MND Australia Research
@mndaus-research.bsky.social
Motor Neurone Disease Australia is the peak body for the MND community in Australia. We have built & sustained Australian MND research for 40 yrs and partner with other Australian and international MND/ALS organisations.
pTDP-43 levels correlate with cell type–specific molecular alterations in the prefrontal cortex of C9orf72 ALS/FTD patients | PNAS pnas.org/doi/10.1073/...
Interesting to see microglia changes are seen first then followed by oligodendrocytes and astrocytes - suggests cascade
Interesting to see microglia changes are seen first then followed by oligodendrocytes and astrocytes - suggests cascade
pnas.org
February 26, 2025 at 12:31 AM
pTDP-43 levels correlate with cell type–specific molecular alterations in the prefrontal cortex of C9orf72 ALS/FTD patients | PNAS pnas.org/doi/10.1073/...
Interesting to see microglia changes are seen first then followed by oligodendrocytes and astrocytes - suggests cascade
Interesting to see microglia changes are seen first then followed by oligodendrocytes and astrocytes - suggests cascade
Rethinking phase 2 trials in amyotrophic lateral sclerosis academic.oup.com/brain/articl...
Well-thought out discussion - basically if you want to run 6-month Phase 2 trials - use biomarkers for your go/no-go decision. If you want to use ALSFRS-R - you need a longer trial
Well-thought out discussion - basically if you want to run 6-month Phase 2 trials - use biomarkers for your go/no-go decision. If you want to use ALSFRS-R - you need a longer trial
Rethinking phase 2 trials in amyotrophic lateral sclerosis
There is a long history of ALS therapies being promoted on the basis of Phase 2 data, which then fail to translate into meaningful benefits in Phase 3 tria
academic.oup.com
February 25, 2025 at 10:58 PM
Rethinking phase 2 trials in amyotrophic lateral sclerosis academic.oup.com/brain/articl...
Well-thought out discussion - basically if you want to run 6-month Phase 2 trials - use biomarkers for your go/no-go decision. If you want to use ALSFRS-R - you need a longer trial
Well-thought out discussion - basically if you want to run 6-month Phase 2 trials - use biomarkers for your go/no-go decision. If you want to use ALSFRS-R - you need a longer trial
Reposted by MND Australia Research
Read our new perspective piece published in @mja.com.au. We explore the role of GCs in genomic medicine and current professional challenges. What started as passionate conference discussions has turned into a great perspective piece!
To read the paper: onlinelibrary.wiley.com/doi/10.5694/...
To read the paper: onlinelibrary.wiley.com/doi/10.5694/...
January 6, 2025 at 10:44 PM
Read our new perspective piece published in @mja.com.au. We explore the role of GCs in genomic medicine and current professional challenges. What started as passionate conference discussions has turned into a great perspective piece!
To read the paper: onlinelibrary.wiley.com/doi/10.5694/...
To read the paper: onlinelibrary.wiley.com/doi/10.5694/...
Characteristic symptoms of MND/ALS are preceded by sleep disturbances
Findings suggest that sleep disorders appear before the onset of motor impairment and respiratory issues
healthcare-in-europe.com/en/news/aml-...
www.science.org/doi/10.1126/...
Findings suggest that sleep disorders appear before the onset of motor impairment and respiratory issues
healthcare-in-europe.com/en/news/aml-...
www.science.org/doi/10.1126/...
AML: sleep problems identified as early warning sign
Amyotrophic lateral sclerosis (ALS), also known as Charcot’s disease, or Lou Gehrig’s disease, is a severe neurodegenerative disease that leads to progressive paralysis of muscles involved in voluntar...
healthcare-in-europe.com
February 21, 2025 at 7:09 AM
Characteristic symptoms of MND/ALS are preceded by sleep disturbances
Findings suggest that sleep disorders appear before the onset of motor impairment and respiratory issues
healthcare-in-europe.com/en/news/aml-...
www.science.org/doi/10.1126/...
Findings suggest that sleep disorders appear before the onset of motor impairment and respiratory issues
healthcare-in-europe.com/en/news/aml-...
www.science.org/doi/10.1126/...
