More than 500 leading researchers and dedicated advocates gathered at the Alzheimer’s Disease Research Summit 2015: Path to Treatment and Prevention with one goal in mind—to ramp up a scientific agenda aimed at treating and preventing Alzheimer’s disease (AD) and related dementias by 2025.
Convened by NIA and the U.S. Department of Health and Human Services with private support through the Foundation for the NIH, the meeting, held February 9-10, 2015, revealed research challenges as wide-ranging and complex as Alzheimer’s itself, as well as determination to surmount the devastating neurodegenerative disorder.
“I think we’ve entered a new era in Alzheimer’s research. There are new models of collaboration and data sharing. There are fantastic advances scientifically,” said NIH Director Dr. Francis Collins in opening remarks to the audience, which included another 500 people tuning in via webcast.
More than 60 speakers (PDF, 4.3M) highlighted new knowledge and directions for AD research, from the disease’s epidemiology and etiology to biomarkers, drug discovery, and clinical trials testing new treatments and prevention strategies. Future directions addressed innovations in Alzheimer’s care, such as using sensor technology to monitor people at home for early indicators of cognitive decline, and the emergence of “citizen science,” a consumer-driven movement to help shape and participate in biomedical research.
“We are at an exciting time in science,” said NIA Director Dr. Richard J. Hodes, noting increased funding for a variety of scientific studies, public-private partnerships, and data-sharing tools to help accelerate discovery of new treatments and better understand the many factors involved in the causes and progression of AD.
Dr. Hodes featured collaborations such as the International Alzheimer's and Related Dementias Research Portfolio (IADRP), a publicly available database of global Alzheimer’s research created by NIA and the Alzheimer’s Association. “IADRP helps us gain perspective on where there are research gaps and where new initiatives are required,” he said, inviting all public and private funders of research on AD and related dementias to contribute to the database.
Partnering to strengthen Alzheimer’s research
Building on the success of the NIA-organized 2012 Alzheimer’s research summit, this year’s summit will result in research recommendations for the National Plan to Address Alzheimer’s Disease. The Plan seeks to overcome Alzheimer’s and other dementias by expanding and improving research, care, and services for affected people, families, and caregivers. Its primary research goal is to find effective therapies to treat or prevent AD by 2025.
The most common cause of dementia, Alzheimer’s affects more than 5 million Americans age 65 and older. Without effective interventions, the number is projected to grow to 13.8 million by 2050 as the population ages. Current Alzheimer’s treatments can temporarily allay symptoms but do not change the course of the disease.
It’s become clear, Dr. Collins said, that no one government agency, university, company, or nonprofit alone can beat Alzheimer’s. “We can work more effectively together than separately,” he said.
Dr. Collins pointed to the Accelerating Medicines Partnership (AMP), a groundbreaking, NIH-led venture that brings together industry, academia, advocacy groups, and government agencies, as a key example. AMP aims to accelerate drug development in three areas, including AD. The newly launched AMP AD Knowledge Portal will enable sharing and analysis of large, complex biomedical data sets by the wider research community.
Other major partnerships featured at the summit include NIH’s BRAIN Initiative to create next-generation neuroimaging tools that deepen understanding of brain circuitry and the Global Alzheimer’s Association Interactive Network, which shares clinical data for more than 280,000 individuals from research organizations around the world.
These and other initiatives, including the ongoing Alzheimer’s Disease Neuroimaging Initiative and the work of the NIA-supported Alzheimer’s Disease Centers, rely on rapid data sharing by scientists to inform each other’s research. Tools that pool, standardize, and analyze large amounts of data—including brain images, tissue and fluid samples, and genetic information—enable large-scale collaborations.
“We are improving the accuracy and speed with which we work in a digital environment,” said Dr. Philip Bourne, associate director of data science at NIH and director of the agency’s Big Data to Knowledge (BD2K) program.
BD2K’s first high-profile study involved an international team of roughly 300 scientists in a network dubbed ENIGMA (Enhancing Neuro Imaging Genetics Through Meta Analysis), who used imaging and genetic data from more than 30,000 individuals to identify eight common genetic mutations that appear to age the brain. The discovery could help chart a path toward the development of targeted therapies and interventions for neurological conditions such as Alzheimer’s, scientists say.
A call to integrate research, develop new models
Despite years of failed attempts to find an effective treatment for AD, scientists at the summit expressed hope that disease-modifying treatments are within reach. Some called for better integration of research areas, already underway in some projects, to reach this goal.
The future of Alzheimer’s research, and specifically drug discovery, may lie in a model that combines clinical and pathologic data, aided by powerful computational biology tools, said Dr. David Bennett, director of the NIA-supported Rush University Alzheimer's Disease Center, Chicago. Such an approach could help scientists better understand the many causes of AD and how they interact—and, ultimately, could more quickly pinpoint better drug targets and find effective interventions.
Preventing or treating pathologic Alzheimer’s disease alone will only prevent a portion of Alzheimer’s dementia and mild cognitive impairment,” Dr. Bennett noted. “It’s a significant portion, and we should continue to push down that road, but we need other novel approaches.
That means combining study of the formation and spread of harmful amyloid plaques, tau tangles, and other biological damage in the Alzheimer’s brain with genetics and clinical factors such as chronic disease, diet, sleep, and the environment, he and others said.
A wide range of research
The Alzheimer’s Disease Research Summit 2015 was organized around major themes of critical importance for identifying successful interventions:
- The complex biology of Alzheimer’s. This session addressed key questions about the complexity of AD, such as the role of cerebral microvasculature and inflammation. Approaches to identify and quantify disease trajectories and risk and ways to harness the power of Big Data were also discussed.
- Transforming AD therapy development. This session featured new approaches to target and biomarker discovery for AD and clinical trial design. It highlighted successful use of systems-based, data-driven approaches to drug repositioning and combination therapy development. For example, “systems pharmacology” can help inform future trials by analyzing why past anti-amyloid clinical trials have failed, said Dr. Julie Stone of Merck.
- New approaches to prevention. Reducing AD risk and/or delaying disease onset will greatly reduce the socioeconomic burden of Alzheimer’s. This session reviewed the current understanding of disease progression and featured advances in genetics, epigenetics, epidemiology, and cognitive/behavioral sciences that can inform the development of prevention strategies. “A more comprehensive view of environmental exposure is needed to discover the major causes of diseases today,” noted Dr. Chirag Patel of Harvard Medical School.
- Innovative monitoring, assessment, and care. Advances in technology offer unprecedented opportunities to assess and monitor patients’ well-being in real time and to optimize and customize the delivery of care. This session focused on these innovative possibilities, as well as research gaps in integrating care. “We want to expand our models of care to capture this whole continuum,” said Dr. Christopher Callahan of Indiana University. “We’re hoping technology makes some of these models scalable” so they can reach more people.
- Empowering participants, engaging citizens in research. The acceleration of AD therapy development in large part depends on citizen awareness and engagement. This session addressed research-volunteer recruitment, health disparities, and citizen participation in creating new methods for informed consent, data sharing, and trial design. “The number one reason African Americans don’t participate in clinical trials is because they’re not being asked,” said Stephanie Monroe, director of the African American Network Against Alzheimer’s. “Our job is to make it easier for them to get to yes.”
- Partnerships for open innovation. This session highlighted transformative programs and public-private partnerships that promote an open-source philosophy in biomedical research and drug development. Speakers discussed how these new approaches can accelerate AD therapy development.
To learn more about the Alzheimer’s Disease Research Summit 2015, view the NIH videocast for Day 1 and Day 2.