide industry-standard assays researchers can use to test compounds,” says Thomas Durcan, another Parkinson’s researcher who manages the iPSC platform. “In collaboration with researchers at Université Laval, we reprogram blood cells from patients with neurological diseases into stem cells, and then transform the stem cells into the type of neural cells most impacted in neuropathology.”
This gives researchers the ability to compare normal and diseased states of the cells within one patient, which has long been possible for some diseases but is a novel advance for neurological research. The Centre for Drug Research and Development, a Canadian nonprofit organization that aims to speed the translation of discoveries into therapies, and the international Structural Genomics Consortium have both formed partnerships with the Tanenbaum Open Science Institute to use the iPSC platform.
“If we can identify potential compounds for targeting diseases such as Parkinson’s and share them so they can be screened and tested throughout the scientific community, we can accelerate the rate of discovery,” says Dr. Fon. “Other researchers can benefit from the resources we develop, and the chances of 100 laboratories finding something useful is much greater than that of one lab working alone. If we can come up with something leading to treatment for Parkinson’s disease, that would be an incredible payback.”