Working to treat and prevent Alzheimer’s disease and related dementias

Dr Richard Hodes, Director of the National Institute on Aging within the U.S. National Institutes of Health, explores the importance of working together to treat and prevent Alzheimer’s disease and related dementias

For some time, scientists have understood that Alzheimer’s disease develops by way of a
complex cascade of events taking place over time inside the brain. These events, influenced by both genetic and non-genetic factors, contribute to changes in the brain that disrupt functioning and are at the centre of the notorious devastation caused by this mind-robbing disease.

Driven by the growing ageing population at risk for Alzheimer’s and related dementias (including Lewy body dementia and frontotemporal dementia) and the staggering costs they impose on individuals, families and society, the sense of urgency to develop an
effective treatment has never been greater. Estimates indicate that some 46.8 million people age 60 and older lived with dementia worldwide in 2015, a number expected to grow to 74.7 million people in 2030 and to 131.5 million in 2050 (Prince et al., 2013).

The National Institute on Aging (NIA) at the National Institutes of Health (NIH), which leads the U.S. biomedical research effort on Alzheimer’s and related dementias, is spearheading an ambitious research agenda to better understand, diagnose, prevent and
treat these diseases, with a goal of finding effective treatments by 2025. Thanks to extraordinary boosts in Congressional appropriations for Federal research in recent years, we can now pursue the answers to many fundamental questions – as well as test some of these hypotheses in translational and clinical studies – that we couldn’t address in the not-too-distant past.

We are now better positioned to squarely focus our attention on the heterogeneity of disease – how dementia manifests differently among individuals and across groups – and to set our sights on a precision medicine approach, targeting disease-relevant processes and delivering the right treatments at the right stage of the disease for each person affected.
While, ultimately, we want to prevent or slow Alzheimer’s disease and related dementias, we are working towards serving the needs of all patients at all stages of the disease.

NIH funding has been applied to a broad multidisciplinary programme where research moves through a pipeline from studies of basic mechanisms to an application in clinical trials and studies.

Our efforts are broad and far-reaching, directed to:

• Enabling precision medicine
• research through advances in genomic sciences and deep molecular
  phenotyping of existing cohorts and the launch of new diverse cohorts;
• Using an open science research model of the Accelerating Medicines Partnership for Alzheimer’s Disease to hasten the discovery of the next generation of therapeutic targets and biomarkers;
• Creating new translational infrastructure programmes to enable rapid sharing of data and research models and enhance research rigour and reproducibility;
• Developing emerging therapeutics in academic centres and in the small business community; and
• Making advances in disease monitoring, assessment and care, powered by the revolution in mobile technology, which are helping us bring in people living with AD/ADRD and their caregivers, as direct partners in research.

Several efforts focus on “open science,” a participatory approach to research in which progress is accelerated by making research data, methods and tools available to all qualified investigators. NIH has been taking a leadership role in promoting the broad availability of research data for quite some time. With the rise of big data and new analytical approaches that can help us to better understand human wellness and disease in a person-specific manner, this focus is vital to our progress.

Reflecting from the halfway mark to the 2025 national goal, we recognise more clearly than ever that Alzheimer’s disease and related dementias are complex and challenging foes. Yet our scientific capabilities and momentum are growing rapidly. We are closing in on the research advances that may ultimately contribute to an end to a public health crisis that has penetrated the United States and the world in a way that few other conditions have.

We also need public support – none of these efforts will be successful unless we engage the public and secure the commitment of people from all walks of life to participate in clinical research.

NIA is uniquely positioned to keep the momentum in discovery going and to push forward not only an agenda but concrete, specific guidance and efforts to turn the tide for the better for millions of affected people – and their families, friends and communities – who need solutions.

Richard J. Hodes, MD
Director
National Institute on Aging
National Institutes of Health
Tel: +1 301 496 1752
nianews3@nia.nih.gov
www.nia.nih.gov