Japan's first allogeneic stem cell treatment drug was born!

Source: from February 24, 2016 to March 29, 2016, JCR Pharmaceutical Co., Ltd of Japan began to sell the allogeneic mesenchymal stem cell product temcell The product is used for the treatment of acute graft-versus-host reaction (GVHD), one of the serious complications after hematopoietic stem cell transplantation, and was approved by the Ministry of health, labor and welfare of Japan in September 2015 Temcell's price is 860000 yen (about 50000 yuan) for one bag One course of treatment is to lose two bags every week, a total of eight bags in four weeks The product will provide treatment for 260 patients in Japan by 2020, with sales forecast of about 3 billion 500 million yen What's the sanctity of temcell, which makes the cell therapy industry boil? Let's review the R & D process of the product 1 Temcell, also known as Prochymal, was first developed by Osiris in the United States Prochymal obtained production approval in Canada and New Zealand in 2012, but it was not officially put into production and sales due to financial reasons In October 2013, Osiris transferred the ownership of the product to mesoblast, Australia In September 2014, JCR of Japan filed a new drug application to the Ministry of health, labor and welfare of Japan in the name of temcell's product In September 2015, it obtained the production approval 2 Introduction of relevant regulations on stem cells: in 2013, Japan issued the management standards on cell products, classifying cell products into new categories: regenerative medical products According to the management method, if the safety and effectiveness of products with different homogeneity can be determined and estimated, they can be recognized by additional conditions and time limit, especially in the early stage Then, the security and effectiveness of the system are verified again The change of this policy makes the management of personalized medical treatment take a big step forward again 3 Temcell is a fully approved drug Temcell's review comments stated that the variety was approved without additional conditions and time limit (meaning full approval) The corresponding full approval is the conditional early approval, also known as semi approval) There are only a few approved cell therapy products (including stem cells and other cells) in the world, among which the allogeneic stem cell products are one hand So what is the significance of temcell's approval and listing in Japan? 1 The feasibility of large-scale production of cell products: most cell therapy in the world has always been based on the source of self cells, and then this personalized medical treatment will inevitably make its treatment cost expensive However, stem cells from allogeneic sources are expected to achieve large-scale production of cell therapy, and the continuous optimization of production process will inevitably reduce the cost of treatment, thus reducing the burden of medical treatment However, another voice questions whether the cell products produced by this kind of "big pot rice" are superior to the customized "own stem cells" from the perspective of curative effect More questions need to be studied and answered one by one 2 Promote the development of China's cell industry: since the great impact on the stem cell industry in 2012, China's stem cell policy has been constantly updated and improved Including the measures for the management of stem cell clinical research (Trial) and the guiding principles for the quality control and preclinical research of stem cell preparations (Trial) jointly issued by the health and Family Planning Commission and CFDA in August 2015; and December 2015 The "notice on carrying out the filing work of stem cell clinical research institutions" issued by the national health and Family Planning Commission in June all indicated that China's stem cell management is more standardized and standardized However, there is still a long way to go before "China's first stem cell product" is approved The approval of temcell will provide us with a reference 3 Coexistence of angels and Demons: Although the immunogenicity of mesenchymal stem cells is low and they are used for GVHD treatment based on the characteristics of immunosuppression, excessive immunosuppression can reduce GVHD At the same time, there are also side effects such as leukopenia, thrombocytopenia, infection, septicemia, kidney injury, tumor growth, etc Therefore, treatment must be carried out in the medical facilities that can implement emergency treatment Therefore, how to control the "double-sided blade" will be the next problem But the gene regulation technology of car-t, a new cancer immunotherapy technology, may solve this problem Although the industrialization of stem cell therapy is full of difficulties, we believe that the spring of stem cell industry is coming, let's wait and see.