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Molecular Profile Indication/Tumor Type Response Type Relevant Treatment Approaches Therapy Name Approval Status Evidence Type Efficacy Evidence References
PTEN loss prostate cancer sensitive Akt Inhibitor (Pan) Ipatasertib Phase I Actionable In a Phase I trial, a patient with castration resistant prostate cancer harboring a loss of PTEN demonstrated an improved prostate specific antigen when treated with Ipatasertib (GDC-0068) (PMID: 27872130). 27872130
PTEN loss colorectal cancer sensitive PIK3CB inhibitor AZD8186 + Vistusertib Phase I Actionable In a Phase I trial, the combination of AZD8186 and Vistusertib (AZD2014) resulted in a partial response in a patient with PTEN-deficient colorectal cancer (J Clin Oncol 35, 2017 (suppl; abstr 2570)). detail...
PTEN loss glioblastoma sensitive PI3K Inhibitor (Pan) PF-04691502 Preclinical - Cell line xenograft Actionable In a preclinical study, PF-04691502 inhibited Akt phosphorylation, resulted in growth inhibition of PTEN-null glioblastoma cells in culture and in cell line xenograft models (PMID: 21750219). 21750219
PTEN loss renal carcinoma decreased response PI3K Inhibitor (Pan) Gedatolisib Preclinical Actionable In a preclinical study, human renal carcinoma cells with PTEN loss had a decreased response to Gedatolisib (PKI-587) in culture (PMID: 21325073). 21325073
PTEN loss melanoma sensitive DETD-35 Preclinical - Cell line xenograft Actionable In a preclinical study, DETD-35 treatment resulted in reduced tumor size in cell line xenograft models of Zelboraf (vemurafenib)-resistant melanoma harboring PTEN loss (PMID: 27048951). 27048951
PTEN loss breast cancer no benefit Akt Inhibitor (Pan) MK2206 Phase II Actionable In a Phase II trial, MK2206 treatment resulted in no objective response and 1 stable disease in 7 patients with advanced breast cancer harboring PTEN loss (PMID: 31277699; NCT01277757). 31277699
PTEN loss glioblastoma sensitive UMI-77 Preclinical - Cell culture Actionable In a preclinical study, UMI-77 treatment led to enhanced apoptosis, and inhibited Akt phosphorylation, proliferation, and colony and spheroid formation in PTEN-deficient glioblastoma cells, and inhibited neurosphere formation in a PTEN-deficient glioblastoma stem cell line in culture (PMID: 32737157). 32737157
PTEN loss glioblastoma sensitive Temozolomide + UMI-77 Preclinical - Cell culture Actionable In a preclinical study, UMI-77 in combination with Temodar (temozolomide) enhanced cytotoxicity and apoptosis, and synergistically inhibited proliferation of PTEN-deficient glioblastoma cells in culture (PMID: 32737157). 32737157
PTEN loss Advanced Solid Tumor no benefit Everolimus Phase II Actionable In a Phase II trial, Afinitor (everolimus) treatment in patients with advanced solid tumors harboring PTEN loss did not reach its primary endpoint, resulting in only stable disease or progressive disease (PMID: 28330462). 28330462
PTEN loss breast cancer no benefit CYH33 Preclinical - Cell culture Actionable In a preclinical study, PTEN loss was not associated with sensitivity to CYH33 in breast cancer cell lines in culture (PMID: 30003928). 30003928
PTEN loss prostate cancer sensitive Akt Inhibitor (Pan) Capivasertib Preclinical Actionable In a preclinical study, treatment with AZD5363 decreased AKT phosphorylation and downstream signaling and reduced tumor burden in mouse models of PTEN-deficient prostate cancer, including both castration-naive and castration resistant models (PMID: 26910118). 26910118
PTEN loss glioblastoma sensitive A-1210477 Preclinical - Cell culture Actionable In a preclinical study, A-1210477 treatment inhibited Akt phosphorylation and proliferation of PTEN-deficient glioblastoma cells in culture (PMID: 32737157). 32737157
