What are the objectives PAD2020?

The campaign addresses two important challenges facing efforts to combat Alzheimer’s disease and other disorders affecting memory, movement and mood.  The growing disappointment encountered with the lack of long lasting treatments for these conditions and the dim prospect for a single dramatic breakthrough-idea under the current paradigms for therapy development.

The Campaign seeks accelerated progress in the discovery of cures for Alzheimer’s and related neurodegenerative disease.  The effort will pursue radical changes in current conceptual models of the disease and the traditional approaches for discovering and developing therapies for prevention.

The key objectives of the campaign are to:

1. Establish a strategic planning process, which will include key opinion leaders (KOLs) from academia, industry, government and voluntary health/family support groups to devise solutions for the problems of therapy development2.    Delineate the comprehensive scope of the challenges in developing interventions that would affect disease progression and prevention
2. Delineate the comprehensive scope of the challenges in developing interventions that would affect disease progression and prevention
3. Develop a new archetype for surmounting the scientific, administrative, financial and legislative hurdles in discovering the treatments to prevent disease
4. Devise and calculate a ten-year business plan with specific deliverable, time requirements and budget
5. Eliminate administrative and organizational impediments by creating a new paradigm for planning, funding and managing the formation of worldwide cooperative research networks - establish a ‘systems approach’ to solve the problem
6. Identify new funds devoted to research through the promotion of a National Strategic Disease Prevention Investment to provide sustained investment of $1 billion additional dollars per year for the next ten years
7. Support rapid advances in science necessary to develop new technologies for early detection and treatments for the prevention of disorders in memory, movement and mood.
   

What is meant by “Prevention”?

PAD2020 defines the concept of ‘Prevention’ in the broadest possible terms.  The ultimate aim of the Campaign is to promote the development of broad spectrum intervention, including but not limited to primary, secondary and tertiary prevention, designed to maintain independent functioning and/or delaying  the onset of disabling symptoms for as long as possible.


What is a “Systems Approach”?

Ludwig von Bertalanffy, ascribed as the founder of general systems theory, wrote:

"Modern science is characterized by its ever-increasing specialization, necessitated by the enormous amount of data, the complexity of techniques and of theoretical structures within every field.  Thus science is split into innumerable disciplines continually generating new sub-disciplines.  In consequence, the physicist, the biologist, the psychologist and the social scientist are, so to speak, encapsulated in their private universes, and it is difficult to get word from one cocoon to the other.”

Von Bertalanffy explains that general system theory—or a Systems Approach—is a general science of the "wholeness”.  He writes,

“The meaning of the somewhat mystical expression, “the whole is more than the sum of its parts” is simply that constitutive characteristics are not explainable from the characteristics of the isolated parts. The characteristics of the complex, therefore, appear as "new" or "emergent".”

Using a Systems Approach to the planning and execution of the PAD 2020 campaign will follow Von Bertalanffy’s construct.  The effort will be based on interactive and collaborative research models that integrate knowledge, methods and perspectives from different disciplines.  The approach promotes rapid exchange and dissemination of information among virtual networks of worldwide collaborators. 

Many other grand challenges (e.g., Transcontinental Railroad, Panama Canal, Manhattan Project, Apollo Program, and the Human Genome Project) have relied on aspects of the Systems Approach.  This model is optimally suited for the aims of PAD2020.   The enterprise will focus its effort to define a goal and then advance solutions on multiple fronts, each with teams of experts working together and among other experts.  Collaboration and interaction are key elements for success.


Will the Campaign involve only basic neuroscience?

The Systems Approach requires integrating knowledge from the broadest possible variety of disciplines and expertise.  The Campaign will involve managing multi-dimensional arrays of knowledge and data sources.  Visually, this could be described as multiple arrays including (note, these examples are representative only, and not exhaustive) basic research, clinical research, and applied science.  Within each array, there are multiple vector covering a variety of disciplines or expertise for example: behavior/neuropsychology, pharmacology, epidemiology, neuroscience, genetics, computational biology, structural chemistry, molecular biology, computer science as well as many other fields.       


Does the state of science warrant such a Campaign?

