Thursday, October 25, 2012

FasterCures launches Sequestration Station

FasterCures today launched Sequestration Station – an online destination for relevant and up-to-date news and resources about how sequestration could impact medical research.

The prospect of automatic spending cuts, or sequestration, which will take effect in January 2013 if Congress does nothing, threatens the future of U.S. leadership in medical research and development and will delay or permanently remove access to life-saving medical treatments. Sequestration would slash federal investments in critical health, scientific, medical, and biological research aimed at discovering treatments, moving safe and effective new medicines to market, and creating innovations to grow our economy.

According to a report released by the Office of Management and Budget, sequestration would have a "devastating impact" on scientific research. Here’s how this will impact two agencies critical to advancing life-saving therapies:
  • The National Institutes of Health (NIH) will face an 8.2 percent cut, resulting in a $2.518 billion loss. In addition to the discretionary cuts, an additional $150 million in mandatory budget authority (for diabetes research) will be subject to a 7.6 percent cut, equal to $11 million, making the total cut to NIH equal to $2.529 billion.
  • $3.873 billion of FDA's budget is subject to an 8.2 percent reduction (merely $67 million of the agency's budget is exempt from the sequester). That cut means that sequestration will erase $318 million from the FDA's budget. Virtually all user fees that were active in FY 12 are considered part of the sequester. Such a loss will devastate the agency, cripple its ability to do its job, and put millions of Americans at greater risk from unsafe food and drugs that come from both inside our borders and abroad
At stake: our economy, national productivity, global competitiveness, and progress against life-threatening disease. Making medical research a national priority benefits all of us.

We’re concerned and you should be, too. But there is something you can do. Contact your Members of Congress and let them know that further cuts to the NIH and FDA will set us back on the path to new therapies and cures for disease, jeopardize our economic competitiveness, and result in loss of jobs in communities throughout the country. Share with them why a strong investment in medical research is important to you, your family, and your community.

Medical research matters – we must continue to fund it.

When it comes to medical research, saving time means saving lives

That's the theme of a new social media campaign by FasterCures.

We launched Time Equals Lives to make a compelling statement about why we must invest in medical research. Time Equals Lives is a platform for personal stories, each one on its own is a strong case for why research matters, and when woven together collectively these stories create an imperative to make medical research a national priority. 
The campaign is anchored around FasterCures’ operating principle that to save lives, we must save time in medical research - the way we search for discoveries, turn these discoveries into therapies, and bring these therapies to patients. 

We are collecting stories from:
  • patients and their families whose lives have been altered by disease,
  • scientists and researchers facing incredible obstacles to advancing their work, but whose relentless efforts are bringing us closer to a cure, and 
  • impatient advocates who know too well that improving a system means disrupting it and playing an active role in reshaping it.
The Time Equals Lives campaign provides our nation with a bird’s eye view of the critical importance of medical research. It is our goal to ensure we share these stories effectively and amplify their messages to leaders and decision makers whose work can chart the course of our future well-being. Here are some of the things we look forward to doing throughout this campaign:
  1. delivering these stories in a creative and compelling ways to Members of Congress;
  2. packaging these stories and sharing them effectively with Administration officials;
  3. preparing these stories for all stakeholders in the medical research advocacy community to use as appropriate to advance their respective goals. 
We built the site in the public domain with the express purpose of making this resource available for the medical research advocacy community to use as appropriate.  
We urge you and those you know to add your stories to the mix. Here's how to get involved:

Wednesday, October 24, 2012

Start planning your Partnering for Cures experience today

The 2012 Partnering for Cures program agenda is LIVE, and packed full of useful and timely content. With 30 innovator presentations, 16 hot-button panels, 18 expert consultations, and countless 1:1 partnering sessions, there's something for everyone

Taking place Nov. 28-30 in New York City, the fourth annual Partnering for Cures meeting brings together senior leaders from across all sectors in medical research with the express purpose of making collaboration happen – collaborations that must happen if we are to speed up the time it takes to turn discoveries into treatments and cures.

Find out what leaders in medical research think about today's hottest topics. Is life sciences still a good investment? Can clinical trials be better, faster, and cheaper? What does the 2012 election mean for medical research? How can we speed translation to human trials?

