Seattle Children's trial pinpoints precision treatment for cancer

An experimental drug to treat a rare form of cancer is on its way to the FDA for approval. The trial included a patient at Seattle Children's, who is in full remission after years of unsuccessful traditional treatment.

Amy Pearcy remembers how she felt when doctors asked to put her son on an experimental drug.

Pearcy's son Connor has been battling Soft Tissue Sarcoma, a rare form of cancer, his entire life.

“So, clearly, it's a roller coaster emotional reaction, right? Like or we might have something here that would work, but you're also facing the unknown,” said Amy.

Dr. Doug Hawkins of Seattle Children's says Connor’s tumor is caused by a series of defective genes, which create a malfunctioning protein that causes tumors to grow.

“He was born with a tumor that took up most of the lower part of his right leg. When you look at his original imaging of the tumor, it kind of filled most of the lower compartment of his leg,” explained Dr. Hawkins, associate division chief of hematology/oncology at Seattle Children’s.

Initially, doctors tried to surgically remove the tumor - a very tricky process because Connor's body began to grow around the mass and because there were blood vessels growing through the tumor.

After two unsuccessful surgeries and little progress with chemotherapy, Connors family worried the only option they left was amputation.

That’s when Dr. Hawkins found a clinical trial that involved genetic testing to identify the abnormal genes in Connor's body.

“Precision medicine. The idea is that instead of treating everyone the same, if they have this type of cancer, we give them the same treatment that you use, features that you know about that person or that particular cancer - the direct the treatment to something that’s individualized. It’s unique to that person's cancer,” says Dr. Hawkins.

The University of Washington identified the abnormal cells, and Dr. Hawkins immediately asked to put Connor on the trial drug called Larotrectinib.

“You don't see oncologist really excited. He came in and had like, you know, his face was all like, happy and he was all excited,” said Amy.

“We dream of drugs that work like this. We dream of having something so specific, but doesn't cause terrible side effects,” said Dr. Hawkins.

Connor didn't experience any side effects, and in just two months of treatment, his tumor had reduced in size by 80 percent.

Now, his mother says the tumor is undetectable by MRI

“It's just kind of an amazing thing to be working at for five years and then all the sudden you get this drug that says, hey, it's gone four months,” explained Amy.

As of right now, Connor will stay on the drug as long as he doesn't experience any side effects.

The company that makes Larotrectinib plans to take the results of the study to the FDA this fall and hopes for approval in 2018.

If you’d like to learn more about Connor’s story go to Seattle Children’s, On The Pulse, where you can find out about their medical milestones. 

© 2017 KING-TV


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