During a relatively brief span of three decades, research on brain aging and Alzheimer’s disease has yielded significant progress.  The advances in its understanding have propelled this disease from a state of obscurity to the forefront of modern biomedical science.  The indices of this remarkable transformation are the exponential increase in scholarly publications and the dramatic increases in the numbers of investigators. The genes involved in early-onset Alzheimer’s disease and a risk (susceptibility) gene have been identified. Traditional thinking on brain aging has been shattered with the discovery that healthy aging nerve cells can regenerate.  The field has learned a great deal about the genetics, synthesis, degradation, aggregation, toxicity, folding and clearance of abnormal proteins involved in the pathogenesis.  Strategies have been developed on how to prevent toxicity from these proteins.  Intensive studies are underway on multiple fronts, from basic science, to genetics, to drug therapy, to care giving.  Infrastructures to conduct large scale multi-site clinical trials or studies to validate promising biomarkers are in place. The field is more optimistic than ever about the prospects of discovering more effective disease-modifying treatments and, ultimately, interventions for preventing the disease entirely.


Is this too ambitious a goal?

A national strategic goal to prevent Alzheimer’s disease and other conditions affecting memory, movement and mood within a decade will be difficult and costly.  However, the challenges confronting the PAD 2020 mission are no less daunting than other great human endeavors of the past such as:

• Transcontinental Railroad 1862-1860; championed by Abraham Lincoln – completed in 7 years,
• Panama Canal 1904-1914; championed by Theodore Roosevelt – completed in 10 years,
• Manhattan Project 1939-1945; championed by Franklin D. Roosevelt- completed in 6 years, 
• Apollo Program 1961-1969;  championed by John F. Kennedy- completed in 8 years, 
• Human Genome Project 1990-2000; championed by William J. Clinton – completed in 10 years and 

Former Speaker Newt Gingrich, co-chair of the ASG, said in his testimony to the 111th Congress:

“The human pain and financial burden of Alzheimer's is so great and the potential breakthroughs in science are so encouraging that a "Manhattan Project," "Apollo Project," or "Human Genome Project" approach to ending Alzheimer's is more than justified. The Alzheimer's Solutions Project is in the best American tradition of solving a big problem with a big vision and a big effort. A public-private partnership is the best collaborative approach to achieve that vision as rapidly as possible. It is the combination of, first, the scale of the crisis and, second, the breadth of the new science which makes this focused, intense investment and project management approach worth implementing.”

The scientific community feels that there is no longer a question of whether there is a scientific knowledge base to ensure the success of such a bold initiative, but rather if there is a national commitment to move forward.


Is Alzheimer’s too difficulty a problem?

The rationale for the PAD2020 is rooted in a national tradition to undertake formidable and necessary endeavors.  Alzheimer’s disease and other disorders of memory, movement and mood represent problems that will require massive mobilizing of resources and stakeholders in seeking a solution.  The importance of this public health problem is understood globally.

Yet, the collective thinking of over 200 worldwide leaders in dementia research concur that a mission to prevent Alzheimer’s disease within a decade is an attainable scientific objective.  Scientists understand that PAD2020 faces several scientific, administrative and financial challenges.  Yet, the scientific community is also convinced that this is not any more difficult, ambitious, or premature than the other great defining national efforts previously mentioned.


We do not have sufficient knowledge about the cause(s) of the disease?

It is possible that science may not ever achieve “complete” knowledge about the causes of Alzheimer’s disease and the many other disease affecting memory, movement and mood.  However, a nearly crystal clear image is emerging about the consequences of these diseases for patients and their families.  Few would argue against the starkness of this reality.

The PAD2020 Campaign will promote the development of new conceptual models of the disease in order to broaden the range of therapeutic targets and increase the pipeline of potential treatments.  One of the major challenges for prevention is the paucity of validated therapeutic targets that affect neural survival. 

The mission is to expand the current pipelines of potential treatments by fostering collaborative drug discovery programs in academia and industry: with particular focus on validation of new targets for protection against synapse loss, prevention of dendrite pruning, and repair/regeneration of dying neurons.   In parallel, there will be the effort to accelerate the pace of translating newly emerging or promising knowledge (such as, stem cells, gene therapy, neural repair and neuronal regeneration) into practical therapies and interventions. 


All other “Great American Projects” involved technical or engineering solutions.

