Welcome everyone to tonight's member May session or second member May session. My name is Christina Ghanem. I'm a member engagement coordinator at AAPMNR. I'm just going to review some housekeeping notes before I pass it on to Dr. Carter. The views expressed during this session are those of the individual presenters and participants and do not necessarily reflect the views of the views and positions of the Academy. AAPNR is committed to maintaining a respectful, inclusive, and safe environment in accordance with our code of conduct and anti-harassment policy available at AAPNR.org. All participants are expected to engage professionally and constructively. This activity is being recorded and will be made available on the Academy's online learning portal. An email will be sent after this activity with a link to bring you to the recording and evaluation. For the best attendee experience during this activity, please mute your microphone when you're not speaking. To ask a question, we'll actually be using questions at the end, but please use the raise the hand feature or the chat to be called upon. Note time may not permit for the panel to field every question. Thank you so much and I'll pass this on to Dr. Carter. Thank you very much. I am honored to be moderating this really excellent group of physicians we have tonight. I am filling in for Dr. Colin Franz, who was originally the moderator. I would like to introduce the speakers. First, we have Dr. Eliana Howard, who is a professor in the Department of Rehabilitation Medicine and medical co-director of the ALS Center of Excellence at the VA Puget Sound in Seattle. Dr. Howard will speak about leading an interdisciplinary ALS clinic as a physiatrist and share insights from her experience delivering coordinated person-centered care for the veterans with ALS and championing a national level advocacy and policy change. Next, we have Dr. Nassim Raad, assistant professor at the University of Washington and director of the Electrodiagnostic Laboratory at UW Medical Center, Montlake. Dr. Raad will present physiatry leadership in spinal muscular artery care and neuromuscular rehab. Dr. Raad also co-directs the MDA Care Center and has been instrumental in shaping interdisciplinary approaches to managing complex neuromuscular conditions, including spinal muscular atrophy, across a lifespan. And then finally, we have Dr. Sabrina Bagannoni, associate professor at Harvard Medical School and co-director of the Neurological Clinical Research Institute at Mass General Hospital. You may have heard of that place. Dr. Bagannoni is a global leader in ALS clinical trials and co-VI on the ELI ALS platform trial. Her job is titled Physiatrist-Led Innovation in ALS Research and Clinical Trials and will highlight how BM&R expertise is shaping therapeutic development and accelerating access to new treatments. After these ladies give their presentation, we will open it up for questions and discussion. And without further ado, I will turn it over to Dr. Howard. Thank you. Thank you so much for the kind introduction, Dr. Carter. And I am going to share my slides here. Okay sorry I'm going to have to be a little bit more clever than that. Let me start over the way I'm sharing. Okay you can hear me now right? Yes. Okay great. So thanks everyone. Again my name is Ileana Howard. I'm on faculty at the University of Washington and I did not dream that I would be an ALS physician when I finished my residency but something that I fell into and started an ALS clinic at the VA Puget Sound which I now co-direct and that launched in 2012 and now I currently lead the ALS system of care within the Veterans Health Administration overseeing the delivery of care to veterans with ALS across 170 medical centers in the country and I leaned really heavily on some of my colleagues that you'll meet here tonight as I was trying to navigate this journey into collaborating with neurology colleagues in the world of neuromuscular physiatry. I remember when I was starting out I asked is there room for me at the table here and I'm happy to come back and report to all of you that not only is there room at the table for physiatry but it's my very biased opinion that physiatry belongs at the head of the table when caring for patients with chronic complex neuromuscular disabilities. Before I go too much into this I have a couple of disclosures. First off I don't have any financial conflicts of interest to disclose neuromuscular physiatry is my passion but it's not very profitable and while I am employed by the Veterans Health Administration my views and opinions that I'm expressing during my presentation today are entirely my own and they're not reflective of my employer. Okay I have just a few minutes to share with all of you and what I hope to cover is what made me decide that focusing my career on ALS as a physiatrist was a worthwhile endeavor. Why I think that physiatry adds value to ALS care particularly why physiatry should be leading the charge in ALS care and that neuromuscular physiatry really is at the core of physiatry. And just a shout out that this is ALS Awareness Month. Here's a picture of me up at Washington DC about 10 years ago with Gail Gleason who's Steve Gleason's mom and also the surviving spouse and daughter of one of my very first patients that I cared for with ALS and it's because of these patients that I decided that I wanted to focus my career on this. And while a picture is worth a thousand words I think that a video is worth a million so I'm going to do a quick changeover and I want to share just a really quick video of one of the patients and families that I care for to hopefully give you a little bit better idea as to how central physiatry is in the day-to-day life with these patients. So give me one second. I just want to make sure it's the audio playing on the video. No, I think Elena when you share. I think to check the box, the way it says share sound. Let me try again. Sorry. There were some titles. My name is Bill Vander Ploeg. I was diagnosed with ALS February 14th, 2017. I was very energetic, mainly working on cars. As I started getting worse, I had to give it up. He has a lot of personal needs. He can't reach up and scratch anything and so he needs a lot of that care during the day. On the days that we don't have a nurse or caregiver, I say be patient with me. I'm doing the best I can. And this might be one day that it's just the two of us, but we know tomorrow is going to be different. We're going to have help and we'll get through this. There are a lot of things that we can do to not only improve the quality of life for our veterans with ALS, but also extend their life expectancy. One of the most important is interdisciplinary care. The entire ALS team comes to where the veteran's at in clinic to provide the best, most coordinated care. With the VA, they provide nursing care. They take care of all his needs. They feed him. They change him. Anything that he needs throughout that time. We're really fortunate in our ability to provide skilled home care for our veterans so that it can remain in their homes with their families. And we have to anticipate needs before the veteran often realizes. He needed the lift in the ceiling. They had that installed. Things that we didn't know he was ever going to need, they immediately had those sent to the house and provided instruction on how to use them before he ever needed them. The veteran had completely remodeled our house. Where I am sitting right now was a sunken living room. And so they had to build that up. They had to give us a second bedroom, and it's designed totally for me. And they got me a new van, which I can get out and go to car shows and stuff like that, see people. Before the VA was involved, I was really depressed. I was like, it's a death sentence, you guys. There's really nothing they can do. I'm not a machine or something. I have feelings. I have hope. I have desires. These are things that keep me going. And at the Veterans Hospital, they give me the compassion and the willingness to say, well, let's give it a try. Let's see what happens. A lot of veterans come into our clinic having been told that there's no hope for them. And that is the furthest thing from the truth. Our ALS clinics not only offer hope, but we also deliver on that promise by providing the