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Family calls on FDA to approve life-saving treatment
One Bellevue family is seeing life-changing results for their son thanks to a new drug. But getting the drug FDA-approved is now faced with challenges and technicalities, ultimately putting the future of the treatment in jeopardy.
BELLEVUE, Wash. - A family in Bellevue is seeing life-changing results for their son, thanks to a clinical trial drug. However, getting the drug approved by the U.S. Food and Drug Administration (FDA) has been faced with "technicalities." Ultimately, the family said the technicalities could put future access to the treatment in jeopardy.
"I would hope that the FDA is seeing this as a situation where they can give people something good to try. And I would hope the focus is on that rather than technicalities about data," said Aidan Leffler, 22.
The backstory:
Aidan was diagnosed with Duchene Muscular Dystrophy (DMD) at the age of three. DMD is a progressive muscle-wasting disease that affects males.
"Kids with Duchenne typically lose the ability to walk sometime in their teens or even slightly before. And then they struggle with the use of their arms and then they have a short lifespan due to cardiac and respiratory issues," said Mindy Leffler, Aidan’s mother.
The outcome of the disease is grim. Mindy explained there is no history of DMD in their family. The Lefflers said Aidan has been in various clinical trials across the country trying to improve his health.
"I don’t think it ever really settled into me how severe this is and how much it means until quite recently. Genuinely, I’m facing a historical pathway. It’s always been the same. It’s always been situations where people get weaker and they die early. So, at a certain point I was like, ‘oh, this is what’s going to happen to me.’ And there was this overbearing fear," said Aidan.
His fear turned to hope when Aidan began a new drug during a clinical trial through a company called Capricor Therapeutics. Mindy described the treatment as a cardiac-derived cell therapy designed to help Aidan improve his arm strength and stabilize his heart.
"There aren’t any safety concerns at this point. So, it’s a really low burden, low risk way for him to kind of maintain the life that he’s been able to live," said Mindy.
"After years of my life that I’ve put into clinical trials, we’re finally seeing something that is paying it back for me that’s giving me the chance to live as normal life as I can," said Aidan.
Aidan started the quarterly treatment during the summer before his freshman year at the University of Washington. Since then, his family said the drug helped him get through college relatively independently. He graduated in June with a degree in political science.
"He’s been able to graduate from college, make plans to get his master’s degree. He’s in a relationship, he just got back from Argentina where he flew down there by himself," said Mindy. "He’s doing things that I never thought that he would have the ability to do when he was diagnosed as a little kid."
The family credits part of his successful health journey to the treatment. In 2024, they were encouraged to hear the FDA initially showed support for the life-changing treatment.
"The FDA was so impressed with that data a year ago that they told [Capricor Therapeutics] to file for approval. So, the company spent millions of dollars, a lot of people hours putting that application together. And then unfortunately, something changed at the agency in the way they were looking at the data, and they turned the application down this summer," said Mindy.
Dig deeper:
The recent changes in FDA leadership garnered national headlines. The Lefflers said one person who was most familiar with Aidan’s clinical trial data left the FDA in June. Mindy said the FDA also changed its data review process, presenting various "technicalities" when processing rare disease drug applications.
"They have statistically significant heart data and it’s a really quality data set. And so, our hope is that FDA will exert the same flexibility that they were willing to share a year ago when they take a look at this Phase Three data that [Capricor Therapeutics] is about to submit to the FDA," said Mindy.
Recognizing the risks if the treatment were no longer accessible, Mindy and Aidan have been hard at work to save it. Mindy has flown to Washington D.C. three times in the past two weeks, speaking with Congressional members and advocating for the treatment. Aidan, alongside Capricor Therapeutics, gave a speech to the FDA directly.
"Kind of about what this drug has meant for me and the paths that I have in front of me, whether I have access to the drug from now on or whether access is taken away and then my life changes from there on out," said Aidan.
"There are boys whose hearts are dealing with irreparable damage while we’re waiting for this to get sorted out," said Mindy. "If he comes off treatment, he’s going to follow much more of a typical progression for boys with Duchenne."
The family is already planning their next trip to the nation’s capital to advocate for the clinical trial and treatment.
"The possibility of losing that is obviously heartbreaking. So, you do whatever you can as a parent. We’re used to bad news. We’re used to dealing with loss and heartbreak. But what I can’t stomach is loss and heartbreak due to technicalities," said Mindy.
During their advocacy, the family also takes time to foster a sense of normalcy and celebrate Aidan’s achievements. After graduating from UW this summer, he was accepted into graduate school to study public policy. He also just returned from a two-week trip visiting his girlfriend in Argentina.
"Seeing all the old architecture and going to some soccer stadiums and all of those interests that I’ve had and experiencing those in completely different countries. A really awesome experience," said Aidan. "I think that is a really empowering and fulfilling feeling to have."
The young man and his family hope for many more life experiences. They said they hope the FDA will understand the need for urgency to support the treatment that is changing Aidan’s life and others like him.
"You never know what’s going to happen in the future," said Aidan. "But I’m starting to get to a point where I feel comfortable enough to be able to plan my life and do the things I want to do without that fear hanging over me quite as much.
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The Source: Information in this story came from interviews with the Leffler family and FOX 13 Seattle reporting.