Reflections on My Experience at the 2024 International ALS/MND Symposium

In February 2021, I was diagnosed with ALS, and as I approach my four-year anniversary, I reflect on the challenges and rewards of being involved in ALS advocacy. Over the past three years, I’ve had the privilege of being selected as a Patient Fellow for the International Alliance ALS/AMD Symposium, where we share the knowledge gained with the broader community to raise awareness and provide accurate information.

This year, after attending virtually the last two years, I was excited to travel to Montreal in person. My husband, older son, and I made the journey from New Jersey, navigating icy roads and freezing temperatures, but the winter beauty of Montreal made it all worthwhile.

A heartfelt thank you to the ALS Hope Foundation and Dr. Terry Heiman-Patterson for introducing me to the Patient Fellow program. I am also deeply grateful to Dr. Sara Feldman, Chair of the Patient and Caregiver Advisory Council (PCAC), for nominating me to join the council. These opportunities have allowed me to contribute meaningfully to the global ALS community.

In addition to attending the symposium, I had the privilege of participating in a panel discussion titled Bridging the Gap: What PALS Want Researchers & Clinicians to Know (and Keep Doing Well!). It was an invaluable chance to share our voices and highlight the importance of patient involvement in research and care. As a member of the Patient and Caregiver Advisory Council (PCAC), I also joined my fellow council members in introducing ourselves, discussing the work of the PCAC, and emphasizing its role in advancing ALS care.

At the conference, I engaged with leading experts in ALS research, allied health, and patient care. One particularly fascinating area was the research on pre-symptomatic gene carriers, which is gaining increasing attention and showing promising results. A standout moment was the debut of the A Day in the Life Suite, a fully accessible apartment that allowed attendees to experience adaptive tools and technologies firsthand.

The conference was an unparalleled educational experience, offering excellent networking opportunities and plenty of time to socialize. I had the privilege of meeting people from all over the world—many of whom I’ve only connected with virtually in the past few years. The camaraderie was palpable, and while ALS brought us together, we left as true friends.

I returned home with renewed hope and optimism. Meeting and learning from others in the ALS community, both patients and researchers, has been one of the most rewarding aspects of my journey.

 Author Dr Stacy Lewin 

Co-Author Scribed and Edited by Daniel Farber 

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Notes from a Virtual Attendee

Todd Kelly also attended the meeting virtually as a Patient Fellow. He’s on the laptop in the photo to the right!

He also shared some takeaways from the meeting.

Some highlights were the following.  Dr. Bedlack is an engaging and effective speaker who always manages to cut through the jargon as he did with his session about the ALS Untangled website.  CALS (and PALS) could not help but be inspired by Linda Levine, who hilariously imparted caregiver advice that she has learned while supporting her husband David.  ALS Hope Foundation’s own Stacy Lewin Farber was part of strong panel of PALS, sharing their expectations of researchers and clinicians.  Probably the most impressive presentation came from the keynote speaker, Leanne Sklavenitis, an Australian woman diagnosed with ALS in 2017 who has lost her ability to walk alone and her voice but whose indomitable spirit is an inspiration to all.  I find the Alliance Meeting and Allied Professionals Forum much more PALS/CALS-friendly than the more technical International Symposium on ALS/MND. 

One element I found fascinating was the “A Day in the Life” Suite, an actual hotel suite set up with a huge range of adaptive equipment for ALS patients.  Even a quick glimpse via a video tour was eye-opening to the possibilities to enhance one’s quality of life and reduce the caregiver’s burden somewhat.  If only something like this could be made accessible to PALS in a virtual reality format!

One drawback of being a virtual attendee of the actual forum is that only one of the three sessions is streaming live, so the choice is made for you. However, I was able to submit a question online about the ADORE clinical trial of oral edaravone and have it answered by the presenter live.  While the ADORE findings were disheartening, the sheer amount of research being done globally and highlighted in the symposium is truly encouraging.  The Poster Hall further supplements the scheduled speakers.  Many of the scientists presenting are very young/graduate students who offer generational continuity to this ongoing fight.

Todd Kelly

Allied Professionals Forum 2024

The Allied Health Professionals Forum, or APF, took place this December 4-5, 2024 in Montreal, Quebec, Canada. The ALS Hope Foundation has been supporting this program for the past 20 years, starting with its second meeting which was held in Philadelphia in 2004.

Physical Therapist Sara Feldman (USA) co-chairs the event along with Rachael Marsden (UK) which brings together allied health professionals from around the globe and this year over 400 people joined both in-person and virtually. We were warmly welcomed virtually by the PCAC members, who thanked all of the allied professionals in attendance for the work they do and the care and compassion they do it with.

