Atypical teratoid/rhabdoid tumor (AT/RT) is a rare and lethal childhood cancer. Dana-Farber has made important discoveries that are creating pathways to promising new therapies for this disease. Learn more about our discoveries at .
AT/RT is this rare form of this highly malignant tumor. There were, in fact, five known survivors of AT/RT in the entire world literature, but we noted that of the five of them, three of them had been treated the same way using a therapy that was completely different from anything that we were doing in neuro-oncology. It’s not a brain tumor treatment at all.
I had had a couple of patients that I had treated with AT/RT. They relapsed incredibly quickly. Two new kids with newly diagnosed AT/RT came in, and we asked the simple question: if we applied that therapy to the two new kids and to the two relapsed kids because they hadn’t gotten anything like it, what would happen?
We treated those four kids and three of them actually ended up doing fantastically well. Basically we had almost doubled the number of known survivors of this disease. So, we decided to start a national trial, and centers from all around the United States said, “We think this is a great idea, and we’re willing to commit to getting the full therapy—not a modified version of it,” and over half of the kids ended up being long-term survivors.
Suddenly we took an incurable disease and cured half the kids right off the bat. Remember: there’s no new experimental drug here. This was just using a different approach of chemotherapy and applying it in the circumstance that we never considered before. It turns out that many adult tumors use the same abnormal pathways to allow the adult tumors in breast, in prostrate, in lung, and in colon to allow cells to get out of control. That’s critical, because it means if we can understand how to treat these kids, we may have inadvertently also developed much more effective therapy for the big adult cancers that kind of ravage a large part of the population in the United States.
It’s an exciting place to be, because we can actually talk to those families the day they’re diagnosed and say, “Actually, we have something to offer you that may really help.”
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