PTEN loss osteosarcoma sensitive Akt Inhibitor (Pan) MK2206 Preclinical - Pdx Actionable In a preclinical study, a patient derived xenograft (PDX) model of osteosarcoma with biallelic loss of PTEN was sensitive to treatment with MK2206, demonstrating reduced tumor growth and increased apoptotic activity (PMID: 30266815). 30266815
PTEN loss breast cancer resistant Alpelisib Preclinical - Cell culture Actionable In a preclinical study, breast cancer cells harboring PTEN loss demonstrated resistance to Alpelisib (BYL719) in culture (PMID: 27604488). 27604488
PTEN loss breast cancer sensitive PIK3CB inhibitor AZD6482 Preclinical Actionable In a preclinical study, AZD6482 inhibited viability of breast cancer cell lines harboring PTEN deficiency in culture (PMID: 23674493). 23674493
PTEN loss lung squamous cell carcinoma no benefit Akt Inhibitor (Pan) Capivasertib Case Reports/Case Series Actionable In a Phase II trial (NLMT), Capivasertib (AZD5363) treatment did not result in a conformed response (0/4) or durable clinical benefit (0/2) in patients with squamous cell lung cancer harboring PTEN loss, thus the cohort was closed due to futility (PMID: 32669708, NCT02664935). 32669708
PTEN loss diffuse large B-cell lymphoma predicted - sensitive Akt Inhibitor (Pan) Idelalisib + MK2206 Preclinical - Cell culture Actionable In a preclinical study, MK2206 partially restored sensitivity to Zydelig (idelalisib) in diffuse large B-cell lymphoma cells harboring PTEN loss with acquired resistance to Zydelig (idelalisib), resulting in increased apoptosis in culture (PMID: 28178345). 28178345
PTEN loss breast cancer sensitive PIK3CB inhibitor GSK2636771 Preclinical Actionable In a preclinical study, GSK2636771 inhibited viability of breast cancer cell lines harboring PTEN deficiency in culture (PMID: 23674493). 23674493
PTEN loss Advanced Solid Tumor sensitive Akt Inhibitor (Pan) Capivasertib Preclinical - Cell culture Actionable In a preclinical study, AZD5363 inhibited growth of various solid tumor cell culture models with loss of Pten (PMID: 22294718). 22294718
PTEN loss acute lymphoblastic leukemia sensitive PI3K Inhibitor (Pan) BGT226 Preclinical - Cell culture Actionable In a preclinical study, BGT226 treatment in PTEN-deficient acute lymphoblastic leukemia cells resulted in inhibition of PI3K/Akt signaling and apoptotic activity in culture (PMID: 23705826). 23705826
PTEN loss prostate cancer sensitive PI3K Inhibitor (Pan) Voxtalisib Preclinical - Cell line xenograft Actionable In a preclinical study, a prostate cancer cell line with PTEN loss was sensitive to XL765 (SAR245409) treatment, demonstrating inhibition of the PI3K pathway and decreased colony formation in culture, and inhibition of tumor growth and reduced tumor vascularization in cell line xenograft models (PMID: 24634413). 24634413
PTEN loss triple-receptor negative breast cancer predicted - sensitive Akt Inhibitor (Pan) Capivasertib + Paclitaxel Phase II Actionable In a Phase II trial (PAKT), addition of Capivasertib (AZD5363) to Taxol (paclitaxel) as first-line therapy significantly improved median progression-free survival (9.3 vs 3.7 months, HR=0.30, p=0.01) and reduced risk (66%, HR=0.34, p=0.04) compared to Taxol (paclitaxel) alone in patients with metastatic triple-negative breast cancer harboring activating mutations in AKT1 (n=1) or PIK3CA (n=17), or inactivating mutations or gene loss in PTEN (n=13) (PMID: 31841354; NCT02423603). 31841354
PTEN loss breast cancer resistant Torkinib Preclinical - Cell culture Actionable In a preclinical study, breast cancer cell lines with PTEN loss demonstrated resistance to Torkinib (PP242) in culture (PMID: 21358673). 21358673
PTEN loss triple-receptor negative breast cancer sensitive PIK3CB inhibitor AZD6482 + Veliparib Preclinical - Cell culture Actionable In a preclinical study, the combination of Veliparib (ABT-888) and AZD6482 inhibited growth of triple-negative breast cancer cell lines with PTEN loss in culture (Mol Cancer Res Feb 2016 14; B12). detail...
