Thinking Impact, Philanthropically

By Elizabeth West, Program Manager, FasterCures

Impact investing (also known in some circles as mission investing) is a blanket term used to describe a variety of investment practices similar in their intent to generate measurable social and environmental impact alongside a financial return. In 2012 the practice of impact investing blew up, and it seems like this trend is only going to get bigger. As FasterCures’ Philanthropy Advisory Service is committed to helping those investing in the life sciences make the most informed decisions possible, we could not pass up the opportunity to learn about this new trend (permanent fixture?) in philanthropy at two recent conferences.

  

Imagination, Innovation and Impact in Philanthropy

The first was the University of Southern California’s Annual Leadership Forum – Philanthropy: Imagination, Innovation, and Impact. The event brought together philanthropists, foundations, academics, and innovators to focus on understanding the passion and values of philanthropy and its possibilities and strategic practices that create greater impact. The highlight of the event was a candid discussion with Tom Steyer and his wife Kat Taylor - who have signed the Giving Pledge - alongside panels that covered an array of relevant topics from the power of collaboration, networks, evaluation, and learning to innovations like games for social change*. On impact investing, speakers highlighted different models (both familiar and unfamiliar), including the Omidyar Network, social impact bonds, community development financial institutions, and the Nonprofit Finance Fund.

Some key takeaways include:

• The impact investing field is still in a proof of concept stage.
• It is import to conduct financial and impact due diligence on the front-end as well as to evaluate investment on the back-end.
• Impact investing is not philanthropy – it tries to accomplish something that philanthropy and government do not know how to do.

Not surprisingly everyone agreed that evaluation is HARD. According to Steve Goldberg of Caffeinated Capital, of the ten largest federally funded programs, only one has demonstrated success. That may be a clear indicator that – at least according to the professionals at USC – impact investing is still in the “proof of concept” stage.

The second conference we attended was the Council on Foundation’s Annual Family Philanthropy Conference, which offered a three session impact investing track led by professionals from Mission Investors Exchange, RSF Social Finance, and Confluence Philanthropy, among others. These panels focused on some of the granular details of MRIs, PRIs, and broader “impact investing”, as well as valuable insights from the audience members from foundations engaged in this area.

The biggest message: 

• The primary challenge is convincing others of the opportunity and value of impact investing. (According to participants, CFOs, accountants, and trustees are the most difficult to convert.)

A particularly engaging presenter was Charles Kleissner, the co-founder of the KL Felicitas Foundation – a foundation that has led the way in putting both annual disbursements and assets into impact-first and financial-first investment vehicles. Kleissner spoke of how he arrived at impact investing – seeing it both as an alignment of his values and intentions, as well as a more efficient use of his capital. He considers himself a “100% folk” (i.e. someone who has 100% of his wealth in impact investing) and says now there are at least fix or six people in the world who are “100% folks” in all asset classes.

Impact Investing in Medical Research

So what are some potential examples of impact investing in medical research? Well it depends on what kind of entity is involved. Individual accredited investors can invest in companies that are developing new therapies for unmet medical needs. A foundation could use a program-related investment (PRI) to make a loan to a nonprofit research institution to finance a potentially high-impact research project. Philanthropic funds could also be invested at market or below market rates in commercial entities that meet a foundation’s mission, such as advancing the development of new medicines. (The Bill and Melinda Gates Foundation did this in the case of Liquidia in 2011).

Obviously, there is a lot of potential for growth and sophistication in impact investing. This is likely just the beginning of what you will hear from PAS on impact investing this year, especially how it relates to medical research. So stay tuned!

*Relevant Links

--- www.playspent.org

--- www.gamesforhealth.org

---  www.gameslearningsociety.org

Automatic budgets cuts could stall cancer progress

By Shawn Sullivan, Program Associate, FasterCures

“When I was growing up, cancer was a death sentence, and today my multiple myeloma is in remission because of a drug that was developed based on NIH-funded research,” said former nine-term U.S. Congressman M. Robert “Bob” Carr at a briefing earlier this month to unveil the American Association for Cancer Research’s Cancer Progress Report 2012.

