TranscripT
Ashley Harris Whaley (00:00:03):
Hello everybody. Thank you all so much for joining us this afternoon. We're so happy to have you here. We'll just do a super quick round of introductions. My name is Ashley Harris Whaley. I'm the Director of Adult Programs here at CPF, and I will be moderating this afternoon with my three trustee colleagues and panelists here, and I'll let them introduce themselves. Jen, you go first.
Jen Lyman (00:00:38):
Who do you want? Do you want me to go first or you want Rachel to go first?
Ashley Harris Whaley (00:00:41):
Whomever wants to go.
Jen Lyman (00:00:44):
Well, I'll let Rachel go last. I'm Jen Lyman. I'm the Director of Clinical Recruitment and Community Resources for CPF, and I'm also the mom of a 21-year-old with complex cerebral palsy.
Sarah Philbin (00:01:01):
Hi everyone. I'm Sarah Philbin. I'm the Program Director of Translational Science at CPF and a health services research and implementation scientist by way of doctoral training. And I also am an adult with CP.
Rachel Byrne (00:01:15):
And hi everybody. It's so exciting to have so many of you joining us here this afternoon. My name's Rachel Byrne. I'm the Executive Director at the Cerebral Palsy Foundation, and I'm thrilled that we can start talking about this initiative on what we've been planning for quite a long time at the foundation. And for those of you that don't know, my background is as a physical therapist, and I've been now with the foundation almost 12 years, believe it or not. And yeah, we'll get into it, but thanks, Ashley.
Ashley Harris Whaley (00:01:45):
Yeah, thank y'all so much. Also, I don't think I said this, but for what it's worth, I'm also an adult with cerebral palsy. So we've got all kinds of representation on this call. But thank you and welcome, and we're happy you're here, and we are excited to dive in. So the first thing up for us is to talk about why? Why are we doing this? Why are we here? Why are we kicking off this program? And why are we doing it right now? So Sarah and Rachel, I'll leave the floor to y'all for a little introduction.
Rachel Byrne (00:02:26):
Sure. No, thanks, Ashley. And I think it is a really good question. Why a cures initiative? And we're going to dive in this afternoon about even what we mean by cures. And you'll notice that there is an S on the end of that, which sometimes doesn't feel grammatically correct, but it's because cerebral palsy is such a complex condition, but also is very different for everyone. We often say if you've met one person with cerebral palsy, you've met one person with cerebral palsy. And obviously as an organization, we are committed to accelerating research, clinical implementation, advocacy and policy, and education and training across the lifespan. But part of that is thinking about, well, what's the pipeline that's going to move forward breakthrough interventions? And breakthrough interventions can mean anything from prevention of cerebral palsy. It could also mean what can we do to actually change the trajectory of somebody's mobility?
(00:03:17):
I think a lot of the time people think about CP and think about mobility, but we also want to think about communication. We want to think about vision. We want to think about reducing pain. We want to think about some of these things that really are instrumental in improving somebody's healthcare. And when we started looking at that, we were like, oh my gosh, there is not a cure pipeline for cerebral palsy. There is for nearly every other disease and disability. And this isn't an erasure conversation. This is absolutely thinking about how do we accelerate this pipeline and give people options? Because at the moment we know there are not enough options available to individuals. If you wanted to participate in research, and Jen does an absolutely extraordinary job of sending out what clinical trials are available monthly, but there is really limited. And it's like, why is that the case?
(00:04:04):
So we're going to talk a little bit more about that today. And I just really want to reassure everybody, this is not about replacing the work that we do. The work that we do at the foundation obviously is embedded in everything that we do. We care about health equity, we care about inclusion, we care about participation. This is about thinking about how can we actually complete that pipeline cycle rather than having big gaps in between. But yeah, I'll now hand it over to Sarah to talk about different other elements.
Sarah Philbin (00:04:38):
Thanks, Rachel. Ashley, if you want to move to the next slide, I'll talk through a little bit of an overview.
Jen Lyman (00:04:46):
Absolutely.
Sarah Philbin (00:04:48):
So the evolution of how we understand early brain injury has really evolved over the last decade or so, maybe a little longer. So we understand now that the injuries look different than we previously understood. So they're not large lesions like we once thought. They're more systemic and more spread throughout the brain than previously thought. We've also learned that cells, not necessarily that they are dead or they have died as a result of injury, but they might be stuck. So that means that we can leverage and maybe potentially reactivate them through different treatment modalities. We've also learned that inflammation that associates early brain injury often persists for a period of time after that initial injury. So that's another mechanism through which new treatments might be developed. And that not only for infants, but also for adults as well, because as many of us know, that inflammation is maybe something that contributes to joint pain in adults.
(00:05:59):
And then additionally, as Rachel mentioned, this work very much intersects with other work of the foundation, particularly the early detection network. So if we can flag a child's risk for CP earlier, we can start to intervene with different treatment mechanisms. So this work is very much cohesive and sort of aligns with the other work of the foundation. And also we are thinking of a lifespan approach. If you follow our CPGU Instagram that Ashley leads, we're in the process of developing preventative clinical practice guidelines. So thinking about how once we implement those, that's going to create an infrastructure to eventually implement new treatment modalities for adults, whether that's in, as I said, inflammation or brain computer interface, which sounds like a lot, but we'll speak to in a little bit in the section about the Breakthrough Summit. So for myself, as I mentioned, I'm an adult with CP.
(00:07:01):
And on one side of my family, we have a lot of joint replacements. Folks in my one parent sibling group, hips, knees, shoulders, and also some spinal stenosis. And then on my other side, I have a grandmother that had very severe arthritis. So just thinking on those musculoskeletal genes and what that means for me plus having CP. I'm very much interested, especially in the inflammation work. So we really much are keeping adults as part of the focus of this initiative as well and not solely focusing on infants.
Ashley Harris Whaley (00:07:42):
Thank you so much, Sarah. I think that's such an important point is that this program and in any work that we do at the foundation, we're very committed to a lifespan approach across everything that we do and looking at cerebral palsy as the lifelong disability that it is. And I think that that is coming through in this program and in this work that's being done as well. But we know that the word cure is, it can evoke strong feelings in a lot of folks for different reasons, all very valid. And we realize that the choice to use that word has been an intentional choice. And we are going to dive into some of that while we're here right now. And so one of the things that Jen and I wanted to do is, not that we're going to spend the whole rest of the afternoon on this, but give y'all a little bit of insight into our personal narratives and our personal journeys and how we feel about that word, how we have felt about that word, and how that has evolved.
(00:09:06):
So Jen, would you want to take it first or do you want me to take it first?
Jen Lyman (00:09:15):
Either way, if you want to go first, that'd be great. Yeah,
Ashley Harris Whaley (00:09:18):
I don't mind at all.
(00:09:21):
And I promise I'm not going to wax too philosophical here, but I can remember the first time that somebody asked me about cerebral palsy, about a cure for cerebral palsy, literally like it was yesterday. And it was 10 years ago, exactly almost. I had just flown on an airplane for the first time. We will not talk about how I was 24 until I flew on an airplane, but that's another story for another time. I had just gone to New York City to meet my very first friend with cerebral palsy who has CP that is spastic diplegia, just like mine, very similar. So this is the first time I'd ever met somebody this similar to me in real life. And we were in this really, honestly, kind of gross and dingy restaurant somewhere near Harold Square. I couldn't tell you. And we were doing this thing where we trade off questions like, "Can you put your pants on standing up or do you put your pants on sitting down?
(00:10:26):
Or can you move your toes one at a time or do they just move as a unit?" And so we were doing this question trading and my boyfriend at the time, who's now my husband and another friend of ours were sitting in the booth with us, kind of just open-mouthed a little bit at this back and forth. And then my friend Kyle, who I'm engaging in this with, he hits me with this question and he goes, "So if there was a cure for CP, would you take it?" And I remember, I think I slow blinked at him a couple times, like a lizard. And then I was like, "No, I like how I am. Nope, I don't think I would." And then I kind of shut that down and we never talked about it again the rest of the night. We actually didn't talk about it for many years after that.
