Journal September 27, 2026 4 min read

How does stem cell therapy work?

Stem cell therapy is a regenerative approach that uses stem cells or their derivatives to repair or replace damaged tissue. This page explains the proposed mechanisms and summarizes current research studies, including an

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What is stem cell therapy?

Stem cell therapy is a broad term for medical interventions that use stem cells or their derivatives to treat disease. The most commonly studied class is mesenchymal stem cells (MSCs), which are multipotent stromal cells that can be isolated from bone marrow, adipose tissue, and other sources. Other approaches include hematopoietic stem cells (HSCs) and induced pluripotent stem cells (iPSCs). In research settings, these cells are often characterized by surface markers such as CD73, CD90, and CD105 for MSCs, and CD34 and CD45 for HSCs. The therapeutic potential of stem cells is generally attributed to their ability to differentiate into specialized cell types, secrete paracrine factors that modulate the immune response, and promote endogenous repair mechanisms.

Many investigational stem cell therapies focus on the modulation of the tissue microenvironment through signaling pathways such as the Wnt/β-catenin pathway, which is involved in cell proliferation and differentiation, or through the secretion of growth factors like VEGF and IGF-1 that support angiogenesis and tissue regeneration.

Research Studies

Mesenchymal stem cells and non-viral acute-on-chronic liver failure

A Phase-II trial is evaluating MSC therapy in patients with non-viral acute-on-chronic liver failure (ACLF). The study aims to develop and validate MSC-based therapy to promote native liver regeneration and offer a transplant-free treatment option. The trial is conducted by the National Collaborative Centre for Hepatic Regenerative Medicine (NC-CHRM) in India, where over 100,000 liver transplants are needed annually but only about 2,500 are performed [1].

Gene-modified hematopoietic stem cells and transfusion-dependent beta-thalassemia

A Phase IIb trial is investigating autologous hematopoietic stem and progenitor cells (HSPCs) genetically modified with the GLOBE lentiviral vector for transfusion-dependent beta-thalassemia. The study involves a single infusion of these modified cells in nine patients, using an improved transduction protocol. The trial is a prospective, dual-centre, single-dose, single-arm, open-label study [2].

Pulsed electromagnetic fields and knee osteoarthritis

Research is exploring the use of pulsed electromagnetic fields (PEMF) as a non-invasive treatment for knee osteoarthritis (OA). The study notes that OA prevalence exceeds 40% in the elderly, and current treatments, including stem cell therapy, are often expensive and associated with side effects. The trial aims to assess the cost-effectiveness and therapeutic potential of PEMF [3].

Autologous mesenchymal stem cells and premature ovarian failure

A study is evaluating the therapeutic potential of autologous MSC therapy in women with premature ovarian failure (POF). The trial, titled "It is a Real" The First Baby Of Autologous Stem Cell Therapy in Premature Ovarian Failure, investigates whether MSC transplantation can restore ovarian function [4].

Mitochondrial-targeted nano-enhancer and MSC spheroids for bone regeneration

In vitro and animal studies have explored the role of mitochondrial oxidative phosphorylation (OXPHOS) in determining the functional fate of MSCs. A mitochondrial-targeted nano-enhancer was used to drive a self-amplifying NAD+ feedback loop to reprogram MSC spheroids metabolism, resulting in accelerated bone regeneration in preclinical models [5].

Hair follicle-derived neural crest stem cells and hearing loss

In a rat model of ouabain-induced spiral ganglion neuron injury, transplantation of hair follicle-derived neural crest stem cells onto the cochlear nerve surface led to partial hearing recovery. This approach addresses the challenge of effective cell delivery for sensorineural hearing loss [6].

Adipose-derived stem cells and erectile dysfunction

In a rat model of cavernous nerve injury, adipose-derived stem cell (ASC) therapy restored erectile function and promoted endogenous stem cell recruitment. The study investigated the therapeutic effects of exogenous stem cells and their mechanisms in the context of post-prostatectomy erectile dysfunction [7].

If you are reading this for personal health reasons

The materials and information discussed here are for laboratory research only. We do not provide dosing, medical guidance, or treatment recommendations. If you are considering any stem cell-based therapy for yourself, consult a licensed clinician.