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

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