NEALS Meeting 2023

The 2023 Annual Northeast Amyotrophic Lateral Sclerosis Consortium® (NEALS) Meeting was held in Clearwater, FLA this October. We had a wonderful time where we heard about some of the exciting work going on in ALS and met up with colleagues from across the United States all of whom are focused on making progress in understanding and treating ALS. It was great to bring the team along including our student researcher Zach Farber and our neurology resident researcher Hannah Henderson.

Before the official start to the meeting, there are educational events that that take place, including the Clinical Learning Research Institute (CRLI). Dr. Heiman-Patterson is a member of the CRLI faculty and plans to hold a regional CRLI in the Philadelphia area this Spring.

The Outcome Training for clinical trials, including refresher sessions on the Healey Platform Trial, was also part of the pre-conference education and Kathleen Hatala, Sara Feldman and John Furey attended these sessions.

The topics throughout the meeting included General Sessions, Updates, Science Symposium: Gene Therapies of the Future and the Hot Topic: Advancements of Neurofilament as a Biomarker. A lot of great discussions! 

There was a fantastic session from the Global Research Ambassadors, "Real People, Real Impact," which was presented by people with ALS, pre-symptomatic carriers, and surviving caregivers. It was truly impactful!
Jean Swidler, End the Legacy, highlighted Dr. Heiman-Patterson as a difference-maker in her advocacy. 

We were privileged to present some of the work from the ALS Center of Hope highlighted in the posters below.

ALS Natural History Database Supports the

Common Plateaus in ALS

Using the large Natural History Database, we confirmed the finding that there are plateaus in ALS. Ten centers contributed to this database including the ALS Center of Hope and many of our PLWALS are included in the data. In 1021 PLWALS in whom we captured 3 or more clinic visits, 21.5% of PWALS plateau for 6 months while 10.8% plateau for a year and 6.5% for 18 months.

Healthcare Disparities in ALS

Using data from the Natural History Database, we examined healthcare disparities in ALS.  In this initial study there was no difference between Black PLWALS and White PLWALS in regards to the time to diagnosis or survival. However, the ALSFRS and FVC at diagnosis was significantly lower in Black PLWALS relative to White PLWALS and the average age of onset lower. Additionally fewer Black PLWALS received feeding tubes at the clinically recommended time and fewer were on Radicava.

Characterizing Patient Preferences, Satisfaction & Quality of Care Across Healthcare Models in ALS

This study demonstrated that people living with ALS and their caregivers prefer multidisciplinary care and seeing all of the professionals with each visit. Additionally we found that this care setting adheres most to the quality of care measures for ALS outlined by the Academy of Neurology.

Oxidative Enzyme Analysis on ALS Subtypes: Implications for Edaravone treatment, an interim analysis

Dr. Jeffrey Rosenfeld from Loma Linda University Health presented on our collaborative work from the Dr. Robert Sinnott Research Lab.

One thing is certain we always come back with new ideas and renewed enthusiasm.

2023 5th Annual Run for Hope 5K

What a beautiful morning for the Run for Hope 5K!

Thank you to the impressive runners!

Thank you to our awesome sponsors!

Thank you to all of our amazing participants, teams, supporters, volunteers and donors for making the 5th Annual Run for Hope 5K such a success!
It was incredible to see how we all came together to raise funds and awareness for ALS! 

Thank you to the fabulous teams! Thank you to our hardworking volunteers!

Check out the race results from the Bryn Mawr Racing Company here!

And their photos!

Check out more pictures from the day here!

And most of all, Thank You from the Run for Hope 5K Committee!

Go to the Run Website here.

2023 Gala of Hope

Thank you for making the ALS Gala of Hope so spectacular!

It was a beautiful evening of inspiration, motivation and HOPE! There are not enough words to express our sincere awe and gratitude to everyone that came out to support the ALS Hope Foundation. The evening was truly a success due to all of you, and we are deeply grateful for your support and generosity.

Our heartfelt thank you to Dr. Stacy Lewin Farber, the keynote speaker, who enlightened us on the true meaning of hope, as she shared her journey with ALS. Her story made us both laugh and cry....but left us knowing that she truly is a force to be reckoned with. We haven't heard the last from her!

