4th Annual Gala of Hope

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Thank you to everyone who supported our 4th Annual Gala of Hope, an evening of inspiration, motivation and hope. We are deeply grateful for everyone's generosity and were overwhelmed by the outpouring of support and love. We cannot adequately express our sincere appreciation to all of you. 

Our heartfelt thanks go to Officer Scott Davis, the keynote speaker. He shared his journey, his dreams and his focus. He reminded us that ALS does not play fair, but neither do people living with ALS. His strength and determination and emotional story had us all up on the podium with him, supporting him. In his words, he is not dying from ALS, he is living with it!

We were moved by the friends and family of Dr. Robert Sinnott who took on the challenge of doing something great in his honor after hearing him speak last year. Exceeding all expectations, the Dr. Robert Sinnott Research Lab Fund has raised over $150,000, ensuring the establishment of the lab and allowing the ALS Hope Foundation to push forward research towards a cure. Unbelievable! 
 
Additional thanks to emcee Stephanie Stahl from CBS3, who was an excellent host. 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's festivities. And, of course, a huge thank you to the staff at Vie who admirably managed a room full of 500 guests with ease and professionalism.
 
And most importantly, a sincere thank you to the people and their families who are living with or who have lived with ALS and came out to the gala. We know it is an immensely emotional and difficult night and your presence demonstrates a willingness to come out to advocate for and support the ALS Hope Foundation. You remain our inspiration as we continue to work hard and push forward our mission of care, education and research to make a difference.  

We hope to see you next year!

See our Gallery for more photos!

30th International Symposium on ALS/MND

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The 30th International Symposium on ALS/MND was held in Perth, Australia December 2019. This Symposium is put on annually by the MND Association of England, Wales, and Northern Ireland and is a full week of meetings, presentations, and posters. This event attracted over 800 attendees, including scientists, clinicians, organization leaders, and people living with ALS and their caregivers from around the world. Justin Kwan, MD, and Sara Feldman, DPT, ATP, spent the week attending the many scientific and clinical sessions and meetings.

Calaneet Balas (ALS Association, USA), Chairman of the International Alliance of ALS/MND Associations, opened the week of meetings on December 1, 2019, with the 27th Annual Alliance Meeting. The Alliance brings together ALS/MND organizations from around the globe, and ALS Hope Foundation has been a member of this international group since 2001. Dr. Sara Feldman completed her second term on the Board of Directors for the Alliance and stepped down this year. Andrea Blackman from the Les Turner Foundation in Chicago was elected to the Board to fill the opening, and we wish her all of the best. Dr. Feldman presented on the work of the PALS and CALS Advisory Council The program from the meeting can be found here.

The ALS/MND Connect (previously Ask the Experts) event is held on the second day of the meetings. This year’s panel included Professor Matthew Kiernan (Australia), Professor Ammar Al Chalabi (UK), Professor Merit Cudkowicz (USA), and was moderated by Professor Merrilee Needham (Australia). The event was live-streamed on social media, and the full video and PowerPoint slides are available via this link. 

The Patient Fellows Program began three years ago as a way to fund people living with ALS/MND to attend the Symposium and add their experiences to the discussion. View a few of their insights here.

The Allied Professionals Forum was held on December 3, 2019, this is a day-long session developed for Allied Health Professionals, and the ALS Hope Foundation has been a committed supporter for many years. Sara Feldman (ALSHF) is a co-chair along with Rachel Boothman (MNDA). The Program Agenda can be found here, and the presentations are on the Alliance website.

The 30th International Symposium on ALS/MND then took place on December 4-6, 2019. Sally Light (MNDA) opened the Symposium, and David Ali from MND Australia, this year’s hosts, welcomed everyone in attendance. It was three full days of presentations and posters. Please see the full program here. Sara Feldman presented two posters, “The Addition of Rotational and Adjustable Flexion Components to Cervical Support” and “Assessing Assistive Technology Use and Needs by Individuals with ALS/MND.” Dr. Feldman also co-chaired the session on Palliative Care with Dr. David Oliver (UK).

The International Symposium on ALS/MND is the largest medical and scientific conference specific to ALS/MND in the world. It brings together everyone, leading researchers, clinicians, PALS/CALS, and associations, who dream and believe that one day there will be a world without ALS/MND. We all leave invigorated with a renewed sense of purpose, inspired with new ideas, and driven with that ever-present urgency, for we know that for those living with ALS/MND, time is all too precious and short.

