Nonprofits Find and Fund Big Ideas in Small Spaces

Katie Hood, Michael J. Fox Foundation (MJFF) CEO in a recent blog post discusses getting higher returns on medical research philanthropy, and asserts that “private philanthropists need to break out of the collective comfort zone of a traditional, 'academics-only' approach.” Her organization and several others were profiled at the recent Biotechnology Industry Organization convention in two panels and via the partnering database. Although the models pioneered by groups such as MJFF, Cystic Fibrosis Foundation (CFF) and Multiple Myeloma Research Foundation continue to garner attention and are looked to as best practices, Ms. Hood also raises the important question of why there are not more examples being proliferated.
Our recently released report, Entrepreneurs for Cures, focuses on the need for and importance of more examples that will lead to medical breakthroughs, and notes that, “The measure of our success is lives saved and suffering diminished – not the number of grants awarded, publications presented, or laboratory space acquired.” This report highlights how nonprofit disease research organizations are finding and funding big ideas, effectively paving the way for developing new business models to accelerate research.
At an all-day session at the Institute of Medicine (IOM) on June 23, 2008, I spent the day hearing speakers on the topic of “Breakthrough Business Models: Drug Development for Rare and Neglected Diseases and Individualized Therapies,” which spotlighted nonprofits as innovators in translating research from basic science to therapies for patients. A wide variety of topics were raised:

• for academic researchers: How can we get beyond the need to only share results via peer-reviewed journals?
• for diseases of the developing world: how can we raise the incentives to address them?

As part of this IOM meeting, I moderated a panel on “Strategies for Facilitating Sharing of Research Materials and Data” where Michael Mowatt, Director of the Office of Technology Development of NIH’s NIAID, talked about the imperative to share via repositories and other means. He highlighted the example of the Bill & Melinda Gate’s Foundations AIDS vaccine collaborations as an example to look to. Sharon Terry, President & CEO of the Genetic Alliance urged the audience to “Learn the rules so you can break them properly” and recounted the experience at the Alliance in “herding cats” to get research not just from bench to bedside but to get it into practice. She reflected that one way they herded cats was by moving the food. That would certainly get my cat’s attention. The Genetic Alliance repository was described as an “ebay” for samples. Laurie Ryan, Program Director of Alzheimer's Disease Clinical Trials at the National Institute on Aging, NIH, discussed the Alzheimer’s Disease Neuroimaging Initiative, an important public-private partnership which is gaining increased international attention.

In a panel on intellectual property, Craig Sorensen, Senior Director of Strategic Research Alliances at Vertex Pharmaceuticals outlined their work and collaborations in cystic fibrosis with CFF, Huntington’s disease with the Cure Huntington’s Disease Initiative, and tuberculosis and said that “industry is hiding behind cliches about failure and is doing a lot of running and staying in place.”

As Queta Bond, President of the Burroughs Wellcome Fund said at the end of the day, “We need to let more flowers bloom.” At FasterCures we’ll keep figuring out if we can help the process along and provide as much fertilizer as we can…
- Margaret Anderson, COO, FasterCures
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Exploring the Progress and Potential of Cancer Care

I had the privilege of hearing some terrific speakers at a recent forum in Washington, DC called “Outlook on Oncology: Exploring the Progress and Potential of Cancer Care” sponsored by Pfizer Oncology. After an overview of the Pfizer oncology portfolio, Dr. Kavita Patel, Deputy Staff Director of the Senate Health, Education, Labor & Pensions committee, spoke about her work on Senator Kennedy’s much anticipated cancer legislation. The legislation will look at the highest priorities for federal action and will take a much needed comprehensive approach so as not to pit one cancer against another. She highlighted many of the unfortunate realities in cancer research that continue to need attention: a biospecimen network that is not entirely networked and not universally accessible to researchers, a clinical trials system that mystifies even the most savvy patients and practitioners, and a need to do more…much more.

She cited the need to look closely at public/private partnerships and to fortify what the public and private sectors can do together to advance cancer research and care. When I asked her if the legislation was going to incent opportunities to fund and target innovative research at the National Cancer Institute she replied that they’d looked closely at existing models that allow this flexibility. Will this cost money? You bet. But Dr. Patel pointed out that if we don’t put money into this now, we’ll lose tremendous opportunities for innovation, not to mention the prospect of losing talent to other nations that are not intimidated by risk and are recruiting scientific talent faster than the American Idol winners are getting scooped up. Finally, she said they want to do health care policy forecasting so they can pre-anticipate what the implications of the legislation will be on all the systems in six months, one year, five years, etc. How refreshing to think about some advance planning in biomedical research! It is inspiring to hear her talk about Senator Kennedy’s renewed commitment to this legislation, and comforting to know that the Lion of the Senate is roaring.

