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In his 1973 book ‘Small Is Beautiful’, the economist EF Schumacher argued that technological development and economic growth should be centred on the needs of individuals and communities, and that bigger (unregulated expansion) was not necessarily better.1 We can make a similar case regarding highly technical interventions to manage people with chronic and progressive neurological disorders, including degenerative ataxia. Spectacular advances in genomics appear to offer incredible opportunities to influence the natural histories of ‘untreatable’ conditions, using technologies such as gene silencing/editing and the therapeutic use of agents such as antisense oligonucleotides. However, these treatments, even if proven effective, will be affordable for only a small proportion of patients worldwide. By focusing on these potentially dramatic treatments, patients (and their families) might be left feeling that only technically advanced and expensive interventions will be beneficial, with consequent therapeutic nihilism. Can we do more for our patients with what we already have?
For patients with ataxia (and probably for all neurodegenerative conditions), there are …
Footnotes
Contributors I am the sole author. I was invited to write this piece by the editors of Practical Neurology, following the peer review of a paper submitted to the journal. The concepts explored here have been developed and moulded by conversations with other neurologists, physicians and occupational therapists.
Funding The author has not declared a specific grant for this research from any funding agency in the public, commercial or not-for-profit sectors.
Competing interests None declared.
Provenance and peer review Commissioned. Internally peer reviewed.
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