Jen (00:06):
Welcome to the Cerebral Palsy Health Podcast. We dive deep into health topics that impact people with cerebral palsy, such as stem cells, genetics, neuroplasticity, exercise and fitness, nutrition, accessibility issues that could be confusing are controversial, and those that offer hope but might not live up to the hype. I'm your host, Jen Lyman. Join me in conversations with leading experts as we separate fact from fiction tackle tough to understand topics and try to shed light on how best to maximize and optimize health participation and quality of life for those with cerebral palsy.
Nathalie (00:43):
Good morning. Good morning, Jen. How are you? Oh, good. It's still, as you know, the holidays,
Jen (00:53):
I feel like I get to see a lot of you. We had a late meeting last night with a phenomenal advisory council of individuals with lived experience. We've got parents and we've got
Nathalie (01:08):
Individuals, two people,
Jen (01:09):
Yes, adults, the cp, and on this amazing research study that you're doing, and I want to talk about research today after having that long meeting last night and really hearing their input into the direction of this study and how it's going and where it will go, it really got me thinking about the questions that families ask me when I'm screening them for some of the clinical trials and how to participate in research. And there's no better person to talk to you about than you. So I thought this would be a great topic for today as both a researcher and a physician and a mom who has participated in research. So we can kind of cover all of those. You're not ambitious.
(02:03):
Not at all, but we've got an hour, right? Okay. And right now, on a regular basis, on a daily basis, I screen families who desperately want to participate in a study so that they can, for most people, help their child or help themselves improve function, improve pain, improve quality of life through research. And a lot of the time people, it's wonderful when I can fit somebody into a study. Other times I might not be able to, but I'm able to offer resources. But a lot of the time I find that studies are not that conducive to participation in them, and they're hard. They're hard to be a part of. They're hard for families to get to.
(03:09):
They're not designed in such a way that is conducive to participation. Then I hear the other side from researchers saying, oh my gosh, we can't recruit, and I'm, well, gosh, can't imagine. Why Have you thought about including families in the design of the study? And that's something that you've done. I've seen you do it with this current one that we're working on, and then of course with Apple's tele, and I wanted to ask you about that and how you design studies to really make it conducive to participation for families. And that's a huge question. I know.
Nathalie (03:54):
So as a researcher, and so everyone has a slightly different process by which you go from that aha idea to conducting a clinical trial. Everyone has a different way of starting. People are like, where do you get the ideas? Well, so I'm really lucky in the fact that I'm a doctor who works with families of children with cp. So just watching them and listening to them, I'm like, we've got to do something about this. So a lot of researchers will have the start of an idea, but the start of an idea can come from your patients. But for those researchers who are not clinicians, it can come from deeply understanding the science of what's behind something and saying, wait, there's something that doesn't make sense here. Or if I could make sense of this, then I think we could make a difference. Because there's all types of researchers.
(05:09):
It turns out that a clinical trial that you might participate in often starts at the very, very beginning in what's called basic science, the science that most people discount because it's so removed from them. But it's so important you can't design a clinical trial unless some researcher has carefully gone over that initial mechanism, the thing that the molecule, the gene, the part that's so at the molecular level that might constitute a reason for why your treatment might work. And so when people see participation in a clinical trial, what they're actually seeing is this incredibly long chain of someone did the science, another person had an idea on how to use that science. Sometimes it's the same person, but often it's not. And then the person who had the idea on how to use that science saw how it could be applied, and then went through all the steps of trying to test it out. And it finally makes it to a clinical trial. And I don't know about other people, but I can tell you, even reading all the time, the basic science and the clinical science that's out there, I'm kind of a gee for that. I'll read two or three papers every day about what's coming out in CP or in other fields. So the thing that happens though is that every day I might have a hundred ideas.
(06:52):
Out of those a hundred ideas, I don't keep track of all of them because otherwise my head would explode. Now, I might keep track of a few that are important. And it used to be that a scientist had to write it on post-its or stuff like that. There was a time when my kids were little where there were yellow post-its falling into the spaghetti sauce because what I was doing is when I was having an idea while I was cooking is I'd stick it on the hood, but it turns out that steam can actually undo the post-it glue. So anyway, you start with
Jen (07:30):
All, would you talk to post-it about that?
