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

Tools of Engagement: Building patient-centered research networks

By Kristin Schneeman, Program Director, FasterCures

Engaging patients in the research process is more complex and exciting now than ever before. Patient-driven organizations are moving beyond simply creating patient registries or serving as a conduit to participants for clinical trials and are marshaling their forces in new and more comprehensive ways. In a FasterCures Webinar on March 20, participants heard about two exciting new models of such “patient-centered research networks,” the T1D (Type 1 Diabetes) Exchange and Registries for All Diseases.

Dana Ball, CEO of the T1D Exchange, began by saying that after much due diligence about where the barriers and inefficiencies were in research and care for Type 1 patients, the Helmsley Charitable Trust set out to create an end-to-end solution to connect the R&D and healthcare enterprises with the patient resources necessary for all to succeed. What it has built in the T1D Exchange is a national network of 69 clinics treating 100,000 T1D patients, a 26,000-patient registry, and a 1,000-patient “living biobank” (i.e., samples are collected to answer specific questions). Integral to the network is Glu, a social network through which the Exchange is collecting valuable patient insight via surveys and discussions and which serves as a “real time access point for community-driven research using an innovative mobile platform.” With all these tools, the Exchange can share patient data, recruit patients, and collect samples quickly. Ball noted that absolutely critical to the success of the entire Exchange is a carefully constructed universal consent from patients.

The Exchange is meant to be self-sustaining. Ball noted that it provides products and services to academic and industry researchers and insisted that these are not “dirty words” for a nonprofit. “We think of ourselves as a high-quality CRO [contract research organization],” he said, with a unique trust relationship with the patient community. Another element of the sustainability model will be a new nonprofit company called Unitio, to be launched mid-year, which will license the platform the Exchange has created on reasonable terms to other disease groups. Ball noted that none of this would be possible without philanthropic support – no other single sector has the incentives to do this work. He was passionate about the need to educate philanthropists about how to invest for impact – the need for resources to hire good people, to create infrastructure that facilitates the whole enterprise but that others won’t create.

Sharon Terry followed with a presentation about Registries for All Diseases (Reg4All), which recently won the Sanofi Collaborate/Activate challenge – a cross-disease, crowdsourced registry that promises to break down the silos between diseases and gather information from patients in a standardized way. Terry began by noting that while recruiting appropriate patients for trials is viewed as “finding a needle in a haystack,” in fact “the haystack is made of needles” – we are all potential trial participants and just need the engagement, education, and trust to get on the information highway.

Reg4All is an evolution of work that Terry and Genetic Alliance have been involved with for a number of years that continues to widen in scope and sophistication. It is built on the platform of DiseaseInfoSearch, a database that provides disease-specific information and resources about 13,000 different diseases. Reg4All is not a disease-specific registry but rather a platform for individuals to enter self-reported medical information via a “gamefied survey” – and eventually to import clinical data from electronic health records – fully identified and protected. The survey gathers information on both common data elements across diseases and on disease-specific data elements. Patients use very sophisticated privacy software to authorize access at a very granular level about what information different types of users can see, e.g. researchers recruiting for clinical studies.

Participants had many and excellent questions for the panelists, beginning with, “We’ve gone from having none to too many cost-effective open source registry choices. How do we choose…?” Terry commented that there are valid reasons for multiple registries to flourish, and that our goal should not be one solution but rather lots of interoperable solutions. In her view, the granular privacy settings that Reg4All offers are very important in getting there. She mentioned that Genetic Alliance will be hosting a series of nuts and bolts Webinars to instruct groups on topics such as how to leverage or build on an existing registry/biobank, and will provide Reg4All modules others can pick up and use.

Ball closed with another plea for philanthropists and philanthropies to support this kind of project, calling on them to be like Ben Franklin, building libraries instead of buying books for everyone.

Resources

• View the Webinar slides
• Watch an archive of the Webinar
• Innovator: Sharon Terry's Global Database for Disease Research

http://train.fastercures.org/

Technology Entrepreneurs Unite at SXSWi

By Lauren Fliegelman, FasterCures Intern

Earlier this month the city of Austin, Texas played host to the best and brightest technology entrepreneurs at the interactive portion (SXSWi) of the South by Southwest (SXSW) 2013 conference and festival.  A magnet for startup companies looking to make an impact, it brought together a wide range of breakthrough IT innovations – some of which have the potential to make waves in the healthcare and medical research industries. 

