Research
- Can we develop a new ‘cholesterol test’ for the brain?
Certain proteins found in the brain, notably amyloid and tau (among others), that are associated with Alzheimer’s disease (AD) and other neurodegenerative diseases (ND) can now be detected in the blood many years before the onset of symptoms. A study funded by donors to our Foundation is testing whether it is possible to collect these proteins from people with varying degrees of risk for AD and other diseases, including Parkinson’s disease (PD), Lewy Body Dementia (LBD) and Frontotemporal Dementia (FTD). The ability to monitor and track these protein markers in people at-risk before symptoms begin may be critical for early detection and treatment.
Just like when doctors suggest ways for people to lower their cholesterol (e.g., exercise, dietary changes, prescription drugs, or supplements) and then use cholesterol blood test values (like LDL, HDL and triglycerides) to assess the effectiveness of those interventions, our team is developing a "cholesterol test for the brain". However, instead of looking at LDL, HDL and other cholesterol markers, we use novel brain proteins like amyloid, tau, neurofilament light chain and more than 120 other new tests that we can now run in our lab in Boca Raton, FL, and also at partner labs around the world. We can use these new tests to measure the effect of the risk reduction interventions that we suggest to people with a family history of AD, PD, LBD and FTD, among other disorders, and can also evaluate the effectiveness of treatments used for people at the earliest symptomatic stages of neurodegenerative disease. Based on some of the test results, we can then refine, or "fine-tune" our personalized treatment plan based on response (or lack thereof).
Our team has used “N-of-1” trials (or single participant clinical studies) that consider an individual person when evaluating the effectiveness of different interventions on ND risk. Using the person as the sole unit of observation, the impact of various interventions, such as lifestyle changes (e.g., exercise, nutrition, stress reduction) as well as certain drugs, vitamins or supplements used for other medical conditions (e.g., diabetes, high cholesterol, peri-menopause, depression, high blood pressure), can be measured based on the change in blood-based markers of ND. Using biomarkers both before and after various lifestyle and medical changes will allow our research team to cost-effectively understand the biological impact of these interventions on AD and other ND pathology (rather than by using tests that are riskier, more cumbersome, and costlier, such as lumbar punctures and brain imaging scans).
It is our goal to enroll over 150 people in our study aged 21+ with a family history of, or with already diagnosed, AD, PD, LBD, FTD and other ND, as well as control subjects with no or minimal cognitive and/or motor complaints, and will follow these subjects over time. For example, we are able to further study the effects of anti-amyloid drugs prescribed for patients with mild cognitive impairment due to Alzheimer's disease, cholesterol treatments for people at risk or for those who are currently symptomatic, hormone replacement therapy for women, as well as multi-modal lifestyle interventions to reduce risk such as exercise and dietary changes. Enrollment in this study is currently closed but we are actively looking for more funding to expand this important work. We have presented initial findings on 75 participants at the 2024 American Academy of Neurology Meeting in Denver, CO, and the 2024 Alzheimer's Association International Conference in Philadelphia, PA. Stay tuned for more results and join our email Newsletter to stay in touch. Also, read our recent Blog Posts below or visit our Newsroom Page to learn more.
- Precision Medicine & Genetics Research Program
A Foundation-supported research project focuses on the genetic factors that play a role in the development of AD and other ND. There are many genes – ranging from those that affect memory to those that affect cholesterol to sleep – that can impact the development of ND. It is our goal to be better able to acquire, store, and analyze whole genome sequencing, as well as partial genome sequencing, in people at risk for or already diagnosed with these conditions. We have enrolled several families and aim to study how genetic analyses may contribute to personalizing risk reduction care in a clinical research study.
