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 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.

Friday, July 17, 2009

Perspectives on Incentive Prizes and Crowdsourcing

We have recently come across two particularly interesting and relevant blog posts that we wanted to share. In this vein, please feel free to share our blog with other networks to which you belong; we are eager to gain more readers and stimulate more conversation in the ALS space. But back to business. To follow up on our recent post about incentive prizes and for more information from the McKinsey & Co. report on them, check out a recent “What Matters” post on the importance of incentive prizes to inspire innovation. Rather than innovation and prizes being peripheral to each other, as they were for many decades, they have recently developed an important relationship that has yielded exciting and rapid advances in many research fields. Prize4Life is proud of its own incentive prizes and proud to be a leader in the field of prize-motivated innovation, continuing the legacy of prize-driven organizations that that have harnessed the power of prizes to effect change. Follow the link above to learn about the history of prizes and their potential for influencing research and development across a wide spectrum of industries.

We also wanted to highlight an interesting reflection on the ongoing shifts in the nature of work. In this recent post, InnoCentive’s CEO Dwayne Spradlin shares his thoughts on the power of crowdsourcing to bring people back to doing the work for which they are best suited. Too many people, Spradlin laments, work jobs they do not enjoy and for which they are not well-suited. But this depressing (and inefficient!) trend can be reversed with the increasing reliance of companies upon public wisdom and expertise. The practice of turning to the crowd to solve a problem or concern allows people to define their own work, which will likely lead to greater productivity and accelerated innovation, which is exactly what Prize4Life is striving for. However, unless crowdsourcing offers compensation for solutions and useful suggestions, this belief in the wisdom of the masses will not replace the traditional work model, despite the fact that it may provide the general population with opportunity to actually apply their passions and expertise, which they may or may not get a chance to do at work. And that’s where the prizes come in! At Prize4Life, we are not invested in changing the traditional work model but rather in using the crowdsourcing model to accelerate cures and treatments. We believe dramatic advances in ALS research can be made when people are encouraged to make connections that wouldn’t come to them ordinarily and when they work within and even beyond their own expertise to address questions of ALS treatment that are particularly interesting to us.

We are highlighting these two posts because they evoke two of the foundational tenets of Prize4Life: incentive prizes and crowdsourcing, and we want to share with you different perspectives on these topics and help you understand why we trust this powerful combination to accelerate efforts to identify new treatments and a cure for ALS.

Sunday, July 12, 2009

Prize4Life News Digest - 07/12

Lou Gehrig’s Retirement From Baseball Remembered This Week
This past Saturday, July 4, 2009, marked the 70th anniversary of Lou Gehrig’s immortal retirement speech, in which he declared himself the luckiest man on the face of the Earth. A single man facing his own ALS diagnosis moved the MLB to take a move active role in supporting ALS research efforts, and his endeavor is celebrated by Newsweek. Read a little bit more about remembering Gehrig and his imminently sportsmanlike tradition in the NY Times. In addition, you can find the MLB press release on Saturday’s events here. There was also extensive local coverage for each MLB event around the country—including in the Boston Globe—if you’re interested in further reading.

ALS Drug Trial Put on Hold by FDA
CytRx has filed a report to the FDA protesting the January 2009 partial hold on a phase 2b efficacy clinical trial of arimoclomol, a CytRx ALS drug candidate. Arimoclomol is a molecular chaperone regulator, based on the finding that correcting cellular protein repair pathways with the help of molecular chaperones can prevent the misfolding of proteins that can make them toxic to the body. CytRx is strongly protesting the hold and insists that it has cooperated fully with FDA regulations regarding animal toxicity reports. There are no reports of adverse effects in human trials.

New Microelectrode Technology Heralds Advances in Translating Brain Signals
Researchers at the University of Utah have made advances in microelectrode technology that bode well for the further development of BrainGate-like technologies that translate brain signals into movement in paralyzed patients. BrainGate researchers and their peers have been using the Penetrating Utah Electrode Array to read signals from brain cells, but now there is a newer system. The new electrode technology allows for the same kind of mental control but relies on electrodes placed on the brain, rather than in the brain. By significantly decreasing the risk of brain damage, this non-invasive electrode placement may make such treatment safe for wide use.

