subtype-trials

ABC-06 was the first randomised trial to show that a second round of chemotherapy, FOLFOX, helps people with bile duct and gallbladder cancer live somewhat longer once gemcitabine and cisplatin have stopped working.

ALCANZA showed that brentuximab vedotin produced lasting improvement in the skin disease of far more people with CD30-positive cutaneous T-cell lymphoma than the standard tablets methotrexate or bexarotene.

APL0406 showed that acute promyelocytic leukaemia can be cured without conventional chemotherapy: retinoic acid plus arsenic trioxide did at least as well as, and in fact better than, the standard chemotherapy-based treatment.

AQUILA showed that treating high-risk smouldering myeloma with the antibody daratumumab, rather than waiting for it to turn into active disease, delayed that progression, making it the first drug shown in a randomised trial to hold back myeloma before it causes symptoms.

ARAMIS showed that darolutamide, an androgen receptor blocker that barely enters the brain, delayed metastases and lengthened life in men whose prostate cancer was rising on hormone therapy but not yet visible on scans, with side effects close to placebo.

ARANOTE showed that darolutamide added to hormone therapy alone, without chemotherapy, delayed the progression of newly metastatic prostate cancer on scans, extending the drug's use to men who cannot or do not want to have docetaxel.

ASPEN compared two BTK inhibitors head to head in Waldenström macroglobulinaemia: zanubrutinib did not produce statistically more deep responses than ibrutinib, but it caused far fewer heart rhythm problems and other side effects, which is why many guidelines now prefer it.

ATEMPT tested whether a year of the antibody-drug conjugate T-DM1 could replace chemotherapy plus trastuzumab for the smallest HER2-positive breast cancers; almost no one relapsed on T-DM1, but it was not less toxic overall, so it is an alternative rather than a replacement.

ATTRACTION-2 was the first randomised trial to show that an immunotherapy drug, nivolumab, helps people with heavily pretreated stomach cancer live longer than placebo, and it made nivolumab a late-line option in East Asia before any Western approval.

AUGMENT showed that pairing rituximab with the immune-modulating tablet lenalidomide kept relapsed follicular lymphoma under control for much longer than rituximab alone, giving patients a chemotherapy-free option.

AVAglio tested adding the anti-blood-vessel drug bevacizumab to standard radiotherapy and chemotherapy for newly diagnosed glioblastoma; the tumours took longer to grow on scans, but people did not live any longer, so bevacizumab is not used up front for this cancer.

AZA-001 was the trial that showed a drug could help people with higher-risk myelodysplastic syndromes live longer: azacitidine beat the best conventional care, and it has been the backbone of treatment for this disease ever since.

BOLERO-2 showed that adding the mTOR inhibitor everolimus to the hormone drug exemestane more than doubled the time that advanced breast cancer stayed under control after aromatase inhibitors had stopped working, the first targeted drug to overcome endocrine resistance.

BrighTNess showed that adding the platinum drug carboplatin to standard chemotherapy before surgery makes triple-negative breast tumours vanish completely more often, and later that fewer people relapsed, while the PARP inhibitor veliparib added on top made no difference.

CABONE showed that the tablet cabozantinib could shrink or hold back both Ewing sarcoma and osteosarcoma that had come back after standard chemotherapy, the first drug to show meaningful activity in both bone cancers.

CALLA tested adding the PD-L1 antibody durvalumab to chemoradiotherapy for locally advanced cervical cancer and found no significant benefit, in contrast to the later positive KEYNOTE-A18 trial of pembrolizumab in a higher-risk population.

CARMENA showed that removing the kidney before starting sunitinib did not help people with kidney cancer that had already spread live longer, which ended the routine practice of upfront nephrectomy for intermediate- and poor-risk patients.

CARTITUDE-6 asks whether a single CAR-T infusion can replace the stem cell transplant that has anchored myeloma treatment for thirty years, when both follow the same four-drug induction.

CASSIOPEIA showed that adding the CD38 antibody daratumumab to a three-drug induction before stem cell transplant put more people with myeloma into deep remission and kept the disease away for longer.

CheckMate 025 was the trial that brought immunotherapy to kidney cancer: nivolumab helped people live longer than everolimus after anti-angiogenic drugs had failed, with fewer side effects.