Platform Trial to Begin Enrollment in Early 2020→

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There is exciting news from the Healey Center for ALS at Massachusetts General Hospital responsible for developing a novel trial design, called the Platform Trial, to accelerate finding a cure for ALS https://www.massgeneral.org/als/research/researchlab.aspx?id=1840&display=FirstTreatments.   A traditional clinical trial will examine only one drug at a time and requires lengthy start-up and execution times. Platform trials, instead, are trials where multiple drugs are tested at the same time, using specialized statistical tools. Additional drugs can be incorporated as they become available, thereby decreasing or eliminating the gap in time from identification of a rationale therapy to testing it in PALS.  The platform remains open long-term until successful cures are found and will test multiple promising experimental therapeutics using an efficient and informative early-phase design with increased access for people with ALS. There are now 54 sites across the US who are participating, allowing wide access and the Center of Hope at Temple is proud to be one of them. We are anticipating beginning enrollment early in 2020.

 The first drugs to be tested include:

 Pridopidine (Prilenia) is a highly selective S1R agonist (sigma-1 agonist), shown to exerts neuroprotective effects in numerous models of neurodegenerative disorders mediated via the S1R.  Pridopidine exerts neuroprotective effects via activation of the S1R. This drug increased neuron survival and reduced the clumping of abnormal proteins. There is also previous information suggesting that S1R activation may enhance bulbar and speech function in ALS patients. Prior clinical data with pridopidine demonstrates a favorable safety and tolerability profile and provides evidence for a beneficial effect on functional outcome in another neurodegenerative disease.  https://www.businesswire.com/news/home/20190918005530/en/Prilenia%E2%80%99s-Pridopidine-Chosen-Participate-ALS-Platform-Trial

 Zilucoplan (Ra Pharmaceuticals) is a small peptide C5 (Complement component 5) inhibitor. The complement system, as part of the innate immune system, is a group of proteins that evolved to protect humans from bacterial infections. Inappropriate complement activation and deposition can occur damaging tissues and organs. There has been evidence that complement activation and deposition in the central and peripheral nervous system in ALS plays a role in the damage. Zilucoplan has been designed for convenient “in-home” use by patients and is self-administered as a small volume, subcutaneous administration.  https://www.businesswire.com/news/home/20190918005167/en/Ra-Pharmaceuticals-Announces-Selection-ALS-Platform-Trial

 Verdiperstat (Biohaven Pharmaceutical Holding Company Ltd) is a potent, selective, brain permeable molecule that inhibits myeloperoxidase (MPO). MPO is a potent brain pro-oxidant enzyme that is activated in immune cells, including microglia, the primary immune cells of the central nervous system.  In ALS, there are large numbers of activated microglia, and they are a hallmark of the neurodegeneration.  MPO, within the microglia, is believed to increase oxidative stress and inflammation levels in the brain and spinal cord. By inhibiting MPO, the investigators hope to ameliorate these damaging processes to slow disease progression.  https://www.prnewswire.com/news-releases/biohavens-verdiperstat-an-oral-myeloperoxidase-inhibitor-selected-for-platform-trial-collaboration-at-massachusetts-general-hospitals-healey--amg-center-for-amyotrophic-lateral-sclerosis-als-300920335.html

 Bioenergetic Nanocatalysis (CNM-Au8, nanocrystalline gold) (Clene Nanomedicine, Inc) is designed to provide an energetic assist to impaired motor neurons in ALS, leading to improved function. Motor neurons require significant energy to function but in ALS, corrupted energy metabolism together with increased cellular stress lead to motor neuron degeneration. CNM-Au8 provides an energetic assist to damaged motor neurons leading to improved function. It both supports bioenergetic reactions inside cells and eliminates the harmful waste byproducts of cellular metabolism. Oral delivery of CNM-Au8 resulted in both neuroprotection and remyelination in multiple animal studies. CNM-Au8 is a concentrated, liquid suspension of pure gold nanocrystals that travel through the body and enter the brain and motor neuron cells where they enhance the ability of these cells to survive and communicate by supporting cellular metabolism. CNM-Au8 was demonstrated to be safe and well-tolerated by healthy volunteers in a Phase 1 study.    https://www.benzinga.com/pressreleases/19/09/n14450362/clene-nanomedicine-inc-announces-selection-of-cnm-au8-for-the-harvard-led-healey-als-platform-tria

