The 20th International Conference on Alzheimer’s Drug Discovery, a two-day interdisciplinary forum held September 16-17, 2019 in Jersey City, NJ, brought together 20 top researchers to present a diversity of approaches and the latest on innovative treatments and diagnostics for Alzheimer’s disease, related dementias and cognitive aging.
Hosted by the Alzheimer's Drug Discovery Foundation (ADDF), the 20th year of this meeting is an important landmark for ADDF – reflecting progress in the field over the past two decades.
“When you look back at our earliest meetings, programs were primarily focused on early stage preclinical drug development,” said Dr. Howard Fillit, founding executive director and chief science officer of the ADDF. “Today, we are showcasing results from novel preclinical targets, completed clinical trials, and new biomarker modalities such as blood and eye tests. Research advances are rapidly bringing drug discovery closer to important and practical applications.”
Several presentations by ADDF-funded investigators and other guest speakers presented findings on both novel and repurposed drug programs and biomarkers at various stages of the pipeline. Additionally, perspectives from other neurodegenerative diseases were shared, such as frontotemporal dementia and ALS, alongside academic and startup drug development programs targeting a wide variety of areas including vascular pathology, neuroprotection and inflammation.
Conference highlights included:
In a session focused on clinical trials, Jeffrey Cummings, M.D., director emeritus of Cleveland Clinic Lou Ruvo Center for Brain Health, director of the Center for Neurodegeneration and Translational Neuroscience and research professor at the University of Nevada, Las Vegas department of Brain Health, presented findings of the “Rasagiline Rescue (R2): A Double-Blind Placebo Controlled Trial for Mild to Moderate Alzheimer’s Disease.” Rasagiline is an FDA-approved Parkinson's drug that holds promise for slowing the progression of Alzheimer's disease. Repurposing a drug is an application of an already approved drug to treat another disease/medical condition as a means of accelerating treatments. The ADDF funded the repurposing trial of rasagiline in patients with mild to moderate Alzheimer's disease, which showed positive results.
In the phase two, double-blind placebo controlled study, rasagiline met its primary target and increased metabolism in the frontal regions of the brain compared to placebo. Additionally, consistent trends toward improvement in cognitive, functional and quality of life measurements were observed in patients on active treatment. These findings indicate that significant changes can be anticipated in larger studies, and final analyses of other biomarkers will support advancing rasagiline into a larger trial that will confirm its effects on cognition. The ADDF awarded Dr. Cummings with the 2019 Melvin R. Goodes Prize for Excellence in Alzheimer's Drug Discovery for this research.
Novel biomarker approaches being used in the field were also shared, including neuroimaging (both PET and MRI), fluid biomarkers and ocular tests, as well as biomarkers for frontotemporal dementia (FTD).
In a keynote address, “Extracellular Vesicle Biomarkers Revolutionize Preclinical Diagnosis of AD and Assessment of Treatment Responses in Clinical Trials,” Dimitrios Kapogiannis, M.D., of the National Institutes of Health, discussed the emerging role of biomarkers as tools for identifying patients early in the course of Alzheimer’s disease. Like targeted approaches in other diseases, such as cancer, these biomarkers can help measure multiple signs of disease and show if a patient is responding to a drug treatment. These biomarkers could lead the way to more effective, precise treatments for Alzheimer’s disease.
In a biomarker session, Swati More, Ph.D., University of Minnesota, presented research focused on “Ocular Approaches for Alzheimer’s Diagnosis.” In collaboration with Professor Robert Vince at the Center for Drug Design, Dr. More has developed a retinal hyperspectral imaging technique that has shown promise as an early diagnostic tool for Alzheimer’s disease. This technology has been licensed to RetiSpec, a medical device company focused on Alzheimer’s detection.
This year for the first time the ADDF held a special session devoted to novel approaches for FTD. The session highlighted the needs in drug discovery and biomarkers for FTD, and emphasized approaches used in different neurodegenerative diseases.
In a presentation titled, “Non-Invasive Brain Stimulation to Restore Cortical Connectivity in FTD,” Alberto Benussi, M.D., of the University of Brescia, Italy, presented results from a clinical trial testing non-invasive brain stimulation in FTD patients, a study led by Barbara Borroni, MD, professor at the University of Brescia. This research is supported by an ADDF partnership with The Association for Frontotemporal Degeneration (AFTD) to advance the search for FTD treatments. Non-invasive brain stimulation has the potential to restore brain plasticity and improve symptoms in multiple types of dementia. There are currently no approved treatments for FTD, the most common form of dementia for individuals under age 60. The promising findings presented at the conference provide hope that a non-invasive intervention could help improve symptoms in patients with FTD.
“Thanks to the hard work of all these researchers, the field is finally shifting toward more innovative targets for treating Alzheimer’s and related dementias. Each year brings us one step closer to accomplishing our mission and maintaining our singular focus on the science that’s needed to conquer Alzheimer’s disease,” noted Dr. Fillit.