resources and equipment that those veterans and their family need to have the highest quality of life. Okay. Thank you for humoring me, and I'm sorry for my technical glitches and getting that to play. But I hope that it gives you just a little bit of perspective about the things that matter most to patients with neuromuscular disease, specifically ALS. I'm going to go back to my regular slides here. And how well equipped we are as physiatrists to address that. I have been talking to patients with ALS and their families in my clinics for over 13 years. And when I ask these individuals what matters most to them, it is about being able to continue to communicate with the people that they love, to be able to get out of bed and get out of their home and still engage in their community. Not to be bed bound, to minimize the burden on their family, and to stay at home with their families for as long as they possibly can. And all of those things are precisely what we are the most well equipped to do as physiatrists. It's really core to the bread and butter of being a physiatrist. Just to give another example of ways that physiatrists have incredible impact in caring for these individuals. One of the most important aspects of care that will prolong the life of individuals with ALS is providing proactive and comprehensive management of neuromuscular respiratory weakness. In ALS, the majority of patients have healthy lungs, but they have weakness in the muscles of the chest wall and the diaphragm. Unfortunately, this is not familiar territory for most pulmonologists across the country. But as physiatrists, we understand muscle weakness, we understand stiffness from muscles that don't get full range of motion. And therefore, we are the best equipped to be able to address muscle problems that are life or death for the patients that we serve. So providing range of motion through using a cough assist device in coordination with pressure support through bi-level. Addressing the barriers to performing oral care when someone has lost use of their arms. This often just flies, it's out of the radar of a non-physiatric provider who's caring for these patients. And yet, this is really crux of some of the most powerful interventions that we can provide for these families. Now, just to beat a dead horse, neuromuscular physiatry was the foundation of the field of PM&R in the United States. Many of the leaders of our field were trained with funding from the March of Dimes after the polio epidemic in the 1950s. And as our specialty has sub-specialized over years, it feels like we've gotten away from understanding that the core principles of physiatry are really the essential key characteristics that are needed to provide the most comprehensive and supportive care for these individuals. Other countries, such as Canada and the Netherlands, apply general physiatry to these patient populations. In some countries, physiatrists are the primary physician that are responsible for care of individuals with ALS. And I think that patients would be well-suited to having more physiatric involvement. So, to sum up and allow time for my colleagues to have a chance to share their stories with you, I hope that none of us sell ourselves short as physiatrists in the care of patients with ALS. We are not only the leaders of the interdisciplinary team when it comes to rehabilitation, I think we're very well-suited to leading ALS care beyond that. And we need to see beyond being a musculoskeletal or spasticity or pigeonholed in our role in caring for these individuals. And hopefully that video was a good illustration of that. There is not a conflict in who gets to be at the table. All hands are needed. It's a five-alarm house fire when we're talking about ALS. And no one is going to turn you away when you're offering to add value to this patient population. And while there's a little bit of uniqueness to ALS compared to exclusively upper motor neuron or exclusively lower motor neuron conditions, this is a very exciting place to be in the fields of PM&R. And I'm certain that we're going to see some developments in treatment, thanks to some of the colleagues that we'll be hearing from on this call, that are going to make this even more exciting to work in rehabilitation in the next few decades. So my email addresses are here. I'm always happy to talk ALS and PM&R all the time. Feel free to reach out. And I look forward to the conversation today. Thanks, everyone. Thank you, Dr. Howard. Dr. Rad, I turn the floor to you. Thank you so much. I'm not sure I'm going to be able to do it as beautifully as Dr. Howard. But I thank you, everyone, for joining us today. And a shout out to Dr. Howard, Dr. Paganoni, and Dr. Carter, who really are all pioneers in neuromuscular rehabilitation. So my journey started eight years ago, really, or actually, I did residency before that. But my focus in neuromuscular care started eight years ago. And while I'm sharing that, I'm going to make sure that I can actually share these slides correctly. Is that being shared? Yes, looks good. OK, thank you. And so after completing residency in PM&R, I elected to do neuromuscular fellowship through neurology. That's not the only way to be involved in neuromuscular care. And I think Dr. Howard did such a beautiful job going through the fact that in many places, neuromuscular care is pivotal to what rehab does. I'm a little biased. I think that over the last few years, it's made me sad to see that just a lot of times when people think of physiatry, they think of just MSK, when really it includes so much more than that. So I wanted to dive a little bit into some of the work that we can do for these complex neuromuscular patients. And when I started here at the University of Washington, I really had the pleasure of meeting such wonderful patients with unmet needs. And so I when I started here was during a pretty pivotal time. And we're going to see more of this in the course of neuromuscular medicine where there started to become novel treatments for conditions such as spinal muscular atrophy. And we started seeing a return of these patients from from those that were only being seen by primary care physicians returning back to an academic center and really lost at, you know, where did their care, where does their care belong? And so as we journey through spinal muscular atrophy, and, you know, as a refresher, this is inherited lower motor neuron disease that was once thought of as just a, I guess, an I advance these slides, maybe, just thought of as a childhood disorder. But with not just with the new novel medication approaches, but with the changes in respiratory advancement as well, it's no longer a pediatric disorder. We are seeing them through adulthood. I have the fortune of seeing about 55 adult SMA patients. And what became really apparent is the standards of care really revolve around a multi or interdisciplinary going to steal that word from Dr. Howard approach to treatment. Of course, at the center, we have this patient with SMA and and their family and as the center and we have all these spokes and pulling this, you know, schematic from the standard of care that were written for SMA. We see here neuromuscular and rehab lumped into one one kind of spoke off of this. And the truth is, is that and as I go through some of these slides, I hope you see why physiatry is really I don't know who would be better than to lead a clinic like this. And so we talk about, you know, their pulmonary care, their acute care, medication management, including those novel therapies, nutrition, orthopedics care. We are not unique to a multidisciplinary approach. We're not unique to managing complex diseases as well. But I think what makes us even more unique is that we are very proactive in our way of thinking as physiatrists. And so with and I apologize because with a short time, I'm not telling you as much as I want to about what SMA is and the different types of it. So I hope you can kind of go back and refresh yourself on that. But this is a disorder that, you know, for type one SMAs, we've never seen them in adulthood. They would pass away at age two years old prior to any interventions. But we now have therapies where we're going to be seeing type one, more type twos in our adult clinics. We're going to see more milder, more severe