Nathalie (07:31):
Yeah. Well, I don't think that's what they designed it for. So then you get all these ideas and you weed through them. Some of them, it turns out, don't make sense once you poke holes in them and then you finally get to an idea that you think that could make a difference, that's the key time you think it could make a difference. But do your patients and families actually think it could make a difference? And you asked me about the first step.
(08:04):
The first step is could this make a difference to families? Is it more than an exercise in understanding what is going on? Because if it is an exercise in understanding mechanisms and the science behind something, then it belongs to basic scientists and preclinical researchers. And these are the types of scientists who can work with everything from cells to organoids, which are these collections of cells that create mini organs or animal research, or even now AI models, which are crazy in helping researchers simulate what might happen. So you've got that whole bit of science, and what should make it to people is first of all, only the ideas that are going to make a big difference to the people that they concern. And that's the hard part, right? Yeah. Because a lot of people don't ask. They, they're convinced.
Jen (09:15):
I talk to families a lot about, we'll be reading, I have a small group that looks at some of the studies that are coming out and decides whether families would be interested in them. And a lot of these articles that we read are, we look at 'em, we're like, duh, of course we could have told you that this was what was going to happen and what are you going to do about it? And I think that in participating in trials or getting families involved in how to, I guess when you're talking about the basic science versus it's the what are you going to do about it? How are you going to help and change things? And at what point do you engage the families and say, okay, here's the idea that's percolating with me, and how do you want to see this implemented in a study? How does that happen? How do you get to that point?
Nathalie (10:18):
So it's actually really long and complicated because I told you about the ideas and the basic science and the preclinical models that need to happen. Let's say you've got a valid concept, and let's say that your families, the people you work with have told you that yes, it would make a difference to them. Sometimes you know that, but sometimes you don't. So sometimes what you have to do is you have to do focus groups or you have to do surveys. And the CPF has been great about at least helping me and some other researchers I know to just identify their priorities over the lifespan. That's very helpful.
(11:01):
You can do it through systematic reviews. Systematic reviews are a way where you put all the evidence together and it lets you see where the gaps are. And then you can actually, instead of just stopping there and asking just a few parents, what the CPF has helped me do is say, okay, but if I asked 400 parents of children with CP across the us, what are their preferences from new surgeries, new medicines, new parent driven interventions, new therapist driven interventions, what are their priorities for us to look at next? And so that's one way focus groups is the other is simply asking, I mean, I remember I was asked by our Australian colleagues at the last A-C-P-D-M to do this. What is an advice you would give a young therapist taking care of a child with cp? What's the first question you should ask? And to me it's always, how can I be of help? If you just start with How can I be of help? You'll probably get some really unexpected answers instead of saying, does this help? Does that make sense? It's the difference between saying, how can I IB of help to you without limiting it? And then they'll start talking to you about what matters.
(12:43):
So that's one way large, large surveys is another and one-on-one. And then identifying gaps in the literature saying, whoa, all these amazing scientists have done all this stuff. And it seems to me that here's the missing piece between all of them. That takes a while too. Then when you go from that, the next thing you have to do is test it in a pilot. And that's not easy because you need funding all of that. Exactly. And the pilot is the first thing that has to happen. And then it goes through different phases as depending, and it turns out that I know most people think about the FDA, right?
Jen (13:33):
Right. So I was just going to say that. So I don't know. Let's say you start out with your pilot and then how do you get from pilot to FDA clinical trial or a phase one trial? A phase two? I think that's something that a lot of families ask me about. And you've got your pilot, you've done the basic science, so now you're at the, how do you get there? Is it do company you
Nathalie (14:03):
Usually, it's not straightforward a pilot, what it usually lets you do is simply test whether the concepts might be valid, but you're usually not going to what's called a phase one. So phase one in an FDA trial at least. And those are really for drugs and therapeutic device groups. They're not supposed to be for behavioral interventions like therapy or pt, ot, things like that. The FDA designations are very much for certain types of interventions. One of the easiest to understand is most definitely medicine like a drug. And for the first thing you need to show is safety. And the second part is going to look at, and the first part can look at safety and dosage. The second part can also look at dosage and usually efficacy. The last part is going to be the third phase is a multicenter clinical trial. So each phase of the clinical trial gets bigger,
(15:23):
And the key to the last phase is that they need to be generalizable. So even once you have shown that something, let's say I show something works in a clinical trial that's a big randomized control trial, but it's only at one institution. The problem is people could then say, okay, you've got an intervention that works, but what are you going to tell your individuals with CP or your families that it worked at this one place? It's not totally fair, right? Because we're so desperate to get treatments that we might think this is the be all and end all. So you have two choices. It's not true. Just because it worked in one place with one population doesn't mean it's going to work with everyone.
