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Kline-Tilford, A. M., & Haut, C. (2020). Cases in pediatric acute care: Strengthening clinical decision making. Wiley-Blackwell.
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Gator PICU Podcast
How Personalized mRNA Teaches A Child’s Immune System To Fight Cancer
What if a child’s own tumor could teach their immune system how to fight back? We sit down with Dr. Liggon from the University of Florida to unpack a first‑in‑kids, personalized mRNA lipid nanoparticle therapy designed for high‑grade glioma and osteosarcoma. Instead of chasing a single target, this approach isolates mRNA directly from each patient’s tumor and packages it into lipid nanoparticles, training the immune system against a wide range of cancer proteins. That personalization is the point: every dose is built for one child, aiming to overcome the limits of one‑size‑fits‑all immunotherapy.
We walk through the basket trial design and why starting with these two cancers matters, especially when relapse rates are high and outcomes are poor. Dr. Liggon shares early adult data from glioblastoma patients showing rapid immune activation within hours—fevers, chills, and transient hypotension—signs that the therapy is doing its job without dose‑limiting toxicities. We also get practical: the six‑week manufacturing timeline from tumor to vaccine, the use of radiation or a non‑specific priming LNP to bridge that gap, and how the team measures pharmacodynamic markers at two and six hours to understand dose response over repeated treatments.
Collaboration is the backbone of this work. Our PICU team partners closely with oncology and immunotherapy colleagues to deliver infusions safely, manage inflammatory side effects, and collect the data that will refine dosing and duration. Behind the scenes, a funding story comes to life: local seed backing from Stop Children’s Cancer of Gainesville sparked momentum that grew into support from the V Foundation, National Pediatric Cancer Foundation, Alex’s Lemonade Stand Foundation, and the National Cancer Institute. Looking forward, the plan is to expand this platform to medulloblastoma, DIPG, Ewing sarcoma, and rhabdomyosarcoma as phase one findings guide the next steps.
Tune in to hear how personalized mRNA can move from lab to bedside, what signals we’re tracking, and where this platform could go next. If this resonates, follow the show, share it with a colleague, and leave a review with the questions you want us to ask in future updates.
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References:
Kline-Tilford, A. M., & Haut, C. (2020). Cases in pediatric acute care: Strengthening clinical decision making. Wiley-Blackwell.
Additional Resources:
PICU Essentials on the App Store (apple.com)
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Welcome to the Gator Pick You podcast where pediatric critical care is real world learning. Whether you're grabbing coffee, charting at the bedside, on shift, or on the go, we've got you covered. Our episodes are short, focused, and designed to keep you sharp, informed, and inspired. Let's get into it.
SPEAKER_04:Dr. Ligan is an assistant professor in the Department of Pediatrics here at the University of Florida. He is currently leading a groundbreaking clinical trial testing a personalized mRNA lipid nanoparticle therapy. Correct? Yes. Yes. Absolutely. Or RNALP for Dr. Liggan, or excuse me, for children and young adults with high-grade gliomas and osteosarcomas. So Dr. Liggan, thank you so much for being here. If you want to go ahead and introduce yourself and give us a little background.
SPEAKER_01:Yeah, thank you so much. I'm happy to be here and talk to your listeners about our platform. As you said, I've uh I'm an assistant professor at the University of Florida. I came here about three and a half years ago and was recruited to work with my mentors, um, Elias Sayer, Dwayne Mitchell, and their immunotherapy group to help bring some of these new immunotherapies forward for patients with very difficult to treat types of cancers and hopeful that we will find a new treatment that will help them overcome their cancer.
SPEAKER_04:That's incredible. Very good. And Alyssa, do you want to introduce yourself?
SPEAKER_03:Hi, my name is Alyssa Hood. I am a nursing professional development specialist with nursing education focusing on the pediatric and women's units.
SPEAKER_04:And my name is Amanda Bradshaw, and I'm a clinical leader here in RPICU. And I uh help with quality uh improvement projects here as well. So, Dr. Ligan, we're gonna go ahead and um get started with the big picture. So, what makes this new clinical trial so innovative?
