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Gene Name Source Synonyms Protein Domains Gene Description Gene Role
Therapy Name Drugs Efficacy Evidence Clinical Trials
131I-MIBG 131I-MIBG 2 4
Abaloparatide Abaloparatide 0 0
Abaloparatide + Bevacizumab Abaloparatide Bevacizumab 0 1
Ado-trastuzumab emtansine Ado-trastuzumab emtansine 50 24
Afatinib Afatinib 157 25
Alclometasone Alclometasone 0 1
Apalutamide Apalutamide 3 13
Arsenic trioxide Arsenic trioxide 1 4
Atezolizumab Atezolizumab 13 100
Atezolizumab + Bevacizumab + Rucaparib Atezolizumab Bevacizumab Rucaparib 0 1
Atezolizumab + Hu5F9-G4 Atezolizumab Hu5F9-G4 3 2
Atezolizumab + Ramucirumab Atezolizumab Ramucirumab 0 1
Avelumab Avelumab 10 52
Axitinib Axitinib 24 24
Azacitidine + GSK3326595 Azacitidine GSK3326595 0 1
Belinostat Belinostat 4 10
Bendamustine Bendamustine 0 9
Bevacizumab Bevacizumab 7 120
Bicalutamide Bicalutamide 1 19
Blinatumomab Blinatumomab 2 8
Blinatumomab + Inotuzumab ozogamicin Blinatumomab Inotuzumab ozogamicin 0 2
Bortezomib + Ibrutinib Bortezomib Ibrutinib 0 1
Bosutinib Bosutinib 5 10
Bosutinib + Ruxolitinib Bosutinib Ruxolitinib 0 2
Busulfan Busulfan 0 13
Busulfan + Fludarabine + Romidepsin Busulfan Fludarabine Romidepsin 0 1
Cabozantinib Cabozantinib 45 56
Celecoxib + Interferon alpha-2b + Rintatolimod Celecoxib Interferon alpha-2b Rintatolimod 0 1
Ceritinib Ceritinib 115 18
Cetuximab Cetuximab 65 43
Cobimetinib Cobimetinib 18 6
CPX-351 CPX-351 1 18
Crizotinib Crizotinib 161 33
Cyclophosphamide + Doxorubicin + Paclitaxel + Pembrolizumab Cyclophosphamide Doxorubicin Paclitaxel Pembrolizumab 0 3
Cyclophosphamide + Nivolumab Cyclophosphamide Nivolumab 0 1
Dabrafenib Dabrafenib 59 14
Dacarbazine Dacarbazine 0 9
Daratumumab Daratumumab 1 16
Dasatinib Dasatinib 75 46
Daunorubicin Daunorubicin 1 14
Daunoxome Daunoxome 0 1
Decitabine + Quizartinib Decitabine Quizartinib 0 0
Dexamethasone + Mercaptopurine + Methotrexate + Vincristine sulfate liposome Dexamethasone Mercaptopurine Methotrexate Vincristine sulfate liposome 0 1
Dinutuximab Dinutuximab 1 2
Divalproex sodium Divalproex sodium 0 0
Doxorubicin + Gemcitabine + Pembrolizumab + Vinorelbine Doxorubicin Gemcitabine Pembrolizumab Vinorelbine 0 1
Durvalumab + Monalizumab Durvalumab Monalizumab 0 2
Epirubicin Epirubicin 0 2
Erlotinib Erlotinib 22 50
Etoposide + Gemtuzumab ozogamicin + Mitoxantrone Etoposide Gemtuzumab ozogamicin Mitoxantrone 0 1
Everolimus Everolimus 46 60
Everolimus + Itacitinib Everolimus Itacitinib 0 1
Fingolimod Fingolimod 1 1
GDC-0084 + Trastuzumab GDC-0084 Trastuzumab 0 1
Gefitinib Gefitinib 22 5
Gilteritinib + Venetoclax Gilteritinib Venetoclax 0 1
Hydroxychloroquine + Trametinib Hydroxychloroquine Trametinib 1 3
Ibritumomab tiuxetan Ibritumomab tiuxetan 1 1
Idarubicin Idarubicin 0 7
Idelalisib Idelalisib 9 9
Imatinib Imatinib 164 19
Imatinib + Prednisone + Vincristine Sulfate Imatinib Prednisone Vincristine Sulfate 0 1
Ipilimumab Ipilimumab 5 86
Lapatinib Lapatinib 171 13
Lenalidomide Lenalidomide 3 29
Letrozole Letrozole 1 38
lutetium Lu 177 dotatate lutetium Lu 177 dotatate 0 7
Melphalan Melphalan 0 30
Mogamulizumab Mogamulizumab 3 4
Moxetumomab pasudotox-tdfk Moxetumomab pasudotox-tdfk 1 0
Nilotinib Nilotinib 33 23
Nivolumab Nivolumab 31 282
Obinutuzumab + Vemurafenib Obinutuzumab Vemurafenib 0 1
Ofatumumab Ofatumumab 1 4
Olaratumab Olaratumab 0 5
Omacetaxine mepesuccinate Omacetaxine mepesuccinate 1 0
Orlistat Orlistat 1 0
Osimertinib Osimertinib 100 36
Paclitaxel + Ramucirumab Paclitaxel Ramucirumab 2 7
Palbociclib Palbociclib 44 36
Panitumumab Panitumumab 30 18
Panobinostat Panobinostat 2 11
Pazopanib Pazopanib 30 41
Pegylated liposomal-doxorubicin Pegylated liposomal-doxorubicin 0 11
Pembrolizumab Pembrolizumab 43 398
Pertuzumab Pertuzumab 8 9
Ponatinib + Prednisone + Vincristine Sulfate Ponatinib Prednisone Vincristine Sulfate 0 1
Ramucirumab Ramucirumab 10 14
Regorafenib + Trifluridine-tipiracil hydrochloride Regorafenib Trifluridine-tipiracil hydrochloride 0 1
Romidepsin + Tenalisib Romidepsin Tenalisib 0 1
Ruxolitinib Ruxolitinib 43 41