phenotypes, mimicking milder phenotypes. And this is going to evolve into more chronic disease management. And so we're going to need a more proactive approach to being able to deliver this care. And seeing this is where we instituted a dedicated adult multidisciplinary care center for these patients in our clinic. And so we'll kind of go through this in what happens in these clinics. So when I first approached the hospital into why we even need this and why physiatry should run this, they were like, well, what happens in these clinic visits? And so I'm just very briefly going to touch on some of the things that happen in these clinics and in hopes that these kind of resonates with your skill sets as even general physiatrists. So, of course, we focus on function for these patients. So we address orthoses needs. We talk about contracture risk, supporting these at risk joints. For those that are still ambulatory, we talk about how we can support them to continue to ambulate with appropriate orthotic devices. We talk about power wheelchairs. We talk about custom supports and seating positions and what's going to be best suited for their needs. We talk about assessing them for physical therapy and outpatient and occupational therapy. And we talk a lot with their caregivers about how best to support their care with continued caregiver needs. A lot of what we do in a system that involves insurance approval and unfortunately not as much caregiver support as we would like involves being extra thoughtful into how we can provide these cares to make these patients as independent as possible. Unfortunately, respiratory is the leading cause of death in SMA patients with type 1 and type 2. And Dr. Howard has already mentioned why physiatry is so well versed. We spend a lot of time working with noninvasive ventilation in our clinics. We actually have the PFTs done in clinic and I'm the provider reading them with our respiratory therapist. And it's not uncommon that all these patients have our direct line of contact so that when they get admitted to maybe a community hospital center and they're trying to put oxygen on them, that we're the ones reinforcing why it is that they need noninvasive ventilation. Because this is not well known in general pulmonary condition. And that can really mean a matter of life and death when they're getting admitted for acute pulmonary infections. We talk to them about vaccinations. We talk to them about preventative care. Again, I put some more nitty gritty slides, but the goal in this presentation is to really kind of advertise the role of neuromuscular care in physiatry. And so I think, you know, the uniqueness of how we manage this condition, I won't get into those specifics. We talk a lot about swallow and nutrition. So we talk about, you know, swallow evaluations, how to have appropriate conversations when feeding tube is needed. What are the multiple indications? How do we mitigate the obesity risk in a patient population that may not be able to have as much functional exertion in a disease that also has its own metabolic derangements? And, you know, for those of you, you know, these are similar things that get talked about with spinal cord injury patients, with other general rehab patients. And so not just unique to neuromuscular diseases. We talk about bone health. We talk about scoliosis in SMA. It's unique that the SMN protein itself affects osteoclasts. So not just the fact that they're not weight bearing and how do we address that bone health. We work closely with endocrine and oftentimes we manage the bone health on our own, just given our specialty and expertise in this. And then we manage everything else. And so we have a lot of anxiety and depression with chronic illness. We talk about good news as well. We talk about pregnancy counseling and what are the specific things that we need to monitor and review to make sure that they have a healthy and safe delivery. What does that look like for postpartum? What does that look like for childcare with them? How can they continue driving? How can we do better job in managing bladder management in females? SMA patients are, as with many neuromuscular disorders, cognition is not involved. In fact, there's a little, not antidote, because I find it very true, but antidote for a lack of a better term that our SMA gene must code for higher intelligence. That has not been proven, but most of my patients with SMA have very high IQs and just have such meaningful lives. And so our goal is how I kind of describe my work is I almost feel like I am their primary care for this disease, right? Everything about this disease is what I manage. What I didn't talk about necessarily, and this might pivot into some discussions afterwards, is what about that aspect of the neuromuscular fellowship and how does that play a role into my ability to lead this clinic? Because as we talked about, good general physiatry is involved in the bread and butter of these complex conditions. And I think that there is still, as we become very subspecialized within our specialty, there becomes a more desire for there to be fellowships, similar to subspecializing into any field into physiatry. But I think one of the unique aspects may be that it does allow me to also be the provider that does their gene therapy and gene modifying medications. And it's not to say that general physiatry should shy away from that. I think there may be other hurdles into that in terms of coverage and how you can play a role into that. And that's just one piece of the puzzle for this. And so I don't think that this has to be just a neuromuscular trained physiatrist, but I do think that that does add a little bit to the piece of this complex care model. But I kind of take it back and hope that this echoes with everybody. Who better to lead this clinic that I described than physiatry? Can you think of another specialty that might be able to manage function, bone health, speech, swallow, gene modifying therapy without sending like 10 different referrals, right? Without sending to have so many unique referrals that wouldn't understand the whole picture, right? If you were just sending off to have one specialty address one niche in this disease. And so I think there's gonna be a growing need as there's more novel treatment in neuromuscular for, I've had residents rotate with me and say, really their exposure to neuromuscular before coming to adult clinic was pediatrics. And I think that's helpful to hear because I think those diseases are gonna live longer and it's not just gonna be pediatric PMNR that sees these conditions. And so I hope that gets you excited about the field and no matter whether you're considering doing neuromuscular specifically or are a general physiatrist that you are motivated and equipped with what you need to provide great care for these patients. Thank you. Thank you, Dr. Wright. And our final speaker is Dr. Eganoni. Sabrina, I will turn it over to you. Thank you so much. It is a real pleasure to be here with all of you and I'm really happy that we're having this session about physiatry and the role in neuromuscular care. So in terms of what I, I'm gonna add a little bit more about the research aspect of this. And so I've been working in ALS care now for more than 10 years and really everything that we do is teamwork and that's definitely another aspect of training in PMNR that makes us particularly suited for working and leading neuromuscular clinics. In my case, I work exclusively on ALS. In the ALS clinic, one day a week, every Tuesday, I have the pleasure of meeting people living with ALS and their families. And I always say that I could not do the rest of my job which is designing and leading large clinical trials without the experience of my Tuesday clinic. Because whenever I see people in clinic in addition to scientific ideas and sort of kind of general learnings, it's really like the motivation. Seeing the need and why we're doing what we're doing in research, I always say I had the best job in the world because I do get to see patients and then I get to work in the lab in collaboration with colleagues working on clinical trials. And the two things are very much complimentary. So just wanted to tell you where I started and everyone has a different journey and gets to PMNR or neuromuscular medicine