Stubborn facts on biotech financing

By Kristin Schneeman, Program Director, FasterCures

“Facts are stubborn things,” was a saying that originated in the defense of the British soldiers at the Boston Massacre trial, meaning facts are facts and can’t be disputed.  But facts can also be stubbornly difficult to interpret sometimes. 

A new National Venture Capital Association survey was released last week, reporting that venture capital invested in biotech was up in 2011 by 22% from 2010, but that first-round funding was at its lowest level in 15 years, with only 153 drug and device companies getting seeded.

But as the folks at InVivo blog reported, the other major source for data on venture investment, DowJones VentureWire, also released its year-end totals and came to slightly different conclusions: Total biotech funding rose by only 11% -- all of it on the device side.  And InVivo’s own data on early-stage financing showed that slightly more companies received Series A financing in 2011 than in 2010, giving them a more positive spin on 2011. 

Bruce Booth with VC Atlas Ventures took the media to task for making positive data into a negative story.  He looked at the NVCA’s own data (though through a slightly different lens) and came to some different conclusions as well:

• Total VC funding of first rounds in biotech last year was one of the four highest annual totals since 1995;
• The number of first-round startups in 2011 was in line with long-term averages; and
• The average size of a first round investment hit a 16-year high.

So whose facts are the right ones, and whose interpretation of the facts is correct?  What metrics are most important here – the total amount of funding that went to early-stage biotechs, or the number of companies that got funded?  Are bigger individual deals a sign that small, innovative companies are out of luck, or a reflection of a realization that the longer time horizons for a return in life sciences might require bigger up-front investments?  What timeframes are most important – trends over the last 12 months or over the last 12 years? 

And what on earth does any of this mean for patients hoping these companies hold the key to the treatments they’re waiting for?  Perhaps it’s less about the total amounts of money that are flowing into the system and more about how to make sure the right kind of money (venture, government, foundation) is lined up at the right time for the right ideas so inventors and companies aren’t wasting time “circling the airport” looking for the next infusion of cash. 

Do we need some new models or infrastructure to try to make financing more efficient?

Revving the Engine that Propels Us

by Gillian Parrish, Manager, Alliance Development and Communications, FasterCures

Over the past few decades, the U.S. has created and refined an extremely productive framework for developing new biomedical innovations and bringing them to the marketplace.  In fact, the biomedical sector directly and indirectly accounts for some 5 million U.S. jobs, including 1.2 million high-wage private sector jobs in pharma, biotech, medical devices, research and testing. 

However, for the first time since the 1980’s, U.S. preeminence in biomedicine is in jeopardy, vulnerable to falling behind growing powers in Europe and Asia thanks to regulatory, policy, and funding efforts in those nations that offer incentives for innovation and support entrepreneurship in a way the U.S. does not.

The history of our country’s rise to leadership in biomedical innovation, and a suggested roadmap for overcoming the factors that now challenge our dominance in the global market, is the focus of a new Milken Institute Study, The Global Biomedical Industry: Preserving U.S. Leadership.  Released last week at a Washington D.C. briefing co-hosted by the Council for American Medical Innovation, the report offers specific policy recommendations that will ensure the industry continues to grow and flourish here, including:

• Increase R&D tax incentives and make them permanent.
• Cut corporate tax rates to match the OECD average.
• Extend support for emerging biomedical research fields.
• Provide adequate resources for the FDA and NIH to expedite regulatory reviews and clinical trials.
• Leverage existing strengths in medical devices.
• Build human capital for biomedical innovation.
• Promote and expand the role of universities by adopting best practices in technology transfer and commercialization.

It makes a compelling case for action, and reinforces FasterCures’ long-held position that no one sector can do this alone.  It will take collaboration – universities and businesses, corporations working together, private equity entrepreneurs willing to invest in high-risk, high-impact R&D models, innovative intellectual property arrangements, etc. – to maintain U.S. leadership in biomedicine. 

“As recently as 1980, European companies dominated the industry,” said Ross DeVol, Chief Researchers at the Milken Institute. “Unless we act soon and act smartly, we’ll wake up in five to ten years with no products and nothing in the pipeline. This is our industry to lose.”

“The alarm this report sets off is very real,” said Jim Greenwood, President and CEO of the Biotechnology Industry Organization (BIO), at last week's event. “The fear that the U.S. is losing its competitive edge is well-founded.  If we want to have more cancer cures than iPads, we have to invest in them.”

