Alzheimer's Disease-Related Dementias (ADRD) Summit 2016
Prioritized Research Milestones
National Institute of Neurological Disorders and Stroke
November 2016
Printer Friendly Version in PDF Format (21 PDF pages)
Table of Contents
- Topic 1: Multiple Etiology Dementias (MED)
- Topic 2: Non-Governmental Organizations (NGOs)
- Topic 3: Health Disparities (HD)
- Topic 4: Lewy Body Dementias (LBD)
- Topic 5: Frontotemporal Lobar Degeneration (FTD)
- Topic 6: Vascular Contributions to Cognitive Impairment and Dementia (VCID), Including Vascular Cognitive Impairment and Vascular Dementia
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Topic 1: Multiple Etiology Dementias (MED) | ||
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Focus Area 1: Improved Diagnostic Skills in the Community | ||
Milestone | Success Criteria | Timeline (Timeframe)1 |
1. Detect cognitive impairment when patient or relative voices a concern to health care providers. |
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3-7 years (2017) |
2. Improving differential diagnosis of symptomatic cognitive impairment. |
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3-7 years (2017) |
3. Increase training of health professionals to meet the expanding demand for cognitive impairment and dementia diagnosis and care, as well as the critical challenges of and need for human-based research. |
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7-10 years (2017) |
4. Develop diagnostics/biomarkers in asymptomatic individuals. |
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3-7 years (2017) |
Focus Area 2: Basic and Clinical Research in Interactions between Dementia Pathophysiologies | ||
Milestone | Success Criteria | Timeline (Timeframe)1 |
5. Promote basic and clinical research in multi-etiology dementia. |
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3-7 years (2017) |
Focus Area 3: Determining the Role for Screening for Cognitive Dysfunction | ||
Milestone | Success Criteria | Timeline (Timeframe)1 |
6. Determining the value of screening for clinically relevant cognitive impairment in the absence of a cognitive complaint. |
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7-10 years (2017) |
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Topic 2: Non-Governmental Organizations (NGOs) | |||
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Focus Area 1: Catalyzing Research through Unique Programs and Partnerships | |||
Milestone | Success Criteria | Timeline (Timeframe)1 | |
1. Establish more effective communication between NIH and NGOs on activities and progress toward ADRD goals in the off-years between triennial ADRD Research Summits. |
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Ongoing activity (2016) |
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Special Joint NGO & MED Topic: Addressing Nomenclature for Discussing Cognitive Impairment and Dementia | |||
NGO Focus Area 2: Nomenclature Standards when Discussing Dementia MED Focus Area 4: Revisiting the Nosology of Cognitive Impairment in Late Life | |||
Milestone | Success Criteria | Timeline (Timeframe)1 | |
2. (NGO) Organize a working group of dementia stakeholders, including founding partnerships with health disparities communities, to review the current nomenclature used in public awareness, clinical care services and research and to propose strategies to help advance early differential diagnosis and the understanding of dementia and its underlying causes. |
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Ongoing activity (2017) |
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7. (MED) Developing a consistent nomenclature in Dementia Research and Care. | |||
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Topic 3: Health Disparities (HD) | ||
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Focus Area 1: Treatment and Prevention Strategies | ||
Milestone | Success Criteria | Timeline (Timeframe)1 |
1. Assess epidemiology and mechanistic pathways of disparities in health burden of AD/ADRD. |
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3-7 years (2016) |
2. Enrich the design of trials of vascular health interventions to improve their application to AD/ADRD among aging diverse populations. |
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3-7 years (2017) |
Focus Area 2: Monitoring Changes in AD/ADRD Disparities | ||
Milestone | Success Criteria | Timeline (Timeframe)1 |
3. Develop a system to monitor the magnitude and trends in health disparities in incidence of AD/ADRD. |
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3-7 years (2017) |
Focus Area 3: Assessment | ||
Milestone | Success Criteria | Timeline (Timeframe)1 |
4. Improve tools for assessment of disparities in risks, preclinical disease characteristics, and costs of AD/ADRD among health disparities populations by leveraging existing data and cohorts, designing targeted studies, and using advanced psychometric analyses for improving tools for assessment of disparities in risks, preclinical disease characteristics, and costs of AD/ADRD among health disparities populations. |
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1-3 years (2016) |
5. Increase utilization of culturally- and linguistically-appropriate assessment tools within ongoing and newly generated studies of AD/ADRD and vascular health intervention trials. |
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1-3 years (2016) |
Focus Area 4: Community Partnerships, Recruitment, and Retention | ||
Milestone | Success Criteria | Timeline (Timeframe)1 |
6. Generate an AD/ADRD Health Disparities Task Force that is specifically designed to provide guidance and expertise for community engagement, study design, recruitment and retention into sites to ensure recruitment of diverse populations into newly generated epidemiological studies and clinical trials. |
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1-3 years (2016) |
7. Develop novel community engagement and outreach methods and identify existing methods to facilitate engagement, understanding and partnership with health disparities populations. |
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1-3 years (2016) |
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Topic 4: Lewy Body Dementias (LBD) | ||
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Focus Area 1: Establish Longitudinal Diverse Cohorts with Common Measures, Culminating in Autopsy | ||
Milestone | Success Criteria | Timeline (Timeframe)1 |
1. Initiate clinical trials for motor and non-motor manifestations of Lewy Body dementias (LBD), which is meant to include both dementia with Lewy bodies (DLB) and Parkinson’s disease dementia (PDD), in diverse populations using existing and newly developed therapies that address symptoms that have the greatest impact on patient function and caregiver burden. |
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1-3 years (2016) |
2. Create longitudinal clinical, biological, and imaging resources for LBD from the earliest stages to autopsy to improve accuracy of detection and diagnosis of DLB at the pre-dementia or prodromal stage and to detect PD patients with a high risk of cognitive decline leading to PDD. |