(00:11:16):
But over the years, I've checked in with myself quite a few times about how do I actually feel about that? How do I feel about a cure for cerebral palsy? What could that look like? What could that mean for my life? And if there was a hypothetical magic pill, would I take it? A few years ago, Rachel and I were in New York together, and we had been walking around all day long. And at that point, my feet were screaming, my body was screaming. All I wanted to do was lay down. If I wasn't such a germophobe, I might've just laid down on the sidewalk right there where we were. We were walking along and she asked me if I was tired. I don't really remember what I said, but she remembers what I said because she's brought it up to me several times in the intervening years is I said something to the effect of, yeah, I'm tired because I have to think about every single move I ever make ever.
(00:12:21):
And I know if you have CPN, you're on this call, you might feel very similarly. And since I've been in this work and been leading adult work at the foundation for the past almost five years, I think about aging with cerebral palsy every single day, partly because I'm just a little bit of a worry wart, partly because my work sort of forces me to think about it every day. But the more I think about aging, the more I come to realize how little we do know, how little information we have, how the information that we do have isn't all that great. The more I think about it, I kind of do maybe wish there was a magic pill. And I think the biggest pivot for how I feel about the word cure came when I became a mom. So I have two kids. They're three and one, so they're very busy.
(00:13:24):
They keep me on my toes twenty four seven. And what I've realized in the last three years is, and it's uncomfortable to say, I'm uncomfortable saying it right now, but it's true. And that's that cerebral palsy makes my life harder. It makes it so hard. It has always made it hard. Now I can kind of realize that and look back. But as a parent to two very young children who need me in very intense ways around the clock, oh my goodness, the simple, what probably seems pretty simple act of getting them out of the house, putting them in the car, getting them in the car seat, getting them out of the car, getting them in the stroller, because I can't carry them into a store. I can't go to a grocery store, pull up in a parking spot, take them out of the car and carry them inside.
(00:14:18):
There's about seven different steps, additional steps that have to happen to make that possible. And now I've thought about a cure more than I ever have. If there was a magic pill to answer the question from 10 years ago, would I take it tomorrow? Heck yeah. Sign me up. Put me in line.
(00:14:40):
I like being disabled. I don't mind it at all. I find it a huge part of my identity. Having cerebral palsy is as inextricable from myself as having curly hair or having the accent that I have or whatever other defining characteristic you want to label. It is me and I am it. But at this point, cerebral palsy has kind of shaped me into the person that I am. We've done the shaping. The shaping has been accomplished. The identity is there. The identity is set. So if there were some therapeutic option that were to come along in all seriousness that would make my life easier, absolutely. If there were a therapeutic option that would come along that would make the lives of babies with CP easier, kids with CP, those who have yet to be born and diagnosed with it easier, I'm all for it. I'm all for the pursuit.
(00:15:45):
I just kind of wanted to take a minute to share with y'all the last 10 years of an evolution of thought on a cure for CP, how that has formed for me and how I've decided that a support of that can absolutely coexist along with my disabled self. Those things can exist together. They don't have to preclude, but I'm going to stop talking. I've talked plenty, and I'm going to hand the floor over to Jen. I don't
Jen Lyman (00:16:23):
Know how to follow that.
Rachel Byrne (00:16:25):
And just before you jump in with your story, there is a couple of questions that are happening in the chat. So we didn't mention, if you do have any questions for any of us as panelists, please put them into the chat and we'll either try to answer them in writing on the chat, but we'll also have opportunities throughout where we'll actually discuss them and talk about them as well. So please, anybody that has questions, please put them in the chat. And I think we want this to be a really interactive discussion with all of you. So just wanted to throw that out there, Jen, and I'll hand it back over to you.
Jen Lyman (00:16:53):
Yeah, absolutely. And Ashley, that was really hard to follow. I ain't hard to follow. It's hard to follow that explanation because it is so beautiful and it makes a lot of sense to me. And thinking about that magic pill, and I think about it more like magic pills and all of the opportunities to prevent issues that have occurred since the birth of my son. And if there was something that could have helped when he was much younger that would've perhaps prevented the necrotizing enterocolitis that ultimately caused his brain injury. And I'll get into all of that later when we're talking about it, but all of the, and it's horrible to say, but there have been so many times when he has been near death for a variety of reasons, whether it's been infection or whether it's been choking or just really scary, horrible experiences. And it's all due to his CP.
(00:18:03):
And to watch that as a parent and to watch him suffer and to watch him have pain and not be able to express himself and not be able to see. Sorry, I'll cry. But anything that we can do to reduce the impact for him or for any of these kids and young adults, and not at all to take away that identity. I've worked with people with disabilities my entire career well before I had my son, and I will do anything to advocate for people with disabilities to be included and to participate and to have the very best quality of life humanly possible. And this is part of that. And it's part of that continuum to ensure that as adults, you're not having more pain just because you've got CP on top of your arthritis.
(00:19:03):
And I look at all the caregivers, all of us who are taking care of our adult children who need full-time, twenty four seven care, who need to be fed, who need to be changed and diapered. And I respect every moment of that, but it's really hard. And I think that if my son had the choice, he wouldn't want his mom to be changing his diaper at 21. And I'd love for him to have that choice. So that's my answer. I don't have much more to say because I'll cry more. So I feel strongly about that though.
Rachel Byrne (00:19:43):
I think this is so powerful that both of you shared your stories because there is a lot of nuance to this conversation and what cure means to different people will be very different, whether it be improving an outcome, whether it be preventing something for the future or whether it be presenting something for now. But Jen, you do bring up a really good point because a lot of the time we talk about cerebral palsy and it doesn't have urgency attached to it. We talk about, okay, we're here to manage different components, but there is absolutely a subgroup of individuals where it is urgent, where it is life and death. And the mortality rate for those with cerebral palsy, severe CP is nine times higher under the age of 26. And so these are the things that we need to consider when we're having this conversation, that it is obviously a spectrum of all of these components, but we cannot forget about those children.
(00:20:36):
They are not just a statistic. All of everybody's lives matter and how do we have this with grace, but how do we also make sure we're pushing the pipeline along? And I'm so fortunate to actually work with all of these incredible women who are on this call, and we get to have these conversations daily. We have a lot of nuance around the foundation, around the work that we do, around our why, personal whys, but then also around the greater collective as a community. So we are so thrilled actually that we can have these conversations with all of you. And as I said, we want to continue it. So please put these questions in the chat. And maybe you all disagree with us. That's okay too. We're not here to be critical of everyone's thoughts or opinions. But I'll hand it back over to you, Ashley.
(00:21:22):
But thank you both for sharing.
Ashley Harris Whaley (00:21:24):
Yeah, no, thank you, Jen. Beautifully said as always. So earlier this year we convened what we call the Breakthrough Summit, which was sort of a launching pad for our Cures Initiative. So what I would love, Rachel and Sarah, is for y'all to tell us a little bit more about the summit, about how that feeds into the program structure. If you could walk us through the six areas of emphasis for the program, and then speaking to how our networks of change approach is applied and how the collaborations with different researchers and institutions are working within this program as well.
Rachel Byrne (00:22:17):
Yeah, thanks, Ashley. So obviously you can see those with cerebral palsy and lived experience are at the center of this work, and we are absolutely committed to making sure not only it stays that way, but how do we embed all of these different pieces in it? So the Breakthrough Summit that we held in January was we got together a group of clinicians, researchers, those with lived experience, as well as those in basic science to think about, well, why hasn't this pipeline happened? Why has there been such big gaps in research up until now? And I will be honest with you, some of it was because cure even for researchers is a word that they struggle to come to terms with. And I think the exciting thing is over the last five years in understanding the biology of cerebral palsy, understanding the advancements that we have had, we've been able to really move that conversation along.
