Category Archives: Basic Research/Basic Science

Member Spotlight: United Therapeutics Corporation

By Martine Rothblatt, PhD, Chairman and CEO of United Therapeutics Corporation.

MRothblatt Official photo 2#59583CFounded in 1996, United Therapeutics Corporation is a biotechnology company focused on the development and commercialization of unique products to address the unmet medical needs of patients with chronic and life-threatening conditions. We have four approved products on the market today and we are not stopping there! From the United States to Europe to the Asia Pacific, we are proud of our multicultural business environment where employees can collaborate with people all over the world. As a group, we are relentless in our pursuit of “medicines for life”® and continue our research into treatments for pulmonary arterial hypertension, cancer, and some of the world’s most complicated viral illnesses.

We are proud to partner with Research!America to promote better medical advancements, biomedical research, and overall greater global health initiatives.  We have seen first-hand how tireless research and dedication to a cause can change the lives of thousands of patients and their loved ones. We began our story by conducting extensive research on a treatment for a deadly disease so rare, Pulmonary Arterial Hypertension (PAH), that other medical companies had abandoned any pursuits for treatments or a cure. Continue reading →

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A Wish List for the 114th Congress to Consider

By Caleph B. Wilson, Ph.D., a biomedical researcher at the University of Pennsylvania, logistics director of the National Science Policy Group, science communicator and STEM outreach advocate. Follow him on Twitter as @HeyDrWilson.

CalephWith the 114th Congress underway, the scientific community is looking forward to sharing new research breakthroughs and advocating for STEM during a series of congressional visits to Capitol Hill. In some instances, scientists and trainees will assist writing congressional briefs and give testimony to House and Senate committees on science, technology and health.

While Congress is considering science policy initiatives, positions and funding, there are a few things in the early-career scientist “wish list” that would make improvements and maintain the United States’ leading position in the scientific enterprise.

Throughout 2014, early-career scientists discussed specific issues in science policy groups, on social media and in articles that need to be addressed. These are some of the highlights of the conversations that have been put in a “Wish List” that hopefully Congress and policymakers will strongly consider.

  • National Science Foundation (NSF) and National Institutes of Health (NIH) funding that is predictable and keeps pace with inflation.

In the early 1990s, the NIH budget increased dramatically. However, over the last 10 years the NIH budget has flat-lined and even decreased at times. Unfortunately, the budget has not kept pace with inflation and rising costs of executing experiments. With changes in the economy and the sweeping budget cuts that came in with sequestration, government agencies, institutions and investigators can better plan with predictable budget appropriations that keep pace with scientific opportunity. Continue reading →

A Weekly Advocacy Message from Mary Woolley: Innovation today doesn’t guarantee innovation tomorrow

Dear Research Advocate:

Research!America yesterday released our recommendations for the top five science priorities the new Congress should address in its first 100 days: end sequestration, increase funding for our nation’s research agencies, advance the 21st Century Cures Initiative legislation, repeal the medical device tax, and enact a permanent and enhanced R&D tax credit. See the full press release here. Among these priorities, ending sequestration is the steepest uphill climb – but what a difference it would make for the future of health and the nation’s economy! That’s the focus of this editable message to members of Congress. Please weigh in!

Securing meaningful increases in funding for our federal research agencies will take the same kind of leadership and bipartisan commitment that propelled the FY98 – FY03 doubling of the NIH budget. A recent CQ Healthbeat interview with Congressman Tom Cole (R-Okla.), the new chairman of the House Appropriations “Labor-H” Subcommittee, suggests there is reason to hope for just that kind of momentum. During his discussion with CQ Healthbeat reporter Kerry Young, Chairman Cole indicated that he plans to establish an aggressive hearing schedule, with the goal of facilitating the bipartisanship that was long the hallmark of the Appropriations Committee. He said: “If we talk enough, maybe we find some common ground and some unusual alliances and some places where instead of being at one another’s throat, we can actually work together …” Cue research to save lives and combat disability.

Fareed Zakaria writes in the Washington Post that, “federal funding for basic research and technology should be utterly uncontroversial,” and I couldn’t agree more. However, robust federal funding is only a part of the equation. Tax and other policy reform is crucial to the vitality of domestic innovation. In his op-ed, Fareed identifies troubles faced by American start-up companies, with their number trending down alarmingly as they face so many barriers to entry. He notes that yes, American innovation is still a wonder of the world, but it is becoming less and less unique. Innovation today doesn’t guarantee innovation tomorrow. Success in both the public and private sectors relies on updating of creaky national policies to reflect the excitement and potential of 21st century science and technology.