Ruxolitinib + Venetoclax Ruxolitinib Venetoclax 0 1
Siltuximab Siltuximab 0 1
Sirolimus Sirolimus 21 32
Sorafenib Sorafenib 91 51
Sulindac Sulindac 7 0
Sunitinib Sunitinib 112 54
Tagraxofusp-erzs Tagraxofusp-erzs 1 3
Temsirolimus Temsirolimus 12 20
Tofacitinib Tofacitinib 21 0
trastuzumab and hyaluronidase-oysk injection trastuzumab and hyaluronidase-oysk injection 2 0
Tretinoin Tretinoin 2 5
Valproic acid Valproic acid 4 10
Valrubicin Valrubicin 1 1
Vandetanib Vandetanib 34 8
Vemurafenib Vemurafenib 138 23
Vinorelbine Vinorelbine 1 16
Vismodegib Vismodegib 54 19
Vorinostat Vorinostat 9 29
Drug Name Trade Name Synonyms Drug Classes Drug Description
Abaloparatide Tymlos BA058|BA-058|BIM44058|BIM-44058 Abaloparatide (Tymlos) is a peptide analog of human parathyroid hormone-related protein (PTHrP), which stimulates bone formation and potentially increases bone mineral density (PMID: 25393645, PMID: 29260289). Abaloparatide (Tymlos) is FDA approved for osteoporosis (FDA.gov).
Apalutamide Erleada ARN-509|JNJ-56021927 Hormone - Anti-androgens 28 Erleada (apalutamide) is a second-generation antiandgrogen, that binds to the androgen receptor (AR) and inhibits downstream signaling, potentially resulting in decreased growth of AR-expressing tumors (PMID: 22266222, PMID: 23337756). Erleada (apalutamide) is FDA approved for use in patients with non-metastatic castration-resistant prostate cancer and metastatic castration-sensitive prostate cancer (FDA.gov).
Asparaginase Erwinia chrysanthemi Erwinaze Crisantaspase Erwinaze (asparaginase Erwinia chrysanthemi) is an bacterial derived enzyme that converts L-asparagine to L-aspartate and ammonia, which decreases L-asparagine levels, potentially leading to reduced leukemic cell growth (PMID: 25098829, PMID: 26698391). Erwinaze (asparaginase Erwinia chrysanthemi) is FDA-approved for use in a chemotherapeutic regimen for acute lymphoblastic leukemia (FDA.gov).
Atezolizumab Tecentriq RG7446|MPDL3280A Immune Checkpoint Inhibitor 98 PD-L1/PD-1 antibody 68 Tecentriq (atezolizumab) is a monoclonal antibody against PD-L1 (CD274), preventing activation of its receptor, potentially enhancing T-cell-mediated immune response to neoplasms and inhibiting T-cell inactivation (PMID: 29449897). Tecentriq (atezolizumab) is FDA approved for use in PD-L1 positive advanced or metastatic urothelial carcinoma not eligible for cisplatin-containing chemotherapy, in advanced or metastatic urothelial carcinoma not eligible for chemotherapy or progressed on platinum-based chemotherapy, in metastatic non-small cell lung cancer (NSCLC) progressed on platinum-containing therapy, as first-line therapy in metastatic NSCLC with high PD-L1 expression (TC>=50% or IC>=10%) and without EGFR or ALK alterations, in combination with bevacizumab, paclitaxel, and carboplatin as first-line therapy for non-squamous NSCLC with no EGFR or ALK aberrations, in combination with paclitaxel protein-bound and carboplatin in metastatic non-squamous NSCLC with no EGFR or ALK aberrations, in combination with paclitaxel protein-bound in advanced or metastatic triple-negative breast cancer expressing PD-L1, in combination with carboplatin and etoposide in extensive-stage small cell lung cancer, in combination with bevacizumab in hepatocellular carcinoma without prior systemic therapy, and in combination with cobimetinib and vemurafenib in BRAF V600-mutated melanoma (FDA.gov).
BCG solution TICE BCG solution Bacillus Calmette-Guerin solution TICE BCG solution (BCG solution) is an attenuated live culture of Bacillus Calmette Guerin that induces anti-tumor immunity in bladder cancer (PMID: 26000263). TICE BCG solution (BCG solution) is FDA approved for carcinoma in situ of the bladder (FDA.gov).
Balsalazide Carbadox Balsalazida Carbadox (balsalazide) is a 5-aminosalicylate (5-ASA) type drug that is FDA approved for the treatment of inflammation resulting from ulcerative colitis (FDA.gov), however, has also been demonstrated to induce apoptosis and decrease cell proliferation in cancer cells (PMID: 28108625).
