July 23, 2024

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What is the difference between cancer immunotherapy and gene therapy?

What is the difference between cancer immunotherapy and gene therapy?

What is the difference between cancer immunotherapy and gene therapy?  With the advancement of cancer medicine, more and more new therapies are used to treat cancer. Gene therapy and immunotherapy are among the best. 

In fact, the two popular new therapies, immunotherapy and gene therapy, represent two different types of treatment ideas, but there are also some overlaps. So, what is the relationship between the two? 

Gene therapy

Most diseases are not caused by mutations in a single gene, but by changes in the DNA sequence. But some rare genetic diseases may be caused by mutations in a single gene. Gene therapy is a relatively new treatment method. It has taken a pioneering step in the treatment of these rare genetic diseases, but it is still in the experimental and exploratory stage to a large extent.

The principle of this treatment is to use genetic engineering technology to replace the wrong gene in the body that causes the corresponding disease. These gene mutations cause cells to produce ineffective or dangerous proteins, which can lead to diseases such as severe combined immunodeficiency, sickle cell anemia, hemophilia, cystic fibrosis, spinal muscular atrophy and rare genetic blindness.

Gene therapy can also be used to inactivate or knock out dysfunctional mutant genes to reduce the harmful effects of the gene. Today, most gene therapies are still in clinical trials, but have been approved in the United States for the treatment of two genetic diseases. In 2017, the US FDA approved Luxturna (Voretigene Neparvovec-rzyl) for the treatment of hereditary vision loss that can lead to blindness. This is the first gene therapy product in the United States.
In May 2019, the US FDA approved the new drug Zolgensma to be marketed for specific spinal muscular atrophy. This therapy is the first gene therapy for spinal muscular atrophy. Its price of more than 14 million yuan has attracted great attention and it is called “the most expensive drug in history” by the media.
At present, clinical trials on the effectiveness of gene therapy for several genetic diseases (such as severe combined immunodeficiency and blood diseases such as sickle cell anemia) are also underway.
The rapid development of gene therapy is beyond people’s imagination, giving hope of curing some previously helpless diseases. However, how to ensure the safety of new therapies remains to be studied for a long time.


Immunotherapy is a method of treating cancer by improving the body’s immune system’s ability to recognize and attack cancer cells. Scientists have spent decades exploring why the immune system can sometimes successfully fight cancer, but more often it cannot recognize cancer cells and cannot effectively fight cancer cells.

Due to key discoveries in the 1990s, scientists have a certain understanding of how cancer cells use naturally occurring molecules to shut down the immune system’s response to tumors. The exploration of using drugs to restart the immune response has been rapidly advanced and has become a frontier area of ​​cancer research and treatment, although this type of treatment is only effective for a small number of patients.

In the past few years, immune checkpoint inhibitor drugs have been widely used, which has significantly improved the treatment of melanoma, lung cancer and other types of cancer. At present, the U.S. FDA has approved 6 PD-1/L1 inhibitors, including: PD-1 inhibitors Nivolumab (Opdivo), Pembrolizumab (Keytruda), Cemiplimab (Libtayo), PD-L1 Inhibitors Atezizumab (Tecentriq), Duravulumab (Imfinzi), Avelumab (Bavencio) are used to treat more than 10 different types of cancer, and their efficacy is being evaluated in more than 2,250 clinical trials. In addition, a CTLA-4 inhibitor ipilimumab (Yervoy) has been approved.

In addition, CAR-T cell therapy is also an inevitable “net celebrity” immunotherapy. It is an immunotherapy that uses genetically modified T cells, which can be understood as the use of gene therapy for immunotherapy. The patient’s own T cells have been genetically modified in the laboratory to form a protein called chimeric antigen receptor (CAR), which is equivalent to “scud missiles” for T cells. When this modified T cell is returned to the patient, the CAR-T cell can target and destroy the cancer cells in the patient.

Currently, two CAR-T therapies Kymriah (Tisagenlecleucel) and Yescarta (Axicabtagene Ciloleucel) have been approved by the US FDA for the treatment of B-cell lymphoma and acute lymphoblastic leukemia. In addition, research on CAR-T therapy in the treatment of solid tumors is also underway.

However, according to the FDA, CAR-T therapy is included in the category of gene therapy. Former FDA Commissioner Scott Gottlieb once commented: “We are entering a new field of medical innovation-reprogramming patients’ own cells to attack deadly cancer.”

(source:internet, reference only)

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