Monday, December 21, 2009

Boston Blazers and Prize4Life are teaming up to fight ALS!


Prize4Life is pleased to announce a new partnership—with the Boston Blazers, Boston's professional lacrosse team!

Still looking for a holiday gift for that special someone? How about taking them to see a professional lacrosse game?

The Boston Blazers will be highlighting the fight against ALS during their "Heroes Awareness Night" home game on February 6, 2010 at the legendary Boston Garden. The Blazers are partnering with P4L to bring attention to our cause and also help raise money to further our mission. They have generously agreed to donate half of the tickets purchased through this promotion to Prize4Life.

We'd love to have all of our supporters in attendance at the event. Come out and support Prize4Life, and enjoy the evening! Tickets are $30 and can be purchased directly from us, or through a special website: click here. You have to enter the code: P4L in order to be part of the group (ticket purchasers also have an opportunity to meet the team afterwords).

Alternatively, if you’d prefer to buy your tickets through us directly, just call (617) 914-4956. We’re looking forward to seeing you there!

Thursday, December 17, 2009

NIH Approves Additional Stem Cell Lines For Funding

On December 2, the National Institutes of Health (NIH) declared that 13 human stem cell lines met new NIH ethics criteria, reports USA Today.

This approval re-opens of a source of funding closed to researchers using human embryonic stem cells since the Bush Administration’s 2001 executive order. Since 2001, researchers otherwise supported by the NIH have had to raise private monies to derive stem cells from the fertilized embryos left over from fertility clinics, reports the New York Times. In March 2009, President Obama signed an executive order which lifted that ban.

The first thirteen lines is merely the beginning of several hundred lines to be reviewed by the NIH in the next several months. NIH Director Francis S. Collins, MD, PhD, says that the NIH is reviewing new applications with extreme caution to ensure they meet ethics guidelines.

Eleven of the first 13 newly-approved lines were derived by Children’s Hospital Boston. They will be used to study rare congenital diseases. The other two stem cell lines were derived by Dr. Ali H. Brivanlou at Rockefeller University in New York.

This week (on Monday, December 14), the NIH approved another 27 human embryonic stem cell lines from Harvard University for federal research funding. The decision, however, limited funding to diabetes-related pancreatic cell experiments.

The second batch of cells, from Harvard University, have been used in studies of ALS, Alzheimer's disease, Huntington's disease, Parkinson's disease, and spinal injury, supported by other funding sources. A report in the Proceedings of the National Academies of Science last year called them the 'gold standard', says USA Today. Let us hope that these 'gold standards' lead rapidly to treatments and a cure for ALS.

Tuesday, December 8, 2009

The Evolution of Crowdsourcing

At Prize4Life, we distribute a weekly Digest of all sorts of relevant news: ALS news, news on philanthropy, news on trends in crowdsourcing, and news on industry developments that are relevant to ALS and neurodegenerative diseases. Like many organizations, we do this to keep our staff and constituencies informed of developments that can potentially affect us; on any given week we may read about how heavy metals could present a therapy option for ALS, about crowdsourced philanthropy, or about how women receive less research funding than men (by the way, if you’d like to receive the Digest, you can request to be added to the mailing list here).

In assembling the Digest recently, I have been struck by the diversity of organizations using crowdsourcing tactics to meet their goals. Prize4Life was the first neurodegenerative disease-oriented organization to establish an incentive prize and draw on the power of crowds for solutions, but it looks like we won’t be the last!

Crowdsourcing takes projects traditionally performed by employees and outsources them to a group of people in an open call for submissions. The public may be invited to develop a new technology, create a design, or analyze data. Prizes are often awarded to compensate winners, and clearly, Web 2.0 facilitates this kind of interaction. Prize4Life's two prizes are based on this very concept—we open our competition to the creativity and expertise of researchers the world over.

And crowdsourcing has a wide reach. Companies and non-profit organizations are crowdsourcing everything from purchase of a beer company to lightbulbs to maps. Yellow Tail is crowdsourcing a name for its newest wine. The first crowdsourced car hits the production line this month. Check out the list below for some of my favorite examples of crowdsourcing:

Philanthropy

Philanthropic communities are increasingly harnessing the power of crowdsourcing for social change. Take TUGG, for example (Technology Underwriting Greater Good), a foundation organized by New England’s venture capital and entrepreneurial communities, which helps youth in areas of entrepreneurship, education, and experiences. TUGG relies on the broader community for ideas on social innovation projects, to select fundable projects, and to raise money through micro-initiatives.

Translation

Facebook, Twitter, and IBM have all used crowdsourcing for large-scale translation projects. The IBM application, n.Fluent, translates between English and 11 other languages, and it embodies contributions from the company's 400,000 employees. Facebook and Twitter have relied on their users to translate entire areas of their respective sites.

Technology

Crowdsourcing has even crept into government agencies. The US Department of Energy launched the L Prize in 2007, to spur development of high-quality, high-efficiency LED light bulbs as replacements for the common light bulb.

NetFlix recently awarded a $1 million prize to a team of mathematicians who came up with a recommendation software that could do a better job accurately predicting the movies that customers would like.

NASA and TopCoder, Inc. are holding a competition to develop algorithms which would help NASA flight surgeons make better decisions on what to include in the medical supplies kit of future long-term human space missions.

Prize4Life is proud to be the first disease organization to use these concepts in our work (click here to read more about our model). We invite as wide a pool of participants as possible with no restrictions. We want to attract ALS researchers, clinicians, other disease researchers, emerging scientists, established scientists, domestic researchers, international researchers worldwide, and anyone in between, to participate in the pursuit of understanding of ALS better and finding treatments and a cure for this devastating disease.

--by Meghan Kallman

Monday, November 30, 2009

A Million-Dollar ALS Fundraiser At Northport High School

Students from Northport High School on Long Island have created their own ALS charity, "A Midwinter Night's Dream". The charity was founded in 2004 by 16 teenagers after two of their teachers were diagnosed with ALS. Students took their cues from heavyweights in the business world: Harold J. Garrecht, president of Eastshore Partners, donated to the project, and offered to teach the young people how to do some major-league fundraising. He provided guidance on how to put on a gala dinner, coached them on cold-calling potential contributors, and gave them a list of wealthy donors to approach. As a result, "A Midwinter Night's Dream" has become a million-dollar success story in only five years. The New York Times recently ran a piece detailing the teenagers' exciting progress; read it here. Northport High School student and "A Midwinter Night's Dream" participant Blair Ingraham shares perspectives on the project below.

I could not stop smiling. I had finally heard the words that I had been waiting to hear for eight months: “We have surpassed the one million dollar mark.” I could not believe we had finally accomplished the goal we had been working towards for so long. I looked to my right only to find all 550 guests standing and clapping with tears brimming their eyes. We were all hugging and screaming and crying with each other, all thinking to ourselves that this was the most amazing night we have ever experienced and wishing we could live in this moment forever.

Who would have known that a phrase as simple as “we have surpassed the one million dollar mark” could have so much meaning? To a group of forty-two high school students dedicated to finding a cure for ALS, it meant everything. At that moment, we bound together and celebrated a major feat for A Midwinter Night’s Dream: raising one million dollars for ALS Research over five years.

That moment has inspired our school and our community to keep fighting and has given hope to all of the ALS patients we visit. There are no words to describe how I feel every time that moment is replayed. Whether it be the first time or the hundredth time I have seen it, only the goose bumps on my arms can express my emotions. It amazes me every day to see how passionate, dedicated, and selfless the committee members are. I can honestly say that I am part of the most influential organization I have known, and I couldn’t be prouder.

From the moment I was told I would be a member of A Midwinter Night’s Dream after a long selection process, I knew it would be a great experience, but I never knew how much impact it would have on me, my school, and the ALS community. This committee has taught me to be a strong, confident leader and a good communicator; two qualities that will help me succeed in the real world. Members of our committee are role models for our school and for our small town. We have made a name for ourselves not only locally, but throughout the country. How many groups of high school students can say that?

Our committee’s efforts will not stop until a cure is found. We will not give up and not back down until this horrible disease is gone. We do it for all those afflicted by ALS. We do it for our heroes. There are a million reasons why we continue to fight… choose one and join us!

Thursday, November 19, 2009

E-patients

On Monday, November 16, NPR did a piece on what it terms called “e-patients”. According to the article, “61 percent of adults say they look online for health information. There's a term for them: e-patients.”

The article goes on to note that most e-patients go online to read about a health condition or a potential health condition. To be sure, there are a plethora of sites offering information on diseases and conditions (such as WebMD and HealthCentral). This approach has raised concerns over the possibility of patients self-diagnosing and misdiagnosing, due to incomplete information found online or a lack of expertise.

But there has been a new trend in the behaviors of e-patients. A Pew study notes that approximately 20% of those e-patients now go to social networking sites where they can talk to medical experts and other patients, says the Pew Internet and American Life Project.