Jen (16:17):
And that makes a lot of sense to me where families could be choosing that particular location. And that would bias the study in that sense that you might have families of different means that are just going, or that particular location only chooses these families to be part of, so it's going to impact their results. So it would make sense that you would have, so
Nathalie (16:49):
What's interesting is that if the FDA has approved after that phase three, you can then go to a phase four and phase four at the FDA looks at generalizability. And this is what I love about generalizability is knowing that it's going to work in different settings with really diverse types of populations. It's really hard to do. It's really expensive. And sometimes it needs to also look at another thing which researchers don't necessarily explain to you, which is the difference between efficacy, which is how a drug or a treatment works under research conditions and effectiveness, which is how it works in a real world setting. So you might participating in a clinical trial and we're going to talk about that, Jen, but, and that drug works for you in the clinical trial, and you think you want to tell your friends, oh my God, this is so amazing. It works.
(18:04):
But it worked under research conditions with someone carefully monitoring that you were doing everything right at every step in a precise timing with precise assessments and in the conditions that they told you you needed to do it in effectiveness is real world. And it means that sometimes someone might miss a treatment because life happens. Sometimes the treatment had side effects and you had to stop it for a while and restart it, even though those side effects are not necessarily really bad. They're just uncomfortable. There's all these other things that play into effectiveness, which is real world, and people don't tell you about that necessarily. But most researchers stop at measuring efficacy and they don't go on to test what's called effectiveness. But some do. Some do.
Jen (19:05):
It would make sense that people, especially if it's a drug company or something like that, wouldn't necessarily look at effectiveness beyond, because it's more money, like you said, it's very expensive to do. And once they get that FDA indication that they can use that drug for a particular indication going on to do the next phase, wouldn't financially make sense. Is that the case or? Well,
Nathalie (19:32):
No. I mean a lot of drugs actually go into long-term safety and effectiveness as well as a lot of them actually do the ones that are going to be used at a really large level usually. And then one of my friends, he's John Williams, he's the chair of pediatrics at University of Madison, Wisconsin. Anyway, he has this soapbox that he gets onto, it's so funny, he calls it vaccines and Viagra. Okay. Oh my gosh, are we allowed to talk about this? Absolutely. Okay, so he says, so Viagra is a medicine that helps with male sexual dysfunction. Let's put it that way. In some ways, do you know how much money has gone into testing Viagra and its various phases, all its effectiveness, it's everything, right? I mean, we're talking millions of new, we're talking hundreds of millions, right? I can
Jen (20:55):
Imagine. Yeah.
Nathalie (20:56):
So however vaccines he uses that as an example. It starts with a V. Almost nothing goes into them. And certainly, I mean, to try to get the funding for long-term safety and effectiveness, there are giants in the field like Kathy Edwards and stuff who've done it, and they're remarkable. The CDC is really great actually about funding this type of research to make sure it's okay, but it's a laborious process and you have to fight to get the funding to look at it. So wait, if you're studying Viagra and you're helping adult men who want to function, you're going to get a ton of money and it's going to be really easy to do. There's a lot of people who are going to want to participate, and you're just going to get a ton of funding. It's going to be easy to do. Whereas if you are trying to study something that is for kids to try to prevent long-term complications, it's going to be a fight all the way to try to get the support, the funding, the parents on board, all of that. And he calls it this, it's this problem where to look at real world effectiveness. Actually, when we're looking at pediatric research, it's a lot harder than adult research actually. And if you're looking at something that has a lifespan effect, it's going to be harder also than if you're looking at something that has an effect in the moment.
