Experimental Treatments for Scleroderma

Brentuximab vedotin is being investigated to possibly treat people with systemic scleroderma. It is an antibody, a protein designed to bind to a specific target, attached by a linker to a substance called monomethyl auristatin E, a synthetic agent that blocks cell division. The potential therapy is currently being tested in Phase 1/2 trials.

HZN-825 is being developed to possibly treat people with diffuse cutaneous scleroderma. It is a molecule that binds to lysophosphatidic acid (LPA) receptors, with the goal of reducing the formation and buildup of scar tissue in scleroderma patients. A Phase 2 trial testing the therapy is currently recruiting.

IVIG (immune globulin intravenous) is a human plasma-derived solution that contains antibodies to help protect against infection. The therapy is being tested to determine its efficacy in scleroderma. A Phase 2 trial was launched in 2019 but withdrawn for business reasons. The current status of the therapy for use in scleroderma patients is unknown.

Orencia (abatacept) is approved for the treatment of rheumatoid arthritis, juvenile arthritis, as well as psoriatic arthritis, and is being investigated for the treatment of scleroderma. It is a modified antibody that binds to a specific target. It blocks part of the immune system by interfering with the activity of immune cells called T-cells. A Phase 2 trial testing Orencia in diffuse systemic scleroderma patients has been completed.

Phosphodiesterase type 5 (PDE-5) inhibitors are oral medications used to treat pulmonary hypertension and erectile dysfunction. They are currently being investigated as an alternative to the calcium channel blockers used to manage Raynaud’s phenomenon and pulmonary arterial hypertension, both of which are often associated with systemic scleroderma. They have been studied in a Phase 2/3 trial for the treatment of secondary Raynaud’s.

Statins are a group of medications that act to regulate the level of certain lipids, or fat molecules, in the body. They are prescribed to patients with high levels of cholesterol, a type of lipid, and are under investigation as a possible treatment for scleroderma due to their potential to protect the vascular systems, possibly easing symptoms of scleroderma-related conditions.

Stem cell transplants are being investigated as a possible way to treat several autoimmune diseases, including scleroderma. It is hoped that a stem cell transplant may be able to modify or reset the immune response, potentially reducing or preventing scleroderma progression. This type of therapy has been studied in a Phase 2/3 trial and has shown potential in a variety of other studies.