Children with acute lymphoblastic leukemia (ALL) for whom other treatments no longer work have been treated with CAR T-cell therapy since 2019. For this form of immunotherapy, the own T-cells, a type of white blood cells, are filtered out of the blood. The T-cells are changed in the lab so that they recognize a code on the leukemia cells and specifically attack them. The treatment has to be manufactured for each individual child, currently by a pharmaceutical company in the United States or in other locations abroad. That is not only expensive, but also time-consuming – it takes about four weeks before the modified cells are back in Utrecht. In the meantime, the child receives treatments to keep the leukemia under control.
Taking matters into your own hands
‘We have chosen to take matters into our own hands, in order to make CAR-T available to increasingly many children with cancer,’ says dr. Friso Calkoen, pediatric oncologist and closely involved in the CAR-T program at the Princess Máxima Center. ‘Last year, thanks to the Princess Máxima Center Foundation, we purchased a device to make CAR T cells ourselves. Since then, we have been working with UMC Utrecht on a careful process to commission and test the device, the Prodigy. In 2023, we expect to start a study on CAR T-cell therapy for children and young adults with ALL or non-Hodgkin's lymphoma (NHL) who are not yet eligible for CAR-T treatment or who don’t respond well to that treatment.’
It is expected that, thanks to research, CAR-T can be used in increasingly many types of cancer in children and adults. Partly for this reason, the Princess Máxima Center Foundation is raising funds for the purchase of a second Prodigy. The Prodigy can currently be found in the UMC Utrecht: the device is used in collaboration with the adult hematology department there. With the recently announced construction of the sixth and seventh floors of the research building, the Princess Máxima Center is making room for a dedicated cell therapy facility – also in collaboration with UMC Utrecht – to house the production of and research into these treatments.
Cleanrooms
Dr. Lidwien Hanff, Head of the Pharmacy at the Princess Máxima Center: ‘With the cell therapy facility on the sixth floor of our research building, we will have our own ‘immunotherapy factory’. It will be a production environment with four so-called cleanrooms in which we can manufacture our own immunotherapy. Unlike conventional drugs, cell therapies are made from living cells from each individual patient. That is why the safety requirements are very strict. This aims to prevent contamination at all costs and to guarantee quality. Specialized technicians, researchers and quality officers will soon be working in the completely sterile rooms. They are well-trained, both on quality and safety requirements and on the complex techniques used to make CAR T-cells and other cell therapies.’
Research
Research will also play an important role in the cell therapy facility. While CAR-T has led to a cure for some children, the treatment does not work well for other children. Studies are also already underway to develop cell therapies for different forms of childhood cancer. This research will be further expanded in the cell therapy facility.
Friso Calkoen: ‘For the children for whom CAR-T has already worked, this treatment is a real game-changer: we can now even cure some children with advanced ALL. It is great to see the Máxima Center’s expansion plans focusing on cell therapy as a treatment of the future. With the construction of our own facility to manufacture and further study this therapy ourselves, we are taking a big step towards bringing this future closer to an increasing number of children with cancer.’
The month of June is all about new perspectives at the Máxima. This means that in the coming weeks we will tell you all about the developments in our research hospital. In the Máxima, care providers and researchers work closely together to constantly improve treatments. Our mission: to cure every child with cancer, with the best possible quality of life.