FasterCures was not there in person, but we watched the events unfold from afar and were quite enthused by some of the things we saw.

Trends

Trending topics at this year’s event included the potential of 3D printing to revolutionize U.S. manufacturing and product design. Already, examples of the use of 3D printing can be seen in the orthopedic industry. Earlier this month, Oxford Performance Materials demonstrated how this technology is able to replace 75 percent of a patient's skull, with the approval of U.S. regulators, using 3D-printed implants that take the place of bone damaged by disease or trauma. Several discussions at SXSWi addressed this technology and explored its continued development, practical applications, and safety implications.

Additionally, the “Quantified Self” movement – a trend towards collecting health data from our bodies in real time and tracking its impact on our overall wellness – was a highlight of the week. The case for collecting  big data has been made in previous years at SXSWi, however, more advanced wearable technology has since been developed allowing each and every one of us to record and measure our everyday bodily functions – from fitness impact to sleep quality to metabolic  performance – allowing for greater analysis and insight.

Technologies of note

The newly formed Neurotrack was the winning healthcare startup at the event. Neurotrack’s work towards earlier Alzheimer’s detection is rooted in 25-year-old research from neuroscientists at UC San Diego. Its test can now identify those at risk for Alzheimer’s disease six years before the onset of symptoms, which is traditionally when an initial diagnosis is made. CEO Elli Kaplan said that 100 percent of those who scored lower than 50 percent on the test went on to develop Alzheimer’s disease.

Higi, a medical screening startup, had its own lounge at the event where Higi Stations, which measure different aspects of your health and give you a score out of 999, were readily available for use. A higher score means better physical, mental, and social health. The Higi philosophy is that many of the US’s major health ailments such as obesity and diabetes could be cured through prevention. According to the company, if you know more, you’re more likely to do more. Armed with their score, people have the ability to make significant changes in their lives.

Other cool stuff

Other notable mentions from the conference included Highlight, an app that alerts you when people with similar interests are nearby, the Memoto “life blogging” camera, that clips on to a shirt or jacket and snaps a photo every 30 seconds, and Leap Motion, a small device that allows you to control your computer by the wave and motions of your hand rather than a mouse or track pad.

For more information on SXSWi 2013, and to see a list of guest speakers and participants, visit http://sxsw.com/interactive.

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.

The long-term effects of sequestration on medical research

“I worry about the treatments that we’re not going to discover as a result of these cuts. What does this mean downstream; what does it mean in terms of the unmet needs of patients who have diseases with no treatments or cures? The NIH investment is a very powerful driver to this entire enterprise.” 

- Margaret Anderson

Last week, FasterCures Executive Director Margaret Anderson appeared on the BioCentury broadcast with host Steve Usdin to examine the long-term ramifications on medical research and health outcomes if sequestration is not appealed. Watch the whole segment here. Anderson was joined on the Sunday, March 10 segment by Lynn Marquis, Director of the Coalition for the Life Sciences, who talked about the effects that these cuts will have on the scientific workforce.

“We’re going to lose an entire generation of scientists moving forward,” Marquis cautioned. “We’re already not seeing kids pursuing scientific careers because there is no future for them.”

Though optimistic about the possibility to reverse this alarming trajectory, Anderson and Marquis warned that medical science is an area in which the United States cannot afford to fall behind.  They emphasized the outsized impact that investments in research have on both human health and the health of our economy, and noted that it is hard to compare these types of cuts with others.

“This is a powerful investment in the future - for patients, for the economy, for jobs, for industry. I don’t think it’s the same as cutting other programs,” Anderson said. Marquis agreed and offered a bit of hopefulness, “I think we see from both Republican and Democrats…there is huge support for NIH’s biomedical research, primarily because of the human health and economic benefits that we reap from our investment.”

So if Congress understands that medical research is vital to the future of our country, what can be done to ensure we don’t lose our preeminence in the life sciences?

Anderson thinks there’s hope for the NIH to avoid budget-desolation. “I’m an optimist. I think that people need to speak their mind to their members of Congress about how they feel about this investment.” She also spoke about the power of team science and cross-sector collaboration in moving promising research from lab to patient faster.

