Lurie Children's Hospital of Chicago found that these tumours' growth and tendency to metastasize are regulated by a protein kinase called Polo-like kinase 4 (PLK4), which is increased in AT/RT.
They also have demonstrated that an experimental drug, a PLK4 inhibitor, stopped tumour growth.
'This is the first time that PLK4 has been described as a therapeutic target for brain tumours or in paediatric cancer,' said lead author Simone T.
Sredni and team were able to identify PLK4 as a potential target for treatment by using a novel gene editing technology called CRISPR/Cas9.
Sredni and colleagues also found that the PLK4 inhibitor (CFI-400945) was safe for normal tissue, while attacking the cancer cells.
In the first case study we end up with the marker genes for (i) the proliferation activity composed of 12 genes, CDC20, TK1, KNL1, CENPE, STIL, ANLN, NDC1, NUF2, KIF20A, PLK4
, CCNB1, and CCNA2, and (ii) the quiescence state composed of 12 genes: COL5A1, TGFBI, TCEA2, WNT9A, MMP11, LAMB1, KRT14, LTBP1, PHLDB1, TIMP3, LRP1, and COL18A1.