8 December 2009. Could foresight be 2020 for Alzheimer disease? Zaven Khachaturian has proposed a national strategic plan of preventing the disease within 11 years. Prevention, or even just delay of onset, is a lofty goal that topped the agenda at the Clinical Trials on Alzheimer’s Disease (CTAD) meeting held 29-30 October 2009 in Las Vegas, Nevada, and at the Leon Thal symposium also held there the day before. Presentations dealt with the logistics of prevention trials and with ideas for identifying at-risk subjects (see ARF related news story). But is prevention of Alzheimer’s by 2020 feasible?
Khachaturian is president emeritus of the Lou Ruvo Brain Institute (now the Cleveland Clinic Lou Ruvo Center for Brain Health; see ARF related news story). He formed a not-for-profit called PAD2020 (prevent AD by 2020) to push forward the recommendations of the Alzheimer’s Study Group (ASG). This task force of political, medical, philanthropic, and industry leaders formulated a national strategic plan for tackling Alzheimer disease (see ARF related news story). It issued its report last April (see ARF related news story) and then disbanded. In an appendix to the report, 139 thought leaders in the field endorsed the goal of developing the capability to prevent AD by 2020 provided that the effort “is backed by sufficient funding and pursued with an appropriate, disciplined strategy.”
The ASG had no power to appropriate funds to put its strategy into action. “A lot of these reports tend to be received by Congress and the Administration but never lead to any specific program or legislation,” said Khachaturian. “The idea [of PAD2020] is to carry forward the ASG recommendation into an implementation plan that would have specific targets and a specific budget request that would be sent to Congress,” he said. The Alzheimer’s Breakthrough Act of 2009, which is stuck in committee stage in Congress, would authorize $1 billion in funding for Alzheimer’s research, caregiving, and prevention awareness, but would not actually appropriate funds. “That is basically a hunting license,” said Khachaturian. “You can have a license but no game.”
Khachaturian is hoping some of the top minds in neurodegenerative research will bring their best game to PAD2020. Currently, he has enlisted the help of Ron Petersen at the Mayo Clinic, Rochester, Minnesota; John Trojanowski and Virginia Lee at the University of Pennsylvania, Philadelphia; Stanley Prusiner at the University of California, San Francisco; and Peter Snyder from Brown University, Providence, Rhode Island. Khachaturian envisions workgroups of 10-15 people that will consider a variety of potential projects, including identification of new drug targets, creation of an international patient registry, establishment of a national Institutional Review Board, a tool box for primary care physicians to assess risk, and new models to finance clinical trials.
Robert Egge, who directed the ASG, is now Vice President, Public Policy and Advocacy for the Alzheimer’s Association, which partly funded the ASG report. “If the idea is to convene thought leaders to really figure out, from a scientific perspective, what it would take to do a prevention initiative that the ASG calls for, then I think that next step in thinking and planning is essential,” he said. Egge said that the Association supports Khachaturian’s approach, though it has not, as yet, struck a formal partnership with PAD2020. Doug Galasko, University of California, San Diego, told ARF, “There have been a fair amount of discussion and the occasional conference aimed at new approaches to prevention, but relatively few organized efforts to put infrastructure in place and to think through what all of the barriers might be to actually implementing appropriate prevention studies.”
Khachaturian believes that to realize the goal of AD prevention, funding mechanisms may have to change. He wants to consider programs that are difficult to fund through the NIH, and to pool resources from different government agencies, industry, private foundations into a common fund. (The existing Foundation for the NIH already develops public-private partnerships.) Khachaturian said that he considers the current model for federal appropriations for AD research inadequate. “The current model is that the NIH appropriates a set amount, and we are expected to solve a problem within those budget constraints,” he said. He wants PAD2020 to take a different approach. “PAD2020 would build the budget from the ground up. It would insist that to solve the problem of preventing the disease within 10 years, we are going to need the following infrastructure, the following kind of scientists, the following resources. We would present that to the government and say these are the things we need.” Whether that would sit well with Congress or the Administration is unclear, but Khachaturian envisions a bold mindset, the likes of which have led to the splitting of the atom, the Apollo space missions, or the building of the Panama Canal. These were large national projects that took 10 or fewer years to achieve. “We think that kind of approach will make us go faster and further with Alzheimer’s than the random walk model we use now, where very little planning takes place and not enough resources are placed in a systematic way to solve the problem,” he said.
For more on this initiative, see Khatchaturian and Khatchaturian, 2009 [.pdf].—Tom Fagan.