PTEN loss prostate cancer sensitive PIK3CB inhibitor AZD6482 Preclinical Actionable In a preclinical study, AZD6482 inhibited viability of prostate cancer cell lines harboring PTEN deficiency in culture (PMID: 23674493). 23674493
PTEN loss breast cancer sensitive Akt Inhibitor (Pan) MK2206 + Palbociclib Preclinical - Cell culture Actionable In a preclinical study, the combination of Ibrance (palbociclib) and MK2206 inhibited growth of PTEN-deficient breast cancer cells in culture (PMID: 31594766). 31594766
PTEN loss glioblastoma sensitive PI3K Inhibitor (Pan) PKI-402 Preclinical - Cell line xenograft Actionable In a preclinical study, PKI-402 inhibited tumor growth in PTEN-negative glioblastoma multiforme cell line xenograft models (PMID: 20371716). 20371716
PTEN loss breast cancer sensitive OSI-027 Preclinical - Cell line xenograft Actionable In a preclinical study, OSI-027 induced tumor regression in PTEN-null breast cancer cell line xenograft models (PMID: 21673091). 21673091
PTEN loss triple-receptor negative breast cancer sensitive PIK3CB inhibitor AZD6482 + Talazoparib Preclinical Actionable In a preclinical study, the combination of Talazoparib (BMN-673) and AZD6482 inhibited growth of triple-negative breast cancer cell lines with PTEN loss in culture (Mol Cancer Res, Feb 2016 14; B12). detail...
PTEN loss prostate cancer no benefit CX-5461 Preclinical Actionable In a preclinical study, CX-5461 treatment did not result in histological change in PTEN deficient transgenic animal model of prostate cancer (PMID: 27486174). 27486174
PTEN loss breast cancer resistant Everolimus Preclinical - Cell culture Actionable In a preclinical study, 10/12 breast cancer cell lines with PTEN loss demonstrated resistance to Afinitor (everolimus) in culture (PMID: 21358673). 21358673
PTEN loss breast cancer resistant Fulvestrant + Ribociclib Preclinical - Cell culture Actionable In a preclinical study, PTEN-deficient breast cancer cells demonstrated resistance to the combination of Kisqali (ribociclib) and Faslodex (fulvestrant) in culture (PMID: 31594766). 31594766
PTEN loss prostate cancer sensitive AT13148 Preclinical - Cell line xenograft Actionable In a preclinical study, AT13148 inhibited tumor growth in a PTEN-deficient human prostate cancer cell line xenograft model (PMID: 22781553). 22781553
PTEN loss prostate cancer sensitive PIK3CB inhibitor AZD8186 Preclinical Actionable In a preclinical study, AZD8186 inhibited tumor growth of PTEN deficient prostate xenografts models (PMID: 25514658). 25514658
PTEN loss Advanced Solid Tumor no benefit PIK3CB inhibitor SAR260301 Phase I Actionable In a Phase I trial, SAR260301 demonstrated acceptable safety but undesirable pharmacokinetics, and resulted in no response in patients with advanced solid tumors harboring PTEN loss (J Clin Oncol 33, 2015 (suppl; abstr 2564)). detail...
PTEN loss lung squamous cell carcinoma predicted - sensitive PIK3CB inhibitor AZD8186 Phase I Actionable In a Phase I trial, AZD8186 demonstrated preliminary efficacy in patients with tumor types with prevalent PTEN-deficiency, including squamous non-small cell lung cancer (AACR; Cancer Res 2015;75(15 Suppl):Abstract nr CT329). detail...
PTEN loss ovarian mucinous neoplasm no benefit KX2-391 + Oxaliplatin Preclinical Actionable In a preclinical study, KX2-391 and Eloxatin (oxaliplatin) combination treatment did not show improved tumor suppression in xenograft models of ovarian mucinous carcinoma harboring PTEN loss when compared to single agent treatment (PMID: 24100628). 24100628
PTEN loss uterine corpus sarcoma sensitive AT13148 Preclinical - Cell line xenograft Actionable In a preclinical study, AT13148 inhibited tumor growth in a PTEN-deficient human uterine sarcoma cell line xenograft model (PMID: 22781553). 22781553
PTEN loss lung non-small cell carcinoma sensitive PI3K Inhibitor (Pan) Pictilisib Preclinical - Cell line xenograft Actionable In preclinical studies, the PI3K inhibitor GDC-0941 demonstrated efficacy against NSCLC tumor cell lines in both culture and xenograft models harboring alterations in the PI3K pathway (PMID: 23136191). 23136191
PTEN loss prostate cancer sensitive Acalisib Preclinical - Cell line xenograft Actionable In a preclinical study, Acalisib (GS-9820) resulted in tumor growth inhibition in xenograft models of prostate cancer with PTEN deficiency (Mol Cancer Ther 2009;8(12 Suppl):B136). detail...
