Search impact stories
Video Content: 
0
April 2022

Consortium removes barriers to drug discovery, raises hope for rare disease cures

At a glance
The team

Structural Genomics Consortium (SGC), a Canadian-led consortium of big pharmaceutical companies, clinicians and academia, supports open-access drug discovery around the world. 

The challenge

The market is too small to justify the costs for research into cures for rare diseases using the traditional model for drug discoveries – meaning many scientists do not conduct research into them and treatments can be prohibitively expensive.  

The solution

The SGC is building an innovative research ecosystem with the goal of providing researchers with the tools and knowledge needed to study rare diseases and emerging viral infections. The group makes its research output available to the scientific community patent-free, with no strings attached. It is also committed to affordability. As pledged by consortium spin-offs, any new treatments will be priced to be accessible to anyone who needs it. 

The outcome

SGC is identifying chemical probes for understudied protein families in the human genome. The chemical probes can be used by researchers all over the world as tools to unlock new areas of disease biology, supporting the identification of new proteins that are likely to lead to cures and treatments for common diseases like cancer, Alzheimer’s and Parkinson’s – as well as rare diseases.

When it comes to finding cures for rare diseases and addressing emerging viral infections — like COVID-19 — the traditional pharmaceutical business model just doesn’t cut it. Solutions require global collaboration and open-access research, and that means a new research innovation ecosystem — exactly what the Structural Genomics Consortium (SGC) is building.

Structural Genomics Consortium (SGC), the Canadian-led consortium of pharmaceutical companies, clinicians and academia, supports open-access drug discovery around the world. And it’s bringing hope to the 300 million people afflicted by rare diseases and a world ravaged by COVID-19. 

“SGC is building an innovative research ecosystem whose goal is to empower scientists all over the world with tools and knowledge to explore challenging diseases, so one day rare disease patients may have access to affordable treatments,” says Dr. Arij Al Chawaf, former SGC Director of Strategic Alliances and current Executive Director for Strategic Initiative Development at an SGC partner, the University of Toronto. 

“The market is too small to justify the costs for rare disease cures, according to the traditional model for drug discoveries,” she says. “But when we provide the right tools to researchers all over the world, these discoveries are within reach.” 

Mitacs is a funding partner with the consortium — which includes Bayer Pharma AG, Boehringer Ingelheim, Bristol Myers Squibb, Genentech, Genome Canada, Janssen, Merck KGaA, Pfizer, Takeda, and Wellcome Trust. Several of the SGC’s postdoctoral researchers have provided valuable research contributions to new drug discoveries through the Genes to Affordable Medicine Mitacs supercluster. Through the supercluster, Mitacs researchers acquired valuable experience by participating in open science collaboration with pharmaceutical company partners and AI companies. 

As a result of this experience, many Mitacs-SGC alumni are now academic or business leaders, including professors and chairs in renowned institutions in Canada and abroad or have assumed scientific leadership positions in major pharmaceutical companies. 

A paradigm shift

By removing intellectual property restrictions and freely sharing the building blocks required to carry out experiments on the proteins that make up the human genome, the SGC is driving a seismic shift in the pharmaceutical industry. The consortium makes it feasible to pursue affordable treatments for rare diseases, such as childhood cancers, and respond rapidly to emerging viral infections. 

SGC catalyzes research in new areas of human biology and drug discovery by focusing explicitly on less well-studied areas of the human genome. The consortium accelerates this work by making its research output available to the scientific community with no strings attached, and by creating an open collaborative network of scientists in hundreds of universities around the world and nine global pharmaceutical companies. 

Together, SGC’s network of academic and industry scientists is building a new scientific and drug discovery ecosystem whose primary aim is to advance science — not the pursuit of personal, institutional or commercial gain. And the SGC’s collaborative model has proven to be an excellent training environment for students, offering a seamless transition for trainees between industry, not-for-profit organizations and academia. 

“The goal is to better equip and empower scientists everywhere to explore challenging diseases, so one day rare disease patients may have access to affordable treatments,” says Dr. Al Chawaf. The SGC has approximately 200 scientists in six major research labs at universities in Canada, the United States, and Europe, with the largest hub located at the University of Toronto. 

Accelerating drug discovery efforts around the world 

Founded in 2002, the organization’s current focus is on identifying chemical probes for understudied protein families in the human genome. In using the open science model of offering data patent-free, the chemical probes can be used by researchers all over the world as tools to unlock new areas of disease biology, supporting the identification of new proteins that are likely to lead to cures and treatments for common diseases like cancer, Alzheimer’s and Parkinson’s — as well as rare diseases. 

The SGC regularly releases chemical probes to the research community to support and accelerate drug discovery efforts around the world. These probes are the foundation of a more recent SGC initiative known as Target 2035, which aims to develop and publicly 

release probes for every protein in the human proteome in the next 13 years. Through Target 2035, SGC plans to identify chemical compounds to modulate each of the roughly 20,000 proteins in the human body. 

The group’s open science model has already laid the intellectual foundation for more than 50 clinical trials. Another SGC differentiator is a commitment to affordability, pledged by consortium spin-offs. Any new treatments will be priced to be accessible to anyone who needs it. 

In recognition of its success in providing those tools, the SGC were awarded the 2021 Mitacs Award for Exceptional Leadership — Industry during a ceremony in November 2021