Human umbilical cord mesenchymal stem cell (HUCMSC)-derived treatment of severe pulmonary arterial hypertension (PAH) is a promising novel therapeutic concept, according to a new study presented at the 20th World Congress in Fetal Medicine by Constantin von Kaisenberg, MD, PhD

Additional prospective clinical studies are warranted to confirm and further explore the benefits of HUCMSC-derived therapy for PAH, said Dr. von Kaisenberg, of the departments of obstetrics, gynecology, and reproductive medicine at Hannover Medical School in Germany.

He presented the case of a female patient aged 3 years who had heritable PAH associated with hereditary hemorrhagic telangiectasia. She was in critical condition following 2 syncopal afebrile seizure episodes. Her 6-minute walking distance was 270 m. Her oxygen saturation was above 95%, she had thrombocytopenia, and her N-terminal pro-b-type natriuretic peptide (NTproBNP) was elevated.

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She had a 10-month history of fatigue, repetitive nosebleeds, and mucocutaneous telangiectases at the lips, chest, and lower extremities. Her X-ray and CT scans revealed severe dilation of the right ventricular and pulmonary arteries and she was short-listed for lung transplantation.

She was treated with serial intravascular infusions of conditioned media from allogenic HUCMSCs. The properties of the cells were validated by untargeted label-free quantitation of the cell and conditioned media proteome, suggesting increased activity of regeneration, autophagy, and anti-inflammation.

The treatment improved her clinical and hemodynamic parameters and decreased her blood plasma markers of vascular fibrosis, injury, and inflammation. 

Dr. Von Kaisenberg said that a comparative analysis of single-cell RNA sequencing data collected from 3 HUCMSCs and 2 human umbilical vein endothelial cell lines identified 8 common cell clusters with regenerative potential specific for HUCMSCs. 

PAH is a rare and progressive type of pulmonary hypertension characterized by the narrowing and thickening of the pulmonary arteries leading to increased resistance to the flow of blood. There is currently no cure for the disease.


von Kaisenberg C. Human umbilical cord mesenchymal stem cell-derived treatment of severe pulmonary arterial hypertension. Oral presentation at: 20th World Congress in Fetal Medicine; June 27, 2023; Valencia, Spain.