Cell-based immunotherapy uses a patient's own immune cells, or donor cells, as a treatment. The best-known kind is CAR-T therapy. Doctors collect a patient's T cells, change them in a lab so they can find and kill cancer cells, grow them into millions of copies, and infuse them back. It is sometimes called a "living drug".
A short history
The first CAR-T therapy, Kymriah (tisagenlecleucel), was approved in the US on 30 August 2017 [1]. Since then, several CAR-T products have been approved for blood cancers such as some leukaemias, lymphomas and multiple myeloma. They target proteins called CD19 or BCMA on cancer cells [2].
Why it matters
Some patients with blood cancers that came back after many treatments have gone into long remissions after one CAR-T infusion. For these patients, there may be few other options. Cancer caused nearly 10 million deaths in 2024 [3], so new ways to treat hard cases are urgently needed.
How treatment works
- The patient's T cells are collected from the blood.
- Cells are shipped to a specialist lab and genetically modified.
- The modified cells are grown over several weeks.
- The patient may receive chemotherapy to make room for the new cells.
- The CAR-T cells are infused, and the patient is watched closely.
Serious risks
The Kymriah label carries a boxed warning for cytokine release syndrome, neurological toxicities and secondary blood cancers [1]. Cytokine release syndrome is a strong immune reaction that can cause fever and low blood pressure. Neurological effects can include confusion and seizures. Patients must be treated in centres able to manage these problems.
In 2024, the FDA required a boxed warning for T cell cancers after treatment with all approved BCMA- and CD19-directed autologous CAR-T therapies [2]. These cases have led to hospital stays and deaths [1]. The overall benefit is still judged to outweigh the risk for approved uses, but patients need long-term follow-up.
Cost and access
- Each treatment is made for one patient, which makes it expensive.
- Only specialist centres can give it, so many patients must travel.
- Manufacturing can take weeks, and some patients get sicker while waiting.
- Health systems must decide which patients to fund.
What comes next
- Donor ("off-the-shelf") cells that can be made in advance.
- Solid tumours, which have been harder to treat with CAR-T.
- Autoimmune diseases, such as lupus, being tested in trials.
- Faster, local manufacturing near hospitals.
- Other cell types, such as natural killer cells.
Key numbers at a glance
- First US CAR-T approval: 30 August 2017.
- FDA boxed warning for T cell cancers after CAR-T: 2024.
- Cancer deaths: nearly 10 million in 2024.
Questions for patients and families
- Am I eligible for CAR-T, and when?
- Where is the nearest treatment centre?
- How long will I need to stay nearby?
- What follow-up will I need for years afterwards?
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Who gives CAR-T
CAR-T therapy is only given at certified treatment centres with trained teams [1]. Under the FDA's risk programme for Kymriah, hospitals must be able to manage cytokine release syndrome and neurological side effects quickly [1]. Patients usually need to stay near the centre for several weeks after infusion, with a caregiver. These rules protect patients but limit access for people far from big cities.
Long-term follow-up
Because CAR-T cells are genetically changed and can stay in the body for years, patients need long-term monitoring. The FDA's 2024 boxed warning on T cell cancers advises lifelong monitoring for new cancers [2]. Follow-up also checks blood counts, infections and whether the original cancer has returned.
Paying for one-time treatments
CAR-T is given once, but its price is very high. Health systems are trying new ways to pay, such as paying in instalments or linking payment to results. These models aim to share risk between payers and makers while keeping access for patients who need it.
Questions for health systems
- How many certified centres are needed to serve the population?
- How will travel and housing for patients be supported?
- Which patients benefit most, and when should they be referred?
- How will long-term outcomes be tracked?
Outside the US
CAR-T therapies are also approved in Europe, China, Japan and other countries. Some countries, including India, have developed their own lower-cost CAR-T products. Local production may make these treatments more affordable in middle-income countries.
Research to watch
Trials are testing CAR-T earlier in treatment, rather than only after other options fail. If earlier use proves safe and effective, many more patients could benefit, and demand for treatment centres would grow.
What this means
Cell therapies have changed what is possible for some blood cancers. The next challenges are making them safer, faster, cheaper and useful for more types of cancer and disease.
Where to read more
All figures in this article come from public sources listed below. For more detail, start with the FDA's CAR-T safety communications and the Kymriah prescribing information. Each is free to read online. If anything here raises a question about your own health, take this article to your doctor or pharmacist and talk it through together. Buyers, investors and policy makers can also use these sources to check claims in sales materials and compare options on the same public evidence. Where public sources disagree or update their figures, we note the date of each number so readers can see how the picture changes over time. Readers who spot an error or a newer source are welcome to contact our team, and we will review it.
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Topics: Oncology · Biologics and biosimilars
Sources
- US FDA: KYMRIAH (tisagenlecleucel) prescribing information
- US FDA (Apr 2024): FDA requires boxed warning for T cell malignancies after CAR T cell immunotherapies
- WHO fact sheet: Cancer
Banner image: Diatoms through the microscope by Prof. Gordon T. Taylor, Stony Brook University, Public domain, via Wikimedia Commons.
The data behind this story
Topics: Oncology · Biologics and biosimilars
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