Normal scar tissue forms to heal an internal wound and quietly retreats when the job is done. But in many common diseases — kidney, liver and lung fibrosis — the scar tissue goes rogue and strangles vital organs. These diseases are largely untreatable and ultimately fatal.
A new Northwestern Medicine study has newly identified a trigger of some fibrotic diseases and an experimental compound to treat it.
Fibrosis — a progressive scarring and hardening of internal organs — is estimated to cause 35 to 40 percent of deaths in the world. Fibrotic diseases include diabetic kidney fibrosis, alcoholic liver cirrhosis, hepatitis C, pulmonary fibrosis and nonalcoholic fatty liver disease, which may lead to fibrosis of the liver, the leading cause of liver transplant.
In one subset of human fibrosis cells, scientists discovered a delinquent gang of molecules that continually shouted at an immune receptor — the antennae on the cell — to produce scar tissue instead of quieting down and allowing the scar tissue to go back to sleep.
Scientists collaborated with a University of Colorado researcher who used crystallography and computer modeling to predict a molecule that could block the receptor that leads to the uncontrolled scarring. When they tested the molecule, T53, in three different mouse models of fibrosis, the abnormality was significantly reversed.
“Our study opens a new door into fibrosis by looking at it as an aberrant innate immune response and suggesting a novel approach to treat it,” said senior author Dr. John Varga, director of the Northwestern Scleroderma Program and the John and Nancy Hughes Distinguished Professor of Rheumatology at Northwestern University Feinberg School of Medicine.
The paper was published July 12 in the Journal of Clinical Investigation Insight.
“The leading cause of liver failure in western world is obesity and that’s because of liver fibrosis,” Varga said. “In the U.S., many of these diseases are lifestyle or age dependent. As we get fatter or older, they get worse.”
Most fibrotic disease likely begins as normal repair of an injury, scientists said. “But if the immune system produces too much of an initial scar, it can’t go back to normal,” Varga said. “You have an unhealed scar that keeps growing and can wipe out the entire organ.”
Not everyone’s fibrosis is caused by the same abnormality, Varga said. If the compound, T53, is eventually developed into an approved drug, it would be targeted to patients with the specific genetic signature identified in the study.
“There is an emerging direction for treating fibrosis with precision medicine,” said first author Swati Bhattacharyya, research associate professor of medicine in rheumatology and scientific director of the Scleroderma Research Laboratory at Feinberg. “Some people live with fibrotic disease for 30 years while others die in two years. We need to identify the rapid progressors from the slow progressors. That’s where precision medicine becomes really critical.”
“The results of this study are encouraging,” Varga said. “We are not saying this compound is ready to be a drug. It’s an initial compound that would need to be developed and tweaked. It would need significant funding to go to the next step.”
Learn more: Why internal scars won’t stop growing
The Latest on: Fibrosis
via Google News
The Latest on: Fibrosis
- APAC Idiopathic Pulmonary Fibrosis Market to 2027 - Size, Share & Industry Trends Analysis Reporton May 19, 2022 at 2:50 am
Dublin, May 19, 2022 (GLOBE NEWSWIRE) -- The "Asia Pacific Idiopathic Pulmonary Fibrosis Market Size, Share & Industry Trends Analysis Report By Drug Type, By Distribution Channel (Retail Pharmacies, ...
- MHRA approves Infex Therapeutics’s study for treatment for non-cystic fibrosis patientson May 19, 2022 at 2:39 am
The treatment, for Pseudomonas aeruginosa, will address a chronic and debilitating respiratory condition that has no approved available treatments ...
- Louisville siblings with cystic fibrosis advocate for the Make-A-Wish Walk for Wisheson May 18, 2022 at 6:21 pm
SO IN 2018 WHEN MAKE-A-WISH GRANTED 11 YEAR OLD FTHAI HER WISH TO GO TO DISNEY WORLD THE ENTIRE FAMILY BENEFITED. IT WAS A KID’S PARADISE. I MEAN EVERYTHING WAS BASED ON KIDS. WE’RE SO FOCUSED ON THE ...
- Siblings with cystic fibrosis advocate for the Make-A-Wish Walk for Wisheson May 18, 2022 at 6:20 pm
Faith and Canon Tabor are not only siblings, but they share a connection through a diagnosis they both received as babies.
- ReCode Therapeutics Presents New Preclinical Data from mRNA-based Program for Cystic Fibrosis at the ATS 2022 International Conferenceon May 18, 2022 at 12:46 pm
ReCode Therapeutics, a biopharmaceutical company powering the next wave of genetic medicines through superior delivery, today presented encouraging new preclinical data from the company’s inhaled mRNA ...
- Mucus Clearance Devices For Cystic Fibrosis Market Analysis, Size, Share, Growth, Trends and Forecast 2022-2028on May 18, 2022 at 12:13 pm
The Mucus Clearance Devices For Cystic Fibrosis market report covers all the drivers of growth and opportunities that affect the profit chart, and also provide important information about the ...
- Gene therapy is already saving lives and could revolutionise cystic fibrosis and Huntington’s treatmenton May 18, 2022 at 5:50 am
It is hoped advances in gene therapy will eventually allow scientists to cure hereditary diseases by neatly clipping out faulty genes and replacing them ...
- Come out to celebrate Cystic Fibrosis Foundation's '5 finest' annual honoreeson May 18, 2022 at 5:38 am
Foundation- Wisconsin Chapter has announced its 10th annual class of “Milwaukee’s Finest” honorees.The program was created to emphasize the importanc ...
- Bacteriophage therapy successful in treating cystic fibrosis patient’s Mycobacteria lung infectionon May 17, 2022 at 4:37 pm
Bacteriophages – viruses that kill bacteria–were successfully used for the first time to treat an antibiotic-resistant Mycobacterium abscessus lung infection to allow a cystic fibrosis ...
via Bing News