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

Cancer drug helps treat tuberculosis by restoring leaky blood vessels

April 26, 2018
Biomedical engineers have discovered an unlikely potential ally in the global fight against tuberculosis—an FDA-approved drug originally designed to treat cancer.

Molecule may help tame virulent bacteria and prevent infection

April 26, 2018
According to the U.S. Centers for Disease Control and Prevention, about one in three humans carries Staphylococcus aureus, or "staph," in our noses, and 2 percent of us carry the dreaded methicillin-resistant (MRSA) strain ...

Antibody 'cocktail' can prevent Zika infection but is not effective for treatment of fetuses

April 26, 2018
A "cocktail" of monoclonal antibodies that can prevent Zika virus (ZIKV) infection in primates was not effective for treatment of fetuses, according to a new collaborative study led by a University of Miami Miller School ...

E. coli—are we measuring the wrong thing?

April 25, 2018
A sepsis awareness and management programme has demonstrated overall success in terms of improved sepsis detection, but has led to an increase in the number of E. coli blood stream infection cases presented, calling into ...

Malaria study reveals gene variants linked to risk of disease

April 25, 2018
Many people of African heritage are protected against malaria by inheriting a particular version of a gene, a large-scale study has shown.

Commonly prescribed heartburn drug linked to pneumonia in older adults

April 24, 2018
Researchers at the University of Exeter have found a statistical link between pneumonia in older people and a group of medicines commonly used to neutralise stomach acid in people with heartburn or stomach ulcers. Although ...

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.