Methods for tracking the progression of Alzheimer's...

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Reexamination Certificate

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C800S003000, C800S013000, C800S018000, C428S840500, C435S325000

Reexamination Certificate

active

06374130

ABSTRACT:

FIELD OF INVENTION
This invention utilizes imaging techniques and other methods to measure the activity in a specific region of the brain in genetically modified mice to provide an indicator of the progression of Alzheimer's disease; furthermore, it utilizes this indicator of Alzheimer's disease progression to identify treatments with the potential to halt the progression or prevent the onset of this disorder.
BACKGROUND OF INVENTION
Alzheimer's disease is the most common form of memory and thinking problems (i.e., “dementia”) in older people. According to one community survey, Alzheimer's dementia affects about 10% of those over the age of 65 and almost half of those over the age of 85. As the average life span continues to increase, Alzheimer's disease is expected to take an increasing toll on affected persons, family caregivers, and the communities in which they live.
Little by little, Alzheimer's disease robs affected individuals of their memory, judgment, and reasoning, their ability to recognize objects and familiar faces, their language skills, and their ability to perform routine tasks. In the most severe stage of the illness, individuals may be bed-ridden, totally confused, unable to move around or communicate with others, and unable to control their bladder or bowel functions. Affected persons commonly die from the complications of infections, accidents, or malnutrition. Indeed, Alzheimer's disease is the fourth leading cause of death in the United States.
Alzheimer's disease takes an extraordinary toll on the affected person's family. Family caregivers commonly feel frustrated, helpless, and physically exhausted. Half of them become clinically depressed. Many must become impoverished before the affected person is eligible for long-term residential care.
The financial impact of Alzheimer's disease on the community continues to grow. Currently, it costs about $175,000 to care for each person with Alzheimer's dementia. By the time today's young adults grow old, there will be four times as many people over the age of 65, eight times as many people over the age of 85, and a smaller proportion of individuals in younger age groups who will be expected to provide care for those affected by Alzheimer's disease.
For all of these reasons, researchers must find ways to halt the progression and prevent the onset of this devastating disorder. Scientific discoveries in the last few years have raised the hope that researchers will find effective ways to treat and prevent Alzheimer's disease.
For instance, autopsy studies reveal several characteristic brain abnormalities (often called “histopathological features”) in patients with Alzheimer's disease: plaques, tangles, and a loss of brain cells. Researchers have begun to characterize the cascade of molecular events that lead to plaques (e.g., the aggregation of an unusually long form of a protein called “&bgr;-amyloid”); they have begun to characterize the molecular events that lead to the formation of tangles (e.g., the binding of phosphate molecules to a protein called “tau”); and they have begun to characterize the cascade of molecular events that lead to a loss of brain cells called “neurons” and their connections called “synapses” (e.g., inflammation, oxidation, and over-excitation of brain cells). Treatments that target one or more of these molecular events might be able to halt the progression of Alzheimer's disease or even prevent the onset of Alzheimer's disease altogether.
Furthermore, researchers have identified specific genes that appear to account for about half of all Alzheimer's disease cases. Rare mutations of the amyloid precursor protein (known as “APP”) gene on chromosome 21, the presenilin 1 gene on chromosome 14, and the presenilin 2 gene appear to account for about half of those individuals who develop a form of Alzheimer's disease that runs in families and leads to the onset of memory and thinking problems prior to the age of 60 (“early-onset”). In addition to these rare mutations, the apolipoprotein E (APOE) &egr;4 gene on chromosome 19 (found in about one-fourth of the population) increases the risk and hastens the onset of memory and thinking problems in about half of those who develop Alzheimer's dementia after the age of 60. As researchers determine how the products of these genes lead to the development of Alzheimer's disease, they will provide additional targets for treatments to halt the progression and prevent the onset of this disorder.
Finally, researchers have already identified clinical treatments that might slow the progression or delay the onset of Alzheimer's disease. Promising treatments include, but are not limited to, anti-oxidants like vitamin E, anti-inflammatory medications like indomethacin, estrogen replacement therapy, recently developed amyloid blocking agents, and a potential Alzheimer's disease vaccine. A treatment that delays the onset of Alzheimer's dementia by only 5 years has the potential to cut in half the number of new cases of this devastating disease.
Despite these promising observations, one of the greatest obstacles to the discovery of Alzheimer's disease treatments has been the absence of a marker of disease progression in laboratory animals. A marker of Alzheimer's disease progression in laboratory animals could be used to screen pharmaceutical compounds and other treatments for their ability to halt the progression and prevent the onset of this disorder. This screening procedure could identify promising treatments, give chemists direction in the refinement or new design of promising pharmaceutical compounds, and give the pharmaceutical industry direction in selecting which treatments to test in expensive and time-consuming clinical trials. Companies need to have confidence in a potential treatment before they are willing to spend the time, effort, and millions of dollars needed to establish its efficacy and safety and merit approval from the United States Food and Drug Administration (FDA).
Capitalizing on the discovery of specific genetic risk factors for Alzheimer's disease and refinements in genetic engineering, research groups have recently begun to produce strains of genetically modified mice that develop some of the characteristic brain abnormalities found at autopsy in persons with Alzheimer's dementia, such as amyloid plaques. For instance, some research groups have developed transgenic mice that over express abnormal forms of human APP found in certain families with early-onset Alzheimer's disease; some have developed transgenic mice that over express abnormal forms of the human presenilin 1 protein found in certain other families with early-onset Alzheimer's disease; some have developed transgenic mice that over-express human the APOE &egr;4 protein found in many persons with late-onset Alzheimer's disease; and some have developed transgenic mice with a combination of genetic modifications (e.g., transgenic/knockout mice that contain a mutant form of the human APP transgene and the human APOE &egr;4 transgene and lack the mouse APOE gene). Researchers continue to work on the development of genetically modified mice that could serve as a laboratory model of Alzheimer's disease.
To date, the most promising animal models of Alzheimer's disease have been transgenic mice who overexpress abnormal forms of APP and develop amyloid plaques (e.g., the PDAPP mice first described by Dora Games and her colleagues). While these mice represent an important advance in the effort to find Alzheimer's disease treatments, uncertainties remain about the validity of these and other animal models and the best way to monitor disease progression in the absence of symptoms. First, transgenic mice generally lack some of the characteristic histopathological features of Alzheimer's disease, such as tangles and neuronal loss. Second, it remains possible that amyloid plaques are insufficient to account for the clinic

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