in different ways. So for me, I actually, when I was growing up, to be honest with you, I grew up in Northern Italy, Lake Como, my father was, still he's a primary care physician. And my idea was to take care of people because all I knew was a lifestyle of a great physician was there for his patients. And so I thought I would go into medicine to become an internist or a primary care physician because all I knew was being there for people who needed me in different aspects of their life. That was the model that I was following. And then in medical school, I also did enjoy research and I met a few wonderful researchers. And so in Italy to graduate from medical school, you actually had to write a thesis. So you have to spend some time doing some research. And when I did that, I really fell in love with research. And so I had almost like these two souls, these two things that I loved, being a general physician and thinking about internal medicine or something more general, but also going deeper into one specific mechanism, one specific disease. And then for personal reason, more than anything else, I moved to the U.S. because my husband had an offer to do a PhD in Northwestern. And so I said, sure, I'll do a PhD too. And so I went to Northwestern and I got my PhD in neuroscience. And those five years working with mice and rats were kind of tough because that was not exactly why I went into medical school for, but at the same time, they were incredibly informative because they gave me a glimpse into how fascinating human biology and pathophysiology are and how we can actually work with patients, but also keeping in mind the molecular mechanisms of disease and trying to see if there's something that we can do about it. And so for residency, I chose PM&R again because I really have that desire to keep the patient front and center more than a specific disease or piece of the patient. But then when I, so I moved to Boston, I did my residency at Spalding. And then something unexpected happened when I was a resident at Spalding. Actually, this picture is from our class in 2008. And I am here, different hair color, as always in my different pictures through the different years. But the most important person in this picture is Dr. Lisa Krivikas, who I met the first few weeks when I was a resident there. And Dr. Krivikas, a few months after I started my residency and I started actually following her because I was really fascinated by her lab. You know, I was coming from a deep research experience, preclinical science. And so she was the one who had the leading lab, preclinical lab at Spalding at the time. And so I began following her and sure enough, after a few weeks, she shared that her research in ALS had become deeply personal because she herself had developed ALS. And one thing that I will never forget is that although she knew exactly the natural history of ALS and she knew what the future would be for her, she continued to mentor me. And so I remember her, you know, mentoring me. She was coming in with a cane and then with a scooter and the wheelchair. And then at some point she said, you know, I cannot continue to mentor you, but I can introduce you to a good friend of mine who can be your mentor moving forward. And so that, you know, she was actually one of the very first physiatrists at the time, again, we're talking about 2008, 2009. She was one of the very first few physiatrists who was working in an ALS clinic. And she was working closely with the director of the clinic, Dr. Mary Sukovich, who would later become the chair of neurology. And so she asked her friend, Dr. Sukovich, to take me as a mentor. And so because of that, you know, that really what changed my career. And so with that, you know, armed with that background in physiatry, I had this opportunity to focus on ALS. And so that's why I did two fellowships, one in neuromuscular and one in ALS. And for the ALS fellowship, it was a ALS research fellowship where for one year, I literally followed Dr. Sukovich around. Everywhere she went, I went. And that one year allowed me to learn clinical research. Previously, I'd done a lot of bench research. And so because of that experience, I was able to, again, go deeper into the methodology and I could understand sort of the clinical research because I had the previous experience in the lab. And then little by little, Dr. Sukovich started a center called the Neurological Clinical Research Center, asked me to co-direct the center. And so that's what's called the Neurological Clinical Research Institute that today has over 200 people and I'm one of the co-directors. And we function as a clinical research center where we design trials for ALS and lead trials. And these are large multi-center trials. So tonight, just in the few minutes that we have, I just wanted to give an example of one trial that's been my main occupation over the last five years. And that's a trial where we, I think we really got a lot of inspiration from the PM&R method of working together in teams. And so that's kind of what I want to highlight from that experience. So we started again with the patient at the center. So in 2018, so, you know, compared to my residence, you know, fast forward a few years after I'd done all this extra training and learned about research. In 2018, Mr. Sean Healy, who is in the picture here, was diagnosed with ALS and he was a very successful, prominent businessman from Massachusetts. And he had actually led a number of companies very efficiently. And when he was diagnosed and realized that time was not on his side, he wanted to do something transformative and disruptive, just like he had done the same in different types of business. And he became a patient of Dr. Sukovic and said, you know, I'm going to give you a transformative donation, but we need to do something new to accelerate the way we do research. And so we started with something called the Platform Trial, which is a type of trial design that we learned from cancer. So this was not our idea in terms of the trial design itself but we went around the world, literally learning from people who had done platform trials for ALS. Platform trials mean that you focus on the disease, not on any single drug. And so you create a platform for that particular disease where you can test multiple drugs at the same time and sequentially. And so I'm going to also have a video that I hope the sound will be shared, let me know if not. So platform trials have several advantages over traditional trials, and they do make sense if you have multiple drugs in development for a single disease. So the focus is not on drug A or drug B, the focus is on the disease. Can we be very efficient, keeping patients at the center, understanding their urgency? Can we be efficient and work together with multiple companies at the same time to test multiple drugs? And so we worked again with multiple companies, the FDA and different stakeholders to try to start something new that would basically harness the power of collaboration. And that's how this works. The fight against ALS, the first ever ALS platform trial, designed to accelerate the development of effective and breakthrough treatments for people with ALS. So what's different about the Healy ALS platform trial? Instead of testing just one drug, the Healy ALS platform trial evaluates multiple treatments at the same time. This means more opportunities to find groundbreaking therapies in a faster timeframe. Imagine having to build a new dock every time a different ship came to port with new treatment cargo. This is how traditional trials work. In platform trials, there is one dock to receive all the cargo and connect the treatments to their final destinations. So we built a dock in 2020, and since then we have continued to grow. And so far we have tested seven regimens. Every regimen is a different drug. And in order to design this trial and maintain this trial, you need a very large collaboration, because it means that so far we have worked with seven companies. We have two more that will start in 2025. We have enrolled over 1,300 participants at 70 enrolling sites. And so over the last few years, I learned a lot of new skills. Again, I would say that PM&R prepared me well, because concepts like collaboration, understanding the different disciplines and what everyone