After the big wave of sand and sand, CAR-T therapy research still has a long way to go CAR-T therapy "is high hopes", but there is still a lot of room for improvement in the rational view of new targets, and CAR-T research has a long way to go

According to a report released by Frost & Sullivan in June this year, China's CAR-T market is expected to start to grow from 2021, with a market size of 200 million yuan, and the market size will further grow to 5.3 billion yuan and 28.9 billion yuan in 2024 and 2030, respectively.

However, CAR-T therapy should be viewed rationally. "In China, there are many studies on clinical projects related to cell therapy, and homogenization is also very strong, although it is currently an effective and popular method for treating malignant tumors of the blood lymphatic system, but it cannot cure all patients." Professor Ma Jun, director of the Harbin Institute of Hematology and Tumor Research, told the Health Times reporter.

<h1 class="pgc-h-arrow-right" data-track="7" >CAR-T therapy "has high hopes", but it should be treated rationally</h1>

"The listing of CAR-T cell therapy is the gospel of the majority of cancer friends, but I hope that the price of treatment is not too expensive, so that this hope becomes despair!" Lymphoma patient Xu Feng (pseudonym) lamented with the Health Times reporter that this year is the 5th year since she was diagnosed with lymphoma, and she hopes to be able to use the new treatment as soon as possible.

According to the data of Industrial Securities Research Report, at present, the United States and China have the largest number of research on cell therapy in the world. From 2020 to 2021, the number of INDs (Clinical Trial Research Applications) in China increased from 264 to 420. Among them, the attention of CAR-T products is the highest, from pharmaceutical giants such as Novartis, BMS, gilead, etc., to emerging domestic biopharmaceutical companies represented by Legendary Biologics, WuXi Junuo, and Fuxing Kate have successively entered the competitive track.

"At present, the treatment of tumors is still dominated by chemotherapy, whether it is for lymphoma, leukemia or solid tumors, chemotherapy is still the cornerstone of tumor treatment." Ma Jun told the Health Times reporter that now due to the formation of tumor treatment to cell drug therapy and immunosuppressive point treatment-based change, so the related innovative therapy is also very much, of which, small molecule inhibitors now have more than 4,000 targets for the treatment of malignant tumors, related drugs have now been applied to more than 40 tumors, is the future direction of progress, most of the tumor treatment or need to be solved through chemotherapy.

Zhu Jun, secretary of the Party Committee of Peking University Cancer Hospital, told the Health Times reporter that due to the characteristics of CAR-T cell therapy itself, the current clinical research results in this field are mainly focused on the treatment of blood tumors, especially in the clinical treatment of refractory blood tumors, which have considerable potential and many possibilities. When the current treatment is not particularly effective, there will still be a 50% to 70% effective rate with this method, bringing lasting relief to patients, and even a small number of patients can be completely cured, so this treatment method is placed on high hopes.

However, "CAR-T cell therapy has very high requirements from the collection and transportation of cells to the culture, reserve, quality control, etc. of the entire technical background, and there are still some specifications and requirements in the process of clinical use, and the drug process must be strictly controlled and controlled." From this point of view, I don't think that every doctor everywhere can apply this clinical treatment, and not every patient is suitable for this treatment, for example, some patients have a weakened immune system due to early treatment, and their T cells cannot be used to prepare CAR-T. Zhu Jun said.

<h1 class="pgc-h-arrow-right" data-track="9" > there is still a lot of room for improvement in new targets, and CAR-T research has a long way to go</h1>

Health Times reporters combined with the national drug evaluation center and Clinicaltrials database data statistics found that there are currently 21 domestic CAR-T product clinical trials in research, most of the indications are blood tumors. And most products are still in the early stages of development, and many trials are underway but not yet recruited.

"R&D is overcrowded, and there will inevitably be excessive competition in the follow-up." Chen Kaixian, an academician of the Chinese Academy of Sciences, expressed his concerns at the 7th International Symposium on Biopharmaceutical Innovation and Research and Development.

Zhu Jun told the Health Times reporter, "At present, most OF the CAR-T products are concentrated on the CD-19 target because this technical route is relatively mature, but this also shows that there is still a lot of room for improvement in the new targets of global CAR-T drug research and development in the future." ”

"At present, China's CAR-T product research is still a long way to go. The biggest difficulty in target selection is to find its disease-causing gene, and only in this way can the tumor be overcome. Ma Jun mentioned that at present, there are 68 pre-clinical targets for CAR-T, and because everyone competes with each other for different targets, there may be a situation where the targets are more crowded.

In December 2017, the "Technical Guidelines for the Research and Evaluation of Cell Therapy Products (Trial)" was introduced, marking that China's cell therapy research and development has officially entered a new stage of development. The principle points out that the applicant can formulate the clinical research staging and research design according to the specific characteristics of the proposed product, which can generally be divided into two parts: the early clinical trial stage and the confirmatory clinical trial stage, which can greatly shorten the time to market of CAR-T therapy.

In February 2021, the State Food and Drug Administration issued the "Technical Guidelines for Clinical Trials of Immune Cell Therapy Products (Trial)", which provides more detailed technical guidance for immune cell therapy products that are developed and registered in accordance with the Drug Administration Law of the People's Republic of China, the Measures for the Administration of Drug Registration and other relevant laws and regulations on drug administration for the purpose of domestic registration and listing, and provide more detailed technical guidance in the overall planning, design of trial plans, trial implementation and data analysis of clinical trials.

"The introduction of two technical guidelines, which clearly regulate cell therapy as a drug, has brought unprecedented opportunities for the standardized development of the field of cell therapy in China." There are still many known and unknown risks in the clinical use of cell therapy products, and listing declarations in accordance with the process of drug research and development can make the efficacy and safety of the product more controllable, reduce the risk of clinical use of users, and truly benefit patients. Zhu Jun said.

In Zhu Jun's view, the listed products in the field of CAR-T cell therapy in China have changed from "none" to "have", behind which the regulatory policy in the field of cell therapy has become "orderly".

"At present, there are more than 100 kinds of disease tumor research directions are different, innovation era multi-target research or homogeneous research, I think this is not wrong, there is no harm, but for poor quality products, must be eliminated, in the future the relevant target research more and more, the big wave of sand can always find the best quality products." Ma Jun said.