We were pleased to have several members of the MDA/ALS Center of Hope and ALSHF Team, attend this year's APF. Sara Feldman, Donna Harris, Jamey Piggott, Donna Sickles, Julie Lichtman were able to attend in person with Mary Holt-Paolone and Jennifer Doto attending virtually.

Leanne Sklavenitis (PALS/Australia) was the keynote speaker for the APF and her story and attitude were both inspirational and heartwarming. We were all truly moved with Donna Sickles, RT, commenting that her presentation was one of her favorite things about the conference. "She filled my soul with hope." You can read more about Leanne's journey on her website. #beawesomeeveryday 

The two days were packed full of presentations from a variety of disciplines on a wide range of topics. We will highlight a few below.

The programming began with encouragement to meet up with others and share your information and network. Don’t wait to connect! This was echoed in another presentation, The Pass it On Project, which is an informal way to connect new clinicians with those who are more experienced. Jen Doto, our new OT, has benefitted from connecting with Sara Solomon (Australia) in this way! (PTs & OTs and the SLPs networked!) Other presentations focused on this including two describing their Community of Practice, one of which was based on a previous presentation, a survey of nurses and allied health professionals in the UK and ways to address their educational needs, and a look at mental health for allied health professionals who work in the ALS/MND community.

Donna Harris, SLP, noted, “One of the best things for me about attending the International Alliance of MND/ALS conference is connecting with other speech language pathologists. It's amazing to share ideas and concerns with others in your profession who specialize in ALS. One of the first things I did was to ask our conference moderator, our own Sara Feldman, to request on my behalf to have all SLP's meet in a designated area at our break. I met 8-10 other ALS SLP's who I immediately collected names and emails for all of us to have.  Already I have received a request to start a research project on EMST protocols. I have requested information from another SLP for her handouts on laryngospasm to incorporate in our clinic. I was also offered to have 1:1 voicebank training in our clinic by the amazing John Costello from Boston Children's Hospital who specializes in this area. This exciting idea sharing will continue all year long until we meet up again next year.”

There were presentations focused on symptom management and new ways to measure or augment abilities such as spasticity management, using AI for good, using digital health technology to track progression, tips on eye tracking in ALS and driving evaluations. There were also updates on NIV use and individually designed neck supports. There was a fun and engaging take on pureeing food and an excellent presentation on the Art of Living Well which looked at ways to bring creative arts into the picture.

Julie Lichtman, RD, commented “The conference featured a wide range of topics, and one session I initially anticipated would be less relevant—on nutrition and texture—ended up being one of the most engaging. I was pleasantly surprised by the passion and enthusiasm demonstrated by the speech therapists and dietitians. An example that stood out as surprisingly insightful was the simple yet effective strategy of heating food before blending to improve its consistency and ease of digestion—such a practical innovation!” She also loved the Creative Arts presentation, “I was thoroughly impressed by the creative therapies being explored for PALS, such as wheelchair marble painting and virtual reality interventions. These forward-thinking approaches demonstrate the extraordinary potential for improving quality of life for individuals living with ALS.”

Dr. Rick Bedlack kicked off the presentations with an update on the ALS Untangled website and how we as allied health professionals could use it, too. Racial disparities in both diagnosis and prognosis were discussed by the CDC. The experience of Tofersen for SOD1 and the importance of rehab as concomitant care was presented by Dr. Riccardo Zuccarino, a physiatrist in Italy. Interesting takes on Interdisciplinary Care and Palliative Care were presented.

Several presentations looked at care and support for PALS/CALS including bereavement care, moving forward after loss, and online support for adult children of PALS. One presentation was by a caregiver, Linda Levine, and was titled Angels in Anguish. It was an amazing presentation and touched all of us. Jamey Piggott wrote “One presenter who captured my mind and my heart was Linda Levine. She presented two sessions about Caregiving and Mindfulness.  She spoke of the difficulties in being a caregiver and the toll it takes mentally, emotionally, and physically as well as financially.  She validated the difficult life changes it causes on the caregiver and their loved one. In her sessions, she encouraged both caregivers and PALS to use their voice with a focus on expressing their thoughts, to ask for help, and to acknowledge that everything changes and takes more time. She reiterated that self-care is essential, but put no limit on what that looks like to each individual.”

Stacy Lewin Farber took part in a panel discussing Bridging the Gap: What PALS Want Researchers and Clinicians to Know (and Keep Doing Well!). This amazing panel helped to inform and educate the clinicians in the room.

Julie Lichtman, RD, shared “what resonated most deeply with me were the personal stories shared by the PALS (People with ALS), particularly Stacy, who spoke so candidly about her journey. Leanna's presentation, having traveled all the way from Australia, was especially impactful. Her perspective and the opportunity to sit with her, meet her caregivers, and hear firsthand how she navigates life with ALS, truly shifted my perspective on living with resilience and grace.”