A lipid nanoparticle system capable of delivering messenger RNA (mRNA) to the brain via intravenous injection
Better alternative to intrathecal (spinal) injections
Potential for MND gene therapies?
Only tested in mice so far...
www.nature.com/articles/s41...
www.news-medical.net/news/2025021...
Better alternative to intrathecal (spinal) injections
Potential for MND gene therapies?
Only tested in mice so far...
www.nature.com/articles/s41...
www.news-medical.net/news/2025021...
Blood–brain-barrier-crossing lipid nanoparticles for mRNA delivery to the central nervous system - Nature Materials
A lipid-nanoparticle-based formulation incorporating engineered lipids containing a blood–brain-barrier-crossing moiety effectively delivers mRNA to neurons and astrocytes following systemic administr...
www.nature.com
February 21, 2025 at 7:04 AM
A lipid nanoparticle system capable of delivering messenger RNA (mRNA) to the brain via intravenous injection
Better alternative to intrathecal (spinal) injections
Potential for MND gene therapies?
Only tested in mice so far...
www.nature.com/articles/s41...
www.news-medical.net/news/2025021...
Better alternative to intrathecal (spinal) injections
Potential for MND gene therapies?
Only tested in mice so far...
www.nature.com/articles/s41...
www.news-medical.net/news/2025021...
Neurological Alliance Australia (NAA) (MND Australia area member) are advocating for the creation of a Taskforce for Neurological Conditions to urgently address six critical areas of need.
It takes just a few minutes to send an email to your local MP
countusin.good.do/naa/count-us...
It takes just a few minutes to send an email to your local MP
countusin.good.do/naa/count-us...
Join me in supporting the Count Us In Campaign
Lorem ipsum dolor sit amet, consectetur adipiscing elit. Integer purus lacus, feugiat id efficitur sed, interdum at velit. Nunc diam tellus, fringilla ac tellus a, laoreet rhoncus risus. Donec nec imp...
countusin.good.do
February 21, 2025 at 6:55 AM
Neurological Alliance Australia (NAA) (MND Australia area member) are advocating for the creation of a Taskforce for Neurological Conditions to urgently address six critical areas of need.
It takes just a few minutes to send an email to your local MP
countusin.good.do/naa/count-us...
It takes just a few minutes to send an email to your local MP
countusin.good.do/naa/count-us...
Not specifically MND but the concussion/brain injury association with neurodegeneration is still not finalised
Sports-related concussion not associated with long-term cognitive or behavioural deficits: the PROTECT-TBI study
jnnp.bmj.com/content/earl...
Sports-related concussion not associated with long-term cognitive or behavioural deficits: the PROTECT-TBI study
jnnp.bmj.com/content/earl...
Sports-related concussion not associated with long-term cognitive or behavioural deficits: the PROTECT-TBI study
Background The cognitive effects of sports-related concussion (SRC) have been the subject of vigorous debate but there has been little research into long-term outcomes in non-athlete populations.
Met...
jnnp.bmj.com
February 20, 2025 at 3:58 AM
Not specifically MND but the concussion/brain injury association with neurodegeneration is still not finalised
Sports-related concussion not associated with long-term cognitive or behavioural deficits: the PROTECT-TBI study
jnnp.bmj.com/content/earl...
Sports-related concussion not associated with long-term cognitive or behavioural deficits: the PROTECT-TBI study
jnnp.bmj.com/content/earl...
Reposted by MND Australia Research
Genetic testing for #ALS #MND should go beyond identifying a single mutation. Even if a known genetic cause is found. A comprehensive analysis provides a clearer understanding of the condition and can help guide care &research efforts. Happy to help in this fantastic project.
February 19, 2025 at 10:26 AM
Some challenging ideas - a very well thought out hypothesis;
Amyotrophic lateral sclerosis represents corticomotoneuronal system failure - Eisen - Muscle & Nerve - Wiley Online Library onlinelibrary.wiley.com/doi/10.1002/...
