From rock stages to research labs: An Austin father's quest to save his daughter

AUSTIN (KXAN) - In the heart of Austin, where music and innovation often intersect, beloved local artist Casey McPherson's story has struck a chord with families across the world.  His journey from alternative rock frontman to genetic disease researcher is driven by an unwavering love for his daughter, Rose. 

Leaving a thriving musical career  

McPherson was living the dream of many musicians, playing at Austin's biggest venues, receiving support from local radio stations and fans across Central Texas and the country. 

"I was classically trained as a kid.  I moved up to Austin to start a band, Endochine, and then Alpha Rev and the city supported us. We played ACL Fest, had the major label record deal — the sort of the dream story of a local band," McPherson explained.  "It was what I thought I was going to do the rest of my life." 

He became the lead singer and songwriter of Flying Colors, released multiple albums and toured the United States and Europe.

As his career was soaring, he got married and eventually had two daughters.  That's when things changed. 

After Rose was born, McPherson said he noticed she took a little longer to learn how to walk, crawl and talk.  But as time passed, McPherson knew something was wrong when she started missing more and more milestones. 

“She began to choke on her food. She would try to walk, but she'd fall flat on her face without putting her hands in front of her,” McPherson recalled.  “She lost her ability to talk as she was developing her language.  She used to say three words — she had 'dad,' 'mom' and 'outside.'” 

Testing revealed an official diagnosis: her HNRNPH2 gene was mutated, meaning her body wasn't producing the necessary proteins for her to develop properly. 
 

“We brought it [whole exome sequencing results] to the pediatric neurologist, and he says, ‘Casey, your daughter has a rare genetic disease. There's no cure, there's nothing we can do. Good luck,’" McPherson said.  "I was faced with the dead end of our healthcare system for Rose."

A father's fight 

McPherson was at a crossroads.  He had a record deal from Sony on his desk and a daughter with an incurable disease. 

McPherson chose Rose.

"As a dad, your role is to protect your kids," he explained. "Where was the monster to slay? In Rose's case, it was a single nucleotide in a gene in her brain. I was so devastated, because I knew she would never have the freedoms that other kids have, like make friends or go to college or fall in love ... and without a genetic treatment, she'll never be able to talk again.” 

That’s when his journey began. 

“I literally Googled ‘parent cures child of rare disease’, and I saw all of these parents organizing science teams, creating genetic treatments and putting them into the FDA clinical trials. So I knew, look, if they can do it, I know I can do it," McPherson said. 

Creating a new path 

McPherson began researching genetic medicine, learning about drug development and building a team of scientific experts. 

According to the latest data from the National Institutes of Health, fewer than 50 people have been diagnosed with the same condition as Rose. But at least 30 million in the U.S. are living with a rare disease because researchers have identified at least 7,000 different rare diseases.  Of those, only 500 have approved treatments. FDA approval for treatments typically requires large-scale clinical trials which require hundreds or sometimes thousands of people living with the condition to participate. 

McPherson eventually started To Cure a Rose Foundation. The organization brings genetic treatments to children with debilitating rare diseases, many associated with autism. The focus is on "funding researchers and promising technology to help children like Rose. "funding and bringing together determined researchers and promising technology to create a brighter future for children like Rose," according to its website.

“We have incredible science team there.  We built a lab that actually functions at Austin Community College’s bio-incubator space where we could make these genetic medicines without having to contract really expensive companies or universities,” he explained.  

But he realized they needed a commercial company that can take the drugs through FDA clinical trials and bring them to the market at scale.  So he started AlphaRose Therapeutics to develop and commercialize genetic treatments for “suffering patients of small populations” and make personalized medicine and therapies for patients with rare genetic diseases. 

“We have a treatment for Rose’s disease, and we see a profitable path, so that I can have investors come into AlphaRose Therapeutics and then the Foundation can have sustainable revenue so they can make more treatment,” McPherson explained.  “We are trying to make this sustainable circle — right now with medicine, money goes one way: it goes to the investors’ pockets.” 

McPherson said his team could be in the clinic with their drug for Rose’s disease as soon as the end of 2025 or the beginning in 2026 as they raise the investment dollars. 

“Any person can invest in our company. We're doing this in a way where patients can build a biotech company based around patients, and if we can reach our $5 million seed round, we'll be able to treat Rose and other kids as soon as the end of this year,” McPherson explained.  

Beyond his personal mission, McPherson is advocating for systemic changes in rare disease medical research and regulation. He is taking his fight to Capitol Hill this month, highlighting the challenges and impacts of rare diseases on families across the world.  

“I think that we have an opportunity to mold regulatory that's still safe,” McPherson said. “Our hope is that policy makers and some of the politicians there can begin to understand how important it is, and that they have an opportunity to create change that ends up saving lives in perpetuity, once we get these laws fixed and regulations fixed.” 

A message of hope 

McPherson's ultimate goal extends beyond Rose. He hopes to create a new model of personalized genetic medicine that could help those impacted by rare diseases. 

"What Rose and I have made together here will potentially affect thousands, if not millions of kids in the future," he said. 

He and Rose are in a documentary called "Rare" that chronicles their journey and brings attention to the challenges faced by families battling rare genetic diseases. 

Rose is now 9 years old. She continues to lose her ability to communicate, has seizures and her motor skills are impaired, her father said.

"After I give her a bath, we have this window," McPherson said. "I'll sit on the bed, play guitar, give her a harmonica. She'll blow into it, and we're focused on each other. She might be my only fan now, but she's the best fan I could ask for." 

He says Rose gives him purpose. 

“Rose is determined, tenacious, beautiful and kind amidst her incredible amount of disability, confusion, fear and terror and she — every day for her is a challenge, and she faces it head on,” McPherson said.