From Todd Kelly, Patient Fellow and ALSHF PAC member, “Some highlights were the following.  Dr. Bedlack is an engaging and effective speaker who always manages to cut through the jargon as he did with his session about the ALS Untangled website.  CALS (and PALS) could not help but be inspired by Linda Levine, who hilariously imparted caregiver advice that she has learned while supporting her husband David.  ALS Hope Foundation’s own Stacy Lewin Farber was part of strong panel of PALS, sharing their expectations of researchers and clinicians.  Probably the most impressive presentation came from the keynote speaker, Leanne Sklavenitis, an Australian woman diagnosed with ALS in 2017 who has lost her ability to walk alone and her voice but whose indomitable spirit is an inspiration to all. “

Watch our APF Meeting Instagram/Facebook reel here.

Day in the Life Suite: New this year, during the Allied Professionals Forum, the Alliance transformed one of the hotel suites into an apartment for someone living with ALS/MND. It was equipped it with technology and devices that help with everyday tasks and so much more. Participants could get hands-on with the tools, and see what’s available to help the ALS/MND community. Watch Jarnail Chudge, Innovation and Technology Lead at the Alliance, provide us with a full walk-through video. 

The ALS Hope Foundation was a supporter of this Suite and Sara Feldman introduced the Suite and along with Cathy Cummings, Executive Director of the Alliance. This support would not be possible without the foresight of Dr. Jeffrey Deitch, and we believe he would be truly proud of not only the suite, but also what the Allied Health Professionals Forum has become. See Sara's heartfelt words here.

Todd Kelly, Patient Fellow, noted "One element I found fascinating was the Day in the Life Suite, an actual hotel suite set up with a huge range of adaptive equipment for ALS patients.  Even a quick glimpse via a video tour was eye-opening to the possibilities to enhance one’s quality of life and reduce the caregiver’s burden somewhat.  If only something like this could be made accessible to PALS in a virtual reality format!"

“The Day in the Life suite was another highlight of the conference, offering a comprehensive look into the advances being made in adaptive equipment and feeding aids. It was awe-inspiring to see the strides being made in technology, and I am eager to see what the future holds as these innovations continue to evolve.” Julie Lichtman, RD

“The overarching message about the necessity of individualized, compassionate care and emotional support for each patient was profoundly impactful. It reaffirmed the importance of the holistic, patient-centered approach that we, as allied professionals, must continue to champion in our work.” Julie Lichtman, RD

International Alliance of ALS/MND Associations Meeting 2024

The International Alliance of ALS/MND Associations held their Annual Meeting in Montreal, Quebec, Canada this December 2-3, 2024 hosted by the ALS Society of Canada and ALS Quebec. The Alliance is comprised of organizations from around the world and this year's meeting had attendees from 33 countries (see photo below!).

The Alliance is comprised of organizations from around the world and this year's meeting had attendees from 33 countries (see photo above!). The ALS Hope Foundation has been a member of the International Alliance of ALS/MND Associations since 2001 and this year Terry Heiman-Patterson, Jamey Piggott and Sara Feldman represented the Foundation in person at the International Alliance of ALS/MND Associations Annual Meeting.

Members of the ALS Hope Foundation PALS Advisory Council attended as members of the Patient Fellows Program, this year hosted by the Alliance. Stacy Lewin Farber (in person) and Todd Kelly (virtually) attended all three international meetings.

The programming began with a tribute to people living with ALS/MND around the globe with the March of Faces video.  Special thank you to Michail Christakis and Doug Hill who represented the ALS Hope Foundation.

Watch the March of Faces 2024 video here. 

Program chair and PALS Gudjon Sigurdsson (Iceland), Cathy Cummings (Canada), Executive Director at the International Alliance, and Calaneet Balas (USA), Chair of the Alliance, welcomed everyone and kicked off the presentations from members around the globe. We will highlight a few of these presentations and give you an overview of the meeting below.

The first session was an interactive discussion on common challenges facing many organizations and possible solutions from peers. This support and collaboration idea was seen later when collaboration was the focus of a panel that included members from Argentina, Australia, Belgium, Kenya and the USA. Julia Simon from the Alliance educated us on the Alliance’s Knowledge Management Project, a framework to help set the educational foundation for PALS/CALS. The final product will be available to members to help them to ensure they are covering all areas and help strengthen each member organization.

We heard from several organizations with updates on their work over the past year including social issues facing PALS/CALS in Argentina, PALS led volunteer program in Italy, the cost of ALS/MND in Turkey and the newly formed association in Vietnam.