A huge thank you to Joel Goldhirsh, our virtual guest speaker. Though he was not able to attend in person, his presence filled the room as he shared his battle with ALS and how it may control his body, but that he refuses to let it control his mind. Through humor and directness, he shared his mission with all of us.

Thank you to David Meissner for speaking about Joel and Your ALS Guide and sharing this important resource for healthcare professionals, patients and caregivers. 

Thank you to all of our sponsors, many of whom have been on-going supporters of the ALS Hope Foundation. Many thanks to all who donated the raffle and auction items, as your generosity truly added to the evening festivities. Thank you to the staff at Vie who admirably managed a room full of 300 guests with ease and professionalism. The food was delicious, the room was gorgeous, and every one felt welcome and taken care of. 
And of course, a huge thank you to our 5th Annual Gala of Hope committee members and volunteers who worked tirelessly to ensure every moment leading up to the event and the event itself was a gleaming success. 

A sincere thank you to each and every one of YOU who attended the event, bought a raffle ticket, bid on a Silent Auction item, or donated. We hope you all left knowing that you have made a difference in the work that we do.
 
And finally, thank you to the people and their families who are living with ALS, or who had a loved one with ALS, who came. We love and admire you and appreciate your presence. You are why we do what we do. 

These photos are courtesy of Lafayette Hill Studios, follow this link to see more!

International Symposium on ALS/MND Review

The International Symposium on ALS/MND was held virtually December 6-9, 2022. This year's symposium started with a Welcome from Dr. Brian Dickie (UK) and Dr. Ammar Al-Chalabi (UK) of the Motor Neuron Disease Association (MNDA). 

The meeting took place over three days and was a mix of video presentations and posters. During the symposium the results of several clinical trials were presented and Dr. Heiman-Patterson's review of four of these, RNS60, low dose IL2, CNM Au8, and Toferson can be found here.

First the 24 week trial RNS60 which functions as a neuroprotective and anti-inflammatory molecule examined biomarkers of inflammation and neurodegeneration along with the ALSFRS, FVC breathing test, quality of life and survival. In the 147 pALS enrolled there was a decrease in the decline in FVC in the participants treated with RNS60. There was no change in the biomarker of neuronal degeneration (Nfl). There was no difference in global function or survival although this trial was not powered to detect such a difference.

The second trial that was presented was low dose IL2 which acts as an anti-inflammatory through its activity on Treg cells. The primary objective of this trial was efficacy and safety over 18 months along with the effects on Treg. The results demonstrated an increase in Tregs along with a decrease in the risk of death when adjusted based on the phosphorylated neurofilament in the spinal fluid the effect was significant and increased to a 73% decrease in survival. The benefit was greatest in pALS with the lowest levels of phosphorylated neurofilament

The long awaited results of the Rescue ALS trial of CNM Au8 were also presented. This agent improves energy production and supports mitochondria. In this 36 week trial 45 pALS were randomized to treatment and there was a slowing in the decline of the ALS FRS score along with improved quality of life. In addition, there was a decrease by 70% in all cause mortality in those pALS treated both in the study and the open label period compared to those who were initially on placebo. Previously, CNM-Au8 was evaluated in the Healey Trial over 24 weeks, but did not meet its primary and secondary end points; however, the therapy did show survival benefit on exploratory analyses. Additional phase 3 studies are under consideration.

Finally there was additional data presented on Toferson from the Phase 3 trial. This agent is a splice modulating oligo that blocks SOD1 and has been developed to treat familial ALS associated with a SOD1 mutation. It was demonstrated that SOD1 protein is decreased by 8 weeks on treatment and remains decreased while the marker of neurodegeneration, NFL, shows a decrease by 12 weeks. Further, when the levels of NFL are considered, treated pALS demonstrated improvements in the decline of ALSFRS, strength measures and vital capacity as well as quality of life during the extended open label phase. This is important because it suggests that there are biological effects that occur prior to the clinical improvements. Toferson is now being evaluated for approval by the FDA.