SPEAKER_01:Yeah, so I I think what makes this really innovative is that this is you know trying to make a treatment um or a type of treatment that hasn't worked for pediatrics in the past, namely immunotherapy, which is trying to train the body's immune system to recognize and fight cancer, um, we're trying to make that effective for pediatric cancers, which it has not been effective in the past. And so we're hopeful that this um new type of immunotherapy may be successful for pediatric cancers that have not uh previously responded to immunotherapy. In terms of the overall clinical trial, I think another thing that's very exciting about this is that we call this um a basket trial. And what I mean by that is that it's it's not something that's for just one kind of cancer, like bone cancer or brain cancer or kidney cancer. It's really a type of clinical trial where you could enroll patients with many different kinds of cancers and see for any specific kind of cancer, do you see signs that it's safe and potentially effective for these kinds of patients? And so we're using this basket trial to test this new technology that was developed at the University of Florida over the last 10 years called mRNA lipid nanoparticle technology, um, which we are using against multiple kinds of pediatric cancers. And right now we're evaluating how safe it is and how well it can activate the immune system in children with two specific kinds of cancers. One is a brain cancer called high-grade glioma, and one of them is a bone cancer called osteosarcoma.
SPEAKER_04:Wow, wow, that's fascinating. Um so for listeners who may not be familiar, what exactly is RNA-lp and how does it work?
SPEAKER_01:Yeah, so um the RNA-lp uses a version of mRNA technology where we package the the um kind of building blocks of the cancer called mRNA inside of lipid nanoparticles, similar to what people might know from the COVID-19 vaccines. Um what's unique in our approach is that this is really a personalized um treatment using our RNA lipid nanoparticles. Um we create a custom mRNA therapeutic from the patient's own tumor cells. Then using our engineered delivery system, we essentially um encapsulate that mRNA in a way that we think the body will recognize this uh cancer building block, the mRNA, as being dangerous and allow us to reprogram the immune system so the immune cells can more effectively recognize and attack the cancer.
SPEAKER_03:Okay. So that means each individual like r RNA LP that they're getting is made for that patient. It's not a generic thing that they're getting. Oh wow. Okay, exactly. So you're taking what they have, reusing it, and making this so they can then fight the cancer, hopefully.
SPEAKER_01:Exactly. And I think eventually we would love to make kind of a you know, quote unquote off-the-shelf vaccine that would work for anybody. And that's you know, a big um, you know, kind of part of our ongoing research, you know, back in the lab. Um, but right now what we're doing in the clinical trial is we're actually going in and we're taking a pay a piece of that patient's tumor and isolating isolating the mRNA directly from the tumor.
SPEAKER_02:Okay.
SPEAKER_01:So it really is personalized. Like so the what I'm giving back to that patient um could only have been made from that patient's own tumor.
SPEAKER_04:Exactly. Oh, okay. I did not know that part of that. I thought this was more of a non-specific. An off-the-shelf, like you were saying. Yeah, non-specific. Okay, very interesting. Okay.
SPEAKER_03:Okay. That's triggered a lot more questions now. So you talked about this as new in pediatrics. What have we done, whether overall or specifically you have health, what have we done for adults that's similar to this?
SPEAKER_01:Um, yeah. So anytime that you're bringing forward a clinical trial for children, um, almost all of the time you have to do some sort of testing in adult patients first to make sure that at least it's safe in adult patients before you bring it to a vulnerable population like children. Right. Um, and so we've followed that um that pathway here as well, um, and have treated several adult patients now with a essentially fatal brain tumor called glioblastoma. Um and in the first ever human clinical trial using the RNA LP, we've treated now several adult patients who've gotten the treatment. And what we've seen so far is that they have rapid activation of their immune system against glioblastoma. Um, we see that the immune cells move around very quickly within like two to six hours within the patient's body. And we start seeing um signs of inflammation in the patients as well, in terms of like they develop fevers, they develop uh you know, lower blood pressures, they develop chills. And I say these things that even though they sound a little bit scary, in some ways it's encouraging to us as the um the um team taking care of the patient because it tells us that the that our therapeutic is activating the immune system. All these things, fever, chills, it's signs of the immune system, you know, getting revved up.
