Findings could lead to new scleroderma treatment, researchers say by Steve Bryson, PhD | April 15, 2025

​Exosomes, tiny sacs containing biomolecules secreted from stem cells, thinned skin scarring in a mouse model of scleroderma, as well as skin samples from patients, a study reported.
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These findings support “the use of exosomes as a clinical treatment for [scleroderma] skin fibrosis,” the researchers said in the study, “Exosomes carrying adipose mesenchymal stem cells function alleviate scleroderma skin fibrosis by inhibiting the TGF-β1/Smad3 axis,” which was published in Scientific Reports.

Scleroderma is an autoimmune disorder marked by the abnormal accumulation of collagen, the main protein component of scar tissue, in the skin and various organs. It’s called localized scleroderma when it affects just the skin and/or underlying muscle tissue, and systemic scleroderma when organs, including the heart, lungs, stomach, and kidneys, are also involved.
Nearly all scleroderma patients have skin involvement, and the severity of skin scarring, or fibrosis, is typically associated with damage to the vital organs and mortality.

Mesenchymal stem cells, or MSCs, are a type of stem cell found in various tissues, including the bone marrow, adipose (fat) tissue, and the umbilical cord. Due to their anti-fibrotic and anti-inflammatory properties, MSCs have been considered a promising treatment for scleroderma.
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MSCs work by releasing signaling proteins and/or exosomes, tiny membrane-bound sacs that carry proteins and other molecules important for cellular communication. Because exosomes are cell-free, they may circumvent the adverse effects associated with MSC therapy, including immune rejection.

Car T-cell therapy aims for off-shelf cell supply by Marisa Wexler, MS | April 8, 2025

The U.S. Food and Drug Administration (FDA) has granted fast track designation to the cell therapy ALLO-329 to treat active refractory (resistant to treatment) diffuse scleroderma, also known as diffuse systemic sclerosis (SSc).

The FDA also gave ALLO-329 fast track designation as a potential treatment for two other inflammatory diseases: myositis, which is characterized by muscle inflammation, and lupus, which is marked by antibodies that attack the body’s healthy tissues.

Fast track designation is designed to speed the development of new treatments that have the potential to address unmet medical needs for serious or life-threatening diseases. With these designations, ALLO-329’s developer Allogene Therapeutics will get access to benefits including more frequent interactions with the FDA throughout the drug development process.

“Receiving these designations for ALLO-329 underscores the versatility and transformative promise of this next-generation allogeneic CAR T investigational product in redefining the autoimmune treatment landscape,” said Zachary Roberts, MD, PhD, executive vice president of research and development and chief medical officer of Allogene, said in a company press release.
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ALLO-329 is a CAR T-cell therapy. T-cells are immune cells that are able to kill other types of cells. CAR T-cell treatments involve engineering T-cells to equip them with a chimeric antigen receptor (CAR), a molecular weapon that directs a T-cell to attack other cells bearing specific molecular markers. ALLO-329 uses a CAR that targets CD19 and CD70, two proteins that are present, respectively, on the surface of B-cells and T-cells, both of which are immune cells involved in the development of many autoimmune diseases.

I like to go for walks when it's not too hot or cold by Tomisa Starr | April 2, 2025

For those of us with scleroderma, it’s important to stay warm, because changes in body temperature, especially when exposed to the cold, can cause blood vessels to constrict.

I like to go for walks to get exercise and maintain my lung function. My exercise “Goldilocks zone” — my favorite time of the year to go for walks — is when it’s not too cold or hot outside. Where I live in California, that occurs from late fall to spring, when the daily high temperatures are in the mid 50s to 60s. Exercise makes my blood flow, which helps to keep me warm.
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Raynaud’s phenomenon, a condition where the fingers and toes feel numb, prickly, and frigid in response to cold temperatures or stress, is common among people with scleroderma. The constriction of blood vessels can cause color changes in the fingers. My fingers look purple, yellow, and red when Raynaud’s affects me.

Sustained slowing of lung function decline shown for SSc-ILD patients
by Andrea Lobo, PhD | March 25, 2025

Treatment with Ofev (nintedanib) for up to four years was associated with a sustained slowing of lung function decline in people with systemic sclerosis (SSc) associated with interstitial lung disease (SSc-ILD).

That’s according to new results from the open-label study SENSCIS-ON (NCT03313180), a three-year extension of the SENSCIS Phase 3 trial that formed the basis for the therapy’s regulatory approvals. In the long-term extension, Ofev also had a safety profile consistent with previous studies, the new data show.
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“These findings suggest that [Ofev] can be used over the long term to slow the progression of SSc-ILD and so improve patient outcomes,” the researchers wrote.
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Their study, “Continued nintedanib in patients with systemic sclerosis-associated interstitial lung disease: 3-year data from SENSCIS-ON,” was published as a brief report in the journal RMD Open Rheumatic & Musculoskeletal Disorders.