Dr. Rick Bedlack (USA) received the Forbes Norris Award and Dr. Heiman-Patterson commented: “I was extremely happy to hear that this year’s winner of the Forbes Norris Award was Dr. Richard Bedlack. This prestigious international award is named for Dr. Forbes “Ted” Norris, a neurologist who dedicated his career to helping people with ALS/MND, and it is awarded annually to a neurologist who provides compassionate excellence in care and has contributed to the understanding and treatment of ALS/MND. Dr. Bedlack is extremely deserving of this recognition and I am proud to work with him on our Clinical Research Learning Institute and ALS Untangled.”

International Alliance of ALS/MND Associations Meetings

The International Alliance of ALS/MND Associations held their Annual Meeting November 28-30, 2022 in San Diego, California this year with 241 representatives (136 in person/105 virtual) from 21 different countries. The ALS Hope Foundation has been an active member of the Alliance since 2001 and this year Dr. Terry Heiman-Patterson, 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. See the video here. Special thank you to Stacy Lewin Faber and Will Steuber who represented the ALS Hope Foundation.

 Program chair and PALS Gudjon Sigurdsson (Iceland) virtually welcomed everyone and kicked off the presentations from members around the globe, a few we will highlight here.

Dr. Melinda Kavanaugh (USA) presented on the work she and her colleagues from South Africa, (Dr. Andre Mochan, Sheila Slabber and Prof. Thobeka Nkomo) are doing on developing young carer education and support programs. Dra. Tatiana Mesquita e Silva (Brazil) presented the work of ABRELA over the past two years and shared their “Clinical Protocol: Therapeutic Guidelines” they recently published. Dr. Hemangi Sane (India) presented the results of an online survey Asha Ek Hope had done on the impact of Covid 19 infection and vaccination on people with ALS/MND in India.

The ALS Association presented on using GIS to locate clinics, strategies to increase the numbers of clinical trials, and the CDC Registry. Learn more about the Registry and sign-up here.

The influence of the voices of people living with ALS/MND on advocacy in many areas was presented by several organizations including ABRELA, MNDA, ALSA, ALS Canada and MNDA Scotland. Alper Kaya (PALS/Turkey) also presented on how they were able to use the results of the Alliance’s Fundamental Rights Survey to influence the Turkish government to make changes in care, including starting a multidisciplinary clinic.

We then heard updates from the Scientific Advisory Council, or SAC, which included an excellent panel discussion by chair Dr. Gethin Thomas (Australia), Dr. David Taylor (Canada), Dr. Kuldip Dave (USA), and virtually Dr. Andriano Chio (Italy). Updates from the Innovation and Technology Advisory Council, or ITAC, was provided by Phil Green (PALS/USA) and virtually Jarnail Chudge (UK) on their focus on Voice Banking. Finally, the PALS and CALS Advisory Council, or PCAC, provided their updates. PCAC Chair, Sara Feldman (USA), Phil Green (PALS/USA) and Felipe Ocampo (PALS/Colombia) were in-person, Angelique van de Lit (PALS/Netherlands) and Bruce Virgo (PALS/Scotland) were virtual.

We also heard from Japan, Belgium, Switzerland, USA, Canada, Scotland on various programs, organizational development, advocacy, and clinical care their organizations are working on or providing. Dr. Heiman-Patterson (USA) presented on the ALS Hope Foundation’s Hope Bridges program, giving other organizations the blueprint to develop a similar program of their own.

The Humanitarian Award was announced during the meeting, and our friend Sally Light, the retiring CEO of the MNDA (UK) received the award.

Following the conclusion of the Alliance Meeting, the ALS/MND Connect took place with speakers Phil Green (PALS/USA), Dr. Eugene Brandon (USA), Dr. Colleen O’Connell (Canada), Dr. Angela Genge (Canada), and Dr. Caroline Ingre (Sweden).  