SPEAKER_04:Like a desired, uh, a desired effect. Exactly.
SPEAKER_01:Right. With it's we know that the immune cells that are in the patient are um you know kind of getting activated, and we're hoping they're getting activated against that patient's cancer. Um so uh having given that to several adult patients now, um, you know, we've seen that yes, it has these biological effects that are leading to symptoms, um, but it hasn't caused any um toxicity that we call a dose-limiting toxicity, which is like so severe that you can't keep giving the treatment to the patient. Um so having seen that it has been um safe in the adult patient so far, along with these signs that it's activating the immune system, we've now uh decided to move this um into pediatric clinical trials. And I should also mention that before we brought this to adults, we did a lot of other preclinical testing in mice and actually in dogs as well. Not you know, not like we were uh giving the dogs cancer, but uh it uh you know regular pet dogs, osteosarcoma is actually very common. Brain tumors are very common. And so pay um owners of pets whose uh pets developed a cancer would bring their uh dogs to the vet school and enroll on the veterinary clinical trials. So this was the um, you know, kind of the the um building blocks of evidence that we you know generated to show that there's a chance this may really work against real cancer before we brought this forward to um real human patients.
SPEAKER_04:Wow, that is wow, so great. That's such a huge step. That had to have been really reassuring to see.
SPEAKER_01:It was. I mean, and uh yeah, there's been a lot of work um testing new cancer treatments in mice. Um, I think being able to test it in um veterinary clinical trials is something that's actually really unique to the University of Florida. Because I mean, I can walk you know 20 minutes down Archer Road and make it to the Vet School. And it just allows this level of collaboration that I you know haven't seen at other um institutions.
SPEAKER_04:Right. Yeah, an additional resource. Yeah. That's awesome.
SPEAKER_03:So I'm I'm interested that you said it you start noticing symptoms two to six hours, which symptoms are good in this sense because it shows that your body's you know kind of working against what you put in.
SPEAKER_01:We hope they are. Right. But yes.
SPEAKER_03:I feel like I want to ask the question of what was the end result already. I'm already like interested in how I know we're not there yet, but I'm so interested to see like how it progressed with adults and then how it's progressing.
SPEAKER_04:So presumably this is something that they will receive over and over again. So does the if they're having an inflammatory response, does that build? Does it get worse each time?
SPEAKER_01:That's a really great question. And I think to both of your point, it's something that we're really working to understand. Okay. Yeah, and and any time that we're running a clinical trial, um, especially phase one, never been, you know, used in uh humans, never been used in pediatrics. It's our obligation as um researchers not to just find out, does this work or does this not work? That's gonna take years to find out. You'll you'll need to see, you know, are these patients remaining free from their cancer? Um, but we also need to do a lot of work along the way to see, you know, why is it working or is it not working? That way, if it's not working, we can go back to the lab, we can uh try to um change around our formulations, figure out how to make um it work or overcome the limitations of the first design. Because this is the first design. Right. Like the chances that we're going to go, you know, with our you know, first design, obviously we hope it's going to work. Of course. Um, you know, we wouldn't give something to a patient if we didn't think that there was a chance that it would help them. But if it doesn't, you know, we need to learn from that patient's.
SPEAKER_03:That's the point of the trial to kind of figure out what the best option is. Exactly.
SPEAKER_01:Um to at to really answer your question, it will take longer to you know kind of see what these patients' long-term outcomes are. But I will say that we're able to take um, you know, the patient's blood at multiple time points after giving each of these vaccines. So we draw blood at two hours after the um the uh the treatment, and then six hours after the treatment, and we're able to see kind of how the um the activation markers change on those immune cells at those different times. And then we're able to compare, okay, after the third treatment, how does that compare to you know after the first treatment? Do we see a stronger immunologic response? If it's getting stronger, you know, can we actually back off a little bit on the dose so that our patients are not gonna have such a significant um inflammatory response? Okay. Very interesting. Yeah.