(00:23:08):
But just to be clear, some researchers also were like, "Well, it's not the word cure. Let's use the word prevent. It's not the word cure. Let's use the word breakthrough." And I think sometimes we're just getting into semantics around what different words mean to different people. But yeah, it was really important for us to think about if we're launching an initiative like this, can the science back up what we're trying to achieve? Where are we along in that pipeline to say, okay, is this a 30-year initiative? Is this a 50-year output? Is this something that can actually be genuinely achieved for some in the next five years? And I think that's why it was really important to get a really, I suppose, multidisciplinary group of individuals together. And as I said, that included everyone who was participating from a basic science perspective, thinking about what those really initial scientific pathways are to translational science, to clinical trials, to implementation.
(00:24:04):
We made sure we had, as I said, those with lived experience, both parents and adults with cerebral palsy in the room. But this is where this gets a little complicated, and I'm going to hand it over to Sarah in a little bit because it is not going to be a one pill solution. And we talk about this magical kind of piece. It is not going to be that. It is going to be a multi-pronged approach that has pathways that have success and then pathways that don't. We do know that. But I think what we need to do is create that pipeline so that we are actually part of the conversation. And when we look at that, how we do things at the foundation are obviously all encompassing. So you'll see those with lived experiences, as I said, are at the center of this. And then as we expand around it, how do we think about implementation of this?
(00:24:52):
What does that even look like? How do we think about rigorous clinical trials? We know a lot of people currently go out of state or out of country to participate in interventions. What we would really like to see is to have that clinical trial pipeline amongst institutions that have regulations so that not only are you not having to pay out of pocket for different things, but you know that there are safety trials involved. We know that if the work isn't going down that pathway, then it would have to pivot. And I think that's really important because as we talk about all these different opportunities, safety is first and foremost. And sometimes when we're traveling in unregulated spaces, obviously safety is one of our biggest concerns as an organization.
(00:25:40):
We've just got some questions here. Are these trials all in the US? So when we're creating this network of change, as Ashley just mentioned, it will be international. We also know not one organization, not one institution is going to be able to create these pipelines. And so we're really looking at it as an international approach because that will also help us with both health equity and making sure these things can get implemented in lots of different settings. Just sort of looking at another question that we have here in relation to a research perspective. Having tried to get the research done, we were never able to get funding that they get all the funds. Absolutely. So when we think about funding research, that's a huge one, right? If we go back to that previous slide as to why did we get all the researchers together and why have they done these different pieces?
(00:26:35):
Some of that comes into that there is not funding pipelines for cerebral palsy. Well, why is that? Is it because we actually haven't said, hang on a second, we would like to create a cure and breakthrough pipeline? Is it because the researchers and scientists weren't interested in this space or is it because of which comes first? Funding to pursue the idea or the idea and then finding the funding. So the funding mechanisms are a huge issue, but that's where advocacy comes into play and advocacy needs to come into play both at the federal level from a funding perspective, whether it be at the NIH or others. But we also need to bring advocacy in private philanthropy and other opportunities and even in industry. One of the biggest things that we know at the moment is if we're thinking about say drug pipeline development, and drug pipeline development is one of the areas here, as you can see, the pharmacological and neurotherapeutics, there is very little R&D happening at industry level in this space to the point actually that most of them have removed themselves from pediatric indications, but also in this space because is there a monetary value in that piece?
(00:27:47):
So without going into too much detail around that, that can actually become quite complex. But just before we do moving on, so the areas that the research has identified, whether it's opportunity is thinking around pharmacology and neurotherapeutics. It's also thinking around precision medicine. A lot of you would've heard precision medicine as it relates to cancer. And now that people are getting genetic therapy or genetic interventions that are particularly suited to them. So is there an avenue there to understand? Preclinical modeling. What has advanced now in preclinical modeling before you would've thought about animal trials? Now we can think about organoid trials. And when I talk about organoids, we can now develop brain cells and brain tissues in a dish, muscle tissues. And so how can we fast track those preclinical modeling trials so that we can get to clinical trials at a greater pace? Immunology and neuroinflammation.
(00:28:48):
Ashley sort of touched on this and so did Sarah. Thinking about inflammation across the lifespan, we know that happens. And so what can we think about in that space? And then cell-based interventions. Many of you would've heard of stem cell interventions or different types of cells. There's lots of different types of cells, not just stem cells. So thinking around all of those things. And most likely as we look to the future, even for one individual, it is not most likely going to be one of these pathways. It might actually be a multidisciplinary across all of these things. And again, the outcome may not be that cerebral palsy is "cured," but maybe it's removed pain. Maybe it has allowed for mobility to come back for independence. Maybe communication has occurred. Maybe for Jen and her son Bower, it has allowed for vision. And so I think there's lots of things as we're thinking along this pipeline that is just not a one-size-fits-all answer.
Ashley Harris Whaley (00:29:51):
Yeah, thank you so much, Rachel. I think that's super important. It's kind of like a thread running through all of the conversations that we have about this program is that We are never talking about just one thing or just five things that look a very specific certain way. It's all so nuanced and so context dependent and so individual dependent. But Sarah, was there anything that you wanted to speak to while we were on this slide or do you think we're ready to move forward?
Sarah Philbin (00:30:23):
No, I mean I think we're ready to move forward. I think something that's important to keep in mind that I assume some individuals on this call might have questions about is particularly around issues such as insurance coverage in the US, you have public versus private, insurance through your employer versus insurance like Medicaid through state or Medicare federal program. So those are things that we obviously need to think about throughout the course of this development process. So while the basic scientists are working on these cellular avenues and inflammation avenues, what does this look like once something's ready to be taken to market to patients? Not only that, but also thinking about geographic implications. We know that there's a large percentage of individuals with cerebral palsy that live in low and middle income countries, that infrastructure might be a challenge for them to get into a clinical setting.
(00:31:26):
Even in the United States, obviously that's an issue as well. There's a lot of rural communities. So what does that look like in terms of bringing these treatments to patients? So those are things that we need to think about early because if we don't consider them, we'll have the interventions ready to go and then run into implementation challenges. So that's kind of the pipeline that we look at, but I don't want people to think that we're working in a vacuum and not considering those real world challenges, whether it be, again, geographic barriers or insurance barriers. So those are things that we consider as well. So that's what I would add there.
Ashley Harris Whaley (00:32:12):
Thanks so much, Sarah. And in that same vein, would you want to speak to how are we moving this forward? What does the mechanism for that look like and how do both philanthropy and industry co-investment, how do those weave in and what steps are we taking to actually move this forward? I
Sarah Philbin (00:32:38):
Think I'm going to punt that over to Rachel actually.
Rachel Byrne (00:32:42):
Sure, no problems. As I said, I sort of touched on it before, funding for a project like this is a big deal. When we think about funding in CP, we have been very underfunded and under-resourced for a long time. To put it in perspective, I think this year the funding at NIH was 25 million compared to autism that had 325 million. There is a big gap when we think about federal funding and how that looks in this space, but then there is also a very big gap when it comes to philanthropy and industry. As an organization, we are one of the largest funders of research globally, and we can't even touch the edge of the iceberg when it comes to these topics. So we really do need to lean on other philanthropic opportunities as well as industry to do that. Now, part of that, we need to do two things.