Strengthening the biomedical sector should be central to our country’s economic platform. Government, particularly the National Institutes of Health (NIH) and the Food and Drug Administration (FDA), should work with the private sector to get more and better therapies to patients. 

“The 21st century is going to be the century of life sciences,” said Stephen Ubl, President and CEO of the Advanced Medical Technology Association (AdvaMed).  “The question is: will it be made in America or somewhere else?” 

Many countries are building the infrastructure for biomedical research, and courting the companies who drive it.  Our current leadership is in question, and comes with no long-term guarantee.  But if the advocacy and policy communities can come together to tap into our nation’s most unique attribute – our collaborative, innovative spirit – we can take the decisive steps necessary to not only maintain, but grow our leadership in biomedicine.

 

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.

Biomedical Innovation: Can the U.S. Keep Its Edge?

by Adam Clark, Ph.D., Director of Scientific and Federal Affairs, FasterCures
Over the past decade, international investments in life sciences research and development (R&D) in the in the private-sector have shifted away from the U.S. An expert panel at the 2011 Milken Institute Global Conference examined this shift and explored policies that influence biomedical innovation in the U.S.

Ross DeVol of the Milken Institute framed the issue by noting that it was not until the late 1990s that the U.S. began to dominate global pharmaceutical R&D. U.S. leadership can be attributed to a number of factors, he said, including a more predictable regulatory framework and adoption of intellectual property policies such as the Bayh-Dole Act, which encouraged entrepreneurship. However, DeVol said that “our regulatory approval process has gotten more cumbersome,” which has resulted in a risk-averse environment that inhibits medical innovation.

Edward Holmes of Singapore’s Agency for Science, Technology, and Research (A*STAR) said that Singapore has made the biomedical industry a top priority and developed policies that have resulted in a four-fold growth in the biomedical industry in the past decade. Such policies created an environment with sound intellectual property strategies, efficient regulatory programs, effective ethical frameworks for clinical trials, and an educational infrastructure that strongly supports science and math education to secure a strong scientific workforce. Singapore continues to focus on biomedical manufacturing and a strong effort to build up efforts in R&D.

Michael Leavitt, former Secretary of the U.S. Department of Health and Human Services, emphasized that the next decade of U.S. biomedical innovation will be built upon the “Era of the Value Proposition” in which “the future innovation will have to involve a value proposition that will demonstrate to a payor that the innovation will result in a change in the cost curve.”

Bijon Dorri of General Electric, explained GE’s interest in the healthcare sector and highlighted GE’s Healthymaginations program as an area in which GE is increasing its global competitiveness in healthcare. For projects supported through the Healthymaginations, it must address at least one of three pillars of healthcare: reduce cost, improve quality, or increase access. These guiding principles, he said, are “driving a change in the value proposition of healthcare innovation.”

The panel discussed the growing costs of clinical trials as one of the primary drivers to move R&D out of the U.S. to countries where such costs are significantly lower.

Collaboration was another key theme the panel deliberated on. Partnerships between the private and public sectors are key to moving therapies forward. There needs to be open communication channels between industry and the National Institutes of Health, the FDA, and the Centers for Medicare and Medicaid to ensure a seamless continuum of R&D innovation from discovery to development to dissemination and access.

Lesa Mitchell of the Kauffman Foundation moderated this panel. Watch a video of this session.

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.

“Partnering with Patients” at the Biotech Industry Organization Convention

by Kristin Schneeman, Program Director, FasterCures

All I can say is, I wish I’d read Maureen Martino’s FierceBiotech blog before I left for Chicago.

A week and a half ago I was gearing up to moderate a panel discussion at the Biotechnology Industry Organization’s annual convention in Chicago about venture philanthropy and the impact it’s having in accelerating the development of new treatments for patients. We’ve chaired panels similar to this one at BIO in the past, highlighting the financial resources patient organizations can bring to the table and the partnerships they’re increasingly forming with companies to develop products. This year we decided to focus in a little more on the non-financial assets these organizations also bring -- the “patient capital” in the form of tissue banks, medical data, and participation in clinical trials – as well as their ability to serve as “systems integrators” bringing all of the stakeholders together and smoothing over the information and funding gaps in the research process.