Brexucabtagene autoleucel Tecartus KTE-X19 Tecartus (brexucabtagene autoleucel) are autologous T-cells engineered to express an anti CD-19 chimeric antigen receptor, which may potentially target CD-19 expressing tumor cells (PMID: 32242358). Tecartus (brexucabtagene autoleucel) is FDA approved fro use in adult patients with relapsed or refractory mantle cell lymphoma (FDA.gov).
Capmatinib Tabrecta INC280|INC-280|INCB028060|INCB28060|INCB 28060 MET Inhibitor 51 Tabrecta (capmatinib) inhibits MET signaling and may induce cell death in tumors overexpressing MET or expressing constitutively activated MET (PMID: 31853265). Tabrecta (capmatinib) is FDA approved for use in patients with metastatic non-small cell lung cancer harboring MET exon 14 skipping mutations (FDA.gov).
Carfilzomib Kyprolis PR-171 Kyprolis (carfilzomib) binds to the 20S proteasome and inhibits its protein degradation activity, thereby leading to the accumulation of ubiquinated proteins, which may result in apoptosis and growth arrest in tumor cells. Kyprolis (carfilzomib), in combination with lenalidomide and dexamethasone, is FDA approved for multiple myeloma (FDA.gov).
Celecoxib Celebra Celebra (celecoxib) is a COX-2 inhibitor FDA approved as an anti-inflammatory agent and for colorectal polyp reduction (FDA.gov) and also promotes apoptosis of tumor cells (PMID: 17909047).
Cyclophosphamide Cytoxan CPM Chemotherapy - Alkylating 14 Cytoxan (cyclophosphamide) is an alkylating agent, which inhibits DNA replication (NCI Drug Dictionary). Cytoxan (cyclophosphamide) is FDA approved in multiple hematological malignancies, breast cancer, neuroblastoma, ovarian cancer, and retinoblastoma (NCI Drug Dictionary).
Dacarbazine Deticene Biocarbazine R Chemotherapy - Alkylating 14 Deticene (dacarbazine) alkylates and cross-links DNA, which disrupts the cell-cycle. Dacarbazine is FDA approved for melanoma (FDA.gov).
Daratumumab Darzalex JNJ-54767414 CD38 Antibody 7 Darzalex (Daratumumab) is a human antibody against CD38 that induces antibody-dependent cell-mediated cytotoxicity and complement-mediated cytotoxicity against CD38-positive tumor cells (PMID: 30546360). Darzalex (Daratumumab) is FDA approved for multiple myeloma patients as a monotherapy or in combination with lenalidomide and dexamethasone, or with bortezomib and dexamethasone, or with bortezomib, melphalan, and prednisone, or with pomalidomide and dexamethasone, or with bortezomib, thalidomide and dexamethasone (FDA.gov).
Daratumumab and hyaluronidase-fihj Darzalex Faspro Daratumumab/rHuPH20|HuMax-CD38-rHuPH20|darzalex/rHuPH20 Darzalex Faspro (Daratumumab and hyaluronidase-fihj) is a combination of the anti-CD38 antibody Daratumumab with hyaluronidase, which may be administered subcutaneously (PMID: 32213342). Darzalex Faspro (Daratumumab and hyaluronidase-fihj) is FDA approved for multiple myeloma patients as a monotherapy or in combination with lenalidomide and dexamethasone, or with bortezomib and dexamethasone, or with bortezomib, melphalan, and prednisone, and in combination with bortezomib, cyclophosphamide and dexamethasone in patients with newly diagnosed light chain amyloidosis (FDA.gov).
Darolutamide Nubeqa BAY1841788|ODM-201 Hormone - Anti-androgens 28 Nubeqa (darolutamide) is an androgen receptor antagonist that binds to and prevents nuclear translocation of the androgen receptor, thereby inhibiting expression of genes downstream of androgen receptor signaling that regulate proliferation of prostate cancer cells (PMID: 28490267). Nubeqa (darolutamide) is FDA approved for use in patients with non-metastatic castration-resistant prostate cancer (FDA.gov).
Daunorubicin Cerubidine Rubidomycin|daunomycin hydrochloride|rubomycin C|Acetyladriamycin Chemotherapy - Anthracycline 11 Cerubidine (daunorubicin) is an anthracycline, which inhibits DNA replication and repair. Cerubidine (daunorubicin) is FDA approved for AML and MLL (FDA.gov).
Daunoxome Liposomal daunorubicin citrate Chemotherapy - Anthracycline 11 Daunoxome is a liposomal formulation of the anthracyline, daunorubicin and is FDA approved for HIV-associated Kaposi’s sarcoma (FDA.gov).
Decitabine and Cedazuridine Inqovi ASTX-727|ASTX 727|ASTX727 Chemotherapy - Antimetabolite 12 Inqovi (decitabine and cedazuridine) is a combination of decitabine and cedazuridine, which may inhibit proliferation of tumor cells (PMID: 30926081). Inqovi (decitabine and cedazuridine) is an FDA approved chemotherapy agent (FDA.gov).