This solutions-oriented meeting is all about finding partners, sharing ideas, and getting things done.

To view a full program agenda online, click here



Thursday, October 18, 2012

The Future of Alzheimer’s Research

By Shawn Sullivan, Program Associate at FasterCures 

Earlier this month, Congressmen Ed Markey and Chris Smith, co-chairs of the Congressional Alzheimer’s Task Force, together with the Cure Alzheimer's Fund, held a briefing to explain the latest in Alzheimer's and dementia research and what America must do to achieve the goal of preventing and treating Alzheimer's by 2025.

USAgainstAlzheimer’s Founder George Vradenburg started the briefing with an overview of Alzheimer’s current effects on the American population and the immense dangers that lay ahead if new treatments and therapies are not discovered in the next decade:
  • Alzheimer’s currently afflicts more than 5 million Americans, and Alzheimer’s care cost $183 billion in 2010 in the United States. These numbers will triple by 2050 with current trends.
  • For every $400 spent on Alzheimer’s care, only $1 is spent on research.
  • The president’s budget for 2013 includes $80 million for Alzheimer’s research. This is about $0.26 per American citizen.
  • Alzheimer’s is the only disease in the top 10 with no disease-modifying treatment or cure. 
Vradenburg also noted that the large number of baby-boomers entering their highest risk age over the next 10 years could make estimates about future impacts seem conservative. In particular, the effects of Alzheimer’s on Medicare costs could overwhelm an already stressed system. “If funding for research stays at current levels," he said "the chances of us finding an effective treatment before this takes place are slim to none.”

Rudy Tanzi, director of the Genetics and Aging Research Unit of the MassGeneral Institute for Neurodegenerative Disease (MIND) and Chair of the Research Consortium of the Cure Alzheimer's Fund, gave an overview of current research efforts. He started by mentioning two recent failed drug trials and how those failures have most members of the Alzheimer’s research community questioning current strategies. Tanzi is of the opinion that we are going after the right targets but with the wrong drugs and, more importantly, in the wrong patients. Researchers are coming to the conclusion that by the time a patient is showing symptoms of the disease, it may be too late to intervene. This will make forming future clinical trials all that more difficult. Tanzi also noted that researchers have cured Alzheimer’s in countless mice but that none of the results have been predictive of human response.

Tanzi pointed out that stagnant NIH funding is causing America to lose a generation of researchers who cannot gain access to the resources needed for innovative research. “My students are telling themselves that if I have to fight tooth and nail for research funding, what chance do they have?”

Philip Haydon, founder of GliaCure, Inc., then spoke about the exciting research underway at his company which he believes could lead us to new treatments. He noted that despite the promise of these discoveries, there is little interest from investors. “I am still dependent on the NIH if there is to be any translational research done with these discoveries,” he said. "However, NIH funding is becoming more difficult to obtain."

One attendee asked the panelists what effect sequestration – automatic, across-the-board federal spending cuts – would  have on Alzheimer’s research. They agreed that while the current level of Alzheimer’s research funding is inadequate, the cuts in research that would be imposed by the impending “fiscal cliff” would be devastating and could set back efforts to find new treatments for Alzheimer’s by a matter of decades, if not indefinitely.


Interested in learning more?  Come hear George Vradenburg speak about Alzheimer’s research at Partnering for Cures, Nov. 28-30, in New York. He will join a distinguished panel discussing “Rules, tools, and data pools for catalyzing drug development.”

