"…the kids could walk if they put their mind to it."
- Dr. Ronald Farkas,
Clinical Team Leader
U.S. Food and Drug Administration
So expressed earlier this week in Washington, D.C., at an Food and Drug Administration (FDA) advisory panel meeting to discuss a potential life-saving drug known as eteplirsen. You may or may not have heard about the meeting, eteplirsen, or the ill-advised statement above, but the Duchenne community sure has been abuzz.
The D.C. gathering drew a standing room only crowd in support of a favorable recommendation despite a skeptical FDA advisory panel. Many boys and men living with Duchenne attended to express their needs, as did their families, friends, and relatives. The FDA advisory panel listened to more than 50 people share their unique perspective with Duchenne and how eteplirsen could impact their life. The advisory panel patiently listened as speaker after speaker approached the microphone and voiced their individual and collective views of the fatal disease, which currently has no cure. In the end, however, it seemed only an exercise in free speech as the FDA advisory panel DID NOT recommend eteplirsen for accelerated approval by a vote of 7-6 (including three abstentions). Despite the disappointment, the Duchenne community’s thirst for something…anything…to tame the beast known as Duchenne has never been stronger.
Sertepa Therapeutics, Inc., manufacturer of the exon-skipping drug, sought FDA approval for treatment of patients with Duchenne Muscular Dystrophy. Although treatment would apply to only about 13% of all Duchenne patients, it is a positive step. The issue, however, is not eteplirsen or any other drug (note: FDA denied another potential Duchenne drug, drisapersen, in January 2016). The issue is that we never stop our search for a cure to this unforgiving disease.
Don’t think those with Duchenne are unaware. They are aware, desperately. Every year, my son, Alex, meets with his neurologist, Dr. Brenda Wong, at Cincinnati Children’s Hospital Medical Center as part of his Duchenne clinic visits. Every year, Alex asks the same question.
“Have you found a cure yet?”
Every year, Alex hears a similar response.
“No, but….”
Don’t get me wrong, Dr. Wong does her patient and enthusiastic best to explain the latest in Duchenne research, and does so with hopeful seriousness. She compassionately tells Alex they are working hard to find a cure, and getting closer every day. Alex respectfully nods and smiles his appreciation, but you can clearly see his frustration and waning hope. Alex’s letdown is enough to break your heart every time.
So, when a drug such as eteplirsen shows promise, ANY promise, even if remote, hearts within the Duchenne community swell with tempered enthusiasm. We understand a cure will not be found overnight. We understand our sons, like Alex, may not be eligible for its use (eteplirsen applies only to boys missing exon 51; Alex is missing exons 49-54). We understand the drug may not work, as only marginal successes were observed in the few eteplirsen trials. We also understand the irreversibility of Duchenne, let alone the complexity of fixing a genetic-scale issue throughout the human body. We understand the long and difficult road ahead.
But we need something.
So, when a clinical team leader of the FDA says “…boys could walk if they put their mind to it,” while not recommending Sarepta’s request for approval to treat Duchenne,…well, it steals the air from your lungs and hope from your heart as Duchenne so often does to our boys.
Yet, it also makes you think…our sons’ futures are in the hands of those who don’t understand Duchenne?? I hope Dr. Farkas’ words above were misspoken. I hope he wishes his words back (note: Dr. Farkas’ email was shut down due an overload of messages from astonished and angry Duchenne families). Everyone says the wrong thing now and then…right? But, we’re talking about human lives, not a dismissive wave of the hand accompanying an insatiable need for more data and “substantial evidence of effectiveness.”
Despite the setback, the louder issue is renewed Duchenne awareness and our spirit to never give up. For everyone, not just those in the Duchenne community, the issue should be to chase every angle and turn over every stone in the search for a cure. The issue should be to do anything we can to cure this relentless, lethal, disease. Our boys are counting on us.
The issue should not be risk aversion until absolute certainty of benefit. ANYTHING other than Duchenne’s status quo provides benefit! Some said the FDA meeting felt like a trial where families needed to defend their child’s right for treatment. As many are concerned, at least Eteplirsen proves a glimmer of hope. Why not pursue it?
