Loading
One long-form page per target, generated from the graph: biology, prevalence by cancer, mutation hotspots (23 targets so far), every product by modality and phase, trials, resistance routes, pathways, companion diagnostics, preclinical models, open questions and the external identifiers that join OnCo to UniProt, Ensembl, ChEMBL, Open Targets, CIViC, OncoKB and COSMIC. 1674 targets.
EGFR Gene: EGFR A growth receptor that is mutated in some lung cancers and overproduced in others; the first great success of targeted pills. | 51 | 27 | 372 | 12 | 12 | 4 | 2 |
HER2 Gene: ERBB2 A growth-signal receptor. Some cancers make far too much of it, and drugs that block it or use it as a docking site have transformed those cancers. | 44 | 19 | 278 | 17 | 9 | 6 | 2 |
PD-1 Gene: PDCD1 PD-1 is a brake on T cells. Blocking it releases the immune system against the tumour and has cured some previously incurable cancers. | 32 | 17 | 1079 | 2 | 12 | 7 | 3 |
VEGF / VEGFR Gene: VEGFA, KDR, FLT1, FLT4, PGF, FLT1, FLT4 The signal tumours use to grow their own blood supply. Blocking it starves tumours and, surprisingly, helps immunotherapy work. | 29 | 22 | 582 | 3 | 12 | 1 | 0 |
CD19 Gene: CD19 CD19 is a marker on B cells and B-cell cancers, and was the target of the first CAR-T therapies ever approved. | 26 | 12 | 76 | 3 | 2 | 5 | 1 |
PD-L1 Gene: CD274 PD-L1 is the tumour's side of the PD-1 brake, and also the biomarker that decides who gets immunotherapy. | 25 | 12 | 448 | 8 | 5 | 5 | 1 |
CD3 Gene: CD3E CD3 is the switch on every T cell. Bispecific drugs grab it with one arm and the tumour with the other, forcing the T cell to attack. | 24 | 12 | 114 | 1 | 2 | 14 | 0 |
KRAS Gene: KRAS KRAS is the most commonly mutated cancer gene, called 'undruggable' for 40 years until 2021. | 24 | 11 | 108 | 22 | 26 | 4 | 3 |
MET Gene: MET A receptor that is either mutated in some lung cancers or amplified as an escape route when other lung cancer drugs fail. | 22 | 11 | 119 | 5 | 5 | 5 | 1 |
Androgen receptor Gene: AR The hormone switch that drives prostate cancer, attacked by castration and by pills that block the receptor. | 19 | 11 | 116 | 5 | 5 | 2 | 0 |
Estrogen receptor (ERα) Gene: ESR1 The hormone switch that drives most breast cancers. Blocking or destroying it is the oldest and most effective targeted therapy. | 17 | 14 | 140 | 2 | 6 | 4 | 1 |
Claudin 18.2 Gene: CLDN18 A tight-junction protein normally hidden in the stomach lining that becomes exposed in gastric and pancreatic cancers. | 16 | 2 | 29 | 2 | 1 | 0 | 1 |
FGFR2 Gene: FGFR2 FGFR2 is a growth receptor fused in bile-duct cancer and overproduced in gastric cancer. | 16 | 8 | 121 | 5 | 3 | 0 | 1 |
PSMA Gene: FOLH1 A protein on prostate cancer cells that lets doctors both see the cancer on a PET scan and hit it with a radioactive drug. | 15 | 6 | 36 | 3 | 1 | 5 | 1 |
BCMA Gene: TNFRSF17 BCMA is a survival receptor on plasma cells, and the target that made CAR-T and bispecifics work in multiple myeloma. | 14 | 8 | 59 | 1 | 0 | 3 | 1 |
KIT Gene: KIT KIT mutation is the driver behind most gastrointestinal stromal tumours, and the reason imatinib turned a sarcoma with a median survival of about a year into a chronic disease. | 14 | 11 | 159 | 2 | 1 | 0 | 0 |
BRAF Gene: BRAF BRAF is a signalling kinase mutated in half of melanomas; blocking it with two drugs at once became a template for targeted therapy. | 13 | 12 | 68 | 10 | 6 | 1 | 1 |
CD20 Gene: MS4A1 CD20 is a B-cell marker; rituximab against it was the first antibody approved for cancer, in 1997. | 13 | 8 | 144 | 2 | 1 | 8 | 0 |