(22:52):
And yet, when you think about it, the reason I bring this up is with cp, a lot of what we do should think about the lifespan, right? You and I have talked about this, why we shouldn't just think about interventions that are going to change them now, but we should think about how they're going to change the future. The problem is getting funding to look at that kind of research is different. And there are stages, just so you know. And I talked about that's what I was going to ask you, clinical trust. There are stages as well for things like therapy that's considered behavioral, and they're not the same exactly as the FDA ones, but people should actually, if they were doing this right, go through every stage of that research and not try to skip the safety one. Because think about it, would you want to take a medicine for, I don't know, heart disease or anything else? And it hasn't been tested for safety
Jen (23:58):
First. Exactly. And I think that it's, yeah, there's not a chance. And that's always the safety and side effects and those things. When my doctor prescribes a medication for me, I'm a migraine sufferer. And the first thing I'm wondering is what are the side effects and how is and of course safety and has this been tested?
Nathalie (24:25):
And I mean, we now know that we've done stuff. I remember the horror, I mean Rachel, the director of the CP Foundation, she was there with me when I first got the results of a pilot trial that I did for efficacy and safety. So that's called a phase one, even in behavioral and therapy science and my results, I was so shocked. I was actually devastated because I thought this therapy intervention was going to be so
Jen (25:07):
Great.
Nathalie (25:08):
And when I looked at it, even though the effects were eventually reversible, I just want to point this out, I showed that at the level of the brain, even though at the level of the body, I couldn't see it at the level of the brain and sensation, I was actually changing things in not a good way. And I remember it was Rachel and people like Rosalyn Boyd in Australia, and I run Novak in Australia, and they were like, it's okay, Natalie, it won't be forever. I talked to Kathleen Friel, another researcher who she's remarkable. She has cerebral palsy and she's like, it's going to be okay. It's going to be okay. But it's that devastating moment where even in a therapy intervention, you realize that if someone had actually looked very carefully at the safety profile, they would realize that, oh my God, why hasn't anyone ever checked to make sure this is safe?
Jen (26:13):
And I guess that leads me to a question about these therapies. And when we go and a physical therapist is doing a new therapy with our child or is trying a new intervention with our child, how do we know it's been tested for safety?
Nathalie (26:36):
Or you have to ask, right? Yeah. And you have to ask. And so that's part of it. If you think that just because it's therapy, it's safe, it's so hard because when you're a parent of a kid with cp or when you have cp, you trust your therapist because you form this bond with them. And it has to be based on trust and reciprocity.
(27:10):
But at the same time, if you actually want that person to be deserving of your trust, they can't just be helping you. They have to be honest with you. And if you say, alright, so you're working with me and my child on treadmill training, talk to me about has anyone studied if this is safe and if it works and if it works, do you know it works for everyone or just for certain types of kids? And there are different perspectives on safety. There's also a perspective of value. And the only people who can talk about value are actually the people getting it, not the researchers, not the clinicians.
Jen (28:01):
To me, it's wasting time.
Nathalie (28:04):
So that's it value,
Jen (28:05):
Right? Yeah. And if your child is in an intervention that's not necessarily for their specific type of CP or it's working on or that's
Nathalie (28:17):
Not
Jen (28:18):
For them, it's not safe for them or it's not even a functional goal, what is the point? Why would treadmill training would be a good example to me. If the goal for somebody who's perhaps a GMF CS four or a GMF CS five where maybe gate training would be a functional goal, what would the goal of treadmill training be? Is it cardiovascular, is it that, and just getting exercise like we've talked about,
(28:52):
And is that physical therapy or is that exercise? So I wonder, and I think that's really hard for early when you're a young family and you're trying to even, you don't know where your child's going in development. And some of the families that I'm talking to, their babies are so young, so obviously everybody wants their kid to walk or everybody. And I guess it's really hard to sort through which therapies are we going to choose. And if you've gone, I guess kind of going back to you and the research you're doing, this is the question that I all think get is how do we know which study to participate in? Or there doesn't seem to be any studies for my baby or for my child. And I see that a lot with my own son. He is quadriplegic and I don't see as many studies out there.