Amyotrophic lateral sclerosis represents corticomotoneuronal system failure - Eisen - Muscle & Nerve - Wiley Online Library onlinelibrary.wiley.com/doi/10.1002/...
Amyotrophic lateral sclerosis represents corticomotoneuronal system failure
Several decades have passed since the anterograde corticomotoneuronal hypothesis for amyotrophic lateral sclerosis (ALS) was proposed. The intervening years have witnessed its emergent support based ...
onlinelibrary.wiley.com
February 19, 2025 at 4:50 AM
Some challenging ideas - a very well thought out hypothesis;
Amyotrophic lateral sclerosis represents corticomotoneuronal system failure - Eisen - Muscle & Nerve - Wiley Online Library onlinelibrary.wiley.com/doi/10.1002/...
Amyotrophic lateral sclerosis represents corticomotoneuronal system failure - Eisen - Muscle & Nerve - Wiley Online Library onlinelibrary.wiley.com/doi/10.1002/...
Pridopidine in ALS - HEALEY Platform Trial Outcome
"In this 24-week study, pridopidine did not impact the progression of ALS"
Is 6-months enough to see change? Healey now moving to 9-month trials - hopefully that will produce more definitive outcomes
jamanetwork.com/journals/jam...
"In this 24-week study, pridopidine did not impact the progression of ALS"
Is 6-months enough to see change? Healey now moving to 9-month trials - hopefully that will produce more definitive outcomes
jamanetwork.com/journals/jam...
Pridopidine in Amyotrophic Lateral Sclerosis
This randomized platform trial investigates whether pridopidine, a sigma-1 receptor agonist, impacted disease progression among patients with amyotrophic lateral sclerosis.
jamanetwork.com
February 18, 2025 at 10:22 PM
Pridopidine in ALS - HEALEY Platform Trial Outcome
"In this 24-week study, pridopidine did not impact the progression of ALS"
Is 6-months enough to see change? Healey now moving to 9-month trials - hopefully that will produce more definitive outcomes
jamanetwork.com/journals/jam...
"In this 24-week study, pridopidine did not impact the progression of ALS"
Is 6-months enough to see change? Healey now moving to 9-month trials - hopefully that will produce more definitive outcomes
jamanetwork.com/journals/jam...
CNM-Au8 in ALS - HEALEY Platform Trial outcomes
No significant positive effects.
Government funded EAP underway. Previous trials showed efficacy in longer-term follow-up - Healey only 6-months
Larger Phase 3 trial under consideration and Clene in discussions with FDA
jamanetwork.com/journals/jam...
No significant positive effects.
Government funded EAP underway. Previous trials showed efficacy in longer-term follow-up - Healey only 6-months
Larger Phase 3 trial under consideration and Clene in discussions with FDA
jamanetwork.com/journals/jam...
CNM-Au8 in Amyotrophic Lateral Sclerosis
This randomized, multicenter platform trial investigates the efficacy of CNM-Au8, an oral suspension of catalytically active gold particles, on the disease progression of patients with amyotrophic lat...
jamanetwork.com
February 18, 2025 at 10:18 PM
CNM-Au8 in ALS - HEALEY Platform Trial outcomes
No significant positive effects.
Government funded EAP underway. Previous trials showed efficacy in longer-term follow-up - Healey only 6-months
Larger Phase 3 trial under consideration and Clene in discussions with FDA
jamanetwork.com/journals/jam...
No significant positive effects.
Government funded EAP underway. Previous trials showed efficacy in longer-term follow-up - Healey only 6-months
Larger Phase 3 trial under consideration and Clene in discussions with FDA
jamanetwork.com/journals/jam...
Reposted by MND Australia Research
This morning, the National Science Foundation fired 168 probationary employees, effective immediately. The number represents roughly 10% of its workforce.
Follow Science’s coverage of President Donald Trump’s impact on U.S. research and science globally. ⬇️ scim.ag/40XtSSi
Follow Science’s coverage of President Donald Trump’s impact on U.S. research and science globally. ⬇️ scim.ag/40XtSSi
Trump Tracker: Firings, lawsuits, and U.S. science in chaos
Follow President Donald Trump’s impact on U.S. research and science globally
scim.ag
February 18, 2025 at 5:10 PM
This morning, the National Science Foundation fired 168 probationary employees, effective immediately. The number represents roughly 10% of its workforce.
