The first CAR T-cell therapy is approved

Historical milestone — this page describes an event dated 2017. It is not current breaking news.

What was approved on 30 August 2017

FDA licensed tisagenlecleucel, sold as Kymriah and made by Novartis, under license number STN 125646. It was the first CAR T-cell therapy cleared anywhere in the United States.

The first indication was narrow. It covered people up to 25 years of age with B-cell precursor acute lymphoblastic leukemia that was refractory, meaning it never responded to treatment, or in a second or later relapse.

How the treatment is made

This is not a drug taken off a shelf. It is built from one person's own cells.

T cells are white blood cells that kill infected or abnormal cells. They find targets using receptors on their surface. In some cancers those receptors simply do not recognize the tumor.

The engineering step adds a new receptor, called a chimeric antigen receptor, or CAR. It is chimeric because it is stitched together from parts of different proteins. Kymriah's CAR is aimed at CD19, a marker sitting on the surface of B cells, including leukemic ones.

NCI describes the process taking about 22 days. T cells are collected from the person's blood, sent to a facility, given the new receptor, grown to large numbers, and shipped back to the treating center for infusion.

Our overview of immunotherapy sets out how this differs from drugs that attack cancer cells directly.

The evidence in 2017

The approval rested on a single multicenter trial of 63 children and young adults whose B-cell ALL had relapsed or resisted treatment. NCI reported an overall remission rate of 83% within three months.

There was no comparison group. Everyone in the trial got the treatment, so the trial could not measure how those results compared with anything else.

NCI has since published an update from the same trial, called ELIANA. Among 79 participants followed for three years, the cancer had not returned in about half, and 63% were alive. Twenty-nine percent still had serious side effects at least a year after treatment.

Both sets of numbers belong on the page. The first explains why the approval happened quickly. The second is what the treatment looks like over time.

Why it mattered for this disease

Acute lymphoblastic leukemia is the most common cancer in children in the United States. NCI notes that intensive chemotherapy clears the disease for more than 80% of children with the B-cell form.

The problem this approval addressed was the remaining group. For a child whose leukemia never responded, or returned twice, there was very little left to try. That is the group the label describes, and it is why a single-arm trial was accepted as evidence.

The American Cancer Society projects about 6,250 new ALL diagnoses in the United States in 2026 and about 1,600 deaths, and SEER, the federal cancer surveillance program, hosts the figures. From SEER's own records, median age at diagnosis is 18, and across all ages five-year relative survival is 73.2% for people diagnosed from 2016 through 2022.

Our page on leukemia explains how the acute and chronic forms differ.

The risks are on the label in bold

Kymriah carries a boxed warning, the strongest FDA uses.

Cytokine release syndrome is the first item. When the engineered cells begin killing, they release signaling molecules in a rush. The result can be very high fever and falling blood pressure, and it can be fatal. FDA approved tocilizumab, an antibody that blocks one of those signals, on the same day in 2017 as a way to treat it.

Neurological toxicity is the second. It can include confusion and difficulty speaking, and it can happen alongside cytokine release syndrome or on its own.

The boxed warning was updated in June 2025 to add a third item: T-cell cancers have occurred after treatment with CD19- and BCMA-directed engineered T-cell therapies, including this one.

Other label warnings include severe allergic reactions during infusion, serious infections, and blood counts that stay low for a long time.

How the field moved afterward

The label itself is the clearest record. Kymriah now also covers adults with relapsed or refractory large B-cell lymphoma after two or more lines of treatment, and adults with relapsed or refractory follicular lymphoma after two or more lines.

The follicular lymphoma indication sits under accelerated approval, based on response rate and duration of response, with continued approval contingent on confirming benefit.

The label also carries a limitation of use worth quoting: Kymriah is not indicated for primary central nervous system lymphoma. Approvals list what a treatment is for, and sometimes what it is explicitly not for.

What to keep in perspective

• The 2017 approval rested on 63 people with no comparison group. Follow-up at the time was short, and durability was not yet known. The three-year ELIANA figures came later.
• Progress in solid tumors has lagged far behind blood cancers. A receptor aimed at CD19 works because B cells carry a clean, shared marker; most solid tumors do not.
• This is a summary of a historical decision, not advice about anyone's care. Eligibility for a cell therapy is decided by a treating center, not by a page like this one. Our explainer on what cancer is covers the underlying biology.

Sources

• FDA: KYMRIAH (tisagenlecleucel), STN 125646
• FDA: KYMRIAH prescribing information (PDF)
• NCI Cancer Currents: CAR T-Cell Therapy Approved for Some Children and Young Adults with Leukemia
• NCI SEER Stat Facts: Acute Lymphocytic Leukemia

How this article was prepared

An AI-assisted editorial system helped prepare this page. No named medical reviewer has reviewed it unless one is listed.

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