Stem Cell Therapy Offers New Hope for Patients With Rare Autoimmune Disorder

New treatment potentially the first to improve survival in patients with severe scleroderma in more than four decades.

An unusual autoimmune disease that causes skin and lung damage can be treated effectively by stem cell transplant, reports new study published in the New England Journal of Medicine. The approach could represent the first new treatment to improve survival in patients with severe scleroderma in more than four decades.

The experimental procedure uses chemotherapy and radiation to destroy the body's malfunctioning immune system, then replaces it via a stem-cell transplant. The stem cells are taken from the patient's own blood and given back to the patient after the chemotherapy.

Scleroderma - CREST Syndrome - Signs - Symptoms - Diagnosis - Treatment - Prognosis
Scleroderma or CREST syndrome is a chronic, auto immune disease which manifests as thick, dry, fibrous skin. Scleroderma/CREST syndrome is classified as rheumatic and connective tissue disease.

‘After 6 year follow-up, 86% of those who received the stem-cell transplant remained alive, compared with only 51% of those who received infusions of cyclophosphamide.’

In a multisite clinical trial, the approach proved more successful than the existing treatment, significantly improving survival and reported quality of life. "This is a major advance in the treatment of severe scleroderma," said Karen Ballen, MD, a co-investigator on the study and the director of stem cell transplantation at the University of Virginia Cancer Center.

Scleroderma Study

The study compared the most effective existing treatment, the drug cyclophosphamide, with the new approach. Thirty-four trial participants received cyclophosphamide, while 33 received the stem cell transplant treatment. After 72 months, 86 percent of those who received the stem-cell transplant remained alive, compared with only 51 percent of those who received infusions of cyclophosphamide.

Breakthrough Mechanism That Causes Scleroderma Identified
Cells found in the lymphoid organs are activated and infiltrate the skin causing fibrosis and inflammation. These cells could be the target for treatment.

In the New England Journal of Medicine paper detailing their findings, the researchers conclude: "At four and a half years of follow up, participants who received a transplant experienced significantly better outcomes overall than those who received cyclophosphamide. In addition, 44 percent of participants who received cyclophosphamide had begun taking anti-rheumatic drugs for progression of their scleroderma, compared to only 9 percent of those who received a transplant."

Both treatment options carried risks of infections and low blood-cell counts, the researchers reported. The overall infection rates were similar.

Novel Way to Slow Skin Fibrosis in Scleroderma
A possible mechanism behind the fibrosis that occurs in scleroderma has been identified by researchers.

All participants had severe scleroderma that affected their lungs or kidneys. The researchers noted that their study had limitations that suggest the findings may not apply to all patients with scleroderma.

Blocking Estrogen may Help Fight Scleroderma
Scleroderma, which means 'hardskin' is an autoimmune disease affecting multiple organs and which predominantly affects women.

Source-Eurekalert