“They are posting their first-person accounts of treatments and side effects from medications,” says Susannah Fox of the Pew Internet and American Life Project in the NPR story. “They are recording and posting those podcasts. They're tagging content. They are part of the conversation. And that, I think, is an indicator of where we could be going in terms of the future of participatory medicine.” She notes that internet sites have grown voraciously in the past several years, and that a “much deeper level of information” is available now.

The ALS community has clearly been deeply involved with this kind of social-media transformation, and much of it relies heavily upon these mechanisms of communication. Many PALS are familiar with Jamie Heywood’s site, Patients Like Me. The site is a for-profit entity designed to be a “treatment, symptom and outcome sharing community for patients with life-changing conditions.” The platform “enables patients the opportunity to share their personal stories and health information in a way that illuminates great ideas and new knowledge about their diseases.”

Heywood created Patients Like Me because he was trying to find out about research advances that might help his brother (a PALS, who later died of the disease), and the site eventually grew to encompass other diseases. Patients Like Me currently has about 50,000 members.

Building on concepts of sharing and open information, Heywood suggests, is what makes his site revolutionary. “The amazing shift is that we've pushed out this concept of sharing,” he said. “Which is to say: If you share information about your own experience with this disease, then we can facilitate the conversation that you want to have with the person in the world who is just like you — whether they live in Vancouver, Canada or Australia, or down the block.”

A resource like this is meant to enable patients to find someone else who “is on the same treatments, is dealing with the same side effects ... whatever variable matters to you at that moment. To find out whether your concerns are justified, they make sense, whether you're doing the right thing — that's the transformation,” Heywood says.

Prize4Life is not alone in recognizing the importance of such virtual stores of information. In fact, in a project to provide both researchers and patients with a one-stop shop for good information on ALS, Prize4Life recently created the ALSForum, in conjunction with the Alzheimer's Research Forum.

The same Pew study found that 39 percent of e-patients typically use another social media site such as Facebook or Twitter. For ALS patients, assistive technology is clearly a lifeline, and Facebook, Twitter, and Patients Like Me are channels through which patients can more easily interact with each other and with the world.

Prize4Life was, however, pleased to see the NPR piece note that patients say they trust their most traditional resource the most when it comes to the hard questions of disease, diagnosis and treatment: their own doctor.

Wednesday, November 11, 2009

Honoring Veteran PALS in the Fight of their Lives

As we celebrate our Veterans this Veterans Day, let’s especially recognize those veterans who are suffering from ALS. A disproportionate number of them are.

In recognition of this, in September of this year the U.S. Department of Veterans Affairs granted veterans diagnosed with ALS full access to health and disability benefits. The policy was effective immediately, and covers all veterans who have served a minimum of 90 consecutive days. When and where they served is irrelevant to their eligibility for services.

The VA cited a compilation of medical studies in deciding to expand coverage, Amyotrophic Lateral Sclerosis in Veterans: Review of the Scientific Literature, issued by the National Academy of Sciences’ Institute of Medicine, and released in November 2006. It concluded that "there is limited and suggestive evidence of an association between military service and later development of ALS.” Other organizations, such as the ALS Therapy Development Institute (ALSTDI), were responsible for advocating for expanded ALS attention and funding (read ALSTDI’s coverage).

According to the study, veterans within the last century are almost twice as likely to develop ALS non-veterans. “[ALS] continues to take an inordinate toll on our nation's veterans," said Dr. Stanely Appel for ALSTDI in his prepared testimony before the Military Construction and Veterans Affairs Appropriations Subcommittee earlier this year.

It is not clear why ALS occurs more frequently in veterans, though it has been suggested that the anthrax vaccine administered to servicepeople does play a role.

Regarding the decision to expand coverage, VA Secretary James Peake said in a statement, "Veterans are developing ALS in rates higher than the general population, and it was appropriate to take action. ALS is a disease that progresses rapidly, once it is diagnosed. There simply isn't time to develop the evidence needed to support compensation claims before many veterans become seriously ill. My decision will make those claims much easier to process, and for them and their families to receive the compensation they have earned through their service to our Nation."

The VA was in also the news with respect to ALS in late August due to a regrettable oversight—it mistakenly notified 1,200 Gulf War veterans across the country that they suffer from ALS (see the full story here). It subsequently apologized, blaming a coding error for the mistaken letters.

We encourage veteran PALS to take advantage of the services available to you (see a complete list here). On this day, let us support and encourage those veterans who are battling ALS in the fight of their lives.

Monday, November 9, 2009

November is National Family Caregivers Month

National organizations and community and advocacy groups across the country are observing National Family Caregivers Month in November. National Family Caregivers Month was created by presidential proclamation to call attention to the challenges facing family caregivers, and to raise awareness about programs to support them.

The Department of Health and Human Services estimates that approximately 52 million family members, friends, partners, and neighbors in the US care for their chronically ill or disabled loved ones. A study from the AARP calculates that the economic value of the services family caregivers provide for "free" is estimated between $350 and $375 billion yearly in the US.

The AARP also notes that “The unpaid services family caregivers provide are not without cost to caregivers and society. Lost time at work, lost benefits, and declining health can add to the emotional and physical strain of caring for a loved one. The study underscores the need to better support family caregivers through programs that provide respite (a break from caring), tax credits, information, and other supports.”

“This year we are encouraging people to speak up during National Family Caregivers Month,” said Suzanne Mintz, president and CEO of the National Family Caregivers Association, of November’s awareness activities. “One of the most important attributes of being an advocate for your loved one is the willingness and the ability to speak up and keep your eye on the ultimate goal, protecting not only the health and safety of your loved ones, but your own as well.”

Around the country, ALS caregivers such as Kathy Thompson of Virginia Beach, are coming up with their own projects to support awareness this month. Thompson, who cares for her son, put together the “Hopes and Dreams Quilt Challenge for ALS”, which is designed to “warm the hearts and laps of ALS patients” (see the full story here). The ALS Quilt Challenge is receiving quilt submissions for ALS awareness. The winning submissions will be auctioned off, and the proceeds donated to the ALS Association in support of research. Quilts that are not offered for purchase will be donated to people and families living with ALS.

In its own campaign to recognize caregivers, the ALS Association (ALSA) is encouraging caregivers to voice their concerns about their own health as well as the people who live with their assistance. ALSA is also setting up a series of profiles, to be exhibited on its website for the duration of National Family Caregivers Month.

To the CALS who give so generously of their time and energy, thank you for all that you do.

--by Meghan Kallman

Monday, November 2, 2009

CIRM Awards Grants Supporting ALS Research

There has been another big investment to fuel ALS stem cell therapies. The California Institute for Regenerative Medicine (CIRM) has awarded support to 14 pre-clinical projects this year. This comes in the context of other recent support for such projects: Neuraltem was recently approved for a Phase 1 ALS clinical trial using a stem cell approach, and BrainStorm Cell Therapeutics, of Petach Tikva, Israel, a competitor for Prize4Life's Avi Kremer ALS Treatment Prize, also recently secured funding to complete pre-clinical stem cell trials for ALS, and expects to commence in early 2010.

The InVivo blog reports that the CIRM’s international partners contributed funding, bringing the total awards to an unprecedented $250 million.

Particularly interesting for our purposes is the $15.6 million grant given to the Salk Institute for translational research. The support is meant to focus on developing a novel ALS therapy using a stem cell approach. Notably, this award is the first CIRM funding explicitly expected to result in FDA approval for clinical trials.

Dr. Sam Pfaff, investigator at the Howard Hughes Medical Institute and professor at the Salk's Gene Expression Laboratory, has been named Principal Investigator (co-PI's are Dr. Larry Goldstein and Dr. Don Cleveland, both from UCSD). Dr. Pfaff will lead research on the four-year project.

The grant will support research focusing on the role of astrocytes in treating ALS. Astrocytes are star-shaped cells that provide nutrients for nearby motor neurons, jointly acting as a support system. Pfaff, Goldstein, and Cleveland will cultivate astrocyte ‘precursor’ cells—astrocytic ‘seeds’—and identify those best suited to be implanted in animal models. They hope that, by implanting astrocyte ‘precursors’ into animals, the precursors will fully mature into astrocytes and provide support for sick motor neurons. Because astrocytes are also able to ‘clean up’ the toxic areas around diseased motor neurons, they could potentially slow or stop the progression of the disease.

When the astrocyte 'precursors' have been tested for efficacy and safety, and have been approved by the FDA, the team will move into clinical trials—that is, they will test this approach in humans.

