Needing teamwork for translation

Science is like football, said National Center for Advancing Translational Sciences (NCATS) Director Chris Austin during the Partnering for Cures panel, “Molecule to Man (and Woman): Speeding Translation to Human Trials.” Translational science needs true teamwork, everyone in the ecosystem coming together – researchers, pharmaceutical companies, venture capital firms, patients groups, and government – in order for new treatments to be brought to trial faster. But, “the incentive has always been for individual achievement,” Austin said. Instead of playing football, the scientific community is playing golf. And many hope the creation of NCATS will help change that.

“NCATS is a different beast,” Austin said. “It was set up as a collaborative instrument. It’s founding principle is that we have to do these very, very important things that no one else can do.” 

Anna Barker of the Arizona State University School of Life Sciences hopes that mission includes creating systems to speed up translation. “We haven’t thought about creating an end-to-end system that starts with the patient and ends with the patient,” she said. And a key to this system is requiring high quality at every level, for which she feels the scientific community needs standards. “We have not thought about how to deliver molecular findings to patients in a high-quality, controlled way,” she said. 

Todd Sherer of the Michael J. Fox Foundation advised caution. “The challenge is finding the balance between overstandardization vs. innovation,” he said.

Some steps that could speed translation involve solving problems that have no return on investment, so it is difficult for researchers or big companies to justify using the resources. “How we use our resources has to be ever more strategic,” said Dale Edgar of Lilly Research Laboratories, who said his company is pursuing success by identifying “pillars of excellence,” such as maintaining quality standards of practice for the data it stores.

Research decisions can’t be made in a vacuum, said Stelios Papadopoulos of Exelixis, especially not in an investment vacuum. Decisions still must be made to get the maximum return on investment. “I have not seen a compelling investment proposition to go in and engage in [translational research],” he said.

NCATS, which is free from needing to find a cure for a particular disease or make a particular return on investment, can help, said Austin. He feels this gives the agency an obligation to work on the general issues that will help advance translation.

The panelists agreed that true collaboration is key. Every stakeholder has to go “all in” for the collaboration to work, cautioned panel moderator Margaret Anderson of FasterCures. Edgar pointed to the Innovative Medicines Initiative in Europe as a good example of all stakeholders working together to find and overcome critical gaps in driving innovation to the patient. He said the exciting thing about NCATS is “thinking about the patient as the end point and not the next grant.” Innovation, he said, “requires a line of sight to the patient from the earliest point.”

“Something NCATS brings to the table is leadership, galvanizing the industry and all of the players,” said Sherer. He advised Austin to plan for some early accomplishments in the next 18-24 months to prove to naysayers that the agency can affect change.

Edgar encouraged NCATS to continue to foster partnerships, because “great things will happen.”

“The way you make these things work increasingly is to be inclusive,” said Barker. “Get the best input you can up front from as many in the community as you can. Get as much buy-in as you can.”

And Austin is doing just that at NCATS. He reported that he is in talks now with stakeholders in “concentric circles” in the process, including institute directors, academic institutions, patient groups, pharma, biotech, and VCs. As the agency develops, his goal is to keep the patient at the center of everything. “Having patient involvement helps you stay on the critical path,” he said.

Anderson concluded with emphasizing the important role that all stakeholders need to play in medical research. “It is unrealistic to think that NCATS can do it alone. We’ve got to do this together.”

Innovations in Financing Early-Stage Medical Research

Novel models that manage risk and encourage innovation are needed for funding early-stage medical research, according to an expert panel at the 2012 Milken Institute GlobalConference. The discussion focused on a variety of different financing approaches, including innovative partnerships with biopharmaceutical companies and research securitization.

Moderator Melissa Stevens of FasterCures framed the discussion by describing the gap in translational research funding and asking the panelists why they think there is a lack of capital for early-stage research.

Bernard Munos, founder, InnoThink Center for Research in Biomedical Innovation, described the culture change that began in the biopharmaceutical industry 15 years ago that led to increased risk aversion across many of the major companies. He spoke optimistically of the need for these companies to re-harness their innovation infrastructure: “Innovation is in our DNA… Unfortunately, the DNA does not always express itself the way it should.”

Other panelists mentioned organizational structure as well as increased regulatory hurdles as impediments to innovation and changed risk profiles in the industry.

Chris Varma, president and CEO, Blueprint Medicines, described a recent deal around Warp Drive Bio that resulted in a risk reduction for each partner. Novel approaches like this, he said, are indicative of pharmaceutical companies knowing they need to change and having the flexibility to structure creative deals that meet their needs.

Aya Jakobvits, president and CEO, Kite Pharma, and venture partner, Orbimed Partners, also discussed a new model, the Israeli Life Sciences Fund, which features the Israeli government as the silent partner in a recently closed venture fund that focuses on life sciences investment. She described how the government sees the fund as “a pillar in the ability of Israel to gain more value” and offers additional benefits to partners, including downside protection and upside amplification. The panelists discussed the potential of exporting this model to other countries.

