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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.
Autologous T cells transduced with a lentiviral CD19 scFv-4-1BB-CD3ζ CAR; expand in vivo and kill CD19+ blasts. Connects to CD19.
1.Leukapheresis collects the patient's T cells
Source: US prescribing information (DailyMed). Doses are for orientation; the current label governs.
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.
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.
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.
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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First CAR-T approval in history
Treatment of adult patients with relapsed or refractory (r/r) follicular lymphoma (FL) after two or more lines of systemic therapy
Accelerated approval on a surrogate endpoint; the confirmatory requirement was still open 4.3 years later, when the FDA's table was read.
Class boxed warning for T-cell malignancies
REMS requirements removed for approved CAR-T products
| 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.
Read for the class this product belongs to (car-t cell therapy) unless the answer names the product. Population figures from the cohorts named, not a prediction for one person. Blank where no source gives a recovery figure.
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The reason second-line CAR-T is offered with axicabtagene ciloleucel or lisocabtagene maraleucel rather than tisagenlecleucel. The trial is also the strongest evidence in the field that the interval between apheresis and infusion, and what is given during it, is part of the treatment rather than logistics around it.
Chemotherapy-free cellular therapy for follicular lymphoma that has stopped responding, with a toxicity profile mild enough that the treatment is deliverable outside the largest centres.
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.
Query 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.
Shares Report the time from apheresis to infusion as a trial endpoint, not a logistics footnote, Immunoglobulin replacement after CAR-T, bispecifics and long anti-CD20 treatment, CD19 expression (CD19-positive), Cytokine release syndrome and ICANS: grading and management.
Shares CD19 expression (CD19-positive), Cytokine release syndrome and ICANS: grading and management, The CAR-T pathway in lymphoma: referral, apheresis, bridging and the waiting, Autologous CAR-T manufacturing, batch by batch.
Shares Lymphoma treatments that did not work: the negative trials worth knowing, JULIET: tisagenlecleucel for adults with relapsed or refractory diffuse large B-cell lymphoma, Report the time from apheresis to infusion as a trial endpoint, not a logistics footnote, JULIET.
Shares Lymphoma treatments that did not work: the negative trials worth knowing, Report the time from apheresis to infusion as a trial endpoint, not a logistics footnote, JULIET, Second-line tisagenlecleucel or standard care in aggressive B-cell lymphoma.
Shares Report the time from apheresis to infusion as a trial endpoint, not a logistics footnote, JULIET, The CAR-T pathway in lymphoma: referral, apheresis, bridging and the waiting, ICANS (neurotoxicity).
Shares Report the time from apheresis to infusion as a trial endpoint, not a logistics footnote, Immunoglobulin replacement after CAR-T, bispecifics and long anti-CD20 treatment, CD19 expression (CD19-positive), POD24: progression of follicular lymphoma within two years, and why it changes the plan.
Shares ELIANA: the global trial that made tisagenlecleucel the first approved CAR-T therapy for children and young adults with relapsed ALL, Autologous CAR-T manufacturing, batch by batch, ICANS (neurotoxicity), Relapsed and refractory acute lymphoblastic leukaemia in children.
Shares ELIANA, 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, Children's Hospital of Philadelphia.