A new, experimental drug, produced by Roche Holding AG tested well in early stage, Phase I clinical trials. The Roche drug showed promise in treating children’s malignant brain tumors caused by medulloblastoma.
The new medication – named GDC-0449 – belongs to the category of drugs known as Hedgehog signaling pathway blockers, involving proteins, which contribute to cell growth.
The research was released at a gathering of the American Society of Clinical Oncology and showed Phase 1 clinical trials suggested the new drug showed promise for treating drug-resistant and recurrent medulloblastoma.
Encouragingly, a single participant in the study had no progression of the cancer for over a year.
Curis Inc, in a deal with Roche, would profit from the drug’s continued production.
According to the director of neuro-oncology at St. Jude Children’s Research Hospital in Memphis, Tennessee, Dr. Amar Gajjar, “less than 5 percent of these children (with medulloblastoma) survive if they fail primary treatment.”
Gajjar said 20 percent of medulloblastomas are associated with the hedgehog pathway, which will be the focus for further studies of the new medication.
GDC-0449 also proved encouraging for the treatment of adult basal cell carcinoma and there is another Phase II trial in the works to test the drug’s efficacy on adult medulloblastoma.