Finally, an issue where change is crucial, but the path to it uncertain: how to prevent the discouragement and flight of still more young would-be super-scientists. Johns Hopkins University president Ronald J. Daniels takes on this issue in the most recent edition of the Proceedings of the National Academy of Sciences (see press release here). As he explains, increased competition for grant funding and fewer faculty jobs could well choke off the pipeline of young scientists needed to maintain our nation’s research capacity. Daniels’ perspective is an important contribution to a profoundly complex issue that cries out for resolution. It is likely to be on the 21st Century Cures agenda and receive considerable attention during the aforementioned Labor-H hearings. It would serve the research community well for advocates to come to consensus on solutions rather than wait for solutions to be imposed without their input.

We have a lot of work cut out for us, stakeholders in science and lawmakers alike. But at the end of the day, we are all working in the public’s interest – a starting point for agreement even when we might seem miles apart.

Sincerely,

Mary Woolley

World AIDS Day 2014: Focus, Partner, Achieve: An AIDS-free Generation

World AIDS day, commemorated each year on Dec 1, aims to raise awareness about the virus, encourage advocates to redouble efforts to fight the epidemic, and remember those who have died and continue to suffer from the disease.

Photo credit: cdc.gov

Photo credit: cdc.gov

The 2014 World AIDS day theme “Focus, Partner, Achieve: An AIDS-free Generation,” speaks to how combined efforts and collaborations can bring us closer to a cure or vaccine. For example, public and private-sector funded research led to the development of Highly Active Antiretroviral Therapy (HAART), which revolutionized the battle against HIV/AIDS according to Research!America’s HIV/AIDS fact sheet.

Medical research has played a critical role in reducing the risk of transmission and has led to new drugs that have transformed HIV/AIDS from a fatal to a chronic illness for millions worldwide. Patients like Maria Davis, professional entertainer and HIV/AIDS advocate, has benefited from advances in HIV/AIDS treatments.

Research!America member, amfAR, The Foundation for AIDS research and The Centers for Disease Control and Prevention are raising awareness on World AIDS Day by providing up-to-date resources and information describing the human and economic impact of HIV/AIDS. In FY14, U.S. federal funding to combat HIV/AIDS here and abroad and assist those affected by the disease totaled $29.5 billion, but more resources and funding are needed to tackle this global epidemic. Tell Congress that we need more funding for HIV/AIDS research today!

To find out more about the events happening on Dec. 1, visit http://aids.gov/

A Weekly Advocacy Message from Mary Woolley: Deep thoughts occasioned by ice buckets

Dear Research Advocate: 
 
You have by now heard about the ALS “ice bucket” challenge (show support for ALS research by dumping a bucket of ice water over your head and/or writing a check for $100 to the ALS Association, then challenge three others to do the same.)  Whether viewed as a welcome late-summer distraction from imponderables like conflict in the Middle East, on-going clashes in Ferguson, Mo., or the mounting death toll from Ebola, or, rather, as the emergence of a new kind of advocacy similar to what produced the walks, runs and bike-rides for research that are ubiquitous today, the “ice bucket challenge” is worthy of attention.

I think that public attention to the “ice bucket” challenge is not only good for ALS research (and all the patients and their families who cope with this devastating illness), but is an opportunity to engage a newly-interested sector of the public, including all those members of Congress who have accepted the challenge. Think about those freely written $100 checks and consider that the NIH budget buys only about $100 worth of medical research per American, per year, on all diseases as well as vital basic research.  Add to that other federal agencies’ budgets, the private sectors’ expenditures (industry, academia, philanthropy, patient groups) and we can maybe triple that amount (generously computed, and including development along with research). Is that enough to assure better health and prosperity for our nation? I’d say not even close.  Not when brilliant young people are discouraged to the point of leaving the country if they want to work in science;  not when other nations are poised to take over U.S. leadership in R&D; not when we are looking at ALS heartbreak and huge federal debt associated with the costs of Alzheimer’s, as just two crises we should be focused on intently, with all the resources we can bring to bear.  Continue reading →

A Weekly Advocacy Message from Mary Woolley: Can we put a dent in the costly toll of suicide?

