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