In addition to government-backed ventures, philanthropy-backed ventures are increasingly playing an important role in early-stage drug development, both through providing funding and sending a signaling effect to the market. Kathryn Smith, managing director, Fast Forward LLC, described the importance of Fast Forward, founded by the National MS Society, as “opening doors to potential partners, not necessarily because of the money but because of the society and the network of MS experts.” Varma also highlighted the increasingly important role of philanthropic foundations, saying “I think organizations and companies that don’t talk to foundations are at a competitive disadvantage.”

The final model discussed was the research securitization model, which was motivated from observations made during the financial crisis. Andrew Lo, Harris & Harris Group Professor, MIT Sloan School of Management, explained that by creating vehicles of debt financing we can tap into pools of larger capital that can be used to transform the medical research industry. Such a model would create long-term financing for a large pool of research assets with financial products based on different risk/reward portfolios, allowing for patient capital to be funneled toward a variety of projects that are in need of financing at variable time scales. The other panelists were generally supportive of the concept, but raised questions about how to prevent the degradation of science in the portfolio and ensuring that investors can understand how much risk they are undertaking.

Finally, Stevens drew a contrast between models designed to bring more capital to new ventures with models that are designed to decrease overall capital costs, such as open source research and development, product development partnerships, and data sharing.

All of the panelists agreed that a variety of new financing mechanisms are important for early-stage drug development to meet the unmet needs of patients.

Business Is from Mars, Academia Is from Venus

Universities are showing increasing interest in stretching their work into the "Valley of Death" to move their discoveries towards treatments. Companies are rethinking their alliances with universities as sources of innovation to fill their pipelines, beyond traditional sponsored research agreements. At the Partnering for Cures meeting in November, experts discussed where such rethinking is taking place, the replicability and scalability of these models, and the challenges of this new type of relationship.

Moderator Ross DeVol, Milken Institute, noted that innovation is increasingly shifting to universities and, with that, new thinking and practices are developing to ensure that these efforts advance outcomes without distorting academia's core mission.

Annette Kleiser, University of Southern California Stevens Institute for Innovation explained that "making innovations, or I should say inventions, available to the public has become a critical part of what we do."

Pfizer's Tony Coyle pointed out that the wealth of academic understanding and expertise cannot be kept siloed in the usual few organizations. The divide between basic science and clinical practice is where we should be focusing, he pointed out, with the patient's needs always at the center. Coyle suggested industry look to its colleagues in academia to help reflect on what the true unmet medical needs are, and what opportunities exist to design therapeutics that are fundamentally different from the existing standard of care. "We don't need to be so insular," he said. "Success in medicine or chemistry alone will not allow us to develop new therapeutics that are truly meaningful and will have an impact on patients."

Stephen Friend, Sage Bionetworks, who professed himself a "galactic traveler" - having spent a third of his career in Venus/academia, another in Mars/industry and the last "on Earth with the patient" - talked about the need for new models to support all of the data and information that is starting to flow. The linear drug development pipeline is no longer, he said, and industry is "quite awake" to the fact that the old models aren't going to work.

Kleiser described why academia needs private industry: "We need the insight from the industry on how to turn these discoveries into a product. We need the exchange of data, and we need this exchange of expertise." She went on to give an example of a partnership USC has entered into with Johnson & Johnson and how the flexible nature of their IP agreement has opened up new pathways for innovation.

Fernando Kreutz, FK-Biotec, discussed his perspective on Brazil as a blank slate for medical research. "Innovation requires industry," he said. "In Brazil most of the science is actually done inside of universities. How can we channel all of this knowledge into innovation? That's the biggest challenge in Brazil right now."

Todd Sherer, The Michael J. Fox Foundation for Parkinson's Research, talked about what patients could do to help improve outcomes and ensure that development partnerships remain focused on meeting the needs of people with disease. "We do see the model changing in terms of understanding that one entity cannot solve all these diseases." While there is an openness for these collaborations, there are also competing goals and reward structures between industry and academia. Patient organizations can provide a neutral "playing field," allowing partners to come together in a safe space without the complications of either side having a home-field advantage.

Friend then talked about the power of data, and, in particular, the power of patient ownership of data when it comes to sharing and generating information for research. He highlighted several of Sage's projects including The Clinical Trial Comparator Arm Project, a precompetitive disease biology initiative, which is working to push comparator arm data of clinical trials in a variety of therapeutic areas into the public domain .

Recently, Pfizer and the University of California San Francisco began a revolutionary partnership which Coyle discussed. Aimed at more rapidly moving new therapies into human clinical trials, teams from the University and Pfizer work together on experimental therapies developed by the UCSF scientists with a goal of testing them in people with five hard-to-treat, often deadly conditions, including lung and prostate cancer."This is not collaboration from afar, but an example of working together with people that are striving for the same thing. Physically being located in the same lab starts trust, and builds this new ecosystem relationship. Academia cannot do it on its own, and the same goes for pharmaceutical companies."

