Leukaemia — cancer of the blood and bone marrow — affects around 10,000 people in the UK each year, spanning four major types: acute lymphoblastic leukaemia (ALL), acute myeloid leukaemia (AML), chronic lymphocytic leukaemia (CLL), and chronic myeloid leukaemia (CML). UK haematology centres are world-leading in clinical trial innovation, from CAR-T cell therapy and bispecific antibodies to MRD-guided precision treatment and chemotherapy-free regimens.
The UK has one of the strongest leukaemia trial portfolios globally, supported by the NCRI Haemato-Oncology Clinical Studies Group, Blood Cancer UK, and the National Blood Cancer Trials Alliance. The NHS Genomic Medicine Service provides comprehensive molecular profiling that directly informs trial eligibility — including FLT3, NPM1, IDH1/2, TP53, and Philadelphia chromosome status.
There are currently over 150 actively recruiting leukaemia trials in the UK across all four major subtypes, making this one of the largest disease-specific trial portfolios in the country.
Types of Leukaemia Trials
CAR-T Cell Therapy
Engineered T cells targeting CD19 (ALL, CLL) and novel targets in AML — the most transformative advance in blood cancer treatment.
Bispecific Antibodies
Off-the-shelf T-cell engagers (blinatumomab, teclistamab) bridging T cells to cancer cells without needing personalised cell manufacturing.
Targeted Small Molecules
FLT3 and IDH inhibitors for AML, BTK inhibitors for CLL, BCR-ABL inhibitors for CML, and BCL-2 inhibitors across subtypes.
The UK has Europe's largest CAR-T programme for blood cancers, with NHS England commissioning CAR-T for relapsed/refractory ALL and diffuse large B-cell lymphoma:
CD19 CAR-T for ALL — tisagenlecleucel (Kymriah) and brexucabtagene autoleucel (Tecartus) are commissioned for B-cell ALL. UK trials are now moving CAR-T earlier in the treatment pathway — as second-line therapy, and even as a bridge to transplant
CD19 CAR-T for CLL — liso-cel (Breyanzi) and next-generation CAR-T products in UK trials for relapsed/refractory CLL, historically a difficult-to-treat population with CAR-T
AML CAR-T — developing CAR-T products targeting CD33, CD123, and FLT3 for acute myeloid leukaemia, which lacks a single clean target like CD19. UK Phase 1 trials are pioneering multi-antigen targeting
Allogeneic (off-the-shelf) CAR-T — donor-derived CAR-T cells that don't require individual manufacturing, dramatically reducing time-to-treatment. UK trials at multiple centres
Dual-targeting and armored CAR-T — next-generation products targeting two antigens (CD19 + CD22) to prevent antigen escape, and CAR-T cells secreting cytokines to enhance persistence
Bispecific Antibodies
Bispecific T-cell engagers (BiTEs) offer an off-the-shelf alternative to CAR-T, bridging the patient's own T cells directly to cancer cells:
Blinatumomab (Blincyto) — CD3×CD19 bispecific now standard for MRD-positive ALL and established in relapsed/refractory B-ALL. UK trials testing: earlier use in frontline therapy, continuous infusion optimisation, and subcutaneous delivery
Teclistamab (Tecvayli) — CD3×BCMA bispecific approved for myeloma, now being explored in related haematological malignancies
AML bispecifics — CD3×CD33, CD3×CD123, and CD3×FLT3 bispecific antibodies in UK Phase 1/2 trials for relapsed AML
Step-dosing strategies — UK trials optimising the ramp-up dosing to minimise cytokine release syndrome while maintaining efficacy
Targeted Therapies
Molecularly targeted drugs are reshaping leukaemia treatment across all subtypes:
FLT3 inhibitors (AML) — midostaurin (first-line), gilteritinib (relapsed), and quizartinib in UK trials. Next-generation FLT3 inhibitors with broader mutant coverage and better CNS penetration
IDH inhibitors (AML) — ivosidenib (IDH1) and enasidenib (IDH2) for the ~20% of AML patients with IDH mutations. UK trials testing: combination with azacitidine, use in frontline therapy, and as maintenance after transplant
Venetoclax (AML) — BCL-2 inhibitor combined with hypomethylating agents (azacitidine/decitabine) has transformed older/unfit AML treatment. UK trials extending to younger patients, exploring triplet combinations, and venetoclax-based maintenance