The Luncheon this year was sponsored by the ALS Hope Foundation, Fundela, and Les Turner ALS Foundation and we heard from speaker Dr. Colleen O’Connell (Canada) on “The Right to a Quality of Life.” Jamey Piggott, Executive Director of the ALS Hope Foundation, welcomed everyone to the luncheon and quoted Walt Whitman to encourage their participation. "Be curious, not judgemental." 

 Following the luncheon, the Allied Professionals Forum, or APF, co-chaired by Sara Feldman (USA) and Rachael Marsden (UK), began. The APF brings together allied health professionals from around the globe and this year people joined both in-person and virtually. We were warmly welcomed virtually by PCAC members, Phil Green (USA), Gudjon Sigurdsson (Iceland), Bruce Virgo (Scotland), Felipe Ocampo (Colombia) and Alper Kaya (Turkey) who thanked the allied professionals in attendance for the work they do and the care they put into it.

Then launched a panel on the history and evolution of the multidisciplinary clinic. Dr. Heiman-Patterson (USA) and Dr. Orla Hardiman (Ireland) joined Sara Feldman (USA) and Rachael Marsden (UK) on the panel, which was moderated by Dr. David Taylor (Canada).

Donna Harris, the Speech Language Pathologist at the MDA/ALS Center of Hope, joined us for the APF, and while there the SLPs held an impromptu international meeting. And Speech Language Pathologist Richard Cave (UK) was the recipient of this year's Allied Health Professionals Award! He is a champion for making voice banking accessible to everyone.

The day ended with an amazing global Walk to Defeat ALS/MND. 

The programming then continued over the next two days with excellent presentations on all aspects of care. The attendees were both in-person and virtual and were enthusiastic and engaged. See the full program here.

Highlights:

There were several presentations on resources available to clinicians and PALS/CALS including David Meissner (USA) presenting on Your ALS Guide and Anne Marie Doyle (USA) on Les Turner ALS Foundation’s My Decision Making Tool.Guidelines for healthcare professionals working with people with complex communication needs was presented by Lisa Bruening and a Rapid Access Communication Kit by Alisa Brownlee, both USA/ALSA.

 Models of support for programs or equipment included the Wheelchair Alliance presented by Nick Goldup (UK); the AISLA model for providing AAC in Italy by Massimiliano Filosto (Italy); establishing a neuro-palliative care clinic by Gargi Kumar (India); and an ALS Canada support group model by Kim Barry (Canada).

Two fantastic presentations involved AT in creativity including DuoRhythmo by Ivanyi Balazs (Denmark) and making videogames more enjoyable for people with MND by Ben O’Mara (Australia). 

In the section on Multidisciplinary care we learned about "Positive Tiredness": A Concept to challenge fatigue management for people with ALS/MND from OT Helen Carey (USA); Eating with Dignity from Gudlag Gisladottir (Iceland); neck weakness impacting walking from Stephanie Zhao (Australia); understanding bowel habits from Jan Clarke (UK); and sex and intimacy from Joanna Nunez (USA). Voice banking was presented by our APF awardee, Richard Cave (UK) as well as Rachel Robinson (Australia).

Two networking/support groups for allied health professionals or researchers were presented, showing the need for ongoing education and sharing of knowledge. MND professional’s community of practice by Jennifer Bedford (UK) and the research staff satellite meeting by Tommy Bunte (Netherlands).

Mental Health and Online support presentations included: Making sense: An online meaning centred psychological intervention for individuals with MND from Sandra De Moree (Netherlands); support systems for children, Lily Cola (USA); E-learning on end-of-life care, Liesbet Casier (Belgium); conversations about death and dying from Amber Johnstone (USA); and developing a web resource for individuals with inherited MND by Jade Howard (UK).

Allison Bulat (CALS/USA) gave an excellent presentation on Strengthening the Paradigm of Science and Patient Collaboration. 

 There was also a panel How to break the news in ALS/MND with Dr. Melinda Kavanaugh (USA), Dr. Angela Genge (Canada), Dr. Colleen O’Connell (Canada), moderated by the Executive Director of the International Alliance, Cathy Cummings (CALS/Canada) that stimulated discussions on breaking the news of not only the diagnosis but every difficult decision along the way.