Friday, October 5, 2012

FDA as a Global Actor

At the Center for Strategic and International Studies earlier this week, U.S. Food and Drug Administration (FDA) Commissioner Margaret Hamburg, in a discussion with J. Stephen Morrison, stressed the need for the FDA to take on more of a global approach to regulation and to collaborate with scientists, industry, and stakeholders to strengthen supply chain integrities and overall food safety.
Nothing stresses the necessity for a global approach more than these startling numbers:
  • 25 cents of every dollar spent in the U.S. goes to products regulated by FDA.
  • 80 percent of active pharmaceutical ingredients are manufactured abroad.
  • 40 percent of all drugs taken by Americans come from abroad.
  • 85 percent of seafood and close to 50 percent of fruit is imported.
  • Global production of FDA-regulated products has quadrupled over the last decade.
At the event, Hamburg noted that while “the FDA remains the gold standard for regulation, minimum thresholds for standards must be established globally” to effectively regulate today’s intertwined system of supply chains and international competitiveness.
To restructure the system effectively and build capacity, Hamburg underscored the need to:
  • move toward a global coalition of regulators;
  • create new data systems for sharing information;
  • find a strategic risk-based approach and use the best science and experts to target high-risk products and identify vulnerabilities within them; and
  • establish new partnerships with all stakeholders, including industry.
By developing new models, standards will be raised, information can be shared, and regulatory capacity will be built to underline the harmony of interests between the FDA, development agencies, and stakeholders. Embracing our global reality will enable key sector leaders to better work toward the goals of stronger economics, improved infrastructure, a stronger regulatory capacity, and, most importantly, improved health and safety regulations that impact the everyday lives of citizens worldwide.

Wednesday, October 3, 2012

Celebrating Science: Making Science Cool

This is the third in a series of blogs highlighting key themes and outcomes from FasterCures’ Celebration of Science, which brought together over 1,000 scientists, educators, industry executives, policymakers, and patient advocates to celebrate the scientific achievements of the last 20 years and jumpstart a new wave of discovery.

At the recent Celebration of Science, Earvin “Magic” Johnson said one of the keys to inspiring the next generation of scientists is “to keep making it cool for the kids.” Throughout the weekend celebration, leaders in science, policy, and business touted the importance of encouraging young people to go into science, technology, engineering, and mathematics (STEM) fields, and making sure there are positions available for them after their education.

Panelist after panelist shared remarkable advances that are making this such an exciting era of scientific discovery (just check out DARPA’s cheetah robot). However, when young scientists complete their studies, they seem to have a hard time breaking into academic careers – only 14 percent of those who graduate with a doctoral degree in biology or life sciences attain an academic position within five years. As Steven Chu, Nobel laureate and United States Secretary of Energy, put it, “You can’t just educate people; you have to make sure they have jobs.” 

The average age for a scientist to receive his or her first NIH grant is 42, which is more evidence that young people are facing barriers. It takes a lot of motivation for a young scientist to stay inspired that long before they see their job turn into a career.

Budding scientists who are being inspired by their high school chemistry teachers today need to be confident that this field will have opportunities for them tomorrow. “The most important thing we can do is capture the imaginations of the next generation,” said Eric Lander, president, Broad Institute of Harvard and MIT, and co-chair, President's Council of Advisors on Science & Technology.

Encouraging the next generation of researchers was a theme throughout the Celebration of Science, and it was the focus of the panel “STEM Education and Job Prospects for Young Investigators.” The panelists discussed the importance of internships, networking, diversifying the field of science, and the need for adequate funding. No one wants to hop on a sinking ship, much less aspire to be its captain.

That is why the Celebration took every opportunity to honor young scientists (a future Nobel laureate could be pictured at the left). As FasterCures Chairman Michael Milken said, “This is the century of bioscience. Problems that don’t appear to be solvable will be solved by bioscience. To do all of this you need talented people.” By renewing our commitment to science, we can ensure that those talented young people have opportunities in the laboratories and at patients’ bedsides.

Kids idolize sports heroes, who are invited to the White House when they win championships. The Celebration of Science honored scientists and patients who are the heroes to millions by discovering effective treatments for debilitating diseases and inspiring others with their stories of survival. Although they may not be household names, they showed how cool science can be.
And what could be cooler than the Rock Docs?

Celebrating Science: Uncovering the Mysteries of the Brain

This is the second in a series of blogs highlighting key themes and outcomes from FasterCures’ Celebration of Science, which brought together over 1,000 scientists, educators, industry executives, policymakers, and patient advocates to celebrate the scientific achievements of the last 20 years and jumpstart a new wave of discovery.

The brain dictates when to breathe, when to blink, the way our voice sounds, how we remember our loved ones, and all of the other everyday functions we easily complete without consideration. But what happens when your brain begins to let you down? When disease takes over and starts to break down your body’s ability to communicate with itself?

