— Global Clinical Trials Landscape Focusing on Adipose-Derived MSCs (2025 Edition)
table of contents
Introduction: New possibilities in the treatment of autoimmune and inflammatory diseases
Autoimmune diseases often do not improve sufficiently with conventional treatments alone. In recent years, new therapeutic approaches using stem cells have been attracting global attention in the field of regenerative medicine
Autoimmune and inflammatory diseases such as rheumatoid arthritis, systemic lupus erythematosus (SLE), Crohn's disease, and multiple sclerosis (MS) are a group of diseases that cause chronic inflammation and tissue damage due to immune abnormalities. While advances in existing immunosuppressive therapies and biological agents have improved the prognosis for many patients, there are still a significant number of treatment-resistant cases and patients who experience repeated relapses
Against this backdrop, stem cell therapy has attracted considerable attention in recent years. In particular, mesenchymal stem cells (MSCs) are being researched worldwide as a new therapeutic approach because they possess anti-inflammatory, immunomodulatory, and tissue repair properties
A review article published in Medical Science Monitor in 2025, "Global clinical trial landscape of stem cell therapies for autoimmune and inflammatory diseases," comprehensively analyzed 1,136 clinical trials of stem cell therapies for autoimmune and inflammatory diseases. This article will focus particularly on adipose-derived MSCs (AD-MSCs) and discuss their current development status and future prospects.
Data from over 20 years and 1,136 clinical trials worldwide
This study analyzes 1,136 stem cell clinical trials related to autoimmune and inflammatory diseases registered in the TrialTrove database up to October 2024
The number of clinical trials for stem cell therapy has increased rapidly since the early 2000s, reaching 73 in 2019. This reflects the expansion of global research investment in the field of regenerative medicine and the growing expectations for the immunomodulatory capabilities of MSCs
The following items were evaluated in the analysis
- Trends in the number of clinical trials
- Phase-by-phase transition rates
- Regional distribution
- Sponsor Classification
- Disease-Specific Clinical Outcomes
- Characteristics of different stem cell sources
- Safety analysis based on 36 randomized controlled trials (2,076 cases)
A key feature of this paper is that, rather than focusing on a single disease, it provides a comprehensive overview of "the current state of stem cell therapy in autoimmune and inflammatory diseases as a whole."
Why are adipose-derived MSCs (AD-MSCs) attracting attention?
MSCs can be harvested from various tissues, including bone marrow, umbilical cord, and adipose tissue, but in recent years, adipose-derived MSCs (AD-MSCs) have attracted particular attention
Adipose tissue is relatively easy to harvest and has the advantage of yielding a large number of cells. Furthermore, AD-MSCs possess immunomodulatory and anti-inflammatory properties, and are expected to have applications in the treatment of chronic inflammatory diseases
Furthermore, adipose-derived MSCs are thought to have the following advantages:
- It can be collected with relatively little invasiveness
- High cell proliferation capacity
- Cell collection efficiency
- Highly practical in terms of cultivation and manufacturing
- High affinity with regenerative medicine
Therefore, research is being conducted not only in the field of autoimmune diseases, but also in a wide range of fields such as orthopedics, dermatology, and neurology
In recent years, attention has been focused not only on MSCs themselves, but also on MSC-derived exosomes and cytokine secretion mechanisms
"darvadstrocel (Cx601)," an AD-MSC product attracting attention in the field of Crohn's disease
In this paper, darvadstrocel (Cx601) is introduced as a representative example of the results achieved using adipose-derived MSCs
Darvadstrocel is a therapeutic product that utilizes adipose-derived mesenchymal stem cells (MSCs), and its effectiveness has been reported in clinical trials for refractory Crohn's disease-related anal fistulas
In the studies cited in the paper,
- Remission rate: 56.3%
- Placebo group: 38.6%
- P=0.010
The results showed a significant improvement
Safety data: Favorable profile in a meta-analysis of 2,076 RCTs
In stem cell therapy, verifying not only efficacy but also safety is extremely important
This paper presents a meta-analysis of 36 randomized controlled trials (RCTs) involving a total of 2,076 patients, demonstrating a favorable safety profile for MSC therapy as a whole
While significant safety signals are currently limited, the authors point out that careful long-term evaluation is needed regarding the following points
- Long-term immunological effects
- Cellular behavior under chronic inflammatory conditions
- Safety during repeated administration
- Lack of long-term tracking data
Stem cell therapy remains a developing field, and accumulating long-term safety data, in addition to short-term efficacy data, will be crucial going forward
Globally expanding research and development
In terms of geographical distribution, China, the United States, and Europe occupy the major regions, with particularly rapid growth in the Asian region being noteworthy
In the sponsor classification,
- Academic institutions: 48.0%
- Business: 25.9%
- Joint research: 23.8%
This result indicates that while research is based on academia, a transition to industrialization is progressing
On the other hand, the transition rate from Phase II to Phase III remains at only 10.2%, revealing that many studies are not progressing to the practical application stage
It is believed that several challenges exist behind this, including manufacturing standardization, costs, and regulatory compliance
Future outlook: Standardization and technological integration will be key
The paper identifies the following four points as important challenges for the future development of stem cell therapy
① Standardization of manufacturing protocols
Standardization of cell quality and establishment of a GMP compliance system
② Overcoming the Phase II Bottleneck
Establishing a system to smoothly transition promising initial tests to later tests
③ Integration with AI and gene editing technologies
Product quality improvement through advanced technologies such as AI analysis and CRISPR
④ Coordination of international regulations
Promoting international collaborative trials and the standardization of approval criteria
Going forward, it is expected that the importance of quality control, standardization, and long-term safety evaluation will increase even further, moving beyond the mere "research stage" to actual medical implementation
summary
This paper is an important review that comprehensively presents global trends in stem cell therapy for autoimmune and inflammatory diseases through a large-scale analysis spanning over 20 years and 1,136 studies
In particular, adipose-derived MSCs (AD-MSCs)
- Ease of collection
- Cell collection efficiency
- Practicality
- Immunomodulatory effect
These characteristics suggest that clinical applications may advance further in the future
Adipose-derived MSCs have the potential to become even more important in the field of regenerative medicine in the future, and are attracting attention as a new therapeutic strategy for autoimmune and inflammatory diseases
On the other hand, as the 10.2% transition rate to Phase III indicates, there are still many challenges to overcome, such as manufacturing standardization and regulatory development
Stem cell therapy is currently at a turning point, moving from research and development to full-scale clinical implementation, and the progress of future international clinical research is attracting attention
On the other hand, further evidence needs to be accumulated at this point, and large-scale clinical trials and long-term safety evaluations are expected in the future
Supervised by: Yurie Hara (Obstetrician and Gynecologist)