In closing the panelists discussed the one thing they would change in the current environment. Kruetz said, "The one thing I would change is the lack of urgency in academia. It is shocking." Friend made the point that the scientist, the academic institution and industry work pretty well together. It is the patients that are missing. "Until earth joins Mars and Venus, it's not going to work."

What is NIH's Role in Bridging the Valley of Death?

The spending bill referenced in our post yesterday will, if signed into law, establish the National Center for Advancing Translational Sciences (NCATS) at the National Institutes of Health. Good news for our medical research and development enterprise, and for the millions of patients who need treatments and cures, the inclusion of funding for NCATS in the omnibus will help to speed the translation of basic discoveries into real world applications that will improve public health.

Last month at our Partnering for Cures conference, a distinguished group of panelists from academic, advocacy, and government institutions examined the challenges and opportunities to bridging the "valley of death" between preclinical development and clinical research, and specifically explored the impact and appropriateness of NCATS’ role in this effort. Moderator

Anna Barker of Arizona State University (formerly of the National Cancer Institute), challenged the panel to consider the rapid advancement of accessible information - who is the appropriate convener to push forward translational research?

Kathy Hudson of NIH explained the nuts and bolts of NCATS: instead of focusing on individual projects, NCATS will identify bottlenecks in the pipeline that slow the research process for all players. Hudson acknowledged that it is an "awkward time" given the difficult economic climate, but emphasized that the goal of this new center is to collaborate to address shared problems - not to duplicate what is already happening in the pharma/biotech industry.
University of Pennsylvania professor Garret FitzGerald agreed that NCATS will be helpful to "shine a light on problems," and pointed to the erosion of human capital in translational research. NCATS could play a role in rebirthing the discipline through the training of heterogeneous skills, he noted, which could be especially important in the era of comparative effectiveness research. FitzGerald argued that this type of focus would be politically uncontroversial and relatively inexpensive.

Academic scientist and entrepreneur Peter Lansbury said that government had a huge role to play, particularly in neurodegenerative disease. He noted that there a special need for tools that could help facilitate clinical trials for these types of diseases, where progression is erratic and often slow.
Louis DeGennaro of the Leukemia & Lymphoma Society - which earlier this year signed an cooperative R&D agreement with the NIH Therapeutics for Rare and Neglected Diseases (TRND) program and the University of Kansas Medical Center to accelerate the development of potential clinical therapies for rare blood cancers - mentioned that an often overlooked topic during discussions of solution development is the role advocacy and disease advocacy groups play. The "fiduciary responsibility" he and his colleagues feel for patients served by LLS has driven them to develop creative partnerships to engage in translational research.

Chris Varma, Entrepreneur-in-Residence at Third Rock Ventures, noted that although the amount of life science venture capital funding continues to increase, a smaller quantity of individual investments are being made overall. Given this environment, NCATS "makes a lot of sense" in driving efficacy and reducing risk, especially when it comes to identifying failures early on in the development process, he pointed out.

The panelists suggested a few priority areas for NIH's engagement in this area including

• Get the funding and support for NCATS to successfully get up and running
• Develop human capital - increasing the expertise, capabilities, and attractiveness of the research discipline
• Keep focus on patients when bridging the gap from academia to clinical trials
• Take an analytical approach to the data available for new research

Fixes in Financing Needed to Bridge the Valley of Death

Turning medical science into medical solutions requires innovators to leap all kinds of hurdles, but one of the biggest is the financial “Valley of Death,” where many underfunded, early-stage ventures meet their demise.

To help bridge this funding gap, dozens of investors and innovators convened for a Financial Innovations Lab to explore financial tools and other ground-breaking approaches that are being used to advance initiatives in medicine and other industries. Organized by FasterCures; and the Milken Institute, the Lab was designed to explore whether these financial tools could be applied to translational medical research.

The Lab spotlighted models for partnering in research and development, and identified finance instruments and incentives, some of which are described below.

• Distributed Partnering Model utilizes product definition companies (PDCs) that identify and license promising early-stage assets from research institutes, manage product definition research, and sell de-risked assets to later-stage stakeholders. The PDCs make $3 million to $5 million investments aimed at attracting additional funding.


• BioPontis Alliance is a hybrid investment fund and product development company that aggregates technologies from a consortium of universities, screens these assets, develops them through a translational development network, and then licenses them to strategic pharmaceutical partners. Their activities are supported by a $50 million fund. The model encourages sourcing of early-stage assets from academia by pooling and sharing the value of intellectual property, and helps ensure later-stage funding by establishing relationships with pharmaceutical companies. This proposition is made more attractive to universities via a master IP licensing agreement where the process is expedited and universities receive a pro-rata share of the total value created by the assets.


• Fast Forward, a wholly-owned subsidiary of the National Multiple Sclerosis Society, is a venture philanthropy model that provides leveraged, philanthropic funding to translate academic research and further develop biotech research into new treatments. The technologies are identified and evaluated by scientific and business advisors. Fast Forward makes $250,000 to $1 million investments.