brings to the table are core to PM&R. And now my stakeholders may be different companies, attorneys, people who work in different areas, safety experts, et cetera, but it's the same concept of bringing everyone together, keeping the patient at the center. And so one thing that I would like to highlight is that by doing this work with over 70 sites, we really had great opportunities to meet with many people. A few people trained in PM&R, a lot of neurologists as well, all people focused on ALS. And one feature that kind of came to me as a surprise, because I'm not, never kind of, you know, being a person who loves to do public speaking or do webinars or be out there. One thing that became really clear to me over the last few years is that it's important to communicate what we're doing and to talk to patients and have their input. And so we have a fantastic patient advisory committee and we have many opportunities for connecting with the community. And so we actually started the first patient navigation role for clinical trials in ALS. And that has been really fruitful. Again, learning from the patients and doing this as a collaboration. Just to end, you know, this trial is not just about testing drugs. One of the main goals of the trial is actually to advance ALS science. And that's really in recognition of the time and effort that people living with ALS give to research. You know, they want to help the field, but I will say in my experience, most people with ALS participate in research out of generosity and altruism, wanting to contribute to the field. And so what we're doing here, we are doing a lot of biomarker research, trying to learn more about ALS. Again, aside from any specific drug, we actually have a shared placebo pool. And so the shared placebo pool is essentially a natural history study that's embedded in the trial and continues to grow. And that's where we're getting a lot of biomarkers, bio samples of different types. And we can share that with the community for scientific learning. In fact, we are about to release a large amount of data and samples, again, following the principles of open science from the placebo participants in the first few regimens, since they're no longer be part of the trial. And the other thing I want to say is that just like, you know, we follow our patients and we adapt and adjust our treatments based on the current functional status and what the goals of the patients are, the same happens in research and in trials. So the heliplafon trial that I just described is more a late stage trial, more in the phase two slash three phase, you know, of drug development. And then we realized that now in 2025, we have many more drugs that are coming down the pipeline at an earlier stage, more in the phase one to two stage. And that's because of some of the recent breakthroughs and scientific discoveries over the last few years. And so we already created a second trial called ALS MyMatch, which is actually led by a colleague Dr. Suma Babu. And this is very much biomarker focused, trying to tackle the fact that ALS is heterogeneous. And so we probably need to understand more and learn more about the different subtypes. All of you who have seen patients with ALS, you know that there's no two such thing as, you know, no two patients are the same. And so we're really trying to understand by using biomarkers in a careful way, all these differences to better target drugs, because ultimately I think that by better targeting drugs, we'll be able to get new drugs to market. And this is actually we're launching this week, which is why I wanted to at least mention that as a way to say that we continue to grow and do new things. And just like we continue to adopt our clinical framework, we need to continue to adopt what we do in research. So I just want to end by saying that all of these is the result of intense collaboration, not only with pharma, but also with a lot of companies, a lot of foundations and patient groups. And that's another thing that I feel like we learn a lot about in physiatry training, you know, family meetings, community events, ways to interact with patients. That's actually a set of skills that also work very well when you do large multi-center trials. And with that, I'll end here. Thank you. Thank you, Dr. Bancone. We will now have a question. Sorry. We will now have a question and answer session. And I will start off with a few questions that we drafted up. One, this will be for anybody. What unique value do physiatrists bring to interdisciplinary care teams for motor neurone disease? How can we better define or advocate for our roles alongside neurology in different clinical models? Now, you guys touched on that a little bit, but do you want to expand on that at all? Sure. I can go ahead and kick us off. I think that it's been mentioned a couple of times already in this session, but we're collaborative by nature, by our DNA. And collaboration is precisely what is needed to provide the most cohesive support for the patients that we serve, but also to advance the field. We can't work in silos. We have to reach out and collaborate when we have patients that need access to research. We need to be able to help guide them to opportunities. And for a patient, Dr. Bancone, patient Dr. Rad's presentation highlighted so well the many facets of care. It's really unfair to the patients and the families to make them responsible for keeping track of recommendations from every single organ system that's involved. The better care is when a plan of care is cohesive and delivered as one unified plan of care for that patient and family. And that's what we do best. Maybe I'll just add to that, that, I mean, I completely agree. One thing that I would like to make sure that we touch upon is that there's never been a better time to work in neuromuscular medicine. And I know my colleagues alluded to some of the new treatments that have already come down the pipeline now. I mean, my work is mostly on developing new treatments, but the reality is that there's been already a few great success stories and some real breakthroughs, certainly in SMA, you know, the development of new treatments, gene-targeted treatments has completely revolutionized natural history. In ALS, we're starting to see that with the SOD1 gene and antisense oligonucleotides targeting that particular gene, which is only carried by a small minority of patients, but nevertheless provides a great proof of concept that a disease can actually be effectively modified if you target the right mechanism. And all of this to say that, you know, whenever you have these targeted treatments that change disease course, the need for physiatry and care along, you know, probably along, you know, a longer lifespan at this point, thanks to these interventions, that need for physiatry continues to grow. So I just want to say that, especially for the people who are in training, we do expect now, you know, the technology that's coming out in terms of gene-targeted treatments, I do expect that the natural history will continue to improve and with that, the need for physiatry will continue to grow. Dr. Rai, do you want to add anything? I would really be echoing. I absolutely agree. I can even speak from experience for SMA, you know, just the number of SMA patients in my clinic as, you know, the first novel treatment with Neusinersen and ASO was in 2016. And, you know, so we're not at the point yet where I'm seeing a type one patient yet, but, you know, 16 years from now, you know, 16 years from, or 18 years, it's when we take them from 2016, I'm actually really excited for the day that I get to see a patient that would have passed away at age two, right? So we're going to be, it's going to be entirely unique, right? So this patient that would never have existed in an adult population will now be seen. And I don't, I'm very passionate about it. So I don't know if I can express, you know, how unique that is, how pivotal that is, that you would never have seen that patient in any other clinic, you know, prior to that. And so I really do think that the world of physiatry will change. I work a lot also with Duchenne muscular dystrophy and Becker's, and we've seen more novel approaches there, some even for adult management. And as I go to tables where they're usually neurology led, and