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One woman’s fight to find cures for neuroendocrine cancer

Time=Lives Story of the Week: Catherine Cooling Davis

Catherine Cooling Davis is 28, newly married, getting her MBA, and living with metastatic neuroendocrine cancer. But, she’s not letting this life-threatening diagnosis stop her from being her own best advocate in the face of uncertainty.

Neuroendocrine tumors (NET), most recently in the news as the type of cancer that killed Steve Jobs, is slow-growing and can begin anywhere in the body that has neuroendocrine cells. Although these cells exist throughout the body, NET are most commonly found in the gastrointestinal tract and lungs. More than 11,000 people are diagnosed with NET each year – a number that is growing by five percent annually.

Medical research is important when it comes to NET because most patients are only diagnosed in the late stages of the cancer once it has metastasized. In Catherine’s case, her diagnosis didn’t come until her cancer was in stage four, forever changing her busy life. However, faced with endless appointments with a series of doctors, Catherine knew she had to take her treatment into her own hands. “Since my diagnosis, I have traveled to meet specialists all over the country,” she said. “They have no better idea what to do with me than I know what to do with myself. Each of the specialists has a strategy, none are the same, and all say that the other doctors' strategies are also not wrong.”

"As terrifying as it is," she says, "I have to be my own best advocate. I have to choose the medical plan that I feel is best for me."

Catherine chose to have surgery to begin removing multiple tumors from her liver. In addition to this, she is searching for answers to many of her unaddressed questions. “I don't know how long I can live with this disease. I don't know how fast it is growing, how long I have had it, or how long I will continue to feel as good as I do,” said Catherine. But, she has hope. She believes a potential cure for this deadly cancer has been developed but sits idle in a research lab at Uppsala University because it cannot currently be patented by the company that owns it. So, Catherine and her friends and family began fundraising to provide money and support for the researchers to take the drug into phase 1 clinical trials.

To read more about Catherine’s story, visit Let’s Cure Neuroendocrine Cancer, or visit Catherine’s Time=Lives story page. 

Using electricity to get the blood pumping

Time=Lives Story of the Week - Fred Streitz

As director of the Institute for Scientific Computing Research at Lawrence Livermore National Laboratory, and director of the High Performance Computing Innovation Center, the technology Fred Streitz is working on everyday has the potential to save lives. Through the use of high performance computers, Fred and his team have developed a new code called Cardioid, which mimics the electrical currents that naturally make the muscles of the heart pump blood throughout the body.

Watch Fred's Time=Lives story here.

We met up with Fred at last fall’s Partnering for Cures when he presented Lawrence Livermore’s collaboration with IBM Research and learned more about its opportunities for biotech and pharmaceutical companies that offer on-demand access to computation expertise running on high-performance computers.

Researchers at the Lawrence Livermore National Laboratory took on this project to saves the lives of those with heart arrhythmias and other heart complications. When the natural electrical system within the heart malfunctions, it can cause an arrhythmia where blood flows irregularly to the body. As a result, more than 325,000 people die each year in the U.S. from this condition.

Fred, who earned a Ph.D. in Physics from the Johns Hopkins University and a B.S. in Physics from Harvey Mudd College, is a leader in High performance computing at Lawrence Livermore National Laboratory, which specializes in combining advanced science with biomedical research in an effort to strengthen national security and contribute to the major medical issues facing the US. “People’s lives are at stake,” said Fred. “Every time a cure doesn’t work, or a cure gets delayed for lack of funding, experience, or scientific background, those are lives that are at stake.”

Fred’s work at Lawrence Livermore is a great example of the power of technology and innovation to change the healthcare and medical research industries. Just last week, President Obama announced his support for BRAIN (Brain Research through Advancing Innovative Neurotechnologies) a radical national initiative which allows us to "better understand how we think and how we learn and how we remember," said the president. Additionally, the promise of whole genome sequencing is also leading to rapid new discoveries enabled by a decrease in cost and increase in availability.

Check out more stories from researchers like Fred on Time=Lives.

