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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.

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.

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

Time=Lives Story of the Week: Jessica Foley

Encouraging the next generation of scientists

“What motivates me is working in a field where every day it's changing, it’s challenging, and it’s exciting.”

Meet Jessica Foley. She’s the Scientific Director at the Focused Ultrasound Foundation (FUSF) and a long-time advocate for greater use of the focused ultrasound, a revolutionary technology that allows for the treatment of numerous diseases without the danger of invasive surgery or a lengthy recovery time.

Watch Jessica’s story here.

Currently, MR-guided focused ultrasound surgery has been approved by the U.S. Food and Drug Administration (FDA) for the treatment of uterine fibroids, and has obtained CE-mark approval in Europe for uterine fibroids and pain from bone metastases.  However, the possibilities for this technology are countless - with ongoing clinical trials for breast tumors and brain tumors, and plans to begin additional research into it's impact on several other kinds of tumors, stroke, and epilepsy.

For over 12 years, Jessica has had a rich career as a scientist and researcher in this novel field. Before joining FUSF, she worked with InSightec and Medtronic, and also served as a 2011-2012 AAAS Science and Technology Policy Fellow at the National Science Foundation.

We met up with Jessica at last year’s Celebration of Science to talk about what she envisions for future generations of scientists and researchers and the challenges they may face with today’s economic shortcomings. She emphasized the need to elevate the role of science in our society so we can attract and engage tomorrow's innovators.

"Years ago [the public] seemed to get it," said Jessica, who holds a Bachelor’s in Biomedical Engineering from Duke University and a PhD in Bioengineering (emphasis in focused ultrasound) from the University of Washington.  "And maybe not everyone gets it now, but I hope that through all sorts of projects we're trying to work on we can get to that point again where kids want to grow up and be scientists and engineers."

To learn more about the Focused Ultrasound Foundation visit http://www.fusfoundation.org.

And make sure to check out the TIME=LIVES campaign for more stories about the power and promise of medical research, and to help spread the message that research matters and needs to be a national priority.

Time=Lives Story of the Week: Phil Gattone

"There really isn't much logic in sending your child to brain surgery, but we did. Because we knew we were losing him."

President and CEO of the Epilepsy Foundation, Phil Gattone, and his wife Jill began the fight for their son Phillip’s life in 1991 after he suffered his first seizure at the age of four that lasted almost an hour and landed him in the emergency room.

Watch Phil’s story here

“It was a researcher who we probably will never know who helped well before our son started having seizures,” said Phil. “Because of the tremendous efforts of his clinicians and those researchers, there was a sense of hope.” 

Now in his mid-20s, Phillip is a software engineer at Northrop Grumman Aerospace Systems and is able to effectively manage his epilepsy.

According to the Centers for Disease Control and Prevention, epilepsy affects 2.2 million Americans and 65 million people worldwide. Approximately 1 in 26 people in the United States will develop epilepsy at some point in their lifetime, with an aging baby boomer generation reaching retirement age the number of citizens with the disorder is predicted to grow.

Advances in treatment for epilepsy have provided a functionally normal life for people like Phillip, but despite how common it is, epilepsy remains one of the least understood chronic medical conditions. While medications and other treatments help many people of all ages who live with the condition, more than a million people continue to have seizures that can severely limit their school achievements, employment prospects and participation in all of life's experiences

Research is the key to the future for people with hard to control seizures, and the trajectory of epilepsy – and other disease for which there are no cures – depends on attracting the best scientific minds and funding innovative clinical investigation. The science is promising, but without continued support and resources, we risk patients like Phillip falling by the wayside.

To learn more about the Epilepsy Foundation and get involved in their work, go to http://www.epilepsyfoundation.org. 

And check out the Time=Lives campaign to find out what you can do to help speed the medical research process by participating in a clinical trial, sharing your story, or simply spreading the message that medical research must be a national priority.

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

Time=Lives Story of the Week: Michael Kaplan

“I’ve been a Type 1 diabetic since I was 12, so 31 years; and HIV positive for 20 years this March. I’ve been able to maintain good health, keep my viral load down, my t-cells up, which has allowed me to do the work I do.” – Michael Kaplan, President & CEO of AIDS United

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.

