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Welcome to Connecting ALS. Today we take a look at where the fight against ALS currently stands.
Connecting ALS is a weekly podcast produced by The ALS Association in partnership with CitizenRacecar. We aim to discuss research and technology developments, highlight advocacy efforts, and share the personal stories woven through the community.
This transcript was exported on Jan 11, 2023 - view latest version here.
Speaker 1:
I think there's an increasing body of evidence that the solutions exist on this planet. They are with us and they are buried in the minutiae of big data. And it will take enormous computing power and collaboration to unlock some of the keys that will lead to these solutions.
Jeremy Holden:
Hello everyone and welcome to Connecting ALS. I am your host Jeremy Holden. 2023 is shaping up to be a big year in the fight against ALS. In April, the US Food and Drug Administration, is set to decide whether to approve Tofersen for the treatment of SOD1-ALS, a story we will be following closely here on Connecting ALS. In the coming weeks we will talk about the fight to make Tofersen available, how antisense technology could show a path forward in treating ALS more broadly, efforts to expand access to clinical trials, and efforts around the world to make ALS a livable disease.
But before looking ahead, we want to take stock of where we have come in the past year, and to help us do that, we are turning this week to Scott Kaufman, chairman of the ALS Association Board of Trustees. Well, Scott, thank you as always for being with us on Connecting ALS.
Scott Kaufman:
My pleasure, Jeremy, nice to see you again and hear you.
Jeremy Holden:
It's an exciting time. We're bringing this episode to listeners the beginning of January. I think we all say this, I get this feeling every year. It's hard to believe it's 2023. Before we do look ahead though, the association put out in December as it's done for the last several years, a report kind of wrapping up some of the victories and some of the progress that had been made during the previous calendar year. So as you reflect back on 2022, what are you proud of? What do you think worked well, and really sets us up for success going forward?
Scott Kaufman:
Well, it's hard to talk about 2022, and not talk about Relyvrio, the third FDA approved drug on the market today.
Jeremy Holden:
Sure.
Scott Kaufman:
The hope that comes along with any new therapy, and whether it's administered as the sole therapy, or as in many cases for people living with ALS on top of riluzole or something else, it appears to have effect. And so that in and of itself, given the longevity of this disease, and the lack of therapies and certainly the lack of a cure at this point, any step forward in the scientific world, is a huge step forward. I also think about what happened with the Congress, the act for ALS at the end of last year, and some of that money now coming into the market. So it's a time, I think great opportunity and great optimism. Of course, in our world, optimism is always guardrailed by the reality of the challenges of this particular disease.
But I think between what's happening in the research community along with what's happening in areas of assistive technology, this notion of making it a livable disease and extending not just life, but the quality of life is something that we've taken great strides on in the last several years. And it always helps to have new therapies to aid and abet what's happening over in what I'll affectionately refer to as, gadget land around assistive technology.
Jeremy Holden:
Sure. You mentioned making ALS a livable disease, that the commitment that the ALS Association has made. It dovetails nicely. You reference the act for ALS and coming out of that, the National Academies of Sciences announced a research project looking into the fight against ALS, and specifically guided by the concept of making ALS a livable disease. How do you reflect on how that concept has kind of percolated out into the world?
Scott Kaufman:
Well, I come from Silicon Valley in the venture community where you place a lot of bets initially, but you always want to keep some powder dry in the event that any one of your investments is really showing progress. And so the act for ALS, which is over a five year time span, includes money not just for a new original research, but also for early access to existing therapies. And I like this notion of playing the long game in a sense of making sure that the money is still available when something becomes promising. That we of course measure that against the tremendous urgency of trying to get to a solution right now for everyone living with the disease today. So it's a challenge, but I like the way it's been structured and am hopeful that as the community says, more shots on goal will result in another breakthrough of one form or another, and then when that happens, the reserves and the resources will be there to pursue it aggressively and bring it to market.
Jeremy Holden:
Yeah, I want to talk about some of those shots on goals, and one in particular that's on the short term horizon. But before we get to that, three new certified clinics came online in the past calendar year. Another 30 are under consideration. So when you look at kind of expanding the map of multidisciplinary care, and the way that the community can access the best care available, what does that say about the progress that's been made, and where we're going in that front?
Scott Kaufman:
Yeah. One of my driving mantras is that the zip code you're born into shouldn't determine the quality of care that you get. And if you think back just 10 and a half years when my son was diagnosed, and it was still called Lou Gehrig's disease, and look at the map today, the increasing awareness of the disease now referred to as ALS, thanks in large part to the ice bucket challenge, but also the ability to identify it earlier. So you've got a shorter path to diagnosis with greater awareness. And then the clear research about the impact of multidisciplinary care, and the big push forward to certified treatment centers of excellence, feel is that the entire medical community is sort of galvanizing around, what it takes to be smart about how to diagnose, and how to ultimately treat the disease. And so I like to say that we're always punching way above our weight.
