Stem Cell Therapy

Stem cell therapy is a regenerative medicine approach that uses stem cells - haematopoietic stem cells (HSC), mesenchymal stromal cells (MSC), induced pluripotent stem cells (iPSC), umbilical cord-derived cells, or tissue-resident progenitors - to repair, restore or replace damaged tissue. The most rigorously established applications are haematopoietic stem cell transplantation for blood cancers and severe immunodeficiencies, where the therapy is curative for many patients. NIH-affiliated literature also describes emerging applications in cardiac, neurological, pancreatic, ocular and musculoskeletal disorders, with mechanisms ranging from direct differentiation into target tissue to paracrine signalling that recruits endogenous repair. Delivery routes depend on the indication: intravenous infusion (haematological reconstitution, systemic immunomodulation), intra-articular injection (knee osteoarthritis, cartilage defects), intrathecal injection (neurological disorders within trials), and intramyocardial or intracoronary delivery (ischaemic cardiomyopathy in trials). Patient selection should follow rigorous criteria; many non-haematological indications remain investigational and are best delivered within registered clinical trials, as noted by ORIP/NIH. A typical treatment episode involves harvesting or sourcing the stem-cell product, processing in a laboratory under good-manufacturing-practice conditions, conditioning the patient if indicated, and administering the cells. International patients often need 7-14 days in-country for product preparation, administration and immediate monitoring. Long-term follow-up is essential to monitor for engraftment, immune reactions and durability of clinical effect. Sources: NIH ORIP, NIH/PMC peer-reviewed regenerative medicine literature.