Technology was the focus of a presentation from Colombia on their Communication and Voice Preservation initiatives, Steve Saling provided a look into the ALS Residences in the US, Taiwan showed us their amazing use of technology in an almost futuristic look, and Jarnail Chudge, Innovation and Technology Advisory Council, discussed the use of AI and the development of the Patients Needs Matrix to ensure the needs and wants of PALS are being heard.  

We heard the voice of people living with ALS/MND and their caregivers woven throughout the two days. The PALS and CALS Advisory Council, or PCAC, Chaired by Sara Feldman (USA) with member Stacy Lewin Farber (PALS/USA) provided an update on the Alliance’s Fundamental Rights Survey.

Paula Trefiak (FALS/Canada), Leah Stavenhagen (PALS/France/USA), Steven Spangenberg (CALS/Australia), Leanne Sklavenitis (PALS/Australia), and Norman MacIsaac (PALS/Canada) took place on the in person panel.

Several organizations shared information on research including an overview of NEALS, FightMND’s research focused fundraising, ALSA’s survey on PALS perspectives on research, an update on International Collaboration from the Alliance, and a panel on Regional Research Updates led by Martina de Majo, the Alliance’s Science Director.

Genetics was the topic of several presentations including the panel pictured, Les Turner shared their online resource focused on Genetic Counseling and Testing, and there was Gene-Specific Education from End the Legacy.

The Path to Pre-symptomatic ALS/MND Trials Panel Discussion with speakers Michael Benatar (USA), Jean Swidler (USA), Stephanie Fradette (USA), and David Taylor (Canada) was the focus on the ALS/MND Connect Session this year. It was live streamed on fb and the link can be found here. 

We are pleased to note that Evy Reviers (Belgium) received the Humanitarian Award for her work with the ALS Liga Belgium, EUPALS, and the International Alliance. Well done!

Many other connections, collaborations and friendships were shared over the two days that we will know will continue throughout the year. #ALSMNDWithoutBorders #AWorldUnitedAgainstALSMND

Check out our Instagram/Facebook reel from the Alliance Meeting here!

International Symposium on ALS/MND 2024

The International Symposium on ALS/MND took place over three days, December 6-8, 2024, in Montreal, Quebec, Canada. This year's symposium started with a Welcome from Dr. Ammar Al-Chalabi (UK) and Brian Dickie (UK) of the Motor Neuron Disease Association (MNDA) and from Tammy Moore (Canada) CEO of the ALS Society of Canada.

Terry Heiman-Patterson and Sara Feldman attended the International Symposium in person and Sara co-chaired the Clinical Session on Improving Clinical Management with Dr. Christian Lunetta (Italy). 
Watch our International Symposium Instagram/Facebook reel here.

We are pleased to note that the genetic ALS community had a strong presence at the International Meetings both in-person and virtually. Throughout the week of meetings, End the Legacy members Jean Swidler, Cassandra Haddad, and Mindy Uhrlaub gave presentations, participated in panel discussions and presented posters. It is amazing to see how far this amazing group has come in such a short time!

The Veteran ALS Action Committee had two posters displayed at this year’s event, and the VAAC was well represented by Mandi Bailey, Jill Brattain, Tim Abeska, and Katrina Byrd. The ALS Hope Foundation is honored to support this group.

2024 Northeast ALS (NEALS) Consortium Meeting

The 23rd Annual Northeast ALS Consortium (NEALS) Meeting was held October 21-23, 2024, virtually this year due to the effects of two hurricanes in a row. The ALS Hope Foundation is honored to support this important meeting and to have several members of the clinic and lab presenting posters and participating online. MDA ALS Center of Hope

Day 1 started with an introduction from the co-chairs, Dr. James Berry and Dr. Jinsy Andrews. The afternoon session from the Research Ambassadors giving the lived experience perspective was a much anticipated panel discussion and exceeded expectations!

Day 2 included the poster presentations, committee updates, the scientific symposium, and the industry roundtable. There were 12 posters with involvement from our clinic team members, lab team, collaborators, and/or ALSHF! See the posters below this write up.

Day 3 started with a focus on Brain Computer Interfaces and a look at where they are now, a discussion on using Artificial Intelligence for voice banking, Team Gleason was honored with the Gupta Family Award, and Dr. Heiman-Patterson chaired the final platform presentations.

Posters:

The use of remote digital technologies which would enable remote monitoring of progression for clinical care and trials is a topic of great interest. We had three posters in this area, Pison device capture of muscle activity (below left), a machine learning model for predicting dysarthria (below center), and the Capture Proof AI enabled video capture (bottom right). Authors included Terry Heiman-Patterson, Lyle Ostrow, John Furey, Sara Feldman, Donna Harris and Zachary Biles.