While the focus of the briefing was on advancements, such as those that helped Carr, the looming shadow of the sequestration and its deep cuts to both the National Institutes of Health (NIH) and National Cancer Institute (NCI) budgets dominated much of the discussion. Jon Retzlaff, managing director of AACR’s Office of Science Policy and Government Affairs, pointed out that “while the federal government has made large and lasting commitments to cancer research in the past, we are concerned that this commitment has been eroding for some time and is about to suffer a massive blow.”

To put this in perspective, Douglas Lowy, M.D., deputy director of NCI, noted that federal support is critical if we are to keep vital programs moving. The National Lung Screening Trial (NLST), developed by NCI, is a prime example. In this trial, a pool of more than 50,000 patients with a high risk of developing lung cancer went through a randomized trial of helical computerized topography scanning multiple times a year for seven years. This new type of scan caused a 20 percent reduction in mortality over a control group that just received a chest X-ray.

Additionally, Lowy pointed out the limitations NCI is facing. The FY12 budget of NCI was $5.07 billion, but a majority of funds are already committed to ongoing activities. In FY12, the NCI funded fewer than 15 percent of new grant applications. This flat funding, along with the uncertainty regarding the sequester in particular and Congress’ questionable commitment to future research funding in general, is causing a lot of reluctance within NCI to start up new large-scale projects such as the NLST.

Anna Barker, Ph.D., director of Transformative Healthcare Networks at Arizona State University and the former deputy director of the NCI, highlighted much of the hope that is being generated because of exciting new developments, such as the relatively inexpensive cost of gene mapping. It really “is a new day in cancer research,” she said. She described an exciting and ambitious community research project sponsored by NCI and the National Human Genome Research Institute called The Cancer Genome Atlas (TCGA). TCGA aims to analyze the genomic changes in a large number of cancers to discover the spectrum of genes implicated in each form of cancer, and to learn how specific combinations of genes work together in the cancer. This information could then be used to suggest new uses for existing drugs and development of new drugs. A project of this size and scope can only be done by the federal government. While expensive, the cost of cancer itself is much more profound, costing this country approximately $250 billion per year, and expected to rise significantly in the coming years.

To take advantage of the exciting discoveries being made in cancer research and treatment, panelists agreed that it is crucial to increase our nation’s commitment to medical research funding.

To conclude the meeting, Retzlaff previewed the Rally forMedical Research, to be held April 8, designed to reinforce the messages delivered at this briefing. It will be held in conjunction with AACR’s annual meeting.  

The IOM wants to know how you feel about CTSAs (say what?)

By Kristin Schneeman, Program Director, FasterCures

Have you ever heard of a CTSA?  How about NCATS?  Does this feel a little like a word game – how many acronyms can you make out of these letters?

CTSAs are Clinical and Translational Science Awards, a significant program at the National Institutes of Health to fund – you guessed it – translational and clinical science and resources (as distinct from the more basic discovery science NIH has historically supported) at the nation’s most prominent academic research institutions, 60 in all, across the entire country. NCATS is the National Center for Advancing Translational Science, the newest Center at NIH which was created to “catalyze the generation of innovative methods and technologies” to support the translation of promising basic discoveries into new diagnostics and therapeutics for patients.

Last fall, NCATS asked the Institute of Medicine to conduct a review of the CTSA program, which at $461 million is the largest component of NCATS’ $575 million budget and its assets. I attended the most recent committee meeting at the end of January, which focused to a significant degree on the CTSAs’ “community engagement.” There was not much clarity about what “communities” were supposed to be engaged – in most cases they seem to be the geographic communities in which the CTSAs are located, but in some cases they seem to be the patient communities that researchers are interested in studying, which may or may not be local. Community engagement activities and research were in previous years required components of CTSA applications but are no longer; that was the source of some consternation at the meeting, with speakers lamenting that relationships painstakingly built over the course of the first five plus years of the program were at risk (and that new ones would not be a priority) if they aren’t a requirement of funding. Among the most eloquent speakers was Bray Patrick-Lake of the Clinical Trials Transformation Initiative, herself a patient advocate, who said trust and relationship-building are long-term endeavors, and that academic institutions need to invest in patient groups and “not just come running when you need them for a grant.”

