Accelerated evaluation of treatments to prevent clinical onset of neurodegenerative diseases

A method for determining the efficacy of a prospective prevention therapy for a neurodegenerative disease in persons having no clinical symptoms of the neurodegenerative disease, the method comprising:

• receiving at a computing device subject data concerning each of a plurality of subjects in a neurodegenerative disease prevention therapy study, the subject data including an age range and a level of risk of developing the neurodegenerative disease, the subjects each having no clinical symptoms of the neurodegenerative disease and at least one of Down syndrome or a concentration of amyloid plaques or neurofibrillary tangles deposited in a brain of each subject, wherein the concentration is at least equal to a threshold concentration of amyloid plaques or neurofibrillary tangles deposited in the brain, and the subjects being divided based on the subject data into a first group of subjects treated with the prospective prevention therapy for the neurodegenerative disease and a second group of subjects not treated with the prospective prevention therapy for the neurodegenerative disease;
• storing the subject data concerning each of the subjects in memory of the computing device;
• receiving at the computing device a plurality of brain imaging measurements from one or more positron emission tomography (PET) scanners, the brain imaging measurements indicating the concentration of amyloid plaques or neurofibrillary tangles deposited in the brain of each subject, wherein the concentration of amyloid plaques or neurofibrillary tangles deposited in the brain is a surrogate marker found in the absence of treatment to be correlated with clinical severity of symptoms of the neurodegenerative disease;
• and
• executing instructions stored in the memory of the computing device, wherein execution of the instructions by a processor of the computing device:
• determines a correlation between the brain imaging measurements and a plurality of coordinates of a brain atlas,
• extracts from the brain imaging measurements data corresponding to a region of interest identified by at least a portion of the brain atlas coordinates, the data indicating the concentration of amyloid plaques or neurofibrillary tangles deposited in the region of interest,
• calculates based on a predetermined interval a rate of change in the concentration of amyloid plaques or neurofibrillary tangles deposited in the region of interest for each of the subjects in the treated group during or following treatment with the prospective prevention therapy for the neurodegenerative disease and for each of the subjects in the untreated group,
• calculates a difference between the rates of change in the concentrations of amyloid plaques or neurofibrillary tangles deposited in the region of interest calculated for the treated group and the rates of change calculated for the untreated group,
• compares the calculated difference to a predetermined threshold of statistical significance, and
• reports to a user of the computing device by way of a user interface that the prospective prevention therapy for the neurodegenerative disease is efficacious when the calculated difference between the rates of change in the concentration of amyloid plaques or neurofibrillary tangles deposited in the region of interest for the treated and untreated groups meets or exceeds the predetermined threshold of statistical significance.

A system for determining the efficacy of a prospective prevention therapy for a neurodegenerative disease in persons having no clinical symptoms of the neurodegenerative disease, the system comprising:

• at least one positron emission tomography (PET) scanner configured to take a plurality of brain imaging measurements from each of a plurality of subjects in a neurodegenerative disease prevention therapy study, each subject having no clinical symptoms of the neurodegenerative disease and at least one of Down syndrome or a concentration of amyloid plaques or neurofibrillary tangles deposited in a brain of each subject, wherein the concentration is at least equal to a threshold concentration of amyloid plaques or neurofibrillary tangles deposited in the brain, and wherein the brain imaging measurements indicate the concentration of amyloid plaques or neurofibrillary tangles deposited in the brain of each subject and the concentration of amyloid plaques or neurofibrillary tangles deposited in the brain is a surrogate marker found in the absence of the prevention therapy for the neurodegenerative disease to be correlated with a clinical severity of symptoms of the neurodegenerative disease;
• and
• a computing device having a processor and memory storing executable instructions, wherein the computing device is communicatively coupled to the PET scanner by a communications network and is configured to:
• receive the brain imaging measurements from the PET scanner,
• receive subject data concerning each of the subjects, the subject data including an age range and a level of risk of developing the neurodegenerative disease, the subjects divided based on the subject data into a first group of subjects treated with the prospective prevention therapy for the neurodegenerative disease and a second group of subjects not treated with the prospective prevention therapy for the neurodegenerative disease,
• store the subject data concerning each of the subjects in the memory of the computing device, and
• execute the instructions stored in the memory of the computing device, wherein execution of the instructions by the processor of the computing device:
• determines a correlation between the brain imaging measurements and a plurality of coordinates of a brain atlas,
• extracts from the brain imaging measurements data corresponding to a region of interest identified by at least a portion of the brain atlas coordinates, the data indicating the concentration of amyloid plaques or neurofibrillary tangles deposited in the region of interest,
• calculates based on a predetermined interval a rate of change in the concentration of amyloid plaques or neurofibrillary tangles deposited in the region of interest for each of the subjects in the treated group during or following treatment with the prospective prevention therapy for the neurodegenerative disease and for each of the subjects in the untreated group,
• calculates a difference between the rates of change in the concentration of amyloid plaques or neurofibrillary tangles deposited in the region of interest calculated for the treated and untreated groups,
• compares the calculated difference to a predetermined threshold of statistical significance, and
• reports to a user of the computing device by way of a user interface that the prospective prevention therapy for the neurodegenerative disease is efficacious when the calculated difference between the rates of change in the concentration of amyloid plaques or neurofibrillary tangles deposited in the region of interest for the treated and untreated groups meets or exceeds the predetermined threshold of statistical significance.