Linda Ellerbee, a noted journalist and breast cancer survivor gave a captivating luncheon speech about the power of each patient and described her personal journey after her diagnosis 16 years ago. It was remarkable to think back to a time when speaking of breast cancer or any cancer for that matter was a taboo subject. I have worked in the HIV/AIDS movement and have seen the same transformation in that arena, where HIV invoked fear and silence. Ms. Ellerbee talked about how when the public demands something, things can change. I reflected on how AIDS and cancer activists have changed the landscape of biomedical policy. We still need much more change, so her words were an important reminder that there is always more to do. At FasterCures, we are embarking on several exciting new initiatives in our new strategic plan. Stay tuned to hear about ways we want to impact many of these issues and foster change, in areas such as biobanking, the conduct of clinical trials, and in fostering innovation.

- Margaret Anderson, COO, FasterCures

Improving your Risk-Reward Ratio Could Depend on Who Your Partners Are

Breaking away from the traditional model of financing medical research, biotechnology companies have started turning to nonprofit disease research organizations not only for their leadership, patient network and advocacy clout, but also for their financial capital. A panel of nonprofit leaders and biotechnology executives at this year’s BIO convention, organized by FasterCures and moderated by Margaret Anderson, FasterCures Chief Operating Officer, unanimously concurred that new paradigms are needed and that therapeutic outcomes are not likely to come out of academic research centers.

At FasterCures, we believe that one of the most significant impediments to accelerating progress toward cures is the difficulty of transforming research with therapeutic promise into marketable, profitable products that help patients. To address this major roadblock, there are growing numbers of biotech companies beginning to forge new and innovative alliances with nonprofit research foundations to reach new product development milestones. And many of the nonprofit research foundations understand that the only way their work will ultimately help the patients they serve is if they can "hand off the baton" at some point to an entity that makes products and can get them to patients.

Free of the imperatives of publication and career advancement in academia and the bottom-line imperatives of the private sector, disease research foundations are ideally positioned to make relatively high-risk investments that could significantly move a field of disease research forward and increase the likelihood that other parties will also invest.

Panel highlights:

• Katie Hood, CEO at the Michael J. Fox Foundation (MJFF), talked about how they are proactive about project management with their grantees and believe in collaboration. Their work with Elan shows a recognition of the leadership that MJFF has provided in the Parkinson’s research arena. (Read Katie Hood's blog on the Huffington Post: Breaking out of the 'academic comfort zone' for higher returns on medical research philanthropy)

• Ted Yednock, Executive Vice President and Head of Global Research at Elan Corporation, said its relationship with the Michael J. Fox Foundation has "increased the company’s exposure to cutting-edge science."
• Russell "Rusty" Bromley, Chief Operating Officer of the Myelin Repair Foundation said that this new model for medical research allows the foundation to "recognize, overcome hurdles, and remove friction" in the processes of drug discovery.
• Vertex Pharmaceuticals President and CEO Joshua Boger points to its partnership with the Cystic Fibrosis Foundation as a model for closing the gap in treatment for diseases with small or no markets. Additionally, Boger said this partnership energized employees, stimulating renewed commitment to finding a cure for cystic fibrosis.
• Another example of a successful partnership is between the Multiple Myeloma Research Foundation (MMRF) and Proteolix, Inc. Louise Perkins, Director of Research at the MMRF, talked about the many initiatives MMRF is engaged in to aggressively find a cure for multiple myeloma.
• Lori Kunkle, Chief Medical Officer at Proteolix, discussed how this partnership yielded greater enrollment in clinical trials, allowing them to accelerate their schedule, broadened exposure to multiple uses of a particular drug and allowed Proteolix to make early decisions about whether to move a compound forward in he development process.

For-profit biotechnology companies look to nonprofit disease research organizations for strategic and intellectual guidance as well, allowing access to a broad and deep arsenal of disease-specific research, patient networks, and advocacy tools.

Panelists acknowledge that the bottom-line drivers differ for each partner. On the for-profit side, profit drives the quest for innovation, while for nonprofit foundations the ultimate goal is finding a cure for the disease. The need to accurately understand the drivers and pay-offs for each partner is vital in ensuring a successful relationship; a model that’s beginning to change the paradigm of our medical research enterprise.

Many analysts believe that a "paradigm shift" is occurring at many pharmaceutical companies, which are realizing that relying on the blockbuster drug model for profits is no longer a viable business model and that – particularly as personalized medicine becomes a reality – there is a role for therapies for niche markets in their pipelines. These new business models require new partners, and hopefully the BIO meeting continued to nurture some of those existing relationships and the ones that are beginning to grow.

