Alzheimer’s disease is the sixth leading cause of death in the United States, a statistic that underscores the critical need for new insights to help us understand and treat this challenging condition. Among the more exciting research efforts underway is a PCORnet® Study focused on identifying existing drugs that could be repurposed as effective treatments for Alzheimer’s disease.

The PCORnet® Study of Computational Drug Repurposing to Treat Alzheimer’s and Related Dementias uses data from clinical sites in two Clinical Research Networks participating in PCORnet (OneFlorida+ and INSIGHT), which include patients from Florida and New York City. The study team will apply artificial intelligence methods such as machine learning and natural language processing to patient data to identify the key characteristics of patients with Alzheimer’s disease, as well as meaningful outcomes indicating improvement in the disease progression. Then, the study team will use these insights to create and validate a framework that can generate hypotheses about the potential use of a repurposed drug for influencing Alzheimer’s disease progression from real-world data.

Using such a framework could support researchers in understanding the extent that a particular drug may influence Alzheimer’s disease progression. At the end of the study, the study team will disseminate the framework and ensure it is publicly accessible and open source.

The study team has already published two foundational manuscripts that will underpin its work: a systematic review of observational studies exploring the association between newer glucose-lowering drugs and dementia and a publication demonstrating that the team’s natural language processing approach works to extract Alzheimer’s disease biomarkers and cognitive tests from electronic health records.

The promise of using PCORnet to support patients with Alzheimer’s and dementia

Drug repurposing is increasingly coming to the fore of Alzheimer’s disease and dementia research because of its potential to speed up the development of new therapies. Finding effective treatments for Alzheimer’s disease and related dementia is challenging because the condition is slow to progress and manifests differently in each person.

These complexities mean that traditional research approaches often fall short, prompting research teams to increasingly explore the use of distributed research networks like PCORnet, which can connect these teams to broader, longitudinal, and more varied insights than typically allowed through traditional research. The PCORnet infrastructure lends itself to a multitude of impactful research approaches to support Alzheimer’s and dementia. While the Computational Drug Repurposing study is using PCORnet for a retrospective observational analysis of real-world data, other study teams have found success leveraging PCORnet for interventional drug repurposing research. For example, the PREVENTABLE study team used PCORnet resources to support enrollment for one of the largest research studies in adults aged 75 or over. PREVENTABLE is a pragmatic study that is exploring whether taking a statin can help prevent dementia.

Are you interested in using PCORnet resources to power your next research effort? Reach out to the PCORnet® Front Door to start the conversation.