Flipping the switch on scleroderma

April 4, 2014, Michigan State University
The picture on the left shows normal tissue cells. The picture on the right shows a marker for activated fibroblasts that are making collagen related to scleroderma. Credit: Michigan State University

Scleroderma is a rare and often fatal disease, causing the thickening of tissue, that currently lacks a cure and any effective treatments. A group of researchers, including a Michigan State University professor, is looking to change that.

"Our findings provide a new approach to developing better treatment options where few have existed," said Richard Neubig, chairperson of the Department of Pharmacology and Toxicology in MSU's College of Osteopathic Medicine.

Neubig, along with several of his colleagues from the University of Michigan, have identified the core signaling pathway that activates the disease and the chemical compounds that can turn it off.

"There are two kinds of scleroderma – localized and systemic – with the latter often proving to be life threatening," said Neubig, who helped lead the study. "This research shows that by inhibiting this main signaling pathway, we can block fibrosis – the thickening of tissue that occurs with the disease."

For localized scleroderma patients, this process often happens in the skin resulting in loss of flexibility. Systemic sclerosis has the same effect with variable degree of skin fibrosis, but also can spread throughout the body hardening key organs such as the lungs, heart, gut and kidneys.

Scleroderma is an autoimmune disorder. It's estimated 300,000 Americans suffer from the disease with about one-third of those having the systemic form. Localized scleroderma patients usually live normal lifespans. Yet about half of systemic patients, especially with widespread skin involvement and internal organ fibrosis, will see their lives cut short.

"The majority of drug treatments that exist today for fibrosis basically look at reducing just the inflammation," said Dinesh Khanna, associate professor in the Department of Internal Medicine and director of the Scleroderma Program at the University of Michigan. "There are other drugs that block one or two of the signaling pathways that cause the disease, but scleroderma has many of these pathways."

Neubig agrees and adds that this new research could significantly change the quality of life for scleroderma patients and greatly increase the lifespan of systemic patients.

"Our research shows promise for the development of a new drug that can reverse the process by flipping the main switch on all of the signaling pathways," Neubig said. "By validating this core switch as a viable drug target, we can now continue our work to improve the so they will work with doses that are appropriate for people. It's definitely promising."

The research is published in the Journal of Pharmacology and Experimental Therapeutics.

Explore further: New genetic path for scleroderma: Patient biopsies reveal potential new target for therapy

More information: jpet.aspetjournals.org/content/early/recent

Related Stories

New genetic path for scleroderma: Patient biopsies reveal potential new target for therapy

March 19, 2012
A genetic pathway previously known for its role in embryonic development and cancer has been identified as a target for systemic sclerosis, or scleroderma, therapy. The finding, discovered by a cross-disciplinary team led ...

Researchers implicate well-known protein in fibrosis

November 20, 2012
An international multi-disciplinary research team led by Northwestern Medicine scientists has uncovered a new role for the protein toll-like receptor 4 (TLR4) in the development of tissue fibrosis, or scarring.

Updated systemic sclerosis criteria improve disease classification

October 3, 2013
New classification criteria for systemic sclerosis have just been published and are more sensitive than the 1980 criteria, enabling earlier identification and treatment of this disabling autoimmune disease. The 2013 criteria, ...

Researchers identify likely causes, treatment strategies for systemic scleroderma

October 9, 2013
Using mice, lab-grown cells and clues from a related disorder, Johns Hopkins researchers have greatly increased understanding of the causes of systemic sclerosis, showing that a critical culprit is a defect in the way certain ...

Cancer drug may also work for scleroderma

September 22, 2011
A drug used to treat cancer may also be effective in diseases that cause scarring of the internal organs or skin, such as pulmonary fibrosis or scleroderma.

Genes reveal which patients will benefit from scleroderma drug

April 10, 2013
Systemic sclerosis, also known as scleroderma, is a rare autoimmune connective tissue disorder that's difficult to treat. However, thanks to new research at Northwestern University Feinberg School of Medicine and Dartmouth's ...

Recommended for you

New study offers insights on genetic indicators of COPD risk

January 16, 2018
Researchers have discovered that genetic variations in the anatomy of the lungs could serve as indicators to help identify people who have low, but stable, lung function early in life, and those who are particularly at risk ...

Previous influenza virus exposures enhance susceptibility in another influenza pandemic

January 16, 2018
While past exposure to influenza A viruses often builds immunity to similar, and sometimes different, strains of the virus, Canadian researchers are calling for more attention to exceptions to that rule.

Don't hold your nose and close your mouth when you sneeze, doctors warn

January 15, 2018
Pinching your nose while clamping your mouth shut to contain a forceful sneeze isn't a good idea, warn doctors in the journal BMJ Case Reports.

New antifungal provides hope in fight against superbugs

January 12, 2018
Microscopic yeast have been wreaking havoc in hospitals around the world—creeping into catheters, ventilator tubes, and IV lines—and causing deadly invasive infection. One culprit species, Candida auris, is resistant ...

Dengue takes low and slow approach to replication

January 11, 2018
A new study reveals how dengue virus manages to reproduce itself in an infected person without triggering the body's normal defenses. Duke researchers report that dengue pulls off this hoax by co-opting a specialized structure ...

Different strains of same bacteria trigger widely varying immune responses

January 11, 2018
Genetic differences between different strains of the same pathogenic bacterial species appear to result in widely varying immune system responses, according to new research published in PLOS Pathogens.

0 comments

Please sign in to add a comment. Registration is free, and takes less than a minute. Read more

Click here to reset your password.
Sign in to get notified via email when new comments are made.