(00:33:31):
We need to show that actually there is urgency in this conversation, and also there is a need from the community to want this. And then the other piece that I think is really important is to understand that CP is not one thing. And so as we are looking at the different opportunities for interventions, that our expectation isn't that it's going to be one thing. That's why it's so complex and why industry sometimes tries to avoid it, to be honest. But the rare disease community has done a phenomenal job of saying why this is needed for rare diseases. And actually other disabilities have done that as well. So SMA, for example, or just muscular dystrophy have really identified, hang on a second, this is something that we want as a community not to take away that identity. And I do want to make sure, Ashley, that you see Emily's comment in the questions because it is important that we are not trying to take away cerebral palsy and rid CP from the planet in a eugenics manner.
(00:34:28):
That is not the intent at all of this work. And that obviously people's identities and how important they are to how we live our lives, how we bring empathy to conversations, how we understand and participate in the things that we do. We want to make sure that that is maintained throughout of this. And you can see how come autism is more funded than cerebral palsy? Well, that's twofold. A, as an autism community, they mobilized, but there was also a lot of philanthropic funding that's going into that space. And so that sometimes is families that have obviously been impacted by cerebral palsy or autism apologies and seeing that that's a high priority for them. But also this piece, and particularly Jen, I'll lean on your story a little bit as well.
(00:35:15):
When you have somebody who has severe cerebral palsy or severe autism, obviously there are many amazing experiences that you have as family members, but it also can be really difficult and it can be really difficult for that individual. And there is pain and suffering that they experience. And I think we want to remove that from anybody's life. We want to make sure people live long, healthy lives. And that's a big part of all of this, and that's sometimes the message that we need to put forward. I think for a long time as a CP sector or as pieces, we were like, all right, cerebral palsy is here to be managed and nothing can be done about it. What we now know is that the science is caught up and that there are a lot of things that can be done to prevent comorbidities or prevent early aging or even prevent cerebral palsy in babies to begin with.
Ashley Harris Whaley (00:36:06):
So Rachel, I think that may be one of the biggest takeaway points from this whole conversation is that the CP community has existed in a space of status quo for the longest time of just acceptance that this is it, this is what it is. There is no path forward to change or mitigate or do anything of the sort. And now we know that that's not necessarily the truth. And how cool is it to have the knowledge that, yes, we may not have all of the finite detailed answers in every single individual person's case, but what we do know is that we don't have to exist in a state of status quo. And we're on the cusp really of a, to use y'all's word, a breakthrough, all these breakthroughs and figuring out what can that actually look like? How can we really change the landscape of what this disability looks like and the funding landscape and all the pieces that go along with it?
(00:37:16):
I don't know about y'all, you don't have to agree, but I find that personally to be pretty encouraging, honestly. So anyway, to the science of it all, would love to learn and would love to learn with our audience a little bit more about some of this science behind this program and cell development. Sarah, are you going to take this one first?
Sarah Philbin (00:37:44):
Rachel, are you and Jen going to speak to the kind of regulation around cells first?
Rachel Byrne (00:37:50):
Yeah, because I think it's really important. And the reason why we want to talk about cells as part of this, and just to preface this, I am not a stem cell-based researcher or scientist, and neither is Jen. However, we probably have seen these questions happening for a really long time, and we know it's top of mind for a lot of families at the moment from a therapeutic approach. And we want to talk about the hope and the opportunity here because there absolutely is hope and opportunity, and researchers and scientists are really pushing that narrative along. But there is also gaps in our knowledge that we have. And as I mentioned, how important it is for safety. And so obviously in this space, there are a lot of regulated and unregulated treatments, and they come in many different forms. So cell therapy is used, and when I say cell therapy, let's just call it types of stem cell therapy.
(00:38:43):
Again, there are different types of cells that wouldn't even be called stem cells, but for ease, let's go there. There is evidence that cell therapy works for other conditions. We already know that, whether it be for recovery after a surgery, whether it be for a musculoskeletal issue outside of cerebral palsy, whether it be for some cancers, whether it be for other things. And so that has made it readily available sometimes in different spaces. But what we do know is sometimes those are in regulated clinics or unregulated clinics. And as I said, the piece that we're looking at now, particularly when you're looking at cell therapy in relation to brain development or changes in the brain, we are at a gap in our understanding of the biology. Now, that does not mean that there are not some clinical trials that have occurred that have been regulated and that are still occurring globally that have been regulated, but there is a lot of unregulated clinics that are occurring around the world.
(00:39:45):
And so I think it is really important to discuss that and understand potentially the safety risks that occur at those clinics. But Jen, did you want to jump in here as well? I know you have many discussions with families and adults in relation to this topic.
Jen Lyman (00:40:00):
Yeah, it's certainly the number one question that I get from families about are there any recruiting clinical trials with stem cells? And currently there are not, unfortunately. And sadly, I think about it too with this whole sense of urgency. We were very fortunate as a family, and there was the Duke study, a lot of families had been going to Duke for the umbilical cord trials, but things have never moved forward to a phase three clinical trial. And similarly for us, we were participants in the first FDA approved mesenchy, or I'm sorry, I think it was mesenchymal cells, the autologous stem cells that you get from your bone marrow. And we were able to participate in that, but nothing has moved forward since then. But the question that I'm always getting from families is, should we go down to Honduras and pay our $20,000 or $30,000 or Panama?
(00:41:05):
And for years it was China. And back to Rachel's point is we don't know where these cells are coming from. We don't know that they're even human cells, and there have been deaths as a result of that. And I think when I'm looking at, I've been looking at stem cells now personally, just for my own family, for my son for 21 years. And I feel like it's been creeping forward. And now I'm hearing more and more about it. And there's exciting things that we're hearing about. We're hearing about new cells and there's exciting possibility and potential with new cells, but we're not there yet. The research isn't there yet, and I'm sure we'll get more into that. But just thinking about how they work and thinking about the possibilities. And I think what I've come to in the questions and just looking at it is that it's a combination of the mechanism, how they're going to be delivered into the system.
(00:42:09):
And it's also about timing, and it's about the number of times that you're potentially going to have to do this in order to have an effect. And I'm happy to talk about our experience with being in a stem cell trial, in an actual FDA-approved stem cell trial, if y'all would like me to, or I can move on.
Rachel Byrne (00:42:28):
Yeah, Jen, I think that's a good piece. And I think some of the pieces of the stages where we're at in research are based on absolutely strong clinical data. When I say clinical data, I should say preclinical trial data. And so for example, muse-cells, so that was based out of a group in Japan and their work was first published in 2010, but they have not gone to phase three clinical trial yet. So we are not at that stage of saying, okay, this is something then that should be looked at for a implementation component. We're still understanding the how and the why there. And some of these things, we've also been there, done that, didn't work, let's move on. And so just want to be really clear that some of these things are based in science, but they have not progressed. And so just understanding where that discrepancy is, where it comes from, all right, science got this far, and then it's gone into a clinic in a different country.
(00:43:29):
Okay, there's been a big gap in the research and understanding there and knowing if this is going to be safe or if it will work for somebody. But I think where we think about where things stand, and this is sort of trials that you'll see, cord blood studies. So cord blood studies have been shown for early movement gains in young children. Now, when we say that, these are small gains. These are not things that necessarily would be if you understand GMFCS levels or movement levels, these are not going to have a child go from a GMFCS-5 to a GMFCS-1. Some of these are important gains. We all know that small gains in mobility can lead to huge gains in independence or ways of quality of life. So it's not to diminish any of those, but just knowing that cord blood studies have shown that they will not lead to huge mobility gains, but they have been shown to be safe.
(00:44:22):
And so there are trials currently occurring in different parts of the world in relation to that. I think they're
Jen Lyman (00:44:27):
Comparable. Just interrupt you. I think it's comparable to constraint-induced movement therapy. If you do an intensive there, they've shown that some of the stem cell trials are maybe comparable to that. I know in our situation, we did have MRIs. I saw Sierra, you're asking if I could share our story, but it was a two-year study. It was a double-blind placebo-controlled trial. I think there were 16 patients in the trial and we did not know. We were blinded, so we didn't know whether or not he got his stem cells. They were taken from the bone marrow and then reinfused. He had multiple MRIs and GMFM as well as a battery of other tests every six months from the start of the trial until the end of the trial over a period of two years. And what we saw, I absolutely knew that he got his stem cells.