I'm sitting there, oftentimes it's like, okay, great. Now, can you put your rehab hat on? Like, you know, they sometimes like to think of my neuromuscular hat as neurology, which I don't think is fair, but then they're like, okay, well now how are we going to manage, you know, this chronic hip pain that never existed before, right? Or it's like, no, they just didn't age long enough before for you to see this. And so I think now there is a big movement in a lot of these conditions and a lot of these organizations to find rehab providers that want to see these conditions because we're going to be the experts in managing their disabilities. And so there's more need. So highlighting the fact that I think the growth and need of physiatry in this field will grow. And so, you know, I love being able to diagnose them and I love that aspect, but the truth is, is I really love that I get to see them from the beginning and before for many of these diseases, the end, but now just a lifetime. And so I think that that's really, really unique. And I, again, I think physiatry is going to be more sought after for helping to manage these conditions. And again, there is, you know, there is a unfortunate decline, at least in the state of Washington in primary care providers. And I can't speak nationally, but with primary care providers having more burden, less understanding, understandably of these chronic diseases, they're not going to be able to take on the management of an aging SMA patient or an aging Duchenne patient. And so I just also wanted to highlight that point that that not only should be us, I think it's also going to have to be us. Thank you. It does seem like with the advent of all these newer treatments that we're going to be dealing with older SMA patients. So that brings in another role for physiatrists. All right, here's a question that I am personally interested in. Fellowship or no fellowship? And how can someone without a fellowship be more engaged in neuromuscular care? Well, I'm happy to take a stab and I'm sure there are a variety of opinions. So I'm team no fellowship because I fell into neuromuscular physiatry. As I already said, it's an all hands on deck situation. There just simply aren't enough physiatrists that are currently caring for these individuals. And so I don't think it would be too difficult for someone to shift from general outpatient physiatry and learn the ropes of caring for patients with motor neuron disease. I teach a lot of residents in my practice and I often will be talking about what are the pros and cons of pursuing a fellowship. I think there are pretty compelling reasons why an individual would want to pursue a fellowship, particularly if they have a keen interest in an academic or a research focused career, if they would benefit from having the mentorship that would help them find a specific kind of position that's with a more academic focus. I think those are really good reasons to pursue that. But for providing clinical care, I don't think that it is necessary and it could be a limiting factor that could deter people from providing the care that they're well prepared to do in their physiatric training. Thank you. Anybody else want to add to that? I can add a little bit. So I absolutely agree with Dr. Howard. So I'm not saying you absolutely have to do a fellowship, but for those that are in residency, and so I do think that you can pivot and work in neuromuscular using the skillset. Again, that's what we're trained at as a physiatrist. For those that are at the point where they're kind of debating, do I want a career in neuromuscular? And so now they're trying to decide fellowship or no fellowship. I like to think I'm not too far removed from that, although I'm going on nine years. And so I can share why I pursued it. And so for me, it was a unique set of reasons. One, I wanted to be able to diagnose. And so I wanted to be able to go back. And when I was looking for my job, so here I split referral. So if they come to just neuromuscular, we don't have like your neuromuscular physiatry, and this might be unique, your neuromuscular neurology, right? Like the pool is neuromuscular. And so how I like to think about that is, when I was talking to some residents is, so think about sleep medicine, right? So your sleep medicine doctor may be a pulmonologist, your sleep doctor medicine may be an internal medicine doctor, your sleep doctor medicine may be another specialist. But at the end of the day, they're talking to you about sleep medicine, right? And so regardless of what their primary was. So I do think as physiatry, we're better at our job, but at the end of the day, at least right now in my practice, I only see neuromuscular. And so I wanted to be able to diagnose. I do think in finding jobs, I do think that's helpful as well in terms of being able to bring yourself to the table and say, well, I'm no different because I did the same training. I also think, and this might just be my personality where I'm like, I like to focus on very specific things. And I really just really wanted to work in MDA, ALS, is that having a better, in a world where there's so many different evolving treatments, I felt that a fellowship giving me the tools to better understand how the diagnosis happens allows me in my multidisciplinary clinic to kind of recognize like, was that the right diagnosis for them? What am I missing in this? Like pulling back from some zebras. And so with the advent of novel treatment, I have seen patients who walk in and tell me they have SMA and I'm like, no. And, you know, cause they want treatment and I'm diagnosing them with different things. And I'm not, it's not to say that a general physiatrist can't do that. It just really depends on your training and your exposure to that, right? Like what type of residency did you go to? Did you get exposure to diagnosing neuromuscular disorders? I think the clinical management, it's already key. Physiatry is, you know, front and center in that. But how much of that, you know, of that task of rediagnosing or re-examining things that may not be under the rehab umbrella. I also get to do clinical research. I have the privilege of, you know, being a site for the Healy trial and, you know, working on unique SMA clinical trials. And I think that that fellowship really did prepare me for that because, and again, maybe it's unique to what training you did, but I didn't have that in my residency where there were mentors doing clinical research. So I really needed the fellowship, fellowship for that. And in a world where, you know, board certifications become more mandatory and, you know, I worry that the general physiatrist feels like, well, no, I'm just gonna refer them to somebody neuromuscular. I mean, we see that with neurologists, right? Like in the community, like as soon as they think it's, you know, ALS or muscular dystrophy, they don't keep them on, right? They like refer them to the subspecialty. And so I would hope that the fellowship wouldn't intimidate a rehab resident. I guess I would wanna draw more attention into why the fellowship is well suited for a rehab resident and what a rehab resident, why a rehab resident in many ways would be better at it. And, you know, I can share anecdotally, I think this would be okay. So I did my fellowship at the University of Michigan and one of my mentors is Dr. Ava Feldman and she wasn't there on my interview day. And so when she started, she was like, I would have said, no, I don't want any rehab in my fellowship. And at the end of the year, she was my hugest champion and was like, I can admit I was wrong. And I still remember that dinner because her husband was like, I can only count on like barely my hand when she's ever said that. And so I do think that, you know, I'm a little biased in some of the unique things that I'm able to do that I don't think I would have the ability to do in my practice without the fellowship and without some of those mentors. And I just want to, you know, tell those residents that you can do this, right? Like you can do the fellowship, like you shouldn't be intimidated by that, but that's just my thought. I think I probably will add a little bit to that, that one of the aspects is, you know, the differential diagnosis. I think fellowship can help, for example, when we have predominantly