We assembled a phenomenal panel - passionate patient advocates, but also serious and successful R&D professionals -

many of whom had come from industry, started companies, and consulted with companies. They included:

• Howard Fillit, Executive Director of the Alzheimer’s Drug Discovery Foundation (ADDF);
• Dick Insel, Vice President for Research at the Juvenile Diabetes Research Foundation (JDRF);
• James O’Leary, Chief Innovation Officer at Genetic Alliance (GA);
• Louise Perkins, Chief Scientific Officer at the Multiple Myeloma Research Foundation (MMRF); and
• Steve Perrin, Chief Executive Officer and Chief Scientific Officer at the ALS Therapy Development Institute (ALS TDI).

Their organizations have invested more than a billion dollars in medical research, have global reach, have supported hundreds of drug discovery programs and scores of clinical trials, and have played a critical role in development of products that are helping patients today.

Among the many impressive nuggets I jotted down during the session:

• MMRF has demonstrated its ability to activate clinical trials 30-40% faster than the industry standard, through its powerful consortium of 13 leading academic research institutions.
• JDRF is pursuing more than 20 development efforts in partnership with companies -- many of which have been picked up by large industry players – including novel agreements with Johnson & Johnson and Novartis to help those companies fill their product pipelines.
• ADDF has been instrumental in the creation of several startup companies that have gone on to leverage enormous amounts of follow-on investment.
• GA is providing resources to help patient groups in rare genetic diseases marshall the patient assets they need to help them engage companies in developing treatments for those diseases.
• ALS TDI has created a new model, a “nonprofit biotech company,” that is shaving millions of dollars off the development process and de-risking industry investment “by keeping patients in mind from the very start.”
  
  

• • At the end of the session, I offered a question for further reflection and discussion: What are the implications of putting the patient (intermediated by organizations like these) at the center of the research and development process – not as research subjects or consumers, but as partners and decisionmakers?

After I left Chicago I came across a blog posted by FierceBiotech’s Maureen Martino from BIO earlier in the week. In it she recounts her chance conversation with three breast cancer survivors in an airport shuttle. She asked them what they would say if they could talk to the companies developing breast cancer treatments: “A whole lot, as it turns out.”

Among their frustrations was the lack of true communication among patients, their physicians, and particularly the companies developing drugs to help them. “They should pay us to come talk to them,” commented one. She may have said it half in jest, but I suggest we take that idea seriously. And invite the FDA to listen in.

Kudos to Maureen Martino for trying to present a “human face [to] the drug industry.” We’ve been trying to do the same thing for a number of years by supporting the work of innovators like our BIO panelists, who are the trusted intermediaries for meaningful participation by patients in the research process. As a participant at a recent FasterCures meeting has said, “Patients are the only unique resource in the entire continuum … they can create the ultimate bargaining unit.” Where’s Norma Rae when you need her?

Jeff Kindler, Anthony Fauci, Kathy Giusti, Michael Milken among featured speakers at Partnering for Cures

Philanthropists, Medical Research Foundations, and Industry Leaders Gather in New York for Inaugural Partnering for Cures Meeting
First-of-its-kind meeting designed to forge collaborations needed to accelerate the pace of therapeutic development

(November 5, 2009) WASHINGTON, DC – Hundreds of medical research leaders, investors, and decision-makers are coming together at the inaugural Partnering for Cures meeting to explore innovative approaches to pursuing high-risk, long-term investments that could lead to therapeutic breakthroughs.

This first-of-its-kind meeting, to be held December 1-3 at the Millennium Broadway Hotel in New York City, brings together innovators from philanthropy, medical research foundations, and the biotechnology and pharmaceutical industries with the goal of forging collaborations necessary to develop new medical solutions.

