June 5, 2010 / 1:05 PM / in 8 years

Curis, Roche drug combats children's brain tumors

* One patient on the drug for more than a year

* Hedgehog pathway at play in 20 pct of medulloblastomas

CHICAGO, June 5 (Reuters) - An experimental drug being developed by Curis Inc (CRIS.O) and Roche Holding AG ROG.VX 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 promising new class of drugs that block the Hedgehog signaling pathway, which involves several proteins that play a role in cell growth.

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

The hedgehog pathway has been shown to be responsible for 20 percent of medulloblastomas, as well as other cancers, ASCO said in a statement.

    “Some patients have remained on this trial for extended periods of time and our early results are positive and encouraging,” Dr. Amar Gajjar, director of neuro-oncology at St. Jude Children’s Research Hospital in Memphis, Tennessee and the trial’s lead investigator, said in a statement.

    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. (Reporting by Deena Beasley; editing by Andre Grenon)

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