ALS should not be considered a neuromuscular disorder
Capsule: ALS is now recognized to have clinical, histopathological and genetic overlap with FTD. Brain-based pathology is consistently identifiable, yet ALS frequently continues to be classified alongside neuromuscular disorders of the peripheral nerve, rather than the cerebral neurodegenerative disorders, which may have a detrimental impact on research funding and restrict optimal collaboration.
08:30-08:40
Host: Giancarlo Logroscino, Italy
08:40-08:55
Yes: Michael van Es, The Netherlands
08:55-09:10
No: Monica Povedano Panades, Spain
09:10-09:20
Discussion and rebuttals
09:20-10:10
Solving the etiology of Western Pacific ALS-PDC may illuminate understanding of the causes of ALS, atypical parkinsonism and related disorders elsewhere. Do we know the environmental cause of ALS-PDC?
Capsule: Western Pacific amyotrophic lateral sclerosis and parkinsonism-dementia complex (ALS-PDC) is a familial and sporadic neurodegenerative disease featured neuropathologically by a tau-dominated polyproteinopathy. Over the past half-century in the hyperendemic communities of Guam, USA and Kii Peninsula, Japan, disease incidence has progressively declined as age of clinical onset has increased. Japanese investigators view ALS-PDC primarily as a genetic disease, with manifestation of clinical phenotypes modified by other genes or environmental factors.. Similarly, on Guam, it is suggested that family histories with multi-incident pedigrees (ALS, atypical parkinsonism, dementia and PDC) are compatible with genetic transmission of each syndrome, while shifting environmental factors cause disease postponement. Others propose that environmental factors dominate the aetiology of ALS-PDC, the chemical identity of which is relevant to related neurodegenerative disorders (e.g., ALS, progressive supranuclear palsy.
09:20-09:30
Host: Albert Ludolph, Germany
09:30-09:45
Yes: Peter Spencer, USA
09:45-10:00
No:
10:00-10:10
Discussion and rebuttals
10:10-10:25
Coffee Break
10:25-12:05
SECTION TITLE
10:25-11:15
Patients should set the agenda for therapeutic trials in ALS
Capsule: Research being carried out ‘with’ or ‘by’ members of the public rather than ‘to’, ‘about’ or ‘for’ them, underpins an increasing drive of many grant-awarding bodies for applicants to demonstrate public and patient involvement (PPI). ALS patients are understandably desperate for more effective therapy and frequently want to “try anything”. Placebo-controlled trials may be less ethical in rapidly-progressive diseases. ‘Right-to-try’ legislation challenges the traditional model of physician-as-expert, while unfiltered information disseminated through social media by ‘expert patients’ and self-appointed advocacy groups may equally adversely distort the research agenda.
10:25-10:35
Host: Albert Ludolph, Germany
10:35-10:50
Yes: Paul Wicks, USA
10:50-11:05
No:
11:05-11:15
Discussion and rebuttals
11:15-12:05
The study of mice has been detrimental to developing therapy for ALS
Capsule: ALS is a highly-selective neurodegeneration involving motor and extra-motor neuronal networks possibly unique to humans. Twenty-five years since the development of the SOD1 mouse model of ALS, there are currently two only modestly disease-modifying therapies for the human disorder. Transgenic over-expression models may not capture the cellular ageing processes in human neurodegenerative disorders, but rodents offer systems-level readout not currently possible using iPSC-derived models.