SPEAKER_03:Because either way, you have data now to then move forward in whatever direction you need to move forward in since you have been able to trial it. So that's always exciting.
SPEAKER_01:Exactly. Yeah. And that's all we want to do is um, you know, do what's best for the patient that's right in front of us, but also help us to better understand how to give it to the next patient as well.
SPEAKER_04:Interesting. Wow. So you talked about phase one a little bit. So let's let's kind of dive into like what does phase one, phase two, what are we focusing on with this?
SPEAKER_01:Yeah. So anytime that you're talking about different phase uh phases of clinical trials, phase one is the earliest phase, and phase three is the you know, kind of like latest phase. Okay. Phase one means that you're just trying to find out, is this safe and can I actually feasibly give this treatment to a patient? Okay. Um and so usually in phase one, you're testing several different doses of the treatment to see um is it safe at the lowest dose? If yes, you can try a higher dose. If it's still safe, you can go to the higher dose. You're trying to find like what is the highest dose that you can give without causing those dose limiting toxicities that I talked about earlier. Once you find the right dose, then you can move into phase two, which really answers that question of is there a sign that this treatment might be effective against this kind of cancer? And if the answer there is yes, in a relatively small trial, we're talking about like tens to on the high end, maybe a hundred patients, and I don't think we'll do that for uh pediatric cancers, but um, if there's a signal that it might be effective in phase two, that's when um you know you would really think about like a large multi-center hundreds of patients clinical trial. Um uh, you know, then that would be a phase three confirmatory uh clinical trial. We're still very much in phase one. Sure. Okay. Um and it'll take us some time to get through phase one, it'll take us some time to get through phase two. Um, you know, so I think we're we're a ways off from you know a definitive phase three study. Okay. Um and we're still very much trying to figure out what's the right dose of this uh new treatment.
SPEAKER_04:So is each dose, each dose is gonna be contingent on each patient? Considering it's made specifically for them?
SPEAKER_01:Um so when I talk about the dose in this context, I'm talking about like the amount of RNA that's being given. I see. Um And so um when we're enrolling new patients on the trial, like three patients in a row will be given a certain dose of RNA. And then if that is deemed to be safe, then we increase the amount of RNA for the next set of patients and so on and so forth.
SPEAKER_03:Oh, excuse me. Okay. So you're at phase one for the pediatric trial.
SPEAKER_01:We're still in technically in phase one as well for the adults. Okay. Although they're further along in like a phone.
SPEAKER_03:Okay, that's what I was wondering. Okay, so we really don't know what the phase two, phase three is even gonna lead towards yet. So we're definitely in the still figuring out what the how it's gonna go. Let's say it's right.
SPEAKER_01:And uh yeah, I think we're a little closer to you know starting phase two in the adults. But um even the progress is amazing.
SPEAKER_03:Yeah. I agree. You know, I think it's a great step to an answer in some regard, hopefully, of I I just hope that that's what it leads to. I think that's what we all hope that it leads to.
SPEAKER_04:Yeah, and I think it's easy to get really excited about something like that, you know, especially because I did I there was a headline on something in the elevator corridor about the the veterinary clinic having uh success with it. And so um, so I think it's just exciting to kind of see it translate over into the human world.
SPEAKER_03:And I think working in the PICU and seeing patients firsthand, I think also gives us a different perspective and almost makes me even more excited because you've seen the not great side. Yeah. And so I think we want an answer so badly that it's easy to really get kind of excited, emotional about it because you just want an answer. Right. I mean, and we're hoping we're thankful that for people like you that are able to do these things and want to do these things to help other patients and kids, especially, but anybody. So it's really neat.
SPEAKER_01:Right. And I I just want to really highlight something that you said there, uh which was you know that the fact that we're able to give this treatment and the PICU. And I I think that that speaks to a level of collaboration with our intensivists, you know, here at the University of Florida. Um, that yeah, I I it's not like there are no collaborations anywhere else. Right. But I've found it, you know, really um you know gratifying to work with the intensivists and the entire team here because you know, you guys, you know, we're here at this um, you know, big academic setter, we do see a lot of bad outcomes for a lot of different indications. And you know, our charge really as um an academic institution is how are we gonna move the bar for all of our patients? Right. And if that means that you know we have to, you know, find a way to admit our patients to the PICU to you know manage a lot of this, uh, these side effects that I just mentioned, you know, the the fevers, chills, low blood pressure, things like that. Um I'm really gratified that we've been able to work together in a way that um you know makes this really seamless for our patients.
