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.

Medical research investment: an opportunity for bipartisanship?

By Cecilia Arradaza, Director, Communications and Policy, FasterCures

May 29, 2013

Photo courtesy of the NIH

A few weeks ago, House Majority Leader Eric Cantor led a bipartisan group of members to the National Institutes of Health campus to learn more about their work, but also to discuss the future.  Reps. Earl Blumenauer (D-OR), Michael Burgess (R-TX), Renee Ellmers (R-NC), Eliot Engel (D-NY), Chaka Fattah (D-PA), Andy Harris (R-MD), Tim Murphy (R-PA), and Ted Yoho (R-FL) joined the Majority Leader.  Cheryl Jaeger, Megan Whittemore, and Matthew Zackon of Leader Cantor’s office and Karen Summar of Rep. Cathy McMorris Rodgers’s office also participated.

Photo courtesy of the NIH

Seeing is believing – walking through the NIH Clinical Center, the crown jewel of our nation’s research framework, provides you not only with hope for the future but a real sense of optimism that science matters and that it can make a difference in our lifetime. So to see our Members of Congress carve out time to visit the NIH provides the medical research advocacy community with great hope we can reaffirm our commitment to the biosciences.

At the Milken Institute Global Conference, on a panel with Senate Majority Leader Harry Reid and FasterCures founder Michael Milken, Leader Cantor said that “[we] believe that science should be a priority.” He added that he “hopes when [the Congress] gets to the point where there are issues that seem insurmountable, let’s go ahead and try and practice doing things together so that we can get some things done.”

He then cited the creation of the National Center for Advancing Translational Sciences (NCATS), as one of the demonstrable results of him working closely with Leader Reid. “Harry, when you and I did NCATS, it was a great example I think of the ways that we can work together without a lot of fanfare and just practicing and putting points on the board, and maybe we can work our way towards larger goals after that.”

Cantor then alluded to the May 9^th bipartisan trip to the NIH that he was organizing. “Not only will we spend a couple hours together one afternoon in Washington, but also perhaps learn something together we can practice trying to solve problems together,” he said.

During the panel discussion, Leader Reid concurred with Cantor’s statement on the critical importance of supporting science and noted that “if we’re going to make progress in some of the dreaded diseases we have, we have to step up as a federal government to do that. It’s going to take us working together to fund these programs so that our great scientists can continue doing what they know needs to be done. And we can’t do it on the cheap.”

Investing in our nation’s medical research infrastructure is an investment in our future.

From a dollars-and-cents vantage point, consider the economic payoff of the Human Genome Project: the U.S. government’s $4 billion investment in the Human Genome Project spurred an estimated $796 billion in economic growth from 2000-2010—a 141-fold return on investment, after adjusting for inflation.

And, we are living longer, more productive lives, due in large part to research advances that started at the NIH. For example, U.S. death rates from heart disease and stroke have fallen more than 60% in the last half-century.

The case for investing in science and medical research is clear. To punctuate this point, let’s turn to the cost of inaction. Well –

• talk to the brilliant PhD with bleak prospects for a career in science…
• talk to the employer who has lost his or her most productive and talented staffer because that person had to instead become a full-time caregiver…
• talk to the patient who just walked out of the doctor’s office after having been diagnosed with a debilitating disease for which there are no cures or meaningful treatment options…

Point punctuated.

Now to further underscore, consider the global environment we’re in. At a time when other countries are doubling down on their biomedical research spending, the U.S. is cutting our investment (figure 1) in the industry that promises more jobs, greater productivity, and lives saved.

With all these compelling arguments to back us up, let’s remind our elected officials of what’s at stake, and get them to act on making medical research a national priority.

Figure 1

Related links:

• Photos from Leader Cantor’s personal Flickr of his trip to the NIH
• Global Conference 2013 panel discussion with Leader Reid, Leader Cantor, and Michael Milken
• The impact of NIH research

Science takes center stage at world's top finance conference

Make sure to bookmark FasterCures’ new tumblr for more 2013 Global Conference posts and to keep on the pulse of the latest innovations in the medical research ecosystem.

Imagine the possibilities if we could only read your mind…or at least map your brain

"Imagine if no family had to feel helpless watching a loved one disappear behind the mask of Parkinson’s or struggle in the grip of epilepsy.  Imagine if we could reverse traumatic brain injury or PTSD for our veterans who are coming home…,” said President Barack Obama last week as he laid a grand, ambitious new government initiative to map the brain.

Obama said he will ask Congress for $100 million in 2014 to support the BRAIN (Brain Research through Advancing Innovative Neurotechnologies) initiative which allows us to "better understand how we think and how we learn and how we remember." Three government agencies will be involved: the National Institutes of Health, the Defense Advanced Research Projects Agency and the National Science Foundation.

“It's an audacious, bold idea,” said Francis  Collins, director of the NIH. "To understand how the human brain works is about the most audacious scientific project you can imagine," he said. "It's the most complicated structure in the known universe."

In many ways, this initiative is similar to the Human Genome Project which Collins led and completed in 2003, in its vision and ambition. But, unlike the Human Genome Project, the BRAIN initiative has not laid out its primary scientific goals. The lack of detail is worrying BRAIN skeptics and advocates alike. Some scientists were quick to question the motives behind this initiative, noting that a lot of great research on the brain is already underway.”

At a Q&A segment  following the President’s announcement, Collins said that these details would be hammered out by a “dream team” of 15 scientists who will hold their first meeting at the end of the month. This team is led by Cori Bargmann of Rockefeller University and William Newsome of Stanford University, will be charged with coming up with a plan, a time frame, specific goals and cost estimates for future budgets.

Additionally, Obama addressed how this initiative can drive growth and create new job opportunities. As the “rest of the world” is racing ahead in the quest for innovation, he expressed concern that we will lose a new generation of scientists because of uncertainty in research funding. We can’t afford to miss these opportunities, he said.

The President called on companies, research universities, foundations, and philanthropists to get involved.

What we took from sitting in the room with the leaders and innovators in science and research as the president spoke about the promise and potential of this new initiative, was a broader message about the power and value of investing in science, and the important role the federal government plays in ensuring scientific breakthroughs can improve health.  We took such enthusiasm and energy as our cue to be more determined than ever to advance medical progress.

Upcoming Milestones

• As part of this planning process, input will be sought broadly from the scientific community, patient advocates, and the general public.
• The working group will be asked to produce an interim report by fall 2013 that will contain specific recommendations on high priority  investments for Fiscal Year (FY) 2014.
• The final report will be delivered to the NIH Director in June 2014.

Key Resources:

• Remarks by the President on the BRAIN Initiative and American Innovation, April 2, 2013
• Video of Remarks by NIH Director Collins and the President, April 2, 2013
• Fact Sheet: BRAIN Initiative(Whitehouse.gov), April 2, 2013
• Video of Open for Questions with NIH Director Francis Collins(Whitehouse.gov), April 2, 2013 
• WhiteHouse.Gov Blog: BRAIN Initiative Challenges Researchers to Unlock Mysteries of Human Mind, April 2, 2013