Dear Research Advocate: 
 
The loss of American Icon Robin Williams has riveted national attention on suicide, one of the 10 most common causes of death in the United States. Today, we are releasing our updated fact sheet on suicide that you can use when meeting with lawmakers and educating others about the impact research can have. Efforts to prevent suicide rightly draw on research findings. But progress has been painfully slow, stymied by serious gaps – partly due to severely limited funding – in the basic research base that precedes private sector development, and stymied by the equivalent of handcuffs placed on social science research.

The notion promulgated by some in the Congress that social sciences research doesn’t add enough value to merit federal funding is not just unfounded, it’s holding us back. Social sciences research saves lives. Case in point: behavioral research guided the development of a suicide intervention that was pilot tested in schools in Georgia and Connecticut and resulted in a 40% reduction in attempted suicides. It has since been implemented in schools across the country. This is just one example of social sciences research at work.

Research moves faster when patient advocates engage. This is the history of the nation’s commitment to defeating polio, to ramping up HIV/AIDS research, to prioritizing breast cancer research and women’s health research overall. Writing in the New Yorker last month, Seth Mnookin described the impact that “dedicated … well-informed families” can have in pushing progress. In his responsive letter to the editor, Peter L. Saltonstall, CEO of the National Organization for Rare Disorders, focused on the use of social media by patient groups to establish global registries, taking full advantage of abilities we didn’t have just a few years ago, and in so doing, saving lives. But there is another message here. The research community must work more closely with patient advocates in order to drive medical innovation. As one of the researchers in the Mnookin article said, “Gone are the days when we could just say, ‘We’re a cloistered community of researchers, and we alone know how to do this.’” Continue reading →

Communicating Your Science to the Non-Expert

On July 23, the Society for Neuroscience held  its first interactive webinar titled “Communicating Your Science to the Non-Expert.” During the webinar, speakers discussed how to effectively describe the health and economic impact of your research to a nonscientific audience including policymakers and the media. The presentation covers the basics of crafting an elevator speech and a question and answer session with scientists.

Watch the entire recording online or one chapter at a time:

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Medical Research: It’s about you and me

collage fact sheetResearch!America’s newest fact sheet series highlights the personal stories of medical research and the importance of increasing the NIH budget in FY15. We hope you will share these fact sheets with your representatives or congressional candidates, or take it with you on Hill or in-district visits. No one who reads these stories can doubt the significance of medical progress. A stronger investment in research is needed now more than ever!

Here are their stories:

What new discoveries are we delaying and missing when we slow the pace of medical and health research?

We’ve made progress. But the funding to sustain it is eroding.

A Weekly Advocacy Message from Mary Woolley: American values at stake

Dear Research Advocate:

My colleagues at Research!America have shared the role as author of our weekly letter during my recent sabbatical. My thanks to them for providing timely and actionable information to our wide network. As I am “re-entering” the Washington space, I have been struck by (1) the significantly worse condition of the roads — potholes everywhere, and now even sinkholes in DC! I’ve been in several global capitals this spring, including in less-developed countries, and DC doesn’t look good in comparison. Via recent domestic travels, I can attest to the poor condition of our roads nationwide, taking a toll on vehicles and our economy, while eroding public confidence in government. Public goods — like infrastructure, education and science — that we have long nurtured through steady investment cannot continue to be resource-starved without dire consequences. No wonder the American public is angry at Washington! (2) I have come back just in time to witness the appropriations process grind to a halt. The clock is ticking down toward August recess, and appropriators have a new excuse for failure to take action, i.e., the migrant children emergency. There will always be national emergencies. By definition they are unpredictable, and some are more complex than others; it nonetheless cannot be acceptable for Congress to grind to a halt when one occurs. Continue reading →

Bold Public-Private Partnership aims to AMP-lify Medical Innovation

Image credit: National Institutes of Health

Image credit: National Institutes of Health

Technological advances have paved the way for researchers to access a wealth of data about the biological cause of disease. Yet translating these discoveries into treatments remains a challenge. Promising drugs often fail in late phase clinical trials, costing time and money, and leaving patients’ lives hanging in the balance. One reason is that the right biological targets were not chosen from the start.

