Tuesday, April 28, 2009

Prizes Awarded!

Prize4Life Followers - Check out the link below, and join me in congratulating prize winners Dr. Harvey Arbesman and Dr. Seward Rutkove whom Prize4Life awarded, with $100,000 in total, for their significant accomplishments while competing for the ALS Biomarker prize. We'd love your thoughts and comments on the award!

Prize4Life announced today that it is awarding prizes to two research teams who have identified preliminary ALS biomarkers and have made substantial progress in the Biomarker Prize Challenge. Dr. Harvey Arbesman has received the ALS Biomarker Discovery Prize for his discovery of a novel skin-based biomarker, and Dr. Seward Rutkove has received the ALS Biomarker Progress Prize for his adaptation of his current research and technology to ALS. Click here to read the full press release (PDF).

Monday, April 20, 2009

The "Prize" in Prize4Life

Many people have asked us (and continue to ask us) why Prize4Life has embraced the Incentive/Inducement Prize model in our struggle to identify new therapies for ALS.
There isn’t space here to go into a long discourse on the benefits of Incentive Prizes (and many folks have already done a great job of this so if you want to delve further into questions about the model please see: McKinsey Report, X-Prize, and Robert Wood Johnson Foundation Blog) but to put it quite simply, Prize4Life is seeking to use prizes to add to the existing range of resources focused on ALS.

For those who remain skeptical:

Yes of course we know there are fantastic scientists currently working on important ALS-related issues.

Yes, thank goodness there are wonderful donors and foundations already committed to funding ALS research.

Yes the government does donate some money to ALS relevant research as well.

But we at Prize4Life believe that the existing resources: human, financial, and informational are not enough. Not enough scientists, not enough dollars, not enough breakthroughs. Too much suffering from ALS.

So we are pioneering using the Incentive Prize model to try to accelerate disease-related research. The first prize that we launched was the ALS Biomarker Prize Challenge and as we are preparing to make a major announcement related to this prize, the ALS Biomarker Prize will be the main topic of the remainder of this post.

Like all of the Prizes we have/will put forward, the ALS Biomarker Prize Challenge is intended to promote a very specific kind of breakthrough, a breakthrough that would make it easier for promising research findings to cross the gap between academic research and profit-generating industry. This gap (also known as the valley of death) is where the vast majority of ALS academic studies fail to become a product that can benefit patients.

While the valley of death exists for many diseases, it is exceptionally wide for ALS because very basic drug development tools are still missing for ALS and not enough is known about the mechanism of the disease. In May 2006, at Prize4Life’s official founding event, we asked a group of world-renowned thought leaders from academia and industry what would accelerate the slow and difficult process of taking basic ALS research observations and translating them into the creation of new ALS drugs. They responded: An ALS Biomarker.

There are at least two kinds of biomarkers important for ALS: diagnostic biomarkers, which tell you that you definitely have ALS and not another disease; and biomarkers of disease progression, whose appearance, disappearance, or change over time reflect the advancing state of a disease within a given patient (but may not be unique to ALS or any other given disease). We have chosen to focus our efforts on the second, a sensitive marker of disease progression, because identification of such a biomarker would lead directly to faster and cheaper drug trials and the ability to test many more potential ALS treatments then we currently can.

More ALS treatments. Something every PALS, every family member of a PALS, everyone participating in this fight against ALS dreams about. The reality? Currently, a typical proof of concept trial for a potential ALS therapy enrolls approximately 200-500 patients and lasts 15 months or longer, resulting in a total cost of around 8-10 million USD. Small wonder at any given time there are only a handful of drugs being tested for ALS. So to bring things down to hard cold dollars and cents, the underlying objective of the ALS Biomarker Prize Challenge is to reduce the cost of an ALS phase II clinical trial by at least 50%. The less expensive the clinical trial, the more willing companies will be to test new compounds. The more new compounds tested, the better our chances of finding new ALS treatments. As ALSTDI is fond of saying: the more shots on goal, the better our chances of finding something that works. And the biomarker is the key.

