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. 

A Clinical Trials System for the 21st Century

By Samantha Mayberry, Program Assistant at FasterCures

Last month, a host of clinical trial and oncology experts at the Institute of Medicine's (IOM) “Implementing a National Cancer Clinical Trials System for the 21st Century” workshop discussed the great strides that have recently been made in the clinical trials space. The workshop, co-sponsored by the Institute of Medicine and the American Society of Clinical Oncology (ASCO), examined how the National Cancer Institute (NCI) has implemented the recommendations put forth in IOM’s 2010 report “A National Cancer Clinical Trials System for the 21st Century” with the goal of both evaluating progress against these recommendations, and identifying any outstanding gaps.

A January 2012 ASCO report details much of the progress discussed at the workshop, including the development of a centralized patient registration system amd a clinical trials data management system, as well as advancement in gaining coverage for trials through the Affordable Care Act.  However, the issues that remain clearly demonstrate that the current system is still being built to accommodate science as it was, not as it will be.

Specific issues include the fact that many NCI-run trials are not currently equipped for the rapidly-advancing science available in cancer therapeutics, particularly precision medicine, and the need for a clinical trials system run and regulated on an international scale rather than national systems for individual countries.

The NCI cooperative group program promotes and supports clinical trials for cancer therapies by coordinating researchers, cancer centers, and physicians to identify crucial issues in cancer research and design trials to answer these issues. Their goal is to make clinical trials more coordinated and efficient.

More than 30 experts from across NCI, academia, major pharmaceutical companies, cancer research institutes and biotech participated in the workshop. In a presentation to the group, FasterCures Executive Director Margaret Anderson pointed to the many innovative solutions being applied by the venture philanthropy community -- nonprofit disease research organizations that are moving away from the more conventional public charity model and instead are adopting the tools and techniques of venture capital finance, and the strategies and tactics of high-technology business management. One prime example is the Leukemia and Lymphoma Society’s Therapy Acceleration Program (TAP), which targets therapies in areas of high unmet medical need, specifically funding investigators who are performing research in those areas at all stages of clinical investigation.

 

Another trend prevalent among these organizations is the prominence of clinical trial matching tools and trial databases. Many provide opportunities for patients to register their data and/or run complete registries for trials within the disease they focus on, helping to alleviate some of the stress that academia and industry often face with patient enrollment. Finally, another unique ability of these organizations is the capacity to run clinical centers for research.

As NCI, industry, and academia seek out new models to help improve and accelerate the system, these venture philanthropies – many of whom are members of The Research Acceleration and Innovation Network (TRAIN) at FasterCures – provide a fertile nesting ground of ideas.

We look forward to seeing the official outcomes from the workshop and continuing to work with IOM and ASCO on this important issue.

Relevant Links:
• The webcast and presentations from the workshop can be found here.

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.  

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

Relevant Links
* Facts about prostate cancer
* The struggle to employ young investigators

Time=Lives Story of the Week: Jamie Goldfarb

“We found out I had Stage IV Melanoma the day before I was supposed to go back to work from maternity leave. So Kai was three months when we started this, and definitely the most difficult part emotionally is worrying about not being there for him.”

Meet Jamie Goldfarb: wife, mother, blogger and Melanoma survivor. 

After being diagnosed with metastatic cancer in early 2011, Jamie enrolled in the National Cancer Institute’s TIL Trial, hoping the progressive immunotherapy treatment would stimulate her body’s ability to fight the disease.  Documenting her experiences in a blog she initially created for close family and friends, Jamie unwittingly became a source of hope and inspiration for Melanoma patients around the country, and a living breathing example of the power of clinical trial involvement.

Nearly two years later, thanks to the pioneering efforts of her medical research team, and Jamie’s own resolve to be there for her son, all that remains of the more than 20 tumors that once riddled her liver and pancreas is one, seemingly harmless subcutaneous tumor.

Jamie’s is one of nearly 100 powerful personal stories featured through FasterCures’ Time Equals Lives campaign, a social media effort designed to humanize the toll of disease and explain in real terms why medical research MUST be a priority NOW. 

"We cannot be satisfied with things that work ok," says Jamie. "That can't be the goal. There are life threatening illnesses and we have to figure out a way to find cures for them, not just several treatments that may or may not work." FasterCures couldn't agree more.

“My thoughts are now dominated by planning for my after-cancer future, rather than by wondering if there will be a future. . . . I realize over the past weeks that I have officially started thinking of myself as a survivor rather than as a patient. And the difference between those two definitions is astounding.”

– Jamie’s Melanoma Mom Blog

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 

It’s about Cancer, Stupid.

Lance Armstrong’s cancer forum is putting the wrong people under the lights. Even if the presidential candidates found a cure for cancer right there on stage, with the way the media covers politics the rest of us would never know. How is it possible that after discussing their approaches to winning – dare I say it – the war on cancer -- the headlines are about which candidate is willing to take which lobbyists’ money? I thought journalists were taught not to bury the lead. Well the lead is the toll in death and suffering cancer is taking, not the campaign contributions candidates are taking.

The press continues to confuse healthcare economics with curing diseases. The benefit of Lance’s gathering is it forces candidates to go beyond the “more money” argument and into how they would change something. In a discussion about curing and surviving cancer, it is folly to debate which lobby groups are “acceptable” as donors. The last time major health players were left out of the game, they defeated a popular President by putting two actors playing man and wife sitting around a kitchen table shaking their heads. If we are going to create medical solutions for cancer, we will need everybody at the table with open minds, collaborative natures, and valuable human and financial resources. And not shaking their heads.

Lance was right to ask the field of future Presidents how they would attack cancer. Now let's start asking the press to cover the answers and not let them change the subject to pick silly fights.

Greg Simon, President, FasterCures