A single gene gone wrong. That is the core problem with rhabdoid tumours. And it is the core clue that a Spanish research team is now chasing.
Alfonso Alba Bernal, a researcher from Rincón de la Victoria on the Costa del Sol, is working at La Paz University Hospital in Madrid. His focus: paediatric rhabdoid tumours.
These are rare. They are aggressive. They strike very young children.
Most cancers are genetic messes. They accumulate mutation after mutation.
Rhabdoid tumours do the opposite. They depend primarily on the loss of one gene. That makes them both vicious and, potentially, vulnerable.
“Unlike other cancers full of mutations, rhabdoids depend primarily on the loss of a single gene, which makes them aggressive and difficult to treat,” Alba Bernal said. Current treatment is blunt. Surgeons cut.
Chemotherapy poisons. Radiotherapy burns.
For infants and toddlers, the side effects are severe. And the survival rate is brutal. Only 20 percent of patients live more than five years.
“But it’s insufficient and carries significant side effects for such young children. The problem is that we don’t have specialised treatments for these types of tumours and with current therapies, only 20 per cent of those who suffer from them survive more than five years,” Alba Bernal said.
The research team is asking a different question. If the tumour is genetically simple, does it display simple, recognisable signals on its surface? And if so, can the child’s own immune system be trained to find those signals?
That is the premise behind CAR-T therapy. The patient’s own T cells — the immune system’s hunters — are extracted. They are genetically reprogrammed to recognise the tumour.
Then they are infused back into the child. The hope is that these engineered cells will hunt down and destroy the cancer.
Alba Bernal is leading the project. He works with a highly qualified team and in collaboration with Antonio Pérez Martínez, head of the Pediatric Hematology-Oncology Department at La Paz. The work is postdoctoral research within the paediatric oncohematology group at the hospital.
The tumours can appear in different places. Some are in the brain — atypical rhabdoid teratoid tumours.
Others arise in the kidney or liver. Those are called extracranial malignant rhabdoid tumours. The genetic simplicity is the paradox.
It makes the tumour hard to treat with conventional tools. But it also gives researchers a clear target. One gene.
One surface signal. One chance to train the immune system.
The stakes are high. These are children, often in their first years of life. The current 20 percent survival rate is a wall.
The team is trying to break through it. CAR-T therapy is not new.
It has been used for some blood cancers. But applying it to solid tumours in children, especially rare ones like rhabdoids, is still experimental. The Madrid lab is pushing into that territory.
The work is happening now. Alba Bernal is based in Madrid, but his home is Rincón de la Victoria. The shift in childhood cancer treatment, if it comes, will start inside that lab.
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