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Targeted therapies are drugs designed to switch off a specific broken protein that a cancer depends on.
Small-molecule kinase inhibitors, monoclonal antibodies, hormone-pathway agents, PARP inhibitors, degraders (PROTACs, molecular glues), and synthetic-lethality approaches. Matched to a molecular alteration by a companion diagnostic. Resistance is the central problem; next-generation and combination strategies are the response.
Anti-angiogenic therapy cuts off the tumour's blood supply; it is now mostly used to help immunotherapy work better.
An ADC that carries a gene-silencing strand instead of a chemotherapy, so it can switch a protein off rather than poison the cell.
Drugs that disable the checkpoint cancer cells use to pause and repair DNA under replication stress, pushing tumours with faulty DNA repair into collapse.
Drugs that switch off BCL-2, the protein that lets cancer cells refuse to die; venetoclax transformed treatment of chronic lymphocytic leukaemia and acute myeloid leukaemia.
A bispecific antibody is one antibody with two different grabbing arms, so it can block two targets at once or pull an immune cell onto a cancer cell.
Tumours recruit nerves and use nerve signals to grow. Blocking that traffic, with beta-blockers or botulinum toxin, is being tested.
Pills that stop the cell-division engine, added to hormone therapy for the most common type of breast cancer.
Next-generation relatives of lenalidomide that make the cell's disposal machinery destroy the myeloma proteins Ikaros and Aiolos more completely, working after older drugs fail.
Designing a protein from scratch on a computer to grip a chosen target, instead of finding one in an animal or a library.
An ADC that delivers a protein-destroying molecule instead of chemotherapy, hitting targets inside the cell that were previously unreachable.
Folded DNA machines that open only when they touch a tumour, releasing a payload or clotting the tumour's blood supply.
Loading the tiny vesicles cells naturally use to talk to each other with a cancer drug, so the body treats the carrier as its own.
Switching a gene off for good without changing the DNA sequence, by writing chemical marks onto it.
Drugs that block the enzyme that activates Notch signalling; nirogacestat became the first approved, in 2023, for desmoid tumours, a rare but destructive growth of connective tissue.
Pills that block a developmental signalling pathway hijacked by basal cell skin cancer, shrinking tumours that cannot be operated on; also used in some leukaemia.
Small pills that block HER2 from inside the cell, complementing antibody drugs; tucatinib is notable for working against brain metastases.
A harmless molecule that turns into a poison only where there is no oxygen, which in the body means inside a tumour.
Pills that block mutant IDH1 or IDH2 enzymes, which flood cells with a metabolite that blocks maturation; approved in leukaemia, bile duct cancer and low-grade glioma.
In vivo base and prime editing would rewrite a cancer's DNA letter by letter inside the body. It works in the liver for inherited disease; nobody has yet corrected a cancer this way in a person.
First-in-class drugs against the histone acetyltransferases KAT6A and KAT6B, in late trials for hormone receptor-positive breast cancer that has stopped responding to endocrine therapy.
Drugs against the most common cancer gene, considered impossible to target until sotorasib in 2021.
Cells or drugs that fire only when two conditions are true at once, so healthy tissue expressing just one of them is spared.
Drugs against the histone demethylase LSD1, which keeps blood and neuroendocrine cancer cells from maturing; being tested in myelofibrosis, leukaemia and small cell lung cancer.
Drugs that stop MDM2 destroying p53, reawakening the cell's guardian protein in tumours where p53 is intact but suppressed, such as some sarcomas and leukaemias.
Pills that break the interaction between menin and the KMT2A protein that acute leukaemias with KMT2A rearrangements or NPM1 mutations depend on; revumenib was the first approved, in 2024.
Removing an amino acid or nutrient that certain tumours cannot make for themselves, while normal cells can.
Molecular glues are small molecules that stick two proteins together so the cell destroys one of them. They are smaller and more drug-like than bifunctional degraders.
Lab-made immune proteins that lock onto one target, either blocking it or flagging the cell for destruction.
Oligonucleotide therapeutics are short synthetic strands of genetic code that silence a specific cancer gene.
Oral drugs that destroy the oestrogen receptor rather than just blocking it, working even when the receptor has mutated to escape older hormone therapies.
Most targeted cancer drugs are tablets. Turning a powder into a tablet that dissolves the same way every time is its own craft, and when patents end the same craft lets generic makers sell the drug for a fraction of the price.
Pills that block a DNA repair backup, killing cancer cells that already lost their main repair system (BRCA).
