Learn about the FDA Approval of Larotrectinib

VIDEO | 02:16

Hear from our doctors, who say that the FDA approval of larotrectinib delivers on the original promise of precision medicine. Published November 26, 2018

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I totally believe I've got the miracle of being alive through this treatment.

With the approval of larotrectinib what we have is the first targeted therapy ever approved for patients with a specific genetic alteration, regardless of the type of cancer they have.

In technical terms, larotrectinib is a tyrosine kinase inhibitor. And in simple terms, it's a drug that inhibits the protein that's responsible for cancer growth in these tumors. We started this trial at Memorial in 2015.

I started the medication, which is a pill. I take it twice a day. I don't have any hard side effects.

You sound good.

The majority of our patients who started three years ago are still on treatments, seeing me in clinic once a month, doing well with no signs of substantial disease progression.

The types of cancers that we saw in this clinical trial include cancers that we all know-- lung cancer, colon cancer, pancreas cancer, breast cancer, thyroid cancer, rare pediatric cancers. We were really waiting for a drug like this to come into our program. A drug that really worked not in just subsets of cancers, but in any cancer that had that particular mutation.

You could tell even before these patients had scans that they were responding to treatment. There were a few cases of patients that had externally visible tumors, that very nicely receded with therapy.

We did these PET scans and CAT scans before starting the treatment. And two months later, we repeated these CAT scans and PET scans, and the scans were clear.

The most exciting part of my job is that a-ha moment when you realize something is working and you're really on to something. This is delivering on the original promise of precision medicine, which was that we were going to be able to characterize a mutation that drives an individual patient's tumor and treat that mutation, and the patient would respond regardless of their cancer type. And that's finally what we've been able to achieve with larotrectinib.

This is, as a doctor, completely fulfilling to see that the research that we've done-- we say on the bench, so in the laboratory-- can find its way into the clinic and really help someone turn things around.

Thank you.