Imagine the possibilities if we could only read your mind…or at least map your brain

"Imagine if no family had to feel helpless watching a loved one disappear behind the mask of Parkinson’s or struggle in the grip of epilepsy.  Imagine if we could reverse traumatic brain injury or PTSD for our veterans who are coming home…,” said President Barack Obama last week as he laid a grand, ambitious new government initiative to map the brain.

Obama said he will ask Congress for $100 million in 2014 to support the BRAIN (Brain Research through Advancing Innovative Neurotechnologies) initiative which allows us to "better understand how we think and how we learn and how we remember." Three government agencies will be involved: the National Institutes of Health, the Defense Advanced Research Projects Agency and the National Science Foundation.

“It's an audacious, bold idea,” said Francis  Collins, director of the NIH. "To understand how the human brain works is about the most audacious scientific project you can imagine," he said. "It's the most complicated structure in the known universe."

In many ways, this initiative is similar to the Human Genome Project which Collins led and completed in 2003, in its vision and ambition. But, unlike the Human Genome Project, the BRAIN initiative has not laid out its primary scientific goals. The lack of detail is worrying BRAIN skeptics and advocates alike. Some scientists were quick to question the motives behind this initiative, noting that a lot of great research on the brain is already underway.”

At a Q&A segment  following the President’s announcement, Collins said that these details would be hammered out by a “dream team” of 15 scientists who will hold their first meeting at the end of the month. This team is led by Cori Bargmann of Rockefeller University and William Newsome of Stanford University, will be charged with coming up with a plan, a time frame, specific goals and cost estimates for future budgets.

Additionally, Obama addressed how this initiative can drive growth and create new job opportunities. As the “rest of the world” is racing ahead in the quest for innovation, he expressed concern that we will lose a new generation of scientists because of uncertainty in research funding. We can’t afford to miss these opportunities, he said.

The President called on companies, research universities, foundations, and philanthropists to get involved.

What we took from sitting in the room with the leaders and innovators in science and research as the president spoke about the promise and potential of this new initiative, was a broader message about the power and value of investing in science, and the important role the federal government plays in ensuring scientific breakthroughs can improve health.  We took such enthusiasm and energy as our cue to be more determined than ever to advance medical progress.

Upcoming Milestones

• As part of this planning process, input will be sought broadly from the scientific community, patient advocates, and the general public.
• The working group will be asked to produce an interim report by fall 2013 that will contain specific recommendations on high priority  investments for Fiscal Year (FY) 2014.
• The final report will be delivered to the NIH Director in June 2014.

Key Resources:

• Remarks by the President on the BRAIN Initiative and American Innovation, April 2, 2013
• Video of Remarks by NIH Director Collins and the President, April 2, 2013
• Fact Sheet: BRAIN Initiative(Whitehouse.gov), April 2, 2013
• Video of Open for Questions with NIH Director Francis Collins(Whitehouse.gov), April 2, 2013 
• WhiteHouse.Gov Blog: BRAIN Initiative Challenges Researchers to Unlock Mysteries of Human Mind, April 2, 2013

Time=Lives Story of the Week: Sienna Otto

“Sienna can’t plant a flower, or skip, or twirl like a ballerina, or even hold her mom’s hand when they walk. But since Sienna can’t plant a flower herself, we’re going to do it for her…and in doing so, we’re going to save this little girl’s life.”

Fibrodysplasia Ossificans Progressiva or F.O.P. is an extremely rare genetic disorder where bone forms unexpectedly within muscle and other soft tissue. Over time, this can cause joints to lock-up and leave them unable to move. So, F.O.P. is often referred to as “stone man syndrome.”

Although two-year-old Sienna Otto was diagnosed with F.O.P. in 2012, neither Sienna nor her family have let this rare disorder affect their positive outlook on life. Sienna loved to plant flowers outside with her parents, however, F.O.P. makes it impossible for her to lean down to the ground and kneel to the grass. So, with the help of their friend Natasha Lam O'Rourke and her Boston-based advertising agency, Connelly Partners, Sienna's family started Sienna’s Flower Garden (@CureSienna), a virtual garden where each donation is recorded as a digital flower. All funds go towards finding a cure for F.O.P. for children like Sienna. 

