Molecular Profile Detail

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Molecular Profile Indication/Tumor Type Response Type Relevant Treatment Approaches Therapy Name Approval Status Evidence Type Efficacy Evidence References
PIK3CA M1043V Advanced Solid Tumor sensitive mTORC1 Inhibitor Sirolimus Preclinical Actionable In a preclinical study, Rapamune (sirolimus) inhibited proliferation in cultured cells expressing PIK3CA M1043V (PMID: 17376864). 17376864
PIK3CA M1043V head and neck squamous cell carcinoma sensitive Celecoxib Preclinical - Pdx Actionable In a preclinical study, Celebra (celecoxib) treatment reduced tumor growth rate in a patient-derived xenograft (PDX) model of head and neck squamous cell carcinoma harboring PIK3CA M1043V (PMID: 30683736). 30683736
PIK3CA M1043V head and neck squamous cell carcinoma sensitive Sulindac Preclinical - Pdx Actionable In a preclinical study, Clinoril (sulindac) treatment reduced tumor growth rate in patient-derived xenograft (PDX) models of head and neck squamous cell carcinoma harboring PIK3CA mutations, including PIK3CA M1043V (PMID: 30683736). 30683736
PIK3CA M1043V head and neck squamous cell carcinoma predicted - sensitive PIK3CA inhibitor Alpelisib Preclinical - Pdx & cell culture Actionable In a preclinical study, treatment with Piqray (alpelisib) resulted in inhibition of proliferation and increased apoptosis in a head and neck squamous cell carcinoma (HNSCC) cell line expressing PIK3CA M1043V in culture and led to inhibition of tumor growth and decreased phosphorylated Akt levels in a patient-derived xenograft (PDX) model of HNSCC with PIK3CA M1043V (PMID: 34779417). 34779417
PIK3CA M1043V histiocytosis predicted - sensitive PIK3CA inhibitor Alpelisib Case Reports/Case Series Actionable In a clinical case study, Piqray (alpelisib) treatment resulted in symptom improvement and a complete metabolic response lasting at least 9 months in a patient with Langerhans cell histiocytosis harboring PIK3CA M1043V (PMID: 37874915). 37874915