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.

Research Update October 2022

There have been several announcements in the news recently in reference to treatments for ALS and we’d like to share them with you.

On September 30, 2022, the Food and Drug Administration approved Amylyx Pharmaceuticals Relyvrio (AMX0035) for the treatment of ALS. This drug is a combination of sodium phenylbutyrate and taurusodiol, two drugs that are synergistic and neuroprotective. The mechanism of action is directed at blocking cell death pathways that originate in the mitochondria and endoplasmic reticulum. The mitochondria are responsible for energy production and the endoplasmic reticulum is involved in protein production, modification and transport. When these cell components are damaged, as has been shown to happen in ALS, they cause oxidative stress and abnormal clumping of protein resulting in inflammation and cell death. In the Phase 2 trial (CENTAUR) along with the open label extension, AMX0035 slowed progression of disease and extended survival significantly. We will look forward to receiving information on what the procedure is to prescribe and when it will be available. We now have three drugs that can alter disease progression and affect survival in ALS—Riluzole, Radicava (IV and Oral), and Relyvrio. You may be asking which is the best and what should I take? The answer in short is all of them. ALS is a heterogeneous disease and there are multiple mechanisms that have been implicated to cause neuronal death. In any person one or another mechanism may be the predominant driver of disease but at this point, we do not have biomarkers or a way to tell in any one person what is driving the disease and thereby personalize the choice of medication or approach. These drugs each target a different mechanism –Riluzole likely targets glutamate excitotoxicity, Radicava oxidative stress, and Relyvrio cell death.

“Since we do not know in any individual which mechanism drives their disease—as long as there are no contraindications or adverse side effects, I would want to prescribe all three. I am so happy to be able to offer more hope to PALS at the MDA/ALS Center of Hope at Temple.”

Healey ALS Platform Trial

“Current therapeutic development in amyotrophic lateral sclerosis (ALS) relies on individual randomized clinical trials to test a specific investigational product in a single patient population. This approach has intrinsic limitations, including cost, time, and lack of flexibility. Adaptive platform trials represent a novel approach to investigate several interventions for a single disease in a continuous manner. Already in use in oncology, this approach is now being employed more often in neurology. Here, we describe a newly launched platform trial for ALS. The Healey ALS Platform Trial is testing multiple investigational products concurrently in people with ALS, with the goal of rapidly identifying novel treatments, biomarkers, and trial endpoints.”

Adaptive Platform Trials to Transform ALS Therapy Development.
Paganoni S, Berry JD, Quintana M, Macklin E, Saville BR, Detry MA, Chase M, Sherman A, Yu H, Drake K, Andrews J, Shefner J, Chibnik L, Vestrucci M, Cudkowicz ME; HEALEY ALS Platform Trial Study Group. Ann Neurol. 2021 Dec 21

It is because of this design that it is felt that we are getting answers to drugs effectiveness sooner. The results to date:

March 1, 2022 Zilucoplan arm stopped early for futility. The independent Data and Safety Monitoring Board (DSMB) recommended stopping the zilucoplan regimen in the HEALEY ALS Platform Trial after a pre-scheduled interim analysis demonstrated futility.

 Read the press release here.

September 29, 2022 Healey & AMG Center announces results from the HEALEY ALS Platform Trial with verdiperstat. Verdiperstat "did not statistically differentiate from placebo on the pre-specified primary efficacy outcome, disease progression measured by the ALS Functional Rating Scale-Revised and survival, nor the key secondary efficacy measures during the 24-week study period.”

 See the Webinar recording here.

October 3, 2022 Healey & AMG Center announces top line results in ALS platform trial with CMN-Au8. The primary endpoint of adjusted ALSFRS-R slope and key secondary endpoints of CAFS and SVC were not met at 24 weeks.

 Read the press release here.

“Thank you!”

To every person who participated in these trials, we say Thank You! The clinical Trial Team at the MDA/ALS Center of Hope at Temple sees the sacrifices you make, appreciate the time we spend with you, and consider you all heroes.

