Kidscan and North Western Cancer Research have partnered up to fund research into a new therapeutic approach to address the unmet need within the medulloblastoma sphere.

Medulloblastoma is a brain tumour that develops in the cerebellum at the back of the brain which is an area associated with coordination and movement. Due to the cancer’s ability to rapidly grow, it can spread to other areas of the brain and the spinal cord too.

Although adults can be affected, it is children who are most commonly diagnosed with Medulloblastoma, in fact it’s the most frequent malignant childhood brain cancer and there are four known subtypes. Although more is now known about the genetic and molecular basis of these subtypes, and outcomes have been improved in patients in comparison to 20-30 years ago, recent years have seen little progress and there remains an unmet need within the medulloblastoma treatment landscape.

Current effective treatments involve highly toxic radiotherapy, and chemotherapy regimes that often lead to severe and lifelong complications. Unlike other treatments for brain tumours, medulloblastoma does not have any effective treatments that specifically target the cancer without causing harm to the body’s cells.

Kidscan have partnered with North Western Cancer Research to jointly fund a 3 year PhD grant with the aim of providing more precise therapies that reduce the side effects that typically accompany the current conventional treatments, and improve the quality of life and survival outcomes for patients.

The project aims to develop a novel treatment strategy for medulloblastoma using PROTACs (proteolysis targeting chimaeras) which are designed to target and destroy specific-cancer causing proteins. Scientists have identified a protein called KBTBD4 which is mutated in a large proportion of children with a poorly understood type of medulloblastoma, and although scientists have identified a chemical compound that binds to it, it needs to be modified to ensure that it also removes the mutated protein from the cell.

The project specifically aims to improve patient outcomes for medulloblastoma patients, but has the broader aim of enhancing the PROTAC-based chemical toolbox for precise and targeted therapeutic approaches for other types of cancer too.

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