A leading advocate for HIV/AIDS patients, policy, and research, Michael Kaplan is the president and CEO of AIDS United.  Born out of the merger of the National AIDS Fund and AIDS Action in late 2010, AIDS United’s mission is to end the AIDS epidemic in the United States by combining strategic grantmaking and capacity-building with national advocacy to ensure access to life-saving HIV/AIDS therapies and services, and advance key policy initiatives.

View Michael's Story Here

“As the successes of early treatment towards prevention merge with health care reform across the U.S.,” says Kaplan” the light at the end of the tunnel is only getting brighter.  I truly believe I’ll see the end of this epidemic in my lifetime."

Kaplan advocates for early testing and treatment as a way to dramatically decrease the number of new HIV/AIDS cases and help those already infected to start managing their illness as soon as possible. Like many currently incurable diseases, access to treatment is crucial. NIH research shows that the right drug cocktail can reduce a person’s ability to spread the virus to another by up to 96 percent.

Medical research discoveries from several fields have helped create many of the current medications HIV/AIDS patients depends upon today. Just as in the past, “fundamental basic research that is being done now is going to lead to things ten or fifteen years from now that we cannot predict,” said Dr. Anthony Fauci at a FasterCures' 2012 Celebration of Science event.

But with funding for research across all diseases at risk because of sequestration – looming, across-the-board budget cuts – support for the work of advocates like Michael and the science he helps to advance is more important than ever. Saving time in medical research means saving lives.  

See more stories about the power and promise of medical research, and tell us why medical research matters to you.

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

Relevant Links:
-- Back to Basics: HIV/AIDS Advocacy as a Model for Catalyzing Change

Time=Lives Story of the Week: Derrick and Meredith Day

“I think I’m the same, except I just can’t see.” 

– Derrick Day, 6 years old

Derrick (6) and Meredith’s (4) story came to Time=Lives via the Foundation Fighting Blindness, an organization dedicated to driving research to prevent, treat and cure people affected by retinal degenerative diseases.

View Derrick & Meredith’s story here.

Born legally blind, the siblings suffer from a rare inherited eye disease called Leber’s Congenital Amaurosis, a disorder that can also create central nervous system abnormalities.

Seemingly simple tasks for a sight-abled person become difficult hurdles or even impossibilities when disease has robbed someone of their sight. Derrick’s dad dreams of a day when his son will be able to drive a car. “I never thought about Derrick’s not being able to drive,” said his mom. “When we got to the stop sign, he presented the question to me: Mom, how are we going to make that sign braille so that I can drive?”

Whether it’s as simple as experiencing a rainbow, or as complex as driving a car, the only way for kids like Derrick and Meredith to be able to achieve these seemingly insurmountable tasks is to be able to see. “And the only way that we can have them see,” says their mom, “is with research, and by funding scientists to find a cure.”

The good news is that retinal disease science is advancing. For example, just yesterday the Foundation Fighting Blindness reported that the Argus II retinal prosthesis, a device that can restore some vision to people who are blind from advanced retinitis pigmentosa (RP), received U.S. market approval from the Food and Drug Administration (FDA). More than 20 years of research went into the development of Argus II, with early support from the Foundation.

Also, earlier this month The Washington University School of Medicine created an innovative method for treating vision-robbing diseases using genetic reprogramming. And while their research is at an early stage in mice, it has revealed valuable clues about how to potentially save vision in people.

But promising research like Washington University is conducting wouldn’t be possible without federal funding.  And with sequestration – mandatory, across-the-board budget cuts – looming, the researchers who study diseases like Leber’s Congenital Amaurosis and others could face debilitating cuts to their research programs that would, at best, slow innovation and progress, and at worst, cause entire labs to shut down.

Want to know more about sequestration? Visit FasterCures’ Sequestration Station for facts, forecasts, and ways to add your voice to the fight for funds. Medical research matters, and must be supported. Derrick, Meredith, and millions of others suffering from debilitating diseases are counting on it.

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

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

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:

• VISIT the campaign Web site
• LIKE the Facebook page
• TWEET with us at #TimeEqLives
• DOWNLOAD and SHARE the Message
• TELL us your story