New study finds hope in understanding and better treating scleroderma

January 10, 2018, Hospital for Special Surgery

Scleroderma is a terribly debilitating disease with no effective treatments and the mortality rates are still upwards of 20%-50%, the highest of any rheumatic disease.

This disabling autoimmune disorder results in inflammation and leading to the thickening of the body's connective tissue, including the skin; and for decades its treatment has been symptomatic and, at best, inconsistently effective. But new research by a team from Hospital for Special Surgery (HSS) in New York City may signal hope for patients suffering from the condition.

The mechanism behind systemic sclerosis is not well understood. However, new research published today in Science Translational Medicine reveals a potential breakthrough into the cause of this disease, and also provides a possible treatment lead. Led by HSS researcher Dr. Franck Barrat—the Michael Bloomberg Chair and Senior Scientist at HSS - along with clinicians of the Scleroderma center of HSS, the work implicates what are called (pDCs) in contributing to scleroderma.

Normally pDCs secrete a compound called interferon to help fight off infections. However, as Dr. Barrat's study revealed, in scleroderma patients these cells are chronically activated and infiltrate the skin causing fibrosis and inflammation.

"Plasmacytoid dendritic cells are known to be activated in many other rheumatic conditions, including lupus," explains Dr. Barrat. "But our findings suggest that they participate in both establishing and maintaining fibrosis in the skin as well. This is a very interesting finding as it opens new ways to tackle this condition."

Dr. Barrat found that depleting pDCs in an animal model of scleroderma prevented the disease from forming, while also reversing already existing fibrosis.

The new research also revealed that a receptor on the surface of pDCs called TLR8 is responsible for their increased activity.

Dr. Barrat fully acknowledges the limitations of the new study in particular the part where the research used animal models of scleroderma, which only partially reflect the complexity of the disease in humans. But he's hopeful that not only will the new findings help illuminate the pathology of a puzzling , they may also represent potential novel approaches to .

Strategies to eliminate pDCs are currently being evaluated by drug companies in diseases other than scleroderma. And though these potential treatments are still years away from being available, Dr. Barrat hopes that "a better understanding of the role pDCs play in fibrosis will open up the possibility of repurposing existing drugs to treat patients with ".

Explore further: New study suggests way to slow skin fibrosis in scleroderma

More information: M.D. Ah Kioon el al., "Plasmacytoid dendritic cells promote systemic sclerosis with a key role for TLR8," Science Translational Medicine (2017). stm.sciencemag.org/lookup/doi/ … scitranslmed.aam8458

Related Stories

New study suggests way to slow skin fibrosis in scleroderma

October 31, 2016
The prognosis for patients diagnosed with scleroderma - an autoimmune disease characterized by fibrosis of the skin - is not typically a rosy one. With limited treatment options available, those suffering from the disorder ...

Drug found that induces apoptosis in myofibroblasts reducing fibrosis in scleroderma

December 15, 2017
(Medical Xpress)—An international team of researchers has found that the drug navitoclax can induce apoptosis (self-destruction) in myofibroblasts in mice, reducing the spread of fibrosis in scleroderma. In their paper ...

New way of classifying scleroderma

December 1, 2015
A new way of classifying scleroderma will greatly help the diagnosis and stratification of the debilitating medical condition, as well as assist in researching other autoimmune diseases.

New clues on tissue scarring in scleroderma

April 18, 2014
A discovery by Northwestern Medicine scientists could lead to potential new treatments for breaking the cycle of tissue scarring in people with scleroderma.

Flipping the switch on scleroderma

April 4, 2014
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 ...

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.

Recommended for you

Creation of synthetic horsepox virus could lead to more effective smallpox vaccine

January 19, 2018
UAlberta researchers created a new synthetic virus that could lead to the development of a more effective vaccine against smallpox. The discovery demonstrates how techniques based on the use of synthetic DNA can be used to ...

Study ends debate over role of steroids in treating septic shock

January 19, 2018
The results from the largest ever study of septic shock could improve treatment for critically ill patients and save health systems worldwide hundreds of millions of dollars each year.

New approach could help curtail hospitalizations due to influenza infection

January 18, 2018
More than 700,000 Americans were hospitalized due to illnesses associated with the seasonal flu during the 2014-15 flu season, according to federal estimates. A radical new approach to vaccine development at UCLA may help ...

Zika virus damages placenta, which may explain malformed babies

January 18, 2018
Though the Zika virus is widely known for a recent outbreak that caused children to be born with microencephaly, or having a small head, and other malformations, scientists have struggled to explain how the virus affects ...

Certain flu virus mutations may compensate for fitness costs of other mutations

January 18, 2018
Seasonal flu viruses continually undergo mutations that help them evade the human immune system, but some of these mutations can reduce a virus's potency. According to new research published in PLOS Pathogens, certain mutations ...

Study reveals how MRSA infection compromises lymphatic function

January 17, 2018
Infections of the skin or other soft tissues with the hard-to-treat MRSA (methicillin-resistant Staphylococcus aureus) bacteria appear to permanently compromise the lymphatic system, which is crucial to immune system function. ...

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.