• Israeli Life Sciences Fund uses a standard venture capital-structured fund with the government and private sector as limited partners. The Israeli government, which takes the first loss before the other limited partners, has committed $80 million to boost returns for private investors.


• Flow-through shares, which originated in the Canadian resource industry, encourage higher-risk investments through tax incentives. Oil and mineral exploration companies issue these shares as a way to pass government tax deductions for exploration on to investors, effectively halving the risk of investment.

There was broad agreement that the medical research system has much to learn from financial models that have worked in other industries such as filmmaking, telecommunications, and oil and gas. But participants cautioned against simplistic analogies, noting the unique circumstances that define the medical research process. Science is unpredictable, and failure to fully vet a product has consequences that are measured in terms of life or death.

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.

TRAIN Group at Center of New NIH Collaborative Effort

Kristin Schneeman, Program Director, FasterCures

FasterCures aims to expedite cures by stimulating innovative collaborations across all sectors. We recognize that a collective approach is needed to successfully address the barriers to innovation. That is why we were fascinated to note this week the announcement that NIH has just signed a cooperative research and development agreement (CRADA) to move promising therapies for rare blood cancers into clinical proof-of-concept studies. This is an excellent example of collaboration that we’d like to see more of in the future.

While NIH has engaged in CRADAs for many years to make government facilities, intellectual property, and expertise available to further the development of marketable products, this one caught our attention because of the involvement of the Leukemia & Lymphoma Society, a major funder of blood cancer research. The press release announcing the agreement explicitly noted the role that philanthropic and patient organizations have in the drug discovery process. Also engaged in the collaboration is the University of Kansas Medical Center.

The agreement is notable to us also because NIH is viewing it as a “Learning Collaborative,” the goal of which is “to bridge the gap in time and resources that often exists between basic research and human testing of potential new treatments.” “We plan to learn more about and improve the drug development process for all diseases,” says Chris Austin, director of NIH’s Therapeutics for Rare and Neglected Diseases program, which is spearheading this effort.

FasterCures, through our TRAIN program, has long been a proponent of the important role that patient-driven philanthropic organizations can play in funding innovative approaches to medical research and in helping move promising discoveries across the translational “valley of death” toward new treatments. They bring not just risk capital but also the voice and priorities of patients into the research process.

This agreement is part of a heartening trend (no pun intended) at NIH to play a more active role in bridging the gap between the many promising discoveries it funds and the point at which biotechnology or pharmaceutical companies are willing to invest in expensive late-stage clinical research. We are pleased to see that Leukemia & Lymphoma Society has a seat at the table in this effort, and we hope to see more collaborations of this kind in the near future. We look forward to learning along with The Learning Collaborative about how government, academia, nonprofits, and ultimately industry can work more efficiently and effectively to get badly needed new treatments to patients.

Purpose and Partners: Yielding Return on Medical Research Investments

by Kristin Schneeman, Program Director, FasterCures

A Global Conference panel of experienced funders, innovators, and analysts described first-hand the growing “Valley of Death” in funding the early stages of research and development of new treatments, explained the novel approaches they are taking to bridge the gap, and called for a complete rethinking of the financial models for funding medical research.

Ali Andalibi of the Small Business Innovation and Research grant program at the National Cancer Institute, Stephen Seiler, CEO of biotech company AesRx (which has benefited from a novel translational research funding program at NIH), and Lt. Col. Daniel Wattendorf of the Defense Advanced Research Projects Agency (DARPA) all represented efforts by the federal government to move beyond its historical role of funding basic discovery, to help push discoveries down the field toward becoming products in the marketplace. Catherine Arnold of Credit Suisse provided the viewpoint of the investment community, which is increasingly risk-averse and unwilling to invest in life sciences companies before the later stages of research.

DARPA is often cited as a role model for how government should fund research. Wattendorf ascribed this to DARPA’s “goal-orientation” rather than interest in contributing to scientific knowledge, its flat management structure and the complete authority of its program directors to control funding. Other panelists commented on the need for NIH to “take some DNA from DARPA.” DARPA is also teaming with FDA to look at regulatory science and whether there are ways to fundamentally change the development pathway that will accelerate progress.

Chris Elias of PATH, a public-private partnership to develop global health technologies, talked about what the global health field is learning about the Valley of Death: philanthropy and government capital can leverage company interest, particularly if it advances their platforms; and there is a significant role for nonprofits to invest in enabling platforms and to create standards to remove stumbling blocks for all parties.

Seiler pronounced the traditional biotechnology development model dead, noting that by 2009-2010 almost no preclinical research was being funded by the public markets. “We aren’t going to go back to the good old days, so what does the new model look like?” He also noted that what’s needed now is not just more sources of funding but “other management modalities – not just is this good science, but does the management know how to get there? This hasn’t been required before, we need to build those skills.”

Watch a video of the session.

