When no medical treatment existed for their children, these fathers built one
Lifestyle
Audio By Carbonatix
11:00 AM on Friday, June 12
By Jianna Lieberman for Kivo, Stacker
When no medical treatment existed for their children, these fathers built one
Every year on Father's Day, the internet fills up with tributes to dads who showed up at recitals, practices, and bedsides during long nights. But there is another kind of showing up that rarely makes the greeting cards: the kind where a father receives the worst news of his life, is told there is nothing medicine can do, and decides that is simply not an acceptable answer.
These are the dads who became founders to try to save their child’s life. They are not scientists, not investors, not pharmaceutical executives, or at least they weren't before the diagnosis. They are fathers who looked at a gap in human knowledge and decided to close it themselves. Like Terry Pirovolakis, who launched a new gene therapy company to save his son's life.
In this story, Kivo, a GxP-compliant document & process management platform, looks at the extraordinary stories of Pirovolakis and two other founder dads, John Crowley and Matt Might.
The Scale of the Problem They're Solving
To understand why these men do what they do, you first have to understand the landscape they are operating in.
Rare diseases, defined in the U.S. as any condition affecting fewer than 200,000 people, are collectively not rare at all. According to the National Organization for Rare Disorders (NORD), 1 in 10 Americans lives with a rare disease, totaling more than 30 million people.
Half of these are children.
The numbers get bleaker from there. Of more than 10,000 known rare diseases, fewer than 5% have an FDA-approved treatment, according to a 2024 report from the U.S. Government Accountability Office. The average time for a family to receive an accurate diagnosis is 4.8 years, per a 2024 analysis published in The Lancet Global Health, years during which a child may be deteriorating and families are searching for answers that often don't come. That same analysis found that approximately 30% of children with a rare disease die before the age of five.
For conditions affecting only dozens or hundreds of people worldwide, the traditional pharmaceutical calculus simply doesn't work. There are no blockbuster revenues to justify a billion-dollar development program. There aren’t large patient pools to power randomized controlled trials. There's no marketing department calculating whether the R&D spend is worth it.
Which is why, remarkably, there are situations where the people developing treatments for rare diseases are not pharmaceutical companies at all. They are parents. Moms. Dads.
Terry Pirovolakis and the Race to Save Michael
On April 2, 2019, Terry Pirovolakis got the call that changed everything.
After 18 months of searching for an explanation for his youngest son Michael's symptoms, he finally had a diagnosis: Spastic Paraplegia Type 50, or SPG50, a rare neurodegenerative disorder characterized by developmental delays, seizures, small head size, and a progressive stiffening of muscles that, left untreated, would eventually lead to full paralysis. Michael was one of only 80 known children in the world with the condition.
The doctors told Terry and his wife Georgia to go home, love their son, and give him the best life possible. There was nothing available to prevent what was coming.
Pirovolakis is not the kind of person who accepts that answer.
Within weeks, he and Georgia had launched CureSPG50, a nonprofit aimed at funding gene therapy research for SPG50. They raised money any way they could: crowdfunding campaigns, car washes, community events. They cold-called researchers. They read everything they could find on gene therapy. Terry, who had no background in pharmaceutical development, taught himself enough molecular biology to have credible conversations with scientists who did.
Less than three years after the diagnosis, a clinical trial developed specifically for Michael took place. He became one of the first children with SPG50 to receive a gene therapy intervention.
Out of that journey, Terry founded Elpida Therapeutics, a name that means hope in Greek, a socially responsible biotech corporation with a single animating principle: develop gene therapies as fast as possible, for as many children as possible. As of 2025, Elpida is pursuing five active gene therapy programs, with plans to dose 8 to 12 children in each over the next two to three years.
John Crowley and the Blueprint That Started It All
If Terry Pirovolakis is the current generation of dad founders, John Crowley is one of the original dads who proved it was possible.
On March 13, 1998, Crowley learned that his daughter Megan had Pompe disease, a severe neuromuscular disorder that destroys muscle tissue, enlarges the heart, and in infantile-onset cases is often fatal within the first year of life. Weeks later, his infant son Patrick was diagnosed with the same condition. The Crowley family was told both children would likely not survive to adulthood.
Crowley, who held a law degree from Notre Dame and an MBA from Harvard Business School, had been building a career in pharmaceutical marketing at Bristol-Myers Squibb. He walked away from it.
