Stem cell are known as “universal cells” because of their unique multidirectional differentiation potential and self-renewal ability, giving them unparalleled advantages in tissue repair, organ regeneration and disease treatment.
After a long journey from basic research to clinical trials, and then to industrialization, what is the application potential and international environment of stem cell therapy at this stage?
What about the current situation and prospects of stem cell therapy?
PART 1: In which convenience stem cells have application potential
Stem cells have strong regenerative and repair capabilities, and have been clinically studied in hundreds of diseases, and have potential applications in the prevention and treatment of many major diseases and rehabilitation.
1) Immune system diseases
Mesenchymal stem cells can be used to treat diseases of the immune system, such as rheumatoid arthritis, red lupus.
By modulating the patient’s immune system, the patient’s symptoms are relieved.
2) graft-versus-host disease
aGVHD patients treated with mesenchymal stem cells had a reduced incidence of cGVHD in later stage.
3) Orthopaedic diseases and sports injuries
Mesenchymal stem cells treat knee osteoarthritis by direct tissue growth, differentiation into specific chondrocytes and promoting tissue repair, and create the optimal environment for joint repair through anti-inflammatory and immunomodulatory paracrine.
Multiple clinical trials have shown that intra-articular mesenchymal stem cell injection can reduce intraarticular inflammatory cells, promote articular cartilage growth, and further improve patient pain and function.
4) Diabetes
Mesenchymal stem cells can secrete a variety of cytokines and growth factors, such as vascular endothelial growth factor (VEGF), hepatocyte growth factor (HGF), fibroblast growth factor (bFGF), transforming growth factor-β (TGF-β) and insulin-like growth factor (IGF-1), etc. It has anti-inflammatory, immune regulation, promoting angiogenesis and cell regeneration and repair.
Mesenchymal stem cells play a beneficial role in the treatment of diabetes by promoting the regeneration of islet cells, protecting endogenous islet cells and improving insulin resistance.
5) Anti-aging and plastic beauty
After many years of basic and clinical research in the field of plastic repair and aesthetic surgery, mesenchymal stem cells and their derivatives have proved that they have good therapeutic effects in promoting skin regeneration and repair, tissue vascularization, soft tissue regeneration, bone and cartilage repair, multi-tissue rejuvenation and hair follicle regeneration.
Through the characteristics of stem cell proliferation, multidirectional differentiation, tissue repair and immune regulation, it is used to improve skin texture, or improve mental state, improve the repair ability of autologous tissue, and slow down the aging process, so as to achieve the purpose of anti-aging.
6) Blood system diseases
Hematopoietic stem cell transplantation can effectively treat a variety of blood system diseases, such as leukemia, lymphoma, multiple myeloma and aplastic anemia, etc.
Healthy hematopoietic stem cells can re-establish a normal blood and immune system in the body, improving the survival rate and quality of life of patients.
PART 2 Global clinical status of stem cells
In stem cell clinical research, the United States dominates, but in terms of listing, due to the open attitude of Japan, South Korea and other countries, more and more stem cell products are listed in Japan and South Korea, and there are 15 mesenchymal stem cell treatment products that have been approved internationally.
(Korea owns 5 products, Japan 3, India 1, Iran 1, Europe 2, the United States 1, China 1, Canada & New Zealand 1, etc.)
Stem cell treatments for disease are becoming a broader reality.
Recently, the field of stem cell therapy has ushered in exciting and significant progress! The United States and China have successfully approved stem cell therapy products, a milestone that has brought new hope to countless patients.
Currently, there 15 MSCs therapies approved by regulators around the world.
These regulators include:
The European Medicines Agency (EMA), the Food and Drug Administration (FDA) of the United States (USA), the Ministry of Food and Drug Safety (MFDS) of the Republic of Korea, the Medicines Regulatory Authority of India (DCGI), the Therapeutic Goods Administration of Australia (TGA) and the Medicines and Medical Devices Administration of Japan (PMDA), China Food and Drug Administration (NMPA), etc.
Among them, the original Prochymal (later renamed Remestemcel-L) product held by Osiris Therapeutics, a company in the United States, in 2010, although it a potential orphan drug for type I diabetes, but the listing process in the United States has repeatedly suffered setbacks.