Crossing Over the Valley of Death

by Angelo Bouselli, Senior Communications Manager

Stem cells. Genes linked to Alzheimer’s, autism, diabetes. Cancer drugs tailored to treat an individual tumor. Every day we see stories in the media about the latest medical “breakthroughs” that could lead to cures for dreaded diseases. And yet, over the years, many breakthroughs like these have yet to bear fruit for patients. Why? FasterCures’ new whitepaper Crossing Over the Valley of Death, examines one significant factor, a gap in funding and support for the critical stage of the research process known as translational research.

Many basic discoveries barely get to start the journey down the therapeutic development pipeline because structural, intellectual, technical, and funding barriers make it difficult to advance any further. They get stuck in an ever-widening gap, known as the "Valley of Death," between a promising scientific discovery and the point at which a company is willing to pick it up and move its development forward.

According to a recent Wall Street Journal article, “the Food and Drug Administration (FDA) approved about 21 drugs in 2010, a relatively modest figure that shows the pharmaceutical industry hasn't yet escaped its drought in recent years.” Monthly drug-approval reports on the FDA's website show the number of new drugs approved in 2010 is down from 25 in 2009 and 24 in 2008, but slightly higher from a recent low of 18 in 2007.

The National Institutes of Health (NIH) states that 80 to 90 percent of research projects fail before they ever get tested in humans. By industry’s reckoning the number may be even higher— for every 5,000 compounds tested, only five make it to clinical trials, and only one is ever approved by the FDA. Half of all experimental drugs in Phase III trials never become approved medicines.

In Crossing Over the Valley of Death, FasterCures, in collaboration with the Parkinson’s Action Network, reviews the drug development pipeline from the most basic research conducted at academic research centers and supported by the NIH to the large-scale Phase III clinical trials conducted by pharmaceutical companies. It also features case studies from the sickle cell and neurodegenerative disease communities that illustrate the Valley of Death’s impact on cutting edge therapeutic development.

In the great film “Lawrence of Arabia,” T.E. Lawrence had a gift for making overcoming apparently insurmountable barriers seem merely a matter of taking them on. Confronted by a seemingly impassible desert, Sherif Ali says, “There is the railway. And that is the desert. From here until we reach the other side, no water but what we carry with us. For the camels, no water at all. If the camels die, we die. And in twenty days they will start to die.” Replies Lawrence, “There’s no time to waste then, is there?”

To learn more about the importance of translational research in the therapeutic development process, some promising approaches to trying to bridge the gap, and what more needs to be done, read: Crossing Over the Valley of Death.

Medical Research: When Scientific 'Breakthroughs' Get Lost in Translation

Margaret Anderson
Executive Director, FasterCures, The Center for Accelerating Medical Solutions

Every day we see stories in the media about the latest medical "breakthroughs" that could lead to treatments or cures for dreaded diseases. We are overwhelmed with snippets about stem cells, genes linked to Alzheimer's disease, autism and diabetes. We hear that cancer drugs are being tailored to treat an individual tumor. And yet, many breakthroughs like these have not borne fruit for patients.

Whether it's because science is hard and unpredictable, or that resources are limited, or there is lack of prioritization -- too many great scientific ideas sit at the laboratory bench waiting for translation. But, there are successful models that have turned a basic discovery into an effective therapeutic option. These models can provide pathways to a healthier future.

We are at a critical inflection point in current discussions within the biomedical research establishment about what actions need to be taken to push the science toward cures where possible. We need to take advantage of this moment, and we need to bring patients, the public and policymakers into the conversation. Next week from December 13-15 FasterCures is convening in New York City all of the sectors involved in medical research to do just that. Partnering for Cures is a meeting like no other, a place to forge collaborations and participate in outcome-focused dialogue about the challenges facing medical research. We have always maintained that each of the sectors plays a vital role, whether it is government, industry, philanthropy, academia, finance or the non-profits. And the need for their ability to seamlessly pass the baton from one to the other has never been greater or the stakes higher.

Successful models have integrated all of these sectors. Everyone needs to be at this table. Few life-saving therapies have come to market without the resources of private industry. Increasingly, patients have become more sophisticated and disease groups are ever innovating with new models for collaboration with academia and industry. The U.S. government is recognizing that it can play a special and critical role in providing an environment where successful partnerships can grow and proliferate. Partnering for Cures provides an opportunity for all sectors to productively collide, creating an ultimate open source opportunity to shine a light on these models so others can learn and build on them.

One area of keen interest to us is identifying solutions and models to cross the so-called "Valley of Death" -- an ever-widening gap in funding and support for the kind of research that moves basic science down the path toward treatments.

In a new report released today by FasterCures, "Crossing Over the Valley of Death," we highlight the productivity gap that currently exists today, how research moves from molecule to marketplace, how we can traverse the Valley of Death and what all of the sectors are doing towards that end. Many players are marching into that valley, but we are far from reaching the other side.

The need to keep marching with the resources required to make the passage is recognized by advisors to U.S. National Institutes of Health (NIH) Director Dr. Francis Collins, who have recommended that a new translational medicine and therapeutics development center be created by the federal agency post-haste. This proposed center would bring together existing NIH activities in translational research and medicine and allow greater coordination and collaboration internally as well as externally, and ultimately, we hope, produce greater outcomes.

