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Child cancer. Treatment success. Precision medicine therapy. Clinical case. 10
Precision medicine means we have to select targeted cancer treatment based on unique tumor mutations. Treatment is not based on only tumor’s tissue or organ origin. Top pediatric cancer expert shares remarkable patient’s story. It illustrates how tumor genomic sequencing can change the course of cancer treatment and result in cure. Professor Izraeli, is there a clinical case about pediatric cancer patient that you could discuss? Dr. Anton Titov, MD. A situation that illustrates some of the topics that we discussed about pediatric cancers, and, in particular, child leukemias? Dr. Shai Izraeli, MD. Maybe I’ll give you an example that is not from leukemia case. But I’m very moved by this child cancer treatment example. We were very excited by this clinical case. It just tells you the general importance of precision medicine across different types of tumors. We do precision medicine pediatric cancer therapy here in our hospital. We are not a leukemia department only. We treat all types of cancers. Dr. Shai Izraeli, MD. We had a child with a congenital tumor. He was born with a tumor. But he was diagnosed only four months later. Then he had a tumor almost like a small football attached to his chest. The type of tumor is not so important. But his cancer was not operable. We treated this tumor with chemotherapy. But it grew and grew despite chemotherapy. Then we decided to do a genomic testing of the tumor. In the cancer genome we found a genetic abnormality in a gene called TRK, “Trek”. I was very excited when I saw this genetic abnormality in this child’s cancer. I was very excited because a couple of months before I saw a clinical report from another hospital on an extremely rare case of leukemia. It had exactly the same TRK genetic abnormality. At that time there was no cancer medication available for this abnormality. But oncologists transplanted the leukemia into mice. Dr. Anton Titov, MD. Then they treated the mice with a TRK inhibitor. The mice were cured from leukemia. Then, as you know, I’m an important doctor, right? What are important doctors doing? They go to doctor Google, right? Dr. Shai Izraeli, MD. I went to Dr. Google. This TRK cancer inhibitor was already in clinical trials. Now we returned to this child. He had a very big tumor attached to his ribs. We treated him with this new TRK inhibitor [larotrectinib] in a clinical trial in Heidelberg in Germany. Now this clinical trial is opened here. The tumor disappeared completely! TRK inhibitor cancer medication is given as a syrup, by mouth, orally. Already within two weeks 90% of cancer tumor was gone. Now, couple of months later, the tumor almost disappeared. The rest of cancer we can remove by surgical operation. Dr. Shai Izraeli, MD. Now, why do I decide to give you an example of a solid tumor? You asked me to give a clinical vignette of leukemia? Dr. Anton Titov, MD. You already understand it. Because I knew about this TRK inhibitor from leukemia. But this already is the hallmark of precision medicine cancer therapy today. Because these TRK genetic mutations in the tumor are rare. These abnormalities are not common. But they are not unique to the type of tumor that I described. They are not unique to leukemia, where they are extremely rare. These tumor mutations are also found in brain gliomas. TRK mutations exist in kidney cancer and other cancers. These cancer genetic abnormalities happen in a small percentage of tumors. But this example tells you why it’s really important today to test every child with cancer for general genetic abnormalities. Because we may find a genetic abnormality that can treated by a specific targeted cancer medication. Then we should use a targeted cancer medication and not something else. Dr. Shai Izraeli, MD. That is what we are going to do in the upcoming year. That’s an example of modern precision medicine. Dr. Anton Titov, MD. Classification of tumors is not done only by the type of tissue that the tumor comes from. Examples are leukemia, soft tissue tumors, carcinomas, brain tumors. Today tumors are classified based on a genetic abnormality that causes cancer. That’s one example. Another example of targeted cancer therapy is this. We had a child with acute myeloid leukemia. Leukemia didn’t respond to any treatment. Dr. Shai Izraeli, MD. Then we found out that there is activation of a signaling molecule called FLT3 in AML. This child also entered remission of leukemia after the treatment with high dose FLT3 inhibitor [Quizartinib]. He is cured now. I believe we are living in a very exciting era of precision medicine. We now have some magic bullets. Dr. Anton Titov, MD. These are targeted precise cancer medications. There is not only magic. There are also many problems with cancer treatment side effects. It is very important not to make excessive promises. But I think the era where every child with cancer will be cured is certainly in the lifetime of a doctor who just now starts working in medicine.
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