New Compound Shrinks Skin Cancers

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But another researcher warned that it is premature to get too excited about the results.

"It's great to see something with so much potential, but it really is potential," said Dr. Clifford Perlis, director of Mohs Micrographic Surgery and Dermatologic Surgery at Fox Chase Cancer Center in Philadelphia.

However, he added, "there are other companies developing Hedgehog inhibitors as well, so I think people should be paying attention to this."

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The Hedgehog gene, so named because it was first discovered in flies with hair resembling the spikes of hedgehogs, "is really important during early embryonic development in pretty much all animal species from flies to mice to humans, and for pretty much every tissue you can imagine," explained Frederic de Sauvage, also an author on both papers. "But remarkably in adults, it seems to be mostly turned off."

De Sauvage is vice president of research, molecular biology, at Genentech, which developed the molecule and funded the study.

Some 1 million Americans get basal cell carcinoma each year. It often doesn't spread but, once it has, there are no approved treatments. Medulloblastoma is an aggressive form of brain tumor.

The first trial enrolled 33 individuals whose basal cell carcinoma had spread locally or to distant organs.

Half of the participants who had distant metastases saw a reduction in tumor size, as did 60 percent of those with locally advanced cancer. The rest had either stable or progressive disease after 10 months of follow-up.

But "stable" in this population may not mean much, Perlis pointed out, as it is generally a very slow-growing cancer.

The man with medulloblastoma also saw a significant shrinkage of his tumor, along with vastly improved quality of life, but only for two months. He later died.

A third study by some of the same authors, this one published online Sept. 2 in Science Express, discovered that treatment with GDC-0449 actually spurred another mutation in a gene called SMO, which caused the brain tumor to become resistant to the drug.

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