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FDA Approves Unique, DNA-Targeting Cancer Drug, Potentially Changing Care for Thousands

By November 29, 2018No Comments

Today, Connor Pearcy is an average 7-year-old boy. He loves dinosaurs, Pokemon and tetherball — so much so that the Pearcy family home has a tetherball setup in its backyard.

But until about two years ago, Connor also had something most kids don’t: A tumor just below his left knee that resisted chemotherapy and surgery and forced Connor’s family to consider amputating his leg.

Connor’s tumor was wiped out by a drug called larotrectinib, a unique new medicine that on Monday was approved by the FDA to treat a huge variety of soft tissue tumors.

Larotrectinib was developed by Connecticut-based Loxo Oncology and has been tested at sites around the U.S. for several years. It is approved to treat both children and adults whose cancer expresses a specific genetic change that occurs in an estimated 2,500 people each year.

It has the potential to wipe out stubborn cancers with few to no negative side effects, making it a unique entrant into the cancer treatment world.

Seattle Children’s Hospital has operated the largest arm of the drug’s pediatric trial, enrolling more patients than any other site, including Connor.

“Just giving larotrectinib alone, we can physically see with our own eyes — and then also confirm with doing diagnostic imaging— that tumors are really melting away,” said Dr. Katie Albert. Albert is a pediatric physician and researcher who has overseen Seattle Children’s arm of the larotrectinib trial. She said the drug could have big ramifications for children like Connor.

“We really are pretty dramatically, potentially, changing the standard of care for these patients,” Albert said.