CheckMate 227 showed that a chemotherapy-free pair of immunotherapy drugs, nivolumab and ipilimumab, helped people with advanced lung cancer live longer than chemotherapy alone, which made it a first-line option for tumours with any PD-L1 expression.

CheckMate 358 gave the immunotherapy drug nivolumab to people with cancers caused by viruses; in Merkel cell carcinoma, a short course before surgery made the tumour disappear entirely in nearly half of those operated on, the first sign that immunotherapy could work before surgery in this skin cancer.

CheckMate 915 asked whether adding a low dose of ipilimumab to nivolumab after melanoma surgery would prevent more recurrences; it did not, and added toxicity, so single-agent PD-1 blockade remains the adjuvant standard.

CHRYSALIS was the first human study of amivantamab, an antibody that grabs both EGFR and MET; its cohort of people with EGFR exon 20 insertion lung cancer responded well enough to earn the drug its first approval.

CODEL is the intergroup trial meant to settle whether oligodendroglioma patients should get temozolomide or the older three-drug PCV combination with their radiotherapy; its first phase already showed that temozolomide on its own, without radiotherapy, lets the tumour come back sooner.

AALL0434 was the largest trial ever run in childhood T-cell leukaemia; it showed that adding the drug nelarabine to standard chemotherapy cut relapses, and that most children could be cured without radiation to the brain.

ACNS0332 found that giving carboplatin alongside radiotherapy improved survival for children with the most aggressive group 3 medulloblastomas, while isotretinoin, tested in the same trial, did nothing and was dropped.

AREN0532 tested treating the lowest-risk Wilms tumours with surgery alone and no chemotherapy, and giving more treatment only to children whose tumours carried particular chromosome losses; both ideas worked and now guide how much therapy each child gets.

COMET asked whether women with low-risk DCIS could safely be watched rather than operated on; after two years the rate of invasive cancer in the monitored group was no worse than with surgery, so careful monitoring is now a reasonable choice for some women.

COMMANDS showed that luspatercept freed more people with lower-risk myelodysplastic syndromes from blood transfusions than the erythropoietin injections that had been the first-line standard for decades.

DASISION showed that the second-generation drug dasatinib brought chronic myeloid leukaemia under control faster and more deeply than imatinib when used from diagnosis, though after five years patients lived equally long on either.

DESTINY-Gastric01 showed that trastuzumab deruxtecan shrank tumours far more often than standard chemotherapy, and helped people live longer, in HER2-positive stomach cancer that had already progressed on trastuzumab.

DESTINY-Gastric02 repeated the trastuzumab deruxtecan study in patients from Europe and the United States, after the first trial had been run only in Japan and Korea, and confirmed that the drug produces meaningful responses in HER2-positive stomach cancer after trastuzumab.

DESTINY-Lung01 showed that the antibody-drug conjugate trastuzumab deruxtecan shrank tumours in more than half of people whose lung cancer carried a HER2 mutation, which led to the first approved HER2-targeted treatment for lung cancer.

DETERMINATION asked whether people with newly diagnosed myeloma still need a stem-cell transplant when modern three-drug therapy and long maintenance are available; transplant kept the disease away for longer, but after several years the two groups had not lived different lengths of time, so the transplant can be delayed for some.

ECHELON-2 was the first trial in decades to improve on CHOP chemotherapy for T-cell lymphoma: swapping vincristine for the antibody-drug conjugate brentuximab vedotin helped patients live longer, especially those with anaplastic large cell lymphoma.

ENZAMET showed that adding enzalutamide to hormone therapy from the moment prostate cancer is found to have spread helped men live longer, and it did so in an academic trial that also allowed chemotherapy, which made its results widely applicable.

ESOPEC pitted the two standard ways of treating operable oesophageal adenocarcinoma against each other, chemotherapy before and after surgery or chemoradiotherapy before it, and found that the chemotherapy approach helped people live longer.

EXAM showed that cabozantinib, which blocks RET, MET and VEGF receptors, held back progressive medullary thyroid cancer for far longer than placebo, and gave the disease its second approved targeted drug.

FIGHT-302 set out to test whether the FGFR2 inhibitor pemigatinib should be used before chemotherapy in bile duct cancers driven by an FGFR2 fusion; the trial closed early after the standard of care changed, and its 2026 report showed pemigatinib delayed progression compared with chemotherapy, with similar overall survival.