Follow Science’s coverage of President Donald Trump’s impact on U.S. research and science globally. ⬇️ scim.ag/40XtSSi
Follow Science’s coverage of President Donald Trump’s impact on U.S. research and science globally. ⬇️ scim.ag/40XtSSi
Reposted by MND Australia Research
We're aware there is still debate going on, on the merits of ellorarxine. We've made our position on this clear, which is that from the data published so far, it is one of many drugs or compounds in a similar position. We'll be watching developments closely, as we do with all such situations.
February 18, 2025 at 4:12 PM
We're aware there is still debate going on, on the merits of ellorarxine. We've made our position on this clear, which is that from the data published so far, it is one of many drugs or compounds in a similar position. We'll be watching developments closely, as we do with all such situations.
Reposted by MND Australia Research
We are alarmed by proposed funding cuts that would devastate the fight against ALS. Slashing funding for NIH will hinder efforts to turn ALS from fatal to livable and cure it. Congress MUST reject these cuts! We need your voice NOW more than ever. bit.ly/NIH-funding-...
February 12, 2025 at 5:34 PM
We are alarmed by proposed funding cuts that would devastate the fight against ALS. Slashing funding for NIH will hinder efforts to turn ALS from fatal to livable and cure it. Congress MUST reject these cuts! We need your voice NOW more than ever. bit.ly/NIH-funding-...
Reposted by MND Australia Research
How does EXPERTS-ALS fit into the MND global drug development process?
#als #mnd
united2endmnd.org/2025/02/10/e...
#united2endmnd
#als #mnd
united2endmnd.org/2025/02/10/e...
#united2endmnd
EXPERTS-ALS Accelerating MND Drug Development
EXPERTS-ALS Accelerating MND Drug Development You may have seen the recent news from the UK MND Research Institute announcing that one of its first projects, EXPERTS-ALS, funded by the NIHR from th…
united2endmnd.org
February 10, 2025 at 7:30 PM
How does EXPERTS-ALS fit into the MND global drug development process?
#als #mnd
united2endmnd.org/2025/02/10/e...
#united2endmnd
#als #mnd
united2endmnd.org/2025/02/10/e...
#united2endmnd
Reposted by MND Australia Research
Our study on the safety and tolerability of Trimetazidine in #ALS @braincomms.bsky.social
Trimetazidine reduced oxidative stress markers and energy expenditure, warranting a follow up study
@rpavaneijk.bsky.social @fredsteyn.bsky.social @ammaralchalabi.bsky.social
academic.oup.com/braincomms/a...
Trimetazidine reduced oxidative stress markers and energy expenditure, warranting a follow up study
@rpavaneijk.bsky.social @fredsteyn.bsky.social @ammaralchalabi.bsky.social
academic.oup.com/braincomms/a...
February 10, 2025 at 10:10 AM
Our study on the safety and tolerability of Trimetazidine in #ALS @braincomms.bsky.social
Trimetazidine reduced oxidative stress markers and energy expenditure, warranting a follow up study
@rpavaneijk.bsky.social @fredsteyn.bsky.social @ammaralchalabi.bsky.social
academic.oup.com/braincomms/a...
Trimetazidine reduced oxidative stress markers and energy expenditure, warranting a follow up study
@rpavaneijk.bsky.social @fredsteyn.bsky.social @ammaralchalabi.bsky.social
academic.oup.com/braincomms/a...
Reposted by MND Australia Research
“These are real consequences, longer waits for cures and for diagnosis, slower scientific progress, losing out to competitors… and fewer jobs. Those who are facing any health challenges will suffer from less biomedical research.”
— D. Skorton
Via @nytimes: www.nytimes.com/2025/02/07/u...
— D. Skorton
Via @nytimes: www.nytimes.com/2025/02/07/u...