More discussion of these issues can also be found in our newly released report, Entrepreneurs for Cures, available to download now.

--Cecilia O. Arradaza, Communications Director, FasterCures

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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

Health IT’s Real Savings

Health information technology (health IT) is no panacea for our ailing healthcare system. There is no shortage of analysis weighing the costs and of the benefits an integrated health IT system.

• The RAND study estimates potential savings of approximately $80 billion per year from health IT if it were widely adopted.
• The Congressional Budget Office says health IT “appears to be necessary but not sufficient to generate cost savings and can be an essential component of an effort to reduce cost and improve quality; but by itself it typically does not produce a reduction in costs.”

Wherever the savings may lie, a sense of urgency and momentum is developing around the issue of creating a comprehensive, interoperable health IT system to improve patient care. Officials from Kaiser Permanente and Microsoft on Monday announced that they are developing a patient information exchange pilot program. Google Health: recently began offering personal health records on the Web. These are just two of the more recent efforts that bridge health IT from policy to reality.

Oftentimes, the health IT discussion gets mired in a discussion of its potential for curbing costs or its threat to patient privacy. However, as health IT efforts progress, we at FasterCures are waving the red flag to draw attention to health IT’s promise for speeding the discovery of new therapies by allowing researchers access to the precious data contained in them.

Health IT systems are a critical research resource. The trend toward the capture and storage of patient information in digital form provides researchers with a potentially more efficient and effective means of accessing these data. This paves the way to the discovery new treatments, conducting post-marketing research in real-time, and to making clinical trials more cost-effective and accessible.

So, when the U.S. Department of Health and Human Services released its Federal Health IT Strategic Plan last week (more than three years since President Bush issued an executive order to develop the plan), we were pleased to see that in addition to improving patient-focused healthcare, the plan includes strategies focused on population health that would “enable the appropriate, authorized, and timely access and use of electronic health information to benefit public health, biomedical research, quality improvement, and emergency preparedness.”

We urge all private and public sector leaders to put a high priority on understanding and integrating the needs of medical researchers into health IT solutions. By providing the research community access to a broader and more diverse patient population, health IT presents us with the potential for real savings: in terms of both reducing healthcare costs and, more importantly, in curing disease.

FasterCures is a member of the Health IT Now! Coalition

-- Cecilia O. Arradaza, Communications Director, FasterCures

Chronic Diseases Emerge as a Global Health Concern

Chronic Diseases Emerge as a Global Health ConcernLast week, I joined representatives from the U.S. government, foreign governments, foundations, partners, and individuals from the global health field working in developing countries for five days of discussion during the Global Health Council (GHC) annual conference. The theme was Community Health: delivering, serving, engaging, and leading. Participants heard valuable lessons-learned and experiential evidence from those on the frontlines that would support their own community health programs in resource-poor settings.

Often, society associates developing countries with infectious diseases. The high prevalence of these diseases was reflected in more than 24 sessions dedicated to the “big three”: HIV/AIDS, TB, and malaria; and, the international community continues to agree that infectious diseases remain an enormous barrier country health systems need to battle. Several product development partnerships shared their successes and challenges trying to develop better and more effective diagnostics tools, prevention methods, drugs and vaccines for infectious diseases. The lay world is all too well aware how debilitating infectious diseases can be especially to those living in resource-poor settings.

At the GHC conference however, a packed audience also heard discussions about chronic disease in developing countries – specifically, cancer which is of growing concern. In 2002, there were 5.1 million new cases in developed countries and 5.8 million new cases in developing countries. By 2020, experts predict the majority of the 15-20 million new cases of cancer will occur in developing countries. Panelists emphasized cancer programs must be bolstered by a health system infrastructure, including cancer registries and robust screening programs, all of which require additional human and financial capital.

New capital and innovative, low-cost programs are needed to address many global health issues, including both infectious and emerging chronic diseases. The FasterCures Philanthropy Advisory Service allows for new capital to meet such innovative ideas, particularly with respect to biomedical research. Through a grant from the Bill & Melinda Gates Foundation and the Robert Wood Johnson Foundation, the Philanthropy Advisory Service will create a transparent, information marketplace for philanthropists to learn about effective and high-impact nonprofit disease research organizations. The initiative aims to channel philanthropic investment to areas where there are demonstrated funding gaps both in global and domestic health research. Initially the Philanthropy Advisory Service will focus on malaria and tuberculosis that plague the developing world, as well as Alzheimer’s disease and multiple sclerosis that impact the developed world. Ultimately, the Philanthropy Advisory Service can facilitate giving by providing clear and transparent information and thus direct capital for under-resourced, but scientifically-urgent research programs for all diseases around the world.

--Priya Patil, Global Health Program Manager, 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