Additionally, one San Diego-area biotechnology company was selected to receive a CIRM grant. Academic researchers have historically received the majority of such funding, and industry executives have been displeased with the lack of opportunities available to companies. Though CIRM is one of the largest sources of funding for human embryonic stem cell research in the world, only a handful of its hundreds of grants have ever been awarded to companies.

by Meghan Kallman

Tuesday, October 27, 2009

Enthusiasm and beautiful weather for Inaugural 5K4Life!



Last Sunday, October 25, hundreds of people joined forces in Prize4Life’s first annual 5K4Life, a road race to raise money for Prize4Life and ALS research. We had a beautiful day, a terrific turnout, and lots of enthusiasm. Runners came from colleges and universities, from local running clubs, from corporate, biotech, and research organizations, and from the Prize4Life family.

Prize4Life was lucky to have the help of volunteers from the East Boston High School Key Club, who arrived bright and early to help set up. They distributed t-shirts, helped stuff race bags, and served food. Other dedicated volunteers lent a hand at the registration table, the water stop, and the finish line.



People surged across the start line when the horns sounded, wearing electronic timing tags. Participants ranged in age; most ran the course (11-year-old Eliza from Natick took fourth place in the female 1-19 division and Dr. Seward Rutkove, who won one of Prize4Life's progress prizes, took second place in his age group). Some folks and families with young children walked, including Prize4Life Board members and a 54-year man old recovering from a double lung transplant. Corinne Casey won the overall women’s division, and Robert Cipriano won the overall men’s division. See the full results (including results by age group) here.



'White Collar Crime,' Boston's leading benefit band, played upbeat music as the runners started off, and all through the post-race picnic. After the race runners and spectators mingled, enjoying a picnic lunch and the pleasant day, and discussing Prize4Life with the staff and Board members present.

Sponsorship for the race was generously provided by Genzyme, Biogen IDEC, Shire, Microsoft, Harpoon Brewery, Marathon Sports, Barker Graphics, Recognition Services Incorporated, the Grossman Marketing Group, White Collar Crime, Save That Stuff, and Yogi Cereal.

The event was a tremendous success its first year (see the list of top fundraisers and fundraising teams here). Runners and participants were very positive about the day.

“We all thought it was a really well run, efficient, organized event with a fantastic course! Thanks so much for your work – we had a blast and will definitely be doing it again next year!” --Vanessa

"I thought it was a good race through a great part of the city!" --Allen

We’ll have a picture gallery up shortly, so keep an eye peeled for more great photos. An extra shout-out to all of our volunteers--we couldn’t have done it without you! Thanks to everyone who ran, raised money, or cheered us on, and we hope to see you at the 2010 5K4Life!

--by Meghan Kallman

Tuesday, October 20, 2009

"Trapped" event a great success

On October 14 Allison Kotzin and Peter Bowen, two former Prize4Life Board Fellows, organized and hosted an event at the 28 Degrees Lounge in Boston. It was a screening of the ALS film “Trapped”, held as a fundraiser for Prize4Life, and it raised over six thousand dollars (with donations still coming in).

“Trapped” is a provocative new film shining a bright light on the plight of individuals and families living with ALS. The film chronicles the life of a brilliant young composer who discovers he has ALS after an eerie progression of symptoms. Though he loses his ability to physically write music and play the piano, he refuses to cede his spirit and genius to the disease. The film was written and produced by Jim Mahoney and Zach Lewis, and directed by James Takata. It boasts music by Michael Mollo, and was written at the urging of Amy Yamner, the Chair of Prize4Life’s Board (read an interview with Takata, Mahoney, and Lewis here).

Yamner had seen a film about Alzheimer’s disease that had generated attention and moved Alzheimer’s into the public eye; she thought a similar project could be undertaken with ALS. She was right. The film is evocative and haunting, and showcases both the horrors of ALS and the talent of its writers, producers, and actors.

Allison and Peter put on a great party, and Prize4Life co-founder and CEO, Avi Kremer, was present for the evening. Prize4Life President James Giessler gave a moving speech after the screening, and Prize4Life was fortunate to be able to hold a silent auction as well. The 28 Degrees lounge generously provided the space and appetizers for the event. Prize4Life supporters from near and far came to see the movie, talk with each other and with staff, and to hear about Prize4Life’s new work.

The event showed that, even in these difficult economic times, Prize4Life’s dedicated volunteers are able to convene generous donors and an engaged support base. Donors and activists are willing to support compelling causes, and “Trapped” is an incredibly artistic, moving tool that raises both awareness and funds for ALS and Prize4Life. Do you know a group of people who would be interested in seeing the movie? Could you host a screening for us? Send us a note at contact@prize4life.org, and together we can push for more research, more treatments, and someday soon, a cure.

by Meghan Kallman

Wednesday, October 14, 2009

"Fight Smart, Not Just Hard"

In the September/October issue of Neurology Now, Dr. Richard Bedlack, director of the Duke ALS Clinic in Durham, NC, wrote an opinion piece entitled “Fight Smart, Not Just Hard: How I’d Battle ALS If It Happened To Me.”

“What would I do if I received a diagnosis of ALS, or if one of my loved ones did?” he asks. An ALS researcher with years of experience behind him, Dr. Bedlack advocates ways to live as healthily as possible with ALS, supporting research and awareness on the road to a cure. In the piece he writes,

Like most of the 1,000 or so patients with ALS and their caregivers that I have seen over my last decade in this field, I’m sure I would fight it...But without all my years of scientific and on-the job-training, would I know the best direction to go in?

A Google search on ALS would certainly tempt me to consider that the diagnosis was wrong. Maybe it’s Lyme disease or heavy metal poisoning, I would wonder. Further browsing of the Internet and conversation in online chat rooms would make me want to try a number of alternative or off-label therapies for the disease...but...I have learned that there is a better way. I’ve seen data showing that the diagnosis of ALS, when made by an experienced neurologist, is almost always correct. Therefore, once I had an opinion from an ALS expert, I wouldn’t need to expend a lot of energy considering other possibilities.

In addition, ALS experts have already looked critically at many of the alternative and off-label options being pursued and found no merit to them. Thus, instead of furiously chasing these down, I would pursue the treatments that have been shown to prolong life and/or preserve quality of life; these include riluzole, early bipap, optimal nutrition, and care in a multi-disciplinary clinic.

Realizing that research is the only sure way that I (and everyone else with ALS now and later) will get closer to a cure, I would try to participate in every study that I could, monitoring legitimate Web sites like clinicaltrials.gov to look for opportunities.

Knowing that this is a rare disease, and one in which research funding is desperately needed, I would “burn and rave” as an advocate, doing all I could to raise awareness...I would travel to National ALS Advocacy Day every year to make sure my elected representatives understand how tough this disease is and that they continue to support programs that help ALS patients and caregivers. I would stay in this fight even if my loved one eventually lost his personal battle with the disease.

In the end, would the things I’ve learned ultimately save me or my loved one from ALS? Unfortunately, they might not...experience treating people with ALS simply maps a path which optimizes the possibility that something good will come from all the valiant efforts.

Like Dr. Bedlack, Prize4Life is working hard to accelerate research to find a cure. As ALS Advocacy aptly points out, sometimes it's hard to fight smart when it's not a fair fight. We admire all the brave PALS who have worked and continue to work tirelessly to raise money, awareness, and to promote research to treat and cure ALS. Thanks again for all you do.

by Meghan Kallman

Friday, October 9, 2009

Data Sharing

Andrew Vickers is a biostatistician working on cancer. Throughout his career, he has requested underlying datasets from several researchers at different times to help advance scientific studies, and has been refused more often than not. He was perturbed enough to write an essay in the New York Times about it, in which he advocated making clinical trial data freely available.

Vickers’ piece discussed the various difficulties he has encountered trying to obtain data that could “make an immediate and important impact on the lives of cancer patients". The reasons that researchers were reluctant to share data included:
• the potential that analyses could be undermined
• the original research team might “consider a similar analysis at some point in the future”
• privacy concerns
• unwillingness to co-operate
• the “difficulty of putting together a dataset”
• potential for misinterpretation or misrepresentation

“Given the enormous physical, emotional and financial toll of cancer, one might expect researchers to promote the free and open exchange of information,” Dr. Vickers wrote. “The patients who volunteer for ... trials often suffer through painful procedures and harsh experimental treatments in the hope of hastening a cure. The data they provide ought to belong to all of us. Yet ... researchers typically treat it as their personal property.”

Dr. Vickers cited the work of Dr. John Kirwan, a rheumatologist from the University of Bristol, who has studied researchers’ attitudes on sharing clinical trials data. Dr. Kirwan found that three-quarters of researchers he surveyed, as well as a major industry group, opposed making original trial data available.

In an article published in Nature magazine on the same topic (“Making Clinical Data Widely Available”), Jocelyn Kaiser writes on difficulties of sharing raw data among scientists. The issue is complex; researchers often embrace the idea of sharing, which can open channels for independent scrutiny, foster collaborations and encourage new discoveries in old data. In practice, however, those advantages often fail to outweigh researchers' concerns.

