“In every age group, African Americans and Hispanics are at higher risk for Alzheimer’s disease…Clinical trials should include these higher-risk populations and should oversample in order to test hypotheses.”
Jennifer Manly, Ph.D., spoke emphatically as she delivered this recommendation to the Alzheimer’s Disease Research Summit 2012: Path to Treatment and Prevention. A neuropsychologist who studies minority aging at Columbia University, New York City, Dr. Manly was one of 57 speakers and panelists reviewing, debating, and recommending national research priorities for Alzheimer’s disease at the Summit held May 14-15, 2012, at the NIH campus, Bethesda. Her perspective and that of several other speakers highlighted the need for more research on minorities and Alzheimer’s, among recommendations covering basic biological studies, genetic research, and translational and clinical investigations.
The Summit, hosted by the U.S. Department of Health and Human Services (HHS) and its operating division NIH (with support from the Foundation for the NIH), was a major step toward implementing the National Alzheimer’s Project Act (NAPA) legislation of 2011. Fulfilling requirements set out by NAPA, HHS Secretary Kathleen Sebelius formally released the National Plan to Address Alzheimer’s Disease, the number one goal being to prevent and effectively treat Alzheimer’s disease by 2025. During the Summit specific strategies and action steps were developed to help meet that goal.
The meeting attracted more than 500 international participants, including scientists from academia and industry, advocates, and members of the public invested in the National Plan to Address Alzheimer’s Disease. In addition to those attending the meeting, nearly 500 others followed the proceedings online (videocasts for each day—May 14 and May 15—are available on the NIH website). Speakers, panelists, and attendees urged new or intensified approaches to research. The proceedings resulted in a set of 40 recommendations which are intended to guide research directions nationally and possibly internationally—an effort that requires the involvement of the federal government, academia, industry, advocates, and the many volunteers needed for research trials and studies.
Two of the recommendations focus on higher-risk populations:
Expand large-scale registries and natural history cohorts of healthy individuals from early midlife to late life, as well as individuals with subjective and/or objective cognitive impairment and use the data generated to inform clinical trial design. These cohorts should be population-based and should oversample underrepresented ethnic minorities and groups with lower education.
Develop approaches to stratify and individualize treatments based on the diversity of symptomatic patient populations.
Other Summit recommendations call for a multidisciplinary research agenda, including basic and translational studies. Prevention and treatment for both the disease and its symptoms—cognitive as well as neuropsychiatric—are also a focus. In addition, recommendations address infrastructure, resources, and new partnerships between the government, academia and private industry.
Several overarching recommendations emerged repeatedly in presentations and discussion:
Recognize the heterogeneity and the multifactorial nature of the disease.
Employ new research paradigms such as systems biology and network pharmacology.
Enable rapid and extensive sharing of data, disease models, and biological specimens.
Build new multidisciplinary translational teams and create virtual and real spaces where these teams can operate.
Develop strategies to overcome intellectual property barriers to Alzheimer’s disease drug development.
Develop new public-private partnerships.
Establish a National Institutional Review Board (IRB) for Alzheimer’s disease clinical research.
The NIH, based in part on the Summit recommendations, released funding opportunity announcements (FOA) in October 2012—applications are due in January 2013—that seek research proposals in some key areas. The FOAs are available online:
The opportunities are dependent on available funding, through NIH’s base funding or through an additional $80 million that HHS seeks to direct to Alzheimer’s research in fiscal 2013. This support will be in addition to an extra $50 million earmarked for Alzheimer’s research in the NIH’s 2012 budget.
The additional fiscal 2012 funding allowed NIA to move forward and fund a number of important studies, including two new clinical trials. One is a trial testing whether a nasal spray delivering insulin directly to the brain can improve memory, cognition, and daily functioning. The other is a 5-year prevention trial—the first to test promising therapies on people who are cognitively normal, but at very high risk for developing early-onset Alzheimer’s, a rare, familial form of the disorder.