Nonetheless, Duchenne families are familiar with obstacles and roadblocks. They’re common themes in our lives. Just when you think things look promising, Duchenne smacks reality in your face. Just when you think you’ve made progress, Duchenne knocks you back down. Just when you think you can handle the beast, the leash snaps and you must work to corral it all over again and again and again. It’s discouraging, tiring and, at times, demoralizing.
But, Duchenne families are known to get back up and continue fighting for their son’s lives, even with odds stacked heavily against them. We encourage, lift, and support our sons every day. We keep on fighting this dreaded monster of a disease that steals time, ability, and dreams. We instill hope in our sons. We promote life and happiness whenever possible and make sure our boys know they are loved and not forgotten. We let them know they are no different from anyone else. We let them know we have their back.
In turn, our boys teach us about life. They remind us what is important, like a smile, a hand to hold, laughter, and love. They show us how to live despite any obstacle. They show us to forget about what we are missing and focus on what we have. They teach us that joy can be found in the simplest of things and during the simplest of times. They teach us patience. They teach us perseverance. Most of all, they teach us love.
So, when a potential life-saving drug like eteplersin is advanced and then later denied, despite enthusiastic support from those who would directly benefit, the Duchenne community collectively sighs and shakes its head in frustration. Then, we then pull up our bootstraps and continue marching, hoping, and loving. We also get louder.
It’s what we do.
The FDA may believe our sons simply need to just put their mind to it. But we know differently. Are sons are fighters…
Every day,
Every night,
Every breath, and
Every heartbeat.
It’s what THEY do.
Their only wish for ALL OF US…you, me, everyone…
...is to find a cure.
(Note: Encouraging and nuanced comments from Dr. Janet Woodcock, Director for Drug Evaluation and Research at the FDA, suggest the advisory panel’s recommendation to deny eteplirsen approval may be reconsidered when a final decision is released by May 26, 2016.)
- Dr. Ronald Farkas,
Clinical Team Leader
U.S. Food and Drug Administration
So expressed earlier this week in Washington, D.C., at an Food and Drug Administration (FDA) advisory panel meeting to discuss a potential life-saving drug known as eteplirsen. You may or may not have heard about the meeting, eteplirsen, or the ill-advised statement above, but the Duchenne community sure has been abuzz.
The D.C. gathering drew a standing room only crowd in support of a favorable recommendation despite a skeptical FDA advisory panel. Many boys and men living with Duchenne attended to express their needs, as did their families, friends, and relatives. The FDA advisory panel listened to more than 50 people share their unique perspective with Duchenne and how eteplirsen could impact their life. The advisory panel patiently listened as speaker after speaker approached the microphone and voiced their individual and collective views of the fatal disease, which currently has no cure. In the end, however, it seemed only an exercise in free speech as the FDA advisory panel DID NOT recommend eteplirsen for accelerated approval by a vote of 7-6 (including three abstentions). Despite the disappointment, the Duchenne community’s thirst for something…anything…to tame the beast known as Duchenne has never been stronger.
Sertepa Therapeutics, Inc., manufacturer of the exon-skipping drug, sought FDA approval for treatment of patients with Duchenne Muscular Dystrophy. Although treatment would apply to only about 13% of all Duchenne patients, it is a positive step. The issue, however, is not eteplirsen or any other drug (note: FDA denied another potential Duchenne drug, drisapersen, in January 2016). The issue is that we never stop our search for a cure to this unforgiving disease.
Don’t think those with Duchenne are unaware. They are aware, desperately. Every year, my son, Alex, meets with his neurologist, Dr. Brenda Wong, at Cincinnati Children’s Hospital Medical Center as part of his Duchenne clinic visits. Every year, Alex asks the same question.
“Have you found a cure yet?”
Every year, Alex hears a similar response.
“No, but….”