Some researchers in her article were concerned that open-access could lead to erroneous interpretations. Epidemiologist Bruce Psaty warned that investigators could conduct erroneous risk analyses, or that young researchers could become too dependent on data mining, and neglect designing their own rigorous studies.

Kaiser also notes that, “clinical investigators are understandably reluctant to hand over datasets in which they have invested years and that they hope will generate many papers.”

“They don’t want to be scooped,” says Christine Laine, senior deputy editor of the Annals of Internal Medicine.

Dr. Vickers frames it differently. “This is exactly what...patients need. They want new results to be published as quickly as possible and to encourage a robust debate on the merits of key research findings.” Reidpath and Allotey agree, writing that this “highlights an unfortunate situation where researchers are more concerned with losing an advantage than advancing science.”

“It is worth restating this finding: most scientists doing research on how best to help those in pain, or at risk of death, want to keep their data a secret.” Dr. Vickers finished.

However, the trend of keeping data private is perhaps beginning to change. Data-sharing advocates say that the power to encourage data sharing rests largely with those who have always had the most clout in science: the funding agencies, which can require data sharing in return for support, or journals, which can make sharing a condition of publication. Many such agencies have begun to encourage data sharing through those mechanisms.

In her article, Kaiser points out that: “A law enacted in 2007 will require sponsors of all clinical trials of drugs and devices that were subsequently approved in the United States to post summary results in a federal database. The aim is to ensure that all findings see the light of day, including negative results that often get buried. At the same time, the National Institutes of Health (NIH), which ramped up its data-sharing efforts with genome data more than a decade ago, has been advancing these policies into clinical research.”

This trend appears to be spreading slowly across different parts of the scientific landscape. ResearchGate recently launched a Self­Archiving Repository, which provides members with free access to potentially millions of research papers without the obstacle of library subscriptions or the financial barrier of pay-per-view.

In a broader mandate for sharing Data, the NIH policy states that “NIH reaffirms its support for the concept of data sharing. We believe that data sharing is essential for expedited translation of research results into knowledge, products, and procedures to improve human health. The NIH endorses the sharing of final research data to serve these and other important scientific goals. The NIH expects and supports the timely release and sharing of final research data from NIH-supported studies for use by other researchers.”

Nature magazine’s policy is similar. “An inherent principle of publication is that others should be able to replicate and build upon the authors' published claims. Therefore, a condition of publication in a Nature journal is that authors are required to make materials, data and associated protocols promptly available to readers without preconditions,” says the website.

Similarly, the Annals for Internal Medicine asks that their technical studies include a reproducible research statement explaining the study protocol, dataset, and statistical code. Researchers are not required to do so, and many simply say that data are not available, Kaiser reports. However, Christine Laine “views this policy as a tiny, baby step towards changing the scientific culture.”

by Meghan Kallman

Tuesday, October 6, 2009

"Just being here is already a victory," by guest blogger Aimee Chamernik

Just being here is already a victory.

That’s what I keep telling myself, anyway. As my family and I stand near the starting line of this year’s ALS Walk4Life, basking in the sunshine and the positive vibes emanating from the 100+ people on our team, I’m trying hard to prepare myself for the disappointment of not being able to complete this two-mile Walk.

And while it’s true that just being here at all—-an astonishing five years after my ALS diagnosis—-is something I hardly dared hope for, I’ve also been secretly telling myself that if there’s any way sheer force of will can make a difference, I will cross the finish line on my own two feet.

Because this year, the Walk is more personal than ever. I can’t stop thinking about our ALS support group friends who died this year. I can’t stop thinking about the online friends I’ve made and lost. I don’t know why I lucked out to still be here five years later, and frankly, I feel more than a little survivor’s guilt. I do know that if it’s at all within my power to complete this Walk I will do it to honor them.

As teams take off across the starting line, we see many, many people wearing t-shirts or carrying signs indicating that they are walking in memory of a loved one who has lost the ALS battle.

My mind shifts to the countless other walks held for cancer, AIDS, diabetes, and so many other horrible diseases. I can’t help but wonder about the numbers of teams walking with a patient or survivor of those diseases versus those walking in memory of a patient who has died. Is it possible that any other walk has a worse ratio than we do? Given the merciless pace of ALS and the lack of any effective treatment, I doubt it.

And that leads to one of my major frustrations with this disease, as well as one of my major motivations for trying to raise awareness and money. How is it possible that 70 years after Lou Gehrig died, the prognosis for a newly diagnosed ALS patient is no better than his was way back then? How is it that the life expectancy hasn’t budged? How is it that so many other diseases—as heartbreaking and insidious as they are—at least have treatment options? That they have survival odds somewhere above 0 percent?

As we walk, we are confronted with signs quoting familiar statistics many ALS families could recite in their sleep. Each one is more dismal than the last. But as I read them, my anger builds. And, in turn, so does my determination. I can’t tell if my heart rate is rising from exertion (probably) or from impatience (probably that too) at the unchanging stats.

These stats, while true, are too sterile. These stats, disheartening as they are, don’t even begin to convey the harsh reality of the thousands of families living with ALS. They don’t convey what it’s like for a little girl to watch her dad’s strong frame shrink, for her to help feed him when he can no longer lift a spoon, for her to bat away mosquitoes that he can’t. They don’t convey what it’s like for the boy who remembers his mom playing tag and follow-the-leader with him, who sees her choke and sputter and gasp after taking a simple sip of water, who wonders whether she can hang on until he can grow up and find a cure.

I pause to catch my breath and take in the scene. Teams have been passing us all along, and our group is likely the last one on the course. I want so much to quit, to succumb to the fat cushion on my wheelchair’s seat that’s been taunting me for nearly two miles. We’re in the home stretch of the Walk now, and I’m barely moving.

As I take a deep breath and stumble onward, I see a kind of parallel between this stage of the Walk and the ongoing search for that elusive treatment or cure. Where are we in terms of progress toward our goal? Are we in the home stretch? Or barely moving? Seventy years since Gehrig’s death, I sometimes wonder.

Finally, mercifully, the finish line comes into view. I tumble into my wheelchair, out of breath but exuberant, and look around at all the young kids from our team cheering along the sidelines.

And it is in their faces that I see the other reason I wanted to finish this Walk. When those children grow up, will they have friends who have ALS? Will the prognosis still be the same? Or will the breakthrough finally have come? Will they have friends who are managing ALS while living fairly normal lives? Or friends who had ALS, were treated, and now are ALS-free?

That would be the sweetest victory of all.

by Aimee Chamernik

Friday, October 2, 2009

The "Stealing Pieces" exhibit travels Florida

One of the more moving ALS awareness campaigns of 2008 came from the ALS Society of Canada, showing a father’s deterioration. “Sarah’s Story”, an extremely powerful ad by the UK MND Association, was banned earlier this fall. In 2009, the Florida chapter of the ALS Association (ALSA) launched its own moving ALS awareness installation entitled “Stealing Pieces.” The exhibit has been travelling across the state since early 2009, and is booked into 2010.

At first glance, the “Stealing Pieces” installation looks like an experimental fashion project. More than one hundred mannequins, dressed only in black t-shirts, are positioned in precise rows across parks, malls and other public areas, spaced to resemble a graveyard.

Upon closer inspection the shirts read: “Lou Gehrig's disease is stealing our loved ones piece by piece,” (see a close-up photo here). On the back of each shirt is the name of a Floridian affected by the disease, their birth year, and sometimes their year of death. The mannequins are armless, legless, headless, and scarred with use. The ultimate impression is eerie and haunting.

The installation was conceived by Tampa’s Dunn & Co. advertising agency, who donated its time and resources to the creation of the exhibit. Conceptually, the campaign was intended to mimic the disease progression in ALS patients.

“So the idea of the mannequins is that ALS patients are literally, completely paralyzed. They lose their arms and their legs,” said Kamden Kuhn of the ALS Association. “I think that's why we’ve seen some people absolutely moved to tears...Symbolically they have already been stolen, piece by piece by Lou Gehrig’s disease. We want people to see that and we want it to affect them emotionally, but also affect them to take action.”

This exhibit has moved observers, and provided more dimension to an ongoing debate on shock tactics in public awareness campaigns. One online commenter observed, “Definitely creepy. But, you know, it got my attention, which is tough these days. And it's probably going to stay with me awhile. So...kudos.”

“The display is powerful and so very true,” said another. “Lou Gehrig's disease stole my loved one piece by piece.”

As occurred with “Sarah’s Story”, the ALS Association of Florida saw a significant increase in the number of volunteers and donors during the first few months of the exhibit. ALSA has said that more than 60% of donations to the exhibit come from first-time donors, indicating that this project is indeed powerful.

by Meghan Kallman

Monday, September 28, 2009

NIH Gives 115 Research Awards to Encourage Innovation

Supported in part by the American Recovery and Reinvestment Act, the National Institutes of Health (NIH) announced last week that it is awarding $348 million to encourage investigators to explore bold ideas that have the potential to catapult fields forward and speed the translation of research into improved health.

