Results of lab study make MSCs 'ideal candidate' for SSc therapy
by Andrea Lobo, PhD | August 19, 2025

Modifying mesenchymal stem cells (MSCs) — a type of stem cell found in various tissues and, in this case, taken from the umbilical cord of mice — and using clumps of them in a mouse model of systemic sclerosis (SSc) was found to ease signs of skin and lung fibrosis, or scarring, and to reduce inflammation in the animals.

In a laboratory study, a team of scientists in China found that treatment with these clumps, or aggregates, alleviated both fibrosis and inflammation in the mice.

“These results suggest that [aggregates of modified MSCs] are ideal candidates for SSc therapy and optimize the clinical utilization of MSCs,” the researchers wrote.
The study, “Engineered MSC Aggregates with E/N-Cadherin and IL-6 Preconditioning for the Treatment of Systemic Sclerosis,” was published in the journal Advanced Healthcare Materials.

Results of separate trial for pulmonary sarcoidosis expected next month
by Marisa Wexler, MS | August 12, 2025

Efzofitimod was found to be generally safe and well tolerated by patients with systemic sclerosis-associated interstitial lung disease (SSc-ILD) in a proof-of-concept Phase 2 study. No treatment-related serious side effects were reported, with efzofitimod showing early signs of efficacy.

In addition, the last patient visit has been completed in EFZO-FIT, a Phase 3 clinical trial testing aTyr Pharma’s investigational therapy in people with pulmonary sarcoidosis.

Results from the Phase 3 study are expected next month, according to a corporate update from aTyr.
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“With the recent completion of the last patient visit in our Phase 3 EFZO-FIT study of efzofitimod in pulmonary sarcoidosis … we are on track to report topline data in mid-September,” said Sanjay S. Shukla, MD, president and CEO of aTyr. “This upcoming readout represents a major inflection point for aTyr, our clinical program for efzofitimod … and the broader sarcoidosis community, and we look forward to sharing the results.”

Levels tied to disease activity, digestive problems, inflammation and scarring
by Steve Bryson, PhD | August 5, 2025

The immune signaling protein interleukin-40, or IL-40, may be involved in developing systemic sclerosis (SSc), a study from the Czech Republic suggests.
Elevated levels of IL-40 were found in the bloodstream of adults with SSc and correlated with worse disease activity, digestive problems, and markers of inflammation and fibrosis. Although higher IL-40 levels were generally resistant to treatment in some patients, pre-treatment levels and reductions in IL-40 during treatment correlated with improvements in several clinical outcomes.

“These findings might pave the way for further research into IL-40, including it’s potential as a marker of early detection,” the researchers wrote in the study, “Serum IL-40 is elevated in systemic sclerosis and is linked to disease activity, gastrointestinal involvement, immune regulation and fibrotic processes,” which was published in Arthritis Research & Therapy.
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SSc, or scleroderma, is an autoimmune disease marked by the excessive production of collagen, a protein and the main component of scar tissue. Excess scar tissue formation, or fibrosis, builds up in the skin and internal organs, including the heart and blood vessels, the lungs, stomach, and kidneys, causing damage. The disease is thought to arise from an abnormal immune response that’s driven by the release of TGF-beta, a signaling protein that activates fibroblasts, the cells that produce collagen. Other immune signaling proteins altered in SSc include IL-4 and IL-6.

Study to enroll up to 180 adults with scleroderma, other autoimmune diseases
by Andrea Lobo, PhD | July 29, 2025

The first patient with systemic sclerosis (SSc) has been dosed in a Phase 1 trial of Adicet Bio’s investigational CAR T-cell therapy for autoimmune conditions, called ADI-001, the company has announced.

Recruitment in the open-label trial (NCT06375993) is ongoing at one site in California. It’s expected to enroll up to 180 adults, ages 18-80, with SSc or other autoimmune diseases that include ANCA-associated vasculitis, idiopathic inflammatory myopathy, stiff-person syndrome, systemic lupus erythematosus, and lupus nephritis, a common complication of lupus. Preliminary results are expected in the second half of the year.

“There remains a critical unmet need for safe and effective treatment options to combat debilitating autoimmune diseases, including SSc, for which we believe ADI-001 has the potential to offer transformative benefits,” Julie Maltzman, MD, chief medical officer at Adicet Bio, said in a company press release. “We are pleased with the continued momentum of our Phase 1 trial of ADI-001 in autoimmune diseases as we announce the dosing of the first SSc patient, a key development milestone for Adicet.”
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SSc is caused by an overactive immune system that leads to inflammation and the accumulation of scar tissue, or fibrosis, in the skin and internal organs, including the heart, kidneys, lungs, and digestive tract. Like other autoimmune conditions, SSc is marked by the production of self-reactive antibodies by immune B-cells that attack healthy tissue.