Don’t get me wrong, Dr. Wong does her patient and enthusiastic best to explain the latest in Duchenne research, and does so with hopeful seriousness. She compassionately tells Alex they are working hard to find a cure, and getting closer every day. Alex respectfully nods and smiles his appreciation, but you can clearly see his frustration and waning hope. Alex’s letdown is enough to break your heart every time.
So, when a drug such as eteplirsen shows promise, ANY promise, even if remote, hearts within the Duchenne community swell with tempered enthusiasm. We understand a cure will not be found overnight. We understand our sons, like Alex, may not be eligible for its use (eteplirsen applies only to boys missing exon 51; Alex is missing exons 49-54). We understand the drug may not work, as only marginal successes were observed in the few eteplirsen trials. We also understand the irreversibility of Duchenne, let alone the complexity of fixing a genetic-scale issue throughout the human body. We understand the long and difficult road ahead.
But we need something.
So, when a clinical team leader of the FDA says “…boys could walk if they put their mind to it,” while not recommending Sarepta’s request for approval to treat Duchenne,…well, it steals the air from your lungs and hope from your heart as Duchenne so often does to our boys.
Yet, it also makes you think…our sons’ futures are in the hands of those who don’t understand Duchenne?? I hope Dr. Farkas’ words above were misspoken. I hope he wishes his words back (note: Dr. Farkas’ email was shut down due an overload of messages from astonished and angry Duchenne families). Everyone says the wrong thing now and then…right? But, we’re talking about human lives, not a dismissive wave of the hand accompanying an insatiable need for more data and “substantial evidence of effectiveness.”
Despite the setback, the louder issue is renewed Duchenne awareness and our spirit to never give up. For everyone, not just those in the Duchenne community, the issue should be to chase every angle and turn over every stone in the search for a cure. The issue should be to do anything we can to cure this relentless, lethal, disease. Our boys are counting on us.
The issue should not be risk aversion until absolute certainty of benefit. ANYTHING other than Duchenne’s status quo provides benefit! Some said the FDA meeting felt like a trial where families needed to defend their child’s right for treatment. As many are concerned, at least Eteplirsen proves a glimmer of hope. Why not pursue it?
Nonetheless, Duchenne families are familiar with obstacles and roadblocks. They’re common themes in our lives. Just when you think things look promising, Duchenne smacks reality in your face. Just when you think you’ve made progress, Duchenne knocks you back down. Just when you think you can handle the beast, the leash snaps and you must work to corral it all over again and again and again. It’s discouraging, tiring and, at times, demoralizing.
But, Duchenne families are known to get back up and continue fighting for their son’s lives, even with odds stacked heavily against them. We encourage, lift, and support our sons every day. We keep on fighting this dreaded monster of a disease that steals time, ability, and dreams. We instill hope in our sons. We promote life and happiness whenever possible and make sure our boys know they are loved and not forgotten. We let them know they are no different from anyone else. We let them know we have their back.
In turn, our boys teach us about life. They remind us what is important, like a smile, a hand to hold, laughter, and love. They show us how to live despite any obstacle. They show us to forget about what we are missing and focus on what we have. They teach us that joy can be found in the simplest of things and during the simplest of times. They teach us patience. They teach us perseverance. Most of all, they teach us love.
So, when a potential life-saving drug like eteplersin is advanced and then later denied, despite enthusiastic support from those who would directly benefit, the Duchenne community collectively sighs and shakes its head in frustration. Then, we then pull up our bootstraps and continue marching, hoping, and loving. We also get louder.
It’s what we do.
The FDA may believe our sons simply need to just put their mind to it. But we know differently. Are sons are fighters…
Every day,
Every night,
Every breath, and
Every heartbeat.
It’s what THEY do.
Their only wish for ALL OF US…you, me, everyone…
...is to find a cure.
(Note: Encouraging and nuanced comments from Dr. Janet Woodcock, Director for Drug Evaluation and Research at the FDA, suggest the advisory panel’s recommendation to deny eteplirsen approval may be reconsidered when a final decision is released by May 26, 2016.)
Photo by John Boal for The Boston Globe
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