Also notable at this meeting was the lack of focus on what might be done with the CTSAs as a network, as opposed to how the individual institutions are progressing and evolving. This does seem to have been taken up at previous IOM meetings as part of this review, but it was markedly absent during the discussions about future directions for the program that wrapped up this particular day. NCATS itself has made a principle for the next stage of the program that “the whole must be greater than the sum of its parts,” and many observers believe that great things could be achieved by connecting all these major institutions and allowing stakeholders both internal and external to better understand and access the expertise and resources that reside in them. There is a relatively new Coordinating Center which has begun working on developing shared resources such as IRBshare, Contractshare, and the Research Electronic Data Capture (REDCap) platform, which could benefit external stakeholders as well as the CTSAs themselves.

The IOM is accepting comments from the public about the CTSAs until March 1st.  We encourage you to take a look at the questions and to consider commenting.  Even if you aren’t familiar with the CTSAs, that might be worthy of comment in and of itself. Here are a few of the questions we’re focused on:

• Are potential stakeholders aware of the resources available through the CTSA Program and are there barriers to use of those resources?
• Have the CTSA institutions, individually and collectively, played an appropriate and adequate role in involving and interacting with community organizations and patient advocacy groups? 
• Does the balance of CTSA Program efforts across the continuum of research from first phase studies in humans to clinical trials to population-based research on health outcomes and comparative effectiveness need to shift?
• What do you see as successes, challenges, and future directions of the CTSA Program?

Time=Lives Story of the Week: The Hempel Family

“When we realized our kids had a fatal cholesterol disorder we turned to some of the foundations and the researchers to figure out what is the therapy pipeline. What we realized was that that therapy pipeline really wasn’t built.” - Chris Hempel, mother of Addi and Cassi

At age three, identical twins Addi and Cassi (Addison and Cassidy) Hempel were diagnosed with an ultra-rare and fatal cholesterol disease that affects only 500 people worldwide – Niemann Pick Type C – a condition frequently referred to as “Childhood Alzheimer’s.” 

Their parents, Chris and Hugh, noticed a drastic change in their girls and immediately started taking the necessary steps to find a cure. By becoming experts in the disease, advocating for treatment options, and not taking no for an answer, the Hempels have made medical progress happen.

 

 Watch the Hempel’s Story Here

In April of 2009, after months of work by Chris and Hugh to set up a “virtual biotech” in their home, the U.S. Food and Drug Administration (FDA) granted special permission to allow the girl’s doctor to give them intravenous infusions of 2-hydroxypropyl-β-cyclodextrin, a non-toxic sugar compound. In October 2011, the FDA granted permission to allow intratehcal injections of cyclodextrin into their spines to enable the compound to reach their brains. 

Now the family is working on a permanent solution to treat the girls, now 7. Their story is chronicled in the new documentary Here.Us.Now, a film commissioned by the Ewing Marion Kauffman Foundation.

February 25 – March 1, 2013 is Rare Disease Week, where advocates for illnesses such as Niemann Pick Type C (which affects approximately 250 to 500 people in the US) come together to bring widespread recognition of rare diseases as a global health challenge. In the U.S., any disease affecting fewer than 200,000 people is considered rare. There are nearly 7,000 rare diseases affecting nearly 30 million Americans, which means almost one in ten Americans are suffering from rare diseases.

We’re looking forward to seeing Hear. Us. Now. at RDLA’s 3rdAnnual Rare Disease Cocktail Reception & Movie Screening this evening.  Following the movie, our own Margaret Anderson will be leading a panel discussion with Dr. Christopher Austin, Director of the National Center for Advancing Translational Sciences at the National Institutes of Health; Dr. Emil Kakkis, MD, PhD, President of the EveryLife Foundation for Rare Diseases; Marc Boutin, Executive Vice President & Chief Operating Officer,  National HealthCouncil; and Chris & Hugh Hempel.

See more stories about the power and promise of medical research, and tell us why medical research matters to you.

-- VISIT the campaign Web site
-- LIKE the Facebook page
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Relevant Links:

-- Addi and Cassi Fund

A perfect storm brewing over science

A dynamic panel of experts painted a vivid picture of the tsunami of budget and fiscal issues that threaten federal science programs during a FasterCures Webinar on “The 113th Congress and Medical Research: A Perfect Storm Approaching?” moderated by FasterCures Executive Director Margaret Anderson. While the prospects for avoiding widespread cuts seem bleak, the speakers remained optimistic that a loud, unified, focused argument from the medical research community could have an impact right now.

