What's new in autoimmune disease drug development?

Autoimmune diseases have no cures and treatments often involve the management of long-term symptoms. Although there are more than 100 autoimmune diseases, patients often rely on the same set of medications, such as prednisone and immunosuppressants, which can have harsh side effects. Infusions like Rituximab and IVIG can also be effective, although they can be time-consuming and expensive, especially when not approved for off-label use.

Janus Kinase (JAK) inhibitors is a relatively new type of autoimmune disease treatment that can be taken orally. JAK is a family of 4 enzymes that facilitate the signaling of cytokines, which are small proteins that activate and regulate the immune system. Cytokines are crucial in defending the body against infections, but an excess can also lead to inflammation and autoimmune disease.

Conventionally, JAK inhibitors have targeted 1 of the 4 JAK enzymes. However, a new JAK inhibitor, Brepocitinib, is being studied as one of the first such drugs to simultaneously target 2 JAK subtypes, JAK1 and TYK2. It was tested on patients with psoriatic arthritis in a Phase II clinical trial, which showed improved symptoms over 52 weeks. Brepocitinib is also currently being tested on patients with dermatomyositis (as a study on patients with systemic lupus erythematosus is wrapping up).

Dr. Ruth Ann Vleugels, MD, MPH,* of Harvard Medical School is one of the primary researchers studying this new drug, as she points out the need for new treatments: "There is an urgent need for novel, targeted therapies for dermatomyositis, a devastating disease with few safe and effective treatments.” The Autoimmune Registry is also helping to recruit patients for this study.

If you're interested in learning more or participating in the ongoing dermatomyositis study, please contact us. To support the Autoimmune Registry, please share our information with others. Donations are also greatly appreciated!

* Dr. Ruth Ann Vleugels, MD, MPH, is the Director of the Connective Tissue Disease Clinics and Autoimmune Skin Diseases Program at Brigham and Women’s Hospital/Harvard Medical School.