Key points
- Not every cancer has the same treatment. In some cancers a targeted pill or immunotherapy works better, with fewer side effects, than chemotherapy [2, 3, 4].
- The key is a test on the tumour sample. It shows which medicine will help you. The same pill that is excellent for one patient did worse than chemotherapy for another [2].
- Chemotherapy is still needed in many cancers, and sometimes the best treatment is chemotherapy together with a new drug [11, 14].
- Before treatment starts, ask: "Has my tumour been tested for markers?"
How do these treatments differ?
Chemotherapy
Medicines that kill cells that divide quickly. That is why they also harm busy healthy cells such as hair, mouth and bone marrow (chemotherapy side effects (Persian)).
Targeted therapy
A medicine that attacks a specific change in the cancer cell. It only works if the tumour has that change (What is targeted therapy? (Persian)).
Immunotherapy
A medicine that wakes up the body's immune system so it recognises the cancer and fights it (What is immunotherapy? (Persian)).
Hormone therapy
In cancers that grow with hormones (such as most breast and prostate cancers), it blocks the effect of the hormone.
The story of a big change
Until a few decades ago, chemotherapy was almost the only weapon doctors had against cancers that could not be removed by surgery. Chemotherapy hits every cell that divides quickly, healthy or cancerous; that is why it has many side effects, and in many cancers it only held the disease back for a while.
From around 2000, scientists understood that in many cancers a specific "switch" (a faulty gene or protein) drives the tumour's growth. Drugs were made that turn off exactly that switch. In several cancers the result was so large that chemotherapy is no longer the first treatment. Below you can read the story of some of these diseases.
Chronic myeloid leukaemia (CML): from a deadly disease to an almost normal lifespan
With the old treatments (interferon and chemotherapy), CML could only be controlled for a while. In 2001 came imatinib (Persian) tablets, which switch off the faulty protein of this disease. In a study comparing it with the best treatment of the time, imatinib was far better, and after 10 years about 83 out of every 100 patients were alive [5].
Today the life expectancy of people with CML is close to that of the general population; in Sweden, patients diagnosed in 2013 lose on average fewer than 3 years of life because of the disease [27]. The first treatment for CML today is a tablet, not chemotherapy.
Chronic lymphocytic leukaemia (CLL): when chemotherapy stepped aside
Until about 2015, the best treatment for fit patients with CLL was a combination of three chemotherapy and immune drugs called FCR; in its day this was a big advance and helped patients live longer [18]. But it was heavy and caused more infections and low blood counts [18].
Then the targeted tablets arrived. In patients aged 70 or younger, ibrutinib (Persian) tablets with an antibody controlled the disease much better than FCR, and after 3 years about 99 out of 100 patients vs 92 were alive [6, 19]. In older patients, too, ibrutinib was much better than oral chemotherapy [7]. One year of venetoclax (Persian)-based treatment also did better than chemotherapy in fit patients; after 3 years, with venetoclax, an antibody and ibrutinib, the disease was controlled in about 90 out of 100 vs 75 with chemotherapy [20].
Today the European guidelines build first-line treatment of CLL on these targeted drugs [28]. If you have been offered only chemotherapy for CLL, ask why.
Acute promyelocytic leukaemia (APL): from the deadliest leukaemia to treatment without chemotherapy
APL is a type of leukaemia that starts with severe bleeding and can kill within days. In 1988, doctors in Shanghai gave tretinoin (a vitamin A derivative) to 24 patients; all of them went into remission, without their bone marrow being wiped out [21]. Later, arsenic was added.
Today the combination of tretinoin and arsenic, with no chemotherapy at all, is better than the old treatment: after 2 years, 97 out of 100 vs 86 were alive without relapse [9]. The main danger now is starting late: in the US, 17 out of every 100 patients died in the first days, and the researchers said the doctors who see these patients first need to know this disease better [22]. That is why guidelines say tretinoin must be started the moment this disease is suspected [29].
Acute myeloid leukaemia in older people: a treatment that did not exist before
Many older people with AML cannot tolerate intensive chemotherapy and used to receive only a mild drug. Adding venetoclax tablets to that mild drug (azacitidine) helped these patients live longer [8].
