subtype-drugs-wave

Acitretin is a vitamin A tablet licensed for severe psoriasis. In people who have had an organ transplant and keep developing skin cancers, it reduces the number of new squamous cell carcinomas while it is taken.

Aminopterin was the first drug to send childhood leukaemia into remission. In 1948 Sidney Farber reported temporary remissions in children with acute leukaemia, the birth of cancer chemotherapy; its safer cousin methotrexate replaced it within a decade.

Anastrozole is a daily tablet that stops the body making oestrogen after the menopause. It treats and prevents hormone-receptor-positive breast cancer and is an alternative to tamoxifen after surgery for ductal carcinoma in situ.

Brigimadlin is a tablet that frees the p53 'guardian' protein from MDM2, the protein that liposarcomas make in excess to keep p53 switched off. It is being compared with doxorubicin as first treatment for dedifferentiated liposarcoma in a phase 2/3 trial.

Bromocriptine was the first pill that could shrink a pituitary tumour. From the 1970s it turned prolactinomas from a surgical disease into one usually controlled with tablets, and it is still used where cabergoline is unavailable or unsuitable.

Cabergoline is a tablet taken once or twice a week that shrinks prolactin-secreting pituitary tumours and normalises hormone levels in most patients, so surgery is reserved for the few whose tumours resist or who cannot tolerate it.

Carotuximab was an antibody against endoglin, a protein on growing blood vessels and on angiosarcoma cells. Added to pazopanib in the phase 3 TAPPAS trial it did not help, and the trial was stopped in 2019.

Cediranib is a tablet that blocks the blood-vessel growth signal VEGF. In alveolar soft part sarcoma, a rare very vascular sarcoma that ignores chemotherapy, a randomised trial showed it shrinks tumours and delays progression, although it was never licensed.

Celecoxib is a common anti-inflammatory painkiller. In oncology it is paired with low-dose oral methotrexate as a cheap 'metronomic' treatment for advanced head and neck cancer, a regimen from Tata Memorial in Mumbai that beat intravenous cisplatin in a randomised trial.

Danazol is an old hormone tablet, licensed for endometriosis, that can raise haemoglobin and platelets in myelofibrosis and bone marrow failure. It was the comparison treatment that momelotinib beat in the MOMENTUM trial.

Daromun is a pair of antibody-linked immune messengers injected straight into melanoma deposits before surgery. In a phase 3 trial it lowered the chance of the melanoma coming back after the operation compared with surgery alone.

Depatuxizumab mafodotin carried a cell-killing payload to glioblastomas with extra copies of the EGFR gene. It caused serious eye problems and, in the phase 3 INTELLANCE-1 trial, did not help patients live longer, so development stopped in 2019.

Diazoxide is a medicine that stops insulin-producing tumours releasing insulin. It is used to control dangerous low blood sugar from an insulinoma while the patient is prepared for surgery or when the tumour cannot be removed.

GI-6301 was a vaccine made from yeast carrying brachyury, the protein that chordomas depend on. Given with radiotherapy in a randomised trial it did not improve tumour responses.

GSK2256098 is a tablet that blocks an enzyme called FAK, which meningiomas missing the NF2 gene rely on. In the Alliance A071401 trial it slowed progression in recurrent NF2-mutant meningiomas, one of the first positive results for a targeted drug in this tumour.

HB-200 is a vaccine built from two harmless engineered viruses that teach the immune system to attack the HPV16 proteins inside cervical and throat cancers. It has been tested with pembrolizumab in HPV16-positive head and neck cancer.

INCA033989 is an antibody that recognises only the mutant form of calreticulin found in about a quarter of essential thrombocythaemia and myelofibrosis patients. Unlike existing drugs, which control counts, it targets the diseased clone itself and is in first-in-human trials.

Isotretinoin, the acne drug, is given to children with high-risk neuroblastoma for six months after transplant to nudge any surviving cancer cells into growing up into harmless nerve cells. A 1999 trial showed it improved survival and it has been part of standard care since.

Lenzilumab is an antibody that soaks up GM-CSF, a growth signal that chronic myelomonocytic leukaemia cells with RAS mutations are unusually sensitive to. It is being tested with azacitidine in this leukaemia.

Mavorixafor is a once-daily tablet that blocks CXCR4, a receptor that traps white cells in the bone marrow. It is approved for the rare immunodeficiency WHIM syndrome and is being tested in Waldenström macroglobulinaemia, where CXCR4 mutations blunt the response to ibrutinib.

