International Alliance of ALS/MND Associations - Scientific Advisory Council Webinar H1 2025

I tend to ask chatGPT for a summary of the transcript of videos so that I don't have to spend the time watching them. I have done this for the ALS Scientific Advisory Council from about a month ago which follows (the critique is of chatGPT - my critique is that they don't talk about mitochondria, the closest they come to epigenetics is DNA methylation):

International Alliance of ALS/MND Associations
Scientific Advisory Council Webinar – Transcript Summary

Opening & Housekeeping

Jessica Mabe (Programs Coordinator, International Alliance) welcomed attendees and introduced the webinar, noting caption availability (via Zoom chat or QR code) and thanking sponsor Mitsubishi Tanabe Pharma.

The Scientific Advisory Council

Chair & Moderator: Dr Nicholas Cole (Head of Research, MND Association, UK)

Panelists:

• Dr Kuldip Dave – Vice President of Research, ALS Association (USA)
• Dr Nadia Sethi – Co‑chair, NEALS Patient Education & Advocacy Committee (USA)
• Dr Martha Peña – Neurologist, Roosevelt Institute (Bogotá, Colombia)
• Dr Christopher McDermott – Professor of Translational Neurology, University of Sheffield (UK)
• Dr Jinsy Andrews – Assoc. Professor of Neurology & Director, Neuromuscular Clinical Trials, Columbia University (USA)
• Dr Martina de Majo – Scientific Director, International Alliance (Italy)

Attendees were invited to submit questions through the Zoom Q&A box.

1. Optimism & Concerns in ALS/MND Research

Panel reflections

Speaker	Optimistic about …	Keeps me up at night …
Dr C. McDermott	Rapid advances in disease biology; proof-of-concept that targeted SOD1 therapy changes trajectory.	Persistent funding shortages slowing progress.
Dr N. Sethi	Robust global natural‑history data feeding an expansive pipeline and AI‑powered insights.	Urgent unmet treatment need; difficulty translating research for newly diagnosed patients.
Dr M. de Majo	Greater inclusion of under‑represented regions and populations in studies.	Ongoing inequity in trial and therapy access.
Dr M. Peña	Progress in exposome studies & epigenetics (e.g., DNA methylation).	High cost of emerging therapies and delivery challenges in resource‑limited settings.
Dr J. Andrews	Gene editing, antisense oligos, AI tools for trial matching, improved trial design.	Imminent research‑budget cuts; limited trained workforce worldwide.
Dr K. Dave	Evidence of functional recovery (SOD1, FUS) suggests broader potential.	Clinical & biological heterogeneity; need for finer ALS sub‑types.

2. SAC Top‑Five Recommendations for Trial Sponsors

1. Publish results swiftly & responsibly (top‑line within months; avoid hype).
2. Share data & biosamples via open repositories (e.g., PRO‑ACT, NEALS biobank).
3. Give clear post‑trial‑access guidance (compassionate or expanded access).
4. Communicate development timelines as transparently as possible.
5. Unblind participants promptly once study integrity allows.

3. Research Funding Landscape

• USA: Proposed 40 % NIH cut + indirect‑cost reduction threatens two decades of work; $300 M in neuroscience grants already halted.
• Global call: Unified advocacy across neurodegenerative diseases to safeguard funding.
• Colombia: Reliance on limited public funds & donations; ~400 patients at Roosevelt Institute, but under‑reporting likely.

4. UK MND Research Institute & EXP‑ERTS‑ALS Platform

Patient‑led campaign secured £50 million (UK). The virtual institute connects discovery science to clinical trials. EXP‑ERTS‑ALS uses neurofilament light chain to prioritise agents before expensive Phase III trials—enabling fewer, faster, smarter studies.

5. Accelerating Diagnosis

ALS remains a clinical diagnosis. Key initiatives to shorten delay:

• Think ALS (USA) and Red Flag (UK) checklists for community neurologists.
• Alliance & My Name’5 Doddie Foundation developing a global e‑learning course for GPs and allied‑health professionals.

6. Audience Q&A Highlights

Is riluzole effective?

Real‑world evidence shows a median survival benefit of 6–19 months, particularly when started within six months of symptom onset (Andrews et al., 2020).

Progress on C9orf72 therapies

• Two ASO trials informed target biology despite mixed results.
• New approaches: CRISPR editing, pathway‑specific drugs, and C9‑enriched multi‑arm trials.
• C9 carriers remain eligible for most broader ALS trials.

Closing Remarks

Dr Cole emphasised that ALS/MND is now demonstrably treatable—progress relies on coordinated effort across researchers, clinicians, people with lived experience, advocates and funders.

Jessica Mabe thanked the panel, sponsor, and attendees, wishing everyone a good day wherever they are.

2  Concise summary

Theme	Key points	Why it matters
Momentum in basic science	Improved disease models and Tofersen proof‑of‑concept establish ALS as a targetable condition.	Shifts mindset from “inexorable” to “treatable.”
Technology enablers	AI analytics, CRISPR, viral vectors, & neurofilament assays accelerate discovery and Phase‑2 decisions.	Could shorten timelines and reduce late‑stage trial failures.
Equity & diversity	Awareness growing, but LMIC access and representation still lag.	Diverse cohorts are essential for globally effective therapies.
Funding headwinds	Proposed 40 % NIH cut; UK secured £50 M by patient advocacy.	Risk of “lost generation” of scientists; infrastructure fragile.
Trial‑sponsor behaviour	SAC “Top‑5” urges rapid data‑sharing, clear post‑trial access, timely un‑blinding.	Maximises scientific value of every participant’s effort.
Diagnosis & early treatment	Median 12‑month delay; tools like “Think ALS” target referral bottlenecks; early riluzole adds 6–19 months.	Earlier therapy improves outcomes and enriches trials.
Pipeline snapshot	>180 investigational agents; C9 pivoting to CRISPR & pathway drugs; EXPERT‑ALS prioritises via neurofilament.	Indicates broad but rationalising global activity.

3  Critique

Strengths

• Diverse, high‑calibre panel blending academic, clinical‑trial, charity and LMIC viewpoints.
• Candid discussion of funding politics, heterogeneity and equity rather than pure success stories.
• Action‑oriented outputs – “Top‑5” checklist; diagnostic e‑learning in development.
• Patient‑centred framing – emphasis on burden, un‑blinding and compassionate access.
• Realistic optimism – gene‑editing & AI presented with caveats, not hype.

Weaknesses / Gaps

Issue	Comment
Limited LMIC airtime	Latin‑American voice present but Africa & Asia absent; discussion returned to US/UK quickly.
Data granularity	Funding‑cut figures lacked sourcing; a slide/table would boost credibility.
Over‑reliance on neurofilament	Alternative or complementary biomarkers weren’t explored in depth.
Audience engagement	Live Q&A felt rushed; few pre‑submitted questions covered.
Production	Auto‑captions contained errors; speaker labels missing; human‑edited captions would improve accessibility.

Suggestions for next iteration

1. Publish a one‑page fact‑sheet of funding statistics with references.
2. Invite additional LMIC investigators (e.g. Sub‑Saharan Africa, South‑East Asia).
3. Dedicate a webinar solely to biomarker development beyond neurofilament.
4. Reserve a 15‑minute live Q&A block, grouping questions by theme.
5. Release human‑edited English & Spanish captions plus a cleaned transcript post‑event.

Bottom line: The webinar paints a 2025 snapshot of cautious optimism: genuine scientific breakthroughs tempered by funding threats and persistent inequities. Implementing the panel’s own transparency standards – and broadening the global lens – could make future updates even more valuable.