PIK3CA / PI3K-alpha Gene: PIK3CA PIK3CA is the most commonly mutated gene in hormone-driven breast cancer. Drugs against it work, but hitting it cleanly without raising blood sugar took years. | 13 | 8 | 66 | 10 | 14 | 6 | 0 |
ALK Gene: ALK ALK is a gene fusion driver in about 4 to 5% of non-small-cell lung cancers that responds to a succession of ALK inhibitor pills. Lorlatinib kept about 60% of patients progression-free at five years, alectinib is approved after surgery, and neladalkib targets compound resistance mutations. | 11 | 9 | 51 | 5 | 2 | 2 | 1 |
PARP Gene: PARP1 PARP is a DNA repair enzyme. Cancers that have already lost one repair system (BRCA) die when this second one is blocked; healthy cells survive. | 11 | 6 | 97 | 4 | 4 | 9 | 0 |
CTLA-4 Gene: CTLA4 The first immune brake ever targeted for cancer; releasing it won a Nobel Prize and cures a fraction of melanomas. | 10 | 3 | 145 | 1 | 3 | 0 | 0 |
RET Gene: RET RET is a kinase altered in thyroid cancer and a small slice of lung cancer, treatable with one selective pill regardless of where the tumour is. | 10 | 8 | 122 | 4 | 2 | 0 | 0 |
CDK4/6 Gene: CDK4, CDK6 CDK4/6 is the engine that pushes a cell to copy its DNA. Blocking it alongside hormone therapy roughly doubled the time hormone-driven breast cancer stays controlled. | 9 | 5 | 94 | 2 | 13 | 4 | 1 |
Somatostatin receptor 2 Gene: SSTR2 Somatostatin receptor 2 is a hormone receptor densely present on neuroendocrine tumours, and was the first theranostic target to reach routine care. | 9 | 5 | 22 | 2 | 0 | 0 | 0 |
BCR::ABL1 (Philadelphia chromosome) Gene: BCR, ABL1 The fusion that defines chronic myeloid leukaemia and a quarter of adult acute lymphoblastic leukaemia; the first cancer driver ever switched off by a pill. | 8 | 8 | 25 | 1 | 1 | 0 | 0 |
BTK (Bruton tyrosine kinase) Gene: BTK The signalling enzyme that B-cell cancers use to survive. Blocking it turned chronic lymphocytic leukaemia into a disease controlled by a daily pill. | 8 | 5 | 83 | 1 | 1 | 4 | 1 |
HER3 Gene: ERBB3 HER3 is a cousin of HER2 that cancers use as an escape route when HER2 or EGFR are blocked. | 8 | 2 | 92 | 4 | 0 | 1 | 0 |
IDH1 / IDH2 Gene: IDH1, IDH2 A metabolic enzyme whose mutant form produces a molecule that scrambles how genes are read; blocking it slows brain tumours and leukaemias. | 8 | 5 | 17 | 5 | 5 | 0 | 1 |
Folate receptor alpha Gene: FOLR1 Folate receptor alpha is a vitamin receptor that ovarian cancer cells carry in large numbers, used as the docking site for the ADC mirvetuximab. | 7 | 2 | 26 | 3 | 0 | 1 | 1 |
TROP2 Gene: TACSTD2 TROP2 is a surface glycoprotein present at high levels on most epithelial cancers (breast, lung, urothelial, gastric, pancreatic) and at low levels on normal tissue. It does not drive the cancer; it is a delivery address, used by the approved ADCs sacituzumab govitecan and datopotamab deruxtecan and by sacituzumab tirumotecan, with a TROP2 PET tracer in development to pick patients. | 7 | 3 | 134 | 6 | 2 | 7 | 1 |
BRCA1 / BRCA2 (HRD) Gene: BRCA1, BRCA2 DNA repair genes. Inheriting a broken copy raises breast and ovarian cancer risk, but tumours that lose them become uniquely vulnerable to PARP inhibitors and platinum. | 6 | 5 | 71 | 12 | 7 | 9 | 1 |
DLL3 Gene: DLL3 A protein that appears on the surface of small-cell lung cancer cells, now hit by a drug that pulls T cells onto them. | 6 | 1 | 26 | 2 | 4 | 1 | 1 |
