NCATS: Increasing the odds of getting to faster cures

By Margaret Anderson, Executive Director, FasterCures
This is old news: the drug development paradigm takes too long, it costs too much, and it’s laden with failures.

But for the thousands of people diagnosed each day with a disease for which there are no cures, or limited treatment options, the reasons – real as they may be – pale in comparison to the great need for living a healthy and productive life.

Of course we need to continue spotlighting the “broken R&D system” and need for a “new business model”. Identifying the problem is half the battle. But what are solutions to these problems? That is what matters most.

Solutions can be tough to develop, and tougher to implement. In the pursuit of solutions, we may second guess: Have we picked the “right” one? How can we hedge against risks? What about failure? Who is on board? Is it the right time to implement?

The road to success is paved with good intentions. It is clear that actions to address these vexing challenges will require bold vision, steadfast leadership, and diverse support. Thanks to our vitally important national investment in scientific discovery, we now know more about disease and biology than ever before.

The opportunity we must seize now is contingent upon having systems in place that will allow these ideas and discoveries to be translated into effective products and therapies that will ultimately improve patients’ health and quality of life. If indeed we are in an era where the scientific knowledge and possibilities are abundant but they are getting stuck in the translational pathway, we have to get moving.

The proposed NIH National Center for Advancing Translational Sciences is one way to get moving. Knowing full well that there are no guarantees in the pursuit of treatments and cures, this proposed center was conceptualized to reengineer the process of developing diagnostics, devices, and therapeutics to increase our odds of success, and streamline a process to make it work better for all sectors and across all diseases.

In a paper published last week, NIH Director Collins laid out the possibilities that his proposed center could yield to reengineer the process of translational research . Among the specifics, he noted that the proposed NCATS will:

• support broadly applicable rather than disease-specific target-validation approaches and the investigation of nontraditional therapeutic targets that are considered too risky for industry investment.


• encourage innovations in chemistry for drug delivery, such as nanoparticles; imaging agents for use as biomarkers; and detection technologies for use in diagnostics.


• aim to develop more reliable efficacy models that are based on access to biobanks of human tissues, use of human embryonic stem cell and induced pluripotent stem cell models of disease, and improved validation of assays.


• serve as an honest broker for matchmaking between compounds that have been abandoned by industry before approval and new applications for which these compounds might show efficacy.


• support innovative designs for testing combination therapies, as optimal treatment of many diseases is likely to require multiple therapeutic agents.

All of these are essential to all the sectors, and the NIH has the ability to share the insights and system improvements coming out of this work across every entity in the medical research system. But the NIH can’t go it alone. The entire medical research community must come together to ensure this proposed center does not become part of the road paved with good intentions. And we urge our Congressional leaders to support the establishment of this important effort.

Patients need these improvements in translational research, and that means you and me.



Relevant Links:1) Science Translational Medicine Commentary: Reengineering Translational Science: The Time Is Right
2) Podcast (MP3 - Dr. Collins summarizes the goals and functions of the proposed National Center for Advancing Translational Sciences)

Building Bridges across the Valley of Death

Gillian Parrish, Manager of Alliance Development and Communications, FasterCures
I spent the early part of last week at the BIO2011 Convention, where the message on medical research seemed to be that there is cause to be optimistic about the future of drug development, but that in order for patients to see the payout of groundbreaking science, truly collaborative, outcomes-oriented partnerships – and a willingness to take risks – are key. On Wednesday afternoon, a panel moderated by our own Margaret Anderson explored strategies for crossing the valley of death – the gap between a promising discovery in the lab and the point at which a company is willing to pick it up and moved its development forward – to fund innovative new medicines.

“What’s missing today is a sense of urgency,” said Dr. Ellen Feigal, Vice President for R&D at the California Institute for Regenerative Medicine. “Research cannot just be about fostering knowledge and understanding anymore. It has to be about translating that knowledge into products, and then working with industry to move those products into the clinic.”

Neil Warma, President and CEO of Opexa Therapeutics, emphasized the need to communicate around the long-term horizons of science – describing to funders and partners in clear terms the impact of research on patient’s lives five or more years down the road, and then demonstrating discernable progress along the way to keep enthusiasm and energy up. “The education process is long,” he said, “so it’s important to bring an entrepreneurial mindset to everything you do.”

“If you don’t have any failures, then you aren’t taking enough risk,” noted Dr. Bob Beall, president and CEO of the Cystic Fibrosis Foundation. He talked about how CFF’s successes – more than 30 drugs in the development pipeline, four of them already FDA-approved – have been a result of holding its partners’ feet to the fire, and keeping patients at the table every step of the way to create the urgency Feigal talked about.

But it goes beyond just having a seat at the table. While patient presence is critical to humanizing research and speeding progress, patient voices can’t just be loud, they also have to be smart on the science and knowledgeable about the regulatory environment. The recently released paper Back to Basics: HIV/AIDS Advocacy as a Model for Catalyzing Change, co-authored by FasterCures and HCM Strategists, distills lessons learned from the HIV/AIDS movement that can be replicated to address today’s medical research advocacy challenges. HIV/AIDS activists were successful because they refused to accept that the system was unchangeable, and instead took the time to figure out what it should be and how it should work.

“You have to keep going in the face of failure,” said Charles Queenan, Senior Vice President of New Ventures at the Juvenile Diabetes Research Foundation. “Failure is inevitable, but progress takes time.”

By taking a step-wise, incremental approach to risk, managing expectations from the get-go, and embracing the fact that research is not unidirectional, progress is not only possible, but fully attainable.

As John Mendlein of aTyr Pharma and Fate Therapeutics has proven time and again – you have to make a big bet by finding technologies that can shake up medicine, adding capable people and adequate venture financing, and creating a team-oriented culture to get promising science across the finish line and into the hands of patients.