In recent years, with the development of stem cell technology, deepening knowledge and accumulation of experience, stem cell therapy has gradually become a hot topic in the field of biomedicine. In order to standardize and guide the non-clinical research and evaluation of human-derived stem cell products, the Center for Drug Evaluation (CDE) of the State Drug Administration issued a notice on October 7, 2023 to publicly solicit opinions on the “Technical Guiding Principles for Non-clinical Research of Human-derived Stem Cell Products (Draft for Comment)” (hereinafter referred to as the “Guiding Principles”), which clarifies that organoid technology can provide useful supplementary information for the evaluation of the non-clinical effectiveness and safety of human-derived stem cell products.
New technologies such as organoids and microfluidics are included in the “Guiding Principles”
“Technical Guiding Principles for Non-clinical Research on Human Stem Cell Products (Draft for Comments)” is in “Important points of concern when formulating non-clinical research strategies” related chapters point out:
Each animal species/model has its strengths and weaknesses, and no single model can fully predict the effectiveness and safety of stem cell products in patient populations. Therefore, when selecting an animal species, the relevance and limitations of the animal species/model used to humans should be evaluated. If there are multiple relevant animal species/models and these animal species/models can provide complementary evidence, it is recommended to use multiple animal species/models to conduct research. When relevant animal species/models are missing, cell- and tissue-based models (such as two-dimensional or three-dimensional tissue models, organoids and microfluidic models, etc.) may provide useful supplementary information for the assessment of non-clinical efficacy and safety.
In recent years, stem cell therapy has gradually become a hot topic in the biomedical field, providing new potential treatments for many refractory diseases. More than ten stem cell products have been launched overseas. At present, the number of domestic stem cell product applications is increasing, involving a wide range of cell types and a wide range of indications.
Stem cell products are an important type of cell therapy products, and they have their own particularities in non-clinical research. However, there are currently no relevant guiding principles and technical requirements for non-clinical research on stem cell products in China. In this case,It is urgent and urgent to explore non-clinical research and evaluation strategies for stem cell products, establish regulatory standards, and scientifically guide the research and development of stem cell products.
Non-clinical research is an important part of drug development. The active ingredients of stem cell products are cells with certain biological functions and different degrees of differentiation potential. Compared with conventional therapeutic drugs, their therapeutic mechanisms, in vivo activity and toxicity may be significantly different. The interaction between cells and the human body is also more species-specific. Therefore, compared with conventional therapeutic drugs, non-clinical research on stem cell products has its own particularities.
The purpose of CDE publishing the “Technical Guiding Principles for Non-clinical Research of Human Stem Cell Products (Draft for Comment)”It is to standardize and guide non-clinical research on human stem cell products, based on the technical guidelines for non-clinical research of cell therapy products and gene-modified cell therapy products that have been published and implemented in China, based on the characteristics of stem cell products, combined with the relevant guiding principles and technical requirements of international regulatory agencies/industry associations, as well as the research progress and current understanding of stem cell products, the draft was drafted based on the current research and development status of my country, and specific requirements for non-clinical research of stem cell products were proposed.
The scope of application of this “Guiding Principles” includes: adult stem cells (ASCs), human embryonic stem cells (ESCs) originating from humans and induced pluripotent stem cells (iPSCs), stem cells and their derivative cell products obtained through a series of in vitro operations involving stem cells, generally including amplification, genetic modification, induced differentiation, trans (differentiation), etc.
In 2021, the Ministry of Science and Technology issued the “Notice on Soliciting Comments on the 2021 Project Application Guidelines for the Six Key Special Projects of the “14th Five-Year Plan” National Key Research and Development Plan”, which will“Organoid-based malignant tumor disease model”Listed in the “14th Five-Year Plan” national key research and development planThe first batch of key special tasks to be launched;
In 2021, the Drug Evaluation Center of the State Drug Administration issued the “Technical Guiding Principles for Non-clinical Research and Evaluation of Gene Therapy Products (Trial)” and “Technical Guiding Principles for Non-clinical Research and Evaluation of Gene Modified Cell Therapy Products (Trial)”,For the first time, organoids are included in the guiding principles for gene therapy and gene-modified cell therapy products;
In 2022, the Ministry of Science and Technology issued the “2022 Annual Application Guidelines for the Key Special Projects of Stem Cell Research and Organ Repair” on the National Key R&D Plan (Draft for Comments)”,Highlight the great value of organoid disease models in tumor research.
Beike Biological Organoid R&D Platform
Organoids, as an in vitro research model derived from three-dimensional culture of stem cells, can be used in basic research such as regenerative medicine and disease occurrence mechanism, drug sensitivity testing, personalized treatment and drug screening.
The organoid R&D platform led by Professor Cai Cheguo, chief scientist of Beike Biology is one of the seven R&D platforms of Beike Biology.
Accurate results
Future direction
In the future, we will build a tumor organoid library of various tissues and conduct multiple diseases
bed research and technology transformation.