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
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