Thousands of rare drugs are stalled in what is often called the “Valley of Death” — the phase where private companies abandon a drug development project because of its high costs coupled with the high risk of failure.
The National Institutes of Health (NIH) and research universities such as Northeastern are at the forefront of a new movement to overcome those obstacles to drug development. It is based on interdisciplinary collaborations between the public and private sectors to discover new therapies, said Dr. Christopher Austin, the scientific director of the NIH’s Center of Translational Therapeutics and the senior advisor for the director of translational research at the National Human Genome Research Institute.
“This is an area of tremendous opportunity and need,” said Austin, explaining that, of about 6,000 rare diseases that affect humans, only about 200 have treatments. “These are areas that have been made scientifically tractable, in some cases, for the first time ever because of advances due to the Genome Project and other scientific breakthroughs so that we now understand the underlying science and physiology of these disorders that we simply didn’t 10 years ago.”
The ecosystem of translation and therapeutic research is changing, Austin said, from the traditional system — in which the nonprofit sector does the basic science while private industry pursues the real-world applications and payoffs — to a new model based on partnerships and collaborations across disciplines and organizations.
Institutions such as Northeastern are at the forefront of this solution-focused research, for which the NIH is developing milestones to help drug developers move through the necessary steps ahead of human drug trials. Teams work across disciplines — and with researchers from other universities and institutions — to achieve tangible goals to benefit society.
“I think what is going on at Northeastern is a perfect example of this,” Austin said. “This translation of research is obligatorily a team sport and obligatorily a collaborative enterprise in which tremendously skilled researchers from a number of disciplines are required to get a discovery from its basic, mechanistic stage to a point where it would be useful in the real world.”
Universities are also important to the NIH’s work, Austin said, because they train the next generation of scientists and researchers who will work in both the private and public spheres on problems relating to disease and public health.
“The training of people in these areas of chemistry, engineering, bioengineering, information technology and other explicitly deliverable research — applied research — is a huge opportunity and an important mission,” he said.
This shift toward results-based research also leads to outcomes that are easier for political leaders and policy makers to digest than basic, fundamental scientific advancements without real-world applications, allowing for more resources to be devoted to these important projects.
“As someone who works in the public policy space and spends a lot of my time advocating for NIH funding, these paradigm shifts to a focus on applications is extremely helpful because it makes scientific research more tangible for policy makers,” said Tim Leshan, Northeastern’s vice president for government relations and a former colleague of Austin at the NIH.