Sudip Parikh, vice president and general manager of Battelle Health & Analytics and a former senior staffer on the Senate Appropriations Committee, walked participants through how the budget process is supposed to work and how it’s currently working – or not working, as the case may be. An across-the-board spending cut or “sequester” is set to take place on March 1, the government is operating on a continuing resolution set to expire at the end of March, the government could shut down if that is not resolved, and the national debt ceiling looms once again in mid-May. The budget process is about setting priorities, Parikh said, and the current breakdown is not only in the budget process but also in priority-setting. Priorities within the Labor-HHS appropriation, for example, which includes the National Institutes of Health (NIH), are competing head-to-head this year – medical research, Title I education spending, Pell grants – and they can’t all win. He reminded listeners that this is a long-term discussion and that spending levels set now will set the stage for years to come.

Carrie Wolinetz, associate vice president for federal relations at the Association of American Universities, as well as president of United for Medical Research, took participants on a tour of the members of Congress involved in decisionmaking about all these budget matters, including the leadership of appropriations subcommittees with responsibility for NIH’s and the Food and Drug Administration’s (FDA) budgets as well as the Senate and House leadership responsible for overall budget negotiations. While decisions will be made at the end of the day by those top negotiators, they do still rely on input from the committees. Many of these members can be considered strong supporters of biomedical research, but in this year what has been historically a bipartisan priority is being consumed by the macro-level fiscal arguments. Still, Wolinetz argued that it is critical to continue to cultivate champions, including new ones such as Representatives Kevin Yoder, Ed Markey, Eric Cantor, and Steve Stivers, to replace old friends like Arlen Specter, John Porter, and Tom Harkin who are gone or will soon retire.

Ceci Connolly, managing director of PricewaterhouseCoopers’ (PwC) Health Research Institute and former Washington Post journalist, gave participants a flavor of the political environment in which budget negotiations are taking place this year. In addition to the unusual confluence of the budget, the sequester, and the debt ceiling, this is the beginning of President Obama’s second term, and Connolly noted that second terms can be a “double-edged sword.” On the one hand, they can liberate an Administration to take risks; on the other hand, they can cause the Administration to over-reach. And presidents don’t really have four years in which to accomplish their goals; they really have 12-18 months before attention turns to the next election. The Obama Administration, she said, is trying to get past the fiscal issues to what they consider their potential legacy issues of immigration reform and gun control. The advice she extended to medical research advocates was to focus on the compelling argument that medical research creates jobs and leverages significant economic activity, noting that in a recent PwC survey, 69 percent of Americans agreed that biomedical research is important to economic growth. She also recommended advocates expand the community, and build new alliances and relationships with others who benefit from research investments, including industry.

Parikh urged advocates to come up with an “ask” that’s clear and common across all stakeholders. “This isn’t just about the next three months; we need to set up for the next two years. The Fiscal Year 14 process is underway.”

FasterCures’ Sequestration Station and United for Medical Research’s Web site provide information and resources about sequestration and its impacts on research and the economy.

View an archive of this Webinar here.

Time=Lives Story of the Week: Michael Kaplan

“I’ve been a Type 1 diabetic since I was 12, so 31 years; and HIV positive for 20 years this March. I’ve been able to maintain good health, keep my viral load down, my t-cells up, which has allowed me to do the work I do.” – Michael Kaplan, President & CEO of AIDS United

It has been over thirty years since the emergence of the HIV/AIDS epidemic and thanks to incredible advancements in science, a diagnosis once tantamount to a death sentence is today managed in much the same way as a chronic disease. Great strides have been made in reducing the burden of HIV/AIDS, but this is a war still in progress.

A leading advocate for HIV/AIDS patients, policy, and research, Michael Kaplan is the president and CEO of AIDS United.  Born out of the merger of the National AIDS Fund and AIDS Action in late 2010, AIDS United’s mission is to end the AIDS epidemic in the United States by combining strategic grantmaking and capacity-building with national advocacy to ensure access to life-saving HIV/AIDS therapies and services, and advance key policy initiatives.

