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Trials recruiting now, the landmark trials, the trials held by this cancer's subtypes, the key papers and what they mean, the latest literature, and the milestones year by year.
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AALL1731 brings immunotherapy into the front-line treatment of the commonest childhood cancer, in the group of children where most relapses were occurring despite good initial risk. Blinatumomab was approved for this use in 2024 and paediatric protocols worldwide are being amended. Whether it can allow less chemotherapy, and its effect on very low-risk children, are the next questions.
A lower-middle-income country can design, manufacture, trial and approve an autologous CAR-T therapy. Response rates are in the range of first-generation Western products in similar mixed populations, at a price an order of magnitude lower, which reopens the question of what CAR-T should cost everywhere.
E1910 changed the standard of care for adult B-ALL: immunotherapy is now part of front-line consolidation even for patients with no detectable leukaemia, because MRD-negative by flow cytometry does not mean cured. It also demonstrated that a T-cell engager can improve overall survival in a curative setting. Chemotherapy-light or chemotherapy-free regimens built on blinatumomab and inotuzumab are the next step.
Blinatumomab belongs in the treatment of low-risk marrow relapse in children; isolated extramedullary relapse needs new approaches.
Revumenib proved that a transcriptional dependency, rather than a kinase, can be drugged in leukaemia, opening treatment for two genetic subgroups that together cover roughly a third of AML plus most infant ALL. It is now approved and is being combined with venetoclax-azacitidine and intensive chemotherapy in front-line trials. Single-agent remissions are often short without transplant.
Prophylactic cranial radiotherapy can be limited to central nervous system involvement and the highest-risk patients in childhood T-ALL, and bortezomib is a reasonable addition for T-lymphoblastic lymphoma.
Blinatumomab consolidation is a safer bridge to transplant for children with higher-risk relapsed B-ALL and improved survival; it is the basis for using blinatumomab in place of chemotherapy blocks after relapse.
The UCART19 report was the first clinical evidence that a universal, pre-manufactured CAR-T made from a donor can work, avoiding the weeks of autologous manufacturing and the problem of patients whose own T cells are too damaged. It set the template for later allogeneic programmes (including cemacabtagene autoleucel in the ALPHA studies) and for in vivo CAR generation. Short persistence and the need for deep lymphodepletion remain the central weaknesses.
Query for this cancer: (TITLE:"Acute lymphoblastic leukaemia" OR ABSTRACT:"Acute lymphoblastic leukaemia") AND (treatment OR therapy OR trial OR survival OR diagnosis). Results are unfiltered search hits about Acute lymphoblastic leukaemia, not a curated reading list.
Aminopterin in children with ALL; the birth of cancer chemotherapy.
Pinkel's 'total therapy' at St. Jude: multi-agent induction, cranial irradiation, maintenance; first cures.
NCI/Rome criteria (age, white count); paediatric cooperative groups reach ~70% cure.
First BCR::ABL1 inhibitor; Ph+ ALL survival doubles when added to chemotherapy.
AIEOP-BFM 2000 and UKALL 2003 show end-of-induction MRD outperforms every clinical factor; MRD-directed therapy adopted.
CTL019 at CHOP/Penn; durable remission of refractory paediatric ALL.
Accelerated approval in relapsed/refractory Ph-negative B-ALL.
CR/CRi 81% vs 29%; approval 2017.
ELIANA-based approval for ALL up to age 25; TOWER OS 7.7 vs 4.0 months.
First approval based on an MRD endpoint (BLAST).
Dasatinib then blinatumomab, 18-month OS 95%.
Immunotherapy becomes part of standard first-line therapy for adults and children; second CAR-T for adult ALL; revumenib approved for KMT2Ar leukaemia.
FDA lifts REMS for approved CAR-T products; ziftomenib and revumenib studied in KMT2Ar ALL.
Subcutaneous blinatumomab trials (including the first in mixed-phenotype leukaemia) aim to replace continuous infusion; infant trials integrate blinatumomab.