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Prize4Life: Inducement Prizes for ALS (Lou Gehrig’s Disease) Nicole Szlezak Member, Board of Directors Prize4Life - PowerPoint PPT Presentation


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Prize4Life: Inducement Prizes for ALS (Lou Gehrig’s Disease) Nicole Szlezak Member, Board of Directors Prize4Life. What is ALS (Lou Gehrig’s Disease)?. Progressive paralysis of unknown cause, 100% lethal Death within 3-5 years from diagnosis (respiratory failure)

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Prize4Life:

Inducement Prizes for

ALS (Lou Gehrig’s Disease)

Nicole Szlezak

Member, Board of Directors

Prize4Life


What is als lou gehrig s disease l.jpg
What is ALS (Lou Gehrig’s Disease)?

  • Progressive paralysis of unknown cause, 100% lethal

  • Death within 3-5 years from diagnosis (respiratory failure)

  • ~30.000 individuals are affected in the U.S. at any given time

  • No cure exists, only existing treatment prolongs life by 3 months (Rilutek)

  • http://www.dollar4life.org/?p4l-hbs-004

Source: www.nwabr.org

Source: web


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ALS is an orphan disease. Patients die so fast that total ALS “patient pool” is relatively small (30.000/120.000 in US/world)

Annual investment in ALS research is comparatively low (~$60 million USD worldwide)

Tools that industry needs for systematic, large-scale drug development are not in place

Why is There no Effective ALS treatment?Some Reasons…

We know little about the causes of ALS

ALS CASES

100%

90%

“sporadic”

?

10%

inherited


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ALS Therapy Development is Risky

Human

Trials

Certainty

?

Mouse model

?

target

ALS clinical trials are expensive due to lack of suitable biomarker. Large intra- and interpersonal variablity of ALS symptoms makes disease progression hard to measure.

Most frequently used biomarker = survival time

Requires long (=expensive) trials

Questionable predictiveness of existing ALS mouse model

Causes of ALS unknown,

targets uncertain

Cost


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Universities/

Research

Institutions

Biotech –

Pharma

Health Systems

Hospitals

Health Services

Innovation in Basic Research often doesn’t get translated into direct benefit for patients

Research -–- Development --- Access:

“Valley of Death” = gap between academic research and industry involvement

Without industry commitment, basic research does not get translated into tangible results for patients


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Prizes as “Lighthouse”:

Prize4Life prizes highlight scientific breakthroughs that will accelerate ALS therapy development

Triple mission:

Accelerate existing efforts in ALS research

Bring in new ideas and new minds

Complement existing funding models

Draw attention to ALS research

Raise funding from heretofore untapped sources

Prize4Life’s Approach : Can we make ALS Breakthroughs more likely?


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Founded in 2006; US 501c3 status in 2007

Mission:

To accelerate the discovery of a treatment and a cure for ALS by using powerful incentives to attract new people and new ideas and to leverage existing efforts and expertise in the ALS field

Prize4Life: Background/Structure



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The Prize Design and Selection Process

  • Step 1: Prize4Life develops prize criteria in cooperation with our Scientific Advisory Board and input from the scientific community

  • Step 2: Prize4Life launches Prize

  • Step 3: Scientific Advisory Board selects winner/s according to pre-set criteria


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Robert H. Brown, M.D., D. Phil, Professor of Neurology, Harvard Medical School

Lucie Bruijn, PhD., Science Director and Vice Presiedent, the ALS Association (ALSA)

Valerie Estess, Director of Research and Cofounder, Project A.L.S.

Adrian Ivinson, PhD., Director, Harvard NeuroDiscovery Center at Harvard Medical School

Tom Maniatis, PhD., The Thomas H. Lee Professor of Molecular and Cellular Biology, Harvard University

David P. Meeker, M.D., President, Lysosomal Storage Disorders, Genzyme Therapeutics

Alfred Sandrock, M.D., Ph.D., Senior Vice President of Neurology R&D, Biogen Inc.

Prize4Life’s Scientific Advisory Board represents academia, industry and patient associations


Progress l.jpg

Raised > 4.5 Million USD since 2006 Harvard Medical School

First prize (Prize4Life ALS Biomarker Prize) launched in November 2006 on InnoCentive

$1.000.000 for an ALS Biomarker that will reduce the cost of ALS clinical trials

Teams from different disciplines are currently working to submit their solutions

Deadline: November 2008

Five small “idea prizes” awarded in May 2007 to researchers who submitted theoretical solutions to finding a biomarker

Several of these proposals have generated new, interesting projects for the development of an ALS biomarker (new minds)

Preparing to launch second prize (2008)

Progress


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Outreach to the scientific community Harvard Medical School

Reach researchers from a wide variety of fields and disciplines

Overcome scepticism

Resources

Upfront funding: how can we support researchers interested in winning our prizes in their search to find upfront funding?

Other resources

Challenges


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