Science, Cures and All the Stuff In Between

by Margaret Anderson

Reblogged from the Huffington Post

The outlook for science has never been brighter -- we know more today than ever before about the molecular basis for thousands of diseases that impact millions of patients -- but the outlook for funding has also never been bleaker. With the National Institutes of Health losing a billion and a half dollars to the sequester, traditional investors retreating from early-stage life sciences, industry pipelines shrinking, and philanthropy unable to fill the gap, we are poised to leave a huge scientific opportunity on the table, namely our ability to get important new medicines out of the lab and to patients more quickly. And with millions of lives hanging in the balance, it's just not something Americans can afford to do.

FasterCures hosted over 100 congressional staffers, patient advocates, academics and drug developers at a Capitol Hill briefing about the importance of federal support for translational research, the phase of medical R&D between the birth of a basic scientific discovery made in the lab and when a pharmaceutical company tests out a potential new therapy with patients -- or, more plainly put, "the stuff in between" microscope and marketplace.

"If there is anything I believe can bring this nation together for a cause, it is finding cures for disease," said House Majority Leader Eric Cantor in his opening remarks. "We ought to be about research regardless of political leaning, because it is an investment that yields tangible returns for our country." In an environment where Congress is laser-focused on coming up with ways to spend dollars more wisely, his advice to advocates was to emphasize the following message to policymakers:

1. It is good to be about cures. Disease touches us all, and as compassionate humans we should all care about improving and saving lives.
2. It is good for the economy to invest in research. This leads to job creation and global competitiveness.
3. It is a good way to bend the cost curve of health care. We have mounting deficits and debts, and we should prioritize the allocation of funds to areas like this that have the greatest impact.

"This is really an engineering problem," said NIH Director Francis Collins. "It's about taking a pipeline that is too long and too leaky and fixing it." With each new therapy that comes to market taking up to 15 years to develop and costing over $1 billion, it's more challenging than one might think to successfully navigate the translational stage. There is great momentum in science, and we are poised to start solving several of these problems, particularly with the existence of the new National Center for Advancing Translational Science (NCATS) at NIH, but if we don't continue to invest, we risk losing not only time and lives but a significant driver of economic health.

"NCATS is a unique new entity, purpose-built to solve this central paradox: that we know more than ever before about how the body works and what goes wrong in disease but lack the equivalent ability to fix it," said NCATS director Chris Austin. "We're designed to be perpendicular to how the rest of NIH works, and to focus not on what's different about disease but what is common." He went on to describe the institute as a biomedical equivalent to the Internet, focusing not on creating companies or products but on allowing people with ideas to share them in a more productive way, and de-risking investment by other sectors in this translational space.

Both Collins and Austin said that there is no better time to pursue translational research. Today, we know the molecular causes behind 4,500 diseases, yet there are drugs available to treat just 250 of them.

"We are operating on hope, not hype," said Sharon Terry of the Genetic Alliance. "The network solutions that have overtaken other industries and streamlined them are finally starting to hit the biomedical research space, and we can't afford to lose momentum." She specifically referenced networked programs such as the Clinical and Translational Science Awards at NIH that are significantly advancing systems improvement in the translational phase and are primed, with the proper support, to foster more integrated research across communities. A new report released this week by the Institute of Medicine goes into more detail.

"Industry can't do it alone. ... Companies depend on basic science coming from the NIH," Simeon Taylor of Bristol-Myers Squibb said. He described how Big Pharma relies on the basic science discoveries made early on in the lab to develop drugs for patients who need them so desperately. Just last week, BMS, along with eight other companies, provided 58 compounds for an NCATS pilot program called Discovering New Therapeutic Uses for Existing Molecules, designed to develop partnerships between pharmaceutical companies and the biomedical research community to advance the development of therapeutics.

I had the privilege of moderating this discussion. The message that rose the top was clear: Getting a therapy from discovery to patients requires partnership and collaboration across all sectors in the medical research ecosystem: scientists, regulators, industry, investors, and, most importantly, the patients themselves. An effective R&D system is an efficient one, but reengineering the system in the way we know is possible requires support, and with funding at critically low levels, the ripple effects could be devastating. If we are to improve our odds for success for our patients and for our economy, there is no time to waste.