FLT3 Gene: FLT3 FLT3 is a kinase mutated in about a third of acute myeloid leukaemias, where adding an inhibitor to chemotherapy improves survival. | 6 | 5 | 15 | 1 | 2 | 0 | 0 |
JAK2 Gene: JAK2 The signalling enzyme that tells marrow cells to make red cells and platelets; a single mutation (V617F) leaves it switched on in most myeloproliferative neoplasms. | 6 | 5 | 27 | 1 | 3 | 1 | 0 |
MEK1/2 Gene: MAP2K1, MAP2K2 MEK is the relay in the growth-signal chain that sits just below RAS and RAF. Blocking it starves BRAF- and RAS-driven tumours of their go signal. | 6 | 4 | 31 | 1 | 7 | 0 | 0 |
ROS1 Gene: ROS1 A gene fusion in about 1-2% of lung cancers that responds for years to targeted pills, now in their third generation. | 6 | 6 | 34 | 2 | 0 | 0 | 0 |
Topoisomerase II alpha (TOP2A) Gene: TOP2A The enzyme that cuts both DNA strands to untangle chromosomes before cell division. Etoposide and the anthracyclines hold it in the cut state, filling dividing cells with double-strand breaks. | 6 | 0 | 15 | 1 | 0 | 0 | 0 |
B7-H3 Gene: CD276 B7-H3 is an immune checkpoint-like surface protein found on 60 to 70% of small-cell lung cancers and 80 to 90% of castration-resistant prostate cancers, with little on normal tissue. It is used as an ADC address, chiefly by ifinatamab deruxtecan, now in phase 3 in small-cell lung cancer; whether blocking its immune-dampening role adds anything beyond payload delivery is unresolved. | 5 | 0 | 36 | 2 | 0 | 1 | 1 |
CD38 Gene: CD38 CD38 is a myeloma surface enzyme and the target of daratumumab, which is now given as a quick under-the-skin injection. | 5 | 2 | 45 | 2 | 0 | 0 | 0 |
NTRK Gene: NTRK1, NTRK2, NTRK3 Rare gene fusions found across dozens of cancer types; the first target where a drug was approved for any tumour carrying it. | 5 | 4 | 21 | 7 | 1 | 0 | 0 |
PRMT5 (MTAP-deleted cancers) Gene: PRMT5, MTAP An enzyme that cancers lacking the MTAP gene (about 10-15% of all tumours) depend on more than normal cells do; new inhibitors designed to exploit that difference are in late trials. | 5 | 0 | 11 | 4 | 1 | 0 | 0 |
AKT Gene: AKT1, AKT2, AKT3 AKT is a central survival kinase downstream of PI3K, blocked by capivasertib in breast and now prostate cancer. | 4 | 3 | 43 | 3 | 10 | 6 | 0 |
CD22 Gene: CD22 CD22 is a B-cell surface protein and the docking site for the leukaemia ADC inotuzumab ozogamicin. | 4 | 1 | 8 | 1 | 0 | 0 | 0 |
CD47 Gene: CD47 CD47 is the 'don't eat me' signal: it binds SIRP-alpha on macrophages to stop them engulfing the cell, and over 90% of AML blasts and large B-cell lymphoma cells display it. Blocking it should let macrophages eat tumour cells, but red cells carry CD47 too, so anaemia is built in, and the lead antibody magrolimab was dropped after failed trials. | 4 | 0 | 11 | 2 | 4 | 0 | 0 |
CEACAM5 Gene: CEACAM5 The classic 'CEA' tumour marker measured in blood, also present on the cell surface where ADCs can reach it. | 4 | 0 | 10 | 3 | 0 | 0 | 0 |
CSF1R Gene: CSF1R The receptor that macrophages depend on; blocking it shrinks tenosynovial giant cell tumour (a CSF1-driven tumour) and depletes tumour-supporting macrophages, though the latter has not yet helped patients with common cancers. | 4 | 2 | 12 | 1 | 7 | 0 | 0 |
HLA-A Gene: HLA-A HLA-A is the molecule that holds up short pieces of a cell's proteins for T cells to inspect. Engineered T-cell receptor therapies such as tebentafusp and afami-cel only work in people with the HLA-A*02 variant, because the receptor recognises the tumour peptide sitting in that particular groove. | 4 | 2 | 7 | 0 | 0 | 0 | 0 |
