Thursday, May 28, 2009

Prize4Life News Digest - 5/28


Q & A: Judith Rodin
An interview with Judith Rodin, head of the Rockefeller Foundation. In addition to the foundation’s traditional funding priorities, Rodin has decided to fund innovative new processes, such as crowdsourcing, that can be broadly applied to address a variety of social issues. In essence, the foundation is rewarding organizations that are pioneering cutting-edge techniques to identify unexpected solutions to problems and encouraging innovation.


New institute will study rare diseases
Congress has decided to provide $24M a year for 5 years to establish a new program for Therapeutics for Rare and Neglected Diseases. TRND will be a part of the NIH. It is primarily designed to help drugs cross the “valley of death” from basic discovery to industry investment. It is not yet clear what diseases the TRND will focus on, but ALS would be a good candidate.

Friday, May 22, 2009

Prize4Life News Digest

The Prize4Life team is constantly monitoring news about ALS, Non-profits/Philanthropy, Crowdsourcing/Inducement Prizes, and the Healthcare Industry. We've decided to start sharing the best of those findings via our blog. Here is our first edition:


First 'Neuroprotective' Gene In Patients With Amyotrophic Lateral Sclerosis Isolated
A group of scientists led by Bob Brown have discovered a genetic variant that can improve survival in ALS patients. Having this particular genetic variant appears to slow the rate of disease progression and may improve survival as much as Riluzole. It also provides another possible avenue of exploration in the hunts for treatments and a cure.

ALS Musician Composes CD with One Finger
Ace Noface, a 38 year old musician diagnosed with ALS, has composed a new CD called Toxic Charm. You can hear some of his music here:


70th Anniversary of Lou Gehrig's Farewell Speech: The ALS Association Launches the 'Covering All the Bases Hitting Challenge'
ALSA has launched a new fundraiser as part of MLB’s 4ALS Awareness initiative (commemorating the 70th anniversary of Lou Gehrig’s famous “Luckiest Man” speech): people can make a pledge for every hit made by the baseball team of their choice. All the money raised will support ALSA chapters.

ALS Therapy Development Institute, the World's Largest Non-Profit Biotech, Appoints Steve Perrin, Ph.D., as Chief Executive Officer
Steve Perrin, ALS TDI’s Chief Scientific Officer, has also become the organization’s CEO. Maureen Lister, the organization’s CFO and COO, will also take on the duties of President. These appointments have been made in the wake of the death of Sean Scott, an ALS patient and the former president of ALS TDI.


Strength in numbers
An article about Dr. Brian Tseng, a colleague of Melanie’s who was at the AAN Annual Meeting. Dr. Tseng is studying Duchenne muscular dystrophy with the help of new technology that allows for rapid testing of large numbers of tiny muscles at once. This could be important not just for DMD but for any muscle-related illness.

Bill Gates funds British scientists in unorthodox health research
The Gates Foundation is funding about 80 initiatives (in Britain and elsewhere) that are high-risk, very creative proposals to solve some of the world’s most significant health challenges. The foundation recognizes that as many as 90% of these projects might fail, but they felt that it was important to invest in some high-risk, potentially high-reward ideas and see what happens. The willingness of the foundation to explore funding methods beyond traditional grants is exciting and could revolutionize aspects of global health.

Trophos initiates pivotal efficacy study of olesoxime in Amyotrophic Lateral Sclerosis (Lou Gehrig's disease)
Trophos, a pharmaceutical company focused on underserved conditions in neurology and cardiology, is beginning a study of the efficacy of their drug olesoxime after phase I trials revealed the drug to be safe and well-tolerated. The trial is expected to take 18 months and Trophos is planning to recruit 500 ALS patients.

