Publications by authors named "James A Heywood"
Design, power, and interpretation of studies in the standard murine model of ALS.
Amyotroph Lateral Scler
April 2008
Article Synopsis
- Identification of SOD1 as the mutated protein in familial amyotrophic lateral sclerosis (FALS) has led to the creation of mouse models, particularly the SOD1(G93A) mouse, that are widely used for testing ALS therapies.
- Despite over 50 studies reporting lifespan extension with various therapeutic agents in these mice, only riluzole has shown clinical effectiveness in humans.
- The researchers identified key biological variables that affect study results and found that when these were controlled, several previously reported drugs, including riluzole, failed to demonstrate any significant benefits, leading to recommendations for improved study design in future research.
As new lead discovery technologies of high throughput screening and rational drug design have been incorporated into pharmaceutical and biotechnology drug discovery programs, researchers have focused on the applying these new technologies in diseases traditionally neglected by for-profit drug discovery efforts. This article reviews general trends in orphan disease lead discovery, identifies best practices of orphan market drug discovery and provides an overview of recent ALS lead discovery programs and drug development according to these metrics. Best practices in orphan market drug discovery embodied by programs like the NIH Anticonvulsant Screening Program include the (1) management of timelines and priorities, (2) engagement of for-profit partners, (3) creative application of technology, (4) collaboration, and (5) flexibility.
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