The range of awards is granted under three innovation-based research programs: the NIH Director’s Transformative R01 (T-R01) Awards, the Pioneer Awards, and the New Innovator Awards.

The NIH Common Fund encourages collaboration and supports a series of exceptionally high impact, trans-NIH programs known collectively as the NIH Roadmap for Medical Research. The Roadmap is a series of initiatives designed to pursue major opportunities and gaps in biomedical research that no single NIH institute could tackle alone, but that the agency as a whole can address to make the biggest impact possible on the progress of medical research.

These award programs are part of the NIH Roadmap, funded through the Common Fund and managed by the NIH Office of the Director and the National Institute of General Medical Sciences.

"The appeal of the Pioneer, New Innovator, and the T-R01 programs, is that investigators are encouraged to challenge the status quo with innovative ideas, while being given the necessary resources to test them," said NIH Director Francis S. Collins, M.D., Ph.D. "The fact that we continue to receive such strong proposals for funding through the programs reflects the wealth of creative ideas in science today."

Since no budget cap is imposed and preliminary results are not required for submission, scientists are free to propose new, bold ideas. They also have the flexibility to work in teams if the complexity of the research problem demands it.

The NIH expects to make competing awards of $30 million to T-R01 awardees, $13.5 million to Pioneer awardees, and approximately $131 million to New Innovators in Fiscal Year 2009. The total funding provided to this competing cohort over a five-year period is estimated to be $348 million.

This year's awards make the largest number of Pioneer and New Innovator awards in the programs’ history. Investigators funded via the 2004 cohort, the first year of the Pioneer Awards, have completed their projects. Details on their progress is available here.

Several of this year's awards were given to scientists researching neurodegenerative diseases and neurological disorders. For descriptions of the 2009 New Innovator Award recipients’ research plans, click here. For descriptions of the 2009 T-R01 recipients' research plans, click here. Information on the Pioneer Award is at, including information on this year's awardees, is available here.

by Meghan Kallman

Tuesday, September 22, 2009

Neuralstem, Inc., receives FDA approval for Phase 1 ALS clinical trial

Neuralstem, Inc., a Maryland-based biotech company, announced yesterday that the FDA had approved its application to conduct a Phase I safety trial of its stem-cell based approach to treat ALS (read the Reuters press release here).

The FDA’s approval makes Neuralstem the first company to commence a stem cell trial for ALS. The trial will examine the safety of Neuralstem's cells and the surgical procedures required for delivering injections of these stem cells directly into the human spinal cord. The FDA's approval represents a significant step toward realizing the promise of stem cells for helping damaged neural cells in humans.

This trial will be led by Dr. Eva L. Feldman, M.D., Ph.D., Director of the University of Michigan Health System ALS Clinic and the Program for Neurology Research & Discovery, and Dr. Jonathan Glass, Director of the Emory Neuromuscular Laboratory and Director of the Emory ALS Center. Both researchers are renowned for their study and treatment of ALS patients.

Neuralstem expects to conduct the trial at Emory University with Dr. Jonathan Glass, M.D., as site Principal Investigator (PI) and with Dr. Nicholas Boulis, M. D. performing the neurosurgery. The overall PI for the ALS trial program will be Dr. Eva Feldman, M.D., Ph.D.

The 18 ALS patients participating in the trial will be treated through a series of spinal injections of Neuralstem’s patented human neural stem cells. This first trial will primarily evaluate safety of the cells and the surgical procedure. The FDA has approved the first stage of the trial, which will consist of 12 patients who will receive five-to-ten stem cell injections in the lumbar area of the spinal cord. The patients will be examined at regular intervals post-surgery, with final review of the data to come 24 months later.

Neuralstem focuses on treatments for major central nervous system diseases, including ALS. Pre-clinical work has shown Neuralstem's cells to extend the life of rats with ALS (as reported in the journal Transplantation, October 16, 2006, in collaboration with Johns Hopkins University researchers), and also reversed paralysis in rats with Ischemic Spastic Paraplegia, (as reported in the journal Neuroscience, June 29, 2007, in collaboration with researchers at University of California, San Diego).

Prize4Life congratulates Neuralstem for its impressive work, and is excited to see the FDA take this important step towards actualizing stem cells as a potentially promising therapy.

Prize4Life also congratulates Israel-based biotech company BrainStorm Cell Therapeutics, who recently-secured funding to complete pre-clinical stem cell trials for ALS. A competitor for Prize4Life’s Avi Kremer ALS Treatment Prize, BrainStorm expects to begin Phase I clinical trials in early 2010. BrainStorm’s new funding includes both a prestigious grant from the Israeli government’s Office of the Chief Scientist, as well as private investment.

This FDA approval indicates a major change in the ALS landscape, making it easier for subsequent researchers to gain FDA approval and to move forward with testing other stem cell therapies. Prize4Life is thrilled on behalf of PALS, for whom Neuralstem’s discovery may signal an advance towards lifesaving treatments. Congratulations to all!

by Meghan Kallman

Tuesday, September 15, 2009

AIDS Video Ad Aired--Why Not "Sarah's Story"?

In a publicity push similar to that of “Sarah’s Story”—which Prize4Life blogged on several weeks ago—German public health organization Regenbogen will air a provocative TV ad next week. Hitler, Saddam Hussein, and Josef Stalin figure into a series of highly sexualized video ads as part of a shock campaign promoting World AIDS Day. The provocative commercials, which end with the tagline "AIDS is a mass murderer", aim to scare young people into using condoms by associating the deadly disease with political dictators, reported ABC News (The Telegraph also covered the story).

The ad opens in a darkened bedroom with a man and a woman in bed. The man is a look-a-like of the German dictator. The tagline reads: "AIDS is a mass murderer - Protect yourself!"

The project is the brainchild of German charity, Regenbogen, whose website reads: "Up until now 28 million people have died. And every day there are 5,000 new cases. Which is why AIDS is one of the most effective mass murderers in history."

Some HIV organizations have distanced themselves from the ad, complaining—in a familiar argument—that shock tactics are ineffectual. Some agencies claim also that the tactics serve only to stigmatize the infected and to guilt women especially.

However, the campaign’s producers see the fuss—and the fact that the videos are being watched thousands of times on YouTube—as a sign that their plan is working.

Dr. Amir Afkhami, instructor of psychiatry and behavior sciences and Global Health at the George Washington University, supports the initiatives. "It's effective because it raises awareness of the risk factors...This issue has come up among activists in the U.S. and there have been arguments that there needs to be more shock value," he said.

A 2003 study of by Darren Dahl, published in the Journal of Advertising Research found that shock tactics in AIDS ads significantly increase "attention and retention" of the message. Other studies, including one from Ohio State University, show that appearing to peoples’ fears are "powerful persuasive devices."

"It should encourage conversation about disclosure [of HIV status]," said David S. Novak, a senior public health strategist for Online Buddies Inc. "What is terrible is holding a secret and the secret disease inside of them." Afkhami notes that "The real failure on the part of health care advocates and social advertising has been raising awareness."

Prize4Life is perplexed by the apparent double standard here. These ads—which in some ways are more controversial than the “Sarah’s Story” campaign—have encountered less negative reactions, and have been allowed to remain on the air. The press has commented rationally and academically on the use of shock tactics that Regenbogen employed. Graphic ads warning against the dangers of drunk driving, drug use, and texting while driving, are all considered appropriate fodder for public television.

Given that Regenbogen’s ads have not been removed for their ‘disturbing’ features, we wonder what, exactly, differentiates the shock tactics employed here with the ones used in filming "Sarah’s Story". Is it because HIV/AIDS is well-known, and ALS is not? Is an ALS shock campaign less palatable because ALS' cause is largely unknown, whereas preventative measures are effective in controlling HIV infection?

Whatever the case, we consider it telling that this series of ads, while it has received its fair share of buzz, was allowed to remain public, while “Sarah’s Story” was banned. We support Regenbogen’s initiative, and we believe that “Sarah’s Story” should join it on the airwaves.

As always, Prize4Life believes that sanitized presentations of illnesses are not effective or appropriate—that by ‘cleaning up’ images of HIV or ALS, we are able to ignore them, and that ‘clean’ publicity detracts attention from diseases that could be used to find a cure.

Like ALS, HIV/AIDS is brutal; Prize4Life believes that awareness can help lead to a cure, and that the pain inflicted by disease progression is not ‘too shocking’ to be public, especially in comparison to other things that are publicly aired without controversy (rape, violence, etc).

