09.27.08
My Project 10100 Submission: Mitochondrial Repair
Here is an example of what I think is a passable submission to Google's Project 10100, with a focus on mitochondrial science. I could probably run one up for LysoSENS-like work as well, but one thing at a time.
Your idea's name (50 characters maximum):Bring Mitochondrial Repair to Phase 1 Trials
What one sentence best describes your idea? (maximum 150 characters):
Our mitochondria degrade over the years, contributing greatly to age-related disease and frailty - but medical technology can fix this problem.
Describe your idea in more depth. (maximum 300 words):
I propose that the most promising of nascent mitochondrial repair technologies be funded from their present early-stage standing to readiness for Phase I clinical trials in humans. As a condition of funding, methodologies will be published free of restriction for any group to further develop and bring to market. This will be accomplished with the aid of a non-profit research organization like the Methuselah Foundation, with a history of raising matching funds for large donations, so as to maximize the impact of the funding program.
Mitochondria are tiny power plants inside our cells, churning away to turn food into energy. They were once free-roaming bacteria and have retained their own mitochondrial DNA, distinct from our own nuclear DNA. As our mitochondria fail, however, so do we. The Mitochondrial Free Radical Theory of Aging points to progressive damage to our mitochondrial DNA as an important - and arguably the most important - root cause of age-related degeneration, disease, and frailty.
At present, a range of plausible technologies exist to repair mitochondrial DNA, replace mitochondrial DNA, or make damage to this DNA irrelevant. These technologies stand at varying points between ideation and animal trials: whole-body replacement of mitochondrial DNA was demonstrated in mice as early as 2005, for example, as has the process of allotopic expression: moving a single important mitochondrial gene into the cellular nucleus, such that the necessary proteins are still made, and a damaged mitochondrion continues to function.
These technologies are progressing very slowly and with a paucity of funding, partly because this is the nature of early research, partly because of perverse regulatory incentives. This is unacceptable when considered against a) the comparatively low cost of basic research in this age of biotechnology, and b) the vast potential benefits to humanity. Philanthropic funding can overcome these hurdles.
What problem or issue does your idea address? (maximum 150 words):
Consequences of damaged mitochondrial DNA include failing organs, clogged arteries, neurodegeneration, and much more. This is the Mitochondrial Free Radical Theory of Aging, well supported by decades of evidence. A working repair technology pushed into the clinical system has the potential to entirely remove this large contribution to disease and frailty. But first it must be finalized from the promising beginnings presently in the laboratory.
Regulatory bodies like the FDA restrict all application of medical science to specific, named diseases; this makes early stage research to produce a general repair technology for mitochondria unprofitable. It would be hard to license, as a developer would struggle to make money on that license. Yet it costs little to move established research to Phase I trial readiness - $1 million is a fortune for a single laboratory - and developers leap at license-free medical technology. This is where careful philanthropy can unjam the gridlocked system.
If your idea were to become a reality, who would benefit the most and how? (maximum 150 words):
A mitochondrial repair technology demonstrated to be ready for human trials, free of licensing cost, free from intellectual property restrictions, and unjammed from the system of perverse incentives in early-stage research stands to benefit everyone. It will be as universally beneficial a medicine as aspirin; the elderly will benefit immediately upon availability, we will benefit from it in decades to come, and our children will benefit when their bodies too start to run down.
Everyone has mitochondria, and mitochondrial degeneration is a universal condition, bringing myriad forms of suffering and pain. We got rid of tuberculosis and smallpox as soon as we could, so why not this? Repair of mitochondrial DNA damage is a very plausible near-future win for everyone, given where the science is today. We can make it happen.
What are the initial steps required to get this idea off the ground? (maximum 150 words):
I envisage the opening labor as follows: 1) Identify the existing non-profit research group and volunteer cadre to run this project - my vote is for the Methuselah Foundation, given their record and contacts within the research community, and the way their mission aligns with that of this project; 2) Identify the best groups and laboratories presently engaged in mitochondrial repair and related research; 3) Develop prospective work, milestone, and funding plans with researchers; 4) Start raising matching funds through existing channels; 5) Select the initial funding opportunities from the best of those produced, and get the researchers to work.
From there, I would like to see established a low-overhead but effective volunteer group of researchers and advocates to manage the cycle of grants, matching fundraising, and evaluation of progress and new research opportunities going forward.
Describe the optimal outcome should your idea be selected and successfully implemented. How would you measure it? (maximum 150 words):
The optimal outcome, after the completion of the project, is: a) for one or more different repair technologies to be successfully readied for Phase 1 human trials; b) protocols and methods to be fully detailed and published, free of restriction; c) multiple medical development concerns to be working on bringing applications to market in diverse regulatory regions; d) independently funded follow-on research taking place with the aim of improving upon the initial technology; e) matching fundraising to effectively continue even after the Google grant is complete.
Sample metrics for success include: a) the breadth and effectiveness of the technologies developed; b) the quality of the published material; c) range of developers working on applications; d) the range of independently funded lines of work spawned by this philanthropic funding; and, most crucially, e) the amount of matching funding and independent research and development funding drawn by this philanthropic project.
If you'd like to recommend a specific organization, or the ideal type of organization, to execute your plan, please do so here. (maximum 50 words):
The ideal organization is a research non-profit with existing connections to scientists already involved in mitochondrial repair research, a very low cost of operation for delivered funding, and a history of raising matching funds for large donations. The ideal example is the Methuselah Foundation, as you might have gathered.
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