Just this year, NIH will be forced to reduce its spending by $1.6 billion, significantly slashing the United States’ ability to fund basic scientific research.  Yes, that’s billion with a capital B. Devastating to the medical research community – and more importantly, to the hundreds of thousands of patients suffering from diseases for which there are no cures and few treatment options – does not begin to describe the impact of sequestration.

What Can Be Done?

• 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 competiveness, and result in job loss in communities throughout our country. Share with them why a strong investment in medical research is important to you, your family, and your community.
• Submit your story about why medical research and protecting funding for new research matters to Time=Lives.
• Attend the Rally for Medical Research on April 8th here in Washington, DC. Even if you can't attend, check out their site for more way to get involved!

Building a culture of participation in research

By LaTese Briggs Program Analyst at FasterCures

“I have been waiting 174 days to say this: 'Good Morning America.' "

IDA MAE ASTUTE/ABC

Good Morning America anchor, Robin Roberts, made her triumphant and long awaited return to the talkshow last month after a five month recovery from a bone marrow transplant to treat myelodysplastic syndrome (MDS). The show and her fans have been celebrating and supporting Roberts’ through her courageous fight and recovery. An emotional Roberts’ thanked her fans, colleagues, and doctors for helping her through her battle with the disease, but she also took the time to thank all of the courageous people that participate in clinical trials.

“The standard treatments of today are developing because patients before you participated in clinical trials, and in a certain sense it’s our obligation as physicians and the patients of today to develop the standards of care for the patients of tomorrow.”

By participating in clinical research studies, these giving volunteers play a key role in improving the standard of care for themselves and future patients. Clinical trials are critical to bringing new drugs and interventions to patients in need. In a recent editorial by Francis Collins, Director of the National Institute of Health (NIH), he explains that the wealth of discoveries related to the genetic and environmental causes of disease, have led scientists to numerous new targets for drug development; however, the rate at which new drugs and other therapeutics are reaching patients has not increased proportionately. A key impediment to the translation of these discoveries to new treatments is the poor participation rate of Americans in clinical trials. This problem has been well-demonstrated in cancer where according to the NIH, only 3% of adults with cancer are participating in clinical trials.

According to studies conducted by CenterWatch, 94% of Americans recognize the importance of participating in clinical research in order to assist in the advancement of medical science; however, 75% of Americans state they have little to no knowledge about how clinical trials work and the participation process. This staggering statistic highlights the need to raise clinical trial awareness among the general public. Even small efforts like a simple thank you to clinical trial participants from a beloved morning show anchor, can have a major impact on achieving this goal. Thus involvement from the media and high profile individuals to illuminate the importance of clinical trials can be used as a powerful tool to educate the public on the link between improvements in healthcare and clinical research.

Educating patients and non-patients alike has become infinitely easier with the advent of online registries and databases. Sites like clinicaltrials.gov help doctors, patients, friends, and family members find clinical trials soon to be conducted in their area, their specific disease or a find a call for a person with a particular medical history.

The involvement of patient advocacy groups has also proven to be an effective way to increase clinical trial awareness and patient enrollment. A good example of this is the Love/Avon Army of Women collaboration between the Dr. Susan Love Research Foundation, the Avon Foundation, and the American Association of Cancer Research. The goal of this initiative is to recruit one million women willing to donate time, biospecimens and data to breast cancer research. To date, the organization has recruited nearly 400,000 women.

In addition, the use of outreach workers to educate patients about clinical trials can also potentially have a huge impact. These workers would not only make patients aware of clinical trials, but also work to address concerns and misconceptions about clinical trials that some may have. Common concerns include fear of experimentation, logistical concerns related to costs, insurance coverage, travel, missing work and/or time with family. Some common misconceptions are that clinical trials are last-resort efforts and one should only participate when all other approved treatments have failed, placebo treatment means that no treatment will be provided, and the assumption that intervention or treatment in the clinical trial is more invasive than standard treatment.

Clinical trials are truly the crux of drug development and medical innovation. Most trials evaluate new treatment options for safety and efficacy before making them commercially available; however, clinical trials are also used to understand the root cause of disease, improve disease detection, and compare commercially available drugs to determine which are more effective in certain patients. 

By raising awareness and educating the public about the power of clinical trials, we will be able to move research forward faster such that the many patients plagued by one disease or another will be able receive new and innovative medical solutions that hopefully exceed the current standard of care.