PTEN loss sarcoma sensitive YU238259 Preclinical Actionable In a preclinical study, YU238259 demonstrated increased cytotoxicity in PTEN-deficient sarcoma cell lines in culture (PMID: 26116172). 26116172
PTEN loss osteosarcoma sensitive Sirolimus Preclinical - Pdx Actionable In a preclinical study, a patient derived xenograft (PDX) model of osteosarcoma with biallelic PTEN loss was sensitive to treatment with Rapamune (sirolimus), demonstrating reduced tumor growth and increased apoptotic activity (PMID: 30266815). 30266815
PTEN loss transitional cell carcinoma resistant Everolimus Phase II Actionable In a Phase II trial, 57% (8/14) of transitional cell carcinoma patients that demonstrated uncontrolled disease after treatment with Afinitor (everolimus) harbored PTEN loss compared to 0% of patients with controlled disease demonstrating no evidence of PTEN loss (PMID: 22473592). 22473592
PTEN loss breast adenocarcinoma sensitive Akt Inhibitor (Pan) Gemcitabine + LY2780301 Phase Ib/II Actionable In a Phase Ib trial, a breast adenocarcinoma patient harboring PTEN loss demonstrated a partial response when treated with a combination of LY2780301 and Gemzar (gemcitabine) (PMID: 28750271). 28750271
PTEN loss glioblastoma resistant A66 Preclinical - Cell line xenograft Actionable In a preclinical study, A66 did not inhibit tumor growth in a PTEN-null cell line xenograft model of glioblastoma (PMID: 21668414). 21668414
PTEN loss renal cell carcinoma predicted - sensitive Everolimus Clinical Study - Cohort Actionable In a retrospective analysis, loss of PTEN expression was associated with improved progression-free survival compared to PTEN positive (10.5 vs 5.3 months) in renal cell carcinoma patients treated with Afinitor (everolimus) (PMID: 30327302). 30327302
PTEN loss ovarian cancer sensitive OSI-027 Preclinical - Cell line xenograft Actionable In a preclinical study, OSI-027 inhibited tumor growth in PTEN-null ovarian cancer cell line xenograft models (PMID: 21673091). 21673091
PTEN loss breast cancer sensitive Akt Inhibitor (Pan) MK2206 + Ribociclib Preclinical - Cell culture Actionable In a preclinical study, the combination treatment of MK2206 and Kisqali (ribociclib) induced cell cycle arrest and inhibited cell proliferation of PTEN-deficient breast cancer cells in culture and led to tumor regression in cell-line xenograft models (PMID: 31594766). 31594766
PTEN loss breast cancer sensitive Akt Inhibitor (Pan) Fulvestrant + MK2206 + Ribociclib Preclinical - Cell culture Actionable In a preclinical study, the combination of Ibrance (palbociclib), Faslodex (fulvestrant), and MK2206 inhibited growth of PTEN-deficient breast cancer cells in culture (PMID: 31594766). 31594766
PTEN loss breast cancer sensitive Akt Inhibitor (Pan) Ipatasertib + Palbociclib Preclinical - Cell culture Actionable In a preclinical study, the combination of Ibrance (palbociclib) and Ipatasertib (GDC-0068) inhibited growth of PTEN-deficient breast cancer cells in culture (PMID: 31594766). 31594766
PTEN loss prostate cancer sensitive PI3K Inhibitor (Pan) SF1126 Preclinical - Cell line xenograft Actionable In a preclinical study, a glioblastoma cell line harboring PTEN loss (PMID: 24634413) was sensitive to SF1126, demonstrating a 76% decrease in tumor growth in cell line xenograft models (PMID: 18172313). 18172313 24634413
PTEN loss head and neck squamous cell carcinoma predicted - sensitive PI3K Inhibitor (Pan) Rigosertib Sodium Case Reports/Case Series Actionable In a Phase I trial, Rigosertib (ON 01910.Na) treatment resulted in a partial response with 53% decrease of liver metastasis in a patients with head and neck squamous cell carcinoma harboring PTEN loss (Cancer Res April 15 2013 (73) (8 Supplement) LB-198). detail...
