Platform Trial to Begin Enrollment in Early 2020

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   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.

 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.

 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.

 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.

 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.

Research Lab of Hope Tissue Bank Collection

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

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: or phone: 610-733-5573.

CRLI: One PALS Perspective

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.

Report on RESNA 2019 #RehabWeek

The Rehabilitation Engineering and Assistive Technology Society of North America (RESNA) 's annual conference, #RehabWeek, was held in Toronto, Canada, June 24-27, 2019. Sara Feldman, PT, DPT, ATP, the Assistive Technology Professional at the MDA/ALS Center of Hope, attends the conference every year to keep up on the newest developments in assistive technology. The conference attendees include users, Rehab Engineers, PTs, OT, SLPs, researchers, suppliers, manufacturers, students, and other individuals working with assistive technology. Here Sara shares her experience: 

The RESNA Conference is a combination of keynote lectures, panel discussions, poster presentations, and workshops happening throughout the day. This year's conference, #RehabWeek, involved five other organizations concurrently holding their conferences:

 • the International Functional Electrical Stimulation Society (IFESS) 

• the International Conference on Rehabilitation Robotics (ICORR) 

 the International Industry Society in Advanced Rehabilitation Technology (IISART) 

• the American Congress of Rehabilitation Medicine (ACRM) 

• the International Society for Prosthetics and Orthotics (ISPO Canada)

This collaboration increases the numbers and diversity of both presenters and topics!

The Keynote Speakers:

The first speaker was computational neuroscientist, David Putrino, PT, Ph.D., (Mt Sinai HC, NYC). His lecture on "How to be an Innovator: Exploring rehabilitation and human performance in the 21stcentury" was excellent and showed us what is possible if you have the right mindset. For more on the work, he and his collaborators are doing see Putrino Lab and Not Impossible Labs

Deborah Backus, PT, Ph.D., is the director of MS research at the Shepherd Center (Atlanta, GA) and her topic, "Why Technology Innovation does not Equal Technology Adoption: Strategies for Successful Uptake," discussed opening the communication between the people in research and the people who use assistive technology. When deciding what AT to use, clinicians ranked "Evidence" as number one and users ranked "ease of use" as number one. She wants to bring these two closer together. When it comes to the process of idea to design to test to launch, only 2% of ideas ever make it to the intended users. Our role as clinicians is to help it to happen.

Mark Stephan, Board of Directors, Shirley Ryan Ability Lab, (Chicago, IL) spoke on "Recovery," and told his story of rehabilitation following a spinal cord injury. His story is inspirational and amazing and full of hard work and grit. He was the inspiration behind the Shirley Ryan Ability Lab, a place where "clinicians, scientists, innovators and technologists will work together in the same space, applying or "translating" research real-time."

Edward Lemaire, Ph.D., Ottowa Hospital Research Institute, presented on "Wearable and Wear-Nothing Approaches for Digital Transformation" which described using smartphone Apps to track patients' progress and transforming standard clinical spaces into "smart" spaces.

Katherine J. Kuchenbecker Ph.D. directs the Haptic Intelligence Department at the Max Planck Institute for Intelligent Systems in Stuttgart, Germany. Her lecture on "Haptic Interactions Matter" focused on the tactile interaction between people and the environment. Much of her work focuses on giving tactile feedback to surgeons using robotics, but her ideas also translate to other areas of robotics.

Geoff Fernie CM, Ph.D., PEng, CEng, FCAHS gave RESNA's Colin McLaurin Distinguished Lectureship Award Lecture. His presentation, "Prevention is an Important Topic for Rehabilitation Researchers" was an interesting and informative look at small changes aimed at prevention that can make a huge difference in a person's ability. One example was their research into the treads on people's winter boots that lead to Rate My Treads, a website that rates the slip resistance of footwear….preventing slip and falls!


There were multiple workshop-style presentations throughout the week, here are the highlights with a few links for you to check out! 

In "Adaptive and Therapeutic Approaches to Videogaming" Occupational Therapists M Calder (Stan Cassidy Centre for Rehabilitation) and E Muston-Firsch (Craig Hospital) presented on playing videogames, and how to make them more accessible. The first step is to look at what is already available in the software of the system, then the hardware, to look at adaptive controllers and 3D printing. We were all then given a chance to try the different set-ups and play the games with the adapted set-ups. Very cool!

 "Hands-free Mice: What's Currently Available and How to Make the "Right" Choice" was an excellent presentation by H Koester (Koester Performance Research). She has a blog post where she describes her experience at RESNA this year as well as shares her information. Highlights from the 2019 RESNA Conference.

"Unlocking Your Smartphone's Potential: Considerations and Application," was another excellent presentation by K Engstrom and S Sanderstrom (Craig Rehab) and was an across platform look at smartphone accessibility. We will be pulling this information together for our resource section soon, but here is a peek at Craig Rehab’s site

 The assistive technology that is available for people with ALS is ever-changing and evolving. The RESNA conference is an excellent place to learn about the newest technology and find out what is truly functional and what is hype. This year was no different!