We need to support this recommendation, which if implemented would shine a light on the critically needed but under-resourced and under-appreciated area of translational research. In our jump to embrace this incredible opportunity, however, we need to ensure that Peter is not robbed to pay Paul. NIH's other strengths include supporting the nation's basic biomedical research enterprise, and that focus cannot be lost or diminished in our impatience with the pace of progress. Basic science is still as important as ever, but we also want and deserve concrete outcomes.

Dr. Robert Beall, President and CEO of the Cystic Fibrosis Foundation often talks about the Foundation's own model for de-risking research as providing "more shots on goal." Never has the need been greater to have all of the sectors implement that same approach, to take bold steps to move forward swiftly.

Our nation's wise and prolonged investment in basic science has produced discoveries that now need translation. Knowledge gained from basic discoveries allows us to take more strategic and informed shots on goal. This new and important focus on translation no doubt makes some in the research community uncomfortable, as they worry about focus, changing research priorities and competition over scarce dollars.

We recognize those concerns, but at the same time, the need for accountability and outcomes prevail. Patients need to know that we, the collective "we" in the medical research system, are doing everything we can to get new preventive, diagnostic and treatment options through the pipeline and into the clinic. The alternative to this change is the status quo -- 15 years for an intervention to go from bench to bedside. Clearly that isn't acceptable. Is it?

Join us at Partnering for Cures, December 13-15 in New York City. .

Are we there yet? Navigating the Path through the Valley of Death

by Margaret Anderson, Executive Director, FasterCures

Characterizing the abyss separating basic and clinical research with tangible and intangible landmarks, we held our fifth annual blue-sky brainstorming session in partnership with Esquire magazine. FasterCures hosted a provocative and productive discussion last week on “Crossing the Valley of Death” with a group of thinkers and innovators from across the medical research spectrum. Participants represented large pharmaceutical companies, small biotechnology companies, venture capital funds, universities, and nonprofit foundations that fund research. There was broad agreement on the significant challenges we all face – scientific, financial, and cultural – in moving promising research across the valley and that we are at an inflection point where more action and less talk is required.

The objectives of the session were:

• To hear about new models of R&D collaboration and highlight lessons learned from them, and
• To identify broader applications of existing models and opportunities for new collaboration mechanisms.

10 themes that emerged from the discussion:

1. A fundamental restructuring of the system is what's needed if the biopharmaceutical industry is to survive and thrive and patients are to benefit from innovation. The rest of the world is innovating outside the constraints of the U.S. system.
2. We need to redefine the process to make it cheaper, more sustainable.
3. We need to get beyond buzzwords like “open innovation” and “collaborative research,” and dig in to create standards for intellectual property, precompetitive research, collaborative contracting, or we will never be able to scale up models that exist.
4. We need to find ways to increase the output of valuable intellectual property, not just any intellectual property.
5. While we can’t predict the successful business models of the future, we can create the conditions that will allow for disruptive innovation.
6. We need to change the terms of the conversation – we need to talk more about capital efficiency and not stop at building capital, address the issue of patents but also really focus on productivity.
7. Decision-making in the biotechnology and pharmaceutical industries is not entirely rational or evidence-driven -- how can we change that?
8. There is a crying need for better communication to and understanding by the public and policymakers about the process and roles of the players in medical research. We need to elevate success stories of collaboration as a means to thread the needle more and create a fabric of innovation. It’s time these models transcend the patchwork of case studies and best practices and become the overarching approach that’s sorely missing.
9. We need to have the right incentives in place. Universities need to incentivize their faculty towards commercialization and collaboration with industry. They need to change their internal metrics of success. Federal and state policies need to create incentives for the results we want to see.
10. We need to rationalize the allocation of our resources -- money, time, and human capital.

A variety of actionable suggestions, for FasterCures and other players, also emerged throughout the day. Stay tuned, we’ll summarize those in another blog post.

We will also be producing a meeting report on the topics that were discussed throughout the day. We expect the discussion to inform the program at this year’s Partnering for Cures conference in New York on December 14-15. And the action items will feed into FasterCures’ strategic planning for next year.

Most importantly, this was an opportunity to have a candid, honest discussion of where medical research is and where we need to go, among those steeped in the system but with the foresight and will power necessary to infuse life into the valley of death. Onward.

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Briefing with NIH Director Francis S. Collins: Leveraging Federal Investment to Speed the Development of Promising Therapies for Patients

WHAT: The Cystic Fibrosis Foundation and FasterCures invite you to a briefing that spotlights the nation’s investment in medical research at the National Institutes of Health and examines how these dollars can be leveraged to create new therapies for patients and save lives.

