Method To Use Low-Dose Tocilizumab To Treat Sickle Cell Disease

SUMMARY

• Sickle cell disease (SCD) is a rare genetic disease that affects red blood cells, predominantly seen in African-American patients. Patients with SCD often have various complications, including acute chest syndrome (ACS), a life-threatening condition similar to pneumonia but unique to SCD.

• ACS is not entirely understood, and there are no curative treatments available. Recent data have suggested that increased interleukin-6 (IL-6) is a component of the inflammation in ACS. Specifically, it was found that IL-6 levels are elevated in the sputum of patients with ACS. Tocilizumab, a drug approved for rheumatoid arthritis and other autoimmune conditions, targets IL-6 receptors and lowers inflammation.

• The faculty inventor developed a method for the use of low-dose tocilizumab to treat complications of sickle cell disease, including acute chest syndrome and vaso-occlusive crisis. Moreover, an investigator-lead phase II clinical study is underway at UChicago Medicine to further evaluate the method.

ADVANTAGES

ADVANTAGES

• Improves overall clinical outcomes from patients including oxygen levels, inflammation, and pain

• Mitigates risk of blood transfusions (standard treatment for SCD) including iron overload and immune-mediated hemolytic transfusion reactions

APPLICATIONS

• Sickle cell disease

November 15, 2023

Proof of concept

Patent Pending

Licensing,Co-development

Mark Ratain

• Two-arm, Phase IIA trial underway