Thanks to the continuous research and development of Mesoblast relay, Mesoblast took the lead in Canada, New Zealand, Japan and other countries to make breakthroughs, successfully listed, and finally successfully opened the door of the US market in 2024.
In 2025, China’s stem cell therapy also usher in a historic moment, on January 2, the “Emmetosay” injection of Bosheng Excellence Biology was successfully approved by the State Food and Drug Administration for the treatment of acute graft versus host disease with digestive tract involvement and hormone therapy failure above 14 years old, which is the first stem cell therapy drug in China.
It has set an important milestone in the development of regenerative medicine in China.
PART 3 International environment of stem cell therapy
In terms of clinical research, the United States is the country with the largest number of international stem cell clinical research, and 80% of the world’s approved cord blood hematopoietic stem cell products come from the United States.
Europe is second tier in stem cell research and transformation;
Japan ushered in a new era of induced pluripotent stem cells (iPSC); South Korea listed fast;
China’s stem cell clinical research has now entered the development stage, and 1 product is currently on the market)
Comparison of regulatory models in different countries
United States: Regulated by the U.S. Food and Drug Administration (FDA).
Low-risk products can used directly in hospitals (usually placenta stem cell therapy);
High-risk products must approved by the FDA (for the treatment of rare diseases, critical diseases, etc., a New drug application (NDA) must submitted, and can approved only).
Europe: Mainly by the European Medicines Agency (EMA) and the relevant agencies of the member states are jointly responsible, member states have certain flexibility in supervision, both unified and independent.
All 27 EU member states evaluated and approved by EMA, and pricing and medical insurance benefits decided by each member state.
For medical products declared according to advanced technology, they shall approved and administered by EMA;
Some autologous stem cell treatments may determined directly by the hospital under the “hospital exemption clause”.
South Korea: Regulated by the Korea Food and Drug Safety Agency (KFDA, formerly the Korea Food and Drug Administration), South Korea is open to stem cell technology and provides “special support” for cell approval:
If the phase 1 results of autologous cell products published in professional journals, the submission of data will exempted, allowing the submission of data after the launch of stem cell products that treat life-threatening diseases.
Japan and China have similar regulations on stem cell therapy.
Japan: The final regulatory department is the Ministry of Health, Labor and Welfare (MHLW) Japan’s implementation of the “dual-track” regulation of stem cells, stem cell drugs for the purpose of listing conditional listing, not for the purpose of listing stem cell technology hierarchical management.
Level 1 (high-risk technologies) :
Involving cutting-edge technologies such as embryonic stem cells and induced pluripotent stem cells (iPS cells) that have not applied to human beings, they need to reviewed jointly by the “Special Recognition Regenerative Medicine Committee”, the Health and Health Science Review Council and the Minister of Health, Health and Labor;
Level 2 (medium risk technology) :
For autologous cell therapies that have changed biological characteristics, such as autologous fat stem cells for diabetes and peripheral blood stem cell transplantation for rheumatoid arthritis, the approval process is relatively simplified and is mainly responsible for the “Special Recognition regenerative Medicine Committee”;
Level 3 (low risk technology) :
For autologous cell treatments that do not change the original characteristics, such as the treatment of tumor by autologous immune cells, the treatment of bone diseases by plasma platelet extracts, and the reconstruction of the breast by adipose stem cells after mastectomy, only the review of the “regenerative Medicine Committee” required.
As a drug, stem cells regulated by NMPA, and new drugs must approved by the Center for Drug Evaluation (CDE) of the State Food and Drug Administration for clinical trial approval;
Stem cells as medical technology: regulated by the National Health and Health Commission (NHC), medical technology adopts the filing management method of institutions/projects applying to the Health and Health Commission.
As of December 31, 2024, the mainland provinces and cities have declared about 146 stem cell drugs, a total of 117 clinical trial applications for implied approval.
As of December 4, 2023, 141 stem cell institutions have registered.
In recent years, the stem cell medical technology access “pilot areas” in Hainan, Shenzhen, Yunnan, Beijing, Tianjin and other places have opened, and many places have blossomed, providing policy support for the clinical transformation and application of stem cells, and its believed that 2025 will the first year of stem cell technology transformation and application.
