A particularly promising scientific development for the treatment of advanced solid tumours in children is highlighted in a post by Dr. Loizos Loizou, referring to a study published in August in the New England Journal of Medicine. The study involves experimental targeted therapy with modified T-cells, which resulted in complete remission of a 17-year-old patient with relapsed and widely metastatic nephroblastoma.
The doctor's post is as it is
New hope in Paediatric Oncology: A great scientific development
A highly promising study published in the New England Journal of Medicine (NEJM, August 2026) opens up new avenues for treating advanced solid tumours in children through targeted T-cell therapy.
The clinical case:
A 17-year-old patient with relapsing, widely metastatic nephroblastoma (Wilms tumour) with localizations in the brain, lungs, liver, and abdomen, had exhausted all conventional treatment options.
Innovation – Targeting PRAME Protein:
Researchers at the Heidelberg Paediatric Cancer Center in Germany (KiTZ) applied an experimental approach by targeting the PRAME protein, a marker expressed in some solid tumours:
The treatment:
A single injection of the patient's own modified T-cells, programmed to attack PRAME.
Immediate Response:
A biopsy just 9 days after the infusion showed massive invasion of T-cells into the tumour and widespread necrosis of the cancer cells.
Result:
At 3 months, no live cancer cells were detected. More than a year later, the patient remains in complete clinical and radiological remission.
Why it's important:
Although cellular immunotherapies have revolutionized paediatric leukaemia, their application to solid tumours has hitherto encountered major obstacles.
This success proves that targeted immunotherapy can force even very aggressive solid tumours to completely recede. To further evaluate the treatment, a larger clinical trial (PRAMEtime) is already planned for 2027.
The battle against childhood cancer is difficult, but scientific research proves to us every day that no goal is unattainable.
We continue with faith and optimism!
