An experimental drug being developed by Roche Holding AG showed promising results in a small, early stage trial involving children with medulloblastoma, the most common type of malignant brain tumor in children.

The drug, GDC-0449, is part of a new class of medicines that block the Hedgehog signaling pathway, which involves several proteins that play a role in cell growth. Under a collaboration with Roche, Curis would receive payments related to the drug's development.   

The Phase 1 trial results, presented at a meeting of the American Society of Clinical Oncology, showed the drug was well tolerated by 12 of 13 children with recurrent or drug-resistant medulloblastoma, with one patient remaining on the drug for more than a year without disease progression.   

"Less than 5 percent of these children survive if they fail primary treatment," said Dr Amar Gajjar, director of neuro-oncology at St. Jude Children's Research Hospital in Memphis, Tennessee, and the trial's lead investigator.   

He noted the hedgehog pathway was responsible for about 20 percent of medulloblastomas, and future trials will involve only those patients whose tumors have activated hedgehog pathways.

Previous early stage studies have suggested that GDC-0449 may also be effective against basal cell carcinoma in adults and there is currently a Phase II trial being conducted for adults with recurrent medulloblastoma.   

Curis and Roche will also be presenting safety data at ASCO from a Phase 2 trial of GDC-0449 in patients with colorectal cancer. - Reuters