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Gene Therapy. A New Frontier in Medical Innovation

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Cancer is a disease that is experienced by young and old and by every economic and social segment of the world populace. At some point in everyone’s life, cancer will affect us individually, a member of our family or one or many of our friends and acquaintances. The American Cancer Society projects there will be 1,688,780 new cancer patients and 600,920 deaths due to cancer in 2017, for a disease that does not discriminate by race or national origin.

Cancer is where abnormal cell growths form in the body and interfere with normal, healthy body functions. Not new to the world stage, cancer dates back to ancient times. Today, after millennials of research and discovery, new treatments for a vast array of cancer types are being studied, many with promising results.  One such new treatment called chimeric antigen receptor T-cell therapy (CAR-T) involves taking samples of T-immune cells called T-cells from a patient, genetically engineering them, and putting them back in to fight the cancer. Co-developed with the drug giant Novartis, the therapy, CAR-T, genetically alters a patient’s own immune cells to target and destroy cancer cells.

“We’re entering a new frontier in medical innovation with the ability to reprogram a patient’s own cells to attack a deadly cancer,” said FDA Commissioner Dr. Scott Gottlieb. The process takes about 22 days and begins with the extraction of a patients T cells, exposing them to the vector which genetically transforms them. They’re then expanded and frozen for shipment back to the patient. During the out-of-body processing of the cells, the patient receives chemotherapy to wipe out any remaining T cells to avoid interference with the, soon to be, newly implanted cells.

“This is a major advance, and is ushering in a new era,” said Malcolm Smith, a pediatric oncologist at the National Institutes of Health. The treatment appears to strengthen a patient’s immune system allowing their own body to fight the cancer. The treatment is recommended for cancer patients who have run out of options for traditional therapy. The new therapy is only currently available for patients 25 years or younger. Thus far, 83 percent of patients are experiencing positive survival results.

While overtly touting the success of gene therapy, researchers are tempering their enthusiasm after experiencing some significant life threatening side effects in 47 percent of the studies participants. These side effects have resulted in brain swelling and deaths, casting a shadow over the field. Seizures and hallucinations were also relatively common, but temporary. Though concerning, the side effects have been successfully managed with drugs. Quality control and how to standardize the potency and purity of living cells extracted from each patient are also a concern. Because of the risks, the treatment is only currently available at 20 US hospitals. The individualized nature and relatively small patient population both drive up the cost of the treatment for now.

“This is a big paradigm shift, using this living drug,” says Dr. Kevin Curran, a pediatric oncologist at Memorial Sloan Kettering Cancer Center that will soon be offering the treatment. “It will provide a lot of hope. This is the beginning.”

“The approval of CAR T-cell therapy for pediatric leukemia marks an important shift in the blood cancer treatment paradigm,” says the American Society of Hematology. “We now have proof that it is possible to eradicate cancer by harnessing the power of a patient’s own immune system. This is a potentially curative therapy in patients whose leukemia is unresponsive to other treatments and represents the latest milestone in the shift away from chemotherapy toward precision medicine. Today’s approval is the result of over a decade of hematology research, including research funded by the National Institutions of Health (NIH).”