Only 20 per cent of children with a rhabdoid tumour live past five years. That figure comes from Alfonso Alba Bernal, a researcher from Rincón de la Victoria on the eastern Costa del Sol, who is now working on a different answer in a Madrid laboratory.
Alba Bernal is doing postdoctoral research with the paediatric oncohematology group at La Paz University Hospital. He leads a project aimed at rhabdoid tumours, working with a highly qualified team and with Antonio Pérez Martínez, head of the Pediatric Hematology-Oncology Department at La Paz.
A rare and aggressive cancer
“They are rare, almost always appear in the first years of life and are very aggressive,” Alba Bernal says. Some sit in the brain, where they are called atypical rhabdoid teratoid tumours. Others start in the kidney or liver. Those are the extracranial malignant rhabdoid tumours.
Doctors rely on surgery, chemotherapy and, when they can, radiotherapy. “But it’s insufficient and carries significant side effects for such young children,” he says. “We don’t have specialised treatments for these types of tumours.”
Part of the difficulty is that the tumours are genetically simple. “Unlike other cancers full of mutations, rhabdoids depend primarily on the loss of a single gene, which makes them aggressive and difficult to treat,” he says. Yet he sees an opening in that simplicity. It raises, in his words, the question of whether the patient’s own body can be trained to find a tumour that shows recognisable signals on its surface.
Training the body’s patrols
The team is working on CAR-T therapy, short for Chimeric Antigen Receptor T-cells. Alba Bernal describes T lymphocytes as patrols that move around the body looking for anything that does not fit and eliminating it. Many tumours slip past them unseen.
The method takes T lymphocytes from the patient to the laboratory, where genetic engineering adds a new receptor. He compares it to “a pair of custom-made glasses” that lets the cell recognise a specific signal on the tumour. The trained cells multiply and go back into the patient to hunt the tumour down.
It has already changed the outlook for some childhood leukaemias. Solid tumours such as rhabdoids are “much more difficult”, he says, “and that’s precisely where the team at La Paz is working.” In the Madrid lab he and his colleagues design and test CAR-T cells aimed at signals found on rhabdoid tumours. The group belongs to the CRIS unit for advanced therapies in childhood cancer.
Funded by a mother’s loss
The project exists because of Sara, whose son Manuel died of one of these tumours. According to Alba Bernal, she “transformed part of her grief over her son’s death into strength”, raising money to set up a research line for this type of cancer.
He hopes to develop CAR-T therapies that can be used alone or alongside targeted drugs, to make them work better against rhabdoid tumours. The work is still at an early stage. “Currently the studies are being carried out in cell lines, so there is still a long way to go before reaching clinical trials with patients,” he says.
Rare cancers get little research, he argues. There are fewer patients, less funding and fewer dedicated groups. Projects like this one survive on support from families like Manuel’s and from foundations such as Cris Against Cancer, which step in where industry does not. The problem, he says, is small in number of cases but enormous for each family that lives through it.
Consult your doctor for medical advice.