One of the themes at FasterCures’ Celebration of Science (COS) was the need for progress in how we understand, treat and cure brain disorders. Among the participants at this unique event – which aimed to raise the profile of science and research on the national agenda – were neurodegenerative disease researchers, patients and caregivers dealing with a range of disorders from Alzheimer’s to Schizophrenia to depression, and drug developers working on bringing new therapies to market.

“It’s easy to miss the true impact that these illnesses have on individuals, families, and society at large,” Kafui Dzirasa of Duke University School of Medicine admitted during his session at the National Institutes of Health (NIH.) “If we know that all of those experiences are locked within this organ called the brain, and that disease happens when there are changes with this organ, the question comes, how come we haven’t figured out how to cure these disorders yet?”

Dzirasa explained that the challenge early neurologists faced with the dissection of the brain was that it gave them an understanding of the anatomy of the organ, but “like taking apart a laptop, one cannot truly understand its function unless studied while it is on and processing.” This is where the beauty of brain imaging comes in.

The Power of Imaging

Director of the NIH and Celebration of Science co-host Dr. Francis Collins sat down with Daniel Reich in the NIH MRI center to view images of a brain and the vast detailing available with today’s three-dimensional scans.

“How would this look different on somebody with Alzheimer’s disease?” he asked. The audience watched on the screen as ~30% of the brain image deflated, almost like a sponge losing water. Understanding the functionality and physicality of a healthy versus unhealthy brain through imaging has taught us that there’s an opportunity to detect Alzheimer’s disease early and try to do something about it.

The Human Toll

Judy Bachrach of Vanity Fair shared her mother’s struggle with Alzheimer’s disease and the complications it has created for her family. She painted a picture – through words and photographs – of a vibrant, loving woman full of energy and life. She then shared another picture of the same impeccably dressed lady smiling at the camera. “This” she said, “is not my mother. There is a vacancy there that was never there before.”

Judy went on to talk about how she has had to become her mother’s mother.  From dealing with embezzlement schemes to constantly battling with banks and insurance companies for control of her mother’s accounts, the emotional toll and demands on her time have compounded.

Each year, Judy’s mother must take an Alzheimer’s test for insurance purposes.  As part of this test, she is asked to write a sentence – just one sentence – on any topic she likes.  Judy shared that the last time her mother was able to perform this task, over a year ago, the sentence read: “I am so sorry that my daughter has to take care of me.”

Towards a Cure

One of the final sessions at Celebration of Science was “Alzheimer’s and Other Issues of Aging” led by Pfizer’s Freda Lewis-Hall.  It focused on the steps needed to find meaningful treatments and cures for diseases that plague millions of seniors.

According to the panelists, major pieces of the puzzle that is a cure include:
  - Prevention
  - Funding
  - Learning from failures
  - Patient and clinical trial networking

Dzirasa encapsulated the promise of brain science when he said “I hope you’re one of those sitting in the audience celebrating the progress that we’ve made in the brain, and blown away with this idea that one day these advances may open the doorway to new approaches and new technologies that allow us to see brain illness and treat symptoms before these disorders ever really arise and manifest.”

Tuesday, October 2, 2012

Celebrating Science: Continuing the Fight Against HIV

This is the first in a series of upcoming blogs highlighting key themes and outcomes from FasterCures’ Celebration of Science, which brought together over 1,000 scientists, educators, industry executives, policymakers, and patient advocates to celebrate the scientific achievements of the last 20 years and jumpstart a new wave of discovery.

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.

Anthony Fauci , NIAID
The hard-fought battles already won against this deadly virus, and the importance of the fight that still lies ahead, were a focus of exploration at the Celebration of Science.  In a talk given by Anthony Fauci (Director of the National Institute of Allergy and Infectious Diseases), he shared that the initial years of the HIV epidemic were the darkest of his medical career.

Knowing that there was no cure for this terrible disease and that he could only hope to make patients more comfortable was a heavy weight to bear as a medical professional. The rapid spread of HIV/AIDS along with witnessing the consequent suffering of patients  inspired Fauci and other infectious disease specialists to wage war against the disease and discover therapies that could effectively treat the virus, not just the resulting infections.