PTEN loss melanoma sensitive DETD-35 + Vemurafenib Preclinical - Cell line xenograft Actionable In a preclinical study, DETD-35 and Zelboraf (vemurafenib) combination treatment resulted in further reduction of tumor size in cell line xenograft models of Zelboraf (vemurafenib)-resistant melanoma harboring PTEN loss when compared to single agent treatment (PMID: 27048951). 27048951
PTEN loss prostate cancer sensitive PI3K Inhibitor (Pan) Panulisib Preclinical - Cell line xenograft Actionable In a preclinical study, Panulisib (P7170) inhibited tumor growth in PTEN null prostate cancer cell xenograft models (PMID: 25700704). 25700704
PTEN loss glioblastoma sensitive AZD5991 Preclinical - Cell culture Actionable In a preclinical study, AZD5991treatment inhibited proliferation of PTEN-deficient glioblastoma cells in culture (PMID: 32737157). 32737157
PTEN loss glioblastoma no benefit BLZ945 Preclinical Actionable In a preclinical study, BLZ945 resulted in limited benefit in transgenic mouse models of glioblastoma harboring a loss of PTEN (PMID: 27199435). 27199435
PTEN loss glioblastoma sensitive S63845 Preclinical - Cell culture Actionable In a preclinical study, S63845 treatment reduced Akt phosphorylation and inhibited proliferation of PTEN-deficient glioblastoma cells in culture (PMID: 32737157). 32737157
PTEN loss prostate cancer sensitive Akt Inhibitor (Pan) Abiraterone + Ipatasertib Phase II Actionable In a Phase II trial, the combination of Ipatasertib (GDC-0068) and Zytiga (abiraterone) resulted in a better progression-free survival in metastatic castration resistant prostate cancer patients with PTEN loss when compared to placebo combined with Zytiga (PMID: 30037818; NCT01485861). 30037818 detail...
PTEN loss brain glioma sensitive PI3K Inhibitor (Pan) Gedatolisib Preclinical Actionable In a preclinical study, Gedatolisib (PKI-587) inhibited growth of human glioma cells with PTEN loss in culture (PMID: 21325073). 21325073
PTEN loss prostate carcinoma decreased response PIK3CB inhibitor SAR260301 Preclinical - Cell line xenograft Actionable In a precliinical study, PTEN deficient prostate carcinoma cells demonstrated reduced response to SAR260301 in cell culture and in cell line xenograft models (PMID: 27196754). 27196754
PTEN loss prostate cancer predicted - sensitive CX-5461 + CX-6258 Preclinical Actionable In a preclinical study, CX-6258 and CX-5461 combination treatment resulted in decreased tumor burden in PTEN deficient transgenic animal model of prostate cancer (PMID: 27486174). 27486174
PTEN loss renal carcinoma sensitive Akt Inhibitor (Pan) Capivasertib Preclinical - Pdx Actionable In a preclinical study, AZD5363 inhibited growth of renal cancer Pdx models with Pten loss (PMID: 22294718). 22294718
PTEN loss breast cancer decreased response Palbociclib Preclinical - Cell culture Actionable In a preclinical study, two PTEN-deficient breast cancer cell lines demonstrated a decreased response to treatment with Ibrance (palbociclib) compared to control cells in culture (PMID: 31594766). 31594766
PTEN loss glioblastoma predicted - sensitive Birinapant Preclinical - Cell culture Actionable In a preclinical study, TL32711 (birinapant) treatment inhibited proliferation of PTEN-deficient glioblastoma cells in culture (PMID: 32737157). 32737157
PTEN loss diffuse large B-cell lymphoma predicted - sensitive GSK2334470 + Idelalisib Preclinical - Cell culture Actionable In a preclinical study, GSK2334470 partially restored sensitivity to Zydelig (idelalisib) in diffuse large B-cell lymphoma cells harboring PTEN loss that acquired resistance to Zydelig (idelalisib), resulting in increased apoptosis in culture (PMID: 28178345). 28178345
PTEN loss breast cancer predicted - resistant Ribociclib Preclinical - Cell culture Actionable In a preclinical study, treatment with Kisqali (ribociclib) resulted in a decreased response in two PTEN-deficient breast cancer cell lines in culture compared to control cells and resistance in a cell-line xenograft model (PMID: 31594766). 31594766
PTEN loss prostate cancer sensitive PIK3CB inhibitor GSK2636771 Preclinical Actionable In a preclinical study, GSK2636771 inhibited viability of prostate cancer cell lines harboring PTEN deficiency in culture (PMID: 23674493). 23674493
PTEN loss glioblastoma sensitive PI3K Inhibitor (Pan) LY3023414 Preclinical - Cell line xenograft Actionable In a preclinical study, LY3023414 inhibited Pi3k/mTor signaling and proliferation in PTEN deficient glioblastoma cells in culture, and resulted in tumor growth inhibition in cell line xenograft models (PMID: 27439478). 27439478
PTEN loss triple-receptor negative breast cancer predicted - sensitive PIK3CB inhibitor AZD8186 Phase I Actionable In a Phase I trial, AZD8186 demonstrated preliminary efficacy in patients with tumor types with prevalent PTEN-deficiency, including triple negative breast cancer (AACR; Cancer Res 2015;75(15 Suppl):Abstract nr CT329). detail...
