What is gene therapy?
Gene therapy is the therapeutic delivery of nucleic acid(piece of DNA) into patient's cells to correct the effects of a disease-causing mutation. In fact it's a technique, where researchers replace a mutated gene that causes disease with a healthy copy of the gene or inactivate a mutated gene. Nowadays, it is being tested only in some patients who are in the last 'stage' of their life, but is a promising future treatment.
A new treatment against B-Acute Lymphoblastic Leukemia (B-ALL) has just been aproved by U.S. Food and Drug Administration(FDA).
B-ALL is a cancer of the Bone Marrow, in which the body makes the B-cells(a type of lymphocytes) abnormal. B cells are staying immature and they multiple uncontrolable. Also B-ALL is the most common type of cancer in children!
The treatment involves a drug called 'TISAGENLECLEUCEL' or 'KYMRIAH'. The drug is costumized according to the patient's own T-cells (T- cells is another type of lymphocytes). The patient's T-cells are collected and sent to a special manufacturing center, under the appropriate conditions, where they are genetically modified to include a new gene, that codes for a protein called achimeric antigen receptor (CAR). The new gene directs the modified T-cells to target and kill leukemia cells. The modified T-cells are then infused back into the patient to kill cancer cells.
Image 5
In this picture you can see the genetically modified T-cells (in blue), attacking cancer cells(in red).
That treatment destroyed eighty-three per cent of leukemic cells in three months.
Although China has approved a gene therapy drug and two gene therapy treatments using in Europe, but this is the first to win approval from FDA.
This therapy has shown extraordinary results in patients, but the most difficult part is that the company has to be able to manufacture personalized therapies quickly enough!
Those kind of therapies is what the people needs to survive and have a great and healthy life! We are waiting more than that, we need more research but the first step was done! Congratulations!!!
Thank you for reading and I am waiting for your comments!
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Sources:
- https://www.cancer.org/cancer/acute-lymphocytic-leukemia/about/what-is-all.html
- https://www.sciencedirect.com/science/article/pii/S0168365903004462
- https://ghr.nlm.nih.gov/primer/therapy/genetherapy
- http://www.sciencemag.org/news/2017/08/modified-t-cells-attack-leukemia-become-first-gene-therapy-approved-united-states
- https://www.npr.org/sections/health-shots/2017/08/30/547293551/fda-approves-first-gene-therapy-treatment-for-cancer