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7-10 years (2016) |
Focus Area 2: Discover Disease Mechanisms Through Brain Mapping and Genetics | ||
Milestone | Success Criteria | Timeline (Timeframe)1 |
3. Using well defined cohorts of LBD who have come to autopsy, systemically characterize disease-specific changes in the brain, spinal cord, and peripheral autonomic nervous system with state-of-the-art methods, including genomics, expression arrays, metabolomics, and proteomics to identify underlying disease mechanisms that will guide future biomarker and therapeutic approaches. Data generated in this initiative should be incorporated into an open-access, centralized data management system that links clinical, biological, and autopsy data. |
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3-7 years (2018) |
4. Identify novel common and rare genetic variants, epigenetic changes, and environmental influences that impact the risk for and clinical features of LBD. |
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3-7 years (2016) |
Focus Area 3: Develop and Validate Biological and Imaging Biomarkers | ||
Milestone | Success Criteria | Timeline (Timeframe)1 |
5. Develop imaging approaches to 1) enhance the differential diagnostic accuracy of LBD compared to other dementing illnesses, 2) detect latent and prodromal LBD, and 3) monitor disease progression in natural history and treatment studies by integrating established and new imaging tools. Validate these tools against postmortem neuropathology. |
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3-7 years (2016) |
6. Use new (see 4.1.2.) or existing longitudinal case-control studies of individuals with LBD, longitudinal cohort studies tracking cognitive decline, or studies capturing incident cases of LBD, to develop biomarkers for LBD-related pathologic changes, diagnosis, differential diagnosis, disease progression, and the relative amount of Alzheimer’s and other pathologies. As new markers of molecular disease mechanisms are discovered, incorporate them into biomarker studies for diagnosis of latent or prodromal disease and for monitoring molecular processes and their response to therapies. |
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3-7 years (2017) |
Focus Area 4: Model Disease Processes to Develop Potential Symptomatic and Disease Modifying Therapies | ||
Milestone | Success Criteria | Timeline (Timeframe)1 |
7. Recognizing the importance of alpha-synuclein and AD pathophysiologic processes in LBD, new animal, cellular, and in vitro models are needed that recapitulate key features, including clinical heterogeneity, of these disorders with the ultimate goal of identifying strategies that can be carried forward into clinical trials. |
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7-10 years (2017) |
8. Develop disease-modifying interventions for LBD based on discovering biomarkers, molecular targets, and genetic and environmental modifiers that enhance, delay or prevent the onset of disease. |
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7-10 years (2018) |
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Topic 5: Frontotemporal Lobar Degeneration (FTD) | ||
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Focus Area 1: Basic Science: Pathogenesis and Toxicity | ||
Milestone | Success Criteria | Timeline (Timeframe)1 |
1. Clarify the mechanism of tau pathogenesis and associated neurodegeneration. |
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3-7 years (2016/2017) |
2. Determine the molecular basis for C9ORF72 expansion-and GRN mutation-related neurodegeneration. |
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7-10 years (2016/2017) |
3. Determine the mechanism of TDP-43 and FUS pathogenesis and toxicity. |
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7-10 years (2016/2017) |
4. Develop better FTLD in vivo and cell-based model systems. |
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1-3 years (2016/2017) |
Focus Area 2: Clinical science | ||
Milestone | Success Criteria | Timeline (Timeframe)1 |
1. Expand efforts to genotype patients with FTD and identify new genes and their functional relationship to FTLD pathogenesis. |
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1-3 years (2017/2018) |
2. Develop FTD biomarkers for diagnosis and disease progression. |
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3-7 years (2017/2018) |
3. Create an international FTD clinical trial network. |
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1-3 years (2017/2018) |
4. Understand phenotypic heterogeneity and natural history. |
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>10 years In progress |
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Topic 6: Vascular Contributions to Cognitive Impairment and Dementia (VCID), Including Vascular Cognitive Impairment and Vascular Dementia | ||
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Focus Area 1: Basic Mechanisms and Experimental Models | ||
Milestone | Success Criteria | Timeline (Timeframe)1 |
1. Develop next-generation experimental models and translational imaging methods for VCID. Establish new animal models that: (i) reproduce small vessel disease and other key pathogenic processes thought to result in cognitive impairment; (ii) are easily applicable to both VCID and AD research for advances in mixed etiology dementias; (iii) address vascular contributions to dementia via both white matter and grey matter or (iv) include genetic and acquired conditions that are associated with VCID. |
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3-7 years (2016) |
2. Encourage basic science research that investigates the impact of aging, AD pathology, and genes on peri- and para-vascular clearance mechanisms, the NVU, and cerebrovascular function. |
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3-7 years (2018) |
3. Encourage basic science research that investigates the impact of cerebrovascular risk factors/genes and atherosclerosis on AD-related neurodegeneration. |
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3-7 years (2018) |
Focus Area 2: Human-Based Studies | ||
Milestone | Success Criteria | Timeline (Timeframe)1 |
1. Develop and validate longitudinally tracked noninvasive markers of key vascular processes related to cognitive and neurologic impairment. | Development:
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1-3 years (2016) |
Validation:
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3-7 years (2018) |
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2. Determine interrelationships (cross-sectional and longitudinal) among aging, cerebrovascular disease and risk factors, resilience factors, genetic variants, amyloid, tau, and neurodegeneration. |
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3-7 years (2018) |
3. Identify lifestyle and vascular interventions to treat, prevent, or postpone VCID. |
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7-10 years (2022) |
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