To improve the current model for developing new diagnostics and treatments, the National Institutes of Health (NIH) and several biopharmaceutical companies and non-profit organizations formed the Accelerating Medicines Partnership (AMP), www.nih.gov/amp.  “The good news is that recent dramatic advances in basic research are opening new windows of opportunity for therapeutics…But this challenge is beyond the scope of any one of us and it’s time to work together in new ways to increase our collective odds of success,” NIH Director Francis Collins, MD said in a press release. “We believe this partnership is an important first step and represents the most sweeping effort to date to tackle this vital issue.”  Dr. Collins will be among the distinguished panelists at the “AMP-lifying Innovation” discussion on Wednesday, June 25 at the BIO International Convention in San Diego http://convention.bio.org/  #BIO2014 Continue reading →

A Weekly Advocacy Message from Research!America: 5 by June 5

Dear Research Advocate:

Today, June 5, is a milestone in our Ask Your Candidates! (AYC!) voter education initiative. Today is the culmination of 5 by June 5, a nationwide push to encourage voters to ask their candidates about the priority of medical progress and encourage five others to do the same. There is still time for you to join us! Click here to send a message to the candidates running for House and Senate in your district. You can customize the message to include your personal reasons for supporting medical research or you can just click send on the message we’ve provided. In this case, it doesn’t just take a village, it takes a nation. Please help us reach voters in every state and every congressional district. Should accelerating medical progress be a higher national priority? If our future leaders understand that their answer to that question is truly important to Americans, perhaps they will enter office as research champions.

Last week, we shared a fact sheet about John Hudson Dilgen, a child with a debilitating and potentially deadly disease called Epidermolysis Bullosa. Medical research is about John. It is also about Carrie, a woman living with a severe form of Multiple Sclerosis. We hope you will find this fact sheet about Carrie useful in your advocacy. When we sent John’s story to Congress, the response was truly overwhelming. Carrie’s story will no doubt have the same impact.

Two articles, one in the Washington Post on June 1, and one in today’s New York Times, offer profound examples of the power of medical research. The Post article discusses accelerated approval of a new medicine that can extend life for a subset of patients with lung cancer, and the Times article describes DNA testing that led to the rapid diagnosis and successful treatment of a little boy whose life hung in the balance. Both of these stories involve precision or personalized medicine, a hallmark of modern medical progress.  Continue reading →

A Weekly Advocacy Message from Research!America: The good, the bad, and the ugly

Dear Research Advocate:

Congress continues to pay particular attention to – and make decisions bearing on – the pace of medical progress. To briefly count the ways:

The Senate Labor-HHS Appropriations subcommittee heard testimony yesterday from agency heads within HHS about the significance of health-related spending, including spending on medical and health research. Read our written testimony here.

Congressman Upton (R-MI-06), the Chair of the House Energy and Commerce Committee (which has jurisdiction over authorizing legislation for NIH, CDC, FDA and AHRQ) and Congresswoman Diana DeGette (D-CO-01), a member of the Committee, launched their 21st Century Cures initiative with a roundtable discussion focused on identifying what actions are necessary to maintain our nation’s place as the world’s innovation leader. While Reps. Upton and DeGette are champions of research who should be commended for working to strengthen U.S. medical innovation, there is always the risk that Congress will veer into micromanagement of NIH, stymie FDA’s efforts to ensure that private sector innovators are rewarded for ensuring the safety and efficacy of their medical advances, or “hold off” on providing the funding needed to accelerate medial progress until  longer-term strategies are in place. Your participation can help make this effort a success, and the initiative has established an email address you can use if you wish to give input: cures@mail.house.gov.

So that’s the good. Continue reading →

Will Medical Innovation Be an Afterthought This Election Season?

Excerpt of an op-ed by Research!America President and CEO Mary Woolley published in the Huffington Post.

mary-woolley-webLike it or not, we’re in the midst of another election season. As candidates embark on endless rounds of campaign activities to win the hearts and minds of voters, it’s critical that they not neglect, by choice or lack of awareness, a key issue that has tremendous implications for the health and prosperity of Americans: medical research and innovation.