Lung cancer: one mutation, one tablet
In the early 2000s, with the best chemotherapy, half of patients with advanced lung cancer lived less than 8 months, and after 2 years only about 1 in 10 was alive [23]. In 2004 it was found that gefitinib tablets worked dramatically in a small group of patients: those whose tumour has an EGFR mutation [24].
From then on, in these patients the targeted tablet beat chemotherapy, but in patients without the mutation the same tablet did worse than chemotherapy [2]; so testing for the mutation is the key. With the newer tablet osimertinib (Persian), half of these patients lived more than 3 years [25]. In patients without the mutation, immunotherapy also helped people with high-PD-L1 tumours live longer than chemotherapy [3].
HER2-positive breast cancer: from the worst type to one of the best
In 1987 it was shown that about 3 in 10 breast cancers have a lot of the HER2 protein, and these patients relapse sooner and die sooner [26]. The antibody trastuzumab (Persian) was made to switch off exactly this protein. Adding it to chemotherapy helped patients with advanced disease live longer [13], and after surgery it lowered the risk of death by about a third [14]. Here the drug did not replace chemotherapy; it joined it and transformed the result.
A treatment that was the best 15 or 20 years ago may no longer be the best today. If you or someone you love, especially at a young age, has one of these diseases (CML, CLL, APL, AML in older age, lung cancer, HER2-positive breast cancer) and has been offered only chemotherapy:
- Ask whether genetic or marker tests have been done on the tumour.
- Ask whether targeted therapy or immunotherapy is suitable for you.
- If you do not get a clear answer, get a second opinion from a haematologist-oncologist.
Do not stop treatment on your own; raise these questions with your doctor.
One important step: ask these 3 questions before treatment starts
- 1
"Has a marker (molecular) test been done on my tumour sample?"
The European Society for Medical Oncology (ESMO) recommends this test in advanced cancers of the lung, prostate, bowel, ovary, bile ducts and breast [1]. The sample is the piece taken at biopsy or surgery; a new biopsy is often not needed.
- 2
"With this result, is there a treatment better than chemotherapy for me?"
The stories above show examples where a non-chemotherapy treatment did better in careful studies.
- 3
"If chemotherapy is needed, will another drug be added to it?"
For example, in HER2-positive breast cancer, adding trastuzumab to chemotherapy lowered the risk of death by about a third [14].
Which test for which cancer?
| Cancer | Test on the tumour sample or blood | Why it matters |
|---|---|---|
| Lung cancer (non-small-cell) | EGFR, ALK and PD-L1 (and broad genetic testing where possible) | An EGFR mutation means a targeted pill instead of chemotherapy [2]; high PD-L1 means immunotherapy [3] |
| Advanced bowel cancer | MMR or MSI | In MSI-H, immunotherapy did better than chemotherapy [4] |
| Breast cancer | Hormone receptors, HER2 and, in many patients, BRCA | Shows whether hormone therapy, an anti-HER2 drug or a BRCA-specific drug is needed [10, 14, 17] |
| Chronic myeloid leukaemia (CML) | BCR-ABL (Philadelphia chromosome) | Imatinib tablets are the main treatment [5] |
| Gastrointestinal stromal tumour (GIST) | KIT | Imatinib tablets shrank or stopped most advanced tumours [15] |
| Advanced ovarian and prostate cancer | BRCA and similar genes | With a BRCA mutation, PARP inhibitor drugs are used [1] |
There are other tests too; your doctor decides based on the type and stage of the cancer.
Where has targeted therapy or immunotherapy beaten chemotherapy?