Nathalie (29:49):
Have you participated in any studies?
Jen (29:51):
We have participated in this study. We did actually an FDA approved study,
(29:56):
And it was fascinating. I remember getting a call that it was open and I couldn't believe it, but it was the first FDA approved stem cell trial. It was back in 2015 or so at Texas Children's, Dr. Cox, and he was looking at stem cells from bone marrow. So we did not collect my son's cord blood. So we were looking at, and of course I didn't want to go out of the country or do something that I knew was unsafe. And so this was a study that was looking at your own mesen, Kyle stem cells that come from bone marrow and then an infusion. And it was a double blind placebo controlled study that lasted for two years. So we had to go and do it could have been a shame. So just
Nathalie (30:57):
For anyone listening, double blind means that there's two different levels of blinding. The first is that the patient doesn't know what they're getting, and the second is that the researchers don't know what's being given. So that's why it's called double blind, the patient and the researcher.
Jen (31:16):
Thank you for that clarification. I forget about that sometimes. Yes. So it was double blind. I had no idea whether he got his own stem cells or not. And neither did the researcher, and it was an intensive trial. We had to go back to the site multiple times for MRIs and for blood work, a lot of testing over that two year period of time. The trial was similar to apples, Apple's tele that I do with you in the sense that everybody who was in the trial ultimately got to have their own stem cells. Well, so it
Nathalie (31:52):
Was a waitlisted trial. And by waitlist we mean that everyone in the control arm who didn't get it, when it becomes unmasked has the opportunity to be on this wait list to get the treatment. It's the way we try sometimes to be fair when it's something in two cases, when it's something that has a potential to make a huge difference and that you could not get otherwise or, and it helps incentivize people to be part of it. The other option is when you feel that without that, it might be years until there is an approval even with this trial. And so it's a way, if the results are good to sort of tell people if the results are good, you will not have to wait for all of the other things that happen. I like it in mind because I already know it's safe, as in apples, they get to choose. At the end of it, they get to choose which of the two. But that's because it's already been proven safe. If it hadn't been, we would have to wait until we analyze the results to offer it.
Jen (33:21):
And this study, actually, he did receive his stem cells in the initial after it was unmasked. And I had a hunch I thought that he did. I saw some differences in him, but then of course I kept on questioning myself, is this placebo effect and am I imagining things? But it was fascinating during the study. So after we had done the two years and got the results, saw the results of the MRIs along the way, we really did get some, we got information that I thought was fascinating and helpful to learn about, first his brain injury, but also how the stem cells actually made some differences during trial,
Nathalie (34:10):
What we did see. So did people come back to you after you were done with the trial and say, here are the results in words that were like, I mean, you're highly educated, but did they come back in words after the trial that were in layman's trend that literally anyone could have understood and say, here's what we found. And did someone do that for you?
Jen (34:34):
They did do that for me in the short term. So they did that for me on an individual level. I spoke with the site coordinator and who was a nurse practitioner, and he really went into, these were the results of the MRIs from the beginning all the way to into the end of the two years. And this is what we saw. We saw a short-term reduction in inflammation, but it came back and you could see it. You could see it in the numbers and everything else. So we did have a discussion about that. What we did not get, or what I did not get was the final results of the study. I never saw what happened with all of the other participants that were in the trial and whether it was shown to be effective or not. And I really obviously would've liked to have seen all of that. And I'm actually not sure at this point. It's been 10 years, whether that study has actually been published. I did. Did you look it up? No, I need to, I, I think I assumed that it would get sent to me and it just never did. And I sort of forgot about it. I knew what the results for my son were and what I saw personally, I never even looked it up. I should. So
Nathalie (35:54):
I can tell you, is it autologous cellular therapy for cerebral palsy? A randomized crossover trial by Charles F. Cox? You got it. That's, it was published in 2022 in brain communications. And I can look at some of this. It looks like it was a phase one two, a trial, randomized blinded placebo control crossover study. So that a crossover means that everyone eventually gets the treatment. Some get it first and some get it later.