Funding for Translational Research at NIH

This month, The National Institutes of Health will be accepting its first round of applications for a new funding opportunity focused on supporting collaborative translational research projects.  First announced last fall, this U01 research program – Opportunities for Collaborative Research at the NIH Clinical Center – is designed to provide extramural (non-NIH) investigators a mechanism to establish collaborations with NIH intramural investigators and to take advantage of the unique research opportunities available at the NIH Clinical Center.

It is being offered by 12 NIH institutions and is intended to facilitate the translation of basic biological discoveries into therapeutic candidates for clinical testing.

With the creation of this funding opportunity and the establishment of NIH’s newest Center, NCATS, NIH continues to push forward its vision to transform the translational process for the benefit of patients. Recognizing the critical advancements that have been made in the understanding of basic disease biology, the agency continues to look for ways to infuse new and innovative thinking into not only the scientific discovery process, but also the process of getting important new medicines from discovery to patient.

We’re excited to see the increased focus on collaboration – for entry to the program, extramural research projects must have a collaborating investigator in the NIH Intramural Program as well as a “Collaboration Plan” – and eagerly await the first crop of awardees.

Fully utilizing the NIH Clinical Center was one of the recommendations that FasterCures had put forward in 2008 through its blue-ribbon task force focused on the NIH Intramural Research Program led by Nobel Laureate and FasterCures board member Dr. David Baltimore. In fact, of the five recommendations the task force presented, three have been acted on, with outcomes that will save lives by shortening the time it takes to turn breakthroughs into medical solutions.

To learn more about the application process for this grant program, watch the pre-application webinar or visit the official announcement page.

Resources:
Sequestration Station – Check out FasterCures’ Sequestration Station for more information on how sequestration will affect the National Institutes of Health.

NCATS a Reality

On Sept. 14, FasterCures’ Executive Director Margaret Anderson joined fellow leaders in medical research for the first meeting of the National Center for Advancing Translational Sciences (NCATS) AdvisoryCouncil and the Cures Acceleration Network (CAN) Review Board. FasterCures has been a strong supporter of the establishment of NCATS and CAN and their goal of saving time and effort in the pursuit and conduct of medical research and development across disease areas.

The NCATS Advisory Council is made up of 18 appointed members, and the CAN Review Board is comprised of 24 appointed members. They will both meet on a regular basis to provide guidance, consult, and makes recommendations to improve the translation of basic science into clinical application.

The meeting began with an overview of NCATS’ mission and its role in translational research at NIH, budget details, recent science advances, and major center milestones. Then NIH Director Francis Collins announced Christopher P. Austin as the first NCATS director. "There is no way any one scientific discipline can accomplish what we want to do; we only can be successful if we do this together," Austin said.

The meeting also included a brief history of CAN’s establishment, its functions, and its activities. “Collaboration, coordination, communication, and policy all will play a crucial role in our work to advance new treatments and cures for patients,” emphasized Freda Lewis-Hall, CAN Review Board chair.

The Division of Clinical Innovation led presentations focused on the Clinical and Translational Science Awards (CTSA) program and participated in a discussion of its goals, achievements, and coordination. Austin delivered the final presentation of the day, which focused on the Division of Pre-Clinical Innovation.

In response to a question from a council member about getting medicines to patients faster, Austin replied: “Through the CTSAs. Combining the power of these two groups, we can do some magical things."

Relevant Resources on NCATS and CAN:

Archive andsummary of ‘A Virtual Town Hall with NCATS Leadership’

Factsheet about NCATS

“A Discussion onInfusing Life into the Valley of Death”

3 Reasons You Can't Miss Partnering for Cures

1. It's all about solutions. This meeting is designed to get things done - from dynamic panel discussions about transformative ideas and successful models, to the dozens of case study presentations about innovative paths toward meeting R&D goals.
2. It's all about collaboration. No other meeting of this scale is solely dedicated to forging strategic cross-sector collaborations. You will learn about innovative partnerships that are paving the path toward a more effective and efficient R&D system, and find partners who could make a meaningful difference.
3. It's YOUR meeting. Whether you're looking for an investor or a scientific partner, a customized partnering system allows you to easily connect with potential allies from all sectors of medical research. Experts will be available on-site for one-on-one, free consultation sessions to help address your challenges. It is a unique opportunity to get a pulse on trends and best practices that matter to you and what you do.