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| Cancer | Better treatment | Compared with | What was seen |
|---|---|---|---|
| Chronic myeloid leukaemia (CML) | Imatinib tablets | Older treatments | Out of every 100 patients, about 83 were alive after 10 years [5] |
| Chronic lymphocytic leukaemia (CLL) | Ibrutinib tablets or venetoclax regimens | Chemotherapy | After 3 years, about 99 out of 100 vs 92 were alive [6]; the disease progressed much later [7] |
| Acute myeloid leukaemia in older patients or those who cannot tolerate intensive chemotherapy | Venetoclax + azacitidine | Azacitidine alone | Half of patients lived more than 14.7 months, vs 9.6 months [8] |
| Acute promyelocytic leukaemia (APL) | Tretinoin + arsenic trioxide | Tretinoin + chemotherapy | After 2 years, 97 out of 100 vs 86 were alive without relapse [9] |
| Lung cancer with an EGFR mutation | Gefitinib or osimertinib tablets | Chemotherapy | The risk of the disease progressing was roughly halved [2]; osimertinib was also better than the older generation [12] |
| Lung cancer with high PD-L1 (50% or more) | Pembrolizumab | Chemotherapy | 40% lower risk of death [3] |
| Advanced bowel cancer with MSI-H | Pembrolizumab | Chemotherapy | The disease progressed about twice as late; severe side effects in 22 out of 100 vs 66 [4] |
| Advanced hormone receptor-positive breast cancer | Hormone therapy + a CDK4/6 inhibitor | Combination chemotherapy | The disease was controlled nearly twice as long (about 22 vs 13 months), even in severe disease, with fewer side effects [10] |
This list is not complete. The choice of treatment depends on the stage of the disease, your situation and the test results.
Where does a targeted drug go together with chemotherapy?
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Sometimes the drug does not replace chemotherapy but is added to it and makes the result much better:
- HER2-positive breast cancer: adding trastuzumab to chemotherapy after surgery lowered the risk of death by 33% [14]. In advanced disease, deaths in the first year fell from 33% to 22% [13].
- Early triple-negative breast cancer: adding immunotherapy (pembrolizumab) to chemotherapy before surgery improved results [11].
- GIST after surgery: 3 years of imatinib tablets instead of 1 year raised 5-year survival from 82% to 92% [16].
Where is chemotherapy still needed?
Targeted therapy only works when the tumour has the "target". In a large study, in lung cancer patients without an EGFR mutation, chemotherapy was much better than the targeted pill; with the pill, the risk of the disease progressing nearly tripled [2]. In many cancers chemotherapy is still the basis of treatment [11]. So "newer" does not always mean "better"; the better treatment is the one that matches your tumour.
When should I contact a doctor?
Do not stop or change treatment on your own; discuss your questions with your doctor. Targeted drugs have side effects too; for example, in one study more than half of the patients taking a targeted lung cancer pill had an acne-like rash and nearly half had diarrhoea [2]. Report fever, severe diarrhoea, breathlessness or any new side effect to the care team early.
Frequently asked questions
Does targeted therapy mean a cure?
Not always. In some diseases, such as chronic myeloid leukaemia, patients live normal lives for years on tablets [5]. In many others, the drug controls the disease for a time, and then the next drug may be needed.
Does every cancer have a targeted therapy?
I have already started chemotherapy. Is it too late?
Not necessarily. The tumour test result is also useful at later stages of treatment. Ask your doctor whether your earlier sample has been tested.
Do targeted drugs have no side effects?
Read more
- Complete, on-time, uninterrupted treatment: why delays and missed medicine cost lives
- Late arrival costs lives: finding cancer early in Iran
- 10 cheap steps that save lives (for physicians)
For doctors: before prescribing chemotherapy (for physicians)
Show detailsHide
Test before deciding. ESMO 2024 recommends tumour NGS in advanced non-squamous NSCLC, prostate, colorectal, cholangiocarcinoma, ovarian and now advanced breast cancer [1]. If NGS is not available, at least the single-gene tests that change first-line treatment (EGFR, ALK and PD-L1 in lung; MMR/MSI in colorectal; HER2 and BRCA in breast; BRCA in ovarian and prostate) should be done before treatment starts.
Key evidence. IPASS: progression HR 0.48 favouring gefitinib in EGFR-positive disease and HR 2.85 against it in EGFR-negative disease [2]. KEYNOTE-024 (PD-L1 ≥50%): PFS 10.3 vs 6.0 months, HR for death 0.60 [3]. KEYNOTE-177: progression HR 0.60; grade 3 or higher adverse events 22% vs 66% [4]. E1912 (CLL, age 70 or younger): 3-year overall survival 98.8% vs 91.5% with ibrutinib–rituximab vs FCR (HR 0.17) [6]. VIALE-A: HR for death 0.66 [8]. RIGHT Choice (premenopausal, aggressive disease, 48% visceral crisis): progression HR 0.61 with fewer symptomatic adverse events [10]. NSABP B-31/N9831: adjuvant trastuzumab, DFS HR 0.48 and a 33% reduction in the risk of death [14]. SSG XVIII: 3 vs 1 year of adjuvant imatinib in high-risk GIST, HR for death 0.45 [16].