Divalproex sodium Depakote Valproate semisodium|Sodium divalproex HDAC Inhibitor 38 Depakote (Divalproex sodium) comprises a 1:1 mixture of valproic acid and sodium valproate, which acts as an HDAC inhibitor, potentially resulting in decreased growth and increased apoptosis of tumor cells in combination with other agents (PMID: 25449787, PMID: 20200483). Depakote (divalproex sodium) is FDA approved for treatment of manic episodes associated with bipolar disorder, as well as seizures, and migraines (FDA.gov).
Doxorubicin Adriamycin Adria|ADR|Doxorubicin hydrochloride|Hydroxydaunorubicin Chemotherapy - Anthracycline 11 TOPO2 inhibitor 4 Adriamycin (doxorubicin) is an anthracycline chemotherapeutic, in a non-liposomal formulation, which intercalates into DNA and inhibits topoisomerase II (PMID: 24367159). Doxorubicin is FDA approved for multiple cancer types (FDA.gov).
Duvelisib Copiktra IPI-145|INK1197 PIK3CD inhibitor 24 PIK3CG inhibitor 8 Copiktra (duvelisib) prevents the activation of the PI3K delta/gamma-mediated signaling pathways, resulting in tumor cell death (PMID: 25258342). Copiktra (duvelisib) is FDA approved for use in adult patients with relapsed or refractory chronic lymphocytic leukemia, small lymphocytic lymphoma, and follicular lymphoma (FDA.gov)
Enfortumab vedotin-ejfv Padcev AGS-22ME Padcev (enfortumab vedotin-ejfv) is an antibody drug conjugate that fuses together a human antibody against NECTIN4 and monomethyl auristatin E, which may result in inhibition of tumor growth and tumor regression (PMID: 27013195). Padcev (enfortumab vedotin-ejfv) is FDA approved for use in patients with locally advanced or metastatic urothelial cancer who have previously received a PD-1 or PD-L1 inhibitor, and a platinum-containing chemotherapy (FDA.gov).
Entrectinib Rozlytrek RXDX-101 ALK Inhibitor 23 ROS1 Inhibitor 14 Trk Receptor Inhibitor (Pan) 23 Rozlytrek (entrectinib) inhibits the activity of TrkA/B/C, Ros1 fusion proteins, and Alk, which leads to induction of apoptosis and inhibition of proliferation in cells expressing these kinases (PMID: 27003761). Rozlytrek (entrectinib) is FDA approved for use in patients with ROS1 positive non-small cell lung cancer and in adult and pediatric (12 years and older) patients with solid tumors harboring NRTRK fusions without known acquired resistance mutations (FDA.gov).
Epirubicin Ellence Epirubicin Hydrochloride Chemotherapy - Anthracycline 11 Ellence (epirubicin) is an anthracycline, which intercalates into DNA and inhibits topoisomerase II (PMID: 24367159). Ellence (epirubicin) is FDA approved as a component of adjuvant therapy in patients with evidence of axillary node tumor involvement for primary breast cancer (FDA.gov).
Fedratinib Inrebic SAR302503|TG101348 FLT3 Inhibitor 55 JAK2 Inhibitor - ATP competitive 14 RET Inhibitor 39 Inrebic (fedratinib) binds and inhibits JAK2, FLT3, and RET kinases, which may inhibit cell proliferation and tumor growth (PMID: 17541402). Inrebic (fedratinib) is FDA approved for use in patients with myelofibrosis (FDA.gov).
Fluorouracil Adrucil 5-FU Chemotherapy - Antimetabolite 12 Adrucil (fluorouracil) is an antimetabolite chemotherapeutic agent, which interferes with DNA and RNA synthesis thereby preventing cancer cell growth and is FDA approved for colorectal, breast, stomach, and pancreatic cancer (FDA.gov).
Fulvestrant Faslodex ICI 182,780 Hormone - Anti-estrogens 18 Faslodex (fulvestrant) is a competitive inhibitor of estrogen that binds and deforms estrogen receptors, and causes inhibition of growth in cultured estrogen-sensitive breast cancer cells and is FDA approved for use in patients with hormone receptor-positive breast cancer (FDA.gov).
Goserelin Zoladex Goserelin acetate Zoladex (goserelin) is a synthetic analog of lutenizing hormone-releasing hormone, which causes decreased testosterone production in males and decreased estradiol production in females, and is FDA approved for use in prostate carcinoma (FDA.gov).
Ibritumomab tiuxetan Zevalin IDEC Y2B8 CD20 Antibody 11 Zevalin (britumomab tiuxetan) is Yttrium-90 combined with the anti-CD20 antibody, rituximab, which may deliver radiation to tumor cells (PMID: 27497027). Zevalin (britumomab tiuxetan) is FDA approved for use in patients with low-grade or follicular B-cell non-Hodgkin's lymphoma (FDA.gov).
Idarubicin Idamycin Idarubicin Hcl Chemotherapy - Anthracycline 11 Idarubicin is an anthracycline, which inhibits DNA replication thereby preventing RNA and protein synthesis and, in combination with other approved drugs, is FDA approved for acute myelogenous leukemia (FDA.gov).
Isatuximab Sarclisa SAR650984|isatuximab-irfc CD38 Antibody 7 Sarclisa (isatuximab-irfc) is a humanized monoclonal antibody against CD38 that induces anti-tumor immune reaction against CD38-positive tumor cells (PMID: 24987056). Sarclisa (isatuximab-irfc) in combination with Pomalyst (pomalidomide) and dexamethasone is FDA approved for use in patients with multiple myeloma who have received two or more prior therapies (FDA.gov).