One commenter on the ABC story wrote, “I think the whole point of the ad is to shock and encourage a rethink of your actions. It's supposed to be disturbing. All the complaints prove it's effective. Nicy-nice ads don't work.”

We think that applies to ALS, too.

by Meghan Kallman

Wednesday, September 2, 2009

Translational Research

As with any advance in human health, scientific discoveries must be translated into practical applications. Scientific breakthroughs typically begin at “the bench” with basic research, and then progress to the clinical level, or the patient's “bedside.”

Researchers are increasingly aware that this “bench-to-bedside” approach to translational research must be a two-way street, an exchange of information. Basic scientists provide clinicians with new tools for use in patients, and clinical researchers make observations about the nature and progression of disease that often stimulate basic investigations.

However, barriers between clinical and basic research, along with the complexities involved in conducting clinical research, have traditionally made it hard to translate new knowledge to the clinic – and back again to the bench.

The National Institutes of Health (NIH) and numerous pharmaceutical companies have channeled billions of dollars into basic research, and have realized that their return on investments is lower than anticipated. Translational research often is seen as the missing component, though organizations dedicated to translational research do exist (such as the Center for Clinical and Translational Studies at the University of Texas).

Through the Clinical and Translational Science Award Program (CTSA), the NIH recently created a national consortium that includes 39 centers in 23 states with an annual funding commitment of $500 million by 2012. Though still young, the program was designed to shorten the time required to translate research results into therapies.

In an editorial in Science Magazine, 13 leaders of a diverse range of organizations wrote a collective editorial entitled “Translational Careers”. The editorial’s authors share a deep interest in clinical and translational research, and express a collective commitment to the development of an effective clinical and translational research workforce.

The editorial is positive about the potential of the CTSA program, but cautions against an overreliance on federal design and an under-reliance on support systems necessary for this kind of a program.

“People are the prerequisite for success,” they write. “We need an array of innovative investigators whose expertise spans all the disciplines of basic discovery and medical science. As a counterpoint to federal efforts, our private, nonprofit organizations have addressed the human capital need in robust ways, training and funding physicians and other clinical scientists, and piloting models for interdisciplinary graduate training involving biologists, physical and computational scientists and engineers, as well as a wide range of clinical and public health professionals."

The editorialists argue that beyond the rigorous research education essential for all scientists, translational scientists who will work at the frontiers of discovery and clinical science must possess a wide variety of practical and logistical skills.

"They must understand the processes by which discoveries turn into therapies, as well as the evolving role of private industry." the group writes. "They must navigate the regulatory environment surrounding human-subjects research, work in teams and share the rewards of their work, and defer financial rewards while spending years in extra training to gain this knowledge. Existing investigators must learn new skills, but we must also attract new people and facilitate productive interactions among them.

“If it fulfills its potential, translational research will lead to better health for people. But translation is not one-way; the insights gained at the bedside, and from clinical and population-based studies, will spawn hypotheses, enabling scientists to probe the mechanisms of disease in new ways and ultimately enriching basic biology. Therefore, strengthening the support systems for those who will accomplish this multidirectional translation can only be good for science,” the group finishes.

Prize4Life finds this thoughtful editorial particularly relevant to the ALS field. We fully support the CTSA initiative, and, in keeping with our mission and our current projects, we are also conscious of the need to create a vibrant and supportive arena in which researchers can work effectively and cures for diseases such as ALS can be developed.

Monday, August 31, 2009

Technology Enables ALS Patient to Create Virtual Graffiti

The EyeWriter project is on ongoing collaborative research effort to empower PALS to create visual art using open-sourced creative technology. This project is of particular interest to Prize4Life, as it exemplifies the incredible power of crowdsourcing in an effort to improve the quality of life for ALS patients. The EyeWriter enables PALS to act upon the world according to their passions and skills, less limited by the effects of ALS.

Using creative technologies, the project enables ALS patients to design and project ‘virtual graffiti’ using just their eyes. In 2009 in Los Angeles, members of the Graffiti Research Lab, Free Art and Technology, Open Frameworks and The Ebling Group (open-source tech communities) began a project with legendary LA graffiti writer and activist Tony Quan. Tony was diagnosed with ALS in 2003. Like many PALS, Tony retains use of his eyes, but is paralyzed in the rest of his body.

In August of 2009, artists from London, Hong Kong, Madrid, Amsterdam and New York City, got together in southern California and began to work with Tony on a low-cost, open source eye-tracking system that would allow ALS patients to draw using just their eyes.

The result? The EyeWriter, a system constructed from material found in local hardware and electronics stores (see the videos here and here). The software is written using open-source libraries.

The system enabled Quan to draw his tag for the first time in over 5 years, which was then transmitted in real-time and projected outside on a wall he could see from his hospital window (see a diagram here). The team says its long-term goal is to create a network of software developers, urban projection artists, and ALS patients from around the world who are using local materials and open source research to make eye art.

The group also designed a “mobile broadcast unit,” a low-powered, networked bicycle capable of projecting onto the side of a building (or street sign, or train). The artists and technicians responsible for this “eye vandalism” promise that the manuals and software will be publicly available soon.

The group says that the low-cost system can allow ALS patients (with the help of their family and caregivers) to make visual art. Further, when combined with Mobile Broadcast Units (a low-powered, networked bicycle capable of projecting onto the side of a building (or street sign, or train)), a networked version of the EyeWriter can enable patients to have a large-scale presence in public spaces.

This is another compelling example of ALS patients who have turned a practice of ‘survival’ into the wholehearted pursuit of life.

by Meghan Kallman

Wednesday, August 26, 2009

Veterans Mistakenly Informed they have ALS

ALS has been in the national news lately due to a regrettable oversight by the Veterans Administration last week.

At least 1,200 Gulf War veterans across the country were mistakenly notified by the Veterans Administration that they suffer from ALS, the Associated Press reported (the New York Times also covered the story).

The letters, dated Aug. 12, were intended to notify veterans who have ALS of the disability compensation benefits available to them. The VA blames a coding error for the mistake.

At least 2,500 letters were sent out. Of those, some 1,200 were erroneous, according to the National Gulf War Resource Center. No one knows exactly how many letters were mailed to veterans treated at VA hospitals, and how many were a mistake.

Denise Nichols, vice-president of the National Gulf War Resource Center, said her group has received calls and e-mails from alarmed veterans in Alabama, Florida, Kansas, North Carolina, West Virginia and Wyoming.

Gale Reid of Montgomery, AL, Former Air Force Reservist, put herself through a battery of painful, expensive tests following receipt of her letter. Five days later, the VA admitted it’s ‘diagnosis’ was a mistake. She says she is angry: “I've been through a week of hell, emotionally, physically and financially.”

Former Sgt. Samuel Hargrove said that he cried Sunday after opening his letter. “I can't even describe the intensity of my feelings...With so many health issues that I already have, I didn't know how to approach my family with the news.” Hargrove is a father of two.

So, at first, he said nothing. Hargrove later discovered the mistake after talking with fellow veterans in the resource center and online. He is, to put it bluntly, angry as hell.

“Our fear was this could push somebody over the edge,” said Nichols, who was worried that the news could lead already fragile veterans to commit suicide. “We don’t want that to happen.”

“VA is immediately reviewing the individual claims files for all the recipients of this letter to identify those who received the notification in error," the VA said in a statement Monday night.

The Gulf War veterans group is urging the VA to reimburse any veteran who scheduled additional tests with civilian doctors. Reid reported that her follow-up tests cost approximately $3,000, though it may take weeks before she finds out how much her private insurance will cover.

The VA has already withstood heavy criticism this year. In June, Congress questioned it over flawed colonoscopies at VA medical centers in Florida, Georgia and Tennessee that may have exposed 10,000 veterans to HIV and other infections: read about it here and here. Last month, the VA Medical Center in Philadelphia disclosed that the number of cancer patients receiving incorrect radiation doses had risen to 98 veterans over a six-year period (see the story).

Prize4Life is disturbed by this egregious error, and believes, along with Reid, that this type of letter is a callous way to transmit information which could be misinterpreted as a diagnosis. While news reports state that the letter was to inform patients of ALS-related VA benefits, it was clearly interpreted as a diagnosis by some recipients. The VA website states that:

“VA employees are personally contacting these individuals to ensure they understand the letter should not be confused with a medical diagnosis of ALS, explain why they mistakenly received the letter, and express VA’s sincere apologies for the distress caused by this unfortunate and regrettable error.”

This mistake is particularly disturbing in light of the fact that the VA has not been traditionally responsive to ALS patients in need of rapid and intensive medical care; Veterans Today and the Charlotte Observer have run stories on veteran PALS who have faced tremendous obstacles in obtaining full VA benefits for their conditions.

The question of VA care generally is of larger significance and not the immediate concern of this post; however, increased risk of ALS has been statistically linked to veterans, and therefore deserves explicit and careful attention from the Veterans Association and all doctors who treat veterans. This error did not demonstrate any such sensitivity.