The meeting is designed to facilitate informed investments and cultivate relationships by featuring panels that spotlight solutions to decades-long challenges facing medical research, adapting the outcomes-oriented approach of investor conferences, and building on the networking opportunities at industry "partnering" meetings. Speakers include:

Decision-makers from the biotechnology and pharmaceutical industries:

• Jeffrey Kindler, Chief Executive Officer and Chairman of the Board, Pfizer
• The Hon. Billy Tauzin, President and Chief Executive Officer, Pharmaceutical Research and Manufacturers of America (PhRMA)
• The Hon. James Greenwood, President and CEO, Biotechnology Industry Organization
• Garry Neil, M.D., Corporate Vice President, Corporate Office of Science and Technology, Johnson & Johnson

Venture philanthropy leaders:

• Robert J. Beall, Ph.D., President and Chief Executive Officer, Cystic Fibrosis Foundation
• Debi Brooks, Co-Founder, Michael J. Fox Foundation
• Kathy Giusti, Founder and Chief Executive Officer, Multiple Myeloma Research Foundation
• Hala Moddelmog, President and Chief Executive Officer, Susan G. Komen for the Cure

Philanthropists who are transforming the medical research enterprise

• Michael Milken, Chairman, FasterCures/The Center for Accelerating Medical Solutions; Chairman, The Milken Institute
• Debra Black, Founder and Chair, Melanoma Research Alliance

And, some of the most forward-thinking leaders in medical research:

• Anthony S. Fauci, M.D., Director, National Institute of Allergy and Infectious Diseases
• Douglas A. Melton, Ph.D., Co-Director, Harvard Stem Cell Institute
• Seth Berkley, M.D., President and Chief Executive Officer, International AIDS Vaccine Initiative
• Anne Wojcicki, Co-Founder and President, 23andMe

Philanthropic investment in medical research, though it accounts for only three percent of overall research spending – relatively small compared to government and industry funding –plays an essential role in catalyzing high-risk research, far beyond its proportion of funding. But, it requires a significant investment in effort, resources, and time to find the right research partner. Partnering for Cures streamlines this process by connecting similar-minded organizations and sharing best practices, further amplifying the transformative impact of medical research philanthropy.

Partnering for Cures is convened by FasterCures, the Washington, D.C.-based center of the Milken Institute, that's committed to breaking down the barriers that exist across the research continuum – from basic research to drug development – to clear the path to faster medical progress.

NOTE TO MEDIA:
Complete information about Partnering for Cures, including a full program and an up-to-date list of all speakers, is available at www.partneringforcures.org. Credentialed press wishing to cover Partnering for Cures, please email Cecilia Arradaza, FasterCures Communications Director, at carradaza@fastercures.org.

Alzheimer’s Disease Diagnosis Could be in the Eye of the Beholder

At last week’s BIO conference in San Diego, a panel of scientists and physicians focused on the “Advances in Biomarkers and Diagnostics in Alzheimer’s Disease (AD) and Psychiatric Disorders.” Biomarkers are emerging as a priority research area as they hold the potential for swifter diagnosis of disease even when symptoms are silent. Early diagnosis in combination with early intervention will yield far more effective treatment options and disease management strategies. In addition, biomarkers can facilitate disease monitoring to more efficiently determine patient’s response to current treatment regimens.

According to the Biomarkers Consortium, a collaborative public-private partnership managed by the Foundation for the National Institutes of Health, biomarkers are objective measurements of biological processes that can substantially improve the precision with which we evaluate disease risk, diagnosis, and progression, and guide treatment. They also promise to revolutionize both the development and use of therapeutics. In some cases, they may allow for earlier “go/no go” decisions in developing treatments and help shave years and cost off the development process by providing a clearer indication of a treatment’s efficacy, dosage, or safety. Biomarkers are the foundation of evidence-based medicine, making the practice of medicine more personalized, predictive, and preemptive.

The advancement of biomarker science is particularly important for patients suffering from AD for which the current diagnosis takes approximately two years and consists of a complex “process of elimination” based upon cognitive symptoms. A biomarker for AD would impact this field of medicine dramatically, allowing for earlier treatment and more accurate monitoring -- greatly needed to fight an impending AD epidemic.

AD is characterized by large amounts of plaques of beta-amyloid in the brain. Beta-amyloid is formed when amyloid precursor protein an important protein for neuron growth and repair is severed by enzymes. Four years ago, Lee Goldstein from Boston University, featured on the panel, and his research team found that these amyloid plaques also collect in the lens of eyes in people afflicted with Alzheimer’s disease and causes an unusual cataract that is different from common age-related cataracts.

The first biomarker for Alzheimer’s disease was identified. Goldstein’s discovery established the first evidence of Alzheimer’s-linked pathology outside the brain, and led Goldstein and his colleagues to develop a laser-based diagnostic technology that searches for amyloid protein buildup in the eyes and could aid in early detection of the disease. Goldstein’s team hopes that in the next couple of years physicians will be able to use this laser-based molecular diagnostic screening test for Alzheimer’s disease and do without the costly, two-year, multiple-visit Alzheimer’s disease diagnosis process.

