Mesenchymal Stem Cell Therapies Approved by Regulatory Agencies around the World

Abstract

Cellular therapy has used mesenchymal stem cells (MSCs), which in cell culture are multipotent progenitors capable of producing a variety of cells limited to the mesoderm layer. There are two types of MSC sources: (1) adult MSCs, which are obtained from bone marrow, adipose tissue, peripheral blood, and dental pulp; and (2) neonatal-tissue-derived MSCs, obtained from extra-embryonic tissues such as the placenta, amnion, and umbilical cord. Until April 2023, 1120 registered clinical trials had been using MSC therapies worldwide, but there are only 12 MSC therapies that have been approved by regulatory agencies for commercialization. Nine of the twelve MSC-approved products are from Asia, with Republic of Korea being the country with the most approved therapies. In the future, MSCs will play an important role in the treatment of many diseases. However, there are many issues to deal with before their application and usage in the medical field. Some strategies have been proposed to face these problems with the hope of reaching the objective of applying these MSC therapies at optimal therapeutic levels.

Perinatal Tissue-Derived Stem Cells: An Emerging Therapeutic Strategy for Challenging Neurodegenerative Diseases

“…The results obtained in preclinical studies using animal models, and even some clinical trials support the ability of MSCs to differentiate into neuronal lineage cells and to promote the functional recovery of injured neuronal tissue through different mechanisms including immunomodulation, trophic actions, neuroprotection, and the stimulation of angiogenesis [22][23][24][25][26]. However, naturally, MSCs do not spontaneously differentiate into a specific neural lineage cell type, but their differentiation into neurons can be promoted by combining multiple nerve-inducing factors.…”

“…Nine of the twelve MSC-approved products are from Asia, and the Republic of Korea is the country with the most approved therapies. Among them, there is NeuroNata-R, an autologous bone marrow MSC therapy with neuroprotective effects that relieves the progression of ALS [22].…”

The U.S. Food and Drug Administration, the mechanism of action, and other considerations for cell-based therapy candidates

“…Although cell-based therapies were first commercialized in the 1990s, only about a dozen have been approved for use in Europe and other countries. 45 There are very few cell-based therapies that have been approved in the USA. It should be clear that the use of cell-based therapies is out of context of the established criteria that has been an accepted for small molecule or recombinant drugs.…”

Advancing diabetes treatment: the role of mesenchymal stem cells in islet transplantation

“…Ethical sourcing ensures that donors are fully informed and agree to the use of their cells for research or therapeutic purposes. Regulatory policies for MSC therapies, governed by agencies such as the FDA and the European Medicines Agency, focus on ensuring the safety, efficacy, and quality of MSC-based products ( 45 ). These regulations necessitate rigorous clinical trials and manufacturing standards to prevent risks such as immunogenicity and tumorigenicity, aiming to safeguard patient health while fostering innovation in MSC therapy development.…”