FDA announces approval of the first gene therapy m

2022-08-24
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FDA announces approval of the first gene therapy method in the United States Abstract: from the perspective of the whole field, liquid biopsy and gene testing are the entrances to the precision medicine fields such as car-t. according to the national standard gb/large plastic market, there are currently 10 4156 (4) foreign companies' research and the progress of submitting listing applications of "metal cupping test method", and this year is likely to be the first year of the listing of car-t therapy, so, Next, we also need to continue to pay attention to the promotion of car-t for new drug research and development in China

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the U.S. government approved a treatment for leukemia based on the transformation of patients' autoimmune cells on August 30, which is the first gene therapy approved in the United States. Experts believe that this has opened a new chapter in cancer treatment

the US Food and Drug Administration issued a statement on the same day that Novartis' new therapy has been approved for the treatment of patients with relapsed and refractory B-cell acute lymphoblastic leukemia under the age of 25. This is a "historic move", which will "usher in a new way to treat cancer and other life-threatening serious diseases"

the new therapy is a chimeric antigen receptor T cell (car-t) therapy that has developed rapidly in recent years. It first collects T cells that play an important role in the immune response from the patient, and then reprogrammes them. The obtained T cells contain chimeric antigen receptors, which can recognize and attack cancerous cells, so they can be reinjected into the patient for treatment

the results of a clinical trial involving 63 patients showed that 83% of patients were relieved after receiving the new method for 3 months. One year after treatment, the recurrence rate was 64%, and the survival rate was 79%

Scott Gottlieb, director of the US drug administration, commented, "the ability to reprogram patients' own cells and use them to attack fatal cancer means that we are entering the Xinjiang region of medical innovation. New technologies such as gene therapy and cell therapy have the potential to transform medicine and become a turning point for the treatment and even cure of many thorny diseases."

b-cell acute lymphoblastic leukemia is a type of leukemia that is difficult to treat and easy to relapse. In the past, the treatment plan was relatively limited. The 5-year relapse free survival rate of children and adolescents was only 10% to 30%

Novartis said that the new treatment is a one-time treatment, priced at $475000, but if the effect is not seen in the first month, the patient will not be charged. In contrast, bone marrow transplantation, a common treatment for leukemia, costs between $540000 and $800000 in the first year in the United States. Novartis also plans to apply for the treatment of adult B-cell lymphoma in the United States and Europe this year, and apply for the listing of the treatment outside the United States and Europe next year

Carl Joan, one of the pioneers of car-t therapy and a professor at the University of Pennsylvania in the United States, said that approving the listing of new therapies is a "huge step" forward for personalized cancer therapy. Next, they will continue to cooperate with Novartis to promote the use of this therapy to treat other types of cancer

at present, in addition to Novartis, American kite pharmaceutical company and Juno treatment company are also developing car-t products. But earlier this year, Juno therapy officially terminated the clinical trial of gene therapy for adult recurrent refractory B-cell acute lymphoblastic leukemia due to the death of several patients with brain edema in the clinical trial

Kenneth Anderson, President of the American Society of Hematology, said in a statement that this approval "marks an important shift in the paradigm of blood cancer treatment", but at present it is only approved for the treatment of a small number of young patients, and the importance should not be exaggerated. In general, car-t therapy needs more "the design center will try to connect different industries in one research to ensure that it can effectively treat a wider population and reduce side effects

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