PTEN loss triple-receptor negative breast cancer sensitive PIK3CB inhibitor AZD6482 + Rucaparib Preclinical - Cell culture Actionable In a preclinical study, the combination of Rubraca (rucaparib) and AZD6482 inhibited growth of triple-negative breast cancer cell lines with PTEN loss in culture (Mol Cancer Res Feb 2016 14; B12). detail...
PTEN loss colon cancer sensitive KU-55933 Preclinical Actionable In a preclinical study, KU-55933 induced cell cycle arrest and caspase activation in PTEN deficient human colon cancer cells in culture (PMID: 25870146). 25870146
PTEN loss glioblastoma predicted - sensitive GDC-0152 Preclinical - Cell culture Actionable In a preclinical study, GDC-0152 treatment inhibited proliferation of PTEN-deficient glioblastoma cells in culture (PMID: 32737157). 32737157
PTEN loss prostate cancer sensitive KU-60019 Preclinical Actionable In a preclinical study, KU-60019 blocked tumor growth in PTEN-deficient human prostate cancer xenograft models (PMID: 25870146). 25870146
PTEN loss glioblastoma sensitive AZD5991 + Temozolomide Preclinical - Cell culture Actionable In a preclinical study, AZD5991 in combination with Temodar (temozolomide) synergistically inhibited proliferation of PTEN-deficient glioblastoma cells in culture (PMID: 32737157). 32737157
PTEN loss head and neck squamous cell carcinoma predicted - sensitive PI3K Inhibitor (Pan) AZD8055 Preclinical - Cell culture Actionable In a preclinical study, treatment with AZD8055 resulted in antiproliferative activity in head and neck squamous cell carcinoma cells harboring PTEN loss in culture (PMID: 28446642). 28446642
PTEN loss triple-receptor negative breast cancer sensitive PIK3CB inhibitor AZD6482 + Olaparib Preclinical - Cell culture Actionable In a preclinical study, the combination of Lynparza (olaparib) and AZD6482 inhibited growth of triple-negative breast cancer cell lines with PTEN loss in culture (Mol Cancer Res Feb 2016 14; B12). detail...
PTEN loss triple-receptor negative breast cancer sensitive Akt Inhibitor (Pan) M2698 Preclinical - Cell line xenograft Actionable In a preclinical study, a triple-receptor negative breast cancer xenograft model harboring PTEN loss was sensitive to M2698 (MSC2363318A), demonstrating inhibition of tumor growth and tumor regression (PMID: 27186432). 27186432
PTEN loss breast adenocarcinoma sensitive PI3K Inhibitor (Pan) Voxtalisib Preclinical - Cell culture Actionable In a preclinical study, a breast adenocarcinoma cell line harboring PTEN loss was sensitive to XL765 (SAR245409), demonstrating inhibition of cell proliferation in culture (PMID: 24634413). 24634413
PTEN loss melanoma sensitive PIK3CB inhibitor GSK2636771 Preclinical Actionable In a preclinical study, human melanoma cells with PTEN loss were sensitive to GSK2636771, resulting in decreased activation of Akt and some inhibition of tumor growth (PMID: 26645196). 26645196
PTEN loss prostate cancer no benefit Akt Inhibitor (Pan) MK2206 Phase I Actionable In a Phase I clinical trial, 8 patients with metastatic, castration-resistant prostate cancer harboring a loss of PTEN did not respond to MK-2206 therapy, but prolonged stable disease was observed in 2 patients (PMID: 26187616). 26187616
PTEN loss lung squamous cell carcinoma no benefit PI3K Inhibitor (Pan) Buparlisib Preclinical - Pdx & cell culture Actionable In a preclinical study, lung squamous cell carcinoma patient-derived xenograft (PDX) models harboring PTEN loss did not respond to Buparlisib (BKM120) treatment (PMID: 30093452). 30093452
PTEN loss ovarian mucinous neoplasm decreased response KX2-391 Preclinical Actionable In a preclincal study, mucinous ovarian carcinoma cell lines harboring PTEN loss were less sensitive to KX2-391 induced growth inhibition in culture and tumor suppression in xenograft models (PMID: 24100628). 24100628
PTEN loss prostate cancer predicted - sensitive CX-6258 Preclinical Actionable In a preclinical study, CX-6258 treatment resulted in decreased carcinoma in situ in PTEN deficient transgenic animal model of prostate cancer (PMID: 27486174). 27486174
PTEN loss triple-receptor negative breast cancer sensitive PIK3CB inhibitor AZD6482 + Niraparib Preclinical - Cell culture Actionable In a preclinical study, the combination of Zejula (niraparib) and AZD6482 inhibited growth of triple-negative breast cancer cell lines with PTEN loss in culture (Mol Cancer Res Feb 2016 14; B12). detail...