To find out more information about clinical trials and how you can get involved, check out these links:

-- http://www.armyofwomen.org/

-- http://www.patientslikeme.com/

-- https://foxtrialfinder.michaeljfox.org/

-- http://www.alz.org/research/clinical_trials/find_clinical_trials_trialmatch.asp

-- http://www.cancer.org/treatment/treatmentsandsideeffects/clinicaltrials/app/clinical-trials-matching-service

-- http://clinicaltrials.gov/

FasterCures' Patients Helping Doctors (PHD) Program
The Patients Helping Doctors (PHD) program anchors everything we do at FasterCures. In fighting disease, patience is not a virtue—patients are. Through PHD, we are building a culture of participation in research where patients and healthy volunteers understand the fundamental value they bring to clinical research. We focus our efforts on unlocking patient information—medical records and biological material such as tissue, blood, and DNA—and making these available to clinical researchers in a meaningful way. Learn more here.

Working together towards cures for rare diseases

By Karlee Stewart, Communications Coordinator at FasterCures

7,000 different types of rare diseases and disorders

          1 in 10 Americans suffers from a rare disease

                    95% of rare diseases do not have a single FDA approved drug

                                        The numbers don’t add up.

“Take on the voice that I’m sharing with you today, reach out to your fellow patient advocates who frequently don’t see beyond their own situation and help them understand the bigger systematic challenges we all face.” – Hugh Hempel, The Addi and Cassi Fund 

On February 28, 2013 we joined friends and colleagues in the rare disease community to commemorate Rare Disease Day, the culmination of a week's worth of activities to bring widespread recognition of rare diseases as a global health challenge. 

This year, we were lucky enough to be a part of a major event here in DC kick-starting the week and celebrating the screening of Here.Us.Now a documentary highlighting the Hempel Family and their struggle to save their twin daughters, Addi and Cassi, who are living with Neiman Pick Type C. To learn more about the Hempel’s journey, check out their Time=Lives story.   

The documentary focuses on the pitfalls of an ineffective system for patients with rare diseases who have hit a wall in the search for treatment options or a cure. It features prominent members of the medical research community from academia, pharma, biotech, and patient groups calling for change in this uncommunicative and slow system.

Panel moderated by Margaret Anderson

Following the screening, we heard from National Center for Accelerating Translational Science (NCATS) Director Chris Austin during a panel moderated by Margaret Anderson. “The bad news is that there are at least 5,000 more disease like [Addi and Cassi’s] that when doctors like me see them in the clinic, all you can say is please come back to me when you need supportive care.”

Answers are what the Hempels, and others like them, are looking for. However, because rare disease research today is so siloed and disease-specific, communication between groups is limited, leading to occasional duplication of research efforts and time wasted in a world where there’s no time to waste.

“Reach out to your fellow advocates and rally them together for rare disease research,” Hugh Hempel urged the panel’s audience, “We find ourselves fighting over crumbs from funding from the NIH…let’s work together.” 

Hempel said the biggest issue that smaller, disease-specific groups have is not working together for one ultimate purpose, cures.

Besides an insightful talk and plans for collaboration between advocacy groups, here are some outcomes from Rare Disease Week: 

• Reg4All – a “community of people storing and sharing information about ourselves, our health, and our diseases– to improve health and speed therapies across all disease.” An award granted by the Sanofi US Collaborate Innovate challenge that allowed the Genetic Alliance, CFIDS Association of America, National Psoriasis Foundation and the Inflammatory Breast Cancer Research Foundation to build the registry. 

• Boehringer Ingelheim collaboration with PatientsLikeMe on the rare lung condition idiopathic pulmonary fibrosis (IPF.) The partnership will provide “a customized experience for its users that will allow them to monitor their health and progress over time and connect with other to learn more about the condition.” 

• United States Senate agreed to resolution designating February 28, 2013 as Rare Disease Day

FasterCures was pleased to participate in RDLA's 3rd Annual Rare Disease Cocktail Reception and Movie Screening

Make sure to join FasterCures on Wednesday, March 20 for our FREE Webinar, Building New Patient-Centered Research Networks: The T1D Exchange and Registries for All Diseases and learn about: 

• The T1D Exchange, which consists of an integrated clinic network of more than 65 clinics across the U.S., a biorepository, and Glu, an online community with mobile capabilities for people touched by type 1 diabetes. Its mission is to improve outcomes in the community by facilitating better care and accelerating new therapies on the path to a cure.
• Registries for All Diseases (Reg4All), which recently won the Sanofi Collaborate/Activate challenge – a cross-disease, crowdsourced registry that promises to break down the silos between diseases and gather information from patients in a standardized way.

