148 pages

Date published 
December 2008

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Neurodegenerative Diseases: Next-Generation Drugs for Four Major Disorders Overview


CoverNeurodegenerative Diseases: Next-Generation Drugs for Four Major Disorders

Author: Mark C. Via




Neurodegenerative diseases are drawing immense interest from the pharmaceutical industry and have inspired heavy competition in the race to introduce the next generation of improved drugs. Alzheimer’s disease, Parkinson’s disease, Huntington’s disease, and amyotrophic lateral sclerosis are analyzed in this report, which:

  • Reviews their symptoms and pathology, presumed causes, methods of diagnosis, epidemiology
  • Examines existing drug therapies for each disorder
  • Surveys the R&D picture for each disease
  • Tabulates the approximately 150 compounds in clinical development
  • Discusses particularly noteworthy drug candidates.

Neurodegenerative diseases are caused by the loss or dysfunction of neurons in the brain or spinal cord. These diseases are especially devastating because the affected cells typically cannot regenerate following damage or death. Neurodegenerative Diseases: Next-Generation Drugs for Four Major Disorders deals with chronic neurodegenerative diseases by focusing on four of the most comprehensively studied such conditions: Alzheimer’s disease (AD), Parkinson’s disease (PD), Huntington’s disease (HD), and amyotrophic lateral sclerosis (ALS).


To the pharmaceutical industry, perhaps the most important defining characteristic of these four diseases is the inadequacy of the standard of care. Existing treatments tend to address symptoms as opposed to modify disease course. Several relatively new drugs are available for AD, but they have only modest effects. There is a larger formulary on hand for PD, but treatments are plagued by the issues of side effects and diminishing returns. The landscape is even bleaker for HD and ALS, with only a single, moderately effective drug for each of these conditions. We examine the existing drug therapies for each of these diseases, grouping and discussing treatments according to their mechanism of action.

AD and PD present huge potential markets, with 5 million and 1 million US patients, respectively. HD and ALS are uncommon in comparison, with only about 30,000 US patients apiece, but all four diseases disproportionately affect the elderly, who comprise a steadily increasing share of the population in the developed world. Without an outright cure, most therapies would likely require long-term administration. These factors suggest that a company which can deliver an improved compound for one of these neurodegenerative disorders will earn a rich return on its investment.

Developing effective drugs for these diseases continues to be challenging. AD research has been stung by the recent setbacks of several novel compounds in Phase III trials. PD is an active field, but the greatest progress has been with next-generation versions of drugs that are already available. In HD and ALS, the late-stage clinical candidates are most often non-specific drugs that were originally developed for other indications.

Neurodegenerative Diseases: Next-Generation Drugs for Four Major Disorders surveys the investigational drugs in the pipeline for AD, PD, HD, and ALS. We list clinical-stage compounds, providing details about noteworthy candidates, and examine additional preclinical and research programs. The efforts of more than 115 companies working to develop treatments for these disorders are described.

About the Author
Mark C. Via, an editor at CTB International Publishing, has more than 14 years of experience writing and editing for pharmaceutical trade publications. He holds a BA in history from Williams College. Mr. Via has authored previous Cambridge Healthtech reports, including Monoclonal Antibodies: Pipeline Analysis and Competitive Assessment (www.insightpharmareports.com/reports/2007/88_Monoclonal_Antibodies/overview.asp).