Biotech nonprofits immune to recession — for now
Nonprofit biotech companies (or biotech companies funded primarily by nonprofits) are doing surprisingly well despite the recession. Aeras Global TB Vaccine Foundation is among a few such organizations that is hiring new employees, expanding their facilities, and starting new clinical trials. Experts believe this may be because those who fund such organizations (as opposed to traditional biotechs) may take a longer view of what they are trying to accomplish and may be more willing to fund a company that is working on a cause in which they believe. It would be interesting to see if this holds true for ALS TDI (which is itself a nonprofit biotech company) and if it continues to be true as the situation continues to worsen for most other biotech companies.

Monday, May 18, 2009

Mark Wetzel: My Story

My father-in-law lived with ALS for 4 years. Though he's been gone for over a year now, it still astonishes me to think of how much LIVING he did during the time that he had the disease. And nearly all of it with limited mobility. He was an amazing father, husband, boss, friend, mentor, ALS activist...the list goes on.

He had an unbreakable spirit. He heard about a problem with passing the ALS registry act. "Let's go to Washington." He heard there wasn't enough money for research. "Let's raise some." Duke needed a patient to take 31 pills a day for clinical trials. "I volunteer." Walk his daughter down the aisle? "Wouldn't miss it for the world."

It is from him that I first learned of the indomitable will possessed by so many ALS patients. They are truly some of our greatest teachers, men and women of character through and through.

When my father-in-law was still healthy enough to travel, one of the things that we did as a family was take a vacation to Montana. It was the last big trip we had together, just a few weeks before I started the MBA program at Harvard Business School (HBS). By that time, I had resolved to make a "meaningful contribution" to ALS research while I was earning my MBA. Watching a loved one die a senseless death tends to do that to a guy.

While we were out in Montana, I learned of Prize4Life's story when Avi Kremer (HBS 2006) was awarded the ALS Association's Rand Prize for his work toward finding a cure. From the press release:

"Shortly after his diagnosis in 2004, Kremer set out with the other members of Section I (each of the 10 sections at the school is named after a letter of the alphabet) to raise $550,000 from the HBS community for ALS research."

"Half a million bucks," I thought. "From STUDENTS?" This guy's a fighter.

I resolved to seek Avi out when I got to campus, then put him out of my mind as I enjoyed the rest of the trip with my wife and her family. We settled down in Cambridge, and our long and delightful summer ended with the start of the school year.

At the end of MBA orientation I received my "section assignment," which is an HBS tradition. The section and its membership were to be the most important part of my MBA experience. Finding out the letter of the alphabet which denotes one's section is an exciting moment, arbitrary as it may be. I opened the webpage.

"Section I."

Avi's section. It was a sign.

A few weeks later, I stumbled upon a student organization that was offering fellowships to join non-profit Boards of Directors. I sat in on the presentation, and opened up the list of organizations looking for volunteers with the MBA skill set.


Avi's venture. You're kidding right? Where do I sign?

I was brought on board, and the rest is history. I've been around marketing, strategy, fundraising, and even got a chance to write a history of the organization as part of my MBA curriculum. I started out simply wanting to serve the organization as a volunteer, for the good of the cause. But as it often happens when one gives of oneself, my labors were returned to me in abundance. The lessons that Prize4Life has taught me will last a lifetime.

I've watched Prize4Life grow by multiples over the past two years, always driven by a passion to help the research community arrive at a cure. I have had the privilege of making that "meaningful contribution" to an organization that is close to research, close to industry, and close to patients. Being run by a person with ALS clarifies Prize4Life's purpose in a special way. Especially because he's one of the best managers any of us have ever had. Especially because he's become such a close friend.

And as for that vintage of Section I which I joined, they were anything but "arbitrary." "Second family" is a more appropriate descriptor. I am forever grateful that they chose to join us in the fight against ALS and raise thousands of dollars for a cure.

As I leave Cambridge next month, I'll be taking the cause with me. I've seen it happen to almost everyone who has known anyone with ALS - you get involved with the cause, and you just don't stop. As Avi often says, ALS has taken his speech but it will never take his voice. I've been proud to contribute my voice to Prize4Life and look forward to continuing to do so.

So that's how I got involved. That's MY story.

