2022 SMA Research & Clinical Care Meeting: What to Expect

WATCH TIME: 14 minutes

Transcript below. Transcript edited for clarity.

Mary Schroth, MD: Hello and welcome. I'm Dr. Mary Schroth, chief medical officer at Cure SMA.

Jacqueline Glasscock, PhD: And I'm Jackie Glasscock, Vice President of Research at Cure SMA. We're excited to talk a little bit today about the 2022 Annual Research and Clinical Care Meeting, which will be held June 15, through June 17, in Anaheim, California, at the Grand Californian Hotel.

Mary Schroth, MD: So we will have three days of research and clinical care. We want to point out to you that the first day on Wednesday, June 15, we will have concurrent sessions, one session will be the clinical care session. And in parallel to that will be a basic science session, the clinical care session is will be accredited for multiple disciplines. For the duration that day, the remainder of the meeting will be research and clinically focused with a lot of collaboration back and forth between the interplay of research and care. We look forward to seeing you please join us.

Jacqueline Glasscock, PhD: This is our first kind of post-pandemic—although I don't know if we can say post quite yet—but this is our first in-person meeting since 2019. And so we're very much looking forward to having the whole community be back together in person. I'll also mention that the research and clinical care meeting runs in parallel with our Annual SMA Conference that's intended for individuals affected by SMA and their loved ones, caregivers, and families, etc. So, there's opportunity for interaction, although the meetings are to two separate events.

One of the themes that we're emphasizing this year is kind of an emphasis on basic research and a commitment to continued investment into basic research with three approved therapies. For SMA, we've certainly made really historic strides and treatment and things that were that are available to people living with SMA. But our work is certainly not finished and to continue to ensure a robust drug pipeline. Basic research into key biological questions is very important, and so we're continuing to be sure that we continue that investment as Cure SMA, and that we promote basic research in the community as a whole. Mary, do you want to say anything about clinical care?

Mary Schroth, MD: Absolutely, absolutely. So we are focused on research. But we also know that even with these three treatments that are available, that care is critically important, you have to have excellent clinical care in order to optimize the outcomes for these treatments. So part of this meeting is also dedicated to providing updates regarding clinical care and management. The clinical care session of the research and clinical care meeting will occur on Wednesday, June 15, and that will occur over the entire day.

Our special sessions are focused on ethics. Our clinicians, very much talk about ethics and some of the challenges involved with making decisions, especially in view of the fact that we don't have all the information, all the research that we need to make the best decisions about who should be on which treatment and how to use these treatments, should treatments be used together? As Jackie said, we're we're not done, we don't have a cure for SMA, we have three treatments, and they're and they're very good. But they're not curing this disease. And we still have a lot to learn. So the clinical care session really allows clinicians to get together, talk about the issues that are really important to them, and teach each other about where we are with clinical care.

I'm just going to go on a little bit more. In addition to ethics, we also will be focused on nutrition this year, we'll have an hour presentation by several nutritionists, three nutritionist who are part of medical advisory council, plus additional abstracts that were submitted to the conference related to nutrition. So we're very excited about that. And we're going to talk about newborn screening, we have an hour long session on updates regarding newborn screening around the world. I'm going to turn it back to Jackie just so that she can talk more about the Thursday morning session that we're really excited about.

Jacqueline Glasscock, PhD: Thanks! We're having a what we call a special session. So the rest of the talks in the meeting are abstract-driven, they're all selected from submitted abstracts. But our special session is an invited speaker session. And so we have curated a session that we're calling reverse translation, so this idea of taking what we're learning in the real world and in the clinic and then allowing that to inform our basic research questions. We have an exciting lineup of clinicians and scientists that will be speaking on various aspects of clinical and real world observations, kind of over the treatment and age and disease spectrum as well, so what we're learning from children who were diagnosed and treated early and what we're learning from adults who are treated later in an age and disease progression. Then, how those things that we're learning and what questions do we need to ask to answer the underlying biological questions of how patients are responding to treatment and such.

Following those talks, we will have a panel discussion, where everyone in the audience in the audience will be both clinicians and scientists for that session, we'll be able to ask questions interact with the speakers, and we're hoping to really promote a good dialogue that will carry on even after the session and the meeting ends, and inform people's research and the kind of thoughts about, about the science moving forward. We routinely hear from scientists that they're very curious about what is happening in the clinic, and I think the clinicians as well, are curious about what's happening in the lab. So it's a chance to really encourage that dialogue.

Mary Schroth, MD: The crosstalk that crosstalk is so critically important. And I'm again, I'm just really excited for this Thursday morning session. So one of the very unique parts of our research and clinical care meeting is that it occurs in parallel with the Annual SMA Conference, which is our family and community conference, where all of our individuals affected by SMA, their family, their friends, their caregivers all come together to hear special sessions directed to them as an audience.