SPEAKER_02:It's awesome.
SPEAKER_03:I think we feel the same. I think that we've whether it's your team, whether it's another research team that we've you know used and seen up in the PICU, I think we've, as their research grows, we see them more frequently. And it's very interesting. You get to know them, you talk to them about what what's going on and new things that are happening. So it is, it definitely grows from a small collaboration to a very big collaboration very quickly, which is really nice and really interesting to be a part of.
SPEAKER_04:And I think it's really helpful, it's uh very special to see somebody very passionate or a team who's very passionate, especially about something so specific or niche or whatever. Um because I think it helps A, it helps when you're passionate about something, you're gonna help deliver that information. So you help educate these people. But sometimes you spark a passion within somebody else to also be um a part of that change. And so that's really special. We're excited. So um why were high grade gliomas and osteosarcus? The chosen uh cancers?
SPEAKER_01:Yeah, that's a a a really great question. Um and I think um you know uh there are a couple of reasons for one, both are extremely difficult to treat when they return. Okay. Um, you know, so essentially um for a patient um uh with osteosarcoma when it spreads beyond the the initial bone site, um especially to the lungs, um less than 25% of patients are gonna be still alive at five years later. For patients with these high-grade gliomas, these account for over um for our almost half of brain and spinal cord tumors in children. Um and if they don't respond to the upfront treatment, which is surgery and radiation therapy, it's essentially an incurable cancer. Um so we urgently need new treatments for these patients with brain tumors and with bone tumors. Um and uh at least in our preclinical testing, these were two pediatric cancers that had the strongest level of um uh you know preliminary findings, both in mice and in uh the canine trials, um, that they um supported us in moving it forward for these um pediatric cancers.
SPEAKER_04:It's almost like a call to action. Exactly.
SPEAKER_03:That's amazing. Yeah, I'm just fascinated. It is fascinating. Um we did talk about the we talked about the personalized nature of this therapy. So what does that mean for families? Like how does this all sort start for them, the process of that for the families and for the patients?
SPEAKER_01:Yeah, so I I think again, that's one of the really exciting things about this treatment is that it's something that's built specifically for that one patient that's in front of you. So every child that receives this treatment is getting a therapy that's built specifically from their own tumor cells instead of a one size fits all approach. And so the RNA lipid nanoparticle, it we use that to teach their immune system what their own unique cancer looks like and how to respond to that. Um so it really allows us to move from other kinds of um other kinds of immunotherapy that um, you know, while they they could be effective, they have not in pediatrics. And I think in large part because they're only going after one type of protein that may be expressed on the cancer. Here by taking all of the mRNA, we can uh theoretically train the immune system against all of the proteins that are in that patient's cancer. And so you have many more opportunities to train the immune system against different proteins that are dangerous. Um and hopefully if you're targeting many different proteins instead of just one, it offers the um immune system more targets and gives the cancera less uh chances to kind of evade a um immune attack against just one protein because you're kind of hitting it from every different possible angle all at once.
SPEAKER_04:Okay. That's it's a good way to kind of think about, you know, um, you know, trying to picture it for people who are not as specialized.
unknown:Yeah.
SPEAKER_03:But I like yes, though instead of just the one protein, you've got them all kind of over the place, which makes sense of how it's able to target it more specifically. Yeah. Okay.
SPEAKER_04:Um so f funding uh innovative pediatric research is always challenging. We have probably all experienced that to some degree. So who is who's the one or who are supporting um this the development of this trial?
