An open science model for drug discovery extends to neurodegenerative diseases

Parkinson's disease and amyotrophic lateral sclerosis are the latest frontiers in the discovery of open scientific drugs, a global movement led by Toronto-based academic scientists that places knowledge sharing and affordability ahead of patents and profits.

Drugs 4 Neurodegenerative Diseases (M4ND Pharma) will pursue new promising drug targets for these insoluble nervous system disorders with $ 1.5 million from the Krembil Foundation. This will be the second global drug discovery company engaged in open science after Medicines 4 Kids (M4K Pharma), which launched in 2017 the development of a new drug for the treatment of rare brain cancer but deadly in the child.

Open science is a way for researchers to share their data and knowledge quickly and publicly, without being bothered by patents and the peer publishing process, to accelerate scientific discovery. The movement stepped up in the life sciences in the 1990s with the Human Genome Project and extended to protein structures and then drug discovery at an early stage through the Consortium of Structural Genomics (SGC).

The non-profit SGC has generated considerable private and public investment and several spin-off companies, but the development of advanced drugs remains insufficient.

"When we launched M4K, many people thought that an open approach to late-stage drug development might only apply in the case of rare or neglected diseases," says Aled Edwards, professor of genetics. at the University of Toronto and Director General of the GSC. "But we are getting funding and unexpected scientific contributions from industry, universities and clinical sources, and slowly but surely we are moving a drug into the pipeline." It's time to move the targets back to the pipeline. possibilities of open science. "

Like M4K, M4ND will not seek a patent for its findings. Instead, it will rely on the regulatory protections available in several countries, including the exclusivity of data for drug sponsors to prevent competition from generic drugs, and the exclusivity of drugs. orphans for rare diseases. According to Edwards, these protections, along with other existing protections, are likely sufficient to attract trading partners willing to manufacture, distribute and sell drugs at reasonable prices.

Like M4K, M4ND will belong to Agora Open Science Trust, a Canadian charity that supports open science for the public good. M4ND will donate the proceeds of its sales to the charity through commercial partnerships.

Researchers will share their progress with the scientific community through regular online meetings, open to all and posted on YouTube. They will also make the data available, for example through the initiative of SGC open laboratory notebooks.

"All the players in the system are quicker to appreciate the idea of ​​affordable drugs, but it has been incredibly difficult to make it happen because the system is complicated," Edwards says. "The important thing was to invent a different business model, and we are extremely pleased that the industry, academia and others were delighted to contribute."

Dr. Edwards will present an open drug discovery at the annual meeting of the American Association for the Advancement of Science at 2:30 pm on February 16, 2019, Marriott Wardman Park – Maryland Suite, Washington, D.C.

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