(00:45:24):
I saw that there was a little bit of an increase in his trunk control, not huge, but just a little tiny bit, and some clarity, more alertness and clarity in his speech. And that's saying a lot for Bower because he doesn't have much speech. So there was a little bit of improvement there. But what was more remarkable to me was not either one of those, but it was the MRIs. And what the MRIs showed was that for the first year after he received his MRI, there was a clear reduction in the inflammation where he has specifically periventricular leukomalacia around the ventricles of his brain. And it showed a clear reduction in that inflammation. And that came back after two years. So it was a temporary trophic effect, which is why I think that there's hope because I think there's hope in the delivery and potentially, okay, well, you could do it more often.
(00:46:28):
So we don't know what the frequency needs to be. We don't know how much you need. We don't know whether it's better to deliver it via an infusion or intranasally or some other way. But it did give me hope and it gave me hope that there might be some small gains to be had. And small gains for us is a big deal.
Rachel Byrne (00:46:49):
And Jen, I think that's just so important because you were able to obviously enroll in those trials and have it done in a space that you felt safe and that you knew obviously was regulated and had all the different, I suppose.
Jen Lyman (00:47:04):
It was intense too. If anybody wants to -
Rachel Byrne (00:47:08):
And the commitment. But that is why we want to really develop obviously this pipeline and think about how do we continue to make sure that these options are available to everybody. So again, that you're not having to go to unregulated places because we completely understand why people do that. We hear it all the time. It's like, I don't want to leave any stone unturned. I want to make sure that as a parent or as a family member, I've done anything possible for my child. We understand that feeling. I'm a parent of three children. Ashley's a parent. We do anything for our children. But the piece is that we want to do it in a safe space and we want to do it in a way that doesn't actually harm other elements of people's lives. And we know that that's what's happening right now. So Jen, as I said, thank you.
(00:47:54):
And then the other piece I just want to shout out is, as I said, we are not the researchers and scientists behind these different pieces, but as part of this whole series, what we are hoping to do is have very specific webinars in relation to those six topics that I showed you earlier so that we will have a webinar where we can get researchers and scientists and those who have actually participated in these trials, again, to come on and talk about them. And where are we at with the science? And more importantly, what does this future pipeline look like? To give all of you those sort of details and those answers that you're looking for.
Ashley Harris Whaley (00:48:30):
Yes. I think that we'd love to have as many of y'all to come back for the subsequent webinars where we're really going to do some scientific deep dives into what do these six areas of emphasis look like. But beyond webinar attendance, I think it's important that we talk about everything that the foundation does is community-centered. Every piece of work that we do, and this program of course is no different. So would love to speak, Jen and Sarah, to how are people able to get involved? What are the options? What are the pathways? And what practically does participating in research involve and look like? Because I know that there can be some conversation around registries as well. What are the practical implications of registries and how do those feed into the acceleration of cures and this work?
Jen Lyman (00:49:40):
Do you want to go first, Sarah, or do you want me
Sarah Philbin (00:49:42):
To - Jen, I know we have a question in the chat about finding resources. I don't know if you want to address that first or
Jen Lyman (00:49:48):
Commenting. I saw that being a mom with a child that has CP, how do you navigate this and how do you find the resources that are needed? And number one, I had a partner in crime that was my son's neurodevelopmental pediatrician who I absolutely, what I loved about him was that he was curious and he was willing to talk with me through every one of my wacky, crazy ideas about this therapy or that treatment or whatever we were looking at. He was willing to think through with me and go over the evidence surrounding whatever it is that we were talking about. But also to point me, he knew how to point me in the direction of good resources locally to ensure that Bower got great therapies. And I guess I was fortunate because it was also my background. So I knew early intervention, I knew inpatient rehab, I knew I'd been working as a therapist.
(00:51:03):
So it was helpful to have that background as well and know how to work as part of a team and developing IEPs or developing IFSPs or whatever else we had. But really, I think the main thing is having that partner in crime and also knowing that you are the quarterback really. I mean, you're the mom, you're the quarterback, you need to pull all these things together and knowing that you've got an expert on your side to help you with that and to help you plan for all of the different elements. And I think the other thing is recognizing that half the time I don't even know what I don't know. So having that partner in crime as well, that doctor who you trust to say, "Hey, what are the questions that the other moms are asking? Or what are the things that you're seeing that are really helpful that might be helpful for my kid?
(00:52:01):
And what resources do you know about?" So I really lean on a lot of people. That's how I've been able to develop the resources.This is a team effort and it's a marathon. So I've been very fortunate that way. And I guess the other part is early on, I got involved with reading the research and trying to understand what the research said. And also talking to Dr. Hoon, that's his name, talking to him about what it was and really familiarizing myself with what it was so that I could make informed decisions.
Rachel Byrne (00:52:42):
And Jen, can you now expand on why that has led you to develop the different components on CP Resource as well as then now the clinical trial email obviously that we send out to everybody because I think your experience really makes those be so much more robust and useful for people, but really important.
Jen Lyman (00:53:05):
Thank you, Rachel. I appreciate it. Yeah, I feel like there's so many different elements. I mean, you're looking at this web of care and treatments and the whole shebang. We've got everything from educational resources to legal resources to navigating Medicaid waivers to what are the best treatments? What clinical trials are available? Because CP obviously is such a big umbrella term and obviously captures so many different challenges and needs and also wonderful things. We've got a whole section on the different adaptive sports and camps and things like that that promote inclusion and participation and having the opportunities to lead a rich full life. So Rachel, I don't think I'm answering your question. One of the things that I really have enjoyed doing though is developing the research and clinical trial pipeline. And so one of the things that we've done on CP Resource is there is a way to recruit for clinical trials directly from CP Resource that we have.
(00:54:22):
We partner with researchers in the process of developing the clinical question from the very beginning. What is it that families are interested in that needs to be researched? A lot of the time they come to us and say, "Hey, this is what we want to recruit for and we'll work with them." But in the perfect world, we would be saying, which is what we're saying right now is these are the questions that we want answered. These are the problems that we want solved. We want to partner with you to develop this, and then we want to recruit for you and bring in the potential participants so we can match and through CP resource or through the clinical trials email.
(00:55:07):
I can match people to trials that they might potentially qualify for. And then we've also developed a system where we have a directory, which is essentially it's a link that you can click on and you can put all of your information in our directory. And if researchers have a trial that might be applicable to you, let's say you've got hemiplegia and there's a researcher looking for a bunch of people with hemiplegia, I can go into that directory. It's a HIPAA compliant directory and I can actually match those folks to the criteria of that trial and then let them know first. Obviously reach out to those folks and then with their permission, I can match them to the trial and introduce them to the trial. So it's rewarding, obviously for me as a parent and also working for CPF, it's incredibly rewarding to be able to do that and give people those opportunities.
(00:56:04):
It's really hard when you're trying to navigate clinicaltrials.gov to sort through all of it. So that's another element of all of this is I do go through allofclinicaltrials.org monthly when I'm really on my game. When I'm not on my game, it might be every other month, maybe a little less. But there's currently right now, I think we've got about 90 trials. And some are fabulous. I mean, absolutely fabulous and really exciting. And then there's others where I'm like, "Gosh, I wish I saw more brain computer interface on there that really we're taking kids with CP and we're just not there yet." But I think it's
Rachel Byrne (00:56:47):
Exciting. Thank you. I think it's so important when we're thinking about how to participate. There's many different ways. And Jen brought up a really good point to begin with. We need to be the ones influencing what research is happening as a community and making sure that that research is what you want answered and making sure that those pipelines are accessible. And that's sort of the whole piece of this study. But please, if you haven't already, the resources that Jen has created on cpresource.org are extraordinary. And signing up for that newsletter that does happen monthly around what trials are available. Now, as Jen said, some of those trials are not intervention-based trials. They are in other things, and we are absolutely pushing the narrative to see can we get intervention-based trials and see those be created? But the other piece that Jen does, and she didn't really talk about much, was really around the recruitment piece.