BTK inhibitors (CLL) — ibrutinib, acalabrutinib, and zanubrutinib. UK trials testing: fixed-duration combinations with venetoclax (achieving treatment-free remission), sequencing strategies, and newer non-covalent BTK inhibitors for BTK-resistant disease
Next-generation TKIs (CML) — asciminib (STAMP inhibitor with a novel mechanism) in UK trials for CML after multiple TKI failures, and as first-line treatment aiming for treatment-free remission
MRD-Guided Treatment
Measurable residual disease (MRD) is becoming the most important biomarker in leukaemia, guiding treatment intensity and duration:
MRD-guided de-escalation — trials reducing treatment intensity for patients who achieve deep MRD negativity, sparing toxicity without compromising outcomes
MRD-triggered escalation — for patients with persistent MRD, trials testing early intervention (blinatumomab, inotuzumab, or CAR-T) before overt relapse
MRD as a trial endpoint — UK trials increasingly using MRD as a primary endpoint, accelerating drug development by providing a faster readout than overall survival
Molecular monitoring — NGS-based MRD detection with sensitivity down to 10⁻⁶, replacing older flow cytometry methods in UK trials
Paediatric Leukaemia Research
Leukaemia is the most common childhood cancer, and UK paediatric haematology has a remarkable trial track record:
ALLTogether consortium — the UK participates in Europe's largest paediatric ALL trial, testing therapy reduction for low-risk patients and intensification for high-risk groups, guided by MRD and genetics
CAR-T for children — tisagenlecleucel approved for paediatric B-ALL, with UK trials moving it earlier and testing in infant leukaemia (a particularly challenging subtype)
AML-SRC trial — UK-led paediatric AML study testing novel combinations including FLT3 inhibitors and venetoclax in children
Long-term follow-up — UK studies tracking late effects of childhood leukaemia treatment (cardiac, neurocognitive, fertility) to inform safer treatment protocols
Who Can Participate?
Leukaemia trial eligibility depends on subtype, molecular markers, and treatment history:
CAR-T trials — typically require relapsed/refractory disease after ≥2 prior lines, adequate organ function for leukapheresis, no active CNS disease (for some studies), and CD19-positive disease (for CD19 CAR-T)
Targeted therapy trials — require the relevant molecular marker (FLT3 mutation, IDH1/2 mutation, Philadelphia chromosome, etc.) confirmed by genomic testing
Frontline trials — newly diagnosed patients, often with specific cytogenetic or molecular features. May exclude patients who have started treatment
Transplant trials — require a transplant-eligible patient (age, comorbidities) and often a matched donor identified
General criteria — adequate performance status, organ function, no active uncontrolled infection, and willingness to attend frequent monitoring visits (especially for CAR-T and bispecific trials)
💡 Tip: Request Genomic Profiling
If you have acute leukaemia, comprehensive genomic profiling at diagnosis is essential. The NHS Genomic Medicine Service now provides testing for FLT3, NPM1, IDH1/2, TP53, and many other markers. These results directly determine which clinical trials you are eligible for. Ask your haematologist for a full molecular report — it is your passport to targeted therapy and innovative trial options.
UK Leukaemia Trial Locations
Major UK centres running leukaemia trials include:
London — UCLH (particularly for CAR-T), King's College Hospital, Royal Marsden, Imperial College, Barts Health, Great Ormond Street (paediatric)
Manchester — The Christie NHS Foundation Trust (major CAR-T centre), Manchester Royal Infirmary
Birmingham — Queen Elizabeth Hospital Birmingham, Birmingham Children's Hospital
Leeds — St James's University Hospital (Leeds Cancer Centre)
Glasgow — Beatson West of Scotland Cancer Centre
Edinburgh — Royal Infirmary of Edinburgh
Cardiff — University Hospital of Wales
Bristol — Bristol Royal Infirmary, University Hospitals Bristol
How to Find Your Match
Use our Smart Matcher to find leukaemia trials tailored to your subtype, molecular profile, and treatment history. Whether you are exploring CAR-T, seeking a targeted therapy for a specific mutation, or looking for frontline trial options, we can match you to actively recruiting studies.