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NEALS 2025 Report

Our Advocates Perspective

End the Legacy and two of it's advocates Jean Swidler and Cassandra Haddad were gratified to be accepted to share perspectives and research at this prestigious annual meeting of North America's largest ALS research membership organization, NEALS.  While there we paid rapt attention to the proceedings and are glad to bring back to the genetic community some insights. Of course these are subjective and we do not claim they are especially insightful. If you think we have erred on any interpretation we are happy to hear about it and correct or at least have a good conversation!  Email us at info@endthelegacy.org. 


In the photo collage clockwise from top right, the Research Ambassador Panel Jean was able to speak on with other accomplished patient advocates, our poster session handled expertly by Cassandra, Jean with teams from two new recognized centers of at risk care including Dr Rosow from UCSF ALS Center and Dr Ajroud-Driss from the Les Turner ALS Center at Northwestern, and a candid group photo with ETL co-founder Daniel! 

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3 Things ( Updated the Week of October 13th)

Three Research Updates 

All in the genetic community should hear from the meeting. 


  1. 1) Tofersen 3 year data 
    Dr Timothy Miller shared the amazing 3 year open label extension data from the trial of Tofersen, developed by Dr Miller and Ionis and trialed by Biogen with Dr. Miller as the primary investigator. This means the entire cohort of trial participants, both those on drug from the start and those on placebo from the start, had 3 years of drug on open label before this data was looked at. In the overall group, 25% of trial participants had improvements in the ALSFRS. In the fast progressing group, those on early start had a 1.5 year survival benefit over the placebo start participants. This readout should be enough to convert the accelerated approval Biogen received to a traditional approval, though of course we hope the Atlas trial also reads out positive ushering in an era of maintaining function in all sod1 carriers rather than hoping to restore function in some slower progressing SOD1 carriers after manifest symptoms appear. 

  2. The Uniqure Update The perilous nature of many gene therapies was exemplified by the sad update from the gene targeting pharmaceutical company Uniqure sharing their AAV therapy for SOD1 ALS was on pause following a significant side effect in one of their 5 dosed patients. You may hear our drive towards earlier inclusion in clinical trials for ALS and outcomes such as this are in tension. We are always clear for any novel therapeutic with unclear safety profiles that only in later stage pivotal trials are we pushing for inclusion. For substances of well known safety, much more leeway could be used.  

  3. Innovative Early Stage Trial Systems Dr Suma Babu of Mass General Hospital / Harvard shared about their new ALS My Match platform trial. This is an early stage phase 1 / 2 trial system which will leverage specific targeted subgroups for short trials looking at biomarkers, safety and initial efficacy signals. The first trial in this program is for the substance Digoxin and includes a C9orf72 ALS cohort. This joins the Experts platform trial in the UK where other quick biomarker driven early phase trials will be held. We think this is an excellent development and we cheer all involved. 

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Three Topics We Hope

Evolve in Future Meetings

  1. Cures As a Focus There was an inspiring focus on the history and accomplishments of the NEALS organization on the occasion of it’s 30th anniversary at this year’s conference. We applaud the vision and commitment of so many to propel the research field of ALS so far forward. We hope that this will include attention to treatments with a large impact on the disease that adds significantly to the years a person living with ALS is productive. While we are excited about the present therapies and understand that any treatment slowing the disease is good news, we welcome a strong centralized focus towards cures.

  2. Early Intervention As the disease interventions in conditions that are progressively destructive will work best when administered early in the disease process we look to clinicians and researchers to underscore the importance of early diagnosis and treatment. We would welcome the expansion of considerations that will enable timely access to disease modifying agents for those at genetic risk and including cohorts of at risk genetic carriers with biological signs of disease activity as cohorts in late stage trials with documented safety. In fact an expert review earlier this year discussed this type of trial is possible.

  3. ALL ALS As Priority The tremendous energy of the whole room of dedicated researchers clearly committed to preserving robust research and care funding was a sure highlight of this gathering of esteemed minds. It is so important to see the energy continue and hope to see more discussion regarding the precarious nature of ALL ALS funding. The ACT for ALS has provided dedicated dollars that have benefitted ALL ALS , but this is through a second tier status where dollars first must be available to ALS EAPS. To date, the ALS EAPS that have been funded have allowed for leftover monies to go to ALL ALS. However, what is the risk if there is no monies left after the EAPS are funded? We want to support and assure continued funding for the most impressive natural history study ever in ALS and would hope for widespread discussion regarding this in the ALS Community.

Three Ways

We Were Proud to Contribute to the Meeting

  1.  Poster Session On Wednesday we were proud to have our own original research highlighted , with our slightly updated poster on Informants in Asymptomatic Research presented by our Vice Chair Cassandra Haddad. Many conversations were had with sympathetic researchers who did not quite understand how ill thought out the systems of surveillance had been put into practice in our communities without consideration of the impacts on those being asked to participate and further without warning of the possible effects of the use of behavioral informants via their spouses or other loved ones. We cheer Cassandra for her efforts and thank everyone who took the time to speak to us, including the team from Prevent ALS who confirmed they are adding language on this possible risk to their informed consent documents. 

  2. Panel Participation
    We were so gratified to again have our own Jean Swidler asked to speak on the Research Ambassador panel from the main plenary stage. Connecting the reality of generations being severely impacted by ALS with the work we all are doing together was work well done with many sharing it touched them personally to hear. 

  3. CRLI Engagement
    Earlier in the week a few End the Legacy community members were able to participate in the annual Clinical Research Learning Institute or CRLI. They made sure the topics of at risk carriers and also of the linked disease FTD were not forgotten in the group conversations. We cheer them - shout out to Brooke and Emily for their bravery in speaking up! And we thank NEALS for prioritizing educating community members. 

Three Things

We Loved to See 

  1. NonProfit Roundtable NEALS has made a commitment to help coordinate the many ALS organizations operating in the United States and End the Legacy is happy to participate in that needed effort. From the main stage a few organizations were able to share about their work, and there was a non-proift forum we were able to sit in on. 

  2. Genetic Focus
    It was great to see so many updates on genetic work that is ongoing. Cheers to NEALS for seeing the value of this work, and to scientists such as Drs Clelland , Landers, Miller, Harms , Bennet, Brown, Crooke and many others who shared about the genetic driven work they do. 

  3. International Collaboration It was great to hear from researchers from Canada, the UK, Europe and India who all participated in person or via remote. 

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