Look Who's Talking at Partnering for Cures 2013

Remember to visit us on our new blog: www.fastercures.tumblr.com Partnering for Cures is a meeting focused on innovation, science, and collaboration. It is a platform for big ideas, transformative programs, and tangible results brought forth by some of the most disruptive innovators of our time. A shared sense of urgency binds together meeting participants, because we all know that when it comes to the search for cures, "soon" is just not good enough. JOIN US!  Look who's talking at #P4C2013 Confirmed speakers »  Panel topics »   Partnering for Cures emphasized the urgency that time is lives... We have to be willing to approach [challenges] in a different way to get solutions."  – Academic scientist   It's a place to challenge conventional thinking."   – Start-up company executive   [Partnering for Cures] is the biggest gathering of patient advocacy groups, biotech companies, pharma companies, and regulators."   – Xconomy Have you registered yet?   http://www.partneringforcures.org »

Time=Lives Story of the Week: Sarah and Adam Foye

Kids, DNA and Genetic Testing

Adam Foye, a New-Jersey sixth grader, has lived much of his life with weak muscles and uncertainty. Although his symptoms match most closely with the rare muscular disorder, Centronuclear Myopathy (CNM), his genes tell a different story. The 11-year-old’s tests show no issues with the genes that indicate CNM. Adam’s mother, Sarah, said in a recent TIME story, “it has taken a very long time to get no answers.”

Check out Sarah’s Time=Lives story here.

Last year, Adam was one of three children to take part in a competition sponsored by Boston’s Charity Hospital called the Children’s Leadership Award for Reliable Interpretation and Appropriate Transmission of Your Genomic Information, otherwise known as CLARITY. More than 20 teams of international researchers competed to analyze Adam’s genetic sequence in comparison with someone who does not have CNM and translate the results into easily ready and interpretable information for patients, families, and doctors. The winning team, the Division of Clinical Genetics at Brigham and Women’s Hospital in Boston, took home $15,000 for their combined “analysis, clarity, and utility” in their reports.

Whole genome sequencing’s ability to provide key information for many currently unanswered medical questions holds great promise for the future of the healthcare industry overall. The process, which used to be priced in the billions, has dropped in cost and thus increased accessibility. However, questions still loom regarding what doctors and patients should do with their data, how that amount of data should be stored, and what guidelines need to be established to regulate the process. All of these issues need to be addressed before whole genome sequencing can be used with any regularity.

For the Foye family, it was discovered that Adam’s gene that encodes for the Titan protein is malfunctioning. Titan serves as spring in the muscle to help with contraction and expansion. Although there still is no cure for this problem, the Foyes are hopeful. “As my husband says,” notes Sarah in the TIME piece about the results of the competition, “this is not our final destination on our medical journey, but it’s an important milestone. Now we want to work toward a treatment.”

For more information on the CLARITY competition, watch: http://www.youtube.com/watch?v=T6SpWi0VJ0k&feature=youtu.be. And for further information about the potential uses of whole genome sequencing, read Gina Kolata's excellent t three-part series on the topic in the New York Times (links below).

Additional stories about the power and promise of medical research to find cures and save lives can be found on the Your Stories page of our Time=Lives campaign. Check out the site to find out what you can do to help make medical research a national priority.  After all, we’ll all be patients someday.

Gina Kolata's Three-Part Series on Whole Genome Sequencing in the New York Times 

• http://www.nytimes.com/2012/07/08/health/in-gene-sequencing-treatment-for-leukemia-glimpses-of-the-future.html?pagewanted=all&_r=0
• http://www.nytimes.com/2012/07/09/health/new-frontiers-of-cancer-treatment-bring-breathtaking-swings.html
• http://www.nytimes.com/2012/07/10/health/genetic-test-changes-game-in-cancer-prognosis.html

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

Need Cures? Who Ya Gonna Call?

By Margaret Anderson, Executive Director, FasterCures
As seen on HuffingtonPost 

Do you know anyone whose life hasn't been touched by disease? As I get older I become more aware that the frenetic life I lead and call normal could be interrupted in the blink of an eye.

We all know the drill. People in our lives are just going about their business when something goes amiss -- a twitch in their leg, some dizziness, a lump, a pain, an abnormal result, an accident, an ambulance, a hospitalization, a doctor's visit, a test, a diagnosis. A new reality. A replacement of what once was to a new normal.

I can conjure up times in my life that disease has interrupted life just like that. Here are just a few that were easy to recall; I am sure you have just as many.

• "I didn't see this coming -- I thought it was my heart medication." -- My father after his leukemia diagnosis.


• "This week, I found a lump in my breast." -- My friend when she put her hand on my arm after I asked what's new.


• "My dad is at the end of life after battling Alzheimer's." -- My colleague after her dad began his final days.


• "My doctor had told me I would be dead in three years." -- My friend recounting how in the earlier days of the AIDS epidemic there was no prospect of survival.


• "I also realize that while I have what may be the nastiest cancer out there, I have it easy compared to others." -- My friend describing his fight against pancreatic cancer.

That moment when you or someone you love become a patient, the patient. And then what?

Well, hopefully the wonders of science and medical research will have led to good therapeutic options for you. At our recent Celebration of Science event, we witnessed scientific discovery literally unfold on the stage of the National Institutes of Health as scientists and patients told breathtaking stories of research triumph. Here's a glimpse of how scientific discoveries have improved health and saved lives. It's remarkable. But, consider that of the 4,000 diseases for which we have the exact molecular basis, only 250 have treatments available.

So, while we need to celebrate the success stories in medical research that allow us to carry on our lives -- be those successes through prevention, diagnostics, devices, or medical intervention -- we have more work to do. We must ensure that we continue to have a robust flow of scientific discoveries that we can then translate into better health.

The well-being of too many of our friends and family depends on this.

The pathway to treatments and cures is littered with failure, lack of funding, scientific and regulatory challenges, reimbursement issues, health care delivery issues, and if that is not enough there are immediate challenges like the impending fiscal cliff and sequestration. Decisions that our reelected President Barack Obama and the U.S. Congress will make could substantially impact the future of treatments and cures.

I could cite data to show how medical research matters.

• It saves lives.
• It creates jobs.
• It maintains us leadership in the global economy.
• People's lives depend on it.

We face a major paradox -- that the potential of science is greater than ever but the outlook for funding has never been bleaker. If an agreement on how to prevent sequestration doesn't happen, here's what we'd see:

• U.S. government science funding programs would see their budgets cut by 8.2 percent in 2013.
• $2.52 billion decline in funding at the NIH.
• 2,300 fewer NIH grants -- representing almost a quarter of new and competing grants.
• Total employment supported by NIH awards would fall by 33,704.
• The Food and Drug Administration (FDA) will lose $318 million from its budget, which wasn't adequate to begin with.
• Virtually all user fees paid to the agency to ensure swift application reviews would be considered part of the sequester.
• About 80 percent of the agency's budget is devoted to personnel, so cuts would mean reduced staffing levels or imposing furlough days

So, given that the need is great, the promise is there, and the arguments in support of this funding are strong, now what?

1. Get educated. Go to our website called Sequestration Station to get smart on the issues and see how you can get involved.
2. Tell us your story. Be a part of our new social media campaign called Time Equals Lives. We are collecting personal stories -- from patients and scientists, researchers and caregivers, industry executives and academics -- to make a compelling case about the critical importance of medical research. We'll share these stories with leaders and decision makers to remind them of what is at stake.

Because when you or a loved one needed a cure, we want to make sure you knew you'd have someone to call on.

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For more by Margaret Anderson, click here.
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