Family Raises $4 Million for Urgent Gene Therapy Trial for Kids
UPDATE: A family in Fort Collins, Colorado, is urgently seeking to raise $4 million to fund groundbreaking gene therapy research aimed at helping children with the rare FRRS1L mutation. This mutation severely limits physical movement while preserving cognitive function, leaving affected children trapped in bodies they cannot control.
Everly Green, an 8-year-old girl, has faced a devastating decline in her abilities since her diagnosis. After initially developing normally, Everly began experiencing seizures at age two, leading to the loss of motor skills and the eventual need for a feeding tube. Today, she can only communicate through eye movements and a screen. Her mother, Chrissy Green, emphasizes the urgent need for effective treatment, stating, “These kids are in there, they want to play like other kids, they just can’t move.”
The family, alongside a small community of affected children, is spearheading efforts through the Finding Hope for FRRS1L foundation to gather funds for the next phase of drug development. With only a few dozen children diagnosed worldwide, pharmaceutical companies have shown little interest in investing, leaving families to fund research independently. If successful, the planned clinical trial could start in September 2026.
The gene therapy aims to replace the faulty FRRS1L gene with a healthy version using a harmless virus. While previous animal studies have shown promising results, including significant recovery in treated mice, the path to human trials is fraught with challenges. The foundation must navigate not only the complexities of FDA approval but also the potential financial hurdles with insurance coverage for such expensive treatments.
Chrissy Green shared the emotional weight of the situation, recalling when doctors advised the family to make the most of the time they had left with Everly. Instead of giving in to despair, they connected with other families globally, leading to collaborative research efforts. “We saw major recovery in the animals, so we’re really hopeful for our kids,” she stated.
Despite the daunting obstacles, the Greens are determined to push forward. The next steps include testing for side effects and finding a manufacturer capable of developing the therapy. “All the diseases can kind of help each other move forward,” Chrissy Green believes, highlighting the broader implications of their work for future treatments.
As they work towards the $4 million goal, the family hopes to inspire others to join their cause. “We are not looking for profit; we just want to help our kids,” said Chrissy. This urgent initiative represents not only a chance for Everly but also a beacon of hope for children facing similar challenges worldwide.
For updates on their fundraising efforts and to support the cause, visit the Finding Hope for FRRS1L website. The clock is ticking, and every moment counts for children like Everly.
