Judy Shizuru, MD, Blood and Marrow Transplant Specialist, Stanford University School of Medicine, discusses clinical data from an ongoing phase 1 trial of JSP191 in patients with severe combined immune deficiency (SCID).

SCID is a group of inherited immune system disorders characterized by abnormalities with responses of both T cells and B cells. Common symptoms include an increased susceptibility to infections including ear infections; pneumonia or bronchitis; oral thrush; and diarrhea.

As Dr. Shizuru explains, JSP191 is a humanized monoclonal antibody in clinical development as a conditioning agent that clears hematopoietic stem cells from bone marrow.

Preclinical studies have shown that JSP191 as a single agent safely decreases normal and diseased hematopoietic stem cells, including in animal models of SCID, myelodysplastic syndromes (MDS) and sickle cell disease (SCD). JSP191 is currently being evaluated as a sole conditioning agent in a phase 1/2 trial evaluating the safety and tolerability of JSP191 in patients undergoing hematopoietic cell transplant for SCID. JSP191 is also being evaluated in a phase 1 study in patients with MDS or acute myeloid leukemia (AML) who are receiving hematopoietic cell transplant. 

To learn more about SCID and other rare autoimmune disorders, visit https://checkrare.com/diseases/autoimmune-auto-inflammatory-disorders/