“Is my child going to die?”
I don’t know if I have the words to describe what it feels like to have someone look directly in my eyes and ask that question. Their whole world is hanging on to how my lips start to move to form the next words. Will they move to say “Yes” or will it be a “No”?
If the year was 1960, my answer would be an overwhelming “Yes”. In fact, it hadn’t been too long since Dr. Sidney Farber began treating childhood leukemia patients, with ‘anti-folates’ which you can read about here. After World War II, naval personnel returning home who were exposed to mustard gas were found to have bone marrow damage; this was an important observation because the bone marrow is where leukemia starts. Meanwhile, the Army was trying to form stronger compounds for warfare and developed nitrogen mustard. Nitrogen mustard turned out to be quite effective in treating lymphoma. These nearly simultaneous serendipitous discoveries swiftly ushered in the “chemotherapy era” of the 1970s.
From 1970 to the 1990s, the mortality from childhood cancer decreased dramatically across the majority of the tumor types. The real success story of the chemotherapy era is childhood leukemia. A disease that was previously untreatable, now had many survivors – a true medical success story! We also learned that chemotherapy was very important for solid tumors that had been removed with surgery and we saw improvements in those outcomes too!
From the 1990s to the early 2000s, mortality continued to decrease but at a much slower pace. This decrease was largely related to improvements in supportive care. More robust supportive care measures reduced the mortality from infections and other toxic effects of chemotherapy, but unfortunately we had no new chemotherapy drugs come along. It is now 2018 and we have essentially maxed out the amount conventional chemotherapy that an individual can receive and sometimes it is still not enough to cure them. We need new drugs.
In the graph above, you can easily see the plateaus starting around 2000 for many of the cancers. It’s now 2018 and we are well into the era of immunotherapy and precision medicine. I’m sure you have heard of it in the press with headlines like “Harnessing the power of your immune system” and “Personalized targeted therapy”. It’s happening, it’s here – but like all new drugs and therapies, our kids are the the last to be able to try these drugs. The first version of this blog was written in 2016 and since then, we have opened the NCI-COG pediatric MATCH trial, which is the first cooperative “precision medicine” trial for children. This is a phenomenal step forward. What will come of this groundbreaking trial remains to be seen, but I am happy to report it is happening. I will talk more about what the trial entails in another post.
We’ve already proven we made a difference with the drugs we have available, but it’s not enough. We need more and we can do better. With dedicated funding and brilliant scientists, we can figure this out. They used to say it was impossible to save any of them, now they say it’s impossible to save them all, but I, along with all of my colleagues will die trying because there is absolutely nothing worse than telling a parent “Yes”.
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