Tell me. What's yours?

Thursday, May 14, 2009

Wall Street Journal Science Prize Article

The Wall Street Journal had a very interesting article recently: The Science Prize: Innovation or Stealth Advertising? The author, Robert Lee Hotz, makes a number of good points about the strengths and weaknesses of incentive prizes (and it’s great to see Prize4Life’s name in the WSJ!), but we differ on a few key issues. It’s important to keep in mind that Prize4Life is a unique organization, and the prizes we offer are not the same as, say, the Nobel Prize, Netflix, or even the X-Prize. Of course, this gives us a great opportunity to discuss our model a little more deeply.

There has been a great deal of debate about whether crowdsourcing and incentive prizes prevent participants from claiming all of the benefits of developing a winning solution. We recognize that this can be a difficult hurdle for researchers competing for one of our prizes. That’s exactly why Prize4Life does not make a claim on any of the IP generated in the process of competing for one of our prizes, and we will never try to own or buy it (unless we are dealing with an extremely promising idea that a research team has explicitly said it does not want to pursue, in which case we will make every effort to ensure that the team is compensated fairly while we invite others to move the idea forward). We also try to help competing teams locate the front-end funding and resources they need in order to enter our prize competitions.

There’s also an important distinction between a prize that solves a problem specific to a particular company and one designed for the benefit of a broader population. In Prize4Life’s case, prizes are developed with the sole purpose of accelerating the rate at which treatments and a cure for ALS are found. We try to ensure that competing teams can find the funding they need and are compensated fairly for successful solutions, but we don’t offer any front-end funding ourselves, and we know that the teams competing for our prizes are financing their work through other avenues. If our prize model thus harnesses new funds and resources to address ALS, or if the model somehow saves money in the process of meeting critical milestones in ALS research, we’d call that a success, not just for Prize4Life, but for the hundreds of thousands of people living with ALS worldwide and their loved ones. A cure is a cure, regardless of how it’s found or how much it costs.

Another crucial difference is that between incentive prizes and recognition prizes. Hotz discusses Prize4Life and the Nobel Prize in much the same way, but the design and goals of each are hugely different. Prize4Life understands that recognition is important in the scientific community, and we certainly hope that our awards honor the recipients appropriately for their significant contributions to ALS research, but we do not function like the Nobel Prize. Our highly specific criteria focus exclusively on one critical issue in ALS research, and we will not award a prize until that issue is solved fully. The primary goal of our prizes is to solve a problem. In our case, the recognition is a means to an end.

Hotz also points out that science prizes "affect the conduct of science itself, by prizing some efforts at the expense of others." Organizations like Prize4Life are working toward a targeted scientific goal that will have a very specific outcome for patients. To some extent, we are indeed attempting to alter the landscape in which research is conducted, as Hotz suggests – by bringing more funds and attention to a specific research question, we intend to encourage researchers to focus on that question. It is our hope that some scientists, when faced with the wide array of questions they could focus on, might give special consideration to the few targeted research barriers that, when surmounted, will persuade drug companies to invest real dollars in ALS drug development. If our prizes can motivate some people to think about how their work might apply to ALS, or to consider whether ALS might be an interesting research topic for them, then we can truly accelerate the discovery of the vital breakthroughs that will make a difference for ALS patients.

When we launched our first prize in 2006, Prize4Life was the only organization using incentive prizes for neurodegenerative diseases (and only one of a handful of organizations pioneering the use of incentive prizes in the biomedical field). We have learned a lot over the past few years, and many of the lessons we have learned will surely inform the way that we (and other organizations) approach similar prizes in the future. It’s important, of course, to remind ourselves of all of the complex questions our model raises and to monitor the effects that prizes like ours have on the scientific community. But if we’re to effectively measure our performance and be successful in our quest for treatments and a cure for ALS, we need to be clear about our model and how it differs from so many seemingly similar initiatives.

Divya Vohra
Prize4Life Communications Manager