New treatment for cancer: The Hedgehog pathway

-There is an exciting new treatment for cancer.  The new medication inhibits a cellular mechanism called the ‘HEDGEHOG SIGNALLING PATHWAY’ which is an important regulator of in embryonic development and that becomes reactivated in cancer cells.
Inhibiting this mechanism is like a magic bullet directed at cancer cells.

· Cancer is the second most common cause of death in the United States.  About 1.5 million cases of invasive cancer per year are diagnosed in the United States.

· Investigators report dramatic results in the initial testing of the new medication, published in the latest issue of the New England Journal of Medicine.
Study authors describe 2 medical trials in which they used the new medication.
The first was for Basal Cell Carcinoma of the skin in which the medication was given to 33 patients with very advanced cancer.  In the second study, the new medication was given to patients with a rare form of cancer, metastatic medulloblastoma.
In both studies the preliminary reports suggest dramatic response rates of the cancer to the new medication.

· The new medications appears to block this ‘hedgehog signalling pathway’ which is thought to play a role in as many as 30% of all cancer including:
PANCREATIC CANCER
GASTROINTESTINAL CANCER
COLON CANCER
OVARIAN CANCER
PROSTATE CANCER
LEUKEMIA
MULTIPLE MYELOMA

The new medication appears to be well tolerated and can be given by mouth once a day.  The new medication is being developed by Genentech under the investigational drug name: GDC-0449.

This is an important finding and shows us a new and unique way of treating cancer.  The medication is still in the initial stages of testing and further studies are needed to clarify the effectiveness and safety of the medication.

Cristopher Geiler, M.D.
Reference
N Engl J Med September 2, 2009.

Posted on September 9, 2009

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