WHO:

• Francis S. Collins, M.D., Ph.D., Director, National Institutes of Health
• Senators Richard J. Durbin and Richard C. Shelby
• Robert J. Beall, Ph.D., President and Chief Executive Officer, Cystic Fibrosis Foundation
• Moderator: Margaret Anderson, Executive Director, FasterCures / The Center for Accelerating Medical Solutions

WHEN: Thursday, May 20, 2010: 10:00 am – 11:00 am

WHERE: Dirksen Senate Office Building, G-11

WHY: “The Cystic Fibrosis Foundation has shown the way, has lit up the path… and what’s been learned from CF can be extrapolated, generalized, to hundreds of other diseases.”- Francis S. Collins, M.D., Ph.D., Director of the National Institutes of Health

The past few decades have brought exciting scientific breakthroughs necessary to understand, diagnose, and treat many diseases. However, the ability to translate exciting advancements into treatments that can help patients severely lags behind the pace of innovation. On average, it takes 15 years to turn a scientific discovery into a viable therapy. For the millions of Americans who live with chronic and fatal diseases, this is simply too long to wait.

Fifty years ago, people with cystic fibrosis did not live long enough to attend grade school, but today, there are more than 30 drugs in a CF drug development pipeline and the median life expectancy for someone with the disease is 37 years.

NIH Director Francis S. Collins, Dr. Robert J. Beall of the Cystic Fibrosis Foundation, and Margaret Anderson of FasterCures will address:

• What lessons can be learned from the cystic fibrosis successes that can map the way for other diseases?
• How can federal investment at the NIH and other agencies be leveraged to answer important scientific questions in a way that accelerates the discovery and development of medical solutions for deadly and debilitating diseases?
• How can we bridge the “Valley of Death” between basic science discoveries and the creation of new therapies for patients?

RSVP to Angelo Bouselli at abouselli@fastercures.org by Monday, May 17, 2010.

Accessing "Smart Money" for Medical Innovations

By Kristin Schneeman, Program Director, FasterCures

A panel of international financial innovators in health and nutrition agreed today that there is an urgent need to bridge a widening funding gap between early-stage research and product development, not just with more money but with “smart money.” In describing their models, they demonstrated that government and nonprofit intermediaries can be ideally positioned to play that role, if they take novel and thoughtful approaches.

Moderator Margaret Anderson, Executive Director of FasterCures and Glenn Yago, Executive Director of Financial Research at the Milken Institute laid out the challenges and the urgency in addressing them. The funding “valley of death” in medical research is real and widening; companies and venture capital are increasingly risk-averse, and information gaps between academia and industry are growing. The slow progress in addressing poverty and disease around the world is inhibiting global growth.

Noting that Germany suffers from a lack of entrepreneurialism, especially in life sciences, Stephan Gutzeit, Executive Director of Stiftung Charité, outlined his organization’s model. The foundation makes grants to academic labs, and then when the work is advanced enough to build a start-up company around, a for-profit venture arm invests in those companies, providing wrap-around support to move ideas along. Gutzeit echoed the words of Scott Johnson, founder of the Myelin Repair Foundation, from an earlier panel, when he said that nonprofits can play a unique role as “systems integrators” to bring together the players in the research and development system.

Ora Dar, Head of the Life Sciences Sector of Israel’s Office of the Chief Scientist briefed the group on a new program, the Life Sciences Fund, the Israeli government has embarked on to support its vibrant start-up community, which the government already supports with direct grants to young companies. Four management teams were selected and are in the process of raising funds. In addition to investing directly in all four funds, the government will provide upside sharing of its net profits and downside protection to other investors.

Craig Courtney, Special Advisor, Innovative Finance for the Global Alliance for Improved Nutrition infused the discussion with his learnings from nutrition. GAIN works to address constraints in the market and draw private capital in to improving global food markets. In all cases GAIN is creating partnerships, leveraging its global scale, and de-risking investment by private sources.

While experiences differ from country to country, and between organizations addressing developed world and developing world needs, many of the funding challenges are strikingly similar, and there is a need for more intermediaries to help bridge gaps and address market constraints. One panelist noted that in many cases the models being discussed weren’t terribly novel from a financial markets perspective. Several panelists appealed for a thoughtful role for government and not a knee-jerk reaction to merely inject more capital into the market.

Infusing Life into the Valley of Death

Medical research leaders and patient advocates yesterday participated in a forum that spotlighted the Cures Acceleration Network (CAN) provision of the Patient Protection and Affordable Care Act signed into law by President Obama on March 23, 2010. Speakers and participants were eager to discuss CAN and its potential to transform the medical research enterprise by supporting efforts specifically designed to bridge a gap in the therapeutic development pipeline between basic and clinical research.

Senator Arlen Specter (D-PA) introduced CAN to bridge this gap, often referred to as the “Valley of Death,” where research lies dormant and ideas come to a halt because the necessary next steps to take basic research discoveries and turn it into a safe and effective therapy are not taken. The forum was convened by Parkinson’s Action Network and FasterCures.

