Akademik Veri Yönetim Sistemi
International Symposium Cancer and Stem Cell, İzmir, Türkiye, 02 Aralık 2023, (Tam Metin Bildiri)
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Compliance Rules in Stem Cell Research and Dissemination of Knowledge (KHB Example)
Prof. Alp Can
Ankara University School of Medicine, Department of Histology and Embryology, Laboratories
for Stem Cells and Reproductive Cell Biology
Introduction
The field of stem cells and regenerative medicine represents a promising therapeutic avenue
for patients with limited or no alternative treatments for their conditions. Stem cells offer
diverse therapeutic potential by inhibiting inflammation or cell death, recruiting cells,
promoting blood vessel formation, and specializing into different cell types. This innovative
approach holds promise for several complex human diseases, with recent preclinical studies
showing encouraging results that have led to numerous clinical trials utilizing stem cell
therapies. Transitioning these therapies from laboratory research to market availability involves
adherence to precise regulations and directives outlined by regulatory authorities. This article
aims to guide readers through the translation process, detailing how ideas evolve into clinical
practices specifically concerning stem cell products. It will cover crucial guidelines for securing
approval for clinical trials, focusing on regulations set forth by the United States' Food and Drug
Administration (FDA) and the European Medicine Agency (EMA).
Regulations and Regulatory Bodies
In the United States, the FDA plays a crucial role in certifying clinical trials involving stem-cellbased
products within the United States. When a new drug is presented to a clinical
investigator without prior FDA approval, it may necessitate the submission of an Investigational
New Drug (IND) application (1). This application comprises animal pharmacology and toxicology
data, clinical protocols, and investigator details. Insufficient preclinical evidence, such as
inadequate in vitro or in vivo studies, can prompt the need for modifications or result in
rejection. If the FDA determines that modifications are required for an IND (indicating the
application is seeking approval but hasn't been granted), it signifies that the preclinical studies
provided were deemed insufficient or inadequate for transitioning to a clinical trial.
Consequently, further research is mandated before an amended IND can be resubmitted.
In our country Türkiye, and in many countries around the world, stem cell and cellular therapy
applications are carried out under certain regulations. The type of cell transplanted in stem cell
interventions (such as its level of maturity, stemness and genetic profile, etc.), the method and
frequency of cell administration, cell quantity, and the specific patients/diseases for
transplantation require adherence to established rules. Some of these falls under the control of
official health authorities, and failure to comply with these regulations constitutes a violation of
laws and regulations, thus carrying penal consequences. Meanwhile, others represent universal
recommendations formulated by collective reasoning through civil initiative, independent of
geographical or political boundaries. In our country, the health authority consists of the
Ministry of Health of the Republic of Turkey and its affiliated institutions. Experimenting with
cells, organs, and their derivatives on a human being is addressed under the categories of
"standard treatments" or "experimental treatments."
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Regarding this matter, there exist regulations governing the activities of production centers
(dated October 27, 2010) and regulations pertaining to the licensing of products falling under
advanced therapy medicinal products (dated May 27, 2023). Applications for standard or
experimental treatment by health centers with specific qualifications in the Ministry of Health
and universities are evaluated following submissions to the Directorate of Tissue, Organ
Transplantation, and Dialysis Services under the General Directorate of Health Services.
ISSCR Guidelines
Additionally, the International Society for Stem Cell Research (ISSCR), a civil organization
comprising over 1400 members from approximately 70 countries, has issued a total of four
guidelines since 2006, outlining the rules that should be adhered to in human stem cell
research on an international scale, under the guise of recommendations, provided they do not
contradict each country's respective regulations. Recently, in June 2023, ISSCR updated its
recommendations by releasing a new guide comprising 43 suggestions (Fig 1A) (2, 3).
The Stem Cell E-Bulletin (KHB)
For professionals working in the field of stem cells, a platform for current information
exchange, swift access to local and global news, and particularly a space to discuss local issues
has long been lacking. The Stem Cell E-Bulletin (KHB), initiated in 2014 and still the country's
sole stem cell bulletin, significantly fulfills this need through its free dissemination, exclusive
electronic publication, and accessibility to everyone (Fig 1B) (4). Over the past decade, KHB has
been closely followed not only by experts in the field but also by enthusiasts and students
interested in the subject, sharing numerous articles written by subject matter experts and
covering countless news updates regularly.
Fig 1. (A) ISSCR Standards published in 2023; (B) The Stem Cell E-Bulletin (KHB).
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Acknowledgement
The author thanks to Prof. Gulperi Oktem, and Yigit Uyanıkgil for inviting me to the
International Cancer and Stem Cell Congress.
Conflict of Interest
The author declares no conflict of interest.
References
1. Mousaei Ghasroldasht M, Seok J, Park HS, Liakath Ali, Al-Hendy A. Stem Cell Therapy:
From Idea to Clinical Practice. Int J Mol Sci 23: 2850, 2022.
https://doi.org/10.3390/ijms23052850.
2. Ludwig TE et al. Stem Cell Reports 18: 1744–1752, 2023.
3. Standards for Human Stem Cell Use in Research. ISSCR.
https://www.isscr.org/guidelines.
4. http://www.kokhucrebulteni.com/