Drugs against one of the most commonly mutated growth pathways in cancer, now used with hormone therapy in breast cancer and in kidney and neuroendocrine tumours, with high blood sugar as the shared side effect.
Programmable DNA-targeting therapeutics are an experimental idea: a drug that reads a cell's DNA, recognises a cancer-specific sequence, and kills only cells that carry it. Change the guide, and the same drug becomes a new drug.
Instead of blocking a protein, these drugs tag it for the cell's own garbage disposal, removing it entirely.
Adding a PARP or ATR inhibitor to a radioactive drug so the tumour cannot repair the damage the radiation causes.
Chemotherapy leaves behind zombie cells that will not divide but poison their neighbours. Senolytics aim to clear them.
Before a tablet or a vial exists, the active drug itself has to be made: many chemical steps in reactors, or extraction from a plant, at a handful of factories most patients never hear of. When one of those factories stops, whole cancer drugs disappear.
Pills that block the specific enzyme a cancer relies on. Imatinib in 2001 proved a cancer could be switched off by design.
Finding a second gene that a cancer needs only because its first gene is broken, then hitting the second one.
Some tumours contain bacteria and fungi that shelter cancer cells and break down chemotherapy. Killing them may make treatment work.
Stiff, high-pressure tumours squeeze their own blood vessels shut, keeping drugs out. Softening them is a way in.
The 48 most recent of 52 papers; see them all →
A second randomised confirmation that adding a Bruton tyrosine kinase inhibitor to first-line bendamustine-rituximab delays progression in older patients with mantle cell lymphoma, with a toxicity profile that did not improve as much as the drug's selectivity promised.
The authors' conclusion is that ibrutinib-rituximab should be considered a new standard-of-care option for first-line treatment of older patients with mantle-cell lymphoma. The subgroup split means it is clearly better than R-CHOP and roughly equivalent to bendamustine-rituximab.
Adjuvant treatment for lung cancer is now chosen by genotype. A resected ALK-positive tumour is treated with two years of a tablet instead of four cycles of platinum, which is a different life as well as a different outcome.
Patients newly diagnosed with EGFR-mutated advanced lung cancer now have a first-line option that improves survival over osimertinib, particularly if they have high-risk features. The trade-off is intravenous (now subcutaneous) infusions and considerably more skin, nail and clotting toxicity, so osimertinib alone remains reasonable for those who prioritise convenience and tolerability. Both this regimen and osimertinib plus chemotherapy (FLAURA2) are approved; there is no direct comparison.
Stage III lung cancer is now treated by genotype as well as by stage: an EGFR mutation moves a patient from durvalumab consolidation to osimertinib consolidation. It is also the strongest hazard ratio in the lung cancer literature, which is a reason to read the overall survival data carefully when they arrive.
Patients with KRAS G12C lung cancer that has progressed after chemo-immunotherapy can take an oral KRAS inhibitor instead of docetaxel and gain a somewhat longer time to progression with fewer severe side effects, but should understand that most tumours become resistant within a year and that survival is not improved. KRAS G12C testing is worthwhile, but first-generation inhibitors are a step rather than a cure; combinations and next-generation inhibitors are the active research fronts.
Patients with metastatic colorectal cancer carrying a KRAS G12C mutation (about 3-4% of cases) who have exhausted standard chemotherapy now have a targeted option that works far better than trifluridine-tipiracil or regorafenib. The higher sotorasib dose is clearly superior, and the EGFR antibody is essential because KRAS inhibition alone has little effect in bowel cancer. Responses are still modest and short-lived compared with EGFR or ALK inhibitors in lung cancer.
A third refractory-line option, and the clearest example in colorectal cancer of a drug developed and approved in China going on to a global registration trial.
Open-source software, hardware and data projects catalogued by a third party, the Open Medical Registry, that bear on this front. Listing is not endorsement; check each project's own licence and validation before clinical use.
CancerFoundation: A single-cell RNA sequencing foundation model to decipher drug resistance in cancer
Synthetic lethality (SL) is a promising gold mine for the discovery of anti-cancer drug targets.
Open-source precision oncology platform that ranks FDA-approved and repurposing drug candidates from a patient's variant profile using OncoKB...
Targeted and non-targeted anticancer drugs and drug regimens
From the Open Medical Registry (openmedical.sh), an MIT-licensed catalogue of open-source medicine. Blurbs are one line from each registry record; every project keeps its own licence.