MedPacto's vactosertib shows positive results in US osteosarcoma pre-clinical trial

MedPacto said it published a study on Cancer Communications, an international cancer journal, with regards to using vactosertib, a TGF-β inhibitor, as a possible treatment for osteosarcoma in a  pre-clinical trial.

Osteosarcoma, a malignant tumor originating in bone or cartilage tissue, predominantly affects children and adolescents. A substantial proportion of patients (25-50 percent) experience lung metastasis, which often leads to death, even during chemotherapy.

Current osteosarcoma treatments include surgery, chemotherapy, and radiation therapy. However, conventional chemotherapy methods have shown limited success, and immune checkpoint inhibitors have proven ineffective, underscoring the urgent need for new therapeutic approaches.

The paper, titled "Oral transforming growth factor-beta receptor 1 inhibitor vactosertib promotes osteosarcoma regression by targeting tumor proliferation and enhancing anti-tumor immunity," details collaborative research conducted by MedPacto and Case Western Reserve University in the U.S.

The study found that TGF-β increased the expression of the oncogene C-Myc, promoting tumor growth and metastasis, while also creating an immunosuppressive tumor microenvironment.

Notably, vactosertib effectively activated cytotoxic T cells and natural killer (NK) cells within this microenvironment, leading to a marked slowdown in tumor growth.

Also, in a pulmonary osteosarcoma model, vactosertib significantly inhibited tumor growth and reduced lung metastasis.

Meanwhile, the U.S. FDA has designated vactosertib as an orphan drug designation(ODD) and a rare pediatric disease designation (RPDD) for osteosarcoma, and it has also received Fast Track Designation (FTD). In Europe, vactosertib has been granted orphan medicinal product designation (OMPD).

Vactosertib is currently undergoing phase 1/2 clinical trials in both the U.S. and Korea for patients with recurrent or refractory osteosarcoma.