Jeffrey Teckman, M.D., professor of pediatrics and biochemistry and molecular biology at Saint Louis University, has received a $1.4 million grant from the Alpha-1 Foundation to investigate the natural history and progression of Alpha-1 Antitrypsin Deficiency, an inherited disease that can cause liver damage in children as well as adults.
The multi-center Adult Clinical and Genetic Linkage Study aims to understand the Alpha-1 liver disease in adults and answer various questions about effective methods of treatment. The study will be conducted at SLU, University of Florida, Gainesville and University of California, San Diego.
"Alpha-1 can cause life-threating liver disease, and even cancer in some patients. But we can't predict in which patients it will develop or how fast liver damage will progress if it occurs," said Teckman, who serves as the director of the Division of Gastroenterology and Hepatology at SSM Cardinal Glennon Children's Medical Center. "This study will be helpful to understand how quickly it gets worse and what will be the science behind understanding that process."
Alpha-1 liver disease was discovered almost 50 years ago, but researchers have not yet identified many of the basic questions that will lead to possible treatment options. The study will be helpful in providing information about the progression of Alpha-1 liver disease in patients to companies that are looking to develop pharmaceuticals and other treatments.
"This is an important step in building knowledge about the disease," Teckman said. "And this knowledge will help us develop better treatment."
Adrian Di Bisceglie, M.D., chair of the department of internal medicine at Saint Louis University will also be involved with the study.
Teckman has worked with the Alpha-1 community for 15 years. Two years ago, the Alpha-1 Foundation developed a task force dedicated to finding out more about the liver disease. During the committee meetings, Teckman and other researchers identified the need to study a group of adults in order to discover basic questions about Alpha-1 liver disease to drive research for new treatment. Earlier this year, Teckman also published a paper in which he presents an approach for hepatologists to manage adult patients with Alpha-1 deﬁciency.
This study will have some similarities with The Childhood Liver Disease Research and Education Network (ChILDREN) study, in which Teckman and the others have been monitoring 300 children with the Alpha-1 condition for 10 years, the largest group of kids with Alpha-1 being studied in the world. Each year, some of Teckman's patients travel from various parts of the country, including Florida, Texas, Michigan and Wisconsin, to participate in this study.