KTE-C19 Yescarta KTE-C19 CAR|axicabtagene ciloleucel Yescarta (KTE-C19) is an immunotherapy consisted of peripheral T-lymphocytes engineered to express a chimeric antigen receptor targeting CD19, CD28 and CD3zeta, resulting in immunostimulating and antineoplastic activities (Blood 2015 126:3991). Yescarta (KTE-C19) is FDA approved for treatment of relapsed or refractory large B-cell lymphoma (FDA.gov).
Letrozole Femara Letrozol Aromatase Inhibitor 3 Femara (letrozole) is an aromatase inhibitor, which inhibits estrogen synthesis, and is approved for postmenopausal women with hormone receptor positive breast cancer (FDA.gov).
Lisocabtagene maraleucel Breyanzi JCAR 017 Breyanzi (lisocabtagene maraleucel) comprises equal amount of CD4+ and CD8+ autologous T lymphocytes engineered to express chimeric antigen receptors containing an anti-CD19 fragment fused to the 4-1BB (CD137) signaling domain, which may results in immune modulating and anti-tumor activities (PMID: 32888407). Breyanzi (lisocabtagene maraleucel) is FDA approved for use in patients with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy, including diffuse large B-cell lymphoma, high-grade B-cell lymphoma, primary mediastinal large B-cell lymphoma, and follicular lymphoma grade 3B (FDA.gov).
Lorlatinib Lorbrena PF-06463922 ALK Inhibitor 23 ROS1 Inhibitor 14 Lorbrena (lorlatinib) is a small molecule inhibitor of Alk and Ros1 fusion proteins, which may result in suppression of Alk and Ros1-mediated signaling thereby inhibiting cell growth and causing tumor regression (PMID: 26144315). Lorbrena (lorlatinib) is FDA approved for use in patients with ALK-positive non-small cell lung cancer (FDA.gov).
Lurbinectedin Zepzelca PM01183 Zepzelca (lurbinectedin) is an alkaloid analogue that binds to the minor groove of DNA, resulting in delayed cell cycle progression and cell death (PMID: 20977459). Zepzelca (lurbinectedin) is an FDA-approved chemotherapy agent (FDA.gov).
Margetuximab-cmkb Margenza MGAH22|MGAH-22 HER2 (ERBB2) Antibody 50 Margenza (margetuximab-cmkb) is a monoclonal antibody that binds ERBB2 (HER2) and induces antitumor response against ERBB2 (HER2)-expressing tumor cells (PMID: 22129105, PMID: 32653053). Margenza (margetuximab-cmkb) in combination with chemotherapy is FDA approved for use in patients with metastatic ERBB2 (HER2)-positive breast cancer who had received two or more prior anti-ERBB2 (HER2) therapies (FDA.gov).
Melphalan Alkeran Chemotherapy - Alkylating 14 Alkeran (melphalan) is an antineoplastic alkylating agent, which cross-links DNA and induces cell toxicity and is FDA approved for multiple myeloma and epithelial ovarian carcinoma (FDA.gov).
Methotrexate Abitrexate Amethopterin Chemotherapy - Antimetabolite 12 Methotrexate is an antimetabolite, which inhibits DHFR resulting in decreased immune function and antineoplastic activity and is FDA approved for psoriasis, RA, and several cancers including choriocarcinoma, AML, lung, head and neck and epidermoid (FDA.gov).
Mitomycin gel Jelmyto UGN-101|Mitogel|MP77-08 Jelmyto (mitomycin gel) is a prolonged release gel formulation of mitomycin that may have antitumor activity when applied in the upper urinary tract (PMID: 32670424). Jelmyto (mitomycin for pyelocalyceal solution) is FDA approved for use in patients with low-grade upper tract urothelial cancer (FDA.gov).
Naxitamab Danyelza hu3F8 Danyelza (naxitamab) is an antibody that targets the ganglioside GD2, potentially resulting in increased anti-tumor immune response against GD2-expressing tumor cells (PMID: 22754766). Danyelza (naxitamab) is FDA approved for use in combination with GM-CSF in pediatric patients one year of age and older and adult patients with relapsed or refractory high-risk neuroblastoma in the bone or bone marrow who achieved a partial response, minor response, or stable disease to prior therapy (FDA.gov).
Omacetaxine mepesuccinate Synribo homoharringtonine|CGX-635 Synribo (omacetaxine mepesuccinate) is a semisynthetic alkaloid that inhibits protein synthesis, resulting in increased apoptosis and cell-cycle arrest in tumor cells (PMID: 26935769, PMID: 24516334). Synribo (omacetaxine mepesuccinate) is FDA approved for use in patients with chronic myeloid leukemia (FDA.gov).
Orlistat Xenical (-)-Tetrahydrolipstatin Xenical (orlistat) is a fatty acid inhibitor that also demonstrates anti-tumor activity (PMID: 30667213). Xenical (orlistat) is FDA approved for weight management (FDA.gov).
Oxaliplatin Eloxatin Diaminocyclohexane Oxalatoplatinum Chemotherapy - Platinum 6 Eloxatin (oxaliplatin) is comprised of a platinum complex, which causes DNA-platinum cross-links, inhibition of DNA replication and transcription, and cell toxicity, and is FDA approved for colorectal cancer (FDA.gov).
