NIA Small Business funding seeks to find blood-based diagnostic for Alzheimer’s disease
A test for early detection of Parkinson’s disease, which recently received Breakthrough Device Designation by the FDA, may have the potential to become an early measure for Alzheimer’s disease as well. With NIH funding that includes Small Business grants from NIA, Amprion, a California-based biotech company, has developed a technology to detect very small amounts of proteins in the cerebrospinal fluid and bloodstream that could be used to detect the diseases.
Having an effective, accurate, and easy-to-administer test for diagnosing Alzheimer’s and Parkinson’s diseases is crucial to help develop treatments and potentially find cures for these diseases. Currently, the use of biomarkers for Alzheimer’s disease in a clinical setting, such as a doctor’s office, is limited.
The FDA’s Breakthrough Devices Program is designed to speed up development, assessment, and review of medical devices that could provide more effective treatment or diagnosis of life-threatening or irreversibly debilitating diseases or conditions. The FDA designation puts Amprion’s Protein Misfolding Cyclic Amplification (PMCA) system a step closer to approval for early detection of the alpha-synuclein protein, which would help diagnose Parkinson’s disease. Amprion anticipates market rollout within 18 months. Ongoing efforts funded by NIA grants are dedicated to similarly detecting proteins associated with Alzheimer’s disease.
The ongoing NIA funding is specific to detecting traces of amyloid and tau aggregates. Amprion hypothesizes that detection of misfolded amyloid and tau oligomers circulating in cerebrospinal fluid and blood may be the basis for an early Alzheimer’s biomarker. With the funding, the company plans to adapt the PMCA technology for specific and highly sensitive detection in human cerebrospinal fluid and blood plasma, perform studies of specificity and sensitivity using a large number of samples from people with Alzheimer’s, and evaluate the utility of PMCA for monitoring Alzheimer’s disease progression.
The PMCA technology has been under development, with the support of several NIH grants from NIA, the National Institute of Neurological Disorders and Stroke, and the National Institute of Allergy and Infectious Diseases, for the past 15 years. PMCA, also known as RT-QuIC, is a platform technology to detect misfolded protein oligomers implicated in various neurodegenerative diseases, including Alzheimer's, Parkinson's, and prion diseases. The technique harnesses the way proteins misfold, aggregate, and spread in the brain and then amplifies their quantity so small amounts can be found in biological fluids. The PMCA/RT-QuIC assay to detect misfolded prion protein is already being used to diagnose prion diseases. Amprion was founded, with funding from several Small Business Innovation Research (SBIR)/Small Business Technology Transfer (STTR) grants, to facilitate the commercialization of these assays.
This research is funded in part by NIA grants R42AG049562 and R42AG058333.
These activities relate to NIA’s AD+ADRD Research Implementation Milestone 9.F, "Initiate studies to develop minimally invasive biomarkers for detection of cerebral amyloidosis, AD, and AD-related dementias pathophysiology" and Milestone 9.G, "Initiate studies to link peripheral blood-based molecular signatures and central imaging and CSF biomarkers."