lower motor neuron ALS, and we are trying to tease out any possibility of like multifocal motor neuropathy or other inflammatory treatable condition. Another aspect that can help with the fellowship is sometimes for more expensive treatment, like for SMA, for example, or others, actually the insurance requires a neuromuscular provider to sign off or recommend the treatment. So having that neuromuscular head can help with this. And I think it's like, I always approach that, it really depends how much exposure you have in your residency, but also we can only diagnose what we know. So if many neuromuscular disorders are rare diseases, and now we have more than 800 genes associated with that, seeing the clinical presentations or atypical clinical presentations can actually help. So this is kind of my take on that. And I had very similar experience to what you mentioned, like when I started my fellowship, one of the faculty told me in the beginning, you will never be one of us, I guess. He wasn't correct, completely correct. So yes, we all kind of went through that, I think. It's rewarding at the end, I will say, when you prove them wrong. So I don't want anybody who's considering it to be like, that sounds miserable. It is very rewarding at the end. Can any of you comment on the importance of maybe being a member of the American Association of Neuromuscular and Electrodiagnostic Medicine as a means of getting more education and exposure to neuromuscular. Absolutely. I think this is such a small niche subspecialty that it's really important to have a network of colleagues that you can lean on. And the professional organizations, particularly AAM for those of us in the neuromuscular world, is a great place to forge these relationships that can be helpful as you develop in your career or as your career evolves. And as has already been said during this presentation, this is a rapidly evolving field. So even if I were smart enough to have done a neuromuscular fellowship early on in my career, so many of the treatments that I'm prescribing now weren't even developed. And so I never thought that I would be ordering genetic testing on my patients in clinic. And so having a professional network to keep up on the developments, but also to trade practical experience in managing new treatments is really invaluable in this field. I agree. Oh, go ahead. Oh, no, no. I was just going to say, I agree. It's a nice place to have the focus on everything you're doing. I think in AAPMR, I've seen more focus on trying to get more neuromuscular, which I appreciate. Earlier on, there were a few years where I was like, I love hearing about everything else going on in rehab, but there's not a lot of neuromuscular being presented here. And so I think having an organization. That being said, I also think in addition to AANEM, there are other conferences that I think people think of as neurology, but like World Muscle Society, Peripheral Nerve, MDA does their conferences as well, PPMD. So there are very unique conferences that, depending on how broad or how specific you are in your career, are wonderful in terms of getting further education and, as Dr. Howard said, learning about the novel treatments and then the research going on. I am a firm believer, whether you do it or you don't do it, so much of what patients want to hear is understanding what's going on. So I think we need to stay at least abreast of what's coming down the pipeline, and those specific conferences are really helpful for that. Thank you. I will pause for a second, and please, if anybody wants to jump in and ask a question, you can raise your hand or just come off of mute and ask a question. So we'll pause for a second here. So let me just ask a question, because when I was treating, I was basically an adult MDA provider, also a pediatric MDA provider, when I worked in New Mexico. And I started the Spina Rasa and gave first zolgensma treatment for SMA patients. And then I transitioned them from kind of like SMA 3s from my pediatric clinic to my adult clinic, MDA clinic. But what I wanted to tell you is, and I wonder if you are thinking about it, I always wonder if those SMA patients that we are treating with various basically treatments, either through SMN1 pathway or SMN2 pathway, are they going to kind of show symptoms like what we remember from post-polio, basically? Any kind of like, what are we going to see in the future, with respect to those surviving motor neurons? And I think we have quite a lot of experience as physiatrists. I wonder what you're thinking about it is. Yeah, as a community. So I don't have the privilege of seeing the pediatrics, because we have a wonderful children's hospital. But I do get the privilege of seeing them at 20, all the way to I think my oldest is 60-something. But I know that talking to colleagues, there's been not necessarily the post-polio, although that is a certainly interesting thought. And the re-innervation is more unmasking of the SMN protein in non-motor neuron nerves, because we know it's ubiquitous everywhere, and it's highly concentrated in the motor nerves. And we're talking about function, function, function. We know type 1s do have some cardiovascular symptoms, do have some cardiac abnormalities. We know there's metabolic derangement across the spectrum. So are we gonna start to see chronic illness, was my thought and wonder, like what other non-motor neuron symptoms are we gonna see manifesting? I would be curious in regards to the post-polio, for some of these treatments, since we're not seeing, since we're not, like in polio, they self-regenerated, but in this case, at least, you know, I'm not, I'm thinking, I always think about stability for my patients, but most of them, it'd be different than in the peds world, right? Where they have some motor neurons that probably, or more that are salvageable. In mine, I talked to them about stability and slowing down disease progression. So I guess I haven't really thought about, are there, is there any re-innervation happening? And if there's not any re-innervation happening, then would the true post-polio de-innervation process after so many years occur? It will be interesting to see, I think, especially in pediatrics. But no, I think the world of what this phenotype looks like will need to be studied, even the types that we call them. I mean, we haven't changed that yet, but we can't type them anymore by age of onset and motor milestone achieved. So we're gonna, there's gonna be just a lot of changes. That's really fascinating that you bring up non-motor manifestations, because even in the adult world, as we prolong life for patients with ALS who decide to transition to invasive mechanical ventilation, and I have patients that are 15, 20 years out from diagnosis the non-motor manifestations, the autonomic system involvement is really underappreciated and in many cases suboptimally managed. But we put on our PMNR hat and cape and we're all over recognizing autonomic dysfunction in these patients. And I think that is much to their benefit for managing the symptoms and improving comfort and quality of life. I always found, well, when I was in training, most of the neurology textbooks said these diseases were all painless. And then when I started practicing, everybody was complaining of pain, amongst other things too, fatigue, all kinds of stuff. So I do feel like PMNR really has a role in enhancing the quality of life. And you guys have all done wonderful work in that area. And I'm not being disrespectful to neurology, but I think they're a little more focused on a curative intervention and less concerned with the day-to-day life of the patients. And I think that's going to become increasingly important as we have older neuromuscular patients. So, I mean, when I was a resident, nobody talked about a man with Duchenne dystrophy because they were gone by 18, 20 years old. And now we have a lot of patients that are in their 40s with Duchenne and they have a ton of clinical problems. My oldest here just turned 50. So, hi. Yeah, and I do want to add to another opportunity that we have when we care for these people, especially long-term, is to leverage some of our colleagues from PMNR who went on to subspecialize in other things. And I just want to share a couple of anecdotal stories. I have a patient with ALS