11~帕金森病(Parkinson’s Disease).ppt
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Parkinson's Disease Research Agenda
National Institutes of Health
National Institute of Neurological Disorders and stroke
? A new optimism that Parkinson's disease can be defeated is energizing the research community and patient advocates. Halting the progression of Parkinson's disease, restoring lost function, and even preventing the disease are all realistic goals. This hope is fueled by the accelerating pace of discovery in neuroscience research generally, by advances in understanding what causes Parkinson's disease, and by a wide range of new treatments on the horizon, including stem cell transplants, precision surgical repair, chronic brain stimulation, and natural growth factors, to name a few. Optimism is tempered by the recognition that we cannot yet cure any major neurodegenerative disorder, and defeating Parkinson's disease requires crossing a major frontier of medicine.
? The National Institute of Health conducts a vigorous and expanding program of research focused on Parkinson's disease. At a landmark meeting in November 1999,the directors and staff of the major components of NIH conducting Parkinson's disease research, working together with patient advocates, initiated a planning process to ensure that extraordinary opportunities to move toward a cure are not neglected and that critical obstacles to progress are addressed. On January 4-6,2000 a Workshop including intramural, extramural, and industry scientists and representatives from several Parkinson's advocacy groups discussed an agenda for Parkinson's disease research which formed the basis for this document.
What is Parkinson's Disease
? 1817, James Parkinson first observed this disease and called it The Shaking Palsy. His seminal work was based on his clinical observation of six cases. The cardinal symptoms include tremor at rest, bodily rigidity, marked slowness of move- ment, postural changes, gait disturbances, and difficulty initiating voluntary movement. How- ever, it can cause a wide spectrum of other symptoms, including dementia, abnormal speech, sleep disturbances, swallowing problems, sexual dysfunction, and depression.
UNDERSTANDING PARKINSON'S DISEASE
? Parkinson's disease is a devastating and complex disease that progressively affects the control of movement and also produces a wide range of other problems for patients. The symptoms reflect the gradual loss of nerve cells in particular areas of the brain. Among these, cells that produce the neurotransmitter dopamine die in a small brain area called the substantia nigra. What triggers the death of these nerve cells is unknown.
? Using genetics to understand Parkinson's disease: Although most people do not inherit Parkinson's disease, studying the genes responsible for the inherited cases is advancing our understanding of both common and familial Parkinson's disease. Identifying genes that can cause Parkinson's disease is crucial for understanding the disease process, revealing drug targets, improving early diagnosis, and developing animal models that accurately mimic the slow nerve cell death in human Parkinson's disease. Beyond single genes, we must unravel the complex interactions between genetic predisposition and environmental influences that cause most cases of Parkinson's disease.
? Epidemiology to determine entvironmental risk factors for parkinson's disease: Epidemiological investigations can provide essential clues to what causes Parkinson's disease, to risk factors that predispose people to this desease, and to preclinical characteristics of this disorder. In the short term, case control studies that compare people with and without Parkinson's disease can provide valuable information about environmental risk factors and the interaction of genetic and non-genetic factors. In the long run, a prospective study, which follows people who do not yet have the disease, will help identify the causes of Parkinson's disease and provide other needed epidemiological information. It would be highly efficient in such a study to include other disorders.
? Life and death of neurons involved in Parkinson's disease: Parkinson's disease selectively kills only certain types of brain cells. Understanding the normal biology of neurons susceptible to Parkinson's disease is crucial for understanding this selectivity and for developing new therapies that rescue of even replace those cells. Studying how inherited defects in genes for proteins such as synuclein and parkin can cause Parkinson's disease are important inroads to understanding the disease. Other important areas for research include the role of mitochondrial impairment,protein aggregation, excitotoxicity, the immune system and apoptosis pathways in Parkinson's disease.
? Neural circuits and systems in Parkinson's disease: While there has been considerable progress in understanding how the normal brain controls movement, there is a great deal we do not yet understand about the brain's movement control stystems. Moreover, we do not understand how Parkinson's disease disrupts these systems to produce the major symptoms and other problems associated with this disease. A variety of studies using anatomical, electrophysiological, neurochemical, and imaging methods are needed.
DEVELOPING NEW TREATMENTS FOR PARKINSON'S DISEASE
? Developing therapies to prevent Parkinson's disease, to suppress symptoms, to halt disease progression, and to repair damage are all fundamental goals. Available drugs suppress symptoms early in Parkinson's disease, but progressively fail as more nerve cells die. A wide range of therapeutic approaches are now at various stages of development, including precision surgical ablation, chronic electrical stimulation, cell implantation, and several types of drugs. To achieve therapeutic goals, many separate studies are required, from the first steps in translating basic research advances, animal testing, preliminary safety studies in human patients, and finally large trials to evaluate the effectiveness of a therapy.......（后略) ......