 IC14 immunotherapy (Implicit Bioscience Ltd) is an immunotherapy that targets CD14, a master regulator of the healthy function of the immune response to infection and injury, particularly the response of the brain's most common immune cells, microglia. While there are many contributing pathologies and genetic factors in ALS, the immune system plays an important role in both mitigating damage but also damaging neurons and determining the progression of damage. In ALS, it is thought that chronically hyper-activated CD14 loses this ability to balance the immune response to worsening background triggers, and itself becomes part of the cause of cell death, resulting in neuronal death. In fact, elevated soluble CD14 (sCD14) are associated with more rapid disease progression. By potently targeting and blocking both sCD14 and CD14 on immune cells, IC14 can switch off this damaging hyper-activation and rebalance the immune response to the ongoing disease processes.  It is hoped that this rebalancing of the immune response may result in ALS disease remission similar to immunotherapies in multiple sclerosis. 

https://www.massgeneral.org/als/research/researchlab.aspx?id=1840&display=FirstTreatments

Research Lab of Hope Tissue Bank Collection

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A tissue repository is a collection of blood, urine, and cerebral spinal fluid (called tissue samples) stored for future research in our Center of Hope Research Lab. The goal is to collect tissue samples from 500 participants over the next five years. This biorepository will provide samples to be used to study the biochemical and genetic differences of PALS compared to other neurological disease controls and healthy controls. These samples will be used to expedite future studies of genetic markers, possible disease mechanisms, and pathological changes in the PALS and control tissues. Once the biorepository is established, it will provide a resource with a large number of samples readily available to study; this will expedite research without having to collect the specimens prospectively.

PALS, individuals with other neurological diseases, and healthy volunteers are eligible to participate. Participation is voluntary and requires the participant to sign an informed consent clearly stating the allowed use of the sample. After obtaining informed consent, we collect general medical, family, and demographic information. The collection consists of urine and a blood sample (5 tubes). All samples get coded with a unique GUID number that de-identifies the participant to maintain confidentiality. There are optional choices (which are voluntary) to participate in the following procedures: Permission to use the blood sample to create cell lines, which are cells that are grown in a special lab and stored as an inexhaustible source of DNA. Permission to keep the cell line/DNA for future research use. Permission to collect an additional half of a teaspoon cerebral spinal fluid during an already scheduled lumbar puncture.

During consent, participants are asked if they are willing to provide samples at future routine scheduled clinic visits (4-8 month intervals) to create a longitudinal collection of samples to identify any changes in the same person over time. In the case of PALS, the longitudinal collection will study the progression of their disease. The longitudinal study requires another collection of blood (2 tubes) and urine if the participant consents. Providing future samples is voluntary and not necessary for study participation. Participants have the option to decline to give a sample at any time while participating in the study.

Current participants are informed that we are extending the participation to healthy controls, such as family members or friends. Family members and friends interested in participating in the collection can consent during a clinic visit with their PALS or contact us to schedule a convenient time to join.

The MDA/ALS Center of Hope Team at Lewis Katz School of Medicine at Temple University is passionate about and devoted to finding a cure for ALS through clinical research. Participants consenting to the use of their tissue in research are aiding greatly in furthering the knowledge of ALS and providing researchers with an invaluable resource to expand the study of the disease. If you have not already consented and donated tissue to the biorepository, please ask at your next clinic visit or have your family inquire.

Mental Health Tips

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Mary Holt-Paolone, MSRN 
Clinical Nurse Counselor, MDA/ALS Center of Hope 

During August, known as National Wellness Month, many people focus on improving self-care, managing stress, and promoting healthy routines in their lives. These topics can be helpful for anyone, but when living with ALS, they are even more essential to maintaining some emotional and mental balance from day-to-day. And just as important as they are, we also know it can be very difficult to make self-care and stress management priorities due to the rollercoaster of feelings and experiences you may be facing. In this article, we hope to offer some easily accessible, practical, and useful tools to allow you to positively manage those moments when situations are "getting the best of you." 

