Cancer drug may also work for scleroderma

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.

The drug, with the generic name bortezomib, stopped the production of fibrotic proteins in human cells and the development of fibrous scarring in a mouse model of fibrotic disease, according to a new Northwestern Medicine study published in the journal Thorax.

"This drug appears to put the brakes on abnormal development of in the lungs and skin and may also work in other organs," said lead author Manu Jain, M.D., associate professor of medicine and of pediatrics at Northwestern University Feinberg School of Medicine and a physician at Northwestern Memorial Hospital and Children's Memorial Hospital. "These diseases have a high , and there is no truly effective treatment for them right now."

Scleroderma is an autoimmune disease that causes progressive thickening and tightening of the skin and can lead to serious internal organ damage and, in some cases, death. Scleroderma affects an estimated 300,000 people in the United States, most frequently young to middle-aged women.

Idiopathic is a scarring or thickening of the lungs without any known cause that makes it increasingly difficult to breathe. It may affect up to 200,000 people in the U.S. between 50 and 70 years old.

Jain said the drug appears to inhibit a protein called transforming growth factor beta, which is essential for the growth of the scar tissue. Patients with fibrosis have increased levels and activity of the growth factor. Bortezomib is currently used to treat and lymphoma.

In the study, when researchers gave bortezomib to mice, it prevented the development of a fibrotic-like disease. "The mice that normally get this disease didn't get it," Jain said.

Researchers also took from scleroderma and pulmonary fibrosis patients and incubated those cells with the drug. Fibroblast cells are believed to be important in the development of scarring in humans. The drug prevented the expression of proteins that are necessary for scarring.

Related Stories

New Target Identified for Scleroderma Therapy

Jun 14, 2010

(PhysOrg.com) -- Investigators at Northwestern University Feinberg School of Medicine have identified the molecule Egr-1 (early growth response factor 1) as a new therapy target for scleroderma, an autoimmune disease for ...

Scarred lungs leave trail of beta arrestins

Mar 28, 2011

Targeting a family of signaling proteins called beta arrestins may stop the life-threatening scarring and thickening of lungs associated with pulmonary fibrosis, reports a new Science study in mice.

Tumor suppressor may attenuate fibrotic disease

Feb 17, 2009

New research reveals a critical cellular signaling pathway that is responsible for generating excess connective tissue in multiple organs, similar to what is seen in human patients with scleroderma. The study, published by ...

Study sheds light on deadly lung disease

Apr 14, 2008

Systemic sclerosis (SSc), also known as scleroderma, is characterized by the formation of fibrosis, or scar tissue, on internal organs as well as the skin. Beyond its disfiguring symptoms, SSc is associated with a high rate ...

Recommended for you

WHO: Millions of Ebola vaccine doses ready in 2015

Oct 24, 2014

The World Health Organization says millions of doses of two experimental Ebola vaccines could be ready for use in 2015 and five more experimental vaccines will start being tested in March.

Added benefit of vedolizumab is not proven

Oct 23, 2014

Vedolizumab (trade name Entyvio) has been approved since May 2014 for patients with moderately to severely active Crohn disease or ulcerative colitis. In an early benefit assessment pursuant to the Act on the Reform of the ...

Seaweed menace may yield new medicines

Oct 22, 2014

An invasive seaweed clogging up British coasts could be a blessing in disguise. University of Greenwich scientists have won a cash award to turn it into valuable compounds which can lead to new, life-saving drugs.

User comments