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CAR T-cell therapy created inside the body shows promise in animal trials

New RNA method may cut costs and simplify cancer treatment by skipping lab-grown cell steps

19-Jun-2025

Key points from article :

Scientists may be on the cusp of a major advance in cancer immunotherapy by developing a method to create CAR T-cells directly inside the body. Traditionally, CAR T-cell therapy involves collecting a patient's T-cells, genetically modifying them in a lab to target cancer cells, and reinfusing them into the patient—a time-consuming and costly process, often exceeding $500,000. Now, a research team led by Carl June at the University of Pennsylvania has pioneered a potentially simpler and cheaper alternative.

Instead of modifying T-cells outside the body, the new technique delivers RNA instructions via fatty capsules directly into the bloodstream. These capsules are coated with proteins that help them enter T-cells, temporarily giving the cells the ability to detect and destroy cancerous B-cells. In preclinical experiments, this method worked remarkably well in mice implanted with human cancer cells. Mice that received higher doses showed almost complete eradication of tumour cells within three weeks and experienced no side effects.

The approach also showed promise in 22 healthy monkeys, where the treatment quickly wiped out all B-cells in just one day. Most monkeys tolerated the therapy well, although one experienced a serious inflammatory reaction. Importantly, the therapy only offers a temporary effect, as the RNA instructions degrade within a week, meaning repeated doses may be necessary if the cancer returns.

Experts, including Karin Straathof of University College London, are optimistic about the potential to simplify and reduce the cost of CAR T-cell therapy. However, she notes that long-term protection—a hallmark of traditional CAR T therapy—may be lacking in this approach. Clinical trials will be crucial to determine its safety and effectiveness in humans. Encouragingly, June confirmed that the first human trial has already begun.

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Carl June

Professor at Perelman School of Medicine, University of Pennsylvania

University of Pennsylvania

Private Ivy League research university in Philadelphia, Pennsylvania

Topics mentioned on this page:
CAR-T Therapy, Drug Discovery
CAR T-cell therapy created inside the body shows promise in animal trials