An experimental cancer drug being developed by Genentech has proven strikingly successful in a small trial of patients suffering from two rare forms of cancer. And scientists in the field say that the results demonstrate that the hedgehog pathway can blaze a trail for a new class of therapies that target a variety of hard-to-treat tumors.
In one study the drug was credited with shrinking tumors in 18 of 33 patients with basal cell carcinoma, or advanced skin cancer. And the therapy--GDC-0449--produced a sudden turnaround in the health of a 26-year-old patient who had fought a losing, four-year battle with medulloblastoma, a rare brain cancer. The dramatic effect, though, only lasted a few months and the patient died last fall.
Researchers at Genentech--which is collaborating with drug developer Curis--started out with the knowledge that the vast majority of basal cell carcinoma cases are triggered by a mutation in the hedgehog pathway. And their discovery has implications in the treatment of colon, pancreatic and ovarian cancers, which can also prove hard to treat. The therapy is now in Phase II. Merck and Infinity Pharmaceuticals also have development programs that target the hedgehog pathway.
- read the article in the Wall Street Journal