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  1. Matthew C Kiernan
  1. Correspondence to Professor Matthew C Kiernan, Neuroscience Research Australia and University of New South Wales, Barker Street, Randwick, Sydney, NSW 2031, Australia; M.kiernan{at}unsw.edu.au

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In the Journal of Medical Ethics, there is fascinating and fraught debate about the development and release of potential treatments for rapidly progressive, universally fatal, neurodegenerative diseases. The discussion focuses on the often antithetical views of a patient diagnosed with amyotrophic lateral sclerosis (ALS), his treating physicians, research neuroscientists and a medical ethicist.1 The debate centres on how best to promote drug discovery and the future release of potential treatments for patients struck down by this fatal condition, typically in the prime of their adult lives (figure 1).2 ,3 Despite acknowledgement of the various challenges regarding drug discovery, unfortunately no clear consensus about the best way forward appears to have been reached.

Figure 1

Whither the clinical trial in amyotrophic lateral sclerosis? With typical disease onset during the prime of life, heralded by progressive muscle wasting and weakness (illustrated here involving intrinsic hand muscles), patients may question the traditional trial approach, preferring to seek novel, often untested, remedies.

As noted by the patient with ALS, Les Halpin, the first author of the manuscript, sophisticated clinical trials have been conducted in ALS at least since the 1980s, yet riluzole remains the only moderately efficacious drug to have emerged as treatment for the disorder. There may be many reasons for trial failures,4 but one consequence has clearly been that pharmaceutical companies are now reluctant to invest in rare diseases such as ALS. In an attempt to reverse this trend, Halpin …

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