Abstract
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.
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