Cure Alzheimer's Fund proposes a National Research Strategy to accelerate progress toward a cure. The proposal builds on our own Research Roadmap, and provides a broader path to intervention by 2020.
We have shared this draft with selected members of Congress and their staffs, and with the staff of the Alzheimer's Study Group , a bipartisan effort co-chaired by Newt Gingrich and former Senator Bob Kerrey, and featuring others including Justice Sandra Day O’Connor, Harold Varmus and our own Henry McCance. It is our goal to work collectively with other funding organizations, the National Institutes of Health and related government agencies to avert the looming crisis of Alzheimer’s. If we can agree on the fundamentals of a national research strategy, we can save millions of lives and billions of dollars within the next decade.
National Research Strategy Summary:
The overarching goal of the proposed national AD research strategy is to reach a cure by 2020 based on early prediction, pre-symptomatic detection, and early prevention. History has shown us that this goal will require an acceleration of studies aimed at identifying and investigating all genes that influence susceptibility to AD, placing highest priority on those that will provide the most promising biological targets for drug discovery and development.
Studies of the four known Alzheimer’s disease (AD) genes have provided an unprecedented window into the molecular underpinnings of AD. Prior to the discovery of the four known AD genes, the field was limited to merely guessing at the causes of AD with little, if any, success. All four of the known AD genes have pointed to the excessive accumulation of the neurotoxic peptide, A-beta, in brain as the primary cause of the disease, although the route to nerve cell death also involves tangle formation. Accordingly, most ongoing clinical trials aimed at modifying disease progression (as opposed to just treating the symptoms) are targeted at lowering A-beta and/or tau accumulation in the brain.
Over the next decade, the influx of funds into AD research should
allow for the identification, validation, and characterization all of the genes involved in AD susceptibility. These funds will also be implemented to investigate AD-associated defects in these genes. The resulting data can be employed to guide and accelerate novel therapeutics that can prevent AD.
Our strategy includes the following four steps:
- Identify all genes contributing to risk for or protection from AD (foundational research);
- Determine how AD genes contribute to the disease process and which subgroup of genes comprises the most promising therapeutics targets (translational research);
- Discover therapies that can slow down, stop, or reverse AD progress (drug discovery) using the targets provided by AD gene identification and characterization;
- Develop a subgroup of the safest and most effective drugs for the treatment and prevention of AD in nationally coordinated clinical trials (drug development).