(September 4, 2009 - Insidermedicine)

Blocking a genetic pathway known as the Hedgehog signaling pathway is showing promise for the treatment of some advanced cancers, according to three studies published in the September 3rd issues of the New England Journal of Medicine and Science Express.

Here is some information about basal cell carcinoma:

•    It is a type of skin cancer

•    It is the most common of all cancers

•    Its major cause is sun exposure

Researchers from John’s Hopkins University in Baltimore gave an experimental Hedgehog signaling pathway inhibitor known as GDC-0449 to over 30 patients with advanced basal cell carcinoma. Just over half of the participants responded to the drug, meaning their tumors shrank by at least 30%. After an average follow-up of just over 10 months, the remaining patients had stable disease or saw their cancer progress.

In a separate study, the same team of researchers gave GDC-0449 to a patient with advanced case of a rare form of brain cancer known as medulloblastoma. This patient’s cancer had recurred after initial treatment and was not responding to other therapies. The GDC-0449 resulted in dramatic improvements in the patient’s functioning and pain level, but it was short-lived. Two months later, the cancer started to grow again, and the patient soon died. Additional research identified a genetic mutation acquired by the cancer that led to resistance to the drug.

Today’s research highlights the potential of a new target for the treatment of some types of cancer, although problems with resistance will need to be overcome.