BrightFocus-funded Alzheimer’s Test Gets Encouraging Sign from FDA
A BrightFocus-funded project to develop a blood-based screening test for Alzheimer’s disease has turned a major corner toward reaching the marketplace, with the U.S. Food and Drug Administration (FDA) giving it a “Breakthrough Device” designation which may accelerate this promising diagnostic tool’s approval for use in healthcare settings.
“A vital need exists for an improved diagnostic test to screen individuals with subjective memory concerns. The best chance we have for treating Alzheimer’s disease will come from earlier detection and earlier intervention. Improving the speed and efficiency of diagnosis can also help lower health care costs. We are grateful to BrightFocus Foundation for being such a strong supporter every step of the way,” said Joel Braunstein, MD, MBA, the CEO of C2N Diagnostics, which received a grant from BrightFocus’ Alzheimer’s Disease Research (ADR) program.
Braunstein and his C2N colleagues, including Philip Verghese, PhD, developed this potentially breakthrough technology by building upon previous BrightFocus-funded discoveries at Washington University in St. Louis (WUSTL). The BrightFocus ties to this research date back to early grants to world-renowned WUSTL researchers David Holtzman, MD, and Randall Bateman, MD, the scientific co-founders of C2N.
“BrightFocus is proud to have supported such an innovative project from the beginning. Our mission is to identify and catalyze ground-breaking science like this. Too many promising scientific ideas wither on the vine for lack of financial support, and we’re encouraged by the heightened prospects for this new blood-based biomarker diagnostic tool,” said Diane Bovenkamp, PhD, BrightFocus Vice President, Scientific Affairs.
A Blood-Based Alzheimer’s Screening: Why it is Needed
The Alzheimer’s field has long sought something like this, an easily-administered blood test that, if approved, can be used to screen for brain amyloid pathology in individuals being assessed for a possible diagnosis of Alzheimer’s disease (AD).
The ongoing challenge is that many other conditions can mimic the memory loss, mood changes, and other possible indicators of Alzheimer’s, and clinicians rely on further testing to narrow the cause. However, all the tests currently used to detect Alzheimer’s have major limitations. That includes amyloid imaging scans that use positron emission tomography (PET scans), which are costly and not available everywhere, and may also expose individuals who do not have Alzheimer’s to unnecessary radiation. And while it’s also possible to detect amyloid build-up in cerebrospinal fluid (CSF), those tests require a spinal tap, where a needle is inserted into the lower part of the back, which is more costly and uncomfortable for patients than simply drawing a blood sample.
Blood-based screening measures are envisioned as the first step in a multi-stage process to diagnose AD, improving the speed and efficiency of the overall diagnostic process, and also achieving major cost savings to healthcare systems. For example, the simple blood-based screening test being developed by C2N could be used for initial screening and, if positive, might be followed by PET and other more in-depth screening measures to gather more specific diagnostic information.
“At C2N, we are making strong progress toward these goals and are committed to introducing a test that is widely accessible,” Dr. Braunstein said.
Currently, C2N is completing a clinical trial where the results from their blood test will be compared with those from PET scans as a tool for diagnosing Alzheimer’s in adults with memory complaints or dementia. That trial, called the Plasma Test for Amyloid Risk Screening (PARIS) Study, is also supported by BrightFocus funding. The goal of the test will be to determine if persons age 65 and older with subjective memory complaints have brain beta-amyloid at levels indicating possible Alzheimer’s.
Eventually, blood screening tools like this might be used to assess amyloid build-up in individuals who are at higher than average risk of Alzheimer’s due to their health status, genetics, or having a first-degree blood relative with the disease.
Experts who treat Alzheimer’s generally agree that it’s important to understand one’s amyloid status, even in the absence of a disease-modifying treatment. Early detection can lead to better outcomes by encouraging individuals to take steps to do all that’s possible to slow down the course of disease by minimizing health risks that may contribute to AD, such as cardiovascular disease and diabetes, and making lifestyle changes to sleep, diet, and exercise.
Hope for Improving Clinical Trial Screening
Another foreseen use for the test is to screen individuals who are recruited to participate in Alzheimer’s clinical trials to test potential new drugs for the disease. Currently, up to 80 percent of such individuals fail to meet inclusion criteria. A screening test that preselects trial candidates more likely to have amyloid accumulation could vastly improve on this situation, accelerating the development of new AD drugs and lowering the cost of bringing them to market.