A Need to Change Clinical Research Approaches in Studying Alzheimer's Disease

The Physicians Committee

Good Science

A Need to Change Clinical Research Approaches in Studying Alzheimer's Disease

  February 17, 2017  


A Need to Change Clinical Research Approaches in Studying Alzheimer's Disease

Alzheimer’s disease is the most common form of dementia, affecting more than 5 million Americans. This number is expected to triple in prevalence by 2050, which is estimated to quadruple health care costs to $1 trillion.

To prevent this oncoming public health crisis, in 2011 Congress passed the National Alzheimer’s Project Act (NAPA) to create a national strategic plan coordinating the federal government’s efforts to treat and prevent dementia. An Advisory Council on Alzheimer’s Research, Care, and Services, consisting of members from relevant federal agencies and nongovernmental organizations, meets quarterly to evaluate progress, update the strategic plan, and make recommendations to Congress.

At the Council’s Feb. 3 meeting, Physicians Committee scientist Feng-Yen Li, Ph.D., delivered a public commentary to outline impediments to progress to find an effective intervention to treat or prevent Alzheimer’s disease. For example:

  • Drug candidates are initially tested in animal models that do not recapitulate the human disease, unlike human-based models, which are derived from patients or patient data.
  • Patients with chronic health conditions (e.g., diabetes, cardiovascular disease) that often occur alongside the most common form of Alzheimer’s disease are often excluded from participating in clinical trials, which means important underlying factors may be overlooked and the drugs may fail to work in these patients.
  • Drug targets for clinical trials are often based on genetic defects or pathways characterized in the rare inherited form of Alzheimer’s but are tested in patients with the common form of Alzheimer's who may or may not have genetic risk factors.
  • Alzheimer's drug development is often focused on reducing the signs of pathology such as brain plaques. However, these may just be a consequence rather than a cause of the disease. Little focus is on the underlying lifestyle risk factors of the disease.

Dr. Li strongly recommended the Council direct future funding and research efforts in Alzheimer’s disease to clinical trials based on data derived from humans or human-based models and focused on modifying lifestyle risk factors such as diet. A copy of the full commentary is available here.

To further promote these concepts, the Physicians Committee’s Ann Lam, Ph.D., has organized a discussion panel on Shifting Perspectives on Dementia, Science, and Health Policy at the 2017 American Association for the Advancement of Science meeting, featuring three experts in Alzheimer’s prevention research: Drs. Rhonda Au, a professor at Boston University; Jessica Langbaum, an associate professor at the Banner Research Institute; and Neal Barnard, president of the Physicians Committee.