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Development of new therapeutic treatments targeting cancer stem cells.

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A new approach to destroying cancer

With the support of EU funding, Leukos Biotech has developed and validated a family of drugs for differentially killing leukaemia stem cells while sparing healthy haematopoietic stem cells.

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Every year, nearly 18 000 European adults are diagnosed with acute myeloid leukaemia (AML), a type of blood cancer that is notorious for being difficult to treat. In fact, more than 60 % of patients fail to respond to traditional treatments such as chemotherapy – underlying the critical need for new ways to treat this disease. For Leukos Biotech, the answer may be in stem cells. “Leukaemia stem cells are a distinct population of tumour cells that are capable of maintaining and reinitiating the disease,” explains Leukos Biotech co-founder and chief scientific officer Ruth Risueño. With the support of the EU-funded StemTarget project, Risueño is zeroing in on these stubborn cells. “The idea is to develop a novel approach that specifically eradicates leukaemia stem cells while maintaining normal haematopoiesis,” she adds.

Immune to the effects of chemotherapies

As Risueño explains, what makes AML so hard to treat is that leukaemia stem cells are immune to the effects of chemotherapies. “Instead of dividing, as is typically the case when a cancer cell is exposed to chemotherapy, leukaemia stem cells remain dormant and have the potential to ‘wake up’ and cause a cancer relapse,” she says. “They can even cause the leukaemia to become more aggressive or progress to an anticancer treatment resistant form.” The main goal of the StemTarget project, which received support from the Marie Skłodowska-Curie Actions programme, was to destroy these cancer stem cells.

A promising antineoplastic effect

To accomplish this goal, researchers conducted a comprehensive in silico screening of already human-approved drugs that may differentially kill leukaemia stem cells while sparing healthy hematopoietic stem cells. They also tested various antagonists of dopamine and serotonin receptors, both of which are believed to contribute to AML. After three years of research, the project identified a chemical family of small molecules with a promising antineoplastic effect.

High potential for treating haematological cancers

Using these molecules as a foundation, Leukos Biotech is designing and validating new drugs with high potential for successfully treating haematological cancers. The drugs are unique in that they work by blocking the neurotransmitter receptors of leukaemia stem cells. “We succeeded at validating a new therapeutic target, the serotonin receptor, for haematological cancer,” concludes Risueño. “We also identified a new family of drugs that differentially block this target and, in doing so, eliminate cancer cells.” The company is continuing to work on the clinical development of this family of drugs, with the goal of starting clinical trials in the near future.


StemTarget, leukaemia, stem cells, tumour, disease, acute myeloid leukaemia, cancer, chemotherapy

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