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Is Eteplirsen a Miracle Drug?

By March 1, 2016 October 30th, 2016 Tracy's Blog

For several years now, I have been advocating for the advancement and approval of a new drug called eteplirsen being developed by Sarepta Therapeutics. My fellow advocates and I have been careful not to overblow the promise of this experimental medication. The drug slows disease progression for up to 13% of children with Duchenne, the ones who have the most common genetic mutation. It is not a cure.

Two years ago, we encouraged more than 100,000 people to sign a White House petition urging a swift and efficient FDA review. Throughout the process, we were deliberate and clear: eteplirsen does not eliminate Duchenne. I cringed when some news reporters covering the story called eteplirsen a “miracle drug.” I worried that we would be dismissed by the scientific community and regulators as desperate parents who do not understand the data. I did not want to be characterized as frantically clinging to any hope, falsely claiming “It’s a miracle.”

I have always known that eteplirsen is not a cure. But I recently realized that it is, in fact, a miracle. Let me explain.

Late last year on the first night of Hanukkah, Charley broke his hip (femoral neck). Eleven weeks later — last Saturday — I posted this video of Charley walking a few steps.

I chose a single word to accompany my video: “miracle.” Charley’s miracle is not related to eteplirsen. He he does not have the “right” genetic mutation to be eligible for that treatment; in fact he has been participating in a clinical trial for a different experimental drug. I only raise his example to illustrate my point: Before Charley’s diagnosis and before I understood the reality of Duchenne, never in a million years did I think I would consider it a miracle that my teenager could walk to the kitchen by himself to grab a snack out of the fridge. But in the context of this brutal and aggressive muscle wasting disease, Charley’s ability to walk independently matches the definition of a miracle: “a highly improbable or extraordinary event, development, or accomplishment that brings very welcome consequences.”

It’s also a miracle that 10 out of the 12 boys treated with eteplirsen for more than four years can still walk to this day. When you compare them to a similar group of boys from a matched natural history control, only 1 out of 11 boys still walked after the same period of time. While we usually think of a miracle as a one-time phenomenon brought about by divine intervention, this one is caused by treatment intervention, which means science can make it happen again for more and more children.

Eteplirsen is a miracle, and I am not hesitant to say it. It doesn’t mean I’m desperate. It doesn’t mean I don’t understand the data. It means I understand Duchenne, and I know what a miracle looks like in our world.