SLU researcher helps develop treatment for a type of incurable liver disease

Published: Jul. 18, 2022 at 6:38 PM CDT
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ST. LOUIS, Mo. (KMOV) - Debra Renfro and two of her siblings inherited a gene mutation that put them at risk for developing a liver disease that has no cure.

“My one brother who ended up having a liver transplant,” said Renfro.

White blood cells are so powerful that when they’re sent out to fight off an infection, the body produces a type of protein to protect the tissue of the lungs and other organs from being damaged. For people like Renfro, who inherit a type of gene mutation, the liver produces a defective version of that protein, called alpha-1 antitrypsin (AAT).

Dr. Jeffrey Teckman is a Professor of Pediatrics, Biochemistry and Molecular Biology at Saint Louis University School of Medicine.

“Protein builds up in the liver and it’s toxic to the liver, and in some people, that causes scarring and damage to the liver and even life-threatening liver damage,” said Teckman.

Alpha-1 antitrypsin deficiency is often called a silent killer because it often goes undiagnosed or misdiagnosed.

Dr. Teckman and his team collaborated with Arrowhead Pharmaceuticals and Takeda Pharmaceutical Company to develop a way to turn off the liver’s production of the bad version of AAT.

“And it worked in the lab. And I knew at that time or I was very confident that if we could work with the right partners and design a medicine with low side effects that would do the same thing in people, then it could be a game changer,” said Teckman.

The new drug that came out of the research is called Fazirsiran. Renfro said she’s very grateful for the research by Teckman and others.

“It’s going to impact the quality of life, longevity, especially for that next generation. My daughter and my nieces and nephews, she said.”

It’s estimated around 100,000 people in the U.S. have this type of liver disease.

Fazirsiran is about to begin phase-3 trials and won’t be available for several years.