By Rick Shepherd, Communications Manager, RMHC of Central Ohio
Imagine being just five-years old and already a star in the medical world. Gideon Griffiths of Bellefontaine, Ohio will go down in history as the first child to have received the first-ever dose of an FDA approved gene therapy for Duchenne muscular dystrophy at Nationwide Children’s Hospital. DMD is a severe form of muscular dystrophy that leads to muscle weakness and atrophy. Muscle weakness may affect the skeletal muscle and the heart muscle. DMD typically affects boys and is progressive so over time, children with DMD develop problems with walking, breathing and heart function.
This new therapy, called ELEVIDYS (SRP-9001), was invented, developed, tested, and patented at Nationwide Children’s Hospital in Columbus where Gideon went to get the first infusion just over a month ago. To be well-rested and ready for this historic procedure, Gideon and his parents stayed across the street from the hospital at the Ronald McDonald House.
“I reached out to Nationwide Children’s because, through research, I had realized they had a specialty clinic just for DMD,” Gideon’s mom Erin told us. “When we were given his diagnosis, we knew we would just be learning how to care for our kiddo and advocate for him the best we could. However, as we read what DMD was, it really broke our hearts. We researched all we could and continue to research and keep up with as must as we can each day and just made a promise right there to try and do all we can to help Gideon enjoy life.”
Nationwide Children’s Hospital isn’t new to Jonathan and Erin Griffiths. They have a large family with several of their children who see specialists at NCH regularly. This time was different. Erin and Jonathan would need to stay at the Ronald McDonald House for the first time with Gideon because his procedure was going to be very early in the morning and having to go through the procedure right after a long car ride wouldn’t be feasible. But the Griffiths were already familiar with Ronald McDonald House Charities of Central Ohio too. “Before our stay, we had been involved with some fundraisers for the Ronald McDonald House with pop tabs through area schools and donating to school fundraising,” Erin said. “We know what it is like to make the drive several days a week and to have kiddos in the hospital. We loved the idea of helping families like ours where we can.”
Gideon was very impressed with the House during his family’s stay. Erin said, “The staff was super friendly and ready for us. Gideon was made to feel so special! The walk to our room was so excited getting to look around and see everything. Gideon loved the elevator that was a spaceship!”
Erin told us having the House just a short walk from the hospital on such an important morning for Gideon… and important for all kids who have this type of muscular dystrophy… made the journey much less stressful. “With the therapy being so new and none of us knowing 100% what to expect our nerves were already too much,” Erin explained. “The fact that the staff was able to get a room in such short notice was amazing… the staff all together was so helpful. They are so very friendly, and they make everything easy because they walk you through it all. There is so much available.” she added.
Two of the first eight gene therapies approved by the FDA were invented by Nationwide Children’s. “This is a meaningful day not only for these first families, but for families battling DMD everywhere,” said Gideon’s doctor, Jerry Mendell who’s also the principal investigator in the Center for Gene Therapy at the Abigail Wexner Research Institute, and co-inventor of ELEVIDYS. “It is thrilling to arrive at this moment of getting treatment to a patient population that has waited so long for more hope.”
Though Gideon may have been the first patient to receive this therapy, the Ronald McDonald House is ready to receive more families of children like him who will be receiving this therapy as they travel from around the world to get these life-saving infusions and get the rest they need before their big day.
Editor’s Note: Gideon will be joined by his mom and dad as guests of honor during the luncheon at Friday’s Sporting Clays fundraiser for RMHC of Central Ohio at Mad River Sportsman’s Club in Bellefontaine.