There is also a strong interest in aspects of care for individuals with ALS. Team members were involved in looking at developing a National ALS Home Health Medical Standard Consensus Guidance document (below left), costs of the multidisciplinary clinic (below center), use of a self-administered survey to detect neurodegenerative diseases including ALS (below right), and a clinical prediction tool for respiratory insufficiency (right). Team authors for these include Terry Heiman-Patterson, Sara Feldman, Luisa Enriquez and Latoya Weaver.

The two posters described ways to predict the course of ALS using the change in the ALSFRS-R from disease onset to time of diagnosis (below left) and the King's Clinical Staging System (below center), both using data from the ALS/MND Natural History Database. Authors Terry Heiman-Patterson, John Furey, and Larisa Ibric. Mandi Bailey and the Veteran ALS Action Committee were honored to share their findings on Veterans participation in clinical trials (below right).

We also presented work from the Dr. Robert Sinnott Research Lab (far right) and work from the NEALS Biorepository (near right). Authors include Frank Bearoff, Meghan Smith, Terry Heiman-Patterson and Lyle Ostrow.

It was an excellent conference overall with 3 amazing days of presentations, panels and posters! We are hopeful that it will be in person next year but are thinking of all of those impacted by the hurricanes. Looking forward to more updates on the research no matter where we are!

17th Annual Hope Walks for ALS Family Fun day

The 17th Annual Hope Walks for ALS Family Fun Day was held on a windy and overcast Sunday, September 29, 2024 at Temple’s Ambler Campus but what a beautiful day it turned out to be!!

With over 800 registrants, we raised over an astounding $161,000 to support ALS research and patient care!

This year, we had the honor of celebrating Mike O'Brien as our walk chair. Mike is a proud father of four girls as well as a veteran living with ALS. Mike and his wife Diane, the inspiration for OB'S ARMY, shared his powerful story, reminding us all of the importance of our mission and the impact of the support we provide.

Bob Spurrier was the Top Individual Fundraiser and Team PK was the Top Team! Way to go!

The event was filled with families and friends enjoying a roll or stroll through Temple Ambler’s beautiful gardens, fun activities and games for the kids, and raffles with amazing prizes.

Our clinic staff were on site to thank everyone for their support and cheer everyone on! The sense of hope was evident throughout the day creating a truly unforgettable experience.

We are so incredibly grateful to everyone who participated, donated, sponsored and volunteered. Your unwavering support makes a real difference in our fight against ALS. Together, we can make a difference and bring hope to those affected by ALS.

Missed this year’s walk? Don’t worry! Visit here to see photos from the day!

20th Annual Research Update and Awards Luncheon

The Research Update and Awards Luncheon was held on Sunday, May 5, 2024 11:00AM-2:30PM at The Inn at Villanova

Thank you to everyone who joined us for an excellent day of education, information, and awards!

Dr. Jinsy Andrews, Co-Chair of the Northeast ALS Consortium (NEALS), Associate Professor of Neurology in the Division of Neuromuscular Medicine and the Director of Neuromuscular Clinical Trials at Columbia University in New York City, gave an excellent presentation describing the current state of ALS Clinical Trials with an intriguing look to the future.

Dr. Terry Heiman-Patterson provided an update on the work of the ALS Hope Foundation and the Dr. Robert Sinnott Research Lab. She then took a moment to thank Dr. Guillermo Alexander, upon his retirement, for for his over 25 years of research on ALS.

We then took the opportunity to say Thank You to people or organizations that have supported the ALS Hope Foundation, especially over the past year.

International Symposium on ALS/MND

The International Symposium on ALS/MND was held in-person for the first time in 3 years, December 6-8, 2023. The symposium started with a welcome from Tanya Curry (UK) and Dr. Ammar Al-Chalabi (UK) of the Motor Neuron Disease Association (MNDA). 

Dr. Terry Heiman-Patterson attended the meeting virtually and summarized some of the new strategies that are being developed for new and more effective treatments of ALS/MND.

First, I will summarize some of the new strategies that are being developed for new and more effective treatments. Oxidative stress is an important target for treatment of ALS. Certainly, we already have one therapeutic agent, Radicava, that is aimed at scavenging the free radicals produced by oxidative stress that damage the cells. Investigators presented a different strategy—targeting the mechanisms that regulate the production of free radicals through the protective enzyme PDI, which acts to reduce the clumping (misfolding) of proteins and inhibit oxidative stress. Investigators have created novel molecules containing the active region of PDI and showed that these molecules were protective in cell and animal models.

Another strategy by Athira Pharma is examining the potential of small molecules modulating the neurotrophic hepatocyte growth factor system, which is neuroprotective and anti-inflammatory. One of these molecules, ATH1105, showed both independent and synergistic improvement in disease in a mouse model of ALS.