Imagine the potential biomarker technology could provide the way we efficiently and accurately diagnose diseases, improve quality of clinical care, and enhance disease management for patients and caregivers. At FasterCures, we will continue to keep a close eye on developments in biomarker science and support efforts that encourage, inspire, and fuel innovation in medical research and discovery.

Melissa Stevens, Director of Special Projects, FasterCures

States Compete to be “Home” of Innovation

Economic woes continue to permeate front-page news, presidential candidates’ agendas, and water-cooler conversations. In our knowledge-based economy, states are committing major capital and human resources to remain competitive

A report released last week at the BIO 2008 Convention by the Milken Institute, the State Technology and Science Index 2008, takes a comprehensive look at each state’s ability to deal with the shifting nature of the economy and assesses science and technology assets that could be leveraged to promote economic development.

In the Milken Institute study, the states are ranked based on 77 individual indicators in five categories: research and development; risk capital and entrepreneurial infrastructure; human capital investment; technology and science workforce: and technology concentration and dynamism.

• Massachusetts tops the list and “continues to set the pace for other states in terms of technology and science capabilities.” A hotbed of research, world-renowned research universities and cutting-edge firms fuel the state’s economy. On June 16, Massachusetts Governor Deval L. Patrick signed a bill that commits $1 billion into the state's growing life sciences industry over the next decade. The bill includes $250 million in tax incentives for companies, $250 million in grants, and $500 million for infrastructure, much of which is earmarked for the state university system.
  
  According to the Boston Globe, “though biotech still only accounts for a little more than 1 percent of the state's workforce, Patrick has focused on nurturing the life sciences industry because of its growth potential, high salaries, and ability to pump money into the economy.”
• Ranking a close second is Maryland, which moved up from 4th place in the 2004 index. The same day Massachusetts signed its $1 billion life sciences bill, Governor Martin O’Malley unveiled its BIO 2020 Initiative, an effort that will invest $1.1 billion in the state’s bioscience industry over the next 10 years to attract and grow biotechnology companies in Maryland.
  
  In announcing this new initiative, Gov. O’Malley said, “At the end of the day, our greatness as a State and as a people will be determined by how we meet the essential challenge of the present. How we invest in our capabilities for healing this fragile and ever smaller world of ours.”
• Colorado remains in third place. California places fourth, slipping from 2nd place in the 2004 index. Fifth place goes to Washington. For a complete list of state rankings, download a copy of the Milken Institute report.

“States that have a vision and a plan for building and retaining high-wage jobs and viable industries are finding ways to invest in their science and technology assets,” said Ross DeVol, director of Regional Economics at the Milken Institute, and lead author of the study. “The changes in this year’s Index give a good measure of who is ahead in the increasing competition for scarce human capital and other resources needed for a successful industry.”

The report highlighted a new challenge that arose as an unintended consequence of post 9-11 security restrictions on student visas. International graduate student enrollment in U.S. universities has declined, limiting the opportunity to attract the world’s top talents into American institutions. The nation faces the possibility of losing human capital overseas.

This forces states to compete to draw both human and financial capital from other states.
And, with more states scoring near the mean, competition between states is expected to intensify.

However, the main threat to any state’s position emanates from abroad. It is more important than ever for states to remain focused on fostering innovation and entrepreneurship to ensure the nation’s leadership in this fast-paced global industry – “especially as the national economy enters a challenging period.”

Cecilia Arradaza, Communications Director, FasterCures

Realizing the Promise of Personalized Medicine

Last week, media outlets reported on a study about the use of genetic research to personalize cancer treatment, presented at the 2008 annual meeting of the American Society of Clinical Oncology (ASCO) in Chicago.

The Wall Street Journal reported on data that showed Imclone's Erbitux benefits newly diagnosed colorectal cancer patients and confirmed that patients with a certain gene mutation don't respond to the drug. The gene is called K-ras, a regulator of cell growth that researchers say plays a crucial role in several cancers. The results might reduce the market for the drug but could help doctors identify those patients likely to benefit the most.
Genetic research is allowing doctors to customize the drug treatments most likely to work for each patient. Before, treatment has included some guess work. The end result is that a patient can undergo an inefficient and costly game of hit-or-miss.

