CAR-T Cell Therapy: A Potential Game Changer For Leukemia Treatment

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Car-T Cell Trials

A clinical trial of Car-T therapy on children and adolescent patients with acute lymphoblastic leukemia yielded some very promising results. This trial, led by Dr. Stephan Grupp, Ph.D., M.D., was carried out at the Children’s Hospital of Philadelphia. 27 out of 30 patients who were given the treatment experienced full remission with no indications of recurrence after treatment ended.

More trials are scheduled to be conducted at the University of Chicago C.S. Mott Children’s Hospital to determine if Car-T cell therapy can indeed effectively treat cancer in children with acute lymphoblastic leukemia.

This form of cancer is the most common form of leukemia among children in the United States, accounting for around 25 percent of cancer cases in children under 15 years of age. In this trial, the extracted T-cells were genetically modified to make them attack a type of protein called CD19, which is found on the surface of acute lymphoblastic leukemia cancer cells.

About 80 percent of children diagnosed with acute lymphoblastic leukemia are given conventional treatments, and end up recovering as a result. But some of those who do not respond to traditional treatments make ideal candidates for this trial.

However, Car-T cell therapy is not without risks. Some of the side effects of these treatments have proven to be quite serious, even lethal. One of the most serious is cytokine release syndrome, or CRS.

Cytokines are chemical messengers which are released by cells to give instructions to white blood cells and enact the immune response when a threat is detected. When there is a huge release of cytokines, it can cause a significant drop in blood pressure and severe fever. It can also cause inflammation which must be treated by drugs intended for conditions like juvenile arthritis.

Another serious side effect is the risk of swelling of the brain, a potentially life-threatening condition called cerebral edema. This was quite rare in trials conducted thus far, but did result in a few deaths among patients participating in one trial.

Finally, there is also the risk that the Car-T cell therapy might work too well. As mentioned above, it attacks a protein called CD19. This protein is also found on the surface of B cells which actually help the immune system by producing antibodies. The genetically modified Car-T cells attack and kill these B cells while the patient is undergoing therapy. It’s somewhat ironic that using bioengineering to boost the immune system can actually make it attack itself in this way. However, this problem can be overcome with immunoglobulin therapy.

 

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More research needs to be done, but the majority of results for Car-T cell therapy testing has been quite positive and it is currently on the way to FDA approval. Stay tuned on how this amazing new medical technology develops and continues to offer new hope to patients which have not had success with conventional treatments.

References:

www.uofmhealth.org

www.ncbi.nlm.nih.gov

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