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umbilical cord blood + sirolimus
- Development stage
- Unknown
- Lead developer
- CHA Bundang Medical Center
- Modality
- Stem Cell Therapies → Cell Therapies, Small Molecules
- Administration
- Intravenous
01
Overview
This experimental combination therapy, developed by Bundang CHA Hospital, consists of allogeneic umbilical cord blood (UCB) units administered intravenously alongside oral sirolimus for the treatment of Hutchinson-Gilford Progeria Syndrome (HGPS). HGPS is a rare, fatal genetic condition characterized by accelerated aging due to the accumulation of progerin, a truncated form of the lamin A protein. The therapy utilizes UCB cells for their potential regenerative and anti-inflammatory properties to address vascular pathology. Sirolimus, an mTOR inhibitor, is co-administered to stimulate autophagy, thereby promoting the clearance of toxic progerin from cells. This dual approach is currently being evaluated in a pilot clinical trial (NCT03871972) to assess safety and efficacy in improving the clinical outcomes of HGPS patients.
- Other names
- Umbilical Cord Blood UnitUCB transfusionallogeneic umbilical cord blood
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