Small-molecule targeted drugs
Enter cells and inhibit proteins such as EGFR, ALK, BRAF, KRAS, PARP or kinases driving abnormal growth.
- Needs
- A validated target and a drug able to reach it
- Watch
- Resistance mutations, interactions, organ toxicity
The treatment frontier
Follow the target, the evidence and the person, not the headline. Explore modern cancer medicines, why they work, why they stop working and what to ask next.
What has changed
Chemotherapy, surgery and radiotherapy remain essential. The newer landscape adds medicines aimed at molecular drivers, immune checkpoints, cell-surface targets and radioactive delivery routes.
Two people with the same cancer name may receive different drugs. One drug may work across several organs when they share a biomarker. Precision is improving, but most treatments still help only a proportion of eligible patients.
Six treatment systems
These categories overlap. Modern regimens often combine mechanisms to deepen response or delay resistance.
Enter cells and inhibit proteins such as EGFR, ALK, BRAF, KRAS, PARP or kinases driving abnormal growth.
Bind targets on cancer cells or in their environment. Some block growth signals; others recruit immune destruction.
An antibody carries a potent payload toward cells displaying a target. The promise is selective delivery, not zero collateral damage.
Checkpoint inhibitors, bispecific antibodies and cellular therapies help immune cells recognize or attack cancer.
A targeting molecule carries a therapeutic isotope to cancer cells, combining molecular recognition with local radiation.
Block a backup pathway a cancer depends on, or attack several escape routes together.
Interactive preparation tool
Select what is known. This produces discussion prompts, not a drug recommendation or eligibility decision.
Your discussion prompts will appear here.
Important: a reported mutation may not be driving the cancer; an approved drug may not apply to that tumour, stage or treatment line; and a negative blood test can miss alterations found in tissue.
Headline decoder
A molecule killing cancer cells in a dish is interesting science. It is not evidence that the molecule safely treats cancer in a person.
Where innovation is moving
Promising mechanisms still need trials showing that patients live longer or better, not merely that tumours shrink.
New inhibitors and protein degraders are expanding the set of reachable cancer drivers, including specific KRAS states and transcriptional machinery.
Next-generation ADCs, conditionally active antibodies and nanoparticles aim to release treatment mainly inside or near tumour tissue.
Engineered molecules bring immune cells to tumours; CAR-T, TCR and tumour-infiltrating lymphocyte approaches are moving beyond blood cancers.
Neoantigen vaccines are being tested with checkpoint blockade and other therapies to amplify tumour-specific immune responses.
Alpha emitters and new ligands may deliver higher-energy treatment across more tumour types while limiting exposure elsewhere.
Serial tissue, liquid biopsy and response monitoring may help change treatment as resistant cancer populations emerge.
A medicine with years of safety data may gain a new cancer indication, dose, formulation or combination. Thalidomide-related drugs in myeloma and arsenic trioxide in acute promyelocytic leukaemia show how unusual histories can lead to established oncology roles.
Cheap familiar medicines such as metformin, statins, aspirin or antiparasitic drugs often generate headlines. Routine cancer use still requires convincing human trials for a defined cancer and setting.
Biomarkers may reveal that an existing drug works in another tumour type. Tumour-agnostic approvals formalise this when the molecular feature matters more than the organ of origin.
“Old” and “new” are not measures of quality. The useful question is whether the treatment improves a meaningful outcome for people like the patient considering it, at an acceptable burden and risk.
Why good drugs stop working
Treatment removes sensitive cells while resistant clones may survive. They can alter the target, activate a bypass pathway, hide from immunity or change cellular identity.
Reading an approval
Accelerated approval and surrogate endpoints can bring earlier access, but confirmatory evidence still matters.
Do people live longer? Crossover and later treatment can complicate interpretation.
How long before imaging or clinical progression? A statistical gain may or may not feel meaningful.
How many tumours shrink by a defined amount? Also examine response duration.
Symptoms, function and time without severe toxicity can matter as much as scan measurements.
Cost is a treatment effect
Prices, public reimbursement, insurance rules and compassionate-access programs differ sharply by country and indication. Hospital administration, testing, travel and time away from work add to the drug price.
NCI guide to cancer costsAsk for the expected personal cost, required biomarker test, treatment duration and whether an equally effective lower-cost option exists.
Ask for absolute expected benefit, evidence in your exact setting, likely toxicities and what happens if treatment is stopped early.
Ask about appeal, multidisciplinary review, another funded regimen, manufacturer support, expanded access and suitable trials.
For advanced or resistant cancer
For many people with stage III or IV, recurrent, rare or treatment-resistant cancer, a suitable clinical trial may be the most direct route to a new drug. Ask before another treatment makes you ineligible or too unwell to participate.
Usually establishes dose, safety and biological activity. Potential benefit is possible but uncertain; newer is not necessarily better.
Looks for meaningful activity in a defined cancer or biomarker group and continues safety assessment.
Compares a new strategy with current care, often through randomisation, to test benefit and harms more reliably.
Basket trials group cancers by a shared molecular target; umbrella trials test several matched options within one cancer type.
A trial is not guaranteed access or benefit. Eligibility can depend on exact tumour biology, previous treatment, organ function, blood counts, performance status, measurable disease and timing. Travel, scans and routine-care costs may not all be paid by the sponsor.
Regulatory sources
What this site will not promote
No herb, supplement, special diet or “natural” protocol has been shown to cure cancer. Laboratory mechanisms, testimonials and claims that conventional doctors are hiding a cure are not clinical evidence.
NCI: diets, supplements and cancerSome carefully evaluated approaches may help symptoms, stress, function or quality of life alongside oncology care. They must not replace treatment shown to improve outcomes.
Herbs, concentrated extracts and supplements can change drug absorption or metabolism. St John’s wort, grapefruit and green-tea extracts are among products that can interact with particular cancer medicines.
Paclitaxel, vincristine and other medicines have natural origins, but clinical use relies on purified compounds, controlled doses, manufacturing standards and human trials.
A therapy may help nausea, pain, sleep or anxiety without treating the cancer itself. The site will label that distinction plainly and link to the underlying evidence.
Connected cancer guides
A-Z medicine appendix
These are representative medicines discussed on this site, not every cancer drug. Official US labeling is linked through DailyMed; indications and brands differ by country. Trial medicines have no approved product insert.
Read the exact product label you receive. Formulation, dose, indication and warnings can differ between brands, biosimilars and countries. The oncology team and pharmacist should check interactions, organ function, pregnancy risk and monitoring.
Evidence library