SBIR-STTR Award

For a long time, the study of Alzheimer’s disease (AD) in humans has been hampered by the fact that absolute AD diagnosis could only be ascertained by assessment of brain pathology upon autopsy. More recently, imaging agents have become available that allow direct imaging of the two hallmarks of AD (amyloid plaques and tau tangles) in living humans. However, these imaging agents are expensive and invasive for use in routine diagnostics. The need for low-cost blood based diagnostic assays has never been greater. C2N Diagnostics has been developing mass spectrometry based assays that investigate amyloid beta peptides in biofluids since 2007. Recent advances in sample cleanup, purification of amyloid beta, and mass spectrometry instrumentation have made it possible to reliably quantify amyloid beta in human plasma samples. Preliminary data from Dr. Randall Bateman’s laboratory at Washington University shows that the ratio of two amyloid beta peptides in plasma can predict the presence of amyloid plaques in the brain. This technology could revolutionize the field of AD diagnostics, and when presented at the Alzheimer’s Association International Conference in July 2017, this discovery was pronounced as “by far the biggest news of the meeting” by one of the key opinion leaders in AD clinical research. The specific aims of this SBIR Fast-Track application are to analytically and clinically validate C2N’s plasma assay for use as a diagnostic test for predicting brain amyloidosis.

Public Health Relevance Statement:
NARRATIVE C2N Diagnostics seeks to commercialize a blood test for detection of Alzheimer’s disease brain pathology. Amyloid plaques accumulate in the brain early in the progression of Alzheimer’s disease, and C2N has preliminary data showing that measurement of proteins in blood can predict the presence of these plaques in the brain.

Project Terms:
Alzheimer disease detection; Alzheimer's Disease; Amyloid; Amyloid beta-Protein; Amyloidosis; Autopsy; base; Binding; Biological Assay; Blood; Blood specimen; Blood Tests; Brain; Brain Pathology; cerebral amyloidosis; Clinical; Clinical Research; Clinical Trials; clinically relevant; commercial application; Communities; community setting; Conduct Clinical Trials; cost; Data; Dementia; design; Development; Diagnosis; Diagnostic; diagnostic accuracy; diagnostic assay; Diagnostic tests; Disease; disease diagnosis; Eligibility Determination; Enrollment; Evaluation; experience; FDA approved; Goals; Human; Image; imaging agent; Impaired cognition; In Vitro; instrumentation; interest; International; Laboratories; Leadership; Marketing; Mass Spectrum Analysis; Measurement; Measures; meetings; Methods; Neurofibrillary Tangles; Neurologic; news; Participant; patient screening; Patients; Performance; performance tests; Persons; Pharmaceutical Preparations; Phase; Plasma; Positron-Emission Tomography; predictive test; Preparation; Privatization; Process; prospective; Protein Isoforms; Proteins; Radiation exposure; Radioactive; Reporting; Research; Resource Allocation; Risk; Running; Sampling; scale up; screening; Screening procedure; Senile Plaques; Small Business Innovation Research Grant; symposium; tau Proteins; Technology; Technology Transfer; Testing; Time Study; United States; United States Centers for Medicare and Medicaid Services; Universities; Validation; Washington

For a long time, the study of Alzheimer’s disease (AD) in humans has been hampered by the fact that absolute AD diagnosis could only be ascertained by assessment of brain pathology upon autopsy. More recently, imaging agents have become available that allow direct imaging of the two hallmarks of AD (amyloid plaques and tau tangles) in living humans. However, these imaging agents are expensive and invasive for use in routine diagnostics. The need for low-cost blood based diagnostic assays has never been greater. C2N Diagnostics has been developing mass spectrometry based assays that investigate amyloid beta peptides in biofluids since 2007. Recent advances in sample cleanup, purification of amyloid beta, and mass spectrometry instrumentation have made it possible to reliably quantify amyloid beta in human plasma samples. Preliminary data from Dr. Randall Bateman’s laboratory at Washington University shows that the ratio of two amyloid beta peptides in plasma can predict the presence of amyloid plaques in the brain. This technology could revolutionize the field of AD diagnostics, and when presented at the Alzheimer’s Association International Conference in July 2017, this discovery was pronounced as “by far the biggest news of the meeting” by one of the key opinion leaders in AD clinical research. The specific aims of this SBIR Fast-Track application are to analytically and clinically validate C2N’s plasma assay for use as a diagnostic test for predicting brain amyloidosis.

Public Health Relevance Statement:
NARRATIVE C2N Diagnostics seeks to commercialize a blood test for detection of Alzheimer’s disease brain pathology. Amyloid plaques accumulate in the brain early in the progression of Alzheimer’s disease, and C2N has preliminary data showing that measurement of proteins in blood can predict the presence of these plaques in the brain.

Project Terms:
Alzheimer disease detection; Alzheimer's Disease; Amyloid; Amyloid beta-Protein; Amyloidosis; Autopsy; base; Binding; Biological Assay; Blood; Blood specimen; Blood Tests; Brain; Brain Pathology; cerebral amyloidosis; Clinical; Clinical Research; Clinical Trials; clinically relevant; commercial application; Communities; community setting; Conduct Clinical Trials; cost; Data; Dementia; design; Development; Diagnosis; Diagnostic; diagnostic accuracy; diagnostic assay; Diagnostic tests; Disease; disease diagnosis; Eligibility Determination; Enrollment; Evaluation; experience; FDA approved; Goals; Human; Image; imaging agent; Impaired cognition; In Vitro; instrumentation; interest; International; Laboratories; Leadership; Marketing; Mass Spectrum Analysis; Measurement; Measures; meetings; Methods; Neurofibrillary Tangles; Neurologic; news; Participant; patient screening; Patients; Performance; performance tests; Persons; Pharmaceutical Preparations; Phase; Plasma; Positron-Emission Tomography; predictive test; Preparation; Privatization; Process; prospective; Protein Isoforms; Proteins; Radiation exposure; Radioactive; Reporting; Research; Resource Allocation; Risk; Running; Sampling; scale up; screening; Screening procedure; Senile Plaques; Small Business Innovation Research Grant; symposium; tau Proteins; Technology; Technology Transfer; Testing; Time Study; United States; United States Centers for Medicare and Medicaid Services; Universities; Validation; Washington