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Clinical Implications of Basic Research
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Volume 352:1376-1378 March 31, 2005 Number 13
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Amyotrophic Lateral Sclerosis — A New Role for Old Drugs
Robert H. Brown, Jr., D.Phil., M.D.

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Over the past decade, there have been extraordinary advances in understanding the molecular physiology of the brain and its disorders. Unfortunately, few if any of these insights have been translated into meaningful therapies. This failure undoubtedly reflects the general complexity of the central nervous system and the "orphan" status of many of the disorders, including most of the neurodegenerative diseases. Because the potential market value of drugs for these orphan diseases is small, they are usually viewed as inappropriate targets for drug development by pharmaceutical companies.

In this context, a recent study by Rothstein and colleagues1 is notable. This team . . . [Full Text of this Article]


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From Massachusetts General Hospital, Boston.



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