Then, the researchers and clinicians have an opportunity along with the patients and families in the evenings to get together socially. So one of our most exciting and fun events is that on Thursday evening, we have a meet and greet. The meet and greet is in a sort of carnival-type setting—we didn't share with you that our meeting is at Disneyland this year. So we're really excited about that as well, because it is truly where the magic happens. Our families enjoy being there, and our researchers and clinicians also all enjoy being there. Then, when they're together, it is just incredible synergy and energy in the room. Jackie, do want to talk about the event, that's really special?

Jacqueline Glasscock, PhD: We have what we call our community friendly poster session. And so this is an opportunity for clinicians and researchers to make a poster that's intended for individuals with SMA and their caregivers, all those in attendance at the annual conference to view. It's a little less, kind of, technical than the posters for the research and clinical care meeting. But they contain a ton of data on things like clinical trials, basic research going on, and the latest in clinical care.

We invite about 40 people to participate in this session and the families are able to go around, visit the poster, and speak with the scientists and clinicians presenting them. And it's just a really great way to promote dialogue between the clinicians and scientists there and the the individuals attending the the annual conferences, well, we get great feedback every year, people really enjoy it on both sides of of the equation. It's just a really fun time and a chance for the clinicians and scientists to get feedback from patients and families on what matters to them and what's important to them and the clinicians and researchers to ask questions of the families and vice versa. It's a really great opportunity. We introduced this in 2013, and every year we get emails from people asking if we're doing it again, and so of course, we're pleased to continue the tradition of the community poster session.

This is the longest-running and largest meeting dedicated to SMA, and so is the place for all of the clinicians and researchers who are, you know, in this space together, and I really think that it provides a great deal of networking opportunities and that sort of thing. The content of the meeting is great as well. So we all have an outlet, Mary spoke to the clinical care session, but we'll have updates and basic research. So we'll be able to learn and kind of what the state of the field is from the basic science perspective, we have updates in clinical research things like outcome measures, patient reported outcome measures, newborn screening, that sort of thing.

Perhaps the most anticipated session of the meeting is the drug development or clinical trials session. We'll be able to hear the latest data cuts from each of the companies and industry representatives who have drugs and clinical trials. And so that's a very exciting session and a session that a lot of people look forward to as well to get those those latest updates on especially on some of these drugs that are in later stage clinical trials.

Mary Schroth, MD: I would add to that, that, also critical to this. To come into this meeting is the opportunity to network to network with researchers who have been in this field for a long time and with newer researchers, who were just learning about SMA and how to leverage their areas of interest into something that could produce potentially a treatment down the road. Similar for clinicians, there is an incredible network of clinicians focused on SMA care, and having a better understanding of what it means to have SMA for our patient and family community. So I think the application network along with the educational things that we provide is just tremendous. And for the clinical care session, we also offer continuing medical education and continuing education credits for physicians, nurses, pharmacists, nutritionists, physiotherapists, occupational therapists, genetic and genetic counselors, all of these, this accreditation is has been applied for. And we're in the process of having that by the time of the meeting.

Jacqueline Glasscock, PhD: And I'll just add on to that, that we also will be having two poster sessions with the research and clinical peer meeting. And those are also great opportunities to, to network to talk to people about their clinical practice and their science. And one of them comes with a cocktail reception as well. So that adds to the allure.

Mary Schroth, MD: So in my role as chief medical officer curious to me, I'm looking forward to our in-person conference, because I haven't seen anyone for three years—it feels like it's been for ever. So having the opportunity to connect with my colleagues around the world, to have those conversations have those conversations that are not necessarily as much fun or more difficult to have, when we're doing via video chat or over the phone. That being connected. It's just so important. But then not only just our not only our professional community, but also our patient family community, because they're the ones that teach us the most about SMA. I've been going to this conference for a long time since 1988. And every time I go, I learn new things. Families teach me new things, colleagues, colleagues teach me new things. It's just an incredible experience. I cannot say enough about it. From my first meeting, I have not missed it because it was just something that I felt compelled to attend every year.

Jacqueline Glasscock, PhD: Yeah, I read echo what Mary said, and I'm really excited to be able to be back in person and to see everyone again, I think the SMA community is just such a unique and rare community not only rare in the rare disease sense, but there's such a sense of community and support and wanting to learn from everyone. And really just, it's a great atmosphere, atmosphere of learning and atmosphere of support and connection and just fostering that sense of community. So I'm really just looking forward to being able to to be a part of that again, and then they can't really capture that in Zoom, as Mary was saying. So just being back in person learning, the latest science and clinical updates, but also just seeing everyone and building that sense of community as well.

Mary Schroth, MD: Yes, like, I can't wait to have our community again, together again. It's just it's something you can't replicate. You just have to be there.