Alpha Stories

Published on Friday, June 4, 2010

Anti-seizure drug improves liver disease in Alpha-1 mice

A note from Robert A. Sandhaus, MD, PhD, clinical director of the Alpha-1 Foundation and medical director of AlphaNet:

In the interest of presenting the latest information to our Alpha-1 community, the following press release has been posted here. We would like to point out some issues in considering the information presented in this release.

First, this is not a scientific article, although it refers to one, but rather a “story” written by a communications person at the Pittsburgh medical center. With respect to the research described, this is very preliminary research. The article discusses work done on cells in a culture dish and a mouse model of Alpha-1 Antitrypsin Deficiency.

When they mention that the drug used in this study has a “well-understood safety profile,” it is important to note that Tegretol’s well understood safety-profile includes a “black box” warning from the FDA that it can cause aplastic anemia (loss of the ability of the body to make red blood cells in the bone marrow), agranulocytosis (loss of the ability of the body to make white blood cells in the bone marrow), and skin reactions so severe that they have been fatal.

This is in addition to the many additional side effects that have been described with this drug. Having pointed out these issues, this remains a very promising initial set of experiments published by one of the major experts in the field of Alpha-1 liver disease. We certainly look forward to seeing additional information on this line of research.

Article published on
The liver scarring of Alpha-1 Antitrypsin Deficiency, the most common genetic cause for which children undergo liver transplant, might be reversed or prevented with a medication that has long been used to treat seizures, according to findings from Children’s Hospital of Pittsburgh and the University of Pittsburgh School of Medicine to be published in Science.

The drug, carbamazepine, or Tegretol, was tested in mice.

Because the anti-seizure drug is familiar to doctors and has a well-understood safety profile, clinical trials could begin immediately to see whether it can help patients with Alpha-1, said senior author David H. Perlmutter, MD, physician-in-chief and scientific director, Children’s Hospital.

Complete article