View Michael's Story Here

“As the successes of early treatment towards prevention merge with health care reform across the U.S.,” says Kaplan” the light at the end of the tunnel is only getting brighter.  I truly believe I’ll see the end of this epidemic in my lifetime."

Kaplan advocates for early testing and treatment as a way to dramatically decrease the number of new HIV/AIDS cases and help those already infected to start managing their illness as soon as possible. Like many currently incurable diseases, access to treatment is crucial. NIH research shows that the right drug cocktail can reduce a person’s ability to spread the virus to another by up to 96 percent.

Medical research discoveries from several fields have helped create many of the current medications HIV/AIDS patients depends upon today. Just as in the past, “fundamental basic research that is being done now is going to lead to things ten or fifteen years from now that we cannot predict,” said Dr. Anthony Fauci at a FasterCures' 2012 Celebration of Science event.

But with funding for research across all diseases at risk because of sequestration – looming, across-the-board budget cuts – support for the work of advocates like Michael and the science he helps to advance is more important than ever. Saving time in medical research means saving lives.  

See more stories about the power and promise of medical research, and tell us why medical research matters to you.

-- VISIT the campaign Web site
-- LIKE the Facebook page
-- TWEET with us at #TimeEqLives
-- DOWNLOAD and SHARE the Message
-- TELL us your story

Relevant Links:
-- Back to Basics: HIV/AIDS Advocacy as a Model for Catalyzing Change

Time=Lives Story of the Week: Derrick and Meredith Day

“I think I’m the same, except I just can’t see.” 

– Derrick Day, 6 years old

Derrick (6) and Meredith’s (4) story came to Time=Lives via the Foundation Fighting Blindness, an organization dedicated to driving research to prevent, treat and cure people affected by retinal degenerative diseases.

View Derrick & Meredith’s story here.

Born legally blind, the siblings suffer from a rare inherited eye disease called Leber’s Congenital Amaurosis, a disorder that can also create central nervous system abnormalities.

Seemingly simple tasks for a sight-abled person become difficult hurdles or even impossibilities when disease has robbed someone of their sight. Derrick’s dad dreams of a day when his son will be able to drive a car. “I never thought about Derrick’s not being able to drive,” said his mom. “When we got to the stop sign, he presented the question to me: Mom, how are we going to make that sign braille so that I can drive?”

Whether it’s as simple as experiencing a rainbow, or as complex as driving a car, the only way for kids like Derrick and Meredith to be able to achieve these seemingly insurmountable tasks is to be able to see. “And the only way that we can have them see,” says their mom, “is with research, and by funding scientists to find a cure.”

The good news is that retinal disease science is advancing. For example, just yesterday the Foundation Fighting Blindness reported that the Argus II retinal prosthesis, a device that can restore some vision to people who are blind from advanced retinitis pigmentosa (RP), received U.S. market approval from the Food and Drug Administration (FDA). More than 20 years of research went into the development of Argus II, with early support from the Foundation.

Also, earlier this month The Washington University School of Medicine created an innovative method for treating vision-robbing diseases using genetic reprogramming. And while their research is at an early stage in mice, it has revealed valuable clues about how to potentially save vision in people.

But promising research like Washington University is conducting wouldn’t be possible without federal funding.  And with sequestration – mandatory, across-the-board budget cuts – looming, the researchers who study diseases like Leber’s Congenital Amaurosis and others could face debilitating cuts to their research programs that would, at best, slow innovation and progress, and at worst, cause entire labs to shut down.

Want to know more about sequestration? Visit FasterCures’ Sequestration Station for facts, forecasts, and ways to add your voice to the fight for funds. Medical research matters, and must be supported. Derrick, Meredith, and millions of others suffering from debilitating diseases are counting on it.

See more stories about the power and promise of medical research, and tell us why medical research matters to you.

Here's how to get involved:

-- VISIT the campaign Web site
-- LIKE the Facebook page
-- TWEET with us at #TimeEqLives
-- DOWNLOAD and SHARE the Message
-- TELL us your story

The World According to BIO CEOs

By Kristin Schneeman. Program Director, FasterCures

We look forward every year to attending the BIO CEO & Investor Conference in New York in February, because it gives us a window into what the issues of interest and concern to the biotech community are, as we’re planning our own activities for the year.