While Prize4Life finds this administrative oversight galling—especially given the severity of ALS and the lack of treatments and cures available—we hope that the nation can use this opportunity to raise awareness of the crippling effects of the disease. If you know any veterans who received the letter, please help spread the word that though for them this news was a mistake, for 30,000 other US citizens ALS is their reality. They too are fighting for their lives.

by Meghan Kallman

Wednesday, August 19, 2009

Public Reactions to "Sarah's Story"

Prize4Life was very pleased to welcome Sarah Ezekiel’s guest post last week. Sarah shared her perspectives on the making of “Sarah’s Story”, a public service announcement about ALS sponsored by the UK-based MND Association.

The ad was banned from British television several weeks ago by watchdog organization Clearcast, deemed too ‘shocking’ for public consumption. A heated controversy ensued regarding what should and should not be aired publicly. British newspaper the Telegraph published a story on the debate, and the BBC subsequently hosted a discussion between Sarah, the MND Association, and Clearcast, which can be found here. The Jewish Chronicle also recently covered the story: read it here.

We saw a very interesting tension emerge during this debate—that is, there seemed to be several debates occurring simultaneously.

The ALS community sees and feels (rightfully, we believe), that ALS is a brutal disease, that awareness can help lead to a cure, and that the pain inflicted by this disease is not ‘too shocking’ to be public, especially in comparison to other things that are publicly aired without controversy (such as rape, violence, warfare, etc). The ALS community argues in favor of airing the video, and frames its argument as a question of illness; we are discussing the clip in terms of ALS itself.

The second debate, it seems, is about the general standards which television employs to vet its content. The question is not about ALS specifically, but rather about whether there are images which are too controversial to be made public.

We see these conversations occurring on different levels—in effect, missing each other.

Let’s look at some of the comments on the Telegraph article. One woman objects to the video as inflicting unnecessary pain: “Why is it that some people believe they have some sort of God-given right to inflict a display of their misery on others? Just because you are suffering does not entitle you to illustrate your pain on TV or anywhere else.”

Another rebuts: “It is amazing that society doesn't allow something as strong as this message to be seen. We see acts of violence on TV daily. If I don't like what I'm seeing on TV, I change the channel. Simple. If diseases are publicized more, then there is the potential for raising more money to try and find a cure.”

One writer opposes the presentation of the woman in the ad, seeing it as falsified and dramatized. He writes: “I think the ‘advert’ is inappropriate. The woman is not Sarah Ezekiel but a model, Philippa Jonson. Sexy high heels and skimpy skirt all coming off. I do not understand why she is depicted as climbing backwards up a wall...Inappropriate. It fails to show what patience and endurance can achieve.”

An ALS patient responds, and in her response she connects the images in the video with a reality of living with ALS: “I was diagnosed with MND in October 2008. Since last August I have fallen over 10 times. Twice I have fallen against the wall and it was with such force that it felt exactly like I was being slammed against it, as it is shown in the ad. If you are falling over at speed and you encounter a hard surface, with no chance of stopping yourself, then that's how it feels...When a person with MND falls there is no chance to break the fall, your hands, arms and legs crumple beneath you and contact with the floor or other hard or sharp surface is always very shocking, there is nothing in your body that seems to act as shock absorbers...I can't crawl very well either, so in this respect too, the film is very, very accurate.”

It is clear in this exchange that the discussant are talking about very different things; the first writer objects to the sexualization and dramatization of the woman in the advertisement, while the ALS patient clearly sees the debate as a question of awareness regarding the real-life effects of a specific disease.

Another writer rebukes: “as for those commentators who think that the ad contains some reference to ‘sex’ or ‘sexy’, what sick minds you must have.”

At Prize4Life, we believe that the misunderstanding highlighted in these selected comments and in the larger discussion represents a fundamental lack of awareness about ALS’ effects on the body, and about the dehumanizing reality which the disease imposes upon its patients. For example, the connections between bared limbs and the physical effects of ALS on a patient in such a dramatization would be obvious if ALS were more familiar.

Following the media frenzy about the decision, Clearcast rejected the MND Association’s offer to air the video only after 10 pm and not following a family-oriented program. Clearcast noted, however, that it would allow the film to air if the footage of Sarah’s deformed limbs were cut. We find this point to be one of the most disturbing of the entire debate. Per Clearcasts’ decision, this is not a question of sexuality, provocation, or even the showing of someone else’s pain. It is a question of exhibiting ‘deformity’, something which the organization is unwilling to do.

Regarding this, another contributor observes: “It is incredibly disturbing for me that “[Clearcast] would allow the film if the scenes showing Sarah's deformed limbs were cut”--the fact that it is deemed necessary to hide images from the public eye of limbs that are ‘abnormal’ in some way only reinforces the marginalization that people with disabilities experience in our society. There are many people who find it uncomfortable to see images like this, but hiding them away only serves to reinforce the ignorance that feeds discrimination.”

We believe that Sarah’s story is about ALS, and that it should be discussed in terms of ALS, and that they key to creating this connection is through increased awareness of the disease. We support the MND Association’s decision to air this piece, and we hope that the conversation around it continues to foster increased understanding, exchange of ideas and, someday soon, a cure.

by Meghan Kallman

Thursday, August 13, 2009

ALS Patient Stephen Hawking Awarded Presidential Medal of Freedom

Stephen Hawking, an internationally recognized physicist and mathematics professor at Cambridge University in London, and an ALS patient for 40 years, was one of 16 recipients awarded the Presidential Medal of Freedom by President Obama last night. The Presidential Medal of Freedom is America’s highest civilian honor, originally established by President Truman in 1945 to recognize civilians for their efforts during World War II.

“Each saw an imperfect world and set about improving it, often overcoming great obstacles along the way,” Obama said of the honorees. “Their relentless devotion to breaking down barriers and lifting up their fellow citizens sets a standard to which we all should strive.”

67-year-old Hawking is a familiar figure in the ALS world, and best known in physics for his work on black holes. Scientifically, his name is connected with proofs that the universe must contain “singularities” and with radiation theoretically emitted by black holes (“Hawking radiation”).

Hawking, by any estimation, is no ordinary scientist. His wheelchair-bound figure has become emblematic of the human spirit pressing on against adversity, and has indirectly generated awareness of ALS and its repercussions for many years. Diagnosed at age 21, Hawking has been almost completely paralyzed for decades. He communicates through an electronic voice synthesizer and his computer.

Hawking’s book A Brief History of Time remained on the bestseller list for a record 237 weeks. As he conferred the honor Barack Obama said: “From his wheelchair, [Hawking] has led us on a journey to the farthest and strangest reaches of the cosmos. In so doing, he has stirred our imagination and showed us the power of the human spirit.”

On his homepage, when asked about the study of physics taking him “beyond physical limitations,” Hawking answered, “The human race is so puny compared to the universe that being disabled is not of much cosmic significance.”

An unobtrusive symbol of human perseverance generally, Hawking’s accomplishments clearly demonstrate that ALS patients do not merely survive: they continue to live. Congratulations, Dr. Hawking!

by Meghan Kallman

Tuesday, August 11, 2009

Guest writer Sarah Ezekiel blogs on "Sarah's Story" controversy

Prize4Life is pleased to welcome guest blogger Sarah Ezekiel! Sarah shares with us her perspectives on the making of “Sarah’s Story”, an advertisement about ALS sponsored by the UK-based MND Association.

The ad was banned from British television several weeks ago by watchdog organization Clearcast, deemed too ‘shocking’ for public consumption. A heated controversy ensued regarding what should and should not be aired publicly. British newspaper the Telegraph published a story on the debate, and the BBC subsequently hosted a discussion between Sarah, the MND Association, and Clearcast, which can be found here. The Jewish Chronicle also recently covered the story: read it here.

Prize4Life believes that the issue of ALS awareness is crucial; it affects the entire ALS community and has tremendous implications for research. Stay tuned for a subsequent blog post dedicated to the public reactions which have surrounded the controversy. Sarah shares her views below.


Everything was pretty straightforward for me until the age of 34. I was happily married with a beautiful little girl and pregnant with my much longed for second child. In February 2000, I noticed some weakness in my left arm and my speech was slurring. By April 2000, I had a definite diagnosis of ALS or Motor Neurone Disease (MND) as it’s called in the UK.

It happened so quickly; I was absolutely terrified. My marriage collapsed as I became progressively disabled. I couldn't physically care for my children or myself anymore, and spiraled into deep depression. I'm now a single, disabled parent who is totally dependant on caregivers for everything. I never expected my life to change so tragically. I often think that it's more painful for my family and friends than it is for me.