He and his wife Aileen poured their life savings into a new biotech startup called Novazyme Pharmaceuticals, taking a second mortgage on their home to fund early research. Crowley had no scientific background. He didn't care. He had two children running out of time.
Novazyme went from a $1 million angel round to $27 million in venture capital and was ultimately acquired by Genzyme Corporation for nearly $200 million. The enzyme replacement therapy that came out of that work, Myozyme, later redeveloped as Lumizyme, saved Megan and Patrick's lives. Today, more than 3,000 people worldwide with Pompe disease receive those treatments.
Crowley went on to found Amicus Therapeutics, a global biopharmaceutical company that grew from five employees to more than 600, operating across 27 countries, with a focus on rare genetic diseases. He now serves as CEO of the Biotechnology Innovation Organization (BIO), the largest biotech trade association in the world.
His family's story became a Pulitzer Prize-shortlisted book, “The Cure: How a Father Raised $100 Million and Bucked the Medical Establishment in a Quest to Save His Children,” and a 2010 Hollywood film, “Extraordinary Measures,” starring Harrison Ford and Brendan Fraser.
"I think I did my job," Crowley has said of his role as a father. "As a dad, I did what I had to do."
He did not do it alone. But he was one of the earliest and most visible examples of a dad taking his child's wellbeing into his own hands by launching a pharma company.
Matt Might and the Blog Post That Found Nine More Patients
In 2012, Matt Might's son Bertrand became the first known person in the world to be diagnosed with NGLY1 deficiency, a condition so newly identified that it didn't yet have a name, a research community, or any treatment pathway whatsoever.
Might was a tenured computer science professor. He did what computer scientists do: He tried to solve the problem with data.
He wrote a detailed blog post, "Hunting Down My Son's Killer," describing Bertrand's symptoms, genetic findings, and the biological mechanism of NGLY1 deficiency. His goal was to create what he called a "Google dragnet," a piece of content indexed well enough to surface in the searches of any other family whose child had the same undiagnosed condition.
Within 24 hours, the post had gone viral. Within 13 months, it had helped identify nine more children with NGLY1 deficiency. A patient community formed. Researchers volunteered their time. Funding materialized.
Applying what he knew about computational modeling, Might began identifying FDA-approved compounds that could theoretically help Bertrand. He found a candidate, a common supplement called N-acetylglucosamine available on Amazon, and after testing it himself with no ill effects, began giving it to his son. Three days later, he walked into Bertrand's room to find him crying. Not yelling: crying, with actual tears. It was the first time a child with NGLY1 deficiency had ever produced tears.
"They may have just been tears," Might said, "but they were an ocean of science for the disease. They unlocked so much about this disorder."
Might co-founded Pairnomix, a startup focused on identifying personalized therapeutic options for patients with rare genetic disorders. The company was acquired by Q State Biosciences in 2018. He went on to become Director of Precision Medicine at the University of Alabama at Birmingham and a researcher at Harvard Medical School, and he remains chief scientific officer of NGLY1.org.
Bertrand Might died in 2020 at the age of 12. But the research his father sparked has since helped dozens of other children, and the patient community that blog post built continues to grow.
What These Dads Have in Common
John Crowley had an MBA from Harvard. Matt Might had a PhD in computer science. Terry Pirovolakis had a drive so powerful that his company's name is the Greek word for hope.
None of them had a pharmaceutical background when their child was diagnosed. None of them were supposed to end up here. But the alternative, doing nothing, was the one option they could not live with.
"What we realized was that if we didn't continue to do this ourselves, nobody was going to do it," said Allyson Berent, a fellow parent-founder who spoke alongside Terry Pirovolakis at the 2024 STAT Summit, "because the priorities of parents and patients will never change."
According to IQVIA's Global R&D Trends report via Remington-Davis, 45% of all global clinical trial starts in 2024 were focused on rare diseases, a staggering figure that reflects just how much the field has shifted away from blockbuster drugs and toward the edges of human medicine, where the patients are fewest and the need is most acute.
The dad founders didn't create that shift by themselves. But they are, without question, one of its most powerful engines.
This Father's Day, the men worth celebrating aren't just the ones who showed up to the game. Some of them are showing up to the FDA.
This story was produced by Kivo and reviewed and distributed by Stacker.
