Scientists have made a breakthrough in finding out the cause of a rare childhood cancer which could lead to more effective treatments.
Rhabdomyosarcoma (RMS), which develops in the muscles, affects around 60 youngsters in the UK each year.
Now academics from Harvard University and experts from Aberdeen University have pinpointed a protein that plays a key role in triggering the disease.
The findings of their study, which also involved The Institute of Cancer Research, London, and the Swiss Institute of Bioinformatics, have been published today.thurs
They discovered that excessive activity of the Yap protein caused muscle stem cells to divide instead of becoming normal muscle tissue.
These Yap muscle cells fail to develop into normal muscle tissue and lead to the development of RMS. The scientists were also able to prove that Yap is hyper-active in a large proportion of human RMS cases.
Most RMS tumours occur in children younger than 10 years old.
Dr Henning Wackerhage, from Aberdeen University’s Institute of Medical Sciences, said the discovery is the first step to understanding how the rare disease can be targeted.
It is hoped they will able to find out exactly how the protein works in cancer and how its activity can be controlled.
Dr Wackerhage said: “In the last two years we found that certain drugs had an effect on this protein. The question is, can this drug be used in human cancer to control this protein without having too many side effects.
“If we can achieve the inhibition of Yap locally in the tumours, we could cause the cancer to stop and regress by turning the RMS into normal muscle instead. This would most likely produce significantly less side effects than the current therapies.”
Other research has shown that the Yap protein is also active in liver and skin cancers.
Dr Wackerhage added: “These results could therefore be of wider significance in also enhancing our global understanding of the role of Yap in cancer.”