FLOT4 showed that a docetaxel-based chemotherapy given before and after surgery helped people with operable stomach and junction cancers live longer than the older epirubicin-based regimen, and FLOT became the standard perioperative treatment in Europe.

GARNET showed that the PD-1 antibody dostarlimab produced lasting responses in about four in ten women with mismatch repair-deficient endometrial cancer that had returned after chemotherapy, which won the drug its first approvals.

GATSBY tested whether the antibody-drug conjugate trastuzumab emtansine, which works in HER2-positive breast cancer, would also beat chemotherapy in HER2-positive stomach cancer after first treatment; it did not, and the drug was never approved for this cancer.

GeparSixto showed that adding carboplatin to chemotherapy given before surgery made triple-negative breast tumours disappear completely far more often, and in longer follow-up cut relapses, which put platinum into the standard neoadjuvant regimen for this type.

GRID showed that regorafenib held back GIST that had already escaped both imatinib and sunitinib, and gave patients a third line of targeted treatment.

HERACLES showed that the breast cancer drugs trastuzumab and lapatinib, used together, could shrink bowel cancers that carry extra copies of the HER2 gene, turning a laboratory prediction into a treatment for this small group of patients.

HERBY asked whether adding the anti-blood-vessel antibody bevacizumab to radiotherapy and temozolomide would help children with high-grade brain tumours; it did not delay the tumours returning, so the drug is not part of standard treatment for these children.

IFM 2009 asked whether a stem cell transplant is still needed when myeloma is treated with modern three-drug therapy; the transplant kept the disease away for longer, though patients lived about as long either way because those who relapsed could be transplanted later.

IMerge showed that imetelstat, a drug that blocks telomerase in the abnormal blood-forming cells, freed many people with lower-risk myelodysplastic syndromes from transfusions for months or years after erythropoietin had failed.

IMspire150 tested adding the immunotherapy drug atezolizumab to the two targeted pills used for BRAF-mutant melanoma; the triple combination kept the cancer from growing for longer as judged by the treating doctors, which earned it approval, but it did not help people live longer.

INTELLANCE-1 tested an antibody-drug conjugate that targets the EGFR amplification found in about half of glioblastomas, added to standard treatment; it did not help people live longer, and the drug was abandoned.

KEYNOTE-057 showed that pembrolizumab could clear bladder cancer confined to the lining in a substantial minority of people whose disease had stopped responding to BCG, giving the first drug alternative to removing the bladder.

KEYNOTE-062 found that pembrolizumab on its own was no worse than chemotherapy for survival in PD-L1-positive stomach cancer, but adding it to chemotherapy did not clearly beat chemotherapy alone; the tumours with the most PD-L1, and those with mismatch-repair deficiency, gained the most.

KEYNOTE-407 showed that adding pembrolizumab to chemotherapy helped people with squamous lung cancer live longer whatever their PD-L1 level, and made chemo-immunotherapy the standard first treatment for this type.

KEYNOTE-412 tested adding pembrolizumab to standard chemoradiotherapy for locally advanced head and neck cancer; the improvement in keeping patients free of disease fell short of statistical significance, so chemoradiotherapy alone remains the standard.

KEYNOTE-811 showed that adding the immunotherapy pembrolizumab to trastuzumab and chemotherapy shrank HER2-positive stomach cancers more often and kept them under control for longer, with the gain concentrated in tumours that also express PD-L1.

KLASS-01 showed that removing an early stomach cancer through keyhole surgery is as safe for long-term survival as open surgery and causes fewer wound complications, which made laparoscopic gastrectomy the standard for early gastric cancer in Korea and beyond.

MAIA showed that adding the antibody daratumumab to the standard two-drug regimen kept newly diagnosed myeloma under control for far longer in older people who cannot have a transplant, and later that they lived longer too, which made the three-drug regimen a standard first treatment.

MANIFEST-2 found that adding the BET inhibitor pelabresib to ruxolitinib shrank spleens in nearly twice as many people with myelofibrosis, but the improvement in symptoms did not reach statistical significance, leaving the drug's approval uncertain.

MAVORIC showed that mogamulizumab, an antibody that clears the malignant T cells that carry the CCR4 protein, kept cutaneous T-cell lymphoma under control for longer than the standard tablet vorinostat, and did especially well in the blood-involved Sezary form, which led to its approval.