AASLD/Alpha-1 Foundation Liver Scholar Award
The AASLD/Alpha-1 Foundation Liver Scholar Award was established this year in recognition of the 50th anniversary of the discovery of Alpha-1 Antitrypsin Deficiency.
Dr. Andrew Chu of the Children's Hospital of Pittsburgh of UPMC was selected to receive the 2013 AASLD/Alpha-1 Foundation Liver Scholar Award for his research Mechanisms of action for autophagy enhancer drugs on alpha-1-antitrypsin deficiency-associated liver disease.
Dr. Chu states, "In this grant, I will follow up on recent studies from my mentor's lab that identified a drug, carbamazepine (CBZ), which stimulates liver cells to destroy the mutant protein and reverse liver scarring in a mouse model of the disease. I will use what is known about the chemistry and action of CBZ to discover additional candidate drugs for treatment of this disease."
Further, Dr. Chu plans to, "… make cell lines from patients with (Alpha-1) to test the effect of CBZ and other drugs in a way that is personalized for each patient."
What is Alpha-1 Antitrypsin Deficiency?*
Alpha-1 occurs when there is a lack of a protein in the blood called alpha-1 antitrypsin, or AAT. AAT is mainly produced by the liver. The main function of AAT is to protect the lungs from inflammation caused by infection and inhaled irritants such as tobacco smoke.
The low level of AAT in the blood occurs because the AAT is abnormal and cannot be released from the liver at the normal rate. This leads to a build-up of abnormal AAT in the liver that can cause liver disease and a decrease of AAT in the blood that can lead to lung disease.
- It is estimated that about 1 in every 2,500 Americans have Alpha-1.
- An estimated 19 million people in the United States are carriers of Alpha-1 (they have one normal and one defective alpha-1 gene).
- Alpha-1 is the most common genetic cause of liver disease in children.
*What is Alpha-1?. (n.d.). Alpha1 Foundation. Retrieved November 12, 2013, from http://alpha-1foundation.org/what-is-alpha-1/