IKZF1 (Ikaros) Gene: IKZF1 Ikaros is a transcription factor that myeloma cells depend on. Lenalidomide, pomalidomide and the newer CELMoDs work by gluing Ikaros to the cell's disposal machinery so it is destroyed, which kills the plasma cell and wakes up T cells. | 4 | 3 | 93 | 0 | 0 | 1 | 0 |
LAG-3 Gene: LAG3 LAG-3 is the third immune brake to reach approval, combined with PD-1 blockade in melanoma. | 4 | 1 | 28 | 1 | 2 | 1 | 0 |
Nectin-4 Gene: NECTIN4 Nectin-4 is an adhesion protein plentiful on bladder cancer cells, used as the docking site for the ADC enfortumab vedotin. | 4 | 1 | 24 | 2 | 0 | 0 | 0 |
Smoothened (hedgehog pathway) Gene: SMO The switch in the hedgehog developmental pathway that is stuck on in basal cell carcinoma and some medulloblastomas; three approved pills block it. | 4 | 3 | 15 | 2 | 3 | 0 | 0 |
Thymidylate synthase (TYMS) Gene: TYMS The enzyme that makes the thymine building block of DNA. Fluorouracil, capecitabine and pemetrexed jam it, starving dividing cells of thymidine. | 4 | 0 | 94 | 1 | 0 | 0 | 0 |
TP53 Gene: TP53 TP53 is the 'guardian of the genome', broken in half of all cancers. Fixing it directly has so far defeated every attempt, so drugs exploit what its loss makes cancers depend on. | 4 | 1 | 18 | 14 | 26 | 2 | 1 |
Tubulin (microtubules) Gene: TUBB The protein that builds the microtubule scaffolding a cell needs to pull its chromosomes apart. Taxanes freeze the scaffold and vinca alkaloids and eribulin stop it forming; either way the dividing cell stalls and dies. | 4 | 1 | 14 | 1 | 0 | 0 | 0 |
ABL1 Gene: ABL1 ABL1 is the kinase half of the BCR::ABL1 fusion that causes chronic myeloid leukaemia. The CML drugs bind the ABL1 kinase domain, most in its ATP pocket and asciminib in a separate pocket that locks it shut. | 3 | 3 | 17 | 0 | 0 | 0 | 0 |
Aromatase (CYP19A1) Gene: CYP19A1 The enzyme that turns androgens into oestrogen in fat, muscle and breast tissue after the menopause. Aromatase inhibitors remove the oestrogen that drives most breast cancers in post-menopausal women. | 3 | 1 | 5 | 1 | 0 | 0 | 0 |
BRD4 Gene: BRD4 BRD4 is a reader protein that docks on acetylated DNA packaging and pulls in the machinery that switches growth genes such as MYC on. BET inhibitors such as pelabresib and ZEN-3694 block that docking; in NUT carcinoma the cancer's own driver is a BRD4 fusion. | 3 | 1 | 7 | 0 | 0 | 0 | 0 |
EZH2 Gene: EZH2 EZH2 is an enzyme that silences genes. The first drug against it treated a rare sarcoma and some lymphomas until it was withdrawn in 2026 for causing second blood cancers. | 3 | 0 | 13 | 3 | 8 | 1 | 0 |
FAK (PTK2) Gene: PTK2 FAK is the kinase that tells a cell it is anchored to its surroundings, letting it survive, move and resist drugs. Defactinib, given with the RAF/MEK inhibitor avutometinib, removes that escape route in low-grade serous ovarian cancer; other FAK inhibitors are in trials in meningioma and solid tumours. | 3 | 1 | 16 | 0 | 0 | 0 | 0 |
GnRH receptor (GNRHR) Gene: GNRHR The pituitary receptor that starts the hormone chain leading to testosterone and oestrogen. Agonists overstimulate it into silence and antagonists block it outright, the basis of medical castration in prostate cancer and ovarian suppression in breast cancer. | 3 | 0 | 10 | 1 | 0 | 0 | 0 |
Histone deacetylases (HDAC) Gene: HDAC1, HDAC2, HDAC3, HDAC6 Histone deacetylases tighten the packaging of DNA so that genes are switched off. Drugs that block them loosen the packaging and can wake up genes that make lymphoma cells stop growing or die. | 3 | 1 | 5 | 0 | 0 | 0 | 0 |
IKZF3 (Aiolos) Gene: IKZF3 Aiolos is Ikaros' partner transcription factor in B cells and plasma cells. The myeloma drugs lenalidomide, pomalidomide and golcadomide destroy Aiolos and Ikaros together by rerouting them to the cell's protein-disposal system. | 3 | 2 | 89 | 0 | 0 | 1 | 0 |
JAK1 Gene: JAK1 JAK1 is one of the kinases that pass cytokine and interferon signals inside immune and blood cells. Ruxolitinib and momelotinib block JAK1 together with JAK2 to calm the inflammation of myelofibrosis; golidocitinib is the first JAK1-only inhibitor approved for a cancer, in peripheral T-cell lymphoma. | 3 | 3 | 20 | 0 | 0 | 0 | 0 |
mTOR Gene: MTOR mTOR is the cell's master growth controller, deciding whether to build proteins and divide. Rapamycin-like drugs clamp it down in kidney, breast and neuroendocrine cancers and in rare tumours driven by TSC gene loss. | 3 | 2 | 7 | 1 | 3 | 0 | 0 |
Retinoid X receptor (RXR) Gene: RXRA, RXRB, RXRG The retinoid X receptor is a switch inside cells that vitamin A-like molecules turn on to change which genes are active; bexarotene uses it to make lymphoma cells in the skin mature and die. | 3 | 3 | 5 | 1 | 2 | 0 | 0 |
TIGIT Gene: TIGIT TIGIT is an inhibitory receptor on T and natural killer cells that binds PVR (CD155) on tumour cells, so blocking it was expected to amplify PD-1 and PD-L1 inhibitors. Tiragolumab, domvanalimab and others then failed to add benefit in phase 3 lung cancer trials despite encouraging phase 2 signals, and the lack of a TIGIT-specific biomarker remains a weakness. | 3 | 0 | 30 | 1 | 1 | 1 | 0 |
ACVR1 (ALK2) Gene: ACVR1 ACVR1 (ALK2) is the receptor that raises hepcidin, the hormone that hides iron from the bone marrow. Momelotinib and pacritinib block it as well as JAK2, which is why they improve, rather than worsen, the low red-cell counts of myelofibrosis. | 2 | 2 | 2 | 0 | 0 | 0 | 0 |
B7-H4 (VTCN1) Gene: VTCN1 B7-H4 is a checkpoint-like protein that many breast, ovarian and endometrial tumours carry on their surface. Antibody-drug conjugates such as puxitatug samrotecan and HS-20089 use it as a docking site to deliver a chemotherapy payload. | 2 | 0 | 4 | 0 | 0 | 0 | 0 |
BCL-2 Gene: BCL2 A protein that stops cells from self-destructing. Venetoclax removes that protection and has transformed leukaemia treatment. | 2 | 2 | 69 | 3 | 12 | 5 | 1 |
Cannabinoid receptor 1 Gene: CNR1 The CB1 cannabinoid receptor is how dronabinol and nabilone, synthetic cannabinoids approved for chemotherapy nausea that other drugs fail to control, act on the brain; their place is behind the 5-HT3 and NK1 antagonists. | 2 | 0 | 2 | 1 | 0 | 0 | 0 |
CD123 Gene: IL3RA CD123 is the interleukin-3 receptor alpha chain, present on every blastic plasmacytoid dendritic cell neoplasm and on the blasts and leukaemia stem cells of 70 to 80% of acute myeloid leukaemias. Tagraxofusp and pivekimab sunirine target it in that rare neoplasm, and CAR-T cells against it in AML must spare the normal blood-forming progenitors that also carry it. | 2 | 2 | 4 | 1 | 0 | 0 | 0 |
CD137 (4-1BB, TNFRSF9) Gene: TNFRSF9 4-1BB is a switch on activated T cells that makes them live longer and kill better. Rather than press it everywhere, new bispecific antibodies press it only on T cells that are already touching a tumour cell marked by PD-L1 or HER2. | 2 | 0 | 3 | 0 | 0 | 0 | 0 |
CD25 (IL-2 receptor alpha) Gene: IL2RA CD25 is the part of the interleukin-2 receptor found on activated T cells and on some lymphoma cells; denileukin diftitox uses it as a doorway to deliver a toxin. | 2 | 1 | 4 | 2 | 0 | 0 | 0 |
CD52 Gene: CD52 CD52 is a small protein on nearly all lymphocytes and the target of alemtuzumab, a profoundly immunosuppressive antibody once used in CLL and now mostly in transplant conditioning, T-cell leukaemia and multiple sclerosis. | 2 | 1 | 2 | 2 | 0 | 0 | 0 |
CD70 Gene: CD70 CD70 is a protein normally only on activated immune cells, but permanently switched on in kidney cancer and some leukaemias. | 2 | 0 | 2 | 2 | 0 | 0 | 0 |
CD73 / adenosine axis Gene: NT5E CD73 is an enzyme on tumour and immune cells that converts AMP into adenosine, which switches off T and natural killer cells through A2A and A2B receptors. Oleclumab (anti-CD73) with durvalumab slowed progression in a phase 2 lung cancer trial and quemliclustat is in phase 3 in pancreatic cancer, but A2A blockers gave only modest signals and several were dropped. | 2 | 0 | 3 | 1 | 1 | 0 | 0 |
CXCR4 Gene: CXCR4 A chemokine receptor that anchors blood cells in the marrow and helps cancer cells home to it; mutated in a third of Waldenström patients and targeted by plerixafor for stem-cell mobilisation. | 2 | 2 | 5 | 1 | 0 | 0 | 0 |
CYP17A1 (17-alpha-hydroxylase/17,20-lyase) Gene: CYP17A1 The enzyme that makes androgens in the testes, the adrenal glands and prostate tumours themselves. Abiraterone blocks it, cutting androgen production below what castration alone achieves. | 2 | 0 | 2 | 1 | 0 | 0 | 0 |
Dihydrofolate reductase (DHFR) Gene: DHFR The enzyme that recycles folate so cells can build DNA. Methotrexate blocks it, which is why folinic acid rescue after high-dose methotrexate matters. | 2 | 0 | 87 | 1 | 0 | 0 | 0 |
Dopamine D2 receptor Gene: DRD2 The dopamine D2 receptor is how cabergoline and bromocriptine shrink prolactin-secreting pituitary tumours without surgery, and it is the target of the antiemetics metoclopramide and prochlorperazine used during chemotherapy. | 2 | 2 | 3 | 1 | 0 | 0 | 0 |
EDNRA Gene: EDNRA EDNRA (Endothelin-1 receptor) is a gene. The public catalogues list it as a drug target, and an approved or late-stage drug is recorded against it. Tied to Prostate cancer. | 2 | 0 | 2 | 0 | 0 | 0 | 0 |
EpCAM Gene: EPCAM An adhesion protein on almost all carcinoma cells and the capture molecule for circulating tumour cell tests; the target of the first bispecific antibody ever approved (catumaxomab, 2009). | 2 | 1 | 4 | 3 | 0 | 0 | 0 |
FAP Gene: FAP FAP (fibroblast activation protein) sits on the cancer-associated fibroblasts that scaffold more than 90% of epithelial cancers and is almost absent from normal adult tissue. FAPI PET tracers therefore light up tumours with high contrast, including pancreatic, gastric and low-grade cancers where FDG PET is weak, and FAP-targeted radioligands are in development. | 2 | 0 | 9 | 4 | 4 | 1 | 0 |
G-CSF receptor (CSF3R) Gene: CSF3R The receptor that tells the bone marrow to make neutrophils. Filgrastim and pegfilgrastim stimulate it to shorten the dangerous low-neutrophil period after chemotherapy, cutting the risk of febrile neutropenia. | 2 | 1 | 4 | 1 | 0 | 0 | 0 |
GD2 (disialoganglioside) Gene: B4GALNT1 GD2 is a sugar-fat molecule on the surface of neuroblastoma cells (and some other childhood and adult tumours) with almost none on normal tissue except nerves, which is why anti-GD2 drugs cause pain. | 2 | 2 | 10 | 1 | 0 | 0 | 0 |
| 2 | 1 | 10 | 1 | 0 | 1 | 0 | |
IDO1 Gene: IDO1 IDO1 is an enzyme tumours use to burn up tryptophan, starving T cells and producing by-products that switch them off. Epacadostat blocked it but its programme ended in failure; the IO102-IO103 vaccine instead teaches T cells to attack the IDO1-expressing cells themselves. | 2 | 0 | 6 | 0 | 0 | 0 | 0 |
Interleukin-6 and IL-6 receptor Gene: IL6, IL6R Interleukin-6 is an inflammation messenger. Blocking it treats Castleman disease, a lymph node disorder, and calms the dangerous immune storm that CAR-T cell therapy can trigger. | 2 | 2 | 18 | 1 | 0 | 0 | 0 |
KMT2A (MLL) rearrangement Gene: KMT2A A gene fusion that drives an aggressive leukaemia in infants and adults. It cannot be blocked directly, but the scaffold protein it depends on (menin) can. | 2 | 2 | 96 | 2 | 0 | 0 | 0 |
MDM2 Gene: MDM2 MDM2 is the protein that degrades p53; blocking it reactivates p53 in tumours where the gene is intact, especially the liposarcomas that carry extra copies of MDM2. | 2 | 0 | 5 | 2 | 6 | 0 | 0 |
Menin Gene: MEN1 A scaffold protein that certain leukaemias need to keep their genes switched on; the first drug against it was approved in 2024. | 2 | 2 | 6 | 2 | 3 | 0 | 1 |
Mesothelin Gene: MSLN Mesothelin is a surface protein on mesothelioma, ovarian, and pancreatic cancers, targeted by ADCs, CAR-T, and T-cell engagers. | 2 | 0 | 9 | 3 | 0 | 0 | 0 |
MPL (thrombopoietin receptor) Gene: MPL MPL is the receptor that tells the bone marrow to make platelets. Romiplostim and eltrombopag switch it on to raise platelet counts; in some myeloproliferative neoplasms a mutant partner protein, calreticulin, grips it and keeps it on. | 2 | 0 | 4 | 0 | 0 | 0 | 0 |
Mu-opioid receptor Gene: OPRM1 The mu-opioid receptor is the target of morphine and the other strong opioids that control most severe cancer pain; the same receptor in the gut causes opioid constipation, which methylnaltrexone and naloxegol relieve by blocking it outside the brain. | 2 | 2 | 2 | 1 | 0 | 0 | 0 |
PRAME Gene: PRAME PRAME is a cancer-testis antigen: a protein normally confined to the testis that about 90% of cutaneous melanomas and substantial fractions of ovarian, lung, endometrial and uveal cancers switch on. Because it sits inside the cell, drugs reach it only as peptide fragments displayed on HLA, through T-cell receptor bispecifics such as brenetafusp and TCR-T cells such as IMA203. | 2 | 0 | 4 | 1 | 0 | 0 | 0 |
Retinoic acid receptor alpha (RARA) Gene: RARA The retinoic acid receptor that, fused to PML in acute promyelocytic leukaemia, blocks white blood cells from maturing. All-trans retinoic acid releases the block and arsenic trioxide destroys the fusion protein, turning a once rapidly fatal leukaemia into one of the most curable. | 2 | 2 | 4 | 1 | 0 | 0 | 0 |
TIM-3 Gene: HAVCR2 An immune checkpoint on exhausted T cells and on leukaemic stem cells; antibodies against it failed in lung cancer and MDS after strong preclinical promise. | 2 | 0 | 2 | 1 | 0 | 0 | 0 |
WRN helicase (MSI-high cancers) Gene: WRN A DNA-unwinding enzyme that mismatch-repair-deficient cancers cannot live without; the first WRN inhibitors are in trials as a chemotherapy-free option for MSI-high tumours that fail immunotherapy. | 2 | 0 | 2 | 3 | 2 | 0 | 0 |
5-HT3 receptor (HTR3A) Gene: HTR3A The serotonin receptor on the gut's vagus nerve endings and in the brainstem vomiting centre that chemotherapy triggers. Ondansetron and palonosetron block it, the foundation of modern anti-sickness treatment. | 1 | 0 | 0 | 1 | 0 | 0 | 0 |