UCLA pediatric oncologist Dr. Noah Federman, director of the UCLA Jonsson Comprehensive Cancer Center’s Pediatric Bone and Soft Tissue Sarcoma Program and medical director of the UCLA CTSI’s Clinical and Translational Research Center, is available to discuss the newly FDA-approved targeted cancer therapy, larotrectinib.

Larotrectinib is one in a class of drugs known as tyrosine kinase inhibitors (TKIs) that block the action of target genes. Specifically, the drug targets a pan-tropomyosin receptor kinase (TRK) and is designed to work in a variety of different cancer types that have this TRK fusion.

Federman was one of the clinical trial’s investigators who reported promising results of the drug in the New England Journal of Medicine earlier this year and recently presented the updated integrated sarcoma data set at the Connective Tissue Oncology Society Meeting in Rome, Italy. In the latest update, over 120 patients suffering from different cancers – including common cancers such as lung and colon cancer and rare cancers such as sarcoma – had overall response rates of 81% one year after completion of the trial. Specifically, in bone and soft tissue sarcomas, the response was unprecedented with over 93% of patients having rapid and durable responses, and many other patients having complete responses.

Quotes from Federman on larotrectinib:

“The discovery of this investigational medication that targets the TRK fusion in cancers, both pediatric and adult, is one of the seminal discoveries in oncology in the last decade. We are thrilled to be able to pioneer this groundbreaking research here at the UCLA Jonsson Comprehensive Cancer Center.”

“We are seeing unprecedented responses in advanced-stage cancer patients. Moreover, the robust efficacy of larotrectinib has been successful regardless of cancer type and age. I never thought I would see these results – each time I see patients’ tumors disappearing in front of my eyes, I am in awe.”

“I can say confidently now that we have changed the paradigm in cancer treatment. Soon, we will eradicate the need for the aggressive surgeries and harmful toxins that can put patients at risk in the short term and long term.”

“UCLA has been at the forefront of moving this innovative drug therapy forward. This medication istargeted only to this gene fusion and nothing else. We have seen remarkable responses, as it has been relatively nontoxic for patients and they have experienced very few adverse effects. We are excited to have spearheaded what we consider to be one of the seminal discoveries in the last decade in pediatric oncology.”

“It is amazing that our science has come this far. Therapies such as this one are truly changing how we approach cancer. It is remarkable that I can tell a family that their loved one no longer needs chemotherapy or aggressive surgery to combat these malignancies. Methods for the standard treatment of care are becoming part of the past, and to me, that is inspiring.”

The patient story: “Little Linda”

Federman treated the youngest patient on the trial who was just one month old. “Little Linda” had infantile fibrosarcoma, which is a rare cancer in children that can appear anywhere in the body, and reportedly affects only about 20 patients in the United States annually. Linda had a large mass that took up 50 percent of her scalp and was growing every day. Despite the hopeless situation, Linda started treatment in this clinical trial as soon as she was eligible and within days, the tumor began to shrink. Today, Linda is a rambunctious toddler who loves playing with the animals on her family’s farm. Watch her story.