Scientists have a wealth of exciting questions to focus on, but only a subset of these are likely to truly change the cost-benefit calculations of those with the most dollars to invest in ALS treatments and cures. Because of a biomarker’s ability to potentially influence all future ALS trials, a robust, sensitive, and measurable biomarker of ALS progression is one of these deal-makers/landscape changers. That is why Prize4Life has focused a major effort on accelerating the identification of an ALS biomarker.

We are now excited to announce that we have received 12 submissions for the ALS Biomarker Prize challenge, all of which have been reviewed by our Scientific Advisory Board. We were hoping for a global impact for this prize (ALS observes no geographic boundaries, why should we?) so we were very gratified to see that the teams competing come from seven different countries around the world: Australia, Germany, India, Israel, Italy, Russia, and the US. We will be making a public announcement regarding the status of this prize challenge in two weeks, so tune back into this blog for further updates.

The field of ALS clinical research will be transformed by the identification of a therapeutic biomarker. The ALS Biomarker Prize will help us get there faster, and as we all know, ALS patients can’t wait.

-Dr. Melanie Leitner

Wednesday, April 15, 2009

Prize4Life Introduction from CEO Avi Kremer

Many of you have heard my story by now. In 2004, at the age of 29, I was diagnosed with ALS, also known as Lou Gehrig’s Disease. After months of sensing that something was not quite right with my body, that I was losing strength and control in a way that did not seem normal, my suspicions were confirmed: I was sick. Well, alright then. I had a name for my sickness, and now I needed to know what to do about it. I asked the doctors what my treatment would be and how soon I would recover. Surprised that I didn’t seem to understand the severity of my diagnosis, they said, “You won’t recover. You will almost certainly die within 5 years. There’s nothing we can do for you.”

I’ve done my research. I now understand just how cruel this disease is. I’ve learned that about 600,000 people worldwide are living with this disease – and even though the incidence of ALS is about the same as better-known diseases like MS and Huntington’s disease, patients die so quickly that there are never that many of us living with ALS at any given time. I’ve learned that even though we’ve known about this disease for more than 140 years, and even though it’s been more than 70 years since Lou Gehrig lent it his name, patients have no recourse, no hope. I’ve heard doctors tell patients that the only available treatment will extend life for a few months at best and will do nothing to improve their condition. Worst of all, I have personally witnessed ALS destroy my own body over the past several years, and I’ve been powerless to stop it.

As I learned more about this devastating disease, I found the current situation unacceptable. I knew that there had to be treatments and cures out there, and I believed that if we could help focus research on the most promising and most valuable avenues of exploration, we could make a big difference in the lives of people affected by ALS. With my classmates at Harvard Business School, I began asking ALS experts what their major obstacles were and what needed to be done to jump-start the discovery of meaningful treatments. Thus, Prize4Life was born.

In its almost three years of existence, Prize4Life has already become a major player in ALS. We’re funding two one million dollar prizes that each address significant research gaps. We’re collaborating with the Alzheimer Research Forum to build a web-based informational resource for ALS researchers. We continue to undertake projects to direct attention to the most pressing challenges ALS research faces and to build the infrastructural resources that will make it easier for researchers to enter the field and find what they need. I encourage you to take a look around our site to learn more about our programs. You can also help us celebrate our third birthday by making a contribution to our cause and signing our virtual guestbook.

This blog is one of our newest resources: we’ll be using this space to discuss what we at Prize4Life are up to, the latest news of relevance to ALS, and what you can do to help. You’ll see some regular entries from me as well as the rest of the Prize4Life staff, and we’ll also bring in some guest bloggers from other great organizations. Make sure to check back often and share your thoughts with us in the comments. We want to keep you informed as we steadily make progress in the fight against ALS – and I know we are nearing the day when ALS patients will never have to hear a doctor say, “I’m sorry. There is nothing medicine can do for you.”

