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Today in Mice

April 2, 2009

A New Drug to Fight Portal Hypertension?

The drug sorafenib — already approved in several countries for treatment of kidney and liver cancer — dramatically improves the health of rats with liver cirrhosis and advanced portal hypertension.


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Although portal hypertension, the most significant complication for patients with liver cirrhosis, can become life-threatening, doctors do not have many effective treatment options available to them.

A new study in the April issue of Hepatology, however, suggests the drug sorafenib —  already approved in several countries for treatment of kidney and liver cancer — dramatically improves the health of rats with the condition.

In the experiment, rats took sorafenib orally every day for two weeks and displayed no adverse effects from the treatment. The drug is designed to inhibit the growth of new blood vessels, and researchers observed an 80 percent decrease in the areas around the liver where fibrosis and inflammation also improved.

While the researchers stress that a “very careful approach” is needed in potential human trials of the drug, they encourage their colleagues to more closely examine the role of similar therapeutic agents that block blood vessel growth — called antiangiogenesis therapy — in patients with cirrhosis and portal hypertension.

“Taking into account the limitations of translating animal study results into humans, we believe that our findings will be stimulating for consideration of sorafenib as an effective therapeutic agent in patients suffering from advanced portal hypertension,” write the study authors, led by Marc Mejias and Mercedes Fernandez from the August Pi i Sunyer Institute of
Biomedical Research
in Barcelona.

An editorial in the same issue of Hepatology by Vijay Shah of the Mayo Clinic and Jordi Bruix of Barcelona, titled “Antiangiogenic therapy: Not just for cancer anymore?” hails the findings. “It is obvious that a new avenue for pharmacologic intervention in patients with cirrhosis has emerged,” they conclude.

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