Look who's speaking:

Francis Collins
Director, National Institutes of Health

Janet Woodcock
Director, Center for Drug Evaluation and Research, U.S. Food and Drug Administration

William Chin
Executive Dean for Research, Harvard Medical School

Frank L. Douglas
President and CEO, Austen BioInnovation Institute

Regis B. Kelly
Director, The California Institute for Quantitative Biosciences, University of California

Stelios Papadopoulos
Co-Founder and Chairman, Exelixis

Kim J. Popovits
President and CEO, Genomic Health, Inc.

Jay Schnitzer
Director, Defense Sciences Office, Defense Advanced Research Projects Agency

Scott Johnson
President, CEO and Founder, Myelin Repair Foundation

Todd Sherer
CEO, Michael J. Fox Foundation

... and many more

Learn more about Partnering for Cures

Join >800 forward-thinking leaders:

Biotech and Pharma Senior Executives

• Business Development Teams
• Advocacy and Alliance Leaders
• Federal Affairs Directors

Academic Medical Center Leaders

• Research Hospital Executives
• Institute Directors
• Principal Investigators

Life Science Investors

• Venture Capitalists
• Medical Philanthropists
• Wealth Advisors

Medical Research Innovators

• Patient Advocates
• Disease Foundation Executives
• Venture Philanthropists

Government Decision Makers

• Federal Funders
• Federal Researchers and Regulators
• Research Policy Makers

... and many more

‘Translation is a Team Sport’

The Learning Collaborative, a partnership between the Leukemia & Lymphoma Society (LLS), University of Kansas Cancer Center, and the National Center for Advancing Translational Sciences (NCATS) at NIH, is attempting to break the traditional drug discovery and development paradigm by bringing together blood cancer, drug discovery, and development expertise across several organizations. The Collaborative was formed with the goal of targeting repurposed drugs as well as novel, new drugs for the treatment of rare blood cancers.

Last week at a TRAIN (The Research Acceleration and Innovation Network) Webinar, the leaders of this unique effort shared what they’ve learned since they signed the Cooperative Research and Development Agreement (CRADA) in 2010. CRADA defined the resources and expertise each collaborator brought to the effort.

The three partner organizations bring complementary strengths to this collaborative. LLS has ample experience working with industry partners, an established network of experts, about 400 active research projects to contribute, and the sense of urgency that patient-driven foundations uniquely bring to the table. The University of Kansas Cancer Center excels in “bench to bedside” translation in drug repurposing, leadership in chemistry, and pharmaceutical experience. NIH’s NCATS brings a focus on rare diseases, industrial scale capabilities, and pharmaceutical experience.

“While some progress has been made in combating these cancers – half of the FDA-approved drugs of the last decade were approved for use in blood cancers – 50 percent of those newly diagnosed will die from their disease within five years,” said Louis DeGennaro, PhD, chief mission officer of the Leukemia & Lymphoma Society.

Scott J. Weir, PharmD, PhD, director, Institute for Advancing Medical Innovation, University of Kansas Cancer Center, gave a snapshot of the first Learning Collaborative effort, the auranofin project, and shared key insights:

• Keeping the patient in mind will accelerate the process and ensure their needs are front and center in the research agenda.
• Defining the collaboration, setting collective objectives, and managing expectations are fundamental.
• Project management is critical, both to manage across organizations and to maximize appeal to industry.
• Tech transfer must be integrated into teams to optimize the leverage of the data.
• Regulatory science issues that will impact their chances of success need to be addressed in the course of the research.
• Defining exclusivity paths and reimbursement strategies from the earliest stages is central to the effort. The group is working on what they call a “ValueMaP” (Value Maximization Path) to help define the value proposition for drug repurposing.

Currently, there are four active projects, two of them involving repurposing rheumatoid arthritis drugs for use in rare blood cancers (the other two are in earlier stages). The goal is to advance projects to clinical proof of concept within 14 months and to engage industry partners along the way.

This model, said Christopher P. Austin, MD, director, Division of Preclinical Innovation, National Center for Advancing Translational Sciences, can be applied to other organizations, and other diseases. “Translation is a team sport. It needs to be approached that way, and very consciously and deliberately managed that way.”

Documents critical to the Collaborative formation will be made available on FasterCures’ TRAIN Central Station soon.

