One patient was diagnosed as MBEN in my hospital and received surgical removal, radiation and chemotherapy. After relapse and many cycles of chemotherapy, tumor still regrew and became larger. Now he wants to participate clinical trials of LDE225.
Does anyone know this medicine?
Dear Tao,
LDE225 is an inhibitor of the SHH signaling pathway which is activated in about 25% of all medulloblastomas. As the tumor has MBEN histology it is highly likely that this tumor has SHH activation and can be targeted with LDE225. However, there are different mutations in medulloblastoma that can activate SHH signaling and only tumors that have mutations in Ptch1 or Smo will likely respond to LDE225. Many young children though have mutations further downstream in the pathway in either SUfu or Gli2 and those children will not respond to LDE225. Therefore, it will be important to find out:
A. Is this a ShH activated MB? Based on the MBEN histology, this is highly likely
B. Which gene is mutated? Ptch1, smo, sufu, or gli2?
Best,
Marcel Kool
thank you very much. We didn't know the gene status of this patient. Because of diagnosis of MBEN, I thought that there maybe the possibility for this patient to have a positive effect to LDE225.
In China, we didn''t do gene expression or IHC for MB specimens routinely now. That's why I want do something for MB patients.
I send email to you already. But it's always a failure to your address. Do you have another email address?
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