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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.

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PICU Essentials on the App Store (apple.com)

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