For decades, a deadly type of childhood cancer has eluded the best tools of science. Now doctors have come up with an unusual treatment: dripping millions of copies of a virus directly into children’s brains to infect their tumors and stimulate an immune system attack.
A dozen children treated this way lived more than twice as long as similar patients in the past, doctors reported Saturday at a conference of the American Association for Cancer Research and in the New England Journal of Medicine.
Although most of them eventually died from their illness, some are alive and well several years after treatment, something practically unheard of in this situation.
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“This is the first step, a critical step,” said study leader Dr. Gregory Friedman, a childhood cancer specialist at the University of Alabama at Birmingham.
“Our goal is to improve this,” possibly testing it when patients are first diagnosed or combining it with other therapies to boost the immune system, he said. Study patients were given the experimental approach after other treatments failed.
The study involved gliomas, which account for 8% to 10% of childhood brain tumors. They are usually treated with surgery, chemotherapy, or radiation, but they often come back. Once they do, survival averages just under six months.
In such cases, the immune system has lost the ability to recognize and attack the cancer, so scientists have been looking for ways to turn the tumor into a new target. They targeted the herpes virus, which causes cold sores and stimulates a strong immune system response. A suburban Philadelphia company called Treovir developed a treatment by genetically modifying the virus to infect only cancer cells.
Through small tubes inserted into the tumors, doctors administered the altered virus to 12 patients ages 7 to 18 whose cancer had worsened after standard treatments. Half also received a dose of radiation, which is believed to help the virus spread.
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Eleven showed evidence on imaging tests or tissue samples that the treatment was working. Median survival was just over a year, more than double what has been seen in the past. Last June, the limit to analyze these results, four were still alive at least 18 months after treatment.
The tests also showed high levels of specialized immune system cells in their tumors, suggesting that the treatment had recruited the body’s necessary help to attack the disease.
No serious safety concerns were noted, although there were several complications related to the procedure and mild side effects, including nausea, vomiting, diarrhea, and fatigue.
Jake Kestler received the treatment when he was 12 years old.
“Everything went very well. He lived for a year and four months after that,” long enough to celebrate his bar mitzvah, go with his family to Hawaii and see a brother born, “said his father, Josh Kestler, an executive. Financial Services Department of Livingston, NJ.
Jake died on April 11, 2019, but “we have no regrets” about trying the treatment, said Kestler, who with his wife started a foundation, Trail Blazers for Kids, to do more research.
“It is a devastating disease for these patients and their families,” and the first results suggest that the treatment of the virus is helping, but must be verified in a larger study, which doctors are planning, said Dr. Antoni Ribas, specialist in cancer from the University of California, Los Angeles, and chair of the group that held the conference.
Friedman said studies are continuing in adults as well, and plans are in the works for other types of childhood brain tumors. Grants from the US government and several foundations paid for the study, and several doctors have financial ties to Treovir.
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Currently, only one similar virus therapy is approved in the United States: Imlygic, also a modified herpes virus, to treat melanoma, the most serious type of skin cancer.