From tumour to muscle cell: BTU researcher discovers a new approach to treating childhood cancer

This approach could in future complement chemotherapy and radiotherapy for embryonal rhabdomyosarcoma – one of the most common soft-tissue tumours in children. The study, funded by the Wilhelm Sander Foundation, has been published in the journal “Molecular Therapy”.

A protein that acts as both a brake and a lever

Rhabdomyosarcomas arise from muscle progenitor cells, can occur anywhere in the body and, due to their variable location, are often difficult to remove completely. The team led by Prof. von Maltzahn, who has held the chair for Age-Related Stem Cell Biology at the BTU since 2022, identified one of the reasons for this unchecked growth: the protein TRPS1.

“Through our studies on human tumour cells from embryonic rhabdomyosarcoma, we discovered that the level of the transcriptional repressor TRPS1 is consistently elevated,” explains the researcher. “TRPS1 blocks the expression of precisely those genes that muscle cells normally need to mature. The cells remain stuck in an immature, rapidly proliferating state.”

The idea: halting growth

This finding provided the decisive impetus: “This led us to the idea that by reducing the level of TRPS1 in the tumour cells, we might be able to slow down tumour growth,” says von Maltzahn.

The team succeeded in demonstrating this – both in cell culture and in a mouse model: if TRPS1 is specifically reduced, the tumour cells differentiate into mature muscle cells that can no longer divide. The tumour loses its ability to grow indefinitely.

Prof. Dr Julia von Maltzahn says: “We believe we have found an effective, novel therapeutic approach which, in future – in addition to chemotherapy and radiotherapy – could be used to halt the growth and spread of the tumour.”

BTU as a centre for translational cancer research

The study was conducted at the interface between basic and applied research: Originally initiated at the Leibniz Institute for Ageing Research (FLI) in Jena and funded at the BTU as part of the Wilhelm Sander Foundation, it was further developed into a joint project between the two institutions following Prof. von Maltzahn’s move to the BTU in 2022. It serves as a prime example of how BTU’s relatively new Faculty of Health Sciences is playing a leading role in cancer research.

TRPS1 is now regarded as a promising therapeutic target protein for the treatment of embryonal rhabdomyosarcoma – opening up prospects for new, complementary therapeutic strategies.

Publication: Hüttner SS, Henze H, Elster D, Koch P, Anderer U, von Eyss B, von Maltzahn J. *A dysfunctional miR-1-TRPS1-MYOG axis drives ERMS by suppressing terminal myogenic differentiation.* Mol Ther. 2023. DOI: 10.1016/j.ymthe.2023.07.003