Pegylated liposomal-doxorubicin Doxil ATI-0918|CAELYX Chemotherapy - Anthracycline 11 Doxil (pegylated liposomal doxorubicin) is doxorubicin (intercalates into DNA, disrupting DNA replication) encapsulated, which within a liposome allows better penetration into tumors, decreases toxicity, and increases the period of effectiveness, and is FDA approved for ovarian cancer, AIDS-related Kaposi's sarcoma, and multiple myeloma (FDA.gov).
Pexidartinib Turalio PLX3397|CML-261 CSF1R Inhibitor 25 FLT3 Inhibitor 55 KIT Inhibitor 51 Turalio (pexidartinib) inhibits multiple receptor tyrosine kinases, including KIT, CSF1R, FLT3, and FLT3/ITD, which may inhibit growth in cancer cells (PMID: 22294205, PMID: 25993548, PMID: 25847190). Turalio (pexidartinib) is FDA approved for use in patients with symptomatic tenosynovial giant cell tumor (FDA.gov).
Pomalidomide Pomalyst CC-4047 Pomalyst (pomalidomide) is a thalidomide derivative that inhibits angiogenesis and modulates immune response, potentially lead to antitumor activity (PMID: 30069630). Pomalyst (pomalidomide) is FDA approved for use in patients with Kaposi's sarcoma who failed HAART or are HIV-negative, and in combination with dexamethasone in patients with multiple myeloma who had 2 or more prior therapies (FDA.gov).
Pralsetinib Gavreto BLU667|BLU-667 RET Inhibitor 39 Gavreto (pralsetinib) is a small molecule inhibitor that selectively targets activated Ret, which may result in antitumor activity including inhibition of tumor growth and tumor regression (PMID: 29657135). Gavreto (pralsetinib) is FDA approved for use in patients with metastatic MET fusion-positive non-small cell lung cancer, in adult and pediatric patients 12 years of age and older with advanced or metastatic RET-mutant medullary thyroid cancer, and in adult and pediatric patients 12 years of age and older with RET fusion-positive thyroid cancer who are radioactive iodine-refractory (FDA.gov).
Ripretinib Qinlock DCC-2618|DCC2618 KIT Inhibitor 51 PDGFR-alpha Inhibitor 9 Qinlock (ripretinib) is a dual inhibitor of KIT and PDGFRa, resulting in growth inhibition and anti-tumor effects (PMID: 29439183, PMID: 31085175, PMID: 31201392). Qinlock (ripretinib) is FDA approved for use in patients with advanced gastrointestinal stromal tumor who received 3 or more prior kinase inhibitor therapies (FDA.gov)
Sacituzumab govitecan-hziy Trodelvy IMMU-132|Sactizumab Trodelvy (sacituzumab govitecan-hziy) is an antibody-drug conjugate composed of a monoclonal anti-TROP2 antibody linked to the active metabolite of irinotecan (SN-38), which binds to TROP2 positive cancer cells and induces DNA breakage and apoptosis (PMID: 26541586). Trodelvy (sacituzumab govitecan-hziy) is FDA approved for use in patients with metastatic triple-negative breast cancer who received two or more priotr therapies (FDA.gov).
Siltuximab Sylvant CNTO 328|CNTO328 Sylvant (siltuxmab) is an anti-IL-6 chimeric monoclonal antibody, which inhibits IL6 signaling and may lead to decreased tumor cell growth (PMID: 26170629, PMID: 26744529). Sylvant (siltuximab) is FDA approved for patients with multicentric Castleman's disease who do not have HIV or HHV-8 (FDA.gov).
Sulindac Clinoril MK-231 Clinoril (sulindac) is a non-selective COX1-2 inhibitor, FDA approved as an anti-inflammatory agent (FDA.gov), and Clinoril (sulindac) also reduces intestinal tumor burden (PMID: 19755659).
Tafasitamab-cxix Monjuvi MOR00208|XmAb5574|MOR-208|MOR208 CD19 Antibody 10 Monjuvi (tafasitamab-cxix) is an CD19-targeted antibody with an engineered Fc region, which may result in an increased antibody-dependent cell-mediated cytotoxicity response against CD19-expressing tumor cells (PMID: 23277329, PMID: 32511983). Monjuvi (tafasitamab-cxix) is FDA approved for use in combination with Revlimid (lenalidomide) in patients with relapsed or refractory diffuse large B cell lymphoma (FDA.gov).
Tepotinib Tepmetko EMD1214063|EMD-1214063EMD 1214063|MSC2156119|MSC-2156119|MSC2156119J MET Inhibitor 51 Tepmetko (tepotinib) selectively binds to the proto-oncogene hepatocyte growth factor receptor (HGFR or c-Met), which inhibits c-Met phosphorylation and disrupts c-Met-mediated signaling pathways, thereby suppressing tumor growth in tumor cells with activated or overexpression of c-Met protein (PMID: 32328660). Tepmetko (tepotinib) is FDA approved for use in patients with metastatic non-small cell lung cancer harboring MET exon 14 skipping mutations (FDA.gov).
