THE BLOG
05/12/2013 09:27 GMT | Updated 03/02/2014 05:59 GMT

Cures for the Leading Diseases in the Western World are on the Horizon

If I were to say that Europe may be on the brink of stepping into the world of modern technology, I'm sure I'd be met with more than a few raised eyebrows. Are we not already living in glorious gadget gluttony? How many iPads does one person really need? And how much more virtual do we want our reality to become? Yet ironic as it may sound, there is one area of our lives that remains stuck in a time warp -- human disease research.

All this may be about to change, however, with the launch of Horizon 2020 -- Europe's new €70 billion funding initiative for scientific research and innovation. It provides a critical opportunity for European researchers to apply fresh thinking and cutting-edge technologies to tackle pressing societal challenges. In particular, it opens a door for Europe to lead the world in making truly groundbreaking health advances.

Health science has struggled to make significant progress on important diseases such as a number of cancers, asthma and degenerative neurological disorders in recent decades. Asthma is an example of a chronic debilitating disease that affects 300 million people worldwide. Despite decades of animal-based research, only two types of treatment have become available in the last 50 years. Out of more than a thousand potential drugs for stroke tested in animals, only one of these has proved effective in patients. And who knows what more progress we could have achieved by now in the war on cancer, had we not been focusing the battle on mice? "We have cured mice of cancer for decades," Dr Richard Klausner of the U.S. National Cancer Institute famously once said "and it simply didn't work in humans."

The problem is that there are some medical researchers who remain convinced that mice and other animals are essential "models" of human biology and disease. But a number of recent papers challenge this assumption and leading research centers, like Harvard's Wyss Institute, are pointing the way to different approaches to studying and addressing human disease.

Former U.S. National Institutes of Health director Dr Elias Zerhouni says, "we have moved away from studying human disease in humans...The problem is that it hasn't worked, and it's time we stopped dancing around the problem...We need to refocus and adapt new methodologies for use in humans to understand disease biology in humans." The current NIH Director, Dr Francis Collins has written, "With earlier and more rigorous target validation in human tissues, it may be justifiable to skip the animal model assessment of efficacy altogether."

The model of choice for the career-aspiring, grant-chasing scientist over the past 20 years has been a mouse, preferably one with one or more genes modified. But that may be about to change. If the European Commission is serious about overcoming some of the biggest barriers to reversing the paucity of new therapies coming out of the medical research and pharmaceutical industries in recent decades, it will commit to innovative human biology-based research methods. It will also embrace cutting edge technologies such as human stem-cell methods to create human organs on a chip and high throughput cell-based assays to determine the therapeutic potential of thousands of small molecules in a matter of weeks or months. For these are the changes required to improve the health of all Europeans.

So to resuscitate our health research, out must go the current emphasis on mice and in must come the outputs available from cheap human genome sequencing (now around $6,000 per genome), the explosive growth of computational biology and high-speed robotic screening systems, to name just a few of the latest technologies. The 21st century offers a new way of thinking that can help uncover exactly how drugs disrupt normal processes in the human body and identify the underlying mechanisms of disease right down to the level of the very cells and molecules themselves.

This means that with mathematical modeling, population data and virtual patients, scientists can do better, so much better, than the 90 percent failure rate for new therapies (developed using animal models) approved by the US Food and Drug Administration for human trials.

Instead of gambling our medicines -- and our lives -- upon these dismal stakes, scientists can make more meaningful predictions about the effectiveness of new therapies in humans and about their safety that are relevant to people in the real world, and intercept the progression of disease before a patient even receives their diagnosis.

Plus, Horizon 2020 comes at a time when Europe is experiencing an aging and growing population putting increasing economic pressure upon our healthcare systems. It would seem nothing short of reckless not to prioritise the faster, cheaper, more reliable and results-oriented research methods over the traditional animal-based testing methods that are far more costly and time consuming.

The Innovative Medicines Initiative, a public-private partnership set up between the European Commission and the European Federation of Pharmaceutical Industries and Associations, believes that Horizon 2020 "offer[s] unique opportunities to overcome the hurdles which prevent efficient and safe medicines from reaching patients suffering from debilitating diseases."

And remember that in the areas of research that are truly making progress --like understanding human diseases via breathing lungs and beating hearts on chips and developing new treatments for brain diseases with IT technologies that help us understand what makes us human -- there are no mice in their toolbox.

So please, European Commission and Member States, do seize this chance to transform scientific innovation into tangible health benefits for citizens. Cures for the leading diseases in the western world could indeed lie right there in Horizon 2020. Our health has everything to gain if medical research catches up with the other areas of our lives that have embraced the many exciting new (non-animal) technologies of the 21st Century.