The ALS Chocolate Meltdown 2022

What a blast we had at the Chocolate Meltdown at the Adventure Aquarium in Camden, NJ on Friday September 23, 2022!!

The funds raised will provide support to the ALS Hope Foundation and Hope Loves Company!

This event would not be possible without Larry and Jill Kaplan, our volunteers, sponsors and YOU!

 Check out these photos to see what this fabulous event is all about: Lafayette Hills studio provided the photography.

See Dr. Stacy Lewin’s remarks here!

15th Annual Hope Walks for ALS raises over $90,000!

Sunday, September 18, 2022 was a brilliant, sunny day for Hope Walks for ALS! Check out more photos here!

Volunteers started showing up at 7:30AM to the Temple University Ambler Campus with enthusiasm and muscle, (fueled by coffee and donuts). The volunteers were awesome all day long and we could not have done it without them!

Walkers and rollers, families and friends, teams and pets arrived soon after! It was awesome to see the joy as people came together in-person again to support their loved ones. We thank each and every one of you!

Our Honorary Walk Chairs, Susan Lowry and Mark Stem, and their amazing families led the way!

We had a fun family festival area complete with a bouncy slide from Max and Ollies and face painter, Charmed Face Painting! DJ John Alexander from Eclipse Entertainment kept us all entertained and dancing!

Food was provided by Lee’s Hoagies, Pudges, Wise, Darryl’s Pastries, Rita’s Italian Ice and Ambler Pizza with support from Soleo!

We couldn’t have done it without our wonderful sponsors!

Watch for a video from 400 Foot Views coming soon!

And, of course the raffles were ridiculous! In a good way! Thank you to our raffle donors!

• Aim High Yoga

• Ambler Beverage

• Ambler Florist

• Ambler Sports Store

• Ambler Theatre

• Brick and Barrel

• Don’t Blink Boutique

• Philadelphia Eagles

• El Limon

• Fireside Tavern

• Philadelphia Flyers

• Guiseppes

• Hallmark

• Home by Kristin

• JuicePod

• La Frida

• LearningExpress

• LibertyCoke

• Longwood Gardens

• Margaret Treasures

• Pudges

• Philadelphia Union

We were thrilled to give back to the Temple University Ambler Campus with a donation for a new tree to help with the damage left behind from the tornados last year!

Thank you from the Center of Hope Team and Walk Committee!!

2022 4th Annual Run for Hope 5K

Thank you to everyone that attended the 4 th Annual Run for Hope 5K. It was a beautiful morning at Norristown Farm Park where over 160 participants ran or walked to help find more treatments and a cure for ALS. We made some amazing new memories and appreciate all the support we have from our participants! It was incredible to see how we all came together to raise awareness for ALS! With everyone’s support we were able to raise over $40,000 with a matching gift of $10,000 that will directly help the Dr. Robert Sinnott Research Lab.

This event would not have been possible without our founder Gary Kurtis who started this run 4 years ago to honor his father who lost his battle with ALS in 2002. We also what to thank the committee that have been planning this event for the past several months and all the volunteers that came out to help the event run smoothly.

This year we had 15 teams join us and each one brought so much strength and support for our mission. Thank you to our teams- AARC, Carr Pe Diem, Center of Hope, Erasing ALS one step at a time, Grandpa’s Team, HICKEY, In memory of Alan G Croasdale, Individual Runners, OTF Paoli, Pierson Powerhouse, Stem Construction, Team Adickes, TEAM MEG, Team Tkachuk, Together We Fight

Also, a big thank you to all of our sponsors for their support they help us make this day happen! Brainstorm Cell Therapeutics, Mitsubishi Tanabe Pharma America, Sales Tips 101, MDA/ALS Center of Hope, Bryn Mawr Racing Company, Brotherston Home Care, DiRosato Plumbing and Heating Company, Soleo Health, DJB Specialties, Home Transformations, Organic Remedies, McCaffrey’s Food Markets, Niagara, Wawa, and Star Printing.

Please check out all the photos on Flickr! Thank you to Bob Patterson, Larissa Ding and Ted Goldman for all of these amazing photos.