Scientific evidence
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Scientific evidence
11 itemsLong-Term Outcomes of Imatinib Treatment for Chronic Myeloid Leukemia
Ibrutinib as Initial Therapy for Patients with Chronic Lymphocytic Leukemia
Pembrolizumab versus Chemotherapy for PD-L1–Positive Non–Small-Cell Lung Cancer
Trastuzumab plus Adjuvant Chemotherapy for Operable HER2-Positive Breast Cancer
One vs Three Years of Adjuvant Imatinib for Operable Gastrointestinal Stromal Tumor
Recommendations for the use of next-generation sequencing (NGS) for patients with advanced cancer in 2024: a report from the ESMO Precision Medicine Working Group
Retinoic acid and arsenic trioxide for acute promyelocytic leukemia
Ibrutinib–Rituximab or Chemoimmunotherapy for Chronic Lymphocytic Leukemia
Azacitidine and Venetoclax in Previously Untreated Acute Myeloid Leukemia
Osimertinib in Untreated EGFR -Mutated Advanced Non–Small-Cell Lung Cancer
Pembrolizumab in Microsatellite-Instability-High Advanced Colorectal Cancer
References
29 referencesShow all references, 20 with a study cardClose the reference list
Numbers in the text, such as [1], refer to these sources. Sources with a “Study card” button are summarised in the site’s evidence library.
- 1
- 2
- 3
- 4
- 5
- 6
- 7Ibrutinib as Initial Therapy for Patients with Chronic Lymphocytic Leukemia
- 8
- 9Retinoic acid and arsenic trioxide for acute promyelocytic leukemia
- 10
- 11
- 12Osimertinib in Untreated EGFR -Mutated Advanced Non–Small-Cell Lung Cancer
- 13Use of Chemotherapy plus a Monoclonal Antibody against HER2 for Metastatic Breast Cancer That Overexpresses HER2
- 14
- 15Efficacy and Safety of Imatinib Mesylate in Advanced Gastrointestinal Stromal Tumors
- 16One vs Three Years of Adjuvant Imatinib for Operable Gastrointestinal Stromal Tumor
- 17Adjuvant Olaparib for Patients with BRCA1- or BRCA2-Mutated Breast Cancer
- 18Addition of rituximab to fludarabine and cyclophosphamide in patients with chronic lymphocytic leukaemia: a randomised, open-label, phase 3 trial
- 19Long-term outcomes for ibrutinib-rituximab and chemoimmunotherapy in CLL: updated results of the E1912 trial
- 20First-Line Venetoclax Combinations in Chronic Lymphocytic Leukemia
- 21Use of all-trans retinoic acid in the treatment of acute promyelocytic leukemia
- 22Early death rate in acute promyelocytic leukemia remains high despite all-trans retinoic acid
- 23Comparison of four chemotherapy regimens for advanced non-small-cell lung cancer
- 24Activating mutations in the epidermal growth factor receptor underlying responsiveness of non-small-cell lung cancer to gefitinib
- 25Overall Survival with Osimertinib in Untreated, EGFR-Mutated Advanced NSCLC
- 26Human breast cancer: correlation of relapse and survival with amplification of the HER-2/neu oncogene
- 27Life Expectancy of Patients With Chronic Myeloid Leukemia Approaches the Life Expectancy of the General Population
- 28EHA Guidelines on management of chronic lymphocytic leukemia and Richter transformation
- 29Management of acute promyelocytic leukemia: updated recommendations from an expert panel of the European LeukemiaNet
This page is based on reputable scientific sources and was last updated on 27 October 2026. The content of this website is for educational and informational purposes only and does not replace consultation, diagnosis or treatment by a specialist physician. Consult your treating physician about any treatment decision.