Personalized medicine is beginning to garner buy-in from large pharmaceutical and biotech companies, which are just now coming to terms with the reality that the blockbuster way is not the only way. This smaller market approach promises better patient outcomes. As well, biomarkers such as K-ras paves the way for targeted, more effective research efforts and could potentially lower clinical trial costs and accelerate the advancement of medical solutions.

FasterCures is a strong proponent of personalized medicine efforts and strongly believes that 21st century healthcare relies greatly on advances in genetic research, and other innovative medical solutions that fuel the search for new treatments and cures. The passage of the Genetic Information Nondiscrimination Act (GINA), albeit the 13-year delay, allows patients to confidently participate in studies that search for linkages between genes and disease, to enroll in clinical trials for new targeted drugs, or to provide samples for DNA analysis to optimize their own disease prevention and treatment.

These new discoveries in cancer hold enormous value to the rest of our disease research system. We urge pharmaceutical and biotech companies to continue to invest in this type of R&D. While we acknowledge the business imperative of searching for the next blockbuster drug, we believe the ROI for successfully pursuing customized treatments is important. Lives saved and improved quality of care for each individual should always be the measures for medical progress.

Angelo Bouselli, Communications Manager, FasterCures

The Future of Biobanking Relies on the Quality and Viability of Biospecimens

The theme of collaboration carried over into day three and four presentations at the ISBER Annual Meeting, from the role of industry in biobanking and the importance of collaboration between academia and the corporate sector to the importance of global biobank networking. Throughout the conference, in asides by presenters and conversations among attendees, another theme began to emerge: the lack of information about quality and viability of biospecimens.

• A roundtable led by Dr. Mark Cosentino of NCI Frederick was tasked with addressing the topic "Repository Science: Establishing a New Scientific Discipline." A packed table engaged in a discussion that covered degree types, funding, and oversight of educational programs to train repository scientists, but talk quickly turned to curriculum: how can biobanking scientists be trained without knowledge of appropriate biospecimen processing, handling, and storage for a variety of analyses? Participants were unanimous that much more information on biospecimen science is absolutely essential to train effective biobanking scientists.
• At poster sessions, there was a similar sentiment – not only is more information needed about biospecimen science, it is needed through a consolidated, open access resource. After walking me through her poster on varying RNA levels in breast tumor tissues collected by different protocols, Rebecca Barnes of the British Columbia Cancer Agency’s Tumor Tissue Repository noted that studies like hers were being performed by other researchers around the world, but it is difficult to know who is studying what and what the results mean without a clearinghouse or forum for information sharing.
• Carolyn Compton of OBBR at NCI and David Horsfall of the Australian Prostate Cancer Bioresource presided over a panel of speakers whose talks addressed the impacts of packaging, fixation, specimen type, and length of storage on biomarkers. One of the key panel presentations was given by Barbara O’Brien of Westat; she presented a study that focuses on stability testing of biospecimens in conjunction with the National Children’s Study (NCS). NCS will examine the effects of environmental influences on the health and development of 100,000 children in a 21-year longitudinal cohort, beginning pre-conception with sample collection and analysis from parents. The collection schedule and length of study mean that NCS will collect literally millions of samples in the first years of the study. To ensure the validity of data generated from these biospecimens, Westat will undertake long-term stability testing of samples collected from parents of study subjects. Sample specimens to be assessed include urine, saliva, vaginal secretions, hair, blood and blood derivatives, toenails, and breast milk, and Westat will evaluate the stability of dozens of analytes in each of these matrices over the course of the 21-year study. The results of this assessment will add a considerable body of evidence to the field of biobanking and biospecimen research.

The success of biobanking as a tool in fighting disease requires more insight into the properties of different types of biospecimens and how these should be collected, processed, and stored to preserve key biomarkers. Piece-meal studies alone would not address this need; instead, a coordinated effort, with data sharing and public access to findings, is necessary to ensure that this knowledge is appropriately applied. We look forward to ISBER’s leadership in paving the way to bridging this critical gap in scientific knowledge.

-- Kate Blenner, FasterCures, Program Analyst

Global Biobanking Collaborations: Challenges and Opportunities

Blogging from the ISBER 2008 Annual Meeting

The ISBER annual meeting theme of collaboration rang true at the opening plenary session and echoed throughout Monday’s program. Cooperation and harmonization among biorepository resources could bolster the scientific toolkit for understanding disease cause and treatment response.