Our nation’s research enterprise has endured years of flat federal funding and, more recently, sequestration, the destructive across-the-board federal spending cuts that began in 2013, as well as policies that slow rather than propel forward the pace of commercial innovation and drug development. Research projects are coming to a halt, patients are being denied access to clinical trials, research institutions across the country have reduced their workforce, young scientists are fleeing to other careers or other countries and local businesses that flourish in a research-rich environment are scaling back operations.

Ironically, given today’s unprecedented scientific opportunity, elected officials have not provided the resources it takes to capitalize on monies previously invested, forcing innovators to delay or abandon studies that could lead to new therapies and cures for insidious health threats such as Alzheimer’s, cancer and obesity. During this election season, as candidates answer questions and take positions intended to boost their chances at the polls, it is up to us who care about medical progress to ask candidates whether they perceive medical research as a top national priority.

Read the full op-ed here.

A Weekly Advocacy Message from Research!America: The time for medical progress is never later

Dear Research Advocate:

Innovation will be the buzzword on Capitol Hill next week as Senate appropriators meet with experts, including National Institutes of Health (NIH) Director Francis Collins and National Science Foundation (NSF) Director France Cordova, for a hearing on April 29, entitled “Driving Innovation through Federal Investments.” As Research!America noted in its written testimony, there’s actually a two-part question underlying that theme: What is the significance of innovation to Americans? And, implicitly, does the return on our investment justify current or higher levels of spending? When it comes to medical innovation, the short answers are: 1) it bears on longevity, individual well-being, deficit reduction, national security, global leadership, etc.; and 2) the economic return on investment justifies robust federal funding levels for research. Chairwoman Barbara Mikulski and committee members are embracing the power of social media to engage the public. Join the conversation using the hashtag #innovation.

Advocates for research must seize this opportunity to underscore the importance of innovation by joining our Medical Progress NOW initiative. Take a moment to email your representatives and urge them to support a substantial increase for NIH in FY15. And take a few more minutes to call your representatives (here are phone lists for the House and the Senate) and make the case to their health legislative assistants.  Here is a fact sheet that can help you frame your arguments. The Federation of American Societies for Experimental Biology (FASEB) has new data to help make the case for stronger federal support for innovation. FASEB recently updated their fact sheets highlighting the importance of NIH funding in the local economy of each state and many congressional districts. If you can go the whole nine yards — to secure $32 billion for NIH in FY15 — call, email, and visit your representatives or their state/district directors. Use social media to spread the word.

One more time-sensitive topic: Ask Your Candidates! Please consider partnering with Research!America and the terrific group of organizations who have already joined this national voter education effort. For more information, visit the Ask Your Candidates! website or shoot an email to Tim Tassa at ttassa@researchamerica.org. Since we know how important it is to distinguish this non-partisan educational initiative from a campaign activity, we would be glad to provide a fact sheet about that, as well as materials your grassroots networks can use to get involved. The sooner the better.

The time for medical progress is never later. But there are opportunities to accelerate it now. Let’s make sure they’re taken.

This week’s letter was authored by Mike Coburn, Chief Operating Officer at Research!America.

Sincerely,

Mike Coburn

Tell Congress We Need Medical Progress NOW!

The price of wasted time is too high

The Fiscal Year 2015 Appropriations process marks a period of crucial decisions on how to fund America’s top priorities, including combating deadly and disabling disease.  The funding allocated to the National Institutes of Health (NIH), coupled with private-sector investment, saves young lives; empowers those with disabilities to maintain productive, fulfilling lives; and is an underappreciated force behind local and national economic growth, our national security and America’s status as a global economic and innovation leader.  Appropriators can act this year to restore NIH to a growth path consistent with the significance of medical progress to Americans and the level of scientific opportunity.

The NIH budget is lower today than it was in 2012.  How have we fallen so far behind?  Is it no longer important to conquer diseases that kill children, to do more for wounded warriors or to stop devastating conditions like Alzheimer’s and cancer?

Congress needs to do something NOW to make up for the massive gap between the funding needed to reignite medical progress and the minimal funding allocated to this priority over the last several years. Will funding for NIH receive a real funding boost in FY15 or will medical progress continue to lose ground?

We need Congress to accelerate medical progress, not in five or ten years, but now.  Tell Congress to treat medical progress like the American priority it is and give it the boost in funding for 2015 that it needs. 2012 is our past; 2015 is our future.  Let’s keep moving.  Medical progress NOW.

Take action now.