Resources for Medical Research – We’re Gonna Need a Bigger Boat

By Margaret Anderson, Executive Director, FasterCures

It’s appropriations season in Washington, DC, and we are having an early spring. Why does that make me think of Jaws (the movie)? Because I love the beach and sometimes have nagging anxiety about what swims beneath me when I swim in the ocean? Perhaps. (And yes, I know that sharks attack rarely and they are minding their own business.) More so because I think we have allowed ourselves to forget what the real issue is out there lurking in the water. Resources and the future.

Yesterday, the House Appropriations subcommittee on labor, health and human services, and education chaired by Rep. Denny Rehberg (R-Mont.) convened a distinguished panel that focused on investments at the National Institutes of Health (NIH) and the newly created National Center for Advancing Translational Sciences (NCATS). A lot of the discussion centered on NCATS’ value proposition. And some discussion was about the pros and cons of the NCATS approach. (Let’s remember that NCATS is exactly 2 and a half months old now.)

NIH Director Francis Collins reiterated that NIH support for basic research remains consistent and strong at about 54 percent of the agency’s budget. He said he does not expect that percentage to change. He also emphasized that 98 percent the $575 million funding for NCATS comes from preexisting NIH programs. “We believe we could do a lot with modest resources at this point simply by putting the focus on bottlenecks in the drug development pipeline,” said Collins. Threading the needle? That sounds pretty rational.

Acting NCATS Director Thomas Insel has emphasized that NCATS was created to “complement—not compete with—the private sector.” NCATS pools together existing NIH resources and capacity in translational research to foster greater efficiency. It does not develop drugs; instead, it streamlines and improves processes to increase the odds of getting to therapies faster.

Todd Sherer, CEO of Michael J. Fox Foundation for Parkinson’s Research (MJFF), also providing testimony, noted that in pursuit of a Parkinson's cure, MJFF has funded more than $285 million in research since inception, of which 90 percent has gone straight to translational research. "Based on my experience of what can happen when substantive investments are made in translation, the total contributions NCATS can make to drug development may well be greater than the sum of the parts," he said.

At FasterCures, we support NIH’s efforts to create NCATS. Solutions can be tough to develop, and tougher to implement. Groups like MJFF and other outcomes-driven medical research foundations that are part of our TRAIN network, have demonstrated that innovative approaches to disease research are critical to speeding the R&D process. We must try new, promising avenues. NCATS is one tangible way to get moving. So what about the bigger picture?

We should not forget the larger menace on the horizon, the one that should cause the famous John Williams tune from Jaws to be playing in your head now. And that is the need to continue to keep our eye on the overall NIH budget, and the implications of flat or negative funding for NIH, as well as the threat of sequestration in January 2013. 

NCATS, new and important as it is, only accounts for a very thin slice of the NIH budget. Our national investment at the 27 institutes and centers at the NIH have yielded great scientific discoveries that have and will continue to improve health.

NIH also plays an essential role in sustaining our economy. A report released yesterday by United for Medical Research found that in 2011, the NIH directly and indirectly supported 432,094 jobs around the country. While significant, this is actually 55,000 fewer jobs than in 2010 – which, according to the report, “demonstrates that the lack of sustained investment in the agency is beginning to have an impact.”

The ripple effects of a constrained NIH budget go far and wide. Should sequestration (mandatory budget cuts should Congress not reach a budget agreement) happen, the NIH will lose 7.8 percent of its budget – $2.5 billion. Collins said that this means that “2,300 grants we had planned to give in fiscal year 2013 would not be able to be awarded. It would be devastating.”

Do we need to keep innovating our research system? Of course, but we also need resources. Resources feed that important basic science part of the continuum. Resources keep our young investigators in the field.

As the appropriations season unfolds, we urge all members of Congress and the medical research community to keep our eyes on the prize: a strong NIH will not only protect and sustain nearly half a million high quality jobs. This agency is also our biggest chance of having more “shots on goal” in the search for treatments and cures.

This is about resources. This is also about patients – patients today and tomorrow. This is also about the future. Let’s get our priorities straight. There are big fish – maybe even some really big ones – out there we need to be focused on, and as Roy Scheider who played Amity Police Chief Brody told us in Jaws, “We’re gonna need a bigger boat.”