Childhood brain cancer linked to a hedgehog

Curis, a therapeutic company from Cambridge, MA, US, has released a study demonstrating that human medulloblastoma, the most common childhood brain malignancy, is strongly correlated with deletion of a gene that encodes a suppressor of the Hedgehog signalling pathway.

The authors suggest that elimination of this suppressor results in abnormal activation of the Hedgehog pathway, thus promoting medulloblastoma tumour growth.

Dr Lee Rubin, Curis' vice president and chief scientific officer, said: 'This study provides strong corroborative evidence that the mechanism by which certain cancers promote their tumour growth is by exploiting some means of up-regulating the Hedgehog signalling pathway, in this instance by removal of a normal Hedgehog pathway suppressor. We believe that Hedgehog pathway inhibition technologies will constitute an important and promising approach to the potential treatment of medulloblastoma and other cancers.'

Curis has developed several cancer drug candidates designed to block or antagonise abnormal activation of the Hedgehog pathway. Over the last several years numerous other scientific publications have implicated abnormal Hedgehog pathway activation with the progression of basal cell carcinoma, small cell lung cancer, pancreatic cancer, gastrointestinal cancers, and other cancers.

This current scientific report is entitled: 'RENKCTD11 is a Suppressor of Hedgehog Signalling and is Deleted in Human Medulloblastoma.' In the study, the authors also demonstrate that the REN suppressor can inhibit the proliferation of medulloblastoma cells and can suppress tumour growth in a preclinical model of medulloblastoma. Based on these results, the authors suggest that deregulation or inhibition of the tumour promoting activities of the Hedgehog pathway represents a potential target for therapeutic intervention.