As a starting point, it can be helpful to understand a bit of how the brain/body react under stress. When the brain interprets an experience or thing as a potential threat/stressor to the system (whether physical, personal, financial, time, emotional, etc.), its main focus is survival. In a matter of seconds, regions of the brain are alerted, and stress chemicals are released out into the body to protect us by either fighting against, fleeing from or freezing to the potential danger. Known as the Stress Reaction - very important in real trouble, but can be harmful when chronically activated. An interesting detail as well is that our brains cannot tell the difference between a real saber tooth tiger coming at us versus some negative thinking. The same cascade of events can happen in the body, depending on how we perceive or think about our experiences. 

So what can we do? Here are some tools that can quickly down-regulate the body and help us shift gears on the high-speed stress train. 

  1. Notice: We can't make any changes in our lives unless we are aware of what is happening first. Notice what signs your body gives you when feeling stressed - muscle tension, irritability, stomach/head pains, less tolerance, anxiety, etc. Use these as cues, and once aware, we can do something different if needed/wanted.

  2. Pause (if possible): Pausing can help to interrupt the stress reaction train and give us a moment to refocus.

  3. Breathe: Some people hold their breath when feeling distressed. Deeper, slower breaths can help to activate improved oxygen flow to the brain and decrease heart rate, blood pressure, etc., which can effectively down-regulate the body in just a few minutes.

  4. Five Senses: Repeatedly checking into even just one of our senses (see, hear, taste, smell, touch) can also help us refocus, come back to the here and now, interrupt the stressful mental train of thoughts that can occur and allow our bodies/minds to relax a bit.

  5. Mantra/Phrase: Some people benefit from choosing a positive-feeling phrase they continually repeat to themselves during times of stress to stay focused on hope and engage their inner strength. Examples of such phrases are:

      • "I/We can do this."

      • "What can I do now?"

      • "One step at a time."

With practice, any of these tools can bring about benefits, and when used together, the effects can be even more successful in giving you a bit of calm and balance in those crazy moments of life.

For any questions about these tips or any mental health issues, please feel free to reach out to Mary via email: mary@maryfholt.com or phone: 610-733-5573.

CRLI: One PALS Perspective

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Written by Educator, CRLI Attendee, and PALS Todd Kelly

At the conclusion of the ALS Hope Foundation’s 2019 Clinical Research Learning Institute, the participants (patients, caregivers, and specialists) earned their certificates and bright pink lanyards as ALS Research Ambassadors. More importantly, we left the 2-day conference with new friendships, a deeper understanding of the disease and its particular complexities for research, and a renewed commitment to advocate for multiple issues that the ALS community faces.

    The conference on August 23-24 was free to participants at the Inn at Villanova, including the three meals as well as overnight accommodations if requested.  The seminar’s goal was to educate the participants and arm them with the tools to share this knowledge about clinical trials, current treatments, therapies, and drugs in the FDA “pipeline,” and methods for advocacy. We learned that one factor limiting research is low participation from PALS themselves (with an average of only 2 PALS per center per month). Another obstacle is that ALS, unlike most diseases, lacks clear biomarkers, necessary to prove/test a treatment’s success and efficacy. While the FDA does an excellent job in protecting the lives of patients, this means that the drug discovery process is incredibly expensive and often lasts 12 or more years.

    When some of the topics were scientifically complex and riddled with unfamiliar acronyms, the presenters did their utmost to use effective analogies, answer our questions and supported each other’s presentations with elaboration. The group carefully and critically read a published research paper and together uncovered serious flaws in its methodology and conclusions. We learned to distinguish between false hype and authentic hope. In the end, we shared ways in which we could individually and collectively pursue advocacy goals. And, of course, the opportunity to meet with other ALS patients and their caregivers was extremely valuable as we shared stories, frustrations, strategies, advice, and empathy.

Each presenter covered different topics reflecting their overlapping areas of expertise as practitioners, clinicians, researchers, biopharma representatives, organization advisors and directors, and advocates. All were highly knowledgeable, allowing them to respond to our questions as well as those of their peers. However, what fully united them was their compassion for the families of ALS patients and their passion for fighting this disease. The conference was inspiring, and I would encourage anyone impacted by this pernicious disease to participate in future offerings.