Could this replace typical cancer treatment?
The U.S. Food and Drug Administration recently made huge waves in the medical world by announcing it had approved its first gene-altering therapy for childhood leukemia, a move that could usher in a new age of medicine – so long as people can get access to it.
The agency’s approval of chimeric antigen receptor T-cell, or CAR T-cell, therapy allows for the use of the drug Kymriah for children and young adults whose leukemia doesn’t respond to traditional approaches. The drug utilizes genome editing to use the body’s own immune system to fight cancer.
“Genome editing” is a general term used to describe a set of technologies enabling the precise changing in the DNA of a plant, animal or other living organism, according to the FDA’s website.
Kymriah is a customized treatment created using an individual patient’s own T-cells, a type of white blood cell, according to the FDA. The patient’s T-cells are collected and sent to a manufacturing center where they are genetically modified to include a new gene that contains a specific protein directing the T-cells to target and kill leukemia cells. Once the cells are modified, they are infused back into the patient to kill the cancer cells.
Dr. Fari Barhamand, an oncologist and hematologist at Advocate Good Samaritan Hospital in Downers Grove, Ill, calls gene therapy a potential game-changer that could even replace chemotherapy as the treatment of choice for most cancers – particularly if the estimated $475,000 for Kymriah does not become the industry-standard.
“We do not have any long-term data for the very expensive treatment, but if you add other diseases like chronic lymphocytic leukemia, multiple myeloma, glioblastoma or common cancers like breast cancer, the number of people who could be helped in prolonging their lives when standard treatments fail is mind-boggling,” he says.
The drug does have the potential for severe side effects, including flu-like symptoms, neurological events, hypotension and others that can be life-threatening. Still, Dr. Barhamand was enthusiastic about the treatment’s potential and the potential of other drugs like it.
“I do believe that gene therapy will play a very important role in what we do daily, and could eventually be used in a community setting where most patients are,” he says. “The sheer numbers of future patients could bring the cost down dramatically. Whether it happens in a few years or takes a decade is anybody’s guess, but I hope it happens in my lifetime so I could be a recipient of the treatment one day, if necessary.”
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About the Author
Nathan Lurz, health enews contributor, is a public affairs coordinator at Advocate Good Samaritan Hospital. He has nearly a decade of professional news experience as a reporter and editor, and a lifetime of experience as an enthusiastic learner. On the side, he enjoys writing even more, tabletop games, reading, running and explaining that his dog is actually the cutest dog, not yours, sorry.
Off-label use of cancer drugs won’t be covered by your insurance provider. The manufacturer may have a program where you can receive the drug at no cost.
My late wife was an example. Diagnosed with a Grade IV glioblastoma in March of 2016, the standard of care drug, Avastin, was beginning to fail after two months of use.
It was then we were informed of the Opdivo off-label use. Humana wouldn’t cover it, but BMS had a program where my wife could receive it for free.
She received her initial dose in November, and couldn’t stand the side effects. Her condition worsened in the following week, requiring hospitalization.
It was there she decided she had enough, and would enter into hospice care.
She was discharged into hospice at home, and would pass away within a month.
I guess when dealing with a cancer that has a 90% mortality rate in five years, this was something to expect.
In the end, sometimes cancer has the last word.
I would like to see references for every article posted so I could read further
Half a million dollars? Really? If I had the ability to save someone’s life I’d do it for free. Especially a child with cancer. Demanding a half million from a dying child sort of puts you on par with Satan and Hitler. Good work on the advancement though. Lol
Tom,
According to the article I’m linking; there flexible pricing options for Kymriah from Novartis.
https://www.bloomberg.com/news/articles/2017-08-31/novartis-kymriah-cancer-drug-475-000-but-read-the-fine-print