Another target discussed was the clumping of the protein TDP43, an important regulator of RNA processing to produce protein. TDP43 is found in the nucleus of cells in normal circumstances, but in ALS, it moves from the nucleus to the cell cytoplasm, where it clumps (aggregates), a finding in virtually all ALS neurons. It is hypothesized that the clumps may drive inflammation and degeneration. While there are trials designed to increase the clearance of these clumps, other strategies to reduce the amount of clumping are being developed.

One group of investigators showed that a small piece of a protein that regulates TDP43 improves function and survival in a fly model and mouse model of ALS. Other strategies are directed at ways to clear the clumped TDP43 through increases in a process called autophagy. These molecules showed improved survival in culture models. Other investigations focused on the loss of function that occurs when TDP43 moves out of the nucleus. Nuclear TDP43 is responsible for piecing together RNA properly to code for proteins. However, when it moves out of the nucleus, RNAs are not properly put together (this is called mis-splicing). The resultant proteins are not properly made and do not function as they should. Two important proteins that are affected by mis-splicing in ALS are UNC13 and STMN2. There are now genetic techniques being used to correct the RNA mis-splicing of STMN2 in trials, and we heard about a similar strategy to correct UNC13 RNA mis-splicing in cultured neurons. Finally, we heard about genetic strategies to alter the aggregates of small proteins that occur with the C9ORF72 expanded repeat, the most common genetic cause of ALS.

 Of course, everyone is interested in the results of the clinical trials that were reported at the meetings. We heard some additional results from Valor, the trial of Tofersen (Qalsody) in SOD1 ALS. Qalsody is an antisense oligonucleotide that blocks the production of SOD1 and was recently approved by the FDA. Participants were allowed to continue the drug in an open label phase that has continued for up to 3 years. This has enabled additional observation of the efficacy. This data demonstrated a significant reduction in the neurofilament levels which reflects reduced neuronal degeneration as well as continued slowing of decline that continued over the period of analysis. Additionally, in a subset of participants there was an unexpected improvement in strength. Overall, there was improved survival, and for the rapid progressors, survival was extended by 1.6 years.  

Promising results were reported on pridopidine, a small molecule that stimulates the Sigma 1 receptor and provides a neuroprotective effect. The phase 2 HEALEY ALS Platform Trial of pridopidine demonstrated it was safe and well tolerated. There were significant speech improvements compared to placebo, especially in speaking rate and articulation. Additionally, in the subgroup of participants with definite or probable ALS, early disease or fast progression, there were beneficial effects on measures of progression. This will help to design an upcoming Phase 3 trial.

Co-ALS, a phase 2 trial of colchicine (an already-approved drug that has an anti-inflammatory effect reduces clumping of proteins (TDP43) in cultures) demonstrated a decrease in the decline of function measured by the ALSFRS-R over 30 weeks, supporting further study of colchicine as a therapeutic agent. MIROCALS demonstrated a reduced risk of death when IL2, an anti-inflammatory molecule that drives T-regs (protective white blood cells), was administered with riluzole in a group of newly diagnosed ALS participants. Finally, we heard about apilimod dimesylate (AIT-101), which is directed at improving the clearance of clumped proteins including TDP43 through inhibition of an enzyme PIKfyve, which in turn causes the increase in a protein TFEB that increases the degradation of clumped proteins. This small trial in people with the C9ORF72 genetic variant of ALS demonstrated that AIT-101 was able to penetrate the brain and hit its target, with an increase in the activity of TFEB. This success will enable this molecule to move forward into Phase 2 in both sporadic and genetically-caused ALS.

While everyone has their focus on drugs that modify disease, we are learning more about the important role nutrition plays. Recently, I mentioned a paper that demonstrated omega 3 fatty acids could slow progression of ALS. At the meetings, a study of diet demonstrated that people living with ALS who consumed a diet that had a high glycemic index had slower disease progression and longer survival. The glycemic index reflects how much the sugar levels in your blood rise after eating a particular carbohydrate and is measured relative to pure glucose. Examples of high glycemic foods include white bread, rice, potatoes, and sugary soft drinks. This study underscores the need to maximize the interventions that are available now, including nutrition and respiratory support.

Sara Feldman, PT, DPT, ATP, attended the meeting in-person and co-chaired the Clinical Session on Improving Clinical Practice along with Dr. Mieko Ogino (Japan). 

She shared her thoughts on the Clinical Sessions: The first session focused on respiratory management in ALS/MND. Interesting presentations included Andrew Geronimo (USA) presented on the use of home monitoring respiratory status and how the use of home spirometry can give an early indication of need for Non-Invasive Ventilation. M Grassano (Italy) presented his work showing that Non-invasive mechanical ventilation slows the disease progression in ALS/MND. The NIV provides the support the motor neurons need.