Here are a few nuggets picked up at this year’s conference:

• Reimbursement continues to be a key and thorny issue – and not everyone in the industry is dealing with it.  Companies and deal-makers noted that even in early-stage programs, commercial considerations are “at the table,” and that today you need to have not only a biological and clinical hypothesis in early research but a commercial hypothesis as well.  However, almost immediately after a fascinating panel discussion about “Reimbursement in an ACA World,” which catalogued some of the fundamental ways in which the world of not just healthcare but research is changing, a panel of very accomplished investors was asked how reimbursement factored into their world view right now – and they all fell back on the conventional wisdom that “truly innovative products will always get paid for.” One investor in a later panel mentioned that “the Street is ‘catching on’ to reimbursement” – just catching on?


• Everyone seems to love the FDA this year.  Investors described the regulatory environment as “favorable,” friendlier,” and companies praised the new accelerated approval and breakthrough designation initiatives – while noting that they remain something of a “black box.”  


• Rare diseases are hot, with companies and investors.  They see that these products can command high prices.  Personalized medicine is causing common diseases to be redefined as subtypes that in many cases could be considered “rare,” and clearly companies were embracing this line of thinking.  Hepatitis C, on everyone’s lips last year, was almost nowhere to be found – instead we heard about multiple myeloma, lysosomal storage disorders, and “genetically defined cancers.”  This is great news for patients with rare diseases, but how long will the fad last?  What happens to patients then?  And, as one speaker noted, “just having orphan designation isn’t a get-out-of-jail-free card on pricing anymore.”


• China is attracting not just big pharma companies but smaller biotechs as well – and not only to take advantage of cheaper gene sequencing or clinical research capacity, but to manufacture and sell products as well as to raise capital.  While doing business in China is not an easy row to hoe, you could hear the excitement of the panelists talking about this new frontier for U.S. companies.  Many U.S.-educated Chinese scientists and businesspeople are returning home to make their careers.  Will development of the academic infrastructure to fuel homegrown science and companies be far behind? 


• Sadly, there was almost no discussion about the impact of impending budget cuts on NIH, which fuels much of the science these companies thrive on, or FDA, which must have the capacity to regulate and approve their products. Their own user fees, which go directly to FDA review of their products, are threatened with sequester – where is the outcry?  Besides a promising few talking about the value of these agencies, the policy agenda seemed largely focused on repealing IPAB, the Independent Payment Advisory Board, from the Affordable Care Act.  

As always, an informative and thought-provoking two days, interspersed with fascinating glimpses of the products coming down the pipelines of dozens of companies.

Relevant postings:
--- Medical research delivers cures, saves lives and – oh, by the way – is pretty great for our economy too
--- Cutting-Edge Science, Collaboration, and Sustained Funding Needed to Get New Medicines from Lab to Patient
--- The Next Big Thing In Biotech: BIO CEO Conference
--- Are Drugs for Ultra-Rare Diseases the Future of Biotech? Francois Nader of NPS Weighs In

Medical research delivers cures, saves lives and – oh, by the way – is pretty great for our economy too

Over the past few weeks, Washington D.C. has been abuzz with conversation about the value of medical research to our national economy, collective health, and global competitiveness. Leading up to our own Capitol Hill briefing with Friends of Cancer Research last Wednesday – “The Blueprint of Medical Research: How New Medicines Get from the Lab to the Patient” – FasterCures has attended several events focused on the impact of research and research funding on our nation’s well-being.

We’ve heard unequivocally that scientific opportunity – particularly when it comes to our understanding of disease biology – has never been greater, but the outlook for funding has also never been more worrisome.  If we don’t increase our investment in medical research, we’re effectively cutting it, risking not only a major engine that fuels our economy, but also the lives of hundreds of thousands of people living with diseases that have few or no meaningful treatment options.

