Value Proposition
Unmet Need:
Medulloblastoma is a malignant embryonal tumor of the cerebellum and brainstem that accounts for roughly 15–20% of pediatric brain tumors and is the most common solid tumor in children (Boston Children’s Hospital). Standard-of-care—surgical resection followed by craniospinal irradiation and multi-agent chemotherapy—achieves long-term survival in approximately 70% of patients but is associated with substantial neurocognitive, endocrine, and secondary malignancy morbidities as well as limited efficacy against metastatic disease (Johns Hopkins Medicine). Molecularly targeted therapies that selectively inhibit drivers of tumor growth and dissemination while sparing normal developing brain are therefore a high priority to improve outcomes and reduce long-term treatment toxicity.
Technology Description:
Researchers at Johns Hopkins University and the University of Central Florida have identified miR‑211 as a tumor-suppressive microRNA in medulloblastoma that directly targets long-chain acyl-CoA synthetase 4 (ACSL4). Restoration of miR‑211 expression reprograms tumor cell lipid metabolism, decreases proliferation, and limits clonogenicity and tumor-initiating capacity in vitro. In orthotopic and subcutaneous xenograft models, miR‑211 delivery reduces tumor growth and tumorigenicity, supporting its therapeutic potential. In parallel, UCF investigators have developed a nanoparticle delivery platform that improves stability, cellular uptake, and bioavailability of therapeutic miRNAs in pediatric cancer models. Encapsulation of miR‑based therapeutics in these nanoparticles enhances delivery to tumor cells and increases anti-tumor efficacy in preclinical studies, addressing key barriers to systemic nucleic acid therapy such as degradation, poor tissue penetration, and off-target distribution.
Stage of Development:
In vitro and in vivo proof-of-concept completed: functional studies demonstrating miR‑211-mediated suppression of ACSL4, metabolic reprogramming and reduced tumorigenicity have been published; nanoparticle delivery systems enabling enhanced miRNA stability and tumor delivery have been validated in preclinical pediatric cancer models.
Publications:
