Globally, more than 55 million people are living with Alzheimer’s disease or other dementias, with almost 10 million new cases each year. While there is currently no way to prevent or cure this fatal neurological disorder, researchers are hard at work studying the disease and searching for treatments.
Through its grants and awards, the PhRMA Foundation has supported scientists working at the frontiers of Alzheimer’s disease research, from identifying genetic risk factors and novel therapeutic targets to assessing the impact of health policies on quality of care for people with dementia. We asked several PhRMA Foundation awardees about their Alzheimer’s disease-related research and what they hope to accomplish with their work.
Studying the Genetics Behind Resilience
Almost a decade ago, Timothy Hohman, PhD, received a PhRMA Foundation Postdoctoral Fellowship in Translational Medicine to research genetic markers that might predispose some people toward resilience against Alzheimer’s disease. He used an approach called data harmonization to pool lots of datasets together to enable genetic analysis. “The Foundation provided the start that I needed to demonstrate that harmonization was possible, and that resilience has a genetic basis,” Hohman said.
Today, as an associate professor of neurology in Vanderbilt University’s Memory and Alzheimer’s Center, Hohman’s work builds on those initial efforts as he seeks to identify a treatment based on the innate protection of resilient individuals. “Understanding even a single story of protection in the face of disease can transform our knowledge of disease biology and move the field in a new direction,” he said. “We just need to harness the heterogeneity to find the patterns of protection that exist in the population already.”
Searching for Treatments and Pre-Symptomatic Diagnostics
Mark Ebbert , PhD, assistant professor in the Sanders-Brown Center on Aging at the University of Kentucky, received a 2017 Foundation Starter Grant in Translational Medicine to support his work using breakthrough sequencing technologies and bioinformatics to study the biological mechanisms of Alzheimer’s disease. “Because of the investment the PhRMA Foundation made in my research, I have since successfully competed for nearly $5 million in additional grant funds and have established a full-size genomics lab,” he said.
In the long run, Ebbert hopes to help develop both a meaningful treatment and a pre-symptomatic diagnostic for Alzheimer’s disease. “Alzheimer’s disease is not like other ailments like physical injuries or cancer that our bodies can heal from with treatment; we simply cannot heal from Alzheimer’s disease,” he said. “By the time Alzheimer’s disease symptoms onset, it’s too late to treat. That’s why a pre-symptomatic disease diagnostic is equally important to a meaningful treatment.”
Untangling a Hallmark of Alzheimer’s Disease
Jessica Fortin, DVM, PhD, recipient of a 2021 Foundation Starter Grant in Drug Discovery, is also on the search for an Alzheimer’s treatment. In her lab at the Purdue University College of Veterinary Medicine, she researches the buildup in the brain of tangles of twisted proteins called tau, a hallmark of Alzheimer’s disease. Her team is working to develop novel small-molecule compounds that can inhibit this buildup and disaggregate existing tangles.
“In our lab, there are a few small molecules proven capable of completely halting the process of aggregation of tau (involved in tangle formation) in test tubes,” she said. “Eventually, we hope to use an animal model to test one or more of our anti-aggregate small molecules, which is one step closer to developing clinical trials for a potential cure for Alzheimer’s disease.”
Examining How Health Policies Affect Care for People with Dementia
The recipient of a 2021 Foundation Starter Grant in Health Outcomes Research, Douglas Barthold, PhD, studies the impact of health policies on people with dementia. At the Comparative Health Outcomes, Policy, and Economics (CHOICE) Institute at the University of Washington, he seeks to better understand “how health policies like insurance design affect risk factors for dementia and also the value of health care for people with dementia.”
“There are 6.5 million people aged 65 and older with Alzheimer’s disease in the U.S. This age range coincides with Medicare eligibility, meaning that there is potential for Medicare to influence and improve care for these individuals,” Barthold said. Through his work, he hopes to reduce the burden for both dementia patients and caregivers by informing policies and insurance design to help “modify risk factors for dementia and improve the quality of care.”
Alzheimer’s disease takes an immense toll on patients, families, caregivers, and the health care system. The PhRMA Foundation is inspired by our awardees’ efforts to advance innovative and transformative research in Alzheimer’s disease. We share in the same hope as our awardees that through scientific discovery, earlier diagnosis, new treatments, and better evidence-based care will be able to improve outcomes for Alzheimer’s disease patients.
Read the full Q&A with each researcher: