The Unusual Funding Outfit Hot on the Trail of an Alzheimer's Cure

We love outfits like these.

A couple of venture capitalists or Wall Street guys or otherwise deep-pocketed men (or women) get together, bemoan the sluggish pace of research in some moldering health realm, and decide to do something about it. They say to hell with the NIH, to hell with risk aversion, to hell with reason (sometimes), and they forge a new path.

We’re talking about folks like the Pershing Square Sohn Cancer Research Alliance, and the Damon Runyon Cancer Research Foundation. Oh, and the Cure Alzheimer’s Fund. Them, too.

This last outfit is on our agenda because it's been funding the work of Rudolph E. Tanzi, a pioneering researcher focused on Alzheimer's. The New York Times recently reported on the front page that Tanzi's lab, had, for the very first time, grown Alzheimer’s cells in a petri dish. Which means that researchers can start testing different therapies to shrink and eliminate those cells.   

Do we need to stress how huge that is? 

There’s something a little subversive about the way these organizations operate. The Cure Alzheimer’s Fund was founded by four venture capitalists who put their heads and wallets together and committed to paying the operating expenses for a take-no-prisoners Alzheimer’s research organization (the group’s official title is the Alzheimer’s Disease Research Foundation). As a result, all donations the group receives go directly to research.

The fund has underwritten some truly stunning, daring work.

The real jewel in their crown is the Alzheimer’s Genome Project, started in 2008. Pretty quickly, it picked out four genes linked to Alzheimer’s, and got noticed as one of the top ten medical breakthroughs in the world in 2008. The genetic base of the disease is where the CAF has socked a lot of its money, but, taking a step back, the organization’s four-pronged attack on the disease is about the most well thought-out philanthropic “plan of attack” I’ve ever seen.

First, there’s the Foundational Genetics program. That’s where the Alzheimer’s Genome Project lives. In a nutshell, it wants to find all genes linked to Alzheimer’s, and then figure out how to deal with them, one by one. Active projects in this category include a mission to sequence the genomes of everyone in the NIMH’s Alzheimer’s disease family sample, and a study investigating the link between diabetes pathology and Alzheimer’s.

The second part is about taking the Alzheimer’s genes already discovered and analyzing their pathologies: a field called translational genetics. CAF seems to have done more in this realm in the past than it’s doing currently, but a sampling of 2013-funded projects in this area reveals $100K for vascular regenerative therapy that can slow the progress of Alzheimer’s-related dementia, and $100,000 to study amyloid-beta oligomers—basically, the proteins that often make up senile plaques—and determine which ones are the most toxic.

Moving on, the last two areas of CAF interest are drug discovery and drug development. There are proportionally fewer projects in these categories, and they’re receiving only tens of thousands—not millions of dollars, like projects in the Foundational Genetics realm. Initiatives include examining the effectiveness of anti-cholesterol drugs on Alzheimer’s patients, since cholesterol regulates production of the toxic amyloid-beta that causes senile plaques, and a project studying older people with Down Syndrome, most of whom exhibit Alzheimer’s classic neuropathology by age 60.

CAF isn’t currently running any drug development projects.

It’s pretty clear that for this organization, the emphasis is really on roots-up genetics research. That means the door is wide open for Alzheimer’s researchers pursuing far-out genetic connections between genes—the kinds of connections that could lead to big cures down the road.