Annual Research and Awards Luncheon


The ALS Hope Foundation kicks off May ALS Awareness Month with our annual Research Update and Awards Luncheon. This year’s speaker for the luncheon was Dr. Richard Bedlack from the Duke ALS Clinic speaking on his current research into ALS.

Click here to visit our YouTube page to see his video presentation. 

The second half of the luncheon program recognizes individuals who have provided support to the ALS Hope Foundation, The MDA ALS Center of Hope and to ALS Research. Areas of recognition are:

We were honored to have over 150 attendees to learn about advances and new areas of research being explored by Dr. Bedlack and to honor and recognize this year’s honorees. Thank you for your continued support and help in recognizing those who make a difference for the Foundation and in the lives of those living with ALS. For more information and to "meet" our awardees visit our website

3rd Annual Gala of Hope

3rd Annual Gala of Hope

Over 400 people joined us for the beautiful 3rd Annual Gala of Hope on Saturday, February 23 at Vie on North Broad Street in Philadelphia. Together, we raised over $58,000 to support the MDA/ALS Center of Hope, education and research initiatives. The evening was truly a success  and we are deeply grateful for the generosity of our guests and supporters. Dr. Robert Sinnottthe evening’s guest speaker, shared a very heartfelt message of hope and what it meant to him as a person battling this disease. Most importantly, Bob shared an encouraging message, “To love harder." 

We are appreciative to Hank Flynn from Fox 29, our Emcee, who not only was an excellent host, but who also put a personal note on the evening in memory of his father-in law. Thank you to all of our sponsors, many of whom have been on-going supporters of the ALS Hope Foundation. Thank you to the many who donated items to the Silent Auction as your generosity truly added to the evening festivities. And of course, a huge Thank You to the staff at Vie who admirably managed a room full of 400 guests with ease and professionalism. The food was delicious, the room was gorgeous, and every one felt welcome and taken care of.

The evening brought awareness to the ALS Hope Foundation and the work of the team at the MDA/ALS Center of Hope and recognized their 20 years of dedicated care for people with ALS.

View additional photos from the event on our Facebook page or via this link.
Watch bob Sinnott's speech and see the Team video below!

3rd Annual Gala of Hope

President’s Message from the Gala of Hope

Dr. Terry Heiman-Patterson


The 3rd Annual Gala of Hope marked the twentieth anniversary of the ALS Hope Foundation, which I co-founded with my friend and colleague Dr. Jeffrey Deitch, who passed away a few years ago. Dr. Deitch was a gifted scientist and humanitarian whose ideals and commitment will forever be part of the ALS Hope Foundation mission. 

I want to take this opportunity to acknowledge the challenges faced by people living with ALS and their families every day.  No one understands the urgency of our cause more clearly than PALS and their loved ones. The courage and grace with which PALS like our guest speaker at the Gala of Hope, Bob Sinnott, and their families face this disease is an inspiration to all of us at the ALS Hope Foundation and the MDA/ALS Center of Hope. Together, the ALS Hope Foundation, the MDA, and Temple Health, along with our team of clinicians and volunteers, are committed to making a difference to the ALS community. 

We are proud of our ALS Center which has have made tremendous progress over the last three years at Temple University. Our multidisciplinary team is focused on implementing state of the art care with hope, compassion, and honesty.  We are committed to the future, learning more about ALS, its causes, and drugs that may someday allow us to offer better treatment options. We are also dedicated to educating people about this disease - whether it is people living with ALS, healthcare providers, or the general public We want to accelerate research for the good of all people with ALS through our collaborative efforts within Philadelphia and across the United States. This progress is only possible through the joint efforts of the ALS Hope Foundation, Temple, the MDA and you!

Despite much progress, there is still work to be done. While we have two medications, Radicava and Riluzole, that can slow the progression of ALS, there is an urgent need to find real and tangible treatment while taking care of people living with ALS now. For this reason, the mission of the ALS Hope Foundation includes care, research, and education. Thank you for joining us in this important mission. Please enjoy your evening, and know that your participation in our organization is vital to keep hope on the horizon.

Gala of Hope for ALS - New Venue and Date

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We are thrilled to share this exciting news with you regarding our 3rd Annual Gala of Hope, affiliated with MDA/ALS Center of Hope at Temple University.  We sold over 230 tickets in one month!
After careful thought, we have decided to move the event to a larger venue to better accommodate our guests. This means we also have to change the date.
Please now join us on Saturday, February 23, 2019 at 7pm at Vie 600 N Broad Street in Philadelphia!

All tickets previously purchased will be accepted and no further action need be taken. If you are unfortunately unable to attend, please know that you will be missed.  A response to this email will enable you to receive a full refund. You can also contact Mara at or 215-568-2426 with any questions or concerns. 
Thank you for your continued support of the ALS Hope Foundation community. Your donations and assistance make HOPE possible!
Looking forward to seeing you on February 23, 2019 at Vie!

If you would like to purchase tickets or learn more about our sponsorship and advertising opportunities, visit this page.