Dr. Francis Collins, Director of the National Human Genome Research Institute (NHGRI), an esteemed scientist and luminary, provided the keynote address, outlining several pioneering projects that, with the use of biospecimens and collaboration, would inform our understanding of disease to unlock clues for developing cures. These projects include an international cancer genomics consortium that would collectively sequence 50 different tumor types, yielding molecular data to drive development of more targeted diagnostics, therapies, and prevention mechanisms. It also includes the 1000 Genome Project, an effort between the Wellcome Trust Sanger Institute, Beijing Genomics Institute, and the NHGRI that will sequence genomes from 1000 people. With this detailed information, researchers can better understand at a molecular level why some people contract certain diseases and others don’t, and why some treatments cure disease and others won’t.


Monday’s program highlighted global collaborative efforts, including:

• iBOL (International Barcode of Life) project involving 25 countries that aims to develop DNA barcodes that will allow for automated species identification and thus more active management of biodiversity;
• Asian Network of Biological Resource Centers that serves as a collection of microbial cultures across China, Japan, Thailand, and Korea; and
• Biobanking and Biomolecular Resources Research Infrastructure (BBMRI) that intends to facilitate transnational collaboration, reduce fragmentation in the biobanking industry, and provide access of resources across academia and industry across the European continent.

Perhaps even more uplifting were discussions of not only collaboration, but also current and future activities to promote harmonization across biobanks. The lack of standards and practices across biorepositories is probably the most significant hindrance to realizing these resources' full contribution to the scientific process. An organization called P3G (Public Population Project in Genomics) is an international consortium whose members are public organizations undertaking large-scale genetic epidemiological studies and biobanks across North America, Europe, Asia, and Australia. P3G serves as a repository of information and tools and is developing tools such as their DataSHaPER (Data Schema and Harmonization Platform for Epidemiological Research). P3G notes that DataSHaPER is a comprehensive set of variables that should be collected by large epidemiological studies and biobanks for general-purpose biomedical research. The aim of the DataSHaPER is to provide a template to facilitate harmonization between biobanks and support the design of emerging ones. A future DataSHaPER will be developed for cancer as well.

The first two days of the meeting resonated with it's intended theme and demonstrated some impressive and ambitious collaborative efforts. The introduction of even early harmonization tools shows promise in unifying resources across institutions, countries, and continents. I look forward to tracking the progress of these efforts closely.

- Melissa Stevens, Director of Special Projects, FasterCures

Pre-ISBER Thoughts

In preparing for next week’s International Society for Biological and Environmental Repositories (ISBER) Annual Meeting, I recall some of the key takeaways from March’s Biospecimen Research Network Symposium "Advancing Cancer Research Through Biospecimen Science". The most startling came from Dr. Anna Barker’s opening remarks in which she noted that "of the 350 million specimens collected in the United States, only 30 percent of them were of a quality standard high enough for research purposes." And, we’ve actually heard other experts say that the number of viable samples could be even lower.

What if only 30 percent of the emails we sent everyday actually left our outboxes? What if our metro trains came only 30 percent of the time they were supposed to? That would make for a very unproductive world.

NCI’s Office of Biorepositories and Biospecimen Research (OBBR) brought together leaders in the field to discuss the challenges and recommend solutions. A number of the symposium speakers highlighted the variance in quality and study results across institutions and handling scenarios all underscoring the need for standard operating procedures. Carolyn Compton, Director, OBBR, outlined NCI’s role in elevating quality and Elizabeth Hammond, Professor of Pathology, University of Utah, presented a strong call to action for job aides, standard training, sharing of research, developing financial incentives, and implementing a biorepository “Goodhousekeeping Seal of Approval. ”

I hope the ISBER meeting could bring more focus to these calls to action in a global context. I am looking forward to discussing how international harmonization strategies can contribute in raising specimen quality. I am also very much looking forward to discussing communication strategies for engaging patients who are critical variables in the research equation. Most importantly I am looking forward to understanding how the global community can advance biorepository efforts to in turn accelerate medical solutions.

Check back next week as my colleague Kate Blenner and I bring you highlights from the ISBER meeting.

Melissa Stevens, FasterCures Director of Special Projects