The budgetary decisions we make today are decisions that won’t just have an impact in our lifetimes, but will alter the course of the next generation of patients, family members, and young scientists.  Here are some of the things we’ve heard around town: 

• “If scientists can take a few minutes to put down their microscopes and pick up a microphone it will make a real difference.  As we stand at this budget crossroads, we need your help to make the case.”
• Congressman Ed Markey (D-MA) at American Cancer Society’s Cancer Action Network briefing on Capitol Hill, February 5, 2013

• “The FDA is really a bargain when you look at scope of activities that have exploded without parallel budget increases. FDA costs only about $8 per American per year, and we are determined to maximize the dollars that we have. Drugs are available in the U.S. faster than anywhere else in the world, and with 39 new approvals in 2012, including a number of new advances in personalized medicine, we are making real progress. Sequestration would result in a significant reduction of domestic and international inspections, which makes for a tough environment in which to recruit and retain top people. We cannot walk away from our responsibilities."
• U.S. Food and Drug Administration (FDA) Commissioner Margaret Hamburg at Alliance for a Stronger FDA meeting, February 5, 2013

• The average per capita National Institutes of Health investment is just under $100/citizen/year, and less than 1% of federal budget. Yet these investments are having an outsized impact on our economy and health. The more stories that scientists and patients can tell in their own words, the more likely it is that the message will resonate with our policymakers. 
• National Institute of Health’s Scientific Management and Review Board (SMRB) discussion from January 14, 2013 board meeting

• “We have come so far in cancer science, especially in my field of immunotherapy. We now live in an age where it is possible to take the army in own bodies, and train it to fight against our particular disease. But scientists like me would not be where we are if not for the investments that were made in us at the start of our careers.  If the budget of the NIH is cut any further we will be faced with having to tell the patients who rely on our science: I’m sorry, there’s nothing more we can do.  No one wants to be the giver or receiver of this heartbreaking message.”
• Helen Sabzevari, global head of oncology-immunotherapy for EMD Serono Inc. at American Cancer Society’s Cancer Action Network briefing on Capitol Hill, February 5, 2013

• "The National Institutes of Health (NIH) supported more than 402,000 jobs and $57.8 billion in economic output nationwide in 2012 alone. If Congress fails to prevent the 5.1 percent automatic, across-the-board spending cuts slated for March 1, the nation's life sciences sector could lose 20,500 jobs and $3 billion in economic input."
• Feb. 6 statement by United for Medical Research 

Currently, only five cents of every U.S. health dollar goes to biomedical research; however, 1 in every 3 Americans is living with a deadly or debilitating disease for which there are no cures. The U.S. needs strong investment in biomedical research to deliver cures, save lives, and maintain our global competitiveness. There’s no time to waste.

Relevant links:
-- ACS CAN Report Highlights Critical Need for Sustained Funding for Cancer Research
-- Videocast of Jan. 14 SMRB meeting
-- The State of the FDA—February 2013
-- Report from United for Medical Research about the Economic Impact of Sequester on Life Sciences Sector
-- Sequestration Station
-- Time=Lives

Cutting-Edge Science, Collaboration, and Sustained Funding Needed to Get New Medicines from Lab to Patient

FasterCures and Friends of Cancer Research Host Capitol Hill Briefing

“While I can tell you there’s never been a more exciting time for science, I can also tell you there’s never been a more stressful time,” said Francis Collins, director of the National Institutes of Health (NIH), addressing the issue at hand – tightening fiscal resources that threaten the research and development ecosystem to fully deliver science’s full potential to improve health and well-being. This was the main topic of a Capitol Hill briefing co-hosted by FasterCures and Friends of Cancer Research.

Moderated by FasterCures Executive Director Margaret Anderson, Collins and other medical research leaders representing distinct sectors provided a glimpse into what it takes to turn a scientific discovery into a safe and effective therapy that will improve, and maybe even save, patients’ lives. More than 300 policy decision makers, advocates, and key legislative staffers participated.

“There is no one in this room who hasn’t been touched by disease, whether it’s yourself or a loved one,” said Ellen Sigal, chair and founder of Friends of Cancer Research, opening the discussion. “We cannot let the FDA or NIH become a victim of political polarization.” The discussion kicked off with video highlights from a viral campaign, Time=Lives, that featured images and stories of patients, families, researchers, and executives, reminding everyone in the room about what’s at stake.

Onyx CEO Anthony Coles explains impact
of public investment on drug development 

Underscoring the role of public investment in basic research by the NIH, N. Anthony Coles, president and CEO of Onyx Pharmaceuticals, noted that pharmaceutical companies depend solely on this research to understand biology and disease origins. “Companies like Onyx then take these findings, advance the research, and move it toward delivery to patients,” he said.