Wednesday, July 8, 2009

Pride in the Prize

The inducement prize model, on which Prize4Life is based, is increasingly recognized as a critical tool for making the impossible possible in a wide variety of fields. The X Prize Foundation was an early pioneer in the field and sponsors some of the world’s largest prizes. Paul Jansen, a principal in McKinsey & Co.’s San Francisco office recently interviewed X Prize Founder and CEO Peter Diamandis about the value of prize economics and the philosophy behind prize success. You can access the interview (and transcript) here:
Audio interview with Peter Diamandis

It is important to understand the philosophy of the inducement prize model, and Mr. Diamandis illuminates its purpose quite clearly. By packaging a problem and inviting the world to find its solution, prizes encourage risk taking that might otherwise seem like crazy ideas. They promote a paradigm shift in the way the way that the public conceives of a problem (like ALS) and make the impossible possible. And not only do they promote innovation in the here and now, successful prizes are sustainable endeavors that motivate back-end business plans, in that they often inspire innovations that will result in marketable products, which can serve as further incentive to enter the competition—if you know that more than just the prize is at stake. Moreover, prizes encourage a change in the behavior of individuals as to how they conceive of global problems. As Diamandis says, “when the prize is won is just the beginning."

And the inducement prize model is just the beginning of a new era of understanding how to approach problems. It is no longer the case that you train in a field and then necessarily answer the questions and solve the challenges of that field. Rather, we are entering a new phase of problem solving in which problems from across fields are presented to the entire population, thereby taking advantage of fresh eyes and innovative ideas. This is exactly the kind of “new minds and new ideas” that Prize4Life seeks to inspire in its mission statement.

It is understandable to be at first turned off by the thought of monetary incentives being necessary to motivate research on issues whose solution would garner the thanks of humanity; shouldn’t that research take place without the scourge of a monetary incentive, you might ask? But monetarily incentivizing isn’t a bad thing and is not necessarily indicative of a hopelessly greedy system. The importance and influence of money is undeniable, and the inducement prize model leverages that reality to deeply benefit humanity by motivating solvers to tackle all kinds of problems: the widely recognized (like genomic sequencing) and the more hidden (like the lack of an ALS treatment or biomarker). If prizes influence the direction of research and development in academic and commercial settings, such movement is not to be lambasted as money-grubbing but rather celebrated as the recognition of looming problems that are receiving the attention that they deserve. The inducement prize provides the kind of unbiased and open motivation, with no stipulation for who can solve and how, that is likely to attract innovation and creativity, exactly the thinking that we need to make an ALS biomarker possible.

Monday, July 6, 2009

Lou Gehrig Continues to Give

Watch CBS Videos Online

News wires were abuzz this weekend with updates on the MLB celebration of the 70th anniversary of Lou Gehrig's retirement speech (see below for a reaction to the speech). It was remarkable to see a nationwide endeavor dedicated to acknowledging ALS and honoring those touched by it. With a coordinated effort, ALS organizations around the country were able to bring ALS to the forefront of millions of minds.

Raising awareness about ALS was one of the greatest successes of MLB's 4ALS Awareness campaign. And Katie Couric supported that endeavor with her "Katie Couric Reports" special "Treating Lou Gehrig's Disease," which aired on Thursday night. The report, accessible here, weaves patient narrative together with expert summary of ongoing research endeavors, particularly those associated with skin cell-derived pluripotent stem cells. Doctors from Harvard and Columbia Universities shared their research plans with Couric, and to demonstrate the process of cell donation, Couric herself gave a skin sample--on camera--for stem cell research.

This weekend's news makes this Monday nothing short of exciting. Most major news sources published at least one article on Lou Gehrig, each emphasizing the tragedy of the disease that took his life and the fact that a cure has yet to be realized. And Katie Couric drove the point home with her feature on the CBS Evening News.

But the question remains: what happens now? This is the kind of nationwide event that can so easily be dropped by the wayside with the next news cycle. It is up to us, the ALS community, to perpetuate the national message about ALS and take advantage of a swell of awareness so that ALS enters the national consciousness as a significant medical concern. Lou Gehrig's legacy has lasted for seventy years, and he continues to give to us: presenting us an opportunity to promote research and raise awareness. He is on Katie Couric's mind, and through the CBS Evening News, Lou Gehrig has entered American living rooms. Now, the onus falls to us to engage the fight against ALS, recruit our friends to join us, and stand strong and together to find a cure.

Was Lou Gehrig really the luckiest man on the face of the Earth?

Did you get to hear it? If you didn’t, then you must have been hanging out under a rock all weekend!

I’m talking about Lou Gehrig’s farewell speech—the one he gave at Yankee Stadium on July 4, 1939.

This last weekend marked the 70th anniversary of that speech and in a fitting tribute to #4 and to all individuals and families living with ALS around the world, Lou Gehrig’s “…luckiest man on the face of the earth” speech was read at baseball diamonds all across the country—and then replayed many times over on news reports all weekend long.

