Combination Therapy with Mesenchymal Stem Cells and the Caveolin-1 Scaffolding Domain Peptide as a Novel Therapy for Systemic Sclerosis


Technology: MUSC researchers propose a novel therapy for systemic sclerosis (SSc) combining autologous patient-derived mesenchymal stem cells (MSCs) and the caveolin-1 scaffolding domain peptide (CSD). In a mouse model, this combination significantly improved the efficacy of MSC therapy compared to MSCs alone.

  Researchers have previously shown efficacy of adipose-derived MSCs (AT MSCs) in treating fibrotic diseases. However, AT MSCs from SSc patients exhibit a profibrotic and anti-adipogenic phenotype, which may limit treatment efficacy.  This altered differentiation of SSc AT MSCs can be corrected by treatment with CSD (Figure 1).

In an in vivo experiment, bleomycin-treated mice were administered MSCs subcutaneously with or without CSD co-treatment. As shown in Figure 2, lung fibrosis induced by bleomycin was significantly reduced by co-administration of MSCs and CSD compared to MSCs alone, regardless of whether the MSCs were derived from healthy or bleomycin mice.  In summary, MSCs from SSc patients are partially defective and this defect can be reversed by CSD.  Pretreatment of MSCs in vitro with CSD (prior to injection into bleomycin-treated mice) or co-treating the mice by MSC injection and CSD injection increases the overall beneficial effect compared to the injection of MSCs alone.