Early-onset Alzheimer’s and other dementia: From natural history to clinical trials

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PROJECT SUMMARY Approximately 5.8 million Americans age 65 and older have Alzheimer's disease, and this number is expected to increase to 13.8 million by 2050. While most develop diagnosable symptoms at or over the age of 65 (e.g. late-onset), 5-10% develop symptoms at age 64 or younger and are classified as early-onset. Early- and late- onset Alzheimer's disease share the same pathologic substrate, but there are critical differences in their clinical presentations, biological phenotypes, and life impact. Moreover, early-onset dementia is more likely than late- onset dementia to be caused by pathologies other than Alzheimer's, including vascular, Lewy body, and frontotemporal degeneration. Because of their young age and atypical symptoms, early-onset patients often face a significant delay to diagnosis. Dementia onset at such a young age also has disproportionately devastating financial and emotional consequences for patients, families, and society. Due to age restrictions or absence of memory deficits, early-onset patients are under-represented in ongoing largescale genome and observational biomarker studies and in therapeutic trials. To date, no large-scale clinical trials have specifically addressed early-onset MCI and dementia. The Alzheimer's Association (AA) proposes to create and implement a two-day conference, to be held annually for 3 years, entitled Early-Onset Alzheimer's and Other Dementia: From Natural History to Clinical Trials. Patients, carers, and family members will be involved in the planning, implementation, and evaluation components of the conference. The short-term goal of the conference is to create a forum for discussion of research, trial design, drug development, and other concerns among key stakeholders. The long-term goal is to increase the likelihood, quality and patient-centeredness of future clinical trials addressing early-onset MCI and dementia. A Conference Workgroup made up of the PI and collaborators, additional leading experts in dementia, and individuals with early-onset MCI or dementia and/or their family members will develop, implement, and oversee all conference, evaluation, and dissemination activities. The conferences will be held in coordination with the annual Alzheimer's Association International Conference (AAIC) and offer didactic, panel discussion, and poster based formats, as well as face-to-face networking opportunities, anticipating 250 attendees. Lunch poster sessions dedicated to the early career investigators will be held on both days of the conference, and multiple approaches will be used to ensure diversity amongst speakers and attendees. Dissemination efforts will include webinars, press releases, social media highlights, and manuscripts documenting conference proceedings and findings.