Guy Rouleau is the director of the Montreal Neurological Institute-Hospital, chair of the Department of Neurology and Neurosurgery at McGill University, and director of the Department of Neuroscience, McGill University Health Centre. He is also the recipient of the 2020 Canadian Gairdner Wightman Award. The following members of his team contributed to this essay: Damien Chalaud, Viviane Poupon, Dylan Wade Roskams-Edris and Annabel Seyller.
The COVID-19 crisis has taken medical research from relative obscurity to front-page news. Those of us who have spent years toiling in our labs, trying to understand diseases and searching for treatments, suddenly find ourselves at the centre of the world’s attention. On my street in Montreal, neighbours have taken a new interest in my work as a medical scientist. Only the discovery of an effective treatment or a vaccine will allow us to resume our normal lives.
In labs around the world, we are seeing an unprecedented level of sharing as medical scientists work collaboratively to find solutions to the pandemic, such as a diagnostic test to detect antibodies that confer immunity to the virus, drugs for treatment and, of course, a vaccine. Typically, developing a vaccine for a new viral entity takes, on average, 10 years. It is hoped that this process will be accomplished in as little as 18 months. Physicians, researchers, policy makers, industry and patients are working together to find solutions. This global collaboration, where knowledge is fully shared in real time, is a powerful illustration of medical research at its best.
Unfortunately, until now, this level of open sharing – known as Open Science – has not been the norm in medicine.
What most people don’t know is that the world of academic medical science is handicapped by a system that largely prevents the free sharing of information between scientists, hampering discovery. We medical scientists are all searching for pieces of the complex puzzles that will help us understand and cure human diseases. But if these pieces are not shared so they can be assembled into a meaningful whole, it will continue to be difficult to find the answers we seek.
The term “closed science” is used to describe the current system in which neither data nor reagents are freely shared by academic medical scientists. Data refer to information generated in regard to patients, their diseases and all other relevant biological information. Reagents are the tools, such as antibodies and cell lines, created by scientists to study the biology of disease.
Until COVID-19, the norm in academic medical research was not to openly share critical information and tools, but rather to keep them proprietary and protected in order to extract value from them. On the surface, this approach appears to make sense. One of its objectives has been to generate revenue by monetizing the information or reagents obtained from university and hospital medical research. Another reason often cited in support of this closed system is that it is believed to benefit scientists and institutions by providing them with greater opportunities for recognition in the form of prizes and career advancement. But the closed system has largely failed at cost recovery, nor is it the only way to attain recognition for one’s work.
The closed system’s failure to recover research costs is widely documented. In Canada, the institutional costs for managing scientific information and reagents through universities’ technology transfer offices turns out to be roughly equal to the revenues these activities generate. Then there are the hidden costs associated with this closed system: delayed or even missed opportunities for collaboration, duplication and sometimes even triplication (or more) of the same work, all of which hinder the discovery of disease mechanisms and treatments. Individuals with diseases such as cancer, amyotrophic lateral sclerosis (Lou Gehrig’s disease) or dementia, and those who love them, understand there is no time to waste. In addition, people are continuously developing such diseases. A five-year delay in finding a treatment to prevent dementia means that another 50 million people worldwide will develop this devastating disease.
Most funding for academic medical research comes from taxes or philanthropic donations. However, we would wager that neither taxpayers nor philanthropists would be pleased to learn that the research done with their money is being locked up in a failed closed system. Even if university and hospital medical research labs are doing excellent research – and they are – the research outputs are not being shared in a way that leads to optimal discovery of desperately needed treatments. In this way, these institutions are not fulfilling one of their core missions: the pursuit and sharing of knowledge for the common good.
This closed system simply doesn’t work.
It has been around for a long time. In the early 1990s, the federal government was seeking new ways to finance academic medical research, and decided to encourage universities to take more aggressive patent positions by enabling them to retain title to inventions and take the lead in patenting and licensing discoveries, aiming for a financial windfall. The Canadian government and Universities Canada (the Association of Universities and Colleges of Canada at the time) sealed this policy through their Framework of Agreed Principles on Federally Funded University Research in 2002.
So why are institutions clinging to this failed model? One of the ways our federal and provincial governments measure the success of academic institutions is by the number of patents and licences generated. According to Thomson Innovation, for every 3,000 Canadian academic patents in regenerative medicine, only one new company is formed.
Compare this with California, where one company is formed for every 180 patents. In other words, there is a great deal of patenting in Canadian universities with little to show for it. There must be better ways than counting patents to measure medical innovation and benefits to Canadian society.
