Open-science model for drug discovery expands to neurodegenerative diseases

Open-science model for drug discovery expands to neurodegenerative diseases
Dr. Aled Edwards. Credit: M4K Pharma and Ontario Institute for Cancer Research

Parkinson's disease and Amyotrophic Lateral Sclerosis are the newest frontiers for open science drug discovery, a global movement led by academic scientists in Toronto that puts knowledge sharing and medication affordability ahead of patents and profits.

Medicines 4 Neurodegenerative Diseases (M4ND Pharma) will pursue promising new genetic targets for these intractable nervous system disorders, thanks to $1.5 million from the Krembil Foundation. It will be the world's second drug discovery company committed to after Medicines 4 Kids (M4K Pharma), which launched in 2017 to develop a novel drug for an uncommon but fatal childhood brain cancer.

Open science is a way for researchers to share their data and knowledge quickly and publicly, unencumbered by patents and the peer review publishing process, with the aim of speeding up . The movement gathered force in the in the 1990s with the Human Genome Project, and spread to protein structures and then early-stage drug discovery through the Structural Genomics Consortium (SGC).

The non-profit SGC has generated considerable private and public investment and several spin-out companies, but there remains a gap in late-stage drug development.

"When we started M4K, many people thought an open approach to late-stage drug development might only be applicable to rare or neglected diseases, if at all," says Aled Edwards, a professor of molecular genetics at the University of Toronto and CEO of the SGC. "But we're getting unexpected funding and scientific contributions from industry, academic and clinical sources, and slowly but surely we're advancing a medicine through the pipeline. It's time to move the goal posts again on what's possible with open science."

Like M4K, M4ND will not seek patents on its findings. It will instead rely on regulatory protections available in several countries, including data exclusivity for drug sponsors to prevent generic competition, and orphan drug exclusivity for rare diseases. These and other existing protections, says Edwards, are likely sufficient to attract commercial partners willing to manufacture, distribute and sell medications at reasonable prices.

Also like M4K, M4ND will be owned by the Agora Open Science Trust, a Canadian charity that supports open science for the public good. M4ND will donate any proceeds generated through commercial partnerships back to the charity.

The researchers will share their progress with the scientific community through regular online meetings, open to all and posted on YouTube. They will also make data available, for example through the SGC's open lab notebooks initiative.

"Every player in the system appreciates the idea of affordable medicines faster, but it's been incredibly challenging to make it happen because the system is complicated," says Edwards. "The key was to invent a different business model, and we're extraordinarily happy that industry, academia and others have been delighted to contribute."

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


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Feb 16, 2019
I am deeply distrustful of this. It sounds like a Trojan horse for drug companies many of whose execs have profited heavilyl off taxpayer funded open source science...the ONLY kind of science that ever works, by pricegouging sick people for profit, and in many cases stifling important reserach for decades (BRCA drugs) through technology obfuscation. Its a deal with the devil. What this really seems like is an attempt by drug companies to skirt patent laws by co-opting corrupt governments to fascistically enforce their monopolies while still benefiting from open source science. This will result in technological feudalism and ultimatley a new dark ages for science, even if their is some short term capital infusion by the grifters and robbers from this industyr anyways. If we want human progress to go forward in drug and many other technologies, we should be breaking up these companies and nationalizing some of them as well.

Feb 16, 2019
Let companies do what they do best, competing amongst themselves in making and marketing things, but not owning or worse hiding ideas science or bankrolling it with the sick and desparate people they exploit to maximize their profits. There is more than enough money to be made doing that and doing that well, and we will give far more ethical and hard working people in big pharma if we did that...and many other technologies as well. No company should have the right to obfuscate, hide, or control the use of human knowledge. They can help bring it to good uses and compete amongst themselves to do so...and that is their best use. We need to roll back the first to file laws, limit patents, and increase funding and rewards to the true creators of technology...not their owners, buyers, and grifters. If we don't do that, we will get worse copycat drugs, biased science, and technoogical feudalism. Human progress will be set back thousands of years if we couple feudalism with technology.

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