Researchers at the University of Pittsburgh have developed an approach to inhibit lung vaso-occlusion and lung injury in SCD beyond the current FDA approved treatment of intravenous P-selectin Ab therapy.
Description
Acute chest syndrome (ACS) is a type of acute lung injury and one of the leading causes of mortality in Sickle Cell Disease (SCD). The etiological mechanisms that trigger ACS remain poorly understood. 10-20% of SCD patients hospitalized with acute systemic painful vaso-occlusive episodes develop ACS within the next few days, which suggests that molecular events surrounding vaso-occlusion contribute to lung injury. This is a major problem for patients and presents an opportunity for the development of therapeutics that can halt the development of ACS. Recent clinical trial has shown a ~50% reduction in hospitalization of SCD patients receiving intravenous P-selectin Ab therapy, suggesting that therapies beyond P-selectin inhibition would be needed to prevent ACS in SCD patients.
Researchers at the University of Pittsburgh have developed an approach to ameliorate the remaining (~50%) lung vaso-occlusion associated morbidity in SCD by inhibiting the Gasdermin-D (GSDMD) dependent signaling in leukocytes.
Applications
• Therapeutic for patients with SCD to reduce the occurrence of lung-vaso occlusion, which in turn reduces the likelihood of developing ACS.
Advantages
• Improved outcome for patients with SCD; reduced rates of lung-vaso occlusion.
• Potential to reduce mortality rate for SCD patients.
Invention Readiness
In vivo evidence using P-selectin-deficient SCD mice shows significant amelioration of lung vaso-occlusion and lung Injury following GSDMD-inhibition.
IP Status
https://patents.google.com/patent/WO2023034905A1