PTEN loss Advanced Solid Tumor sensitive Akt1 Inhibitor Akt2 Inhibitor BAY1125976 Preclinical Actionable In a preclinical study, BAY1125976 demonstrated anti-tumor efficacy in multiple xenograft tumor models of different cancers with PIK3CA mutations or PTEN deletions and displayed synergy with other anti-cancer therapies (Cancer Res 2013;73(8 Suppl):Abstract nr 2050). detail...
PTEN loss breast cancer sensitive Akt Inhibitor (Pan) Ipatasertib + Ribociclib Preclinical - Cell culture Actionable In a preclinical study, the combination of Ipatasertib (GDC-0068) and Kisqali (ribociclib) induced cell cycle arrest and inhibited growth of PTEN-deficient breast cancer cells in culture (PMID: 31594766). 31594766
PTEN loss glioblastoma sensitive PI3K Inhibitor (Pan) GDC-0084 Preclinical - Cell line xenograft Actionable In a preclinical study, two PTEN-deficient glioblastoma cell line xenograft models were sensitive to GDC-0084 treatment, resulting in decreased tumor volumes by 70% and 40%, and inhibition of PI3K pathway signaling (PMID: 27638506). 27638506
PTEN loss glioblastoma sensitive Akt2 Inhibitor CCT128930 Preclinical - Cell line xenograft Actionable In preclinical studies, CCT128930 prevented tumor growth in a PTEN-null human glioblastoma cell line xenograft model (PMID: 21191045). 21191045
PTEN loss prostate cancer predicted - sensitive AR-mTOR-26 Preclinical - Cell line xenograft Actionable In a preclinical study, AR-mTOR-26 treatment resulted in antitumor activity in prostate cancer xenograft models with PTEN loss (Cancer Res 2010;70(8 Suppl):Abstract nr 4484). detail...
PTEN loss head and neck squamous cell carcinoma predicted - sensitive PI3K Inhibitor (Pan) Dactolisib Preclinical - Cell culture Actionable In a preclinical study, BEZ235 resulted in antiproliferative activity in head and neck squamous cell carcinoma cells harboring PTEN loss in culture (PMID: 28446642). 28446642
PTEN loss prostate cancer sensitive OP449 Preclinical Actionable In a preclinical study, prostate cancer mouse models deficient for Pten demonstrated inhibition of the PI3K/Akt signaling pathway and a decrease in both tumor size and cell proliferation when treated with OP449 (PMID: 26563471). 26563471
Clinical Trial Phase Therapies Title Recruitment Status Covered Countries Other Countries
NCT02987959 Phase II Sapanisertib Study of TAK-228 (MLN0128) in Soft Tissue Sarcomas Active, not recruiting
NCT01870726 Phase Ib/II Buparlisib + Capmatinib Safety and Efficacy of INC280 and Buparlisib (BKM120) in Patients With Recurrent Gliobastoma Terminated
NCT02761694 Phase I ARQ 751 + Paclitaxel ARQ 751 ARQ 751 + Fulvestrant ARQ 751 as a Single Agent or in Combination With Other Anti-Cancer Agents, in Solid Tumors With PIK3CA / AKT / PTEN Mutations Terminated
NCT01884285 Phase I AZD8186 Abiraterone + AZD8186 AZD8186 + Vistusertib AZD8186 First Time In Patient Ascending Dose Study Completed
NCT02215096 Phase I Enzalutamide GSK2636771 Dose-finding Study of GSK2636771 When Administered in Combination With Enzalutamide in Male Subjects With Metastatic Castration-Resistant Prostate Cancer Completed
NCT04632992 Phase II Ipatasertib Atezolizumab + Capecitabine Entrectinib Atezolizumab + Docetaxel Ado-trastuzumab emtansine + Atezolizumab Capecitabine + Pertuzumab/trastuzumab/hyaluronidase-zzxf Paclitaxel + Pertuzumab/trastuzumab/hyaluronidase-zzxf Alectinib GDC-0077 Pertuzumab/trastuzumab/hyaluronidase-zzxf Docetaxel + Pertuzumab/trastuzumab/hyaluronidase-zzxf Atezolizumab + Paclitaxel Ado-trastuzumab emtansine + Tucatinib A Study Evaluating Targeted Therapies in Participants Who Have Advanced Solid Tumors With Genomic Alterations or Protein Expression Patterns Predictive of Response (MyTACTIC) Recruiting