I remember the first time I heard that speech—or a Hollywood version of it—in The Pride of the Yankees. I have to admit, I had trouble at the time accepting that Lou Gehrig could honestly consider himself, “the luckiest man on the face of the earth,” especially considering the future he was facing. It just didn’t ring true to me.

I’d like to say that in time, I came to understand better what he was trying to say. But, in all honesty, that didn’t happen… I continued to struggle with the concept.

ALS is a terrible disease, and it’s hard to imagine that anyone diagnosed with a fatal neurodegenerative disease could consider himself “lucky.” Then again, perhaps my reaction is colored by my personal connection to the disease. In 2005, my brother-in-law was diagnosed with ALS. He died last November. I don’t think he was very lucky. I doubt that he, or anyone else with ALS, went around thinking how lucky he was for having been diagnosed with an untreatable, incurable disease that was likely to take his life in a mere 3-5 years.

At the same time, I have developed a new appreciation for the incredible strength and courage that is born out of this disease. I marvel at the ALS patients I have met who have not given up, but who have undertaken Herculean efforts to make a difference for future ALS patients. Who have created not-for-profit organizations, raised money for ALS research, and inspired thousands of people to work for the day when the horrors of ALS are a distant memory.

If there is any good fortune in being diagnosed with such a terrible disease, it must be in the unique opportunity to discover the unfathomable well of pure will and determination that most of us never have a reason to find within ourselves. Or perhaps it is in discovering just how many people truly love and care for you—sometimes, people you have never even met.

As I heard the famous farewell speech recited again over the weekend, I was struck by the way Gehrig focused on the “kindness and encouragement” he received from his fans. And the number of dear friends that he mentioned by name. Seeing the footage of him with his former rival, Babe Ruth, was just downright touching. In his speech, he expressed heart-felt gratitude for his parents, and his mother-in-law, and his dear wife, “who has been a tower of strength and shown more courage than you dreamed existed.”

So, maybe I am coming to understand just a bit of what he was saying. Discovering your own personal power to change the world and knowing that you have friends and family who love and care for you and are willing to join you on the quest… that’s pretty amazing!

Perhaps what has helped me to understand this better than anything is my affiliation with Prize4Life—talk about an amazing group of friends and family who have rallied for a cause! The people who created this unique organization, and who continue to support it, are helping me to comprehend whatever bit of good that there can be in this lousy disease.

Maybe the best way to commemorate the 70th anniversary of Lou Gehrig’s farewell speech is to do something—anything—to reach out to ALS patients around the world and let them know that they have friends in far-flung regions. That there are hundreds of thousands of us out here—all around the world—who care and who are working to create a world without ALS. Blog, tweet, volunteer, donate, call, email, write, hug… do something! It may not make every ALS patient in the world feel like the luckiest person on the face of the earth, but it can’t hurt!

So… what are you going to do?

James E. Giessler
President & Chief Development Officer

Thursday, July 2, 2009

Prize4Life News Digest - 07/02

About ALS & prize4life

Stem Cell Banks to Promote ALS Research
Researchers at iZumi Bio are capitalizing on the medical promise of stem cells to promote drug development research. Scientists as iZumi are using the cells of patients with various illnesses (including ALS) to derive pluripotent stem cells, which can give rise to virtually any cell type. These cells can launch laboratory models of disease, and the hope is that the cells will contain the genetics and other factors underlying the disease. The company plans to develop a bank of induced pluripotent stem cells from patients with various diseases and use the cells to screen candidates for drug development.

Brain-Computer Interfaces Offer Exciting Potential for Paralysis Patients
40 people paralyzed by disease or accident have entered into an early trial of brain-computer interfaces at MGH and the Wadsworth Center in New York. BrainGate2 enables patients to move robotic body parts with only their thoughts. By connecting the motor cortex to a computer, the brain’s neural signals can be detected and translated into a code readable by a robot. The device can also be used to mentally type. In addition, there is hope that this technology will enable a mind-to-spoken word device to enable ALS patients to verbally communicate, even if on a ventilator.

Industry news

IPLEX Fails in Phase II Trial for Treating Myotonic Muscular Dystrophy
The pharma world is abuzz with news about IPLEX’s phase II trial failure. The drug failed to achieve statistically significant advances in treating muscular function failure, although it did show some promise for addressing insulin resistance in MMD patients. This is a major blow to the case for IPLEX as a successful neurodegenerative disease treatment option.