3 families spark search for genes tied to Alzheimer's
Frustrated with the slow progress of research on Alzheimer's disease, three prominent families with local connections have formed a nonprofit foundation designed to find a cure for Alzheimer's within a decade.
Operating quietly for two years, the Cure Alzheimer's Fund raised nearly $3 million and awarded one-third to Harvard geneticist Rudy Tanzi and colleagues at Massachusetts General Hospital.
The philanthropists' approach is controversial because they are focusing on identifying all the genes connected with Alzheimer's, rather than spreading their efforts to other possible causes. The founding families, two of whom live in the Boston area, hope this strategy will more quickly and efficiently result in treatments. Currently, there are only a handful of drugs available, none of which significantly affects the disease's relentless destruction of the mind.
The group is going public with its venture today -- almost 100 years to the day after the disease was identified -- with the goal of eventually raising about $15 million a year from foundations, corporations, and wealthy donors.
The initiative is underway as federal funding for Alzheimer's research -- $652 million last year -- is shrinking, despite the growing number of Americans with the disease.
An estimated 4.5 million people have Alzheimer's now, but without effective prevention measures, that could reach 16 million by 2050 because of the aging of the population. The Alzheimer's Association, the largest private funder of research on the disease, spent nearly $21 million this year on a broad range of projects. The pharmaceutical industry is also working to develop new Alzheimer's drugs.
"We thought we'd do a better job," said Jeffrey Morby, an investment banker spearheading the new fund. "We felt we could move faster since our effort is more of a rifle shot -- to find a cure."
Morby, managing director of Amarna Corp. and a former Cambridge resident, is bankrolling the project for the first few years, along with his wife, venture capitalist Jacqueline Morby, developer Phyllis Rappaport, and venture capitalist Henry McCance . Morby said all of the families "have been touched by Alzheimer's." They also have raised funds from about 425 others.
None of the founders is seeking to profit from the research, said Morby, now of Pennsylvania. And the foundation is requiring that the work it supports be made public so that others can build on the findings. The scientists -- and their universities -- will own any rights to treatments developed. Tanzi, who earlier helped found a company working on Alzheimer's drugs, says the company has no rights to the foundation-funded work.
The foundation grew out of a 2004 meeting between Morby and Tanzi arranged by a Harvard researcher, as Morby searched for a project to occupy his semi retirement.
Tanzi was already working on genetic causes of Alzheimer's and convinced Morby that mapping the entire Alzheimer's genome could help find a cure -- but that more money was needed. Morby subsequently asked Tanzi to gather a group of scientists to develop a research plan.
The plan, completed in August 2005, calls for identifying all genes connected with Alzheimer's by the summer of 2008. Next, the foundation will focus on how these gene defects play out in the body and where and how scientists might intervene. A third phase will fund programs that explore potential drug candidates, and the fourth phase will help pay for tests of the drugs in animals and people.
Already, the foundation is funding some of the second and third phases of work, based on the four genes previously identified, which account for a small proportion of Alzheimer's cases. Besides Tanzi and two colleagues, the foundation is supporting work by five researchers at universities across the country who helped develop the research plan. The foundation expects the circle of scientists getting grants to expand worldwide as the work progresses.
Some scientists question whether the foundation has too insular an approach.
The foundation does not accept unsolicited research proposals, and the same scientists who devised the research plan have so far received the funding.
At the National Institutes of Health and the Alzheimer's Association, by contrast, anyone may seek funding and projects are selected after detailed evaluation by outsiders.
Some Alzheimer's specialists are also skeptical about focusing so heavily on genetics, since the disease likely has multiple causes, including lifestyle factors such as poor diet or lack of exercise. In addition, there are still many mysteries about the pathology of the disease.
"We have known the gene for Huntington's for years, but it hasn't translated into a treatment," said Zaven Khachaturian, a former official at the National Institutes of Health, and editor of Alzheimer's & Dementia, published by the Alzheimer's Association. "We have to model how genes interact with lifestyle."
Khachaturian and others say there is a need for more money from funders willing to take risks and make decisions quickly.
"To the extent that they bring new dollars in, that's good," said William Thies, vice president of medical and scientific relations for the Alzheimer's Association. "They have identified a group of scientists they want to support. Our mission is different -- to support and grow the field. In today's world, you're foolish to put all your eggs in one basket."
Tanzi, who believes that 80 percent or more of Alzheimer's cases may be the result of genetic defects, suggests that the fund's approach is broader than it may seem.
Some $600,000 is already supporting investigations of the role an errant protein called beta amyloid plays in the disease. After the initial phase, he said, the fund will reach out to scientists working in many different fields.
"It will be anything but a clique," he said. "This is science - driven." 