NCT02465060 Phase II Erdafitinib Copanlisib Trametinib Crizotinib Sunitinib Sapanisertib Nivolumab AZD4547 Dasatinib Pertuzumab + Trastuzumab Dabrafenib + Trametinib Binimetinib Adavosertib Osimertinib Palbociclib Afatinib Capivasertib Defactinib GSK2636771 Vismodegib Ado-trastuzumab emtansine Larotrectinib Taselisib Targeted Therapy Directed by Genetic Testing in Treating Patients With Advanced Refractory Solid Tumors, Lymphomas, or Multiple Myeloma (The MATCH Screening Trial) Recruiting
NCT04388852 Phase I DS-3201b + Ipilimumab DS3201 and Ipilimumab for the Treatment of Metastatic Prostate, Urothelial and Renal Cell Cancers Recruiting
NCT04586270 Phase I TAS0612 A Study of TAS0612 in Participants With Advanced or Metastatic Solid Tumor Cancer Recruiting
NCT03834740 Phase I Everolimus + Ribociclib A Phase 0 /II Study of Ribociclib (LEE011) in Combination With Everolimus in Preoperative Recurrent High-Grade Glioma Patients Scheduled for Resection Recruiting
NCT02576444 Phase II Adavosertib + Olaparib Olaparib Capivasertib + Olaparib AZD6738 + Olaparib OLAParib COmbinations (OLAPCO) Active, not recruiting
NCT02920450 Phase Ib/II Carboplatin + Gedatolisib + Paclitaxel Study of Paclitaxel, Carboplatin, and PF-05212384 in Advanced or Metastatic NSCLC (UF-STO-LUNG-002) Terminated
NCT04592237 Phase II Niraparib Cabazitaxel + Carboplatin + JNJ-63723283 JNJ-63723283 + Niraparib Cabazitaxel, Carboplatin, and Cetrelimab Followed by Niraparib With or Without Cetrelimab for the Treatment of Aggressive Variant Metastatic Prostate Cancer Recruiting
NCT03624543 Phase II CFI-400945 CFI-400945 in Patients With Advanced/Metastatic Breast Cancer Recruiting
NCT02961283 Phase I ASN003 Study of ASN003 in Subjects With Advanced Solid Tumors Terminated
NCT00770263 Phase I Erlotinib + Temsirolimus Erlotinib and Temsirolimus for Solid Tumors Completed
NCT01458067 Phase I GSK2636771 A Phase I/IIa, First Time in Human, Study of GSK2636771 in Subjects With Advanced Solid Tumors With Phosphatase and Tensin Homolog (PTEN) Deficiency Completed
NCT02549989 Phase II LY3023414 Study of LY3023414 for the Treatment of Recurrent or Persistent Endometrial Cancer Active, not recruiting
NCT03065062 Phase I Gedatolisib + Palbociclib Study of the CDK4/6 Inhibitor Palbociclib (PD-0332991) in Combination With the PI3K/mTOR Inhibitor Gedatolisib (PF-05212384) for Patients With Advanced Squamous Cell Lung, Pancreatic, Head & Neck and Other Solid Tumors Recruiting
NCT03131908 Phase Ib/II GSK2636771 + Pembrolizumab Study of the Selective PI3K-Beta Inhibitor GSK2636771 in Combination With Pembrolizumab in Patients With Metastatic Melanoma and PTEN Loss Active, not recruiting
NCT04251533 Phase III Alpelisib + Nab-paclitaxel Nab-paclitaxel Study Assessing the Efficacy and Safety of Alpelisib + Nab-paclitaxel in Subjects With Advanced TNBC Who Carry Either a PIK3CA Mutation or Have PTEN Loss Without PIK3CA Mutation (EPIK-B3) Recruiting
NCT03317119 Phase I Trametinib + Trifluridine-tipiracil hydrochloride Trametinib and Trifluridine and Tipiracil Hydrochloride in Treating Patients With Colon or Rectal Cancer That is Advanced, Metastatic, or Cannot Be Removed by Surgery Active, not recruiting