According to Amy Rick, CEO of PAN, “the business of taking a discovery and pursuing it for its full therapeutic potential is dramatically different than the business of basic research.” It includes considerations like FDA, legal issues, intellectual property, and the timing structure and milestones are different, as well. Appropriate incentives need to be in place to fill the valley of death.

CAN, as authorized, will be established within the Office of the Director of National Institutes of Health (NIH) and would award grants through NIH to biotech companies, universities, and patient advocacy groups. John Schall, deputy CEO of PAN, provided details of this provision, including:

• CAN is designed to accelerate the development of high need cures, including through the development of medical products and behavioral therapies. “Medical product” in this context means a drug, device, biological product, or any combination of the three.
• A diverse, 24-person board will be convened to advise the Director of the NIH on the conduct of the activities of the CAN. This board will represent multiple sectors including at least four individuals from the venture capital community and at least eight patient advocates.
• CAN is subject to the appropriation of funds.

John Myers, Senior Health and Science Policy Adviser to Sen. Arlen Specter (D-PA), encouraged patient advocacy groups to stay involved throughout the appropriations process already underway and build on the momentum of the health reform law, to ensure the potential of the CAN provision can be realized. Learn about how you can support this effort.

The forum, moderated by Margaret Anderson, Executive Director of FasterCures, elevated questions and discussion items that will likely continue as the process to implement the new health reform law unfolds.

This conversation will continue online at FasterCures TRAIN Central Station. Join the discussion.

Health Reform Act Creates Cures Acceleration Network to Speed Researchers along the Path to New and Better Treatments for Disease

The Patient Protection and Affordable Care Act which President Obama signed into law on March 23rd included a little-discussed, but important provision creating a Cures Acceleration Network (CAN) at the National Institutes of Health (NIH), which will work to reduce the time it takes to move new drugs and therapies from the microscope to the marketplace.

This provision, introduced by Senator Arlen Specter (D-PA) as an amendment to the original Senate bill in 2009, creates new funding mechanisms at the NIH through which contracts will be awarded to accelerate the development of “high need cures” – defined as drugs, biological products, or devices:
• That are a priority to diagnose, mitigate, prevent, or treat harm from any disease or condition
• For which the incentives of the commercial market are unlikely to result in its adequate or timely development.

It will establish CAN within the Office of the Director of NIH and authorize grants expected to more quickly move discoveries from the lab through the development, testing and regulatory review process, into the hands of the patients that need them. This is an important step forward in bridging the ever-widening gap between basic and clinical research, known as the “valley of death” – where promising ideas for new treatments for deadly and debilitating diseases are often slowed and sometimes dropped before they can be fully explored.

FasterCures is committed to helping move basic science down the path towards treatments, and looks forward to supporting NIH in this effort. As we noted in our report “Entrepreneurs For Cures: The Critical Need for Innovative Approaches to Disease Research,” the true measure of success will be in lives saved and suffering diminished.

To learn more, participate in the forum:
Infusing Life into the Valley of Death
Wednesday, April 7, 2010, 8:30-10am
1101 New York Ave. NW Suite 620
Washington, DC 20005
Click here for additional information and to register.

Parkinson's Action Network and FasterCures invite you to participate in a forum about the Cure Acceleration Network, a provision in the health reform law, to learn how it can bridge the chasm between a basic scientific discovery and its application as a medical solution, known as the "Valley of Death." Speakers include John Myers, Office of Sen. Arlen Specter (D-PA); Amy Rick, CEO, Parkinson’s Action Network; John Schall, Deputy CEO, Parkinson’s Action Network; and Margaret Anderson, Executive Director, FasterCures. RSVP by Monday, April 5 to Angelo Bouselli abouselli@fastercures.org or 202.336.8900.

Bridging the Valley of Death: Improving the Federal Commitment to Translational Research

FasterCures invites you to a special presentation and discussion
Wednesday, April 22, 9:30 am – 11:00 am

Featuring:

• Amy Comstock Rick, Chief Executive Officer, Parkinson’s Action Network
• Mary Richards, Deputy Chief Executive Officer, Parkinson’s Action Network
• Margaret Anderson, Chief Operating Officer, FasterCures

It is widely acknowledged that one of the factors behind the slow momentum in clinical discovery and application is the ever-widening gap – referred to by some as a “valley of death” – in funding and support for the kind of research that moves basic science down the path toward treatments. Years of discovery, the genome and information revolutions, the doubling of our national investment in research through the NIH, and a new sense of urgency for progress in curing diseases have created a changed research environment that demands a system with greater flexibility, efficiency, and accountability.

In recent years the federal government has acknowledged the need to enhance its commitment to translational research so that Americans will see a better return on the enormous investment of their tax dollars in the form of improved health and cures for disease. What is needed to promote more effective investments in translational research at the federal level? What can the community of research advocates do to support this goal?

Please join FasterCures for this important discussion on:
Wednesday, April 22
9:30 am – 11:00 am
1101 New York Avenue, NW, Suite 620
Washington, DC 20005

Please RSVP to Patricia Wolf at pwolf@fastercures.org or 202-336-8917.