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Increased apoptotic priming of glioblastoma enables therapeutic targeting by BH3-mimetics

Koessinger, A.; Koessinger, D.; Kinch, K.; Martinez-Escardo, L.; Paul, N.; Elmasry, Y.; Malviya, G.; Cloix, C.; Campbell, K.; Bock, F.; O'Prey, J.; Stevenson, K.; Nixon, C.; Jackson, M.; Ichim, G.; Stewart, W.; Blyth, K.; Ryan, K.; Chalmers, A. J.; Norman, J.; Tait, S.

2021-06-13 cancer biology
10.1101/2021.06.13.448232 bioRxiv
Show abstract

IDH wild-type glioblastoma (GBM) is the most prevalent malignant primary brain tumour in adults. GBM typically has a poor prognosis, mainly due to a lack of effective treatment options leading to tumour persistence or recurrence. Tackling this, we investigated the therapeutic potential of targeting anti-apoptotic BCL-2 proteins in GBM. Levels of anti- apoptotic BCL-xL and MCL-1 were consistently increased in GBM compared with non- malignant cells and tissue. Moreover, we found that relative to their differentiated counterparts, patient-derived GBM stem-like cells also displayed higher expression of anti- apoptotic BCL-2 family members. Surprisingly, high anti-apoptotic BCL-xL and MCL-1 expression correlated with heightened susceptibility of GBM to BCL-2 family protein- targeting BH3-mimetics. This is indicative of increased apoptotic priming. Indeed, GBM displayed an obligate requirement for MCL-1 expression in both tumour development and maintenance. Investigating this apoptotic sensitivity, we found that sequential inhibition of BCL-xL and MCL-1 led to robust anti-tumour responses in vivo, in the absence of overt toxicity. These data demonstrate that BCL-xL and MCL-1 pro-survival function is a fundamental prerequisite for GBM survival that can be therapeutically exploited by BH3- mimetics.

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