FDA Approves First Cell-Based Therapy For Cancer (npr.org)
An anonymous reader quotes a report from NPR: The Food and Drug Administration on Wednesday announced what the agency calls a "historic action" -- the first approval of a cell-based gene therapy in the United States. The FDA approved Kymriah, which scientists refer to as a "living drug" because it involves using genetically modified immune cells from patients to attack their cancer. The drug was approved to treat children and young adults suffering from acute lymphoblastic leukemia, a cancer of blood and bone marrow that is the most common childhood cancer in the United States. About 3,100 patients who are 20 and younger are diagnosed with ALL each year. The treatment involves removing immune system cells known as T cells from each patient and genetically modifying the cells in the laboratory to attack and kill leukemia cells. The genetically modified cells are then infused back into patients. It's also known as CAR-T cell therapy.
The treatment, which is also called CTL109, produced remission within three months in 83 percent of 63 pediatric and young adult patients. The patients had failed to respond to standard treatments or had suffered relapses. Based on those results, an FDA advisory panel recommended the approval in July. The treatment does carry risks, however, including a dangerous overreaction by the immune system known as cytokine-release syndrome. As a result, the FDA is requiring strong warnings.
The treatment, which is also called CTL109, produced remission within three months in 83 percent of 63 pediatric and young adult patients. The patients had failed to respond to standard treatments or had suffered relapses. Based on those results, an FDA advisory panel recommended the approval in July. The treatment does carry risks, however, including a dangerous overreaction by the immune system known as cytokine-release syndrome. As a result, the FDA is requiring strong warnings.
My mother is still alive today thanks to this treatment, and no thanks to you.
Il n'y a pas de Planet B.
The thing is :
- these classes of methods are generic. With very few problems (it's not relying on killing the patient slower than killing the cancer. It's about specifically targetting the cancer) Eventually this method could be adapted to other cancers as well.
so the fact that they used it against leukemia isn't a major drawback for brain cancer.
(Unlike chemotherapies which rely a lot on the general charecteristics of the cancer, to find a way to poison it with a drug faster than the drug posions the rest of the patient. Different type of cancer = different type of characteristics. Poisons will therefore work differently)
- because leukemia is well studied and has already lots of studies done with other treatment, that gives a lot data point to compare against, and to combine with.
- leukemia happens to be a slightly lower hanging fruit here. (everything happens in the blood stream, which is where you'd be injecting the modified cells).
(but again, all fruits *on the same tree*. Not an entirely different tree like chemotherapies).
These are reasons to take it as a first target, before expanding to other cancers.
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