Trump Administration Cuts Put Medical Progress at Risk, Researchers Say
Grants from the National Institutes of Health come with additional money for overhead. A planned $4 billion cut would leave colleges with large budget gaps.
www.nytimes.com
February 9, 2025 at 11:01 AM
“These are real consequences, longer waits for cures and for diagnosis, slower scientific progress, losing out to competitors… and fewer jobs. Those who are facing any health challenges will suffer from less biomedical research.”
— D. Skorton
Via @nytimes: www.nytimes.com/2025/02/07/u...
— D. Skorton
Via @nytimes: www.nytimes.com/2025/02/07/u...
Reposted by MND Australia Research
The NIH’s drastic cut to indirect cost rates is a critical threat to U.S. research infrastructure www.statnews.com/2025/02/08/n... via @statnews.com
The NIH’s drastic cut to indirect cost rates is a critical threat to U.S. research infrastructure
The drastic reduction in indirect costs is not a path to innovation or cost savings — it’s a threat to U.S.’s position as global leader in medical research.
www.statnews.com
February 8, 2025 at 2:23 PM
The NIH’s drastic cut to indirect cost rates is a critical threat to U.S. research infrastructure www.statnews.com/2025/02/08/n... via @statnews.com
Can't do your research in the dark or in the garden - infrastructure funding is vital and much of that comes through indirect costs!
Indirect costs fund the staff and facilities to do research at all universities. To find effective treatments for #ALS #MND scientists across the world work together. We need our NIH colleagues in the team.
The NIH’s drastic cut to indirect cost rates is a critical threat to U.S. research infrastructure www.statnews.com/2025/02/08/n... via @statnews.com
February 9, 2025 at 10:32 PM
Can't do your research in the dark or in the garden - infrastructure funding is vital and much of that comes through indirect costs!
Good to see this Australian-led study ready for the next phase;
Open-label phase 2a study to assess the safety and tolerability of trimetazidine in patients with ALS
academic.oup.com/braincomms/a...
@rpavaneijk.bsky.social @ngoshyuan.bsky.social @fredsteyn.bsky.social @ammaralchalabi.bsky.social
Open-label phase 2a study to assess the safety and tolerability of trimetazidine in patients with ALS
academic.oup.com/braincomms/a...
@rpavaneijk.bsky.social @ngoshyuan.bsky.social @fredsteyn.bsky.social @ammaralchalabi.bsky.social
An open-label phase 2a study to assess the safety and tolerability of trimetazidine in patients with amyotrophic lateral sclerosis
van Eijk et al. report that oral trimetazidine is safe and well-tolerated in amyotrophic lateral sclerosis (ALS) patients, significantly reducing oxidative
academic.oup.com
February 9, 2025 at 10:22 PM
Good to see this Australian-led study ready for the next phase;
Open-label phase 2a study to assess the safety and tolerability of trimetazidine in patients with ALS
academic.oup.com/braincomms/a...
@rpavaneijk.bsky.social @ngoshyuan.bsky.social @fredsteyn.bsky.social @ammaralchalabi.bsky.social
Open-label phase 2a study to assess the safety and tolerability of trimetazidine in patients with ALS
academic.oup.com/braincomms/a...
@rpavaneijk.bsky.social @ngoshyuan.bsky.social @fredsteyn.bsky.social @ammaralchalabi.bsky.social
With Tofersen having the potentially to significantly slow disease - would such treatments as this potentially return some function if used in conjunction (sorry behind paywall)
First-in-human study of epidural spinal cord stimulation in individuals with SMA
doi.org/10.1038/s415...
First-in-human study of epidural spinal cord stimulation in individuals with SMA
doi.org/10.1038/s415...
First-in-human study of epidural spinal cord stimulation in individuals with spinal muscular atrophy - Nature Medicine
Improved muscle function, strength and fatigue measures were observed after electrical spinal cord stimulation in individuals with spinal muscular atrophy.
doi.org
February 7, 2025 at 3:59 AM
With Tofersen having the potentially to significantly slow disease - would such treatments as this potentially return some function if used in conjunction (sorry behind paywall)
First-in-human study of epidural spinal cord stimulation in individuals with SMA
doi.org/10.1038/s415...