(36:29):
And it says at the 12 months post-treatment visits, participants who originally received the placebo received either bone marrow derived mononuclear cells or umbilical cord blood treatment. 20 participants were included, seven randomized initially to placebo, meaning the probably saline solution and 13, randomized to treatment. And then they talk about it. It says none of the participants experienced adverse events related to the stem cell infusion. So no short-term side effects or safety concerns. And then it says it didn't dramatically alter motor function. They looked at the MRI, like you said, and they showed some differences, and then they said there was a significant improvement in corticospinal tract. So the tracks are the tracks, the motor tracks, they go from the brain to controlling your muscles. So it's really interesting. And so the conclusion was there may be an improvement in myelination in some groups of patients that correlate with small improvements in the gross motor function measure scale. A larger autologous cord blood trial is impractical at current rates of blood banking. Interesting. Either increased private banking or match units would be required to form a larger scale trial. But what they showed, what's really, really important is safety. And they looked at both and they said, this is safe. So that's pretty cool. You participated in the trial, the results came out in 2022, see how long it took to get those out.
(38:22):
So that's the other thing. And what I'm telling you is it's pretty great because it was safe if you noticed any improvements. There were some improvements probably on MRI, possibly some improvements in motor function. But you saw the conclusion, which is after all that time and effort, the feasibility and practice is going to be really, really hard to do of a large scale trial.
Jen (38:49):
I know he says that it was due to blood banks. And is that a funding issue for families or is that a
Nathalie (38:59):
Funding issue? No, no. It's a practical issue. So you'd have to blood bank at birth, right? But you said yourself, you didn't. Now I have two children on one of them. It was offered to me and I did it my second child. Unfortunately it wasn't my child who had ccp. So you can't predict necessarily, right, as a child is being born the second they're born when they're asking you about blood banking or right before something is going on. And then the other thing is, I don't know if it's always offered to parents of preterm babies, which represent a large portion of cp. So you've got all these logistical problems. You'd have to think in advance, I need to blood bank in case something were to happen. I don't feel like I was given that option. Well, because he talks about it even in the abstract private versus public blood banks. If it's a private blood bank, you're going to have to choose to bank that in a private bank with all the implications of it.
Jen (40:05):
No, but what I mean is even, and maybe this is just a preterm baby thing, and when you're having a baby preterm and it's an emergency, you wonder how, or at least I wonder and I wonder afterwards. I feel like that should just be standard. People just come in and say, Hey, do you want to bank your cord blood?
Nathalie (40:28):
Yeah, but that's hindsight. Hindsight is 2020. Jen, you know what? When you are going through birth, either you don't expect, it's not like someone says your child is going to have CP when they're born.
(40:42):
And now when you think about it, you are like, I wish it was offered to everyone, but A, is it practical? And B, would people even take, if you're stressed and you're already going through a ton, you may not be thinking enough to go through that. You might be really vulnerable and you might be like, oh my God, I'll mortgage my house to do this. There's so many ethical questions surrounding that. I don't think it's a straightforward answer. I think that that idea, everyone should be offered that. That's lovely. But in reality, I think it's us talking as parents of children who would've needed it. And it's not necessarily what's going on. Think about the debate with vaccinations right now, every child has a right to be vaccinated. Now, parents may choose to not do it, and it will hurt other children and it will hurt other people like older adults who are dependent on everyone being vaccinated. However, it's a parent's choice. And all we can do is say, this is what the a p recommends, and we counsel, but we cannot force people to do this. And even for something that benefits society as a whole, we have trouble. Imagine what it's like in something that might only benefit a tiny section of people. It's even harder.
Jen (42:11):
Makes sense. And to have it as a, you have to buy in, so it wouldn't necessarily be equitable.
Nathalie (42:18):
Exactly. So just like vaccines, you have to buy in, right? The thing about vaccines is that they're free. You don't pay for your vaccines and you offer free core
Jen (42:29):
Banking to everybody,
Nathalie (42:31):
Precor banking to everybody. Even with that, there are some people who would still be like, oh, what are they going to do with this? Is it going to hurt me? And there's still be all these conspiracy theories and everything else. Would everyone do it? No. Beyond conspiracy theories and all of that, they're still the people who would be like, I won't need it. I can't be bothered. I'm too stressed to think about it now. There's so many reasons. It is difficult. Nothing is simple. Nothing is black and white. And I think it's really important for parents to understand, and we can talk some more about research. I know you and I are running out of time right now. What I would tell you is if you're a parent or an individual parent of a child with CP individual who have CP and you're thinking about participating in any research, and it could be physical therapy, occupational therapy, it could be nutrition, it could be anything or a medication or a surgery, I would say, tell me about safety and don't just tell me if it's safe or not.