Thalidomide Thalomid N-Phthalimidoglutamic acid imide Thalomid (thalidomide) is a a racaemic glutamic acid analogue that has anti-inflammatory, anti-angiogenic, immunosuppressive, and anti-tumor activities (PMID: 15172781). Thalomid (thalidomide) is approved for use in combination with dexamethasone for patients with newly diagnosed multiple myeloma (FDA.gov).
Tocilizumab Actemra Atlizumab Actemra (tocilizumab) is a humamized monoclonal antibody that targets IL-6R, resulting in decreased downstream signaling and potentially resulted in decreased tumor growth and progression (PMID: 26751841, PMID: 25658637). Actemra (tocilizumab) is FDA approved for use in treating rheumatoid arthritis, polyarticular juvenile idiopathic arthritis, and systemic juvenile idiopathic arthritis (FDA.gov).
Tofacitinib Xeljanz Tasocitinib|CP-690550|CP-690,550 JAK Inhibitor (Pan) - ATP competitive 3 Xeljanz (tofacitinib) is a pan-JAK inhibitor, which decreases downstream signaling and potentially leads to reduced tumor cell growth (PMID: 26300391, PMID: 21106989). Xeljanz (tofacitinib) is FDA approved for rheumatoid arthritis (FDA.gov).
Trastuzumab deruxtecan Enhertu DS-8201|DS8201a|T-DXd|DS-8201a|fam-trastuzumab deruxtecan-nxki HER2 (ERBB2) Antibody 50 Enhertu (trastuzumab deruxtecan) is an antibody-drug conjugate comprising an anti-ERBB2 (HER2) antibody linked to a derivative of the topoisomerase inhibitor DX-8951, which delivers the cytotoxic agent to ERBB2 (HER2)-expressing tumor cells, potentially resulting in decreased growth of tumors, including tumors with low ERBB2 (HER2) expression (PMID: 27026201). Enhertu (fam-trastuzumab deruxtecan-nxki) is FDA approved for use in patients with ERBB2 (HER2)-positive breast cancer who have received two or more anti-HER2 therapies previously, and in patients with locally advanced or metastatic HER2-positive gastric or gastroesophageal (GEJ) adenocarcinoma who have received a prior trastuzumab-based therapy (FDA.gov).
Tretinoin Aberel|Aknoten|Avita|Renova|Retin-A|all-trans retinoic acid|Vitamin A Acid|ATRA Tretinoin is a form of retinoic acid, which binds to retinoic acid receptors and activates downstream signaling, potentially inducing cell differentiation and subsequently, apoptosis (PMID: 11704842). Tretinoin is FDA approved for patients with acute promyelocytic leukemia harboring PML-RARA (FDA.gov).
Umbralisib Ukoniq RP5264|TGR-1202 PIK3CD inhibitor 24 Ukoniq (umbralisib) is a selective inhibitor of PIK3CD, which prevents Akt phosphorylation and induces cytotoxicity (J Clin Oncol 32:5s, 2014 (suppl; abstr 2513), PMID: 30709431). Ukoniq (umbralisib) is FDA approved for use in adult patients with relapsed or refractory marginal zone lymphoma with one or more prior anti-CD20-based therapy, and in adult patients with relapsed or refractory follicular lymphoma who have three or more prior lines of therapy (FDA.gov).
Vincristine sulfate liposome Marqibo VSL Marqibo (vincristine sulfate liposome) is a liposomal formulation of vincristine with improved bioavailability (PMID: 23212117). Marqibo (vincristine sulfate liposome) is FDA-approved for Philadelphia chromosome (BCR-ABL1)-negative acute lymphoblastic leukemia (FDA.gov).
Vinorelbine Navelbine Vinorelbin Antimicrotubule Agent 12 Navelbine (vinorelbine) is a tubulin binding alkaloid that inhibits tubulin polymerization, thus inhibiting spindle formation, leading to apoptosis (NCI Drug Dictionary). Navelbine (vinorelbine) is approved for use in NSCLC (FDA.gov).
Vorinostat Zolinza SAHA|L-001079038|MSK390 HDAC Inhibitor 38 Zolinza (vorinostat) inhibits HDAC activity, resulting in cell-cycle arrest, decreased proliferation, and increased differentiation of tumor cells (PMID: 9501205, PMID: 11731433, PMID: 11016644). Zolinza (vorinostat) is FDA approved for use in cutaneous T-cell lymphoma (FDA.gov).
Zanubrutinib Brukinsa BGB-3111 BTK inhibitor 24 Brukinsa (zanubrutinib) inhibits Bruton tyrosine kinase (BTK), leading to decreased B-cell activation and reduced growth of BTK over-expressing cells (PMID: 31378456). Brukinsa (zanubrutinib) is FDA approved for use in patients with mantle cell lymphoma (FDA.gov).
Gene Variant Impact Protein Effect Variant Description Associated with drug Resistance
Molecular Profile Indication/Tumor Type Response Type Therapy Name Approval Status Evidence Type Efficacy Evidence References
Unknown unknown Indication other than cancer not applicable Metformin FDA approved Actionable Glucophage (metformin) is FDA approved for use in patients with type-2 diabetes (FDA.gov). detail... detail...
Unknown unknown Indication other than cancer not applicable Fingolimod FDA approved Actionable Gilenya (fingolimod) is FDA approved for use in patients with relapsing forms of multiple sclerosis (FDA.gov). detail... detail...
