Immunotherapy trial will deliver CAR T-cells directly into brain

By Flora Southey

- Last updated on GMT

Getty/Meletios Verras
Getty/Meletios Verras
Infusing CAR T-cells directly into the brain, rather than into the blood intravenously, could improve the immunotherapy’s effectiveness and produce fewer side effects, say researchers.

Scientists at Seattle Children’s are trialling an immunotherapy in children with HER2-expressing relapsed central nervous system tumours that directly delivers chimeric antigen receptor (CAR) T-cells into the brain.

At least 18 patients will be recruited for the BrainChild-01 trial and, depending on the location of their tumour, will have T cells infused through a catheter into the tumour resection cavity or into the central nervous system (CNS) ventricular system.

According to researchers, the direct drug delivery method means T cells will not need to pass through the blood-brain barrier, “which often prevents drugs from getting into the brain at the necessary concentrations.” ​ 

Scientists also hope that by limiting the T cells’ presence throughout the broader bloodstream, patients may have fewer side effects, such as neurotoxicity and cytokine release syndrome.

“To our knowledge, it is the first time it will be tested in children,” ​a Seattle Children’s spokesperson told us.

“BrainChild-01 is the next step in Seattle Children’s quest to harness the immune system and bring better therapies and cures to children worldwide.

“With the vast array of tumour types and more aggressive tumours, this research may open new doors to treatment strategies that involve targeting different or multiple proteins simultaneously,” ​she added.

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