(00:57:39):
So anytime that the foundation is supporting recruitment through clinical trials, we want to make sure that you are supported through that whole process. Sometimes it can be really daunting actually participating in a research trial. Sometimes the expectations by the researchers feel enormous. And so we can be that conduit between both what it's like to be recruited into that trial. Do you make criteria? Are there other things that you want to consider? So Jen always has one-on-one conversations with everybody through the foundation. And then the other piece is going, "Well, what's it like during the trial itself?" I saw somebody ask a question in the chat around constraint-induced movement therapy in one-year-olds. And are there any treatment centers offering this intensive therapy? There absolutely are. There is evidence to show now of its both effectiveness and safety. There has just actually been a very large NIH clinical trial that has just been completed that the foundation was a part of that looked at a telehealth version.
(00:58:38):
So we know that intensives at times are not accessible to people because you have to go a lot of the time. And so this particular study was also built around what is it like to actually be able to complete a study without the burden actually being on the participant. So yeah, that's sort of a piece. And again, we'll try and put all this information around how to participate in different things. And now we'll hand it over to Sarah to talk more about how to actually participate in the CPCURES Initiative as we're building this program out as well.
Sarah Philbin (00:59:16):
Thanks, Rachel. So we're going to be following a model similar to what has been used in the other programs within the foundation. So some of you might be familiar with the CPGU advisory board and then also different focus groups and surveys that we've conducted both with adults and then also with families. So we'll be following a similar model to get input from the community. And the reason for that is because we want to understand what's important to you in your day-to-day life. So from a research standpoint, as researchers, we operationalize outcomes very rigidly often. And a lot Part of that is due to their clinical implication, but sometimes that doesn't always align with what goes on in people's day-to-day life. So that patient or community centeredness is important to ascertain. And the way that we do that is through talking to the community so that we can be sure that as trials move forward, whether that means incorporating a quality of life survey, how does this treatment impact quality of life?
(01:00:29):
And so that those outcomes can be assessed early on. And again, thinking ahead to clinical implementation, that gives us some insight earlier than when the intervention is already out in the real world. And the other question on here related to registries and how that accelerates cures, numbers matter in research. Statistically, the bigger your denominator, the better. And registries, it can be a challenging topic for some people. They're worried about their data safety, understandably. But I can tell you as a researcher and someone that has used claims data, insurance claims data, that there is a lot of red tape around getting access to research. So it's not handed out. Data is not handed out kind of willy-nilly within health systems. So the safety of your data is always front of mind in that respect. But registries really allow us to pull data together and look at outcomes on a population that's larger than we might if we just looked at one clinical site or one hospital, for example.
(01:01:45):
So it's really something that can power us to look at the wider impact of a treatment. So that is why registries are important. It's also a helpful mechanism for folks to be informed about what trials are going on. Often there is a communication modality associated with a registry, so that is why registries are important. So we will certainly keep you informed as our efforts on this front and getting the community involved evolve. And we're looking forward to hearing your input and relaying that to the researchers to ensure that your voices are heard.
Ashley Harris Whaley (01:02:32):
Thank you so much, Sarah. I was wondering, as this program continues to progress and continues to move forward, could y'all speak a little bit to what we're doing to realistically level set and set some expectations both in terms of the program as a whole, but then as we're communicating things out to people? So what's your perspective, Jen, particularly? I'd love to hear how do you manage to hold on to genuine hope and honesty about the timelines and how that all fits together? How does hope line up with how the research world works?
Jen Lyman (01:03:21):
So I feel like I never heard me on a webinar before or any of my podcasts. There's a term that the Michael J. Fox Foundation uses. It's hope is informed optimism. And I think following the research and staying on top of, it almost makes time go by a little bit faster. I was giggling, or not giggling, it's not a giggly thing, but I was thinking about necrotizing enterocolitis. And when Bower was born and he got necrotizing enterocolitis, which caused him to code and they had to resuscitate him, but that is the big event that has caused his CP, caused the periventricular leukomalacia, or that's what I tell myself. But I've followed that now 21 years. And five years ago, and it was all back then we were asking that question, why did this happen? Why do these infections happen in the NICU? Why is this one so particularly horrible?
(01:04:23):
And it was five years ago that a potential way to detect necrotizing or colitis, because there was no way to even detect it until you were already sick, and there was finally a way to detect it. And now just last year, there was a drug approved through rare diseases by the FDA for the treatment of NEC. And so it's been 20 years since that start to finish. And hopefully, I don't even know where that drug is now and where they've gotten, but that was approved through the FDA in 2025. So I feel like it still has a way to go, but they're moving forward. So similarly with the stem cells, we've been looking at them for 21 years now. And I feel like that to me I struggle with because I feel like it should be moving forward faster and I don't know why. And especially because I feel like every parent I talk to is asking that same question.
(01:05:33):
So I feel like this is where maybe it is the community isn't speaking out in the way we're not advocating as a community the way we need to for this. And we're not saying this is something that we need now.
(01:05:54):
Because I think every time I do start looking at, okay, well Taub and CIMT, I mean he started his work in the 1970s. We're just now implementing real CIMT with babies at age, well, for the study I just finished recruiting for, I mean four months to 14 months. So sometimes trying to put my head in the sand and not looking at those numbers and saying, oh my God, it's been 30 or 50 years is probably a good thing, but also staying on top of the research and recognizing that it's going to take time. I'm sure Sarah and Rachel can speak much more eloquently about how you get that pipeline, but I believe it's 10 years for most or more. Rachel, you know this better than I do.
Rachel Byrne (01:06:39):
Yeah, it's actually like the average is 17 years at the moment when we're thinking around that's drug development per se, to have something from an idea to clinical trial, if that's if everything goes well and is funded appropriately, then to market. What we do know though, and there's a question that's just been asked here is this all sounds great, but how do we make sure that this is all aligned when there are a long waiting list for even basic implementation pieces that we know should be getting done? And I think this is where as a foundation, we really try to work in those four buckets. So the research pipeline actually can improve and reinforce what needs to be then funded as a clinical implementation strategy. And I think these are things that are really important. And then advocacy actually makes sure that then that funding is available, whether it be at an institutional level or whether it be at a local level so that these interventions can be done.
(01:07:35):
And then education and training to know, well, what intervention should be done. And so when you think about, and this is from Richard, Richard, when you're thinking about that cyclical loop, a lot of the time there are breaks in those loops. And that's why these things don't go full circle and all the way round. And the research pipeline is one part of that much larger comprehensive implementation loop for making sure that clinical care is received by everyone. And I think equity potentially is always something that is at the forefront of our minds is how can we make this sort of pieces equitable? Just going back to that other question around hope, and there's another question on here in relation to AI. I think one thing that I would like to say is that while AI is going to help us advance understanding, there is still going to be need for other elements in relation to biology.
(01:08:26):
As Jen just said, the necrotizing enterocolitis, can't even say that. It has taken 20 years even to develop the assessment, let alone what the intervention is going to be. And that's because the understanding of these things are very complex. And so we do need to understand what biology before say interventions come to market. And AI will help us fill in some of those gaps. But the other things that will help us fill in some of those gaps is those preclinical trial models, whether it be organoids. I know animal models can be very hard for some people to understand, but they are really important as part of this pathway in understanding how we can help these babies and more importantly, these adults as well. And so I think while it's complex, what I would like to say is as part of this initiative, we are absolutely making sure that we address each of those elements and how do we make sure that this goes from an idea to being available to you?
