New cancer treatment credited for saving the life of 9-year-old boy

"GMA" got an exclusive first look at a cutting-edge new cancer treatment that some medical professionals say will change the way we deal with the disease.
5:23 | 11/27/18

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Transcript for New cancer treatment credited for saving the life of 9-year-old boy
Now that "Gma" exclusive, the fda has just approved a breakthrough cancer drug that could help thousands of people every year. It's already giving hope to a family who thought they were out of options for their 9-year-old son, and erielle reshef is here with this encouraging story. Reporter: Very encouraging. That cutting-edge technology has only been administered in trials but it was just approved by the fda within the last 24 hours and experts say it could save more lives like Ashton's as soon as it hits the market. Looking at energetic, athletic 9-year-old ash Lon, you'd never know just five years ago his chances of surviving cancer were bleak. When Ashton was 4, I noticed a lump on his neck. We took him in to see his doctor. He was sent for a biopsy where they found out that he had stage 4 thyroid cancer. Reporter: His diagnosis, dire and devastating to his family. Slowly we noticed his breathing change and he kind of lost some weight and so we went back to see his doctor and they did another chest x-ray and it showed that the cancer had spread in his lungs. Reporter: He would undergo surgeries and treatment. But that didn't stop the disease from progressing. The worst moment was seeing him after he had his surgery and hooked up to all the machines. Reporter: Doctors said there was nothing more they could do. They did some testing and found out that the cancer had become resistant. We didn't know, you know, how long he had after that. Reporter: Until a phone call last spring about a trial for a cutting-edge new cancer drug changed everything. The medication will be sold as vitrakvi or larotrectinib just approved by the fda brought to the market by Bayer and targets a genetic mutation found in certain cancers inhibiting the protein responsible for cancer growth. Larotrectinib actually specifically targets a change in the DNA of the tumor cells for these specific cancer types. Reporter: The Leeds driving 700 miles every month from their home in Alberta, Canada, for Seattle children's for the treatment. The results, stark. This is his most recent scan, and basically almost everything is totally gone, but as you can see the difference is night and day. When you find a patient that has the mutation that this drug targets, it works almost every time. Reporter: It could provide new hope for thousands like Ashton. This medication has been the best thing that's happened to us and really does give us hope for Ashton's future and everything is going to be okay. It's all looking up for Ashton. While he is not considered completely cure his prognosis is very promising and will continue taking this drug as long as doctors are monitoring his progress but his family says his response to treatment certainly is a good reason to be hopeful. Great to see his smile and that of his family. Erielle, thank you. Dr. Jen Ashton will talk about this. How exactly does this work? Let me show you because it soups confusing. Think of it this way. Think of this mug as our DNA. We have a gene that codes for the handle and the lip, the side, the bottom and so forth. This mug now, there's been a clean break in the gene, okay, and when the gene heals itself which DNA does, it comes on in a completely wrong way, wrong place. The drug works here to block this defect. This is what causes the cancer. It's called a track fusion inhibitor, this class of drugs. It's not for everyone. This drug is for kids and adults with locally advanced cancer that has not responded to other treatment so they're kind of out of options. We heard erielle say, unfortunately, only about 1% of all cancers have this particular genetic mutation, but for that 1% this is a big deal. This is precision medicine targeted therapy. Maybe we can learn from this. Exactly. What are the caveats here? Always caveats. This big trial which is called the navigay trial funded by Bayer, the cost is to be determined. Normally a high cost and remains to be seen whether insurance will cover it and side effects. Always side effects, fatigue, nausea, it can bump up your liver enzymes but generally these are pretty mild. It makes me think of memorial sloan-kettering. How do people find these trials? You know, memorial sloan-kettering has a statistic that only 35% of Americans would enroll in a clinical trial largely because of fear. They don't have the right information. There are a lot of myths. I want everyone listening to this regardless of what kind of cancer you have, you can go to Every clinical trial in the world must be registered there. You can put in the type of cancer you have, where you live, for this particular drug, you can enter it in and find out where this drug is being tested in the country. But this is the backbone. If you want involved in a trial, educate yourself. It can make all the difference. Bless you for sharing that information. Thanks so much. Certainly encouraging news. On a side note, we just got

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