FUMES – Focused ultrasound-mediated drug delivery high-tech engineering solutions
Brain tumors in children are difficult to treat because of the blood-brain barrier (BBB) that prevents most drugs to achieve effective concentrations for long enough time in the brain. Microbubble-assisted focused ultrasound-mediated BBB opening (FUS-BBBO) is a technology that can temporarily and locally open the brain for drugs to pass. Standardization of preclinical FUS-research, both with regard to pharmacokinetics and BBB opening in animal models is highly needed and addressed in this fundamental research project.
This is a partnership of Demcon curonix, Demcon biovitronix and The Princess Máxima Center for Pediatric Oncology. Children with brain tumors, 120 per year in the Netherlands, often have a bad survival, varying from 0% for diffuse midline glioma to 70% in medulloblastoma. Those who are cured often have serious disabilities. New treatments are desperately needed for these cancers treat more effectively and prevent damage to the developing brain, leading to better survival and quality of life. Better drug delivery over the BBB holds high promise to achieve this.
In this fundamental research project, we will open the BBB in animal models in a more stable and predictable way upon microbubble infusion using a controlled microbubble infusion system developed by Demcon curonix. We will simulate real-life pharmacokinetics in the laboratory to ascertain the concentration and timing of drugs in combination with FUS-BBBO, using a system developed by Demcon biovitronix. Ultimately, we will test the validity of this system in tumor-bearing animals in combination with BBB opening.
Upon successful completion of this project, we have generated a clear rationale and potential pathway to early-stage clinical trial development for safety and preliminary efficacy of drugs repurposed for FUS-BBBO-mediated delivery in brain tumor patients. This will hopefully in the future improve survival rates and quality of life for this patient population.