Tisagenlecleucel
Tisagenlecleucel was the first CAR-T therapy ever approved (2017), for children and young adults whose leukaemia had come back after everything else.
ELIANA (n=75 infused, age ≤25): ORR 81% within 3 months, 12-month OS 76%, 5-year EFS ~44% in responders without further therapy (2023 update). Approved 30 August 2017 for relapsed/refractory B-ALL up to age 25; later for DLBCL and follicular lymphoma. 4-1BB costimulation gives long persistence; CD19-negative relapse is the main failure mode. Emily Whitehead, treated in 2012, remains in remission.
1.Leukapheresis collects the patient's T cells
- Route
- Single IV infusion
- Schedule
- 0.2-5.0 × 10^6 CAR+ cells/kg (≤50 kg) or 0.1-2.5 × 10^8 (>50 kg) after lymphodepletion
- Monitoring
- CRS and ICANS (boxed warning; tocilizumab on site), prolonged cytopenias, hypogammaglobulinaemia, secondary T-cell malignancy (class warning 2024)
Source: US prescribing information (DailyMed). Doses are for orientation; the current label governs.
- Medicare
- Part B (clinician-administered)NCD 110.24
Autologous CAR-T covered nationally under NCD 110.24 for FDA-labelled indications at facilities enrolled in the FDA REMS. Inpatient administration is paid under Part A (MS-DRG 018); outpatient administration is a Part B drug. The product itself is bundled into the facility payment. HCPCS Q2042.
- Commercial insurance
- covered with prior authorisation
Covered with prior authorisation and usually a single-case agreement with a certified treatment centre; many plans restrict to centres of excellence and require documentation of prior lines of therapy.
- List price
- $475,000 per one-time treatment for paediatric/young adult B-ALL, launch list price
National Cancer Institute, Cancer Currents, September 2017 (reporting Novartis's announced price) (2017). Net prices after rebates are usually lower.
Medicare patients usually face the Part A inpatient deductible rather than 20% of the product price; the hospital stay, apheresis and bridging therapy drive the bill.
Sources: Medicare.gov: Chemotherapy · Medicare.gov: Prescription drugs (outpatient, Part B) · CMS NCD 110.24: Chimeric antigen receptor (CAR) T-cell therapy · Medicare.gov: Inpatient hospital care (Part A). Not medical or financial advice; verify with your plan.
- Appraised for
- Relapsed or refractory B-cell ALL in people up to 25 years
- Notes
- The first CAR-T NICE recommended, within weeks of EU licensing. DLBCL use (TA567, 2019, CDF) was not recommended for routine commissioning when reappraised in 2023. Delivered only at NHS England-designated CAR-T centres after national CAR-T clinical panel review.
- Cancer Drugs Fund
- Entered the Cancer Drugs Fund under a managed access agreement; check the current CDF list for whether it has since moved to routine commissioning.
- NHS England
- Routinely funded for the appraised indication (or via managed access)
Sources: NICE TA554 · NHS England Cancer Drugs Fund list · SMC advice: tisagenlecleucel. Funding decisions are indication-specific and change monthly; verify with NICE and your treating team.
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- 30 Aug 2017ApprovalUS
First CAR-T approval in history
- 18 Apr 2024Label changeUS
Class boxed warning for T-cell malignancies
- 27 Jun 2025Label changeUS
REMS requirements removed for approved CAR-T products
Approvals
| Region | Year | Indication |
|---|---|---|
| US | 2017 | Relapsed/refractory B-ALL, patients up to 25 years |
| US | 2018 | Relapsed/refractory DLBCL |
| US | 2022 | Relapsed/refractory follicular lymphoma |
| Adverse event | Any grade | Grade 3+ |
|---|---|---|
| Cytokine release syndrome ELIANA, Penn grading | 77% | 46% |
| Neurotoxicity (ICANS) | 40% | 13% |
| Prolonged cytopenias | — | 32% |
| B-cell aplasia / hypogammaglobulinaemia Expected; IVIG replacement | — | — |
Rates read from the US prescribing information. Blank cells mean the figure was not sourced, not that it is zero.