SPEAKER_01:Yeah, so that's one another place that I think we've been very fortunate to have a lot of support because as you mentioned, um the it's it's challenging to you know get funding to um develop new treatments, let alone run early phase clinical trials. So this trial grew um from a challenge grant from the Stop Children's Cancer Foundation of Gainesville. And they basically said that you know, we're gonna give you this money to develop this new treatment for kids with osteosarcoma. Um and your charge is that within X number of years, we want you guys to be in a human clinical trial. And we we met that goal. And their support um helped us to create this therapeutic, and we've now expanded that you know to multiple tumor types. And as a result of receiving that um, you know, initial seed funding from STOP and from other uh um foundations, you know, we've been able to then generate the preliminary data that allowed us to get additional funding um from um other organizations like the V Foundation, the National Pediatric Cancer Foundation, Alex's Lemonade Stand Foundation, and uh even the National Cancer Institute. So getting that like initial seed funding from um local organizations that believe in our vision has really been critical to allowing us to getting that follow-on funding because it allowed us to get to start the process. And once you start the process, you can build momentum and we're hoping that we can keep that momentum going.
SPEAKER_04:Wow, that's incredible.
SPEAKER_03:I think that this is my own thought for a second. I think that when we work here, we see and we we're working every day, we're doing those repetitive things, but then to hear you talk about that and that you're getting this and how large this is getting, you the support that you're getting, the fun you're getting from National Cancer Institutes, things like that is just kind of mind-boggling. Because we we're here every day doing it. And then to hear how far our hospital our little hospital is getting is crazy to think about. It's not a little hospital, but you know what I mean? Like our home is getting so big, it's just in so interesting to think about.
SPEAKER_04:Well, and I think especially for people like us who are raised here and and you know, this has, you know, I think when you're so close to a picture, it's hard to see the full view. And so sometimes when you come in day in, day out, you clock in, you clock out, uh, you don't get to see the biggest part of the picture.
SPEAKER_03:And the impact that we're really hacking and that you uh your team is really making, which is very interesting to hear about.
SPEAKER_01:Yeah, no, I mean, and again, we couldn't do it without um a team that believes in the mission of developing, you know, newer and better treatments for patients. And I think that that's that's why a lot of us we want to be in academics. We want to be where we're moving the the bar. You know, like of course we want to help, you know, the the four-month-old who's coming in with bronchiolitis and you know needs their support. And you know, we have to be a referral center for that. But you know, we we also um you know really have an obligation that if we're doing all this cool stuff in the lab, you know, developing mRNA therapeutics, we need to make sure that we're going to actually bring this forward to patients and find out definitively. Is this gonna work for patients? Yes or no. And um I'm I'm glad that we're at an institution um where we're able to collaborate across uh multiple teams um to deliver this kind of care safely for our patients and get these answers to the questions that people really across the entire country are asking and want to know.
SPEAKER_04:Yeah. And I I'm eager, you know, yes, to see where this heads. Me too. So so looking ahead in all of this, do you expect this trial to expand out to even more cancers?
SPEAKER_01:Yeah, absolutely. So our long-term goal is to add additional trial arms uh using this RNA-lipid nanoparticle platform to create personalized immunotherapies for other um types of relapsed or difficult to peak difficult to treat pediatric cancers. Um, and that includes other types of pediatric brain tumors like medulloblastoma, diffuse intrinsic pontine glioma, and other sarcomas like ewing sarcoma, rhabdomyar sarcoma. Um, so I I anticipate that we're going to open you know more trials you know in the future as we gain additional um experience with these first two kinds of cancers that we've been treating.
SPEAKER_04:Excellent. Wow.
SPEAKER_03:Uh I mean it is. It's a very And I know we're in phase one, so it's it's under I think we have a lot of hope and a lot of encouragement, but we're in phase one. We're trying, we're gonna, you know, go with the trial and follow the trial and and work our way to all those other steps and all those other phases.
SPEAKER_04:It just But I think when you've been, you know, I've been in nursing for a really long time. Right. And I think sometimes, again, you kind of just get blinded by the the day-to-day. And so to see something so big and to know that you know, maybe like the up-and-coming kids that are going, you know, in high school right now, they're gonna go to college and they're gonna grow, they're not in. So I think there's just so much um prospect for what could happen. And I think that's it's it's very exciting.