-Avi Kremer

Monday, April 6, 2009

Prize4Life Interview with Makers of ALS Film Trapped

Welcome to the Prize4Life blog and its first post: an interview with Director James Takata, and Writers/Producers Zach Lewis and Jim Mahoney of Trapped. Trapped is a short film about a brilliant young composer and piano player who discovers he has ALS after an eerie progression of symptoms. And though he begins to lose his ability to phsyically write music and play the piano, his spirit and his genius cannot be taken by this terrible disease. As Director Takata describes, though is body is deteriorating, “his ability is still there.”

Trapped Main Character Steven Plays the Piano

Interview Transcript

Interviewer: I wanted to thank all of you for taking the time to interview with Prize4Life, and wanted to start off by asking what were the roots of this project?

Zach: Amy Yamner (Prize4Life Chairman of the Board) called us in March of 2008 and said she had found a short-film about Alzheimer's disease that got a lot of hits and put Alzheimer's into the public eye, and she thought [we] could do something similar to raise awareness about ALS. Then Jim and I wrote the script and after 2-3 revisions we starting shooting on September 15th, after giving our lead actor [David Rodgers] a month to do research. David Rodgers really did his homework and found stuff on Avi [Avi Kremer, CEO of Prize4Life]. He took the responsibility of capturing someone with ALS accurately.

Amy Yamner (right) and Jhenn Webberly (Trapped film editor) at Trapped Screening Party

Interviewer: How did you guys [Jim, James, and Zach] meet each other before making Trapped?

Zach: I used to be a lawyer and did acting at night. Jim and I met in acting school and started writing during the writers’ strike after getting advice from our coaches that now was a great time to try it.

Jim: I saw Zach’s talent and trusted him right away. Shortly after meeting him, we did another movie together.

James: I actually helped Jim get his third voucher on Ghost Whisperer (CBS Show starring Jennifer Love Hewitt). Without it, he couldn’t join the [screen actors guild] union.

Jim Mahoney (left) and James Takata at the Screening Event

Interviewer: What are your hopes for the film?

James: Even if the film doesn’t do anything beyond raise awareness, it is a success. I’m delighted to help push for a cure to ALS and anything beyond that is a bonus. But we’re really excited about this film and would love to see it go to a festival. I’m glad to be a part of it.

Zach: We wanted to highlight what it’s like for someone to be trapped inside their own body. We worked to show the beauty in Steven’s mind.

Steven's Hands Trying to Write His Music

Steven Composing Music as ALS Sets In

Interviewer: Tell us about the production process? What were the challenges?

Jim: Most of the crew work 80-90 hours per week, but they still managed to work full days on the weekends to make Trapped. All of the crew were volunteers, and James offered up his house for 12 or so long days of shooting.

Zach: At one point, we had 10 people polishing a piano for one of the final scenes. It was great.

James: One of the challenges was trying to portray this fatal disease without being too sappy and sad. This was especially difficult because this is a short film and you have to establish understanding of the characters quickly.

Interviewer: What are some of the highlights of the movie and the process in your minds?

Zach: Michael Mollo’s music. He composed all the scores and soundtracks for the movie. He did this for free and wants to contribute to ending ALS as well. One of the other highlights was hearing from Avi on Skype during the premiere in L.A. People were on their feet and clapping.

Michael Mollo (left) and James Takada

James: One of the things I really liked about the script is that in the end, Steven is the same person as at the beginning of it. Even though the disease is killing his body, the person and his abilities are still in there.

Steven’s Genius Goes on Regardless of His ALS

Interviewer: Thanks guys. Any parting thoughts?

Zach: We did this because everyone enjoys making films, but mostly because people like James' wife's grandfather died from ALS and he couldn’t do anything about it. That’s a tragedy we hope the film will ameliorate.

The Trapped video is a fantastic story about the tragedy of ALS. The music is original and well composed, and the film conveys its messages artfully and powerfully, but in a subtle way. The Prize4Life blog will be tracking the video as it attempts to enter the festival circuit and will keep the Prize4Life community informed. Thanks for participating in the first Prize4Life blog posting, and please join us by becoming a follower, subscribing to the blog, or leaving a comment!