Cancer is one of the leading causes of death worldwide. Brain cancer is the 15th most common cancer in the world, and has one of the worst survival rates of all cancer types, with only approximately half of patients surviving for one year post-diagnosis. This poor survival rate highlights that new treatments for brain cancer are urgently required.

Our recent work has identified a new target, a molecule called Brain and Acute Leukaemia, cytoplasmic (BAALC), which is highly expressed in brain cancer (Figure 1). High levels of BAALC in cancer cells predict that the patient will have a poor outcome, as they will survive for shorter periods of time, and their cancer is likely to spread or return.

We have recently demonstrated that BAALC controls how cancer cells divide, move and invade, as well as cell sensitivity to chemotherapeutics. We have developed a drug (called C310) that targets BAALC. This drug kills a range of cancer cells (primarily leukaemia, breast and prostate cancer cells) in the test tube, whilst leaving normal cells untouched. Additionally, we have shown that C310 is not only safe and well-tolerated in laboratory models of leukaemia, but also reduces tumour burden and increases survival times. This specificity will possibly reduce treatment-associated side-effects and toxicity. Furthermore, we have shown that the sensitivity of cells to C310 depends entirely on the amount of BAALC found in the cell. However, the usefulness of C310 in brain cancer remains to be explored. As BAALC is one of the most highly expressed molecules in brain cancer cells, we hypothesise that these cells will be highly sensitive to C310 treatment.

This project has two main aims: (1) determine the sensitivity of a panel of brain cancer cell lines to treatment with C310 in the test-tube (in vitro) and (2) examine the effectiveness of C310 in laboratory models of brain cancer.

The targeting of BAALC offers a new strategy for the treatment of brain cancers with poor prognosis, which will be more cancer-cell specific and may potentially reduce toxicity and side effects in patients.

This project will identify new potential treatment options for advanced cancers, which will be applicable to a range of cancer types.

This project is funded by the supporters of the Mark Hughes Foundation.

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