Powered by a risk-taking strategy, the Cure Alzheimer's Fund employs venture capital principles to spur Alzheimer's disease research into a new era at MGH and beyond.
Venture Capital spurs new insights into the nature of Alzheimer's disease are emerging from the lab of Rudolph Tanzi, PhD, seen here with CAF founder Jeff Morby.
New insights into the nature of Alzheimer’s disease are emerging from the lab of Rudolph Tanzi, PhD (left), seen here with CAF founder Jeff Morby.

Venture capitalists are known for making bold bets on high-risk projects that have the potential for huge payoffs. Today, driven by that vision, a group of business leaders who founded the Cure Alzheimer’s Fund (CAF) to find a cure for this disease are seeing important returns on their investment in research at Mass General and beyond.

The scientific results the CAF support has brought about are nothing short of game changing as Mass General researchers resolve some long-unanswered questions about how Alzheimer’s disease develops.

Ten years ago, Alzheimer’s research was stalled—it’s goal of preventing or reversing the disease a distant dream. Too distant, thought Jeff and Jacqueline Morby, of Pittsburgh, Pa., both business leaders with experience in finance and venture capital. The Morbys had seen the devastating effects of Alzheimer’s first hand. Jacqueline’s mother suffered from the disease and they became frustrated by the slow pace of research.

As business experts, they also understood the financial burden Alzheimer’s creates for families, the country and, ultimately, the world. “It’s hard to imagine anything we could do that would be better for humanity than to move this field forward,” Mr. Morby says.

Venture Capital Model Spurs Innovative Approach

The scientific results CAF support has brought about are nothing short of game changing as Mass General researchers resolve long-unanswered questions about how Alzheimer’s disease develops.

To jump start Alzheimer’s research, the Morbys partnered with two other business-minded philanthropists, Henry McCance and Phyllis Rappaport, to create CAF, which was designed to accelerate research and find a cure. Mass General, home to some of the most innovative and advanced Alzheimer’s disease research in the world, became the first beneficiary of their venture capital investment strategy, thanks to an early association between Mr. Morby and Rudolph Tanzi, PhD, Mass General’s director of the Genetics and Aging Research Unit and vice chair of Neurology Research.

Traditional sources of funding, such as the government or large foundations, lean toward safe bets, explains Timothy Armour, CAF president and chief executive officer. Such fear of risk-taking means that forward-thinking researchers, like Dr. Tanzi, haven’t been able to get some potentially paradigm-changing studies off the ground by relying on traditional funding sources.

With the support of CAF and its venture capital philosophy, the Tanzi Laboratory conducted the first scan of the entire genome (the genetic code) of people known to be at risk for Alzheimer’s and identified more than 100 new Alzheimer’s genes. CAF then funded a successful study examining how these genes play a role in the disease.

Then, in 2014, Dr. Tanzi and colleagues engineered a new method for growing Alzheimer’s neurons in a gel-filled dish. Their work helped end a nearly 100-year-old debate about which of the hallmark signs of Alzheimer’s disease comes first—the plaques that form in the brain called amyloid or the protein strands called tau that collapse into tangles. They showed that amyloid comes first then produces tau inside the neuron. The result: the tangles that kill brain cells, causing Alzheimer’s.

Trailblazing Research Opens New Frontiers

"Alzheimer's-in-a-dish" images show how amyloid proteins develop from Alzheimer's disease neurons in the Tanzi Lab at Mass General.
“Alzheimer’s-in-a-dish” images show how amyloid proteins develop from Alzheimer’s disease neurons in the Tanzi Lab at Mass General.

“In terms of genetics research, nothing matches what’s going on at Mass General,” Mr. Morby says. “They are the leader in our opinion.” Over the past decade, CAF has invested more than $10 million in Dr. Tanzi’s lab.

Dr. Tanzi, in addition to leading the genetic research funded by CAF at Mass General, chairs the CAF Research Consortium which guides a pioneering, multi-institutional strategy around the nation to attack Alzheimer’s disease.

Since its inception, CAF has grown from an organization supported solely by its founders to one that has about 20,000 contributors worldwide who embrace the venture capital concept. But CAF is totally philanthropic: 100 percent of every donation to CAF goes directly to Alzheimer’s research. The founders and directors pay the general and administrative costs of running the organization themselves.

The organization identifies trailblazing researchers around the world who need seed money for their studies. “We’re very involved in finding projects to fund and in forming the strategies behind the foundation—just like venture capitalists when they invest in a small company,” Mr. Morby says. Many times, the research is still in a proof-of-concept stage, so it’s a long way from getting a grant by a major funder like the National Institutes of Health (NIH).

The foundation is well equipped to apply venture capital principles to their philanthropic goals because all of the founders have extensive experience in venture capital and private equity investing, the Rappaports, in real estate, the McCances in venture private equity and venture, and Mr. Mosby in corporate equity investing.

Hurtling Forward in Search of a Cure

“None of this would have happened without CAF. It’s incredible to have partners that allow us to implement our dreams so we can push faster and harder to solve the mystery of Alzheimer’s.”

With the help of CAF, Dr. Tanzi’s lab plans to test 1,200 FDA-approved and 5,000 investigational drugs on affected brain cells in a dish over the next two years. “Testing on brain cells is 100 times faster than testing in mice and 100 times cheaper,” Dr. Tanzi explains. They hope to single out effective drugs to test in humans.

Dozens of researchers throughout the country are joining the effort, with Mass General as the hub. If they can find a drug to stop plaques from forming in the brain—which happens 15 years before symptoms appear—they can halt Alzheimer’s disease.

In the meantime, they have already identified a potential Alzheimer’s preventative drug known as a gamma secretase modulator (GSM). Expected to go into human trials this year, this drug, if successful, could be taken in pill form—the equivalent of taking a statin drug to prevent high cholesterol. In this case, it would block the over accumulation of beta-amyloid deposits in the brain to help prevent Alzheimer’s disease.

“None of this would have happened without CAF,” Dr. Tanzi says. “It’s incredible to have partners that allow us to implement our dreams so we can push faster and harder to solve the mystery of Alzheimer’s.”

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