Effects of formerly incurable liver disease may be reversed with drug - study

A drug known as NoRA1 activates the Notch pathway that helps orchestrate fundamental biological processes. Children with Alagille causes a reduction in Notch signaling.

 Liver diagram (photo credit: Wikimedia Commons)
Liver diagram
(photo credit: Wikimedia Commons)

A drug may have been found to combat the effects of Alagille syndrome, which has been considered to be an incurable liver disease, according to a recent peer-reviewed study.

The findings were published in the journal Proceedings of the National Academy of Sciences last month - with the research being led by Associate Professor Duc Dong from the Sanford Burnham Prebys’ graduate school.

“Alagille syndrome is widely considered an incurable disease, but we believe we’re on the way to changing that,” said Dong.

“Alagille syndrome is widely considered an incurable disease, but we believe we’re on the way to changing that.”

Associate Prof. Duc Dong 

Alagille syndrome is a mutation that stops the formation of bile ducts in the liver. It also affects more than 4,000 babies that are born every year.

What is the drug that is said to cure this disease?

A drug known as NoRA1 activates the Notch pathway that helps orchestrate fundamental biological processes. Children with Alagille experience a reduction in Notch signaling.

 Illustration photo of Super Pharm drug store and pharmacy at the Hadar mall in Jerusalem, on April 30, 2018.  (credit: HADAS PARUSH/FLASH90) Illustration photo of Super Pharm drug store and pharmacy at the Hadar mall in Jerusalem, on April 30, 2018. (credit: HADAS PARUSH/FLASH90)

“Our research suggests that nudging the Notch pathway up with a drug could be enough to restore the liver’s normal regenerative potential,” said one of the researchers of the study.

Furthermore, animals with mutations in the same gene affected in Alagille see the NoRA1 increasing the Notch signaling and triggering duct cells to regenerate in the liver. This is important because it reverses liver damage.