(43:42):
Tell me, has someone studied it could safe or not? So that when I take my chances, I actually know you can choose to do physical therapy without it having proven to be safe or occupational therapy without it proven to be safe. But you need to be able to make that decision knowing that, and not with someone offering you platitudes. Oh, well, people have been doing it all the time and it's perfectly safe. Well, no. So what you're telling me is that no one has studied whether this is safe, but you think in your opinion as someone who's working with me and you're willing to stay with me, that this is safe. Okay? There's safety and then there's value. And you and I talked a little bit about that. I would say, what have other people said about the value of this to them? And we talked to you and I, and we'll have to talk about it again, about large intensive treatments that supposedly are valuable when people report anecdotes, but that no one has actually published on the value of it.
(44:52):
And value is another thing where it's a very personal thing, right? Is the money, the time, the effort, and what your child or you as an individual thought about it, has that been reported? Not in anecdotes, but has it actually been reported somewhere? And show me that. Show me that other people have as a group seen a tangible value in this. Because value is a tough, tough thing to determine. So is it safe? Does it have value? And then if you want to go into it, tell me about the mechanisms and the proof that this might actually work.
(45:42):
This should all be part of the informed consent, but sometimes all we have to show is that it is faith or that if it's not that we're telling you about the risks in words, you can understand if it's not been shown to work, that we explain that in words that you understand and that we're trying to test that. And then the last part is the value is never explained in an informed consent, not in ways that most people can understand. Now, I will tell you that there are efforts in an informed consent to talk about that as opposed to you, it's called beneficence, and it's the value sometimes to society versus the value to yourself. Listen closely to those, and that's the point at which you as an individual or a parent can say, I want to go back to that one. You talk about value to society. I understand you can't talk about value to me, you don't know yet, but talk to me about the value to society a little bit more. I want to know about that. What do you mean by that?
Jen (46:50):
Sorry, I feel like the, or the study I was in actually has that in a sense, you're looking at it by participating in this. There is sort of, and what you just read to me, it comes back with recommendations that I think do have that idea of how it could be a value or where it lies in the value to society. Does that make sense how I'm interpreting what you're telling me? It may have had, we may have seen a small difference in my son, but the overall benefit to this and how to move that forward.
Nathalie (47:27):
But that has to be articulated to you. And it should be more than simply. While we don't know if this will benefit direct you, it will benefit society through advancing. And I want to know exactly what
Nathalie (47:42):
Advances. And as far as values concern, just answer me, what is this something? Is this an outcome that the people, your stakeholders, people with CP have actually said is valuable? And show me that. Did you ask them, do you have a survey? Did you publish it even if you didn't publish your survey? Can I see it?
Jen (48:07):
Yeah,
Nathalie (48:07):
It's okay because sometimes we do surveys and we don't publish them because journals are interested in it, but we have the surveys to show we did the work.
Jen (48:16):
What about with your studies? Do you share the results with the families who participated afterwards? And how do you,
Nathalie (48:23):
So publishing is important, right? And the other thing is talking about it everywhere at parent forums for apples, the first apples, it was so easy because every parent who participated immediately knew. It was like so instant
(48:42):
Within four weeks, they already knew. And part of it is because this is what we did. We used measures that were measures not just of capacity, which is what you do under ideal circumstances, like you did the GMFM. That's a measure of capacity. But we also did something called a measure of performance, which was how a child does in the home. And the parent reported it to us. And so by doing that, the parent had an internal measure of how their child was doing in the home. So each and every parent at the end of it, by performing this ial, and I still do this in apple's telly, the last measure at 12 weeks is the ial because it allows the parent to report on how their child is doing in everyday situations in the home. And so I think that that's one of the ways that you can immediately let a parent reflect on, oh, my child can do all these things in the home in everyday life.