Tracking our Progress
  • Understanding the HIV replication cycle.  After the disease was discovered, researchers set out to develop an understanding of the HIV replication cycle – the process by which HIV makes copies of its genome, bundles the genetic material with viral proteins, which then go on to infect various cells. By identifying key steps in the cycle, they were able to design drugs that could block these steps, thereby suppressing viral replication. The result was development of the first HIV antiretroviral, AZT, approved in 1987.
  • Targeting treatments to each stage of the cycle.  It was evident that AZT could safely slow down HIV replication in patients; however the virus learned to adapt and began to replicate mutant strains that were resistant to AZT and other first generation antiretrovirals. Patients that once found hope in the new antiretroviral therapy were again faced with despair and ailing health as the drugs became less effective. Thus researchers and physicians took up a new charge to slow down replication even further, attacking the virus at different stages of the replication cycle through multiple antiretrovirals. With this discovery came the development of six classes of HIV drugs, one for each stage of the cycle.
  • The birth of combination therapies. The increased pill burden of multiple antiretrovirals lead to significant decline in treatment adherence. Not only was this damaging the health and quality of life of patients, it also created the propagation of mutant strains that were resistant to the antiretroviral regimen. To combat this challenge, developers began to think of ways to combine drugs into one pill, leading to the birth of market leading HIV drugs such as Truvada, Atripla, Complera, and Stribild – all one pill, once a day regimens that contain two or more drugs. The availability of these convenient dosing regimens has led to significant increases in adherence rates among many patients.
Engaging the Community

Anthony Fauci, Earvin "Magic" Johnson , Greg Simon 
“We now have the wherewithal through behavioral and preventative modalities to turn around the trajectory of the HIV pandemic,” said Fauci.  With today’s course of combination therapies and efforts towards prevention, the worldwide incidence continues to decline, moving from 2.7 million new cases in 2010 to 2.5 million in 2011. The challenge now is to get everyone involved – communities, cities, nations – in screening, testing, and implementing the science.

“We need the black church to get involved; that’s the only way we’re going to break it down in our community” said prominent HIV spokesperson Earvin "Magic" Johnson.  With African Americans representing nearly 50% of all new infections in the U.S., getting the churches and pastors on board to help educate their congregants to get tested and get their results is critical.  There is a lot to be learned from what the gay community did at the start of this epidemic he pointed out, in terms of mobilizing to get information out and provide support.

Winning the War

Thanks to the scientific advancements of the past 30 years, more than eight million people around the world are receiving effective antiretroviral treatment and the life expectancy of an AIDS patient now approaches the life expectancy of people not infected by the virus. The progress is evident, but the fight isn't over. History has shown that when we try to control infectious diseases with non-vaccine intervention, we eventually lose control because of drug resistance. With HIV/AIDS we should expect the same result – if we try to continue to control virus with only antiretrovirals, we risk losing control.

In order to win the war, we must develop a vaccine.

At the end of Fauci’s final session, moderated by HCM Strategists' Michael Manganiello, a member of the audience posed the question, “With the incredible progress that we have made in developing effective antiretrovirals, do we really need a HIV/AIDS vaccine?” Fauci’s answer: "Yes absolutely." It is the heavy artillery we need to eradicate HIV/AIDS altogether.

Heard at Celebration of Science

“I’m functionally healthy, but I still, like many others, need to have a cure. . . . Only then will the promise of life be real.  And it will be because science made it happen.”
-          Moises Agosto, Director of Treatment Education, Adherence, and Mobilization, National Minority AIDS Council
“14 years and one day after the door to motherhood had been seemingly shut I gave birth to a beautiful, healthy, HIV-free baby girl. This was something that was never possible in 1988 [the year I was diagnosed].”
-          Dawn Averitt Bridge, Founder and Chair, The Well Project; Founder, National HIV Awareness Month
“My story represents the very best of cutting edge science. The cure for HIV is my blue rose. When I received my HIV diagnosis in 1995 I fell to my knees, and in 2007 when I was diagnosed as being cured I fell to my knees again.  But this time I prayed that one day the millions of people living with HIV/AIDS worldwide would receive their own blue rose. I will continue to dedicate my life, my blood, my body, my mind and my soul to this expedition.”
-          Timothy Ray Brown, "The Berlin Patient"; Founder, The Timothy Ray Brown Foundation of the World AIDS Institute
Relevant Resources