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€1M KWF funding for research into neuroblastoma and leukemia

The Princess Máxima Center will receive €1 million in project funding from KWF for two lines of research. With this, the KWF supports research by dr. Frank van Leeuwen into a new, targeted treatment strategy for babies with acute lymphoblastic leukemia. Prof. dr. Marc Wijnen will use the money for an early clinical trial into fluorescence-guided surgery for neuroblastoma.

The latest round of grant awards from KWF was announced today. A total of 19 studies were financed in the KWF subsidy round for research in the Development & Implementation phase.

Targeted therapy for babies with leukemia

The majority of children with acute lymphoblastic leukemia (ALL) are cured. But an aggressive form of the disease that usually affects babies is still difficult to treat. In this form of ALL there is a specific fault in the DNA: a so-called MLL fusion gene. In previous research, Frank van Leeuwen's group discovered that this MLL leukemia is much more dependent on methionine than healthy body cells. This nutrient is an amino acid that is needed for cell growth. It’s mainly accessed through food.

‘The question now is whether we can exploit the difference in dependence on methionine in the form of a treatment,’ says Van Leeuwen. He will carry out the KWF-supported research with colleagues dr. Ronald Stam and dr. Laurens van der Meer. ‘We aim to find out whether we can inhibit the growth of the leukemia by limiting methionine uptake. This can be done either with a methionine-restricted diet, or with a drug that breaks down methionine. We also aim to look at targeted drugs that make the leukemia cells even more sensitive to this methionine deficiency. That could help make this treatment strategy even more effective. We expect that this form of treatment will be effective, and will have far fewer harmful side effects than the chemotherapy that is currently used.’

The research by Van Leeuwen and colleagues is still at a preclinical stage, using cells in the lab and studies with mice. Van Leeuwen: ‘Methionine-free food is already approved and available for babies. If we can indeed demonstrate in lab research that our approach works, we can relatively quickly bring the new treatment strategy into the clinic, to help babies with this difficult-to-treat form of leukemia.’

Operate using luminescent tumor tissue

Neuroblastoma is a tumor of the nervous system that mainly affects children under six years of age. Children are treated with a combination of chemotherapy, immunotherapy, and surgery. The operation is difficult: four in ten children have a complication. In many cases, there is still some tumor tissue left after the operation.

Marc Wijnen’s research group, in collaboration with the Rios group, has developed a way to light up the neuroblastoma tissue. A fluorescent substance binds to a neuroblastoma-specific ‘flag’, GD2. With a special camera, the neuroblastoma tissue is lit up and visible to the surgeon, in so-called fluorescence-guided surgery. Marc Wijnen: ‘This allows us as surgeons to more accurately tell neuroblastoma tissue apart from healthy tissue. We hope this enables us to remove as much tumor tissue as possible while minimizing damage to healthy tissue – reducing the number of complications. The KWF grant will support an early clinical study into this approach.’

In this phase I/II study, the researchers will look at the safety and right dose of the fluorescent substance that lights up the neuroblastoma cells. A larger, global study with more children is needed to determine whether fluorescence leads to better surgery. Wijnen: ‘We hope to show that fluorescence indeed helps pick out neuroblastoma cells during surgery. With this research we are taking an important step towards tailor-made surgical treatment for children with neuroblastoma.’