Time=Lives Story of the Week: Blair Van Brunt

“Patient participation is really critical in terms of reaching for a cure, the word that nobody wants to mention by the way.”

Diagnosed at age 2 with Shwachman-Diamond Syndrome (SDS), a rare disease affecting the bone marrow, pancreas and skeleton that makes it extremely difficult to digest food and fight off infection, Blair Van Brunt’s daughter Gracie was lucky to have her biggest advocate in her corner. With determination to help find a cure, Blair, like many other parents, quickly sprung into action and became an advocate, a cheerleader and an authority on the rare disease that her now 18-year-old daughter fights every day. SDS, which is often confused with Cystic Fibrosis, is normally diagnosed in the first few years of life and slows growth dramatically.

After their daughter’s diagnosis, Blair and her husband decided to get involved with fundraising right away. Their continued passion and involvement in the cause eventually translated into Blair’s placement on the board of the Shwachman-Diamond Syndrome Foundation in 2004 and then to her current position as president, a role she’s held since 2008. Founded in 1994, the Shwachman-Diamond Syndrome Foundation is now a national voice and advocate for patients and families with SDS.

Watch Blair’s story here.

Because there are only around 1,000 cases of SDS in the United States, it is difficult to rally patients together for a clinical trial, Blair explains. “We need to populate the path [to cures] with our data, our stories, and our drive and passion to get all of us to move towards the same goal.”

Learning to embrace her disease and fight for others, Blair’s daughter Gracie uses music as both an outlet and a tribute. Make sure to check out her website and listen to some her amazing songs, like Thomas’ Song, a project she’s working on with Pledge Music where 10% of all proceeds will go to the Schwachman-Diamond Syndrome Foundation.

To learn more about SDS and the Shwachman-Diamond Syndrome Foundation, visit http://www.shwachman-diamond.org/. Or Like their Facebook page.

And make sure to check out the Time=Lives campaign to find out what you can do to help speed the medical research process.

-- 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: 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

FasterCures Finds Outcomes, Metrics, and Strategic Leadership Define the Effectiveness of Venture Philanthropy Groups

Two new reports outline the practices and approaches of nonprofit funders of medical research

FasterCures today released two reports that paint a vivid picture of how medical research foundations, also known as venture philanthropy groups, have transformed the medical research enterprise. These entrepreneurial groups are accelerating medical research and development by tackling science where it needs the most effort and resources, and applying innovative business approaches in the pursuit for a cure.

Both resources tell a compelling narrative of how venture philanthropy groups are creating a culture in medical research that is mission-driven, results-oriented, and focused on the true bottom line: preventing, diagnosing, and curing disease.

• The first, Honest Brokers for Cures: How Venture Philanthropy Groups are Changing Biomedical Research, features insights from leaders of 20 venture philanthropy groups. This publication features valuable insights and perspectives from leaders of medical research foundations that are transforming the cure enterprise by virtue of how they find and fund research. In this report, FasterCures dissects the business model that has emerged from the shared sense of urgency and frustration stemming from these patient-driven organizations, and their laser-sharp focus on outcomes.

• The second, Measuring and Improving Impact: A Toolkit for Nonprofit Funders of Medical Research, is a how-to guide for foundations seeking to apply some of the best practices and lessons learned from venture philanthropy groups who’ve demonstrated their effectiveness.  It provides a common framework for assessing and improving organizational effectiveness, and a panoply of ideas, questions, and models to help guide new and emerging nonprofits with strategic and tactical choices.

Venture philanthropy groups play an outsized role in improving the medical research and development system to better meet the needs of patients. Since its inception, FasterCures has continued to shine a light on the successes of these foundations in an effort to amplify their lessons learned and sense of urgency to the rest of the medical research community.

“In the end, the most central characteristic of these groups is their close connection to the disease they are pursuing. For most, it is personal—either they or a family member or close friend is or has been affected by the disease,” said Margaret Anderson, executive director of FasterCures. “It spurs them to find the dollars to meet the challenges and change the trajectory of research. We can all benefit from the lessons they've learned.”


                                                  

To download these publications, visit http://www.fastercures.org/Publications/vp.php

 

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.