Trials
topRecruiting now (live from ClinicalTrials.gov)
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Landmark trials in OnCo
JULIET, with ZUMA-1, showed that CAR-T could rescue a substantial minority of adults with chemotherapy-refractory lymphoma and that complete responders often stay in remission. The lower toxicity of a 4-1BB construct and the option of bridging therapy made it usable in a broader population. The high drop-out between enrolment and infusion remains a lesson about turnaround time.
ELIANA turned CAR-T from a single-centre experiment into a licensed product and created the regulatory and logistical template every later cell therapy has followed. For children with refractory leukaemia it offers a chance of durable remission without transplant. The trial also exposed the gaps: manufacturing failures, patients dying while waiting, and roughly half relapsing within a few years.
This paper is the proof-of-concept for a living drug: a single infusion of a patient's own engineered T cells could eradicate leukaemia that had survived chemotherapy, transplant and antibody therapy. It defined cytokine release syndrome and its antidote, tocilizumab, and revealed antigen-loss relapse. It led directly to the first approved gene-modified cell therapy three years later.
Latest papers
topQuery for this drug: (TITLE:"Tisagenlecleucel" OR ABSTRACT:"Tisagenlecleucel" OR TITLE:"Kymriah" OR ABSTRACT:"Kymriah" OR TITLE:"CTL019" OR ABSTRACT:"CTL019") AND (cancer OR tumor OR tumour OR oncology OR carcinoma OR lymphoma OR leukemia OR leukaemia OR myeloma OR sarcoma OR melanoma OR glioma). Results are unfiltered search hits about Tisagenlecleucel, not a curated reading list.
Pages like this
not linked directly; found by shared links- Key paperZUMA-1: axicabtagene ciloleucel for refractory large B-cell lymphoma, the first CAR-T approved for lymphoma
Shares JULIET: tisagenlecleucel for adults with relapsed or refractory diffuse large B-cell lymphoma, ICANS (neurotoxicity), Follicular lymphoma, Cytokine release syndrome (CRS).
- ProductObecabtagene autoleucel
Shares ELIANA: the global trial that made tisagenlecleucel the first approved CAR-T therapy for children and young adults with relapsed ALL, ICANS (neurotoxicity), Cytokine release syndrome (CRS), CD19.
- PersonBruce L. Levine
Shares Emily Whitehead, Maude 2014: CD19 CAR-T cells produce complete remission in 27 of 30 children and adults with relapsed ALL, ELIANA: the global trial that made tisagenlecleucel the first approved CAR-T therapy for children and young adults with relapsed ALL, CAR-T cell therapy.
- IdeaHospital-made CAR-T under one shared regulatory master file
Shares CD19, Prices and value, Manufacturing cost and time for living and radioactive medicines, Acute lymphoblastic leukaemia.
- ProductLisocabtagene maraleucel
Shares ICANS (neurotoxicity), Follicular lymphoma, Cytokine release syndrome (CRS), CD19.
- ProductBrexucabtagene autoleucel
Shares ICANS (neurotoxicity), Cytokine release syndrome (CRS), CD19, Acute lymphoblastic leukaemia.
- Key paperUCART19: the first gene-edited, donor-derived CAR-T cells in children and adults with relapsed B-cell ALL
Shares ELIANA: the global trial that made tisagenlecleucel the first approved CAR-T therapy for children and young adults with relapsed ALL, Cytokine release syndrome (CRS), CD19, Manufacturing cost and time for living and radioactive medicines.
- InstitutionAbramson Cancer Center, University of Pennsylvania
Shares David L. Porter, Carl H. June, Emily Whitehead, JULIET: tisagenlecleucel for adults with relapsed or refractory diffuse large B-cell lymphoma.