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Review
. 2025 Feb 4;10(3):e70000.
doi: 10.1002/btm2.70000. eCollection 2025 May.

Stem cell therapies in the clinic

Shrinivas Acharya  1   2 , Suyog Shaha  1   2 , Michael Griffith Bibbey  1   2 , Malini Mukherji  1   2 , Zongmin Zhao  3 , Samir Mitragotri  1   2
Affiliations
Review

Stem cell therapies in the clinic

Shrinivas Acharya et al. Bioeng Transl Med. .

Abstract

Stem cell therapies have emerged as a transformative approach in modern medicine, with the potential to address and possibly cure a broad spectrum of diseases. These therapies utilize living stem cells that can perform complex biological functions not replicable by traditional drugs. Stem cell therapies have an expanding therapeutic landscape, with several products already approved and numerous clinical trials underway. Among the various stem cell types, hematopoietic stem cells (HSCs) and mesenchymal stem cells (MSCs) are most widely studied. In this review, we provide a detailed analysis of the current clinical landscape of stem cell therapies. We review 27 stem cell products that have received regulatory approvals and discuss 800 ongoing clinical trials, with a focus on HSCs and MSCs. We also discuss the critical challenges to be addressed to facilitate the clinical translation of stem cell therapies.

Keywords: HSC; MSC; cell; cell therapy; clinical translation; clinical trials; iPSC; stem cell.

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Conflict of interest statement

SM and ZZ are inventors on patent applications in the field of cell therapies (owned and managed by Harvard University).

Figures

FIGURE 1
FIGURE 1
Common types of stem cells used in approved or clinically investigated stem cell therapies. HSCs and MSCs dominate the clinical landscape of stem cell therapies.
FIGURE 2
FIGURE 2
Timeline of regulatory approvals of stem cell products.
FIGURE 3
FIGURE 3
Analysis of approved stem cell therapy products. (a) Categorization of approved products by stem cell type. (b) Categorization of stem cell approvals by region.
FIGURE 4
FIGURE 4
Overall landscape of stem cell clinical trials. Eight hundred stem cell clinical trials were analyzed and classified based on: (a) Type of cell; (b) Phase of trial; (c) Status of trial; and (d) Source of cell. Not Applicable (NA).
FIGURE 5
FIGURE 5
Landscape of HSC active clinical trials. Three hundred and thirty‐eight HSC clinical trials were analyzed and classified based on: (a) Phase of trial; (b) Source of cell; (c) Genetic modification; (d) Administration method, which was further classified into systemic and local routes; and (e) Indication type. Not applicable (NA).
FIGURE 6
FIGURE 6
Landscape of MSC active clinical trials. Three hundred and forty‐six MSC clinical trials were analyzed and classified based on: (a) Phase of trial; (b) Source of cell; (c) Administration method, which was further classified into systemic and local routes; and (d) Indication type. Not applicable (NA).
FIGURE 7
FIGURE 7
Current landscape of other stem cell clinical trials. One hundred and thirteen trials were analyzed and categorized based on the specificity of stem cell.
FIGURE 8
FIGURE 8
Challenges for clinical translation of stem cell therapies. For MSC, HSC, and iPSC cell types, various challenges were identified and categorized based on: (a) Source variability; (b) Safety; (c) Functional heterogeneity; (d) Viability and activity; and (e) Targeting ability.
See this image and copyright information in PMC

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