Memantine is an Alzheimer's drug taken during whole-brain radiotherapy to protect memory. With hippocampal-sparing radiotherapy it has become the standard way to limit the thinking problems the treatment causes.

Metyrapone is an older tablet that blocks cortisol production in the adrenal glands. It is used to bring cortisol down quickly in Cushing's disease before or after pituitary surgery, and in the United States mainly as a diagnostic test.

Mifepristone, best known as an abortion pill and a treatment for Cushing's syndrome, blocks progesterone receptors that most meningiomas carry. A large phase 3 trial found it did not slow inoperable meningiomas.

NeoVax is a made-to-order vaccine: a patient's tumour is sequenced, the mutations most likely to be seen by the immune system are turned into peptides, and these are injected to train T cells against that tumour alone. Early trials in melanoma and glioblastoma showed it raises the intended immune response.

Nicotinamide is a cheap over-the-counter form of vitamin B3. Taken twice a day it cuts the number of new skin cancers in people who have already had several, by helping skin cells repair sun damage.

Obrixtamig is an antibody with two arms: one grabs DLL3, a protein on the surface of most neuroendocrine carcinomas, and the other grabs a T cell, pulling the killer cell onto the cancer. It is in a phase 3 trial for neuroendocrine carcinomas outside the lung.

Osilodrostat is a twice-daily tablet that stops the adrenal glands making cortisol. It controls Cushing's disease caused by a pituitary tumour when surgery has failed or is not possible, normalising cortisol in most patients.

Paltusotine is the first somatostatin-type drug that works as a once-daily pill instead of a monthly injection. It was approved in 2025 for acromegaly and is being tested for the flushing and diarrhoea of carcinoid syndrome from small-bowel neuroendocrine tumours.

Pasireotide is a second-generation somatostatin analogue that hits more receptor types than octreotide. It is approved for Cushing's disease and acromegaly caused by pituitary tumours when surgery has not cured them, at the cost of frequent high blood sugar.

Patidegib is a hedgehog-pathway blocker made into a skin gel. It is being tested in people with Gorlin syndrome, who grow dozens of basal cell carcinomas, to prevent new tumours without the hair loss, muscle cramps and taste loss that the oral drugs cause.

Pegvisomant is a daily injection that blocks growth hormone at its receptor. It normalises the downstream hormone IGF-1 in most people with acromegaly whose tumours were not cured by surgery, although it does not shrink the tumour itself.

Pevonedistat was a first-in-class drug that jammed part of the cell's protein-disposal system. Promising with azacitidine in a mid-stage trial for higher-risk myelodysplastic syndromes, it failed to beat azacitidine alone in the phase 3 PANTHER trial in 2021.

Radiophosphorus was one of the first treatments for polycythaemia vera, from the 1940s: a radioactive phosphate injection that quietened the overactive bone marrow for a year or more. It was abandoned when trials showed it raised the risk of leukaemia.

Poziotinib was a tablet designed to fit the awkward shape of HER2 and EGFR exon 20 mutations in lung cancer. It shrank tumours in some patients but caused severe rash and diarrhoea, and the FDA declined to approve it in 2022.

Setmelanotide is a daily injection that switches back on the brain's fullness signal. Approved for rare genetic causes of obesity, it is being tested for the relentless weight gain that follows damage to the hypothalamus from craniopharyngioma surgery or radiotherapy.

HyBryte is an ointment containing synthetic hypericin, the active pigment of St John's wort. Rubbed on patches of cutaneous T-cell lymphoma and switched on with ordinary visible light, it kills the lymphoma cells in the skin without ultraviolet light.

TK216 was an attempt to hit the fusion protein that causes Ewing sarcoma directly, something long thought impossible for a transcription factor. In early trials with vincristine it produced a few responses but not enough to carry the drug forward.

Xevinapant was a tablet meant to make head and neck cancer cells easier to kill with chemoradiation by removing the proteins that stop them dying. A promising mid-stage trial was followed by a phase 3 that was stopped in 2024 because it was not working.

Zanidatamab zovodotin joined the two-armed HER2 antibody zanidatamab to a cell-killing payload. It was tested in early trials in HER2-expressing cancers; the plain antibody went on to approval, the conjugate did not progress beyond phase 1.