FDA Approves Cutting-Edge Cancer Drug from Inova Schar Cancer Institute Clinical Trial

Inova’s Dr. John Deeken served as the site principal investigator for this prestigious study

In groundbreaking news for the medical and cancer research community, the Food and Drug Administration (FDA) approved a new, breakthrough cancer drug, larotrectinib, or Vitrakvi®, that targets specific genetic mutations in adult and pediatric patients. This is only the second tissue-agnostic drug that the FDA has approved.

Photo of Dr. John Deeken

John F. Deeken, MD

“We were one of the few centers in the country that were participating in the clinical study using larotrectinib, and it was extraordinarily successful from a cancer standpoint,” said John Deeken, MD, acting chief executive officer for the Inova Schar Cancer Institute. “We found that three-fourths of patients had their tumors shrink by the treatment. In cancer, that’s huge.”

Inova Schar Cancer Institute (located in Fairfax, Va. and serving the Washington, DC metro area) was one of a prestigious set of cancer centers selected to participate in the accelerated clinical trial. Dr. Deeken was the site principal investigator for this clinical trial.

Changing the future of cancer care with genetics

According to FDA Commissioner Scott Gottlieb, MD, “We now have the ability to make sure that the right patients get the right treatment at the right time,” which represents a drastic change from traditional chemotherapy and radiation options. Larotrectinib is a cancer treatment based on biomarkers in a patient’s genetics; this differs vastly from the more traditional cancer treatment methods which focus on the part of the body where the tumor originated.

“[Personalized] medicine is not just an idea.
It’s actually working, and it should be part of
your care if you go to the right place.”
– John F. Deeken, MD

This cutting-edge drug is for patients with advanced, solid tumors that develop from a Neurotrophic Receptor Tyrosine Kinase (NTRK) gene fusion. NTRK genes occur in many types of cancer and help cancerous tumors grow. For patients with limited other options, larotrectinib works to shrink tumors to a manageable size.

This cutting-edge clinical trial was open to multiple cancer types, and multiple age groups

The NTRK gene mutation is very rare, but is seen in some head and neck cancers, which is Dr. Deeken’s practice specialty. Due to the rarity of the NTRK mutation, Inova cast a wider net for eligible patients beyond its head and neck cancer patients.

“Anyone, no matter what type of cancer they had, was allowed to be in the trial if their tumor had this mutation in it,” said Dr. Deeken. “And what’s even more innovative, was that it was a trial open to pediatric cancer patients as well as adults, so this is all new to how we test cancer drugs.”

Nationwide, the clinical trial included 55 patients, ranging in age from 4 months to 76 years. After two years of searching for eligible Inova patients with this specific genomic cancer signature, Inova enrolled two patients in the study.

“This is a rare mutation that’s only seen in a couple thousand cancer patients in the U.S. a year, so it’s a needle in a haystack. But if you find that needle for patients, then this drug is extraordinarily helpful,” said Dr. Deeken.

Revolutionary results with minimal side effects

What’s especially remarkable about larotrectinib is the balance it strikes between effectiveness and manageable side effects. Dr. Deeken saw accelerated results of larotrectinib’s effectiveness, unlike what he’s seen in traditional cancer drugs. “We and the other investigators around the country saw results early on, within the first couple months, and many of them were durable,” said Dr. Deeken. “I have a patient now who’s been on this drug for two years, still having benefit. His tumor is almost shrunk down to nothing, and the drug is well tolerated.”

At Inova Schar Cancer Institute, patients receive personalized care that’s tailored to them. Dr. Deeken emphasizes that “personalizing medicine is not just an idea. It’s actually working, and it should be part of your care if you go to the right place.”

“What we’ve built here at Schar, we have the experts and operation to be able to offer these cutting-edge tumor testing and clinical trials to patients,” explains Deeken. “They don’t have to have to go to Houston, Boston or New York for these kinds of options; they can stay here in Northern Virginia.”


American Cancer Society: FDA Approves Vitrakvi (Larotrectinib) for Tumors With Certain Genetic Change

Business Insider: The FDA just approved a drug that targets cancers based on DNA, rather than where the tumor is in your body


  1. Cindy M on December 7, 2018 at 9:25 am

    Wonderful news! Congratulations!

  2. Leonard Brown on December 11, 2018 at 10:32 pm

    Hope it is the first of many breakthroughs for Schar Institute

  3. Miriam Cohen on December 12, 2018 at 7:19 pm

    This is fabulous news! Congratulations!

    Thank you for opening the trial to pediatric patients, that is a huge and much needed new mindset. We appreciate it. Were there any osteosarcoma patients lucky enough to qualify for the study?

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