We're still an orphan disease and that's a good thing. And when it comes to getting drugs approved, that we get the more considered care and the ability for people to broaden the horizon of what efficacy looks like in the way of treatment, or I should say in the way of clinical results. But just the overall awareness level is increasing. And whether it's because people are getting diagnosed more quickly, it just feels to me, I hear about people getting diagnosed more and more. Now, that's a terrible thing. And obviously we'd like the disease to go away. We'd like to close the ALS association as soon as possible. But I think in some respects that's also an indication that there is an enlightened sense of focus on the disease, and that can only benefit people that are going to be dealing with it in the course of their lifetimes.
Jeremy Holden:
Talk about Relyvrio, the third drug for the treatment of ALS big win this year. You talked about it at the top. We already have another PDUFA date on our calendar that we're going to be talking about over the coming months. Tofersen is set for a decision by the end of April. Dr. Kuldip Dave, has been on the show talking about the robustness of the drug treatment pipeline. So castor gaze to the future. What are you hopeful for? What are you excited about in 2023?
Scott Kaufman:
Anything we can do to get to better biomarkers, and an understanding of the genetic component of this disease is certainly going to be beneficial in trying to figure out what's happening with the other 85% of our community, where there is sporadic onset. And so I'm hopeful that, with each step forward, that scientists can build upon that in one way, shape or form, and very hopeful that Tofersen will receive a very swift approval process, back out to the community.
Jeremy Holden:
You were on the show last year, and one of the things you talked about was the role that you see big data playing in the future of the fight against ALS. What have you seen in the past year that encouraged to do that, the community is starting to get that the scientific community, the assistive technology community, all the different components of the fight against ALS, that they are starting to embrace opportunities to lean into big data.
Scott Kaufman:
Well, it's been a big year for big data on several fronts. And that's not just conceptually big data, but better storage technologies, better access technologies, artificial intelligence, all these buzzwords, machine learning and artificial intelligence coming to the forefront. And I think there's an increasing body of evidence that the solutions exist on this planet. They are with us, and they are buried in the minutiae of big data. And it will take enormous computing power and collaboration to unlock some of the keys that will lead to these solutions. And so what I'm seeing... And I can't give you specific examples, and a lot of it is considered proprietary, but what I'm seeing is a lowering of the barriers of the silos of the keepers of that data, that there's an understanding of the importance of sharing and collaborating in both the information that's being derived in each silo, and then what happens when you combine some of these silos and get to a better place.
We're also getting better at more centralized information, more data capture coming in at onset when someone is first diagnosed. So between what tablets can capture in real time for a brand new patient, versus what has been locked away in vaults over decades, I'm hopeful that the informatics element of the solution are really coming to the fore. And it's getting cheaper to store the information. It's getting cheaper to capture the information. You're seeing more work with accelerometers and hopefully all of the backend data that's captured in clinical trials, all leading to additional insights. And then somewhere, someone is going to crack the code on something. And as you know, I don't care where it comes from, and I don't care who gets credit for it, but let's get those issues to the table, get them dissected, and get on with the business of solving this insidious disease.
Jeremy Holden:
And then we can all celebrate together as one community. Scott, before I let you go, about the important work that you have in front of you, any closing thoughts as we look ahead to the fight against ALS in 2023?
Scott Kaufman:
Yeah, I'm thinking about the urgency that we have to come to the table with. It's great to celebrate the wins, and we do, and that's an important part of being part of an organization. We also lost a lot of great people last year, and so I want to maybe end on a slightly more somber note, but just as a reminder to everyone that I work with in the ALS Association, all of the staff and all of the volunteers, we are deeply committed to bringing urgency to everything that we do, and understand that every day is precious. And the one silver lining in this disease, and I'll quote an old Joni Mitchell song, "Don't it always seem to go that you don't know what you've got till it's gone." When you watch something being taken away from someone you love and someone you care about, and you watch almost everything get taken away, and you come to understand just how important it is to appreciate everything that you have. And I think that notion of those that can fight the fight will continue to do so until this disease is eradicated.
Jeremy Holden:
And now, I of course have the difficult task of wrapping up the interview and having to talk over those beautiful words you said. Scott, thanks for the closing message and thanks as always for your time today.
Scott Kaufman:
Thank you, Jeremy.
Jeremy Holden:
I want to thank my guest this week, Scott Kaufman. And be sure to check out the ALS Association's year end report for a deeper dive into the progress made in our fight in the past year. And we can share a link to that in this episode's show notes. If you liked this episode, share it with a friend. And while you're at it, rate and review Connecting ALS wherever you listen to podcasts. It's a great way for us to connect with more listeners. Our production partner for this series is CitizenRacecar. Post-Production by Alex Brower, production Management by Gabriela Montakeen, supervised by David Hoffman. That's going to do it for this week. Thanks for tuning in. We'll connect with you again soon.
ConnectingALS_010423_Ready2_YouTube (Completed 01/05/23)
Transcript by Rev.com
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