Watch Sienna’s Story Here

http://timeequalslives.org/stories/Sienna.php 

According to the International Fibrodysplasia Ossificans Progressiva Association (IFOPA), only 1 in 2 million people have F.O.P., and there are less than 200 confirmed cases in the United States. Three principle researchers and 15 fellows, students, and assistants make up the only dedicated F.O.P. research lab in the country at the University of Pennsylvania. Approximately $1.5 million dollars is spent on research each year - 75 percent of which is comprised of family and patient fundraising and donations. 

Unfortunately, F.O.P. is typically misdiagnosed by doctors because it is so rare. Misdiagnosis of the disease leads to greater pain and suffering of patients because unnecessary biopsies or other tests cause the body to create more bone rather than regenerate tissue. To date, Sienna’s Flower Garden has raised more than $50,000 and continues to be an important resource for families and patients.

There are nearly 7,000 rare diseases affecting ~30 million Americans, which means almost one in ten Americans is suffering from a rare disease.  Traditionally, rare disease research has been relatively siloed, with limited communication between and across research organizations. However, increasingly patients, their families, and the disease-specific organizations that serve them are starting to work together towards the ultimate shared goal of finding cures.

See more stories about the power and promise of medical research to improve and save lives, and tell us why it matters to you. Visit FasterCures’ Time=Lives campaign for more information.

Diving deeper into healthcare costs and breakthroughs

By Shawn Sullivan, Program Associate, FasterCures

Over the past few weeks, Washington has been consumed by conversations about the federal budget. Two recent events we attended, a Center for American Progress discussion and a Congressional Neuroscience Caucus briefing, aimed to cut through the rhetoric and focus on specific aspects of health funding – the affordability of healthcare and the relationship between mental illness and violence.

The March 13 Center for American Progress discussion, “The High Price of Healthcare,” featured the journalist and author Steven Brill, who wrote the recent TIME cover article “Bitter Pill: Why Medical Bills Are Killing Us.” The conversation covered the excessive number of healthcare services and exorbitant prices, as well as reforms to control healthcare spending. “[Brill’s article] has changed the entire conversation surrounding healthcare,” said panelist Giovanni Colella, CEO and co-founder of Castlight Health. “It takes the issue to where the real problem is. This is an industry where the entire incentive system is wrong. We don’t know what we are paying, what we are buying, or what the outcomes are relative to costs. That would never fly in any other industry. Imagine paying for a Ferrari and getting a Toyota.” While much of the debate about the Affordable Care Act has focused on who is responsible for paying for healthcare, Brill pointed out that there isn’t as much focus on the actual costs: “During the debate over Obamacare, the questions being asked were about who pays for healthcare, when the question should have been ‘why does it cost so much?’”

The March 18 Congressional Neuroscience Caucus briefing examined the critical topic of mental illness and violence, as well as the state of mental health services and programs in the United States and scientific breakthroughs on the horizon that can benefit the 60 million Americans who suffer from mental illness. “There are two parallel conversations going on right now,” said Thomas R. Insel, director, National Institute of Mental Health. “One is about taking a fresh look at policy dealing with gun violence due to the recent tragic events at Sandy Hook, and other places. The other, due to things that have happened in only the last few months, is about revolutions taking place in brain research. President Obama mentioned mapping the brain in his recent State of the Union Address. But nobody is bridging these two conversations. If we want to prevent more Sandy Hooks, we have to do more about brain research.” Elizabeth Childs, child and adolescent psychologist and former commissioner of the Massachusetts Department of Mental Health, emphasized the importance of funding mental health research in tough economic times: “I am well aware of the challenges you face here in Washington to balance our budget, but there are investments worth making. Underfunding of the National Institute of Mental Health does not move us in the right direction. The reality is that the lack of investment in mental health is far more costly in the long run, including the increased burden that untreated mental illness has on the nation’s education, public safety and corrections systems. And that is before we factor in the incalculable human cost to families and our society.”

These conversations about the unique aspects and critical impact of federal health funding are pivotal to our nation’s well-being and productivity. As these conversations continue, we must focus on the real-world impact of policy decisions on patients, families, and communities nationwide.