(01:09:24):
And that pipeline has many different players across it, but I think what we're trying to be as the foundation is the glue across that pipeline. The foundation is not a research institute, we are not a service provider. However, we do represent the community in the best way we possibly can to make sure that this pipeline continues to move along that trajectory.
Sarah Philbin (01:09:47):
And also jump in quickly to speak to the role of implementation science. So you might think that sounds like a very wonky term, which some might argue it is, but implementation science is a relatively new field, although it has grown substantially within the last decade or so. And it helps us understand when we know something works, but we don't use it in practice, there's a gap between there. So a gap between what we know works and what we do in practice. And we see this in conditions other than cerebral palsy. People with diabetes, for example, might not get a medication that they need, more common chronic conditions.
(01:10:32):
So implementation science, traditionally it has been used at that later point in the translational science pipeline where the treatments are already approved and out and available to patients at their doctor's office. But there's been a shift recently to start thinking about the role of implementation science, which allows you to operationalize across the pipeline and thinking about that earlier so that we can hopefully ward off some of those challenges down the road if we start thinking about them earlier. So if we start planning earlier, we can start addressing them in a more timely way. So I would just say that that's something we think about very strategically and through a very structured different frameworks. So I felt like it's important to mention that so that folks know that it's not just something that we think through haphazardly. There is a science behind it.
Ashley Harris Whaley (01:11:37):
So much, Sarah. Speaking on behalf of maybe the layperson community right now, I think that's a super helpful reminder. We are going to take some questions, y'all, and we've got some that have come through the chat and then some that came through when folks registered. But I wanted to take a minute to pause because there's a question on the slide previous to this that I think I'd really like to get y'all's answers to before we move forward. And that's what is the one thing that you want both families and individuals with CP to understand? If there's going to be one takeaway from this that you don't want misunderstanding around, what is that? And I think I'm going to give that one to Rachel first.
Rachel Byrne (01:12:27):
Yeah, I think from an organization perspective is that we are not listening to families or adults with cerebral palsy. As you can tell, our foundation is made up of adults and caregivers and families of people with cerebral palsy. And that's really important to us as we design any of our initiatives and also as we move forward across the pipeline. We want you to be part of this work. And so that's why it's amazing actually to have so many of you on this webinar right now and to be interactive and get the answers and get the questions that you've been able to give us. And then I think there's another one in the fact that this is blue sky thinking. Science has advanced enough now that hope can actually come in five to 10 years. Now the hope results are not going to come for every single etiology of cerebral palsy and every single pathway in five to 10 years, but absolutely there are things that we should be preventing now and could actually be curing in that timeline.
(01:13:35):
And so to me, what we need to now do is connect everything together. Can we connect the funding, the researchers, implementation and the community so that we can drive this forward as an urgent issue rather than something that is there to be managed? Sorry, that was way more than one. No,
Jen Lyman (01:13:51):
I think it's that sense of urgency that as a community, what I hear from the families and the adults, I mean I've recruited for a giant FDA trial and the sheer number of adults that reached out asking to be a part of it. That's what I want people to know is that we recognize that urgency. We take that seriously, and we're doing everything we can to bring that pipeline, make it faster and embrace that.
Ashley Harris Whaley (01:14:32):
Sarah, would love to get your perspective on that question as well.
Sarah Philbin (01:14:37):
Sure. No, I think I probably would echo what Rachel has to say that we are listening to the voice of the community and want to relay that and be sure that your interests and concerns are reflected as we move forward or decide what to prioritize as we look at different research avenues. So that's what I would say is our ears are open and we're listening to your feedback and look forward to engaging and having conversations with the community.
Ashley Harris Whaley (01:15:18):
Thanks so much. We do have still about 15 more minutes left, so would like to probably now take some time to answer. We've answered a lot of questions that came through the chat already, but there's a couple more that are hanging out. And then we had two in particular that came through on the registration side that I think kind of speak to the lifespan approach of both this and our other work. So I want to start with this one if it's okay. So it says, "My two and a half-year-old daughter has mixed CP effect in all four limbs. She's a GMS three to four. We've been doing intensive therapy since she was a year old and started Botox recently. What other future options does she have?" And I'm going to let Rachel take the being into that one. Sure.
Rachel Byrne (01:16:08):
I'll put a couple of hats on here. So obviously as a physical therapist, we think about early interventions and obviously this child and this family has been participating in early interventions, which is amazing because we know that sort of reinforces pieces. But the opportunities that she has, oh my gosh, they're endless in a way. And so when I think about participation in school, sport, drama, making friends and all of those things, that all coexists. There's a part of the story that I don't usually tell, but I've got someone who's a big part of my life who's like a little brother and we have traveled the world together. He uses a power wheelchair and we've done all sorts of things. We've gone surfing, we've done all the things that make up what family life should be and what it means to all of us. And so hope in the future looks really bright for her.
(01:17:01):
I will just say that in many other ways. But if you're thinking about the pipeline here for interventions, all of those different things could be a possibility. When we are looking at this, as we said, the lifespan approach is really important to us. So if we are looking at things that are going to improve inflammation, that doesn't necessarily need to be in babies that have just been born. When we're looking at cell therapy, again, that could be done in children at many different ages. Obviously sometimes early intervention we know has the greatest impact, but also thinking around, we haven't spoken about brain computer interface much today, but think about the progress in that field. It was fascinating. I saw something at a conference over the weekend that was around brain computer interface and those with ALS. And the amount of science and the advancement in that as a therapeutic is extraordinary, giving people back the opportunity to connect and communicate that they didn't have prior.
(01:18:07):
And that's everything from one day not being able to even, I suppose, have much movement to be able to communicate, to having 50 words that gets done in real time, to having 125,000 words that gets done in real time. And so when you're thinking about each of those interventions, I would just want to strongly say that all of those have the opportunity to impact people at different points in their lives. The other piece that I see, there's a question here in the chat from Alex, how closely do you work with other CP organizations? We work really closely with other CP organizations. Our world is too small not to partner and work together. CPRN, we've got a major project happening right now to look at early detection across the whole CPRN network. Cerebral Palsy Alliance, we work very closely with. We work closely with international organizations, and of course we partner with most of the institutions and children's hospitals across the country.
(01:19:02):
So we see that partnership as being really important. We want to make sure that we're lifting up everybody's work. There's so much work to be done, and that we do that together.
Ashley Harris Whaley (01:19:17):
Thanks so much, Rachel. Sorry, I was taking just one second to start answering another question in the chat. But if we're going back to thinking across the lifespan, and this is something that obviously you and I talk a lot about and think a lot about as far as things go for adults with CP, but when it comes down to talking about cures, what does that mean for adults with CP? Are we talking prevention? Are we talking restoration? Is it both? What does and could that actually look like? It kind of is a combination.
Rachel Byrne (01:20:00):
Absolutely. And let's talk about the science there for a second. So let's go back to cell therapy. What we know we don't have, we do not have a therapy at the moment that would lead to neural recircuitry of the brain. So if you're thinking about whether it be PVL or IVH or other areas of their brain being injured, we don't have the ability to replace that right now, if that makes sense. We don't have the ability to do a brain transplant in a certain section or what that looks like. And so is there future opportunity there? Potentially. Would there be ability to regrow the part of the brain that's being damaged? Maybe, but that is far down the track. But what do we have is the ability to impact inflammation. Of course. Jen spoke about cell therapy impacting inflammation as part of PVL. That's something that we can absolutely do.
