MSC Therapies: Globally Approved Mesenchymal Stem Cell Therapeutics

As the most common stem cell type being used within regenerative medicine, there is huge potential for growth within the mesenchymal stem cell (MSC) market. Today, MSCs are the most common stem cell type being used within clinical trials, as well as the most studied stem cell type within the scientific literature.

Population aging and an increasing prevalence of chronic disease are also driving interest in MSC-based therapies, with Google Trend data revealing that MSC searches are more than twice as frequent as the next most common stem cell type.

How do Mesenchymal Stem Cells Work?

MSCs occur in nearly all tissues found our body, but they are found most commonly in bone marrow, adipose tissue, umbilical cord tissue, umbilical cord blood, and the placenta. Beyond being multifunctional and differentiating into multiple cell lineages, they secrete a variety of cytokines and growth factors to bring about immune modulation and promote injury healing and tissue regeneration.

MSCs are ideally suited for clinical applications of cell therapy because of their multiple lineage differentiation potential. They can be differentiated in vitro or in vivo into different types of functional cells to replace aged or damaged cells.

Types of Mesenchymal Stem Cell (MSC) Therapies

In some applications of cell therapy, cell types derived from MSCs in the laboratory are transplanted into the recipients in combination with biomaterials as tissue engineering (TE) products. In autologous transplantation, MSCs can home to injured tissue sites in the body and persist for some time.

In the majority of cell-based medicines, mesenchymal stem/stromal cells (MSCs) are used for two major reasons: their potent immunomodulatory activity and their ability to secrete a wide range of trophic and growth factors. Immunomodulation has been reported and well-documented for MSCs derived from multiple tissue sources, including bone marrow, adipose tissue, umbilical cord, and Wharton’s jelly. Because of these immunomodulatory properties, MSCs can suppress both acute and chronic inflammation—an example being the approval of remestemcel-L (formerly Prochymal) for the treatment of steroid-refractory acute GvHD. This therapy is approved in specific jurisdictions, including Canada and Japan. In the United States, remestemcel-L was approved in December 2024 under the brand name Ryoncil for pediatric patients (≥2 months old) with steroid-refractory acute GvHD.

Cell therapy studies in recent years have also been focusing on components derived from MSCs instead of whole MSCs. These components include extracellular vesicles (EVs), microvesicles and exosomes, each exhibiting different biological activities. For instance, exosomes from MSCs have been reported to have functions similar to whole MSCs, including repair of tissue damage, suppression of inflammatory responses, and modulation of the immune system.

Globally Approved MSC-Based Products

As of today, 12 MSC-based cell therapies have received global approvals, including:

Republic of Korea:

• Queencell from Anterogen
• Cellgram AMI from Pharmicell
• Cupistem from Anterogen
• Cartistem from Medipost
• NeuroNataR from Corestem

Japan:

• Temcell HS from JCR Pharmaceuticals
• Stemirac from Nipro Corporation

Europe:

• Holoclar from Chiesi Farmaceutici
• *Previously, Alofisel from Takeda/TiGenix was approved by the EMA, but it was withdrawn in December 2024.

India:

• Stempeucel from Stempeutics

Iran:

• MesestroCell from Cell Tech Pharmed

Canada and the U.S. have approved:

• Prochymal, also branded as Ryoncil (remestemcel-L) from Mesoblast

China has given Conditional Approval to one product:

• Ruibosheng, an umbilical cord-derived MSC product (amimestrocel injection) from Platinum Life Excellence

Market competitors have also developed 17 biomaterial-based MSC and MSC progenitor products, which are predominantly being used for orthopedic indications. The cosmetic industry has released 20 products that utilize MSC-derived conditioned or spent culture media.

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