who was having a lot of back pain, but had to use a non-invasive ventilator to lay down. And because of this, when she went to her usual pain clinic that was run by a non-PMNR specialist, they basically told her that she would have to either, they couldn't do the surgery because she couldn't lie prone. And so the only option was to get surgery, which I thought was a little bit absurd. And so I called a colleague from PMNR who was willing to do the procedure in lateral decubitus. Again, while it's not the preferred position, it can be done as long as you put the patient at the center and are willing to do that. And instead of kind of going outside the usual. And on the same lines, we created a very streamlined pipeline for having patients get steroid joint injections to the shoulder if needed for pain. Because interestingly, some sports clinic are not accessible because they were mostly built for athletes. And so, you know, you do need to create a pipeline that includes access, I mean, physical access to the clinic. And also, you know, having the room that's big enough to accommodate a wheelchair and those type of things. And so I feel like there's a lot that we can do also in collaboration with other colleagues in PMNR who are not necessarily neuromuscular specialists, but are willing to partner. In fact, we have a few preferred people, you know, from spine pain, sports, et cetera, who are willing to work with our patients who admittedly may take a little longer in, you know, because they may need a little bit more attention and personalized care. And so I will say that that's also another way to have the specialty shine across its entire, you know, all the sub-specialties. I also add that, for example, the training that I received from PMNR helped me to facilitate for especially younger patients transferring, you know, from pediatrics to young adult clinic, for example, adaptive driving. That, you know, I really, my hat from PMNR really helped me to, you know, put those patients through the training, through basically appropriately, even think about aspects like that, which is very important for, you know, young adults going through basically to become independent. Just the whole idea of what can we help for them to, you know, become independent, you know, either for short time for ALS patients or for SMA patients or, you know, for other. And along those lines, I do want to mention that we had the opportunity to work with and that we had the opportunity to be leaders and really inspire kind of, you know, neuromuscular center care with our allied health professional colleagues, because, you know, that's also another important aspect that, you know, the care that neuromuscular patients need is more specialized in different ways, depending, you know, on the sub-specialty or sort of the type of therapy that we're talking about. You know, it's, you know, from traditional therapy to physical modalities, to cutting edge technologies, like, you know, AI-based intervention. So, I mean, I feel like we also, I think, have a responsibility of continuing to learn rehab, general rehab and, you know, and anything, any advances that happen in the general field in terms of also our rehab colleagues from physical therapy, speech therapy, and you name it, all the sub-specialties, because there is a lot more that we can do, but we do need to advocate for patient-centered care that takes into consideration the specifics of the disease. Because just like there aren't many physiatrists who are interested or specialized or care for people with neuromuscular disease, the same is true in the community when it comes to therapists of different sub-specialties. And so that's another aspect, I think, we need to continue to work on. Sabrina, I'll ask this of you, because you have such a powerful research background, what advice would you give to someone who is interested in neuromuscular research as a physiatrist? Yeah, I mean, I feel like that there's definitely a lot of potential there. I will say that I know that many ALS clinics and neuromuscular centers actually have a hard time finding faculty and staff. So certainly it's an area where, in addition to being exciting for all the reasons that my colleagues have explained and that I agree with, there's also a lot of need. So certainly there's no shortage of opportunities there. In terms of research specifically, and I want to go back to the question of fellowship, and I know that I discussed that previously with both Ileana and Nassim and you, Greg, in the past on different occasions, but, and I agree with what others have said, it all depends on your original training, so what you have learned specifically in training, and also the setting of care. And so if your focus or main interest is to provide, to work on research, I will say that more time spent in on sort of graduate education, whether it's a fellowship or a postdoc or a PhD or an MPH, you know, or any type of advanced degree, that helps in research, not because you, not that you absolutely need it, not that you cannot do research without it, but the more time you spend working in different areas of research and maybe learning skills like epidemiology or biostatistics or all the way to clinical science, all of that will serve you well. I always say that everything I do today, I didn't learn in any degree I had. And, you know, everything I do right now in research specifically, not so much in clinical care, but the research piece, which is four days a week for me, that I learned on the job by kind of, you know, combining my preclinical training and then sort of the research fellowship that prepared me for clinical research. But it's really, you know, material that's not covered in medical school or residency or fellowship for that matter. So if you're interested in research specifically, I think some additional research training, whether it's a PhD, a postdoc, a research fellowship, I think that's really helpful. Well, we're coming up on seven o'clock and that's 10 o'clock for you guys on the East Coast. Are there any other questions or areas you people want to look into? Well, I found this personally very stimulating conversation. And I want to thank all of our speakers tonight and I hope you enjoyed this conversation. And I hope you have a great rest of your day. I want to thank all of our speakers tonight and thank AABM&R for sponsoring this. Any other items for conversation before we close? I just wanted to thank everyone for joining. And, you know, these type of sessions aren't mandatory. So I love seeing people on it because it shows that you're interested and you want to know more about how to take care of your patients. So I really appreciate you joining and listening to us go on about what we enjoy doing and hopefully you enjoy doing too. Yeah, and I will say that there's a growing number of us working in different areas of neuromuscular medicine and other people work more on the muscle front and other people do more research or, you know, there's all sorts of people out there. And every once in a while we meet, you know, either at conferences or virtually. So if you have, if you're interested, please join us. And I think it's great to continue to mentor and hopefully more people will want to join these, you know, people who are interested in neuromuscular medicine from physiatry. Thank you so much for having us. And I think it speaks volumes about how passionate we are to this field that Sabrina is showing up almost at 10 o'clock at night. But I do like to think that we have the coolest teams and the coolest colleagues in any subspecialty in PM&R. And I'm told by the neurologists that the neuromuscular neurologists are considered the coolest ones too, but I don't know. I've heard that too. Okay. Yeah. I don't know if that's true or not, but. Yes, they definitely are some of the nicest. So I'll give them that. Thank you so much for having me. I hope you all enjoy the rest of your evening. And thank you everyone for dialing in and thank you to our speakers tonight. Thanks everyone. Have a great night.

AAPMNR

physiatrists

neuromuscular medicine

ALS care

interdisciplinary teamwork

spinal muscular atrophy

functional assessment

respiratory care

Healy ALS Platform Trial

gene-targeted therapies

patient-centered approach

collaborative research