Parkinson's Disease Research Agenda
National Institutes of Health
National Institute of Neurological Disorders and stroke
? A new optimism that Parkinson's disease can be defeated is energizing the research community and patient advocates. Halting the progression of Parkinson's disease, restoring lost function, and even preventing the disease are all realistic goals. This hope is fueled by the accelerating pace of discovery in neuroscience research generally, by advances in understanding what causes Parkinson's disease, and by a wide range of new treatments on the horizon, including stem cell transplants, precision surgical repair, chronic brain stimulation, and natural growth factors, to name a few. Optimism is tempered by the recognition that we cannot yet cure any major neurodegenerative disorder, and defeating Parkinson's disease requires crossing a major frontier of medicine.
? The National Institute of Health conducts a vigorous and expanding program of research focused on Parkinson's disease. At a landmark meeting in November 1999,the directors and staff of the major components of NIH conducting Parkinson's disease research, working together with patient advocates, initiated a planning process to ensure that extraordinary opportunities to move toward a cure are not neglected and that critical obstacles to progress are addressed. On January 4-6,2000 a Workshop including intramural, extramural, and industry scientists and representatives from several Parkinson's advocacy groups discussed an agenda for Parkinson's disease research which formed the basis for this document.
What is Parkinson's Disease
? 1817, James Parkinson first observed this disease and called it The Shaking Palsy. His seminal work was based on his clinical observation of six cases. The cardinal symptoms include tremor at rest, bodily rigidity, marked slowness of move- ment, postural changes, gait disturbances, and difficulty initiating voluntary movement. How- ever, it can cause a wide spectrum of other symptoms, including dementia, abnormal speech, sleep disturbances, swallowing problems, sexual dysfunction, and depression.
UNDERSTANDING PARKINSON'S DISEASE
? Parkinson's disease is a devastating and complex disease that progressively affects the control of movement and also produces a wide range of other problems for patients. The symptoms reflect the gradual loss of nerve cells in particular areas of the brain. Among these, cells that produce the neurotransmitter dopamine die in a small brain area called the substantia nigra. What triggers the death of these nerve cells is unknown.
? Using genetics to understand Parkinson's disease: Although most people do not inherit Parkinson's disease, studying the genes responsible for the inherited cases is advancing our understanding of both common and familial Parkinson's disease. Identifying genes that can cause Parkinson's disease is crucial for understanding the disease process, revealing drug targets, improving early diagnosis, and developing animal models that accurately mimic the slow nerve cell death in human Parkinson's disease. Beyond single genes, we must unravel the complex interactions between genetic predisposition and environmental influences that cause most cases of Parkinson's disease.
? Epidemiology to determine entvironmental risk factors for parkinson's disease: Epidemiological investigations can provide essential clues to what causes Parkinson's disease, to risk factors that predispose people to this desease, and to preclinical characteristics of this disorder. In the short term, case control studies that compare people with and without Parkinson's disease can provide valuable information about environmental risk factors and the interaction of genetic and non-genetic factors. In the long run, a prospective study, which follows people who do not yet have the disease, will help identify the causes of Parkinson's disease and provide other needed epidemiological information. It would be highly efficient in such a study to include other disorders.
? Life and death of neurons involved in Parkinson's disease: Parkinson's disease selectively kills only certain types of brain cells. Understanding the normal biology of neurons susceptible to Parkinson's disease is crucial for understanding this selectivity and for developing new therapies that rescue of even replace those cells. Studying how inherited defects in genes for proteins such as synuclein and parkin can cause Parkinson's disease are important inroads to understanding the disease. Other important areas for research include the role of mitochondrial impairment,protein aggregation, excitotoxicity, the immune system and apoptosis pathways in Parkinson's disease.
? Neural circuits and systems in Parkinson's disease: While there has been considerable progress in understanding how the normal brain controls movement, there is a great deal we do not yet understand about the brain's movement control stystems. Moreover, we do not understand how Parkinson's disease disrupts these systems to produce the major symptoms and other problems associated with this disease. A variety of studies using anatomical, electrophysiological, neurochemical, and imaging methods are needed.
DEVELOPING NEW TREATMENTS FOR PARKINSON'S DISEASE
? Developing therapies to prevent Parkinson's disease, to suppress symptoms, to halt disease progression, and to repair damage are all fundamental goals. Available drugs suppress symptoms early in Parkinson's disease, but progressively fail as more nerve cells die. A wide range of therapeutic approaches are now at various stages of development, including precision surgical ablation, chronic electrical stimulation, cell implantation, and several types of drugs. To achieve therapeutic goals, many separate studies are required, from the first steps in translating basic research advances, animal testing, preliminary safety studies in human patients, and finally large trials to evaluate the effectiveness of a therapy.......（后略) ......
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