The medical world is abuzz this month after the FDA unanimously approved a new treatment for leukemia. And this treatment isn’t another round of harsh chemicals or radiation-instead, it uses “gene therapy” to train the body’s white blood cells to attack cancer cells. It’s the beginning of what many are calling the “living drug” era, and leukemia is just the first disease in the crosshairs.
“It’s a pretty amazing new treatment,” explained Dr. David Agus (director of the USC Norris Westside Cancer Center) during an interview on CBS This Morning. “They take the white [blood] cells out of a child with cancer, they send them to [a lab in] New Jersey, and they put in a gene to reprogram these cells to attack the cancer.”
The gene therapy approach also differs from radiation and chemotherapy in that it is typically only a one time treatment, and the numbers so far are impressive. Of the 88 cancer patients given the treatment during clinical trials, 83% had complete remission of the cancer. The side effects, unfortunately, are still considered severe-something the gene therapy has in common with older treatments. One of the worst side effects seen is a condition called “cytokine release syndrome”, where the body attacks its own vital organs, a condition that can be life-threatening. But even with the side effect risks, the results are better than doctors have seen with chemotherapy and even newer cancer drugs.
“Our daughter [Emily, age 12] was going to die and now she leads a normal life,” said Tom Whitehead, of Philipsburg, Pennsylvania, father to one of the young patients first given the gene therapy treatment. “We believe that when this treatment is approved it will save thousands of children’s lives around the world. I hope that someday all of you on the advisory committee can tell your families for generations that you were part of the process that ended the use of toxic treatments like chemotherapy and radiation as standard treatment, and turned blood cancers into a treatable disease that even after relapse most people survive.”
Adding to the excitement of the breakthrough is the realization that gene therapy will eventually be able to treat diseases other than leukemia.
“It’s a new world, an exciting therapy,” said Dr. Gwen Nichols, the chief medical officer of the Leukemia and Lymphoma Society. The next step, she said, will be to determine “what we can combine it with and is there a way to use it in the future to treat patients with less disease, so that the immune system is in better shape and really able to fight. This is the beginning of something big.”
The technology is still young, and there are still many challenges to overcome and many questions left to answer, but the “living drug” breakthrough and approval mean big things ahead-both for patients and for those treating them.
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