new treatment for untreatable childhood cancer Flashcards
1
Q
Stone man syndrome
A
- muscles and ligaments turn to bone
- children only live 9-12 months after diagnosis
- since it occurs in brainstem/pons area of the brain DIPG tumours cant be removed surgically and chemotherapy doesn’t work
2
Q
new treatment
A
- targets the protein molecule produced by mutated versions of the ACVR1 gene found in deadly childhood brain cancer
- these mutations don’t exist in other types of cancer, mutation occurs in 1/4 of tumours
- two drug prototypes were found to be particularly good at blocking signals sent out by ACVR1, kills mutant cells while having little effect on healthy cells
- trial in mice showed survival increase by 25%
3
Q
the activin A connection
A
- looking at cells in the lab it is seen that ACVR1 mutant cells respond inappropriately to a molecule called activin A which sparks a cascade of events that trigger tumour growth\
- activin A is present in high levels during brain development
4
Q
why is this important
A
- illustrates how gaining a detailed understanding of the biology of cancers allows us to move very quickly to the discovery of new treatments