First-in-human study of epidural spinal cord stimulation in individuals with SMA
doi.org/10.1038/s415...
Only 7 patients but encouraging finding
Currently under review for approval by TGA in Australia
Tofersen treatment leads to sustained stabilization of disease in SOD1 ALS in a “real‐world” setting
onlinelibrary.wiley.com/doi/10.1002/...
Currently under review for approval by TGA in Australia
Tofersen treatment leads to sustained stabilization of disease in SOD1 ALS in a “real‐world” setting
onlinelibrary.wiley.com/doi/10.1002/...
Tofersen treatment leads to sustained stabilization of disease in SOD1 ALS in a “real‐world” setting
Objective Patients with amyotrophic lateral sclerosis (ALS) caused by superoxide dismutase 1 (SOD1) gene mutations (SOD1 ALS) treated with tofersen have shown slowing of disease progression, and dis...
onlinelibrary.wiley.com
February 5, 2025 at 10:35 PM
Only 7 patients but encouraging finding
Currently under review for approval by TGA in Australia
Tofersen treatment leads to sustained stabilization of disease in SOD1 ALS in a “real‐world” setting
onlinelibrary.wiley.com/doi/10.1002/...
Currently under review for approval by TGA in Australia
Tofersen treatment leads to sustained stabilization of disease in SOD1 ALS in a “real‐world” setting
onlinelibrary.wiley.com/doi/10.1002/...
Reposted by MND Australia Research
We are aware of recent discussions within the MND community regarding research into ellorarxine. Read our update here 👇
www.myname5doddie.co.uk/whats-on/art...
www.myname5doddie.co.uk/whats-on/art...
February 5, 2025 at 3:58 PM
We are aware of recent discussions within the MND community regarding research into ellorarxine. Read our update here 👇
www.myname5doddie.co.uk/whats-on/art...
www.myname5doddie.co.uk/whats-on/art...
Plenty of caveats but a decent number of participants and to my non-expert eye a reasonable analysis;
Longer disease progression milestone-free time in people with ALS treated vs not treated with iv edaravone: results from an administrative claims analysis becarispublishing.com/doi/10.57264...
Longer disease progression milestone-free time in people with ALS treated vs not treated with iv edaravone: results from an administrative claims analysis becarispublishing.com/doi/10.57264...
Longer disease progression milestone-free time in people with amyotrophic lateral sclerosis treated versus not treated with intravenous edaravone: results from an administrative claims analysis
Aim: To estimate time-to-progression milestones in people with amyotrophic lateral sclerosis
(PALS) treated versus not treated with intravenous (IV) edaravone (Radicava® IV, Mitsubishi Tanabe Pharma A...
becarispublishing.com
February 5, 2025 at 4:22 AM
Plenty of caveats but a decent number of participants and to my non-expert eye a reasonable analysis;
Longer disease progression milestone-free time in people with ALS treated vs not treated with iv edaravone: results from an administrative claims analysis becarispublishing.com/doi/10.57264...
Longer disease progression milestone-free time in people with ALS treated vs not treated with iv edaravone: results from an administrative claims analysis becarispublishing.com/doi/10.57264...
Very useful study - would be great to see further studies adding to the data and develop consensus numbers.
Full article: Estimating the minimum important difference in the ALSFRS-R-instrument in people living with MND www.tandfonline.com/doi/full/10....
Full article: Estimating the minimum important difference in the ALSFRS-R-instrument in people living with MND www.tandfonline.com/doi/full/10....
Estimating the minimum important difference in the ALSFRS-R-instrument in people living with MND
Objective: The Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) is a commonly used outcome measure in clinical trials for motor neuron disease (MND) therapies. As such, understandin...
www.tandfonline.com
February 5, 2025 at 12:00 AM
Very useful study - would be great to see further studies adding to the data and develop consensus numbers.
Full article: Estimating the minimum important difference in the ALSFRS-R-instrument in people living with MND www.tandfonline.com/doi/full/10....
Full article: Estimating the minimum important difference in the ALSFRS-R-instrument in people living with MND www.tandfonline.com/doi/full/10....