Thursday, 20 April 2017

Agents for MGMT Unmethylated, PTEN Mutation, and PDGFRA Amplificayion


My mother got the GBM surgery on Feb 6 this year. She is MGMT unmethylated, PTEN mutation, and PDGFRA amplification.

Does any one know effective agents for treating these disorders or symptoms?

James Zhou


  1. Where is she at in her treatment? Has she finished radiation? Has she started (or will she be starting) monthly maintenance cycles of temozolomide? If so, because of the MGMT unmethylated status, I would suggest levetiracetam (brand name Keppra in North America) as an anti-seizure medication, as there is some evidence that Keppra may also downregulate expression of the MGMT enzyme.

    PTEN deactivating mutations lead to the overexpression of PI3K and downstream kinases such as AKT and mTOR. Rapamycin (an approved mTOR inhibitor) + chloroquine/hydroxychloroquine is an interesting combination that is also in clinical trial in Taiwan.

    There are several approved oncology drugs that inhibit PDGFRA, such as imatinib (Gleevec), but none of these targeted kinase inhibitors are approved for brain tumors and none were designed or developed with the blood-brain barrier in mind.

    What country does your mother live in?

    1. Stephen:
      Thank your for quick response.

      My mother lives in China. She will finish 6 week radiation by the end of this week. The monthly maintenance cycles of TMZ will start from next week.
      We thank you for giving us the above useful information. We will research further and make a decision on agents we will use.

      I live in Vancouver

      Best Regards
      James Zhou