Improving and accelerating research and development requires all stakeholders in the medical research ecosystem to work together – including federal agencies, industry, academia, and patient groups. Collaboration – existing collaborative efforts, and the need for even more partnerships – was a resounding theme throughout the discussion.

Disease research organizations feel the excitement for an improved system. “This is the most robust pipeline we’ve seen. Now is not the time to step back from working together, but to do more,” said Deborah Brooks, co-founder of the Michael J. Fox Foundation for Parkinson’s Research.

“The FDA is the final common pathway to translating science to patients,” said Margaret Hamburg, commissioner of the U.S. Food and Drug Administration (FDA). “If the FDA is not fully funded and supported, the ecosystem will not function optimally. When we engage early with the scientific community, we’re able to cut five years off of the drug development process.”

Roy Jensen, director of the University of Kansas Cancer Center, is already seeing how limited resources are impacting research progress. “We’re not even funding a fraction of the best science. We’re starting to cut to the bone – scientists are having to close labs, they aren’t able to train the next generation… It’s fundamentally altering our infrastructure,” said Jensen.

The importance of supporting the next generation of brilliance and innovation weighs heavily on the minds at the NIH, and researchers are hopeful for the next group of young investigators. “It is very tough right now to be a grad student, or a post-doc. As they look at the landscape of this country, they wonder if there’s room for them,” Collins said.

Coles echoed Collins’ concern with a sobering concept about the future of scientific innovation: “What answers won’t we have in 10 years if we don’t fund this research?”

“The decisions we make today will have implications long into the future. Unlike delaying construction of a bridge that can be resumed in a few years, if we lose a generation of scientists, there’s no way to rebuild that human capital quickly,” said Mike Milken, founder of FasterCures and chairman of the Milken Institute. “We fully understand the need for overall budget restraint in Washington; but short-term cuts in bioscience only assure a less-healthy America down the road. Modest investments in prevention and cures today will help avoid the catastrophic costs of care tomorrow.”

* A video of this briefing is now available.

From L to R: Margaret Anderson, Roy Jensen, Deborah Brooks, Tony Coles, Ellen Sigal, Margaret Hamburg, Mike Milken, Francis Collins

Time=Lives Story of the Week: Andrew Goldstein

“The day that science doesn't get me up in the morning and make me love these questions, it’s not right for me. But, until then, I just keep doing it and keep going for the questions we don’t have answers to.”

Meet Andrew Goldstein. Like many young investigators, the prospect of new discovery and hope to transform lives and cure disease keeps him excited about science and the possibilities it can open up. In addition to being an ex pro lacrosse player, Andrew is also an assistant researcher at Jonsson Comprehensive Cancer Center at UCLA, where he focuses on developing new scientific approaches to tackling advanced prostate cancer. 

Watch Andrew’s story here

In 2010, Andrew was part of a team of scientists who identified for the first time a cell-of-origin for human prostate cancer, a discovery that could result in better predictive and diagnostics tools and the development of more effective targeted treatments for the disease. His passion and drive as both an athlete and a scientist fuel his work towards a cure. 

"Health and disease affects everybody," says Andrew. "Whether it's obesity, cancer, heart disease ... it's something that is in everybody’s life. So investing in research and understanding ‘what is the basis of disease’ is absolutely essential.”

According to the Prostate Cancer Foundation, 1 in 6 men are diagnosed with Prostate Cancer each year, and more than 30,000 lives are cut short in that same time frame because of the disease. That’s 30,000 men that don’t get more time with their families, won’t go to their granddaughter’s recital, won’t go back to work on Monday and don’t get a choice in the matter. And this is just ONE disease.

Science has never been more promising, but the outlook for funding for young investigators like Andrew – the future of the medical research enterprise – has never been more worrisome. Learn what you can do to help make medical research a national priority by visiting Time=Lives.

See more stories like Andrew’s about the power and promise of medical research, and tell us why it matters to you. Here's how to get involved: 

-- VISIT the Time=Lives campaign Web site
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Relevant Links
* Facts about prostate cancer
* The struggle to employ young investigators