I am now 43 and have survived much longer than most ALS sufferers. I have lost almost all my mobility, as well as my speech. I use a program called E Z Keys on my laptop, which enables me to communicate and access the internet. Technology has saved me!

When I was asked to appear in “Sarah's Story”, the ALS advertisement that has been the subject of so much controversy, I didn't hesitate. Donna Cresswell from the MND Association showed me the story boards and explained more about the film. I liked the concept and felt that it was important for the public to see.

On the 3rd of July 2008, I found myself in a studio in Central London. My first scene was the end one, sitting in the huge wheelchair. I was positioned and had to keep very still, and when that finished, I was told that was it.

I asked whether I had just come there to sit in a wheelchair, feeling pretty upset. I asked the director whether I could try some other scenes because I wanted to show the deterioration that ALS causes to the body. I kept insisting until he agreed. I went home feeling pleased and amazed by the dedication and kindness of the creatives, director, actress and the crew, who gave their time for free.

The advertisement that we worked so hard on was recently banned from television by the British watchdog, Clearcast. They told us that it was “too shocking” for people to see.

I was, and remain, very disappointed with this decision. There are many shocking adverts being shown on British TV, so why can’t viewers witness the ravaging effect of a real disease on a real sufferer? That, the television censors have decided, is too disturbing for the public to handle.

Sarah’s Story is shocking but so is ALS. The advert is 90 seconds of powerful, disturbing imagery. It opens as a young woman walks into a room and is confronted by a wheelchair. As she gazes questioningly at it, a steel door behind her slams shut. Her body is immediately smashed against a wall. Struggling for breath, she is tossed around the room like a rag doll and left thrashing on the floor. Her clothes are stripped off by an unseen force, and her limbs, twisted and thin, convulse uncontrollably. In the final scene, she sits slumped in the wheelchair, unable to speak or move.

However, Clearcast claims that “even good-cause advertising is capable of causing widespread general offense.”

I feel that a graphic representation is the only way to ensure that more people are made aware of the horrific physical and emotional impact of ALS. The ban makes me feel as though ALS sufferers must be hidden away. Making the video was tough but so very, very worthwhile. I am extremely proud of it. And I'm afraid the only way to relay the true horror of ALS is in a shockingly vivid way. In fact, the film doesn't really convey the reality of this disease. The reality is far, far worse.

Just after being diagnosed, I spent three years wishing I were dead. Now, I spend every day being thankful that I am not. I am grateful for every moment with my children, although I do wish I could do more with them. My greatest wish is to be around to see them grow up. I am not ashamed to have ALS; I am just different now. I strongly believe that more awareness of ALS can help lead to a cure, and I will continue trying to raise awareness for as long as possible.

You can watch Sarah’s Story here http://www.sarahsstory.org.uk/, and I can be contacted via my website http://www.sarahezekiel.com.

Wednesday, July 29, 2009

New ALS Research Portal

As part of Prize4Life’s ongoing effort to reach out to researchers and provide them with the tools they need to make biomarker and treatment discoveries, Prize4Life has launched a new ALS research portal, the ALS Forum.

A partnership between Prize4Life and the Alzheimer Research Forum (Alzforum), the ALS Forum went live on May 29, 2009, and the initial response has been swift and positive. “This will be the site for all ALS researchers go to,” one researcher commented, in response to the initial launch. The portal was modeled on the Alzforum site, which has been quite successful in accelerating Alzheimer’s research and promoting Alzheimer’s education (see publication highlighting its impact).

Our two organizations have collaborated to provide researchers and interested audiences with a one-stop shop for ALS research, drug, and conference news, bringing together a variety of sources for the benefit of our audiences. In addition, the ALS Forum provides an indexed database of drugs in development with potential relevance for ALS, and a compilation of useful ALS-related links.

The ALS Forum is intended to collect resources and build capacity to help researchers accelerate their work. We hope that the portal will be recognized as a crucial tool to support scientists making developments in ALS research. In addition, it will serve to advance basic understanding of the complex pathology of the disease. By promoting the use of a central resource, we seek to encourage collaborative research within the scientific community.

Prize4Life and Alzforum developed the ALS Forum to meet researchers’ need for a centralized and convenient resource for ALS news, and we have been delighted with the Forum’s early success. In just over two months, the portal has attracted a steady traffic flow and registered close to 1,800 hits. In the first two months, 127 members have registered for further information, and the Forum sees nearly as much traffic, on average, as the main Prize4Life website.

Researchers have responded positively to the concept and content, which makes us optimistic for its continued growth: “It is terrific,” praised one scientist. “Bravo to you for your efforts in launching the ALS forum.”

As part of our effort to continue to expand the Forum’s network, we recently launched the biweekly ALS Forum Newsletter. This week marks the inaugural edition of the newsletter, which contains links to new content along with brief summaries of the articles. We are excited to be able to share this resource with our collaborators in the fight against ALS.

We are looking to you, the Prize4Life and ALS communities, to help us spread the news about this exciting new venture. You can visit http://www.researchals.org to browse the Forum and sign-up for the biweekly newsletter that will bring news updates directly to your inbox. You can blog or comment about the Forum on other ALS sites. You can mention the Forum to doctors and researchers. You can comment below on more strategies for expanding the Forum network or provide us with additional ideas for valuable web-based research tools and resources you would like to see in the future. Help us make the ALS Forum a go-to resource for all members of the ALS research community.

Wednesday, July 22, 2009

Neural Transplants and Neurodegenerative Diseases

Despite hope to the contrary, it may be premature to sing the praises of healthy cell transplantation as a treatment option for neurodegenerative diseases. Earlier this week, the Michael J. Fox Family Foundation interviewed Huntington’s disease researchers about their recently published study on the effects of embryonic neural tissue grafts in Huntington’s-affected brains. A transcript of the interview can be found here. Their study reveals that the promise of fetal neural graft therapy faces unexpected challenges.

In the 1990’s, scientists touted cell transplantation as a therapy option for neurodegenerative diseases. Researchers thought that replacing dying neurons with fresh, unaffected ones would revive neural function and slow, or even stop, disease progression. However, recent findings reported in PNAS and Nature suggest that Huntington’s disease can co-opt implanted healthy neural cells and tissues, leading them to degenerate at the same rate as (or even faster than) the patient’s own disease-mutation-containing tissue.

In the reported study, transplanted neural tissue appeared to follow disease-specific degeneration, even though it did not contain the Huntington’s mutation and was initially unaffected. This means that even without the Huntingtin protein mutation, tissue was susceptible to the same degenerative processes of the disease. In their study, the Huntington’s researchers suggest that diseased tissue may promote a toxic environment that makes it impossible for the healthy tissue to thrive. The kamikaze nature of the diseased neighboring tissue seems to make it challenging for the healthy tissue to survive long term. This finding indicates that using healthy tissue transplantation as a therapy option may be more complicated than originally anticipated.

This is somewhat disheartening news, especially given the media-fueled anticipation of tissue transplantation treatment options. The findings suggest researchers should exercise caution moving forward with basic fetal tissue transplantation therapy. However, there are still variables of transplantation that remain untested (particularly those of environment) and optimism remains that stem cell therapies may have more success in refreshing crucial neural populations.

There are numerous differences between cell-transplantation based therapies and tissue transplantation-based treatments. While the tissue transplants are three-dimensional, differentiated, and further along in development, stem cells are typically delivered in solution, undifferentiated, and at an earlier developmental stage. These differences yield hope that implanted stem cells may not be as susceptible to the degenerative processes presumably provoked by the existing tissue.

Stem cells, being less differentiated, may retain some proliferative capabilities that the fetal transplants do not (they may last longer because they are a potentially renewable source), or they may be less sensitive to the toxic environment created by the diseased neurons. Stem cells may also prove to be less antigenic than tissue transplants, thereby arousing less of a destructive response from the patient’s own immune system. This is particularly true of stem cells derived from the patient (using iPS cells or other sources) as opposed to fetal tissues. Researchers are optimistic that stem cells, which can be induced to develop into neurons, may be able to survive long enough in the diseased neural environment to be able to limit, or even correct, the damage inflicted by the disease.

Hope remains that healthy neural cells and tissue can override disease-imposed degeneration in the affected brain environment. However, these recent findings in Huntington’s patients (which were also observed, to a lesser extent, in Parkinson’s patients), indicate that the seeming silver bullet of neural transplantation may not be quite as simple a solution as was anticipated.

Nevertheless, we at Prize4Life take this news in stride; it serves as another step forward on the road to true ALS treatment development. The more researchers know about the pathology of neurodegenerative diseases, the better they are positioned to direct their experiments to successful treatment options. And the better informed that patients are about potential therapy options, the more educated and healthy decisions they can make about their own treatment. We continue to support research into slowing and stopping neural degeneration in the hope that diseases like Huntington’s, Parkinson’s, and ALS will be cured, and look forward to further advances the field of therapy options.