(01:20:52):
And we know chronic inflammation and immunology plays a role across the lifespan. It plays a very important role very early on as well, but what does that look like across the lifespan? The other piece that I suppose I just wanted to touch base on too is when we are looking at the development of trials, when we look at say pharmacology and where that's going to occur, we also know that safety trials most likely will not get done in young babies. So if you have a look at all the different FDA approved drugs, so say I think there's like 4,000 of them, there is only one that has been approved for the NICU, only one. Everything else gets used off-label. But if we're going to think about repurposing of other drugs or what that might look like, those safety trials are most likely not going to get done in those babies in their most vulnerable moment.
(01:21:42):
They're most likely potentially going to be done as an adult trial or as done as something else. And it may not show efficacy that that drug is going to potentially change different outcomes. It might, and I think that's really important to say. It absolutely could. And there are some very promising. But I think there's pieces there where we can also show it's safe. There's a trial that was able to happen at Hopkins, Johns Hopkins, of which we have supported in nanomedicine. And that trial was able to occur and show safety because of COVID. The design of that nanomedicine looking at how it impacts early brain injury, that's exactly what it was for. But during COVID, it was able to have an indication because of urgency. And so when we're thinking about the development of these trials, what that's going to look like and how as a community will actually impact each other.
Sarah Philbin (01:22:37):
Rachel, do you want to speak to the role and the discussions we've been having around the topic of drug repurposing?
Rachel Byrne (01:22:44):
Yeah, look, I think drug repurposing is a really interesting space. As I said, we look at the drugs that have had approval through FDA, and rare diseases particularly is doing a lot in this space at the moment. You've probably seen a lot of it either advertised through social media or maybe you follow some of the rare diseases networks or influencers. They are looking to say, okay, with all those 4,000 drugs, maybe there's something that's already there. We don't have to create something new that is going to impact a pathway for maybe it's one individual, maybe it's a group of individuals who have a similar etiology. The issue that we've had in the past is that you can only test one drug at a time. So you could imagine that's a really long trial pathway and it's very time-consuming and very expensive. Now what we're able to do is do things that we call high throughput trials.
(01:23:35):
And those high throughput trials can happen in different ways. They can happen with blood samples to see, okay, do any of these have an impact on blood that we might think they can happen in organoids where you can actually do high throughput of say a thousand drugs at the same time, or they could also then happen in animal trials. Now you can't do high throughput testing obviously in people. So these things are really important as we think about what that looks like, because what we are trying to do is find out earlier if something works or doesn't work. The majority of things are not going to work. So if you are looking at a thousand different trials, maybe only one of those works. But what we don't want to have to go is through individually a thousand of those different trials and wait for one to finish before the next one starts because that means none of this would happen in our lifetime.
(01:24:28):
Whereas these technologies and the pipelines that are now being created allow for what is more rapid answers. And again, some of this is going to be the answer is going to be no, and that can be really hard for researchers. They might've spent their whole lifetime trying to work out what this might be. And sometimes knowing what doesn't work is just as powerful as knowing what does work because then we don't have to continue to invest to go down that pipeline. And I think that's sometimes what we've seen in cerebral palsy is actually some ideas we know don't work, but we keep doing them out of hope and just going, "Oh, well maybe if we just change it this little way, it'll come with a different outcome." But yeah, that's something that we really need to consider.
Ashley Harris Whaley (01:25:13):
Rachel, to this point, because I'm not sure if we did, could you define what is an organoid? We've mentioned that quite a bit. What do we mean by organoid when we say that?
Rachel Byrne (01:25:27):
And again, I am not a basic science researcher. I'm not a clinical researcher, so I am probably going to butcher this definition. But when we think about organoids, we can think about how we developing cells or reproducing cells that are in a dish. It sounds a little bit pieces. And so we can get muscle tissue. And with that muscle tissue, we could reproduce more of that muscle tissue with the same biology and the same makeup, the same genetic makeup even. And so that's now what we're able to do across different organs of the body. So one of the last frontiers in that space was thinking about how do you actually duplicate brain tissue and how do you develop in the same way that other brain cells would develop? And how can you make sure that those neural connections actually are there and they're communicating with each other?
(01:26:21):
How can you replicate inflammation? So there's extraordinary labs around the country now that are advancing this technology in ways that is pretty remarkable. And so that's where that space is. It's imagine how are you recreating, I suppose, that tissue.
Ashley Harris Whaley (01:26:44):
Thank you so much.
Rachel Byrne (01:26:45):
I know - And again, I just want to reiterate that this is not my level of expertise and we've got phenomenal people as part of this network that that is their level of expertise. And so that's why we want to continue this webinar series and dive deeper into some of these questions.
Ashley Harris Whaley (01:27:01):
Yes, thank you, Rachel. I think we are coming up on time here. And I think that's a really good thing to remember as we wind this afternoon down is this is not the end of a conversation, y'all. This is the very, very, very, very beginning of many conversations around this work, around what we are doing, around what the network that we are building will be doing, and what this means right now in this moment for our community, but also what it's going to continue to mean for our community as these things progress and develop. And before we each give our own little last parting words, I just wanted to thank all of y'all who are here this afternoon for caring about this and for being willing to invest your time in it, to be willing to listen and ask really thoughtful and engaging questions. Our community is what we're all about, so it's nice to just be reminded of that.
(01:28:10):
Yeah, that's kind of it for me. Do any of the rest of y'all have any last words? And then keep it in mind that we'll be communicating about the next installment in this series very soon.
Rachel Byrne (01:28:27):
I think my final words are, we can't promise that we're going to get this right all the time, whether it be that we choose the right research to invest in or whether we communicate this perfectly all the time. But I can promise you that we are going to try and keep our community up to date and along for the journey as we do it. And that's really important to us. What we don't want to do is have this initiative get done in a silo and have it done that in three years' time you hear about an outcome. We are going to try to inform people as quickly as possible throughout this process while also respecting the research process. There is a fine balance there, but that's something that I can promise as an organization that we're committed to trying to achieve. And just so grateful, as I said, for the incredible team that I get to work with every day.
(01:29:19):
And I'm just so grateful that everyone got to hear from all of you during this process because this is a conversation that has a lot of nuance and it's something that we talk about every day at the organization. And what we're trying to do now is bring all of you who are listening and part of this webinar today into that as well. And yeah, thank you from me.
Sarah Philbin (01:29:39):
Yeah, thanks everyone. Thank you
Jen Lyman (01:29:41):
Guys.
Sarah Philbin (01:29:42):
Go ahead, Jen.
Jen Lyman (01:29:43):
No, you go ahead.
Sarah Philbin (01:29:46):
No, I was just going to say I appreciate everyone joining today. And I think as someone who normally works on the far end of the translational science pipeline And I'm very cognizant. It's great to hear about all this basic science and all these exciting new possibilities, but that doesn't always reflect people's lived experience with the health system and what they go through with insurance or care access. So that's something that I personally care a lot about. It's what I studied. So from my standpoint, that's what I keep in mind as I'm supporting this work. So if that's concerns of yours, I know we heard threads of that in some of the questions. It's something that is forefront of my mind as we move forward.
Ashley Harris Whaley (01:30:38):
Thanks so much, Sarah. Jen?
Jen Lyman (01:30:40):
Yeah, thank you all for joining us. And I guess if you are interested in participating in research and clinical trials, like I mentioned before, we do have a form on CP Resource that you can fill out or to join our research directory, sign up for the newsletter as well. There's lots of great resources on CP Resource from expert videos to this webinar will be on there as well if you want to share it with folks. And of course we've got podcasts and all of those kinds of things. So obviously CP is all encompassing for the families and for those who are living with it. And so we try to address that across all of CP resources. So thank you.
Ashley Harris Whaley (01:31:29):
Thank you all so much. We appreciate you. Have a great evening.
Rachel Byrne (01:31:33):
Thanks so much everybody.
Jen Lyman (01:31:35):
Bye. Bye.