ERBB2 over exp gastroesophageal adenocarcinoma sensitive Trastuzumab-anns FDA approved - On Companion Diagnostic Actionable In a Phase I trial that supported FDA approval, the Herceptin (trastuzumab) biosimilar Kanjinti (Trastuzumab-anns) demonstrated structure, function, and pharmacokinetic profile comparable to Herceptin (trastuzumab) (PMID: 28341959), thus supporting the extrapolation of data from the Phase III trial that supported the approval of Herceptin (trastuzumab) in Erbb2 (Her2) overexpressing gastrioesophageal adenocarcinoma (PMID: 20728210; NCT01041404) for approval of Kanjinti (Trastuzumab-anns) (FDA.gov). 20728210 detail... 28341959 detail... detail...
Unknown unknown Indication other than cancer not applicable Valproic acid FDA approved Actionable Valproic acid is FDA approved for use in patients with seizures, migraines, and bipolar disorder (FDA.gov). detail... detail...
ERBB2 over exp gastric adenocarcinoma sensitive Trastuzumab-anns FDA approved - On Companion Diagnostic Actionable In a Phase I trial that supported FDA approval, the Herceptin (trastuzumab) biosimilar Kanjinti (Trastuzumab-anns) demonstrated structure, function, and pharmacokinetic profile comparable to Herceptin (trastuzumab) (PMID: 28341959), thus supporting the extrapolation of data from the Phase III trial that supported the approval of Herceptin (trastuzumab) in Erbb2 (Her2) overexpressing gastric adenocarcinoma (PMID: 20728210; NCT01041404) for approval of Kanjinti (Trastuzumab-anns) (FDA.gov). 20728210 28341959 detail... detail... detail...
Unknown unknown aggressive NK-cell leukemia not applicable Tagraxofusp-erzs FDA approved Actionable In a Phase I/II trial that supported FDA approval, Elzonris (tagraxofusp-erzs) treatment resulted in complete response/clinical complete response in 53.8% (7/13) of patients with untreated blastic plasmacytoid dendritic cell neoplasm (BPDCN, also known as natural killer cell leukemia/lymphoma) with a median follow-up of 11.5 months, and 1 complete response and 1 clinical complete response in 15 patients with relapsed or refractory BPDCN (FDA.gov; NCT02113982). detail... detail...
Unknown unknown Indication other than cancer not applicable Sirolimus FDA approved Actionable Rapamune (sirolimus) is FDA approved for use in patients with lymphangioleiomyomatosis and for prophylactic immunosuppression in renal transplant patients (FDA.gov). detail... detail...
Unknown unknown Indication other than cancer not applicable Nintedanib FDA approved Actionable Ofev (nintedanib) is FDA approved for use in patients with idiopathic pulmonary fibrosis (FDA.gov). detail... detail...
ERBB2 over exp Her2-receptor positive breast cancer sensitive Docetaxel + Pertuzumab/trastuzumab/hyaluronidase-zzxf FDA approved Actionable In a Phase III trial (FeDeriCa) that supported FDA approval, Phesgo (pertuzumab/trastuzumab/hyaluronidase-zzxf) demonstrated pharmacokinetics, safety, and efficacy comparable to i.v. pertuzumab and trastuzumab (H+P) (Cancer Res 2020;80(4 Suppl):Abstract nr PD4-07; NCT03493854), warranted the extrapolation of data from a Phase III trial supporting the approval of H+P plus docetaxel in Erbb2 (Her2)-positive metastatic breast cancer (PMID: 23602601; NCT00567190) for approval of Phesgo (FDA.gov). detail... detail... 23602601 detail...
ERBB2 amp Her2-receptor positive breast cancer sensitive Docetaxel + Pertuzumab/trastuzumab/hyaluronidase-zzxf FDA approved Actionable In a Phase III trial (FeDeriCa) that supported FDA approval, Phesgo (pertuzumab/trastuzumab/hyaluronidase-zzxf) demonstrated pharmacokinetics, safety, and efficacy comparable to i.v. pertuzumab and trastuzumab (H+P) (Cancer Res 2020;80(4 Suppl):Abstract nr PD4-07; NCT03493854), warranted the extrapolation of data from a Phase III trial supporting the approval of H+P plus docetaxel in Erbb2 (Her2)-positive metastatic breast cancer (PMID: 23602601; NCT00567190) for approval of Phesgo (FDA.gov). detail... 23602601 detail... detail...
Unknown unknown Indication other than cancer not applicable Imatinib FDA approved Actionable Gleevec (imatinib) is FDA approved for patients with aggressive systemic mastocytosis in which c-KIT mutational status is unknown (FDA.gov). detail... detail...
ERBB2 over exp breast cancer sensitive Trastuzumab-anns FDA approved - On Companion Diagnostic Actionable In a Phase I trial that supported FDA approval, the Herceptin (trastuzumab) biosimilar Kanjinti (Trastuzumab-anns) demonstrated structure, function, and pharmacokinetic profile comparable to Herceptin (trastuzumab) (PMID: 28341959), thus supporting the extrapolation of data from the Phase III trial that supported the approval of Herceptin (trastuzumab) in Erbb2 (Her2) overexpressing breast cancer (PMID: 23602601; NCT00567190) for approval of Kanjinti (Trastuzumab-anns) (FDA.gov). 23602601 detail... 28341959 detail... detail...