The public health efforts to combat cholera, dental carries, polio, and neural tube defects are steeped in a tradition that applies biomedical research at the population level. The organization and delivery of scientific knowledge and know-how to vulnerable populations essentially is a technical and engineering solution. 

PAD2020 will follow in this tradition with success assured by the same approach to project management as other great American projects mentioned previously. The keys are:

• Defining clear and specific scientific objectives
• Establishing an efficient organizational and management system – a single centralized administration and coordination center
• Developing realistic research and implementation plans with timelines and deliverables
• Adoption of a Systems Approach to the planning and execution of the effort
• Decisive leadership that yields changes in the governance and organization of research,
• Sustained investment of resources and funds to support the mission; for 10-year with a commitment of $1 billion per year
• Unwavering national commitment to support this mission until completion
  

The “Goal” is too narrow. There are other neurodegenerative disorders. How does PAD 2020 effect other neurodegenerative disorders (in terms of neurology/neuroscience/psychology)?

The launch is envisioned as a benchmark for addressing other life-span brain-behavior disorders that effect memory, movement and mood.  

The mission is much broader than the “Problem of Alzheimer’s”—which is a prototypical example of other chronic disorders that require long-term, labor-intensive, and expensive care.  The ultimate enemy is the problem that patients and families experience every day: the loss of memory and independence.

The goal is to create a new paradigm for planning and supporting the organization of worldwide cooperative research networks to develop new technologies for early detection and treatments for various forms of memory impairments.  In order to accomplish this goal, the federal budget must be increased for research aimed at: a) developing national resources to discover new interventions for memory disorders, and b) creating a streamlined decision-making process for the selection and support of new ideas.


How will this Campaign effect NIH?

The mission of PAD 2020 is to promote and support the programs of NIH by launching activities that are complimentary; particularly in areas where there are gaps or deficiencies in NIH programs or administrative procedures. These include:

• Quick peer-review process and funding of project within 90 days
• Investment/support of high risk innovative ideas/projects
• Commitment to Long-term (beyond five-years) funding commitment e.g., longitudinal studies or prevention trials.
• Supporting entrepreneurial enterprises e.g., public-private partnerships or multi-agency collaborative funding of project
• Multi-site international collaborative studied

 
How will PAD2020 be governed?

On June 24, 2009, Prevent Alzheimer’s Disease, 2020  (or PAD 2020) was incorporated as for the purpose to operate exclusively for charitable, educational, scientific, and literary purposes, within the meaning of Section 501(c)(3) of the Internal Revenue Code of 1986.  The board of trustees and an international advisory board with representatives from academia, industry, government, allied foundations and family support groups will continue to expand.   Presently, the management of PAD 2020 consists of Zaven Khachaturian as President, and Ara Khachaturian serving as Executive Vice-President.

The organizational philosophy of PAD2020 is to create an open forum for the deliberations in planning a national research agenda for ameliorating or preventing neurodegeneration.  The planning process of PAD2020 initiatives will be conducted by a series of “Work Groups” with broad representation of all interest groups and different perspective.  The goal is to provide all interest groups and differing perspectives a level playing field in the deliberations to plan for a national research agenda.


How will quality control (peer-review) be implemented?

PAD2020 will have three levels of control:
a) The ‘Work Groups’, consisting of 10-15 peer-selected team members will evaluate the plans and work products of other Work Groups in an open peer-commentary format.
b) Ad Hoc Committees consisting of key opinion leaders of science, as scientific councilors will evaluate the progress and productivity each Work Group on in an annual open peer-evaluation sessions.
c) External Advisory Boards will evaluation all Work Groups and programs of PAD2020 initiative


Will PAD2020 do away with investigator initiated project?

No, the opposite is true. All projects, programs or initiatives launched or supported by PAD 2020 will be planned, evaluated and managed by teams or individual investigators. The Executive Committee of PAD2020 will manage the process to insure: a) quality control, b) timeliness and c) inclusiveness/representation of differing perspective


What would be the role/relationship with industry?

Industry will become an important partner in developing a national strategic plan. Also PAD 2020 will collaborate with Industry in developing a new model for “Public-Private” partnership in formulating prototypes for academia-industry-government research and development programs in drug discovery and development.


How can I be involved?

Please read, review, and comment on the work of the campaign.