Paula Ragan, PhD, CEO and President of X4 Pharmaceuticals, describes the CXCR4 pathway, and how mavorixafor, a CXCR4 antagonist, can treat certain rare diseases.
The CXCR4 protein is a chemokine receptor that is common to both hematopoietic and nonhematopoietic cells. As Dr. Ragan explains, there is evidence that the CXCR4 receptor is involved with B-lymphocyte trafficking throughout the body. Current theory suggests that in one rare immunodeficiency, WHIM syndrome, the mutations of CXCR4 cause abnormal retention of neutrophils in the bone marrow, preventing them from being released in the blood, resulting in neutropenia, which can present an increased risk for recurrent or severe infections for patients. Mavorixafor is a CXCR4 antagonist that could eliminate some or all of the negative impacts of CXCR4 mutations in patients with immunodeficiencies like WHIM syndrome. According to Dr. Ragan, all individuals (including healthy controls) who are given mavorixafor experience an increased production of immune cells in the bone marrow. In patients with primary immunodeficiencies, this increased production is essential.
To learn more about rare autoimmune disorders, visit https://checkrare.com/diseases/autoimmune-auto-inflammatory-disorders/