NIH-funded research R01 grant

Nanoparticle immune treatment for SHH‑subtype medulloblastoma

Official title: Nanoformulated small molecule immunotherapy for SHH medulloblastoma

Funded at Univ of North Carolina Chapel Hill · NIH-11251558 · Data updated 2026-11-12

This project tests a tiny‑particle version of an immune drug designed to help children with SHH‑subtype medulloblastoma live longer with fewer lifelong side effects.

More Immunotherapy research and trials

Quick facts

Grant typeR01 grant
Study typeNIH-funded research
Funding institutionUniv of North Carolina Chapel Hill
NIH-funded
Lab location1 site (Chapel Hill, United States)
Project startJanuary 1, 2022
Project endDecember 31, 2026
Funding year2026
Award (this year)$388,750
NIH project number5R01NS125073-05
Project IDNIH-11251558 on NIH RePORTER

Who could benefit from this research

Could help

Children with SHH‑subtype medulloblastoma, particularly those with recurrent disease or who are candidates for experimental immune therapy, would be the most relevant participants.

Less likely to help

Patients with non‑SHH medulloblastoma subtypes, other unrelated cancers, or those unable to access the study site likely would not benefit from this specific project.

What this research studies

The team is creating a nanoparticle formulation of an immune‑stimulating drug (resiquimod) to help it reach brain tumors and activate tumor‑associated immune cells in SHH medulloblastoma. The particles are designed to cross the blood–brain barrier and deliver the drug to TAMs that express TLR7/8. Researchers will test delivery, immune activation, and anti‑tumor effects in laboratory and model systems and explore combining the drug with cancer vaccines. The overall aim is to develop a safer, more effective immune approach to reduce toxic standard therapies for children.

Why it matters

Potential benefit: If successful, this could produce immune‑based treatments that better reach brain tumors and lower the long‑term cognitive and physical harms of current therapies for children.

How similar studies have gone: Related TLR7/8 drugs like resiquimod have been tested in clinical trials for melanoma, bladder cancer, and glioma, but a blood‑brain‑penetrant nanoparticle formulation for medulloblastoma remains novel and largely unproven in humans.

Where this research is happening

  • Univ of North Carolina Chapel Hill
    Chapel Hill, United States
    Active

Researchers

  • Principal investigator: Sokolsky-Papkov, Marina — Univ of North Carolina Chapel Hill
  • Study coordinator: Sokolsky-Papkov, Marina

Related research

Other NIH-funded projects on the same topics.

  • Immunotherapy for high-risk pediatric brain cancer using systems immunology

    A program to understand why a dangerous form of childhood brain cancer resists immunotherapy and to develop new immune-based treatments for kids with Group 3 medulloblastoma.

    NIH research St. Jude Children's Research Hospital · Memphis, United States · NIH-11380776

  • Immune-based treatments for aggressive Group 3 medulloblastoma

    Working to unlock the immune system to create new treatments for children with high-risk Group 3 medulloblastoma.

    NIH research St. Jude Children's Research Hospital · Memphis, United States · NIH-11162311

  • New treatment for a type of brain cancer in children

    This study is testing a new treatment for children with SHH medulloblastoma, a type of brain cancer, using a special drug that helps the immune system fight the cancer while aiming to cause fewer side effects than current therapies.

    NIH research Univ of North Carolina Chapel Hill · Chapel Hill, United States · NIH-11004378

  • Understanding how to improve immunotherapy for aggressive brain tumors in children

    This study is looking at a tough type of brain tumor in kids called high-risk Group 3 medulloblastoma, and it's trying to find out how these tumors hide from the immune system so that new treatments can be created to help the immune system fight them better.

    NIH research St. Jude Children's Research Hospital · Memphis, United States · NIH-10924068

  • Using exosomes to improve immunotherapy for brain tumors in children

    This study is testing a new treatment for children with medulloblastoma, a type of brain cancer, by using tiny particles from immune cells to help the body’s immune system fight the tumor more effectively while aiming to reduce side effects from standard treatments.

    NIH research Univ of North Carolina Chapel Hill · Chapel Hill, United States · NIH-10790660

  • Boosting immune response to fight cancer

    This study is exploring a new way to help your immune system better fight cancer by tweaking certain cells in your body, and it aims to improve treatment for people with advanced cancer.

    NIH research University of Missouri-Columbia · Columbia, United States · NIH-9868156

About this research

  1. This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
  2. Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
  3. For full project details, budget, and progress reports, visit the official NIH RePORTER page.
Last reviewed 2026-11-14 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.