Dr. Heiman-Patterson’s work was cited in the presentation.

The next session was on Technology and Telemedicine and there were several discussions on the use of digital technologies for home-based assessments, both in the clinical and research areas. An interesting talk on the use of a self-explanatory ALS Functional Rating Scale (ALSFRS-R-SE) from A Maier (Germany) has many people debating the ALSFRS-R scale’s utility.

The final session of the day was on Caregivers and Families. The presentations on Informal Caregiving from M Galvin (Ireland), exploring parents’ and children’s perceptions of the communication around ALS from M Sommers-Spijkerman (Netherlands) and factors predicting anticipatory grief, A Trucco (UK) highlighted the difficulties families face around the globe.There was also a common thread that it is necessary to keep communication open and ongoing. None of these decisions are a one time discussion!

The next morning started with Nutrition Assessment and Management. Dr. Heiman-Patterson discussed the higher glycemic diet already from I Lee (USA). K Tran (USA) presented on the decision making process surrounding feeding tubes. There was also an interesting presentation from J Chang (Australia) indicating alterations in the brain’s response to food stimuli may impact appetite.

Improving Clinical Practice was next, which I had the honor of chairing with Dr. Ogino. H McDonough (Ireland) and A Puchades (Spain) presented their work on mapping the natural history of ALS/MND in Europe. J Roggenbuck (USA) presented on Evidence-based consensus guidelines for ALS genetic testing and counseling, work which is a hot topic right now and incredibly important.

The final morning began with Clinical Management and a new look at standards of care given the state of multiple treatments and options by C Ingre (Sweden), which included Assistive Technology. M Cavanaugh (USA) brought up the incredibly important topic of how to break the news, helping to educate physicians and allied health professionals on how to have difficult discussions with patients. A Genge (Canada) and Cathy Cummings (Canda) participated in this presentation.

The Symposium ended with awards, a scientific update, and a look forward to next year’s meeting in Montreal. Overall, it was a fantastic week of reconnecting with old friends and making new ones. The air of friendship and collaboration was palpable in the room. We all left with new knowledge and renewed commitment to whatever aspect of work surrounding ALS/MND we are in.

International Alliance of ALS/MND Associations Meeting and Allied Professionals Forum

The International Alliance of ALS/MND Associations held their Annual Meeting December 2-3, 2023 in Basel, Switzerland with representatives from 26 different countries in attendance. The ALS Hope Foundation has been an active member of the Alliance since 2001 and this year Jamey Piggott and Sara Feldman were honored to attend the meeting in person.

Calaneet Balas (USA), Chair of the International Alliance, welcomed the attendees, then the programming began with a tribute to people living with ALS/MND around the globe with the March of Faces video. Special thank you to Wendy Hendrickson and Greg Heydet who represented the ALS Hope Foundation.

See the video here.

Program chair and PLWALS Gudjon Sigurdsson (Iceland) and Cathy Cummings (Canada), Executive Director at the International Alliance, welcomed everyone and kicked off the presentations from members around the globe, a few we will highlight below.

We heard from several organizations with updates on their work over the past year including FightMND, Asociación ELA Argentina, MNDA, the ALS Association, Spain and MNDA Scotland, all of whom shared insights and experiences that may be applicable to other organizations. Several shared specific challenges and how they are meeting them, including Colombia’s partnership with ALSA for communication devices and Dr. Melinda Kavanaugh (USA) presented on the Global Neuro Y Care program.

Several support programs were presented including End the Legacy’s peer support for the Genetic ALS/FTD community, Denmark’s support interventions targeting people with ALS and their caregivers, and Israel’s support groups. Guides to help people navigate ALS were presented including the Dutch ALS Guide, My ALS Journey from ALSA, and the ALS/MND Clinic Locator. We heard about the life of a person living with ALS in Japan and went through her day with her; we heard from Alper Kaya (PALS/Turkey) about the earthquake in Turkey and how it affected PALS; we heard about differences in research and treatment access in different areas of the globe; and we heard the voice of people living with ALS/MND and their caregivers woven throughout the two days.

The PALS and CALS Advisory Council, or PCAC, provided an update on what they have accomplished over the past year, with a focus on the Alliance’s Fundamental Rights Survey. PCAC Chair, Sara Feldman (USA), Gudjon Sigurdsson (PALS/Iceland), Ajay Gupta, (CALS/India), Bruce Virgo (PALS/Scotland) and Paula Trefiak (PALS/Canada) were in-person; Angelique van de Lit (PALS/Netherlands), Albert Koo (PALS/Malaysia), Norman MacIsaac (PALS/Canada), Felipe Ocampo (PALS/Colombia) and Orlando Ruiz (PALS/Colombia) were virtual.

