Taking Science Into Their Own Hands

In the race to treat rare disease, families are leading the charge to find cures themselves

- ALEXIS KAYSER

WHEN CASEY MCPHERSON became a father, his purpose became clear. He needed to raise his daughter, Rose, to be loving, courageous, healthy and strong. He needed to protect her.

So when the pediatric neurologist told McPherson that Rose had a rare genetic disease—so uncommon that it didn’t even have a name—parental instinct kicked in. And when the doctor said: “There’s nothing we can do,” that instinct sharpened.

“Rose was being attacked, but there wasn’t a monster for me to kill or a car for me to throw myself in front of,” McPherson told Newsweek. “It was just one little nucleotide in the gene. And if that’s the monster for me to kill, then that’s the monster I’m going after.”

Rose, now 9, and McPherson, 46, fight against that “monster” disease in little ways each day. Their home in a suburb of Austin, Texas, is “Rosie-proofed,” as McPherson said. She sleeps in a special bed equipped with barriers to keep her from wandering off, and a video camera plus oxygen and pulse monitors scan for signs of seizures in the night. Rose doesn’t have the restraint of a neurotypical 9-year-old, so chemicals and electronics are kept behind three levels of locks. She takes seizure medication twice a day, and goes to a private school for children with special needs (paid for out of pocket, because there was a 15-year waiting list for any sort of financial assistance).

Before, McPherson was known as the frontman of the alternative rock bands Alpha Rev and Flying Colors. After Rose’s diagnosis in 2016, he took on a new role as a founder, launching the nonprofit To Cure a Rose Foundation, the contract research organization Everlum Bio and now the public benefit corporation AlphaRose Therapeutics. All three seek to find and fund cures for children with exceedingly rare genetic diseases, like Rose’s.

McPherson is among a rising number of parents, spouses and patients seeking cures for rare diseases. Many of them are not scientists or doctors.