Rock frontman Casey McPherson walked away from music professionally so he could find a cure for his daughter Rose
The 9-year-old girl was diagnosed with an ultra-rare genetic disorder that caused her to flatline once and has taken away her ability to speak
“I’m devoting the rest of my life to this,” the devoted dad, who launched a company that has created a treatment for his daughter, tells PEOPLE
Casey McPherson, the frontman of the progressive rock band Flying Colors, used to love singing with his 9-year-old daughter Rose. But his daughter, who was diagnosed with an ultra-rare genetic disorder in 2019, has lost the ability to speak.
“She screams and giggles and cries and makes noises and sounds, but she doesn’t have any friends,” says McPherson, 46, of his daughter’s HNRNPH2-related neurodevelopmental disorder. “She doesn’t know how to play with anyone.”
After McPherson learned about his daughter’s diagnosis, he turned down a record deal from Sony, determined to find a cure for his little girl. In 2023, he launched a biotech company, AlphaRose Therapeutics, and has raised more than $1 million since January — and he plans to raise $5 million by June to ensure his daughter gets the precision medicine she needs.
That treatment has been developed by experts in the field and in addition to raising money to fund it, the company is currently working towards getting approval to use it in a clinical trial. If everything works out, Rose is hopefully just six months away from getting her first treatment.
“This is just the beginning for me. I’m devoting the rest of my life to this,” says McPherson, who appears in Rare, a documentary about five families on a years-long search for cures for their kids with rare and ultra-rare disorders.
Carl-Ulrich Bonnekamp
McPherson remembers singing melodies with his youngest daughter
McPherson, who is also the frontman of the band Alpha Rev, hopes the documentary, which was recently presented in Washington, D.C., will raise awareness as he and others push for a “regulatory path” to invest in treatment for the 30 million people in the United States with rare disorders.
Many of the more than 7,000 rare diseases (i.e., conditions affecting fewer than 200,000 people) are life-threatening and do not have treatment options, according to the FDA.
For McPherson, it’s a race against time as he and his family continue to monitor Rose’s health and ability to engage with the outside world. She’s currently testing at the developmental level of an 18-month-old child, but her dad is remaining hopeful.
“Rose is working really hard. She’s giving everything she’s got to stay alive and to exist,” says McPherson. “So, I should be doing everything I can to give her every possibility of unlocking her freedom.”
Carl-Ulrich Bonnekamp
It took Rose a long time to learn how to climb the stairs, her dad says
After Rose was born in 2016, she appeared to be relatively healthy. “She was missing some developmental milestones, but the doctors weren’t that concerned,” says McPherson. That changed when Rose began choking on her food. As Rose got older, she never learned to crawl, instead she’d fall flat on her face while learning to walk, which took years. But Rose did learn sign language and could say the words “mom,” “dad” and “outside.”
McPherson and Rose’s mom had been taking Rose to doctors’ appointments to get a better understanding of her struggles, but they knew something had to change when Rose could no longer speak. “She would start screaming and she looked like she was in pain,” remembers her dad.
McPherson then embarked on a “diagnostic odyssey,” which many parents with children with rare and ultra-rare genetic conditions embark on as they seek a diagnosis and treatment. Rose was three years old when her parents learned the name of her disorder, which is a result of a mutation of the gene HNRNPH2. Rose also has autism, which her dad describes as stemming from the genetic disorder.
Dr. Jennifer Bain, child neurologist at NewYork-Presbyterian/Columbia University Irving Medical Center, has been studying HNRNPH2-related neurodevelopmental disorder for eight years. She tells PEOPLE that individuals with mutations in that gene often have developmental delays that inhibit their ability to walk and talk, most of whom “need full support into adulthood.”
“Many of them have autism spectrum disorder or autistic features,” she adds. Based on her research, Bain says people with the disorder can be at “increased risk of early death, premature death, regression, seizures, and epilepsy.”
She notes, “So, it certainly can be a very devastating diagnosis.”
Carl-Ulrich Bonnekamp
The father and daughter playing outside
Rose has intermittent seizures and McPherson says his family is bracing for the next one. Her last seizure, which happened last year, caused her to flatline for about a minute before her grandmother brought her back with CPR and she was rushed to the hospital.
Rose also struggles with depth perception, so walking can be difficult, she’s not potty trained and her oxygen levels and heartbeat need to be monitored at night. The daily challenges make it incredibly difficult for her parents, who are now divorced, to leave the house or go on vacation. “You are more isolated,” McPherson says.
Rose’s sister, 11-year-old Weston, has also been affected. “She’s had to take the back seat a lot of times,” says her dad.
Carl-Ulrich Bonnekamp
Rose (left) with her sister Weston (right)
Despite Rose’s struggles, she is actively trying to engage with her world and is currently attending a private school. Her dad says she’s “much smarter” than tests suggest, she’s just lost certain functions.
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In honor of his daughter’s strength, McPherson started a nonprofit, To Cure A Rose Foundation, in 2021. The next natural step was to launch AlphaRose Therapeutics, a public-benefit corporation, in October 2023. McPherson plans for the company to develop treatments for rare genetic diseases at scale, filling a void left by the pharmaceutical industry.
Bain says she’s “thrilled” to learn when any rare disorder has a treatment. “Right now, there’s no FDA-approved medications,” says the pediatric neurologist, who is currently working on a separate clinical trial for three people with the disorder, “and so it definitely needs to be pushed forward.”
The end goal is simple: McPherson hopes that Rose and other children like her will be able to walk, swim and learn in ways they haven’t been able to before. “Maybe she’ll make friends. Maybe she’ll sing a song, maybe she’ll say ‘Daddy’ again one day,” McPherson says. “Those are my goals. Those are the things that matter to parents.”
Read the original article on People