Jen (49:49):
Do
Nathalie (49:49):
You see what I mean? So there's that. There's also disseminating research. The CPF is fabulous about doing that on CP resource, which is a really great way to have little blurbs that explain in parent terms. And the other way is to disseminate it at conferences and translate it into applied workshops that let therapists learn how to do it. And as you know, I've done that also to make sure that, so it's implementation funds is something we can talk about another time. It's different than research in some ways, but it has to be done to make sure that your research doesn't just stay in this ivory tower of universities. We can talk about it another time, but I would ask those questions. Tell me, and it's not just for a research trial, it's also for your everyday
Jen (50:42):
Therapies and for going to a
Nathalie (50:44):
New is this say, and if it's say, don't just give me a boil play to answer. Don't just say, oh, yeah, I want to know how do you know it's safe? Is it just through your experience or has someone studied this so I can take the risk? I can take the risk knowing this, does it work? And who does it work for? And tell me about the value of this. And you'll see that some things that are really expensive, they're not able to actually, they'll say, well, we know it's expensive, but our parents have said that this is worth it. Okay, so I want to know where did you publish that? Not just testimonials, because you're only going to pick the testimonials that are in your favor. You're never going to pick a testimonial of someone who says, this is a total waste of money and time.
Jen (51:36):
That is a whole conversation that I want to have with you on, I call it considering the alternatives we both have
Nathalie (51:48):
Done. Yeah, and you and I have talked about that. I call it the good, the bad, the ugly,
Jen (51:51):
The bad, the other. I call it considering the alternatives. But yeah, sometimes I feel like the more expensive things get, the more snake oil it becomes. And trying to look at whether some of these things have actually been thoroughly researched for safety and effectiveness and for your child's actual needs, or is this something that is not appropriate for your kid? And how do you decide? We've gotten a little bit into the research today and what it looks like to get a trial started and how to participate in research and disseminating it later. And then I think the next step of this would be how do you choose these therapies that are happening out there? And there's so many and so few that are actually studied, just throwing it out there.
Nathalie (52:44):
Well, research is hard. Let's be clear. I first started studying the precursor of apples. I could only get $5,000 funding, $20,000 funding to do small pilots. And even that took sometimes a year and a half to get and then to do an NIH funded trial. Oh my god, that's years and years of begging to try to get that funding. And let's be clear, people will tell you that funding for CP research at the NIH is increasing. Not really, it's just that some studies study CP among other disorders, or some people might be studying basic science that is applicable to cp, but funding for cp CP research is still not there. A lot of it, there's more funding. We've talked about this at the CDC for example. There's finally funding for CP, a tiny bit to see what the prevalence is and things like that. But there's more funding for fragile XA disorder that is so much rare than CP than there is for cp.
(53:56):
So there's little funding and it takes time and effort. So be patient with your researchers because anyone who is trying to do research NCP, yes, they have obligations towards you to be rigorous and safe and to not waste your time, but just know that they are also fighting almost near impossible odds to do this work, and they are doing it because they believe. So try to balance that. I want to know, and I have all these rights, and I have all of this with compassion for those researchers who are not doing this for the glamor, there is none and the glory because there will never be a Nobel Prize for cp, unfortunately. It's just too complicated. And I mean, yes, I believe what Rachel believes that someday we might completely prevent it, and someday we might even have a cure for every facet of CP. And if we don't aspire to that, then we'll never get there. If we hadn't said we will land on the moon, then we never would've. Right? It's the moonshot. It is, and we need to do it.
Jen (55:14):
Thanks for listening to the Cerebral Palsy Health Podcast with me, Jen Lyman. If you enjoyed the show, please subscribe wherever you listen to your podcast and follow me on Twitter and Instagram. You'll find the links in the show's description. Please feel free to email me with comments, questions, and topics you'd like to learn more about at JB Lyman at Mac. That's MAC.com. This podcast is for educational purposes only. This podcast is not a substitute for a medical doctor or any other medical provider. This podcast is provided on the understanding that it does not constitute medical advice or services. We encourage all of our listeners to have an open, honest discussion about the topics presented on this podcast or any other medical concerns with their personal medical team.