Molecular Profile Detail

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Molecular Profile	Indication/Tumor Type	Response Type	Therapy Name	Approval Status	Evidence Type	Efficacy Evidence	References
RB1 inact mut	retinoblastoma	sensitive	Fostamatinib	Preclinical	Actionable	In a preclinical study, retinoblastoma cell lines demonstrated sensitivity to Fostamatinib resulting in cell death (PMID: 22237022).	22237022
RB1 inact mut	retinoblastoma	sensitive	BAY 61-3606	Preclinical	Actionable	In a preclinical study, a retinoblastoma cell line was sensitive to BAY 61-3606 in cell culture and in xenograft models, demonstrating increased apoptosis (PMID: 22237022).	22237022
RB1 inact mut	retinoblastoma	sensitive	Nutlin-3a	Preclinical	Actionable	In a preclinical study, Nutlin-3a induced p53 pathway signaling in MDMX mouse models with retinoblastoma and 69% (70/102) of the eyes harvested from the mouse models demonstrated complete response or stable disease after 18 weeks of therapy (PMID: 21515735).	21515735
RB1 inact mut	glioblastoma	resistant	Palbociclib	Preclinical - Cell line xenograft	Actionable	In a preclinical study, Ibrance (palbociclib) failed to inhibit growth of RB1-deficient glioblastoma cell lines in culture and in intracranial cell line xenograft models (PMID: 20354191).	20354191

Profile Name	RB1 inact mut
Gene Variant Detail	RB1 inact mut (loss of function)
Relevant Treatment Approaches	HDAC Inhibitor