The Humanitarian Award was announced during the meeting, and we were pleased to learn that Dr. Lucie Bruijn received the award.

A highlight of the meeting was the preview of the animated film, Luki and the Lights, which tells the story of a robot with ALS.

Following the conclusion of the Alliance Meeting, the ALS/MND Connect took place with amazing speakers Dr. David Taylor (Canada), Dr. Nick Cole (UK), Dr. Kuldip Dave (USA), Dr. Ammar Al-Chalabi (UK), Dr. Gethin Thomas (Australia), virtually Dr. Andriano Chio (Italy) Dr. Jeanine Heckmann (South Africa) and Dr. Nadia Sethi (CALS/USA) from the Scientific Advisory Council, or SAC and moderated by Allison Bulat (CALS/USA).

There were presentations followed by a discussion/question period. The video can be viewed here.

It was both informative and inspiring to learn from other organizations around the globe.

#AWorldUnitedAgainstALSMND #ALSMNDWithoutBorders

The Allied Professionals Forum, or APF, co-chaired by Sara Feldman (USA) and Rachael Marsden (UK), then took place over the following two days. The APF brings together allied health professionals from around the globe and this year over 400 people joined both in-person and virtually. We were warmly welcomed virtually by PALS and CALS Advisory Council (PCAC) members, Felipe Ocampo (Colombia), Paula Trefiak (Canada), Gudjon Sigordsson (Iceland), Alper Kaya (Turkey), Bruce Virgo (Scotland), Angelique van der Lit (Netherlands), and Orlando Ruiz (Colombia) who thanked all of the allied professionals in attendance for the work they do and the care and compassion they do it with. It was a heartfelt reminder to everyone in the room why we do the work we do.

Sarah Solomon (Australia) and Helen Carey (USA), both Occupational Therapists, started the APF with a provocative discussion on Embracing Risk: Enabling doing for people living with ALS/MND, which set the stage for the next two days. There was a real sense of how can we, as health professionals, stretch beyond our usual boundaries and enhance the lives of people with ALS and their caregivers.

The Luncheon this year was sponsored by the ALS Hope Foundation, and Jamey Piggott, Executive Director of the ALS Hope Foundation, thanked everyone for their work and participation. He reflected that the audience lived by Dr. Dale Turner's words, "Extend Yourself."

Donna Harris, the Speech Language Pathologist at the MDA/ALS Center of Hope, joined us for the APF. She was pleased to note there was a presentation on oral care.

Dr. Deirdre Murray, a Research Fellow and Clinical Specialist Physiotherapist in Neurology at Beaumont Hospital, Dublin, was the recipient of this year's Allied Health Professional Award for 2023. This award was initiated in 2018 and recognizes recognize an individual committed to providing exceptional care to people with ALS/MND and the sharing of this knowledge.

We also managed to get all of the previous awardees together for the first time!

The APF was an amazing meeting over two days with excellent presentations on all aspects of care as well as a look to the future of technology and the addition of experiential presentations. Here are some of the highlights of the two days:

There was an excellent presentation from G Gisladottir, G Serrero, and F Mancuso (Iceland) on Eating with Dignity. They take food and puree it to the appropriate consistency for the person, then shape it in molds so it looks like actual food. And it tasted delicious!

There were exciting updates to assistive technology including J Chudge and W Gregory’s presentation on using AI and gaming for quality of life; updates to message banking and where the state of the art is by J Costello (USA) and also N Jackson (Australia); the use of eye-gaze to drive a power wheelchair by L Hills, M Hevicon, and S Fielden (UK); Experiential Apps were demonstrated by R Cave (UK) which included amazing software for making digital art and voice recognition for people with significant difficulty speaking/dysarthria. We will be watching for those to come out!

Several rehab focused presentations were done including an excellent one on ALS and Exercise by C Radcliffe (Australia) and one on healthy teeth and oral care that Donna Harris was pleased to see by M van der Linden (Amsterdam). S Brumby (Australia) gave both a heartfelt and informative presentation on choosing NIV, what the person needs to know ahead of time. One of the PALS in the room said it was the first time he had heard it put that way, and thanked her.

We also heard from D Meissner (USA), our friend at Your ALS Guide on their most recent work. Jamey also commented on the Expanding Access to Genetics Education, Counseling, and Testing: Innovations in ALS Genetics Care by P Wicks (UK), L Dratch and L Webb (USA). M Kavanaugh (USA) told us more about how children are experiencing caregiving. Really, there were so many excellent presentations, it is hard to highlight just a few!

It was a fantastic week of reconnecting with old friends and making new ones. The air of friendship and collaboration was palpable in the room. We all left with new knowledge and renewed commitment.