SPEAKER_03:So I did have one other question that kind of popped up. How long this is how long does it take from you to get the sample from the tumor to making the actual mRNA vaccine for them? How long does that process take?
SPEAKER_01:That's a great question. And that's actually really important for our trials because uh I didn't fully get into kind of how long that takes and what we do while we're making the treatment. Um, but so it takes about six-ish weeks from when we take the tumor out of the patient uh to making the um the lipid nanoparticle. Um and so that gives like there's a gap there of about six weeks where you have a patient who still has cancer and needs to be treated. Um and so for some of our clinical trials, if it's a newly diagnosed patient, what we're able to do is we're able to give them the kind of normal radiation treatment that um we uh we might uh be giving as standard of care. Um and so that gives a good little bridge to get to the patient's personalized lipid nanoparticle. For patients where we're treating them when the cancer has come back or it's recurred, um what we do there is we actually do use an off-the-shelf um, you know, lipid nanoparticle because we think that using this non-specific um uh peptide uh based lipid nanoparticle, while it may not be specific for that patient's cancer, we have shown in uh some of our preclinical testing that it can actually start the process of priming the immune system. We're starting to reprogram um the immune system so that way when we give the personalized treatment, it may respond even better than a a patient that didn't get that those priming doses.
SPEAKER_03:So at least help it it helps bridge that gap, like you were saying, at least for those six weeks where you're kind of waiting.
SPEAKER_01:Exactly. That's the that's the hypothesis um or you know, kind of our our thought process right now. Um I think um, you know, we'll kind of have to you know see how that goes.
SPEAKER_04:Right. Wow. Interesting. Is there um this just kind of stimulated a question for me? Uh is there a length of treatment for the for the um uh mRNA?
SPEAKER_01:That's another great question that we're trying to answer. Okay. Um so at least like right now, you know, we're uh we're thinking like, you know, let's try giving it over a whole year. Okay. Um because we we don't really know like um after we give the lipid nanoparticle, how long is the immunity that you induce against the patient's tumor gonna last? Is it gonna stick around for six months? Is it gonna stick around for one month? Is it gonna stick around for 24 hours? You know, I I'm not entirely sure. And yeah, like to your point earlier, the the other question is like, do you get kind of an amplification of the um immune response with more treatments that you give? If you give three nanoparticles, is that enough to teach the body to permanently recognize uh the cancer? Do you need to give it for a whole year to give it durable immunity or to at least um keep the immune system activated long enough to wipe up any last remaining tumor cells that are there? We're not sure. We know that we can make um you know enough of the lipid nanoparticles to last for a whole year. Um whether they need to get it for that long, or if we can you know kind of cut it off after you know three or five or whatever arbitrary number of doses, um, I think that's an open question as well.
SPEAKER_03:Okay, interesting. So still things to learn obviously there's still things to learn. Okay. Yes. Wow.
SPEAKER_04:Wow, yep. So, Dr. Ligan, thank you so much uh for your work and for kind of taking your time to come and talk with us today to explain all of this. This has been fascinating. Um this trial represents an exciting new chapter, obviously, in pediatric cancer and the care for those kiddos and their families. And we obviously will be watching and cheering for you guys very much as the rest of this unfolds. Um, do you have any parting, any parting words you want to share with us?
SPEAKER_01:Yeah, just again, I'm I'm so thankful for the collaboration, you know, with the the PICU. Uh, you know, we wouldn't, you know, feel as confident as we do about moving this treatment forward if we didn't know that you guys were watching over our patients with a you know very watchful eye and ready to intervene with any um toxicity. And um I think that really only by having a great team that is fully invested can we move new treatments forward like this safely and really answer the important questions that we're all asking. Excellent.
SPEAKER_03:Wow. Well, thanks everyone for joining us in the Gator Picky podcast. And we will see you next time. Thank you, Dr. Ligan. We really appreciate it.
SPEAKER_04:It's been wonderful. Yes.
SPEAKER_01:Thank you guys very much. At least I managed to set up.