Another way universities frequently control data and reagents is by sharing them through what are called material transfer agreements (MTAs). These tend to have many restrictive clauses to guarantee that universities get a piece of any eventual profits. Such restrictive clauses significantly increase negotiation time between institutions and are expensive. Sometimes, when a university asks for more than what is fair, the deal falls through – and no sharing occurs. Recently, physician researchers at a Canadian university hospital were unable, because of the unreasonable requirements of the MTAs, to share a small number of properly consented patient samples with a large international not-for-profit consortium doing important research on a neurological disease. The failed negotiations lasted two years – especially tragic considering the average life expectancy of an individual with this disease is two years. The patients who donated their samples were denied the opportunity to contribute to meaningful research on their disease.
When confronted with information suggesting the closed model has failed, hospital and university administrators invariably defend their reluctance to abandon the current system on the basis of their fear of missing out on windfalls. Gatorade sometimes comes up. It’s a well-known example of a university discovery that made a lot of money. In 1965, medical researchers at the University of Florida were asked by the school’s Gators football team to create a drink to help prevent heat exhaustion. The university’s researchers invented Gatorade, which they patented in 1967, and which not only became the world’s most commercially successful sports drink, but has earned the University of Florida some US$300-million in royalties.
However, there are few such examples in Canada or the world. The most frequently cited Canadian example is Pablum, a cereal containing vitamin D, which prevents rickets in children, and which was developed by a team at Toronto’s Hospital for Sick Children – but that was back in 1930. Still, the remote possibility of winning the windfall lottery keeps universities hanging on to this closed model.
But there is an alternative to closed science – one that has been brought to the forefront by the global pandemic we are currently experiencing.
Open Science is the practice of freely sharing data, information, materials, tools and research results in a timely manner without restrictions. The approach dates back to the 1980s and the information-technology revolution. Computers had been around since the 1950s, but were largely inaccessible to the general public. In the 1980s and 90s, personal computers and the internet gave many of us access to this technology. Around this time, a critically important decision was made by the IT community to openly share software, a practice known as open source. This decision led to the explosion and democratization of IT, changing all our lives.
Paradoxically, the pharmaceutical industry, despite its dependence on patents, is a strong supporter of Open Science. That’s because the industry is results-driven and understands that time matters. It wants diseases solved so it can get on with the business of producing new effective treatments. Big Pharma understands that Open Science accelerates discovery and will more rapidly bring them the information it needs to develop such drugs. And although the pharmaceutical industry’s desire for speed is largely driven by the desire for profits, new treatments benefit all of us.
Patents and licences are not the only ways for universities to be compensated for academic medical research investment. At a recent conference, Marc Tessier-Lavigne, the Canadian-born president of Stanford University, explained that his university does not seek to directly retain value from discoveries made in its labs. Dr. Tessier-Lavigne pointed out, however, that when there are windfalls, the beneficiaries frequently give back to the university in the form of major donations. If Dr. Tessier-Lavigne is right, creating barriers to collaboration for the sake of windfall profit is misguided.
The Open Science approach has been discussed in the biomedical field for decades, but remains the exception when it should be the rule. Since Open Science accelerates discovery, the need for it has never been more obvious than now, during the COVID-19 pandemic.
If sharing openly is critical to accelerate understanding and treating COVID-19, which has already infected more than 12 million people, why not take the same open approach to cure Alzheimer’s disease, breast cancer, prostate cancer, degenerative arthritis, Parkinson’s and all the other diseases that plague us? Billions suffer and die from these diseases.
As a neurologist, I am frustrated by the lack of progress in finding new treatments for my patients. That’s why in 2016, we at the Neuro (Montreal Neurological Institute-Hospital) decided to adopt Open Science principles and, together with the Larry and Judy Tanenbaum Family Foundation, created the Tanenbaum Open Science Institute, making us the first institution of any kind in the world to fully adopt Open Science.
At the Neuro, Open Science enables us to more effectively fulfill our mission: to understand the brain, find cures and effectively treat people with neurological disorders. Our adoption of Open Science has also facilitated our ability to attract a new generation of world class scientists, most of whom were raised in the age of open-source internet. We have also been able to attract greater interest and investment from industry because of our openness to collaborate freely and more rapidly. Philanthropists, patient organizations and patients themselves are all eager to support our work. Many have become important ambassadors for Open Science.
COVID-19 has caused immeasurable suffering to millions of people worldwide. And yet, in all kinds of ways, this pandemic is teaching us valuable lessons. One of them is that medical science must be open.
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