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Neurological disorders often demand treatments that go beyond traditional approaches. Our research innovation ‘Immune, Cell & Gene Therapies' unites cutting-edge research in immunology, genetics, and regenerative medicine to develop targeted interventions that address disease at its source.

By working across disciplines, we aim to repair, replace, or enhance the body’s own systems to restore neurological function and improve outcomes for patients of all ages. These approaches not only target the biology of disease but also open the door to restoring independence and reducing the burden of neurological disorders on families and communities.

Breakthroughs and impact

By using the immune system to fight aggressive brain tumours, delivering healthy genes that replace faulty ones, or using stem cells to repair injured brain tissue, our research explores the frontiers of what’s possible. We also work on new diagnostic tools and treatment delivery methods, making it easier to detect disease early, monitor progress, and personalise care for each patient.

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  Liquid biopsies for pediatric brain tumors

  Treatment of pediatric brain tumors is complex and associated with significant morbidity and mortality. Stratification at diagnosis is often based on tumor biopsy and response monitoring during treatment depends heavily on imaging. However, the clinical condition might not always allow biopsy and imaging may be inconclusive. Liquid biopsies, minimally invasive tests analyzing tumor-derived biomarkers in blood and cerebrospinal fluid, have the potential to overcome these challenges.  

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  Models for high grade brain tumours

  Researchers from Utrecht have developed immunocompetent mouse models for two aggressive childhood brain cancers: diffuse midline glioma (DMG) and pediatric high-grade glioma (pHGG). Using high-dimensional flow cytometry and single-cell transcriptomics, they showed that these models closely resemble the immune environment of human tumors. This makes them valuable tools for studying how the immune system interacts with these cancers. The models are now being used to test innovative immunotherapies, including next-generation CAR T cells. These efforts aim to accelerate the development of effective treatments for children with these tumors. 

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  Nasal drops with stem cells

  The study investigates a novel treatment for newborn brain injury using nasal drops with mesenchymal stem cells. Given shortly after birth, these drops aim to support brain repair and lower the risk of cerebral palsy. Infants will receive either stem cells or a placebo. This translational research bridges lab and clinic, with promising potential for broader applications in newborns at risk of developmental delays, aiming to improve brain development and long-term outcomes. 

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  First genetic treatment for SMA

  Zolgensma® is the first genetic treatment that rebuilds the SMN1 gene in the DNA of infants with Spinal Muscular Atrophy (SMA). When administered at an early stage, the treatment can significantly improve clinical outcome. The process to select which patient is eligible for the treatment was coordinated from Utrecht. Furthermore, the SMA center advised the RIVM to add SMA to the Dutch perinatal screening program (hielprik), allowing newborns to be diagnosed early on and to receive timely treatment. From 2022 onwards, SMA is included in the screening program, a major achievement.