Novel Treatment of Pulmonary Arterial Hypertension (PAH)
Keywords
Pulmonary Arterial Hypertension, PAH, MSCs, hUC-MSCs
Invention Novelty
The invention comprises a novel therapeutic approach for the treatment of Pulmonary Arterial Hypertension (PAH) based on cell culture supernatant derived from cultivated human umbilical cord mesenchymal stem cells (HUCMSC).
Value Proposition
PAH is a disease which is characterized by increased vascular resistance and increased blood pressure inside the pulmonary vessels resulting in an undersupply of oxygen to the organs. Due to the excessive strain on the heart, patients often suffer from right-sided heart failure, progressing to death if left untreated. Currently, PAH is mainly treated pharmacologically with vasodilators, which can extend patients' survival for several years. However, a long-term cure for severe cases has so far only been achieved through lung transplantation.
LC–MS analysis revealed significantly higher levels of PGE2 in HUCMSCs. (Hansmann et al., 2022)
Technology Description
The technology is based on the isolation and subsequent cultivation of HUCMSCs from umbilical cord tissue. The supernatant resulting from cell cultivation is harvested as Conditioned Media (CM). Further analysis revealed that CM contains a significantly increased amount of Prostaglandin E2 (PGE2), as well as a multitude of proteins secreted by HUCMSCs such as DKK1, LRP1 and TGFBR2, which are known for their role in regenerative pathways. Extensive research in relevant animal models of severe PAH was able to demonstrate beneficial effects of CM. Further, a first therapeutic intervention on a compassionate use basis with CM from HUCMSCs was successfully performed. Based on these results HUCMSC-derived CM is a very promising approach for the treatment of severe forms of PAH.
Commercial Opportunity
In-licensing or collaboration for further development is possible.
Development Status
In vitro and in vivo studies have been performed.
Patent Situation
Patent applications in EP and US (based on WO 2023/232914 A1) with priority of 2022 are pending.
Further Reading
Hansmann G, Giera M, Ralser M, Hass R et al., 2022, Human umbilical cord mesenchymal stem cell-derived treatment of severe pulmonary arterial hypertension. Nat Cardiovasc Res 1, 568–576. doi.org/10.1038/s44161-022-00083-z
Fukumitsu, Suzuki (2019), Mesenchymal stem/stromal cell therapy for pulmonary arterial hypertension: Comprehensive review of preclinical studies. J Cardiol. 2019 Oct;74(4):304-312, doi.org/10.1016/j.jjcc.2019.04.006
Institute of Origin
