For years, Jameson struggled with a number of challenges including not being able to walk or speak. Until he was 5 years old, his doctors didn’t have a name for what was happening to him. Then, he was diagnosed with Bachmann-Bupp Syndrome (BABS), an ultra-rare neurodevelopmental disorder caused by mutations in the ODC1 gene. With only about 20 known cases worldwide, treatment options for Jameson were extremely limited.
Jameson’s mom, Kayla, soon connected with Drs. Andre Bachmann and Caleb Bupp, who suggested he start taking DFMO, a drug originally developed for African Sleeping Sickness. Within weeks, Kayla recalled seeing improvements. “Developmental progress. Slow, incremental improvements. More understanding. More communication,” she said. “He’s in fifth grade now. He uses sign language, an augmentative and alternative communication device, and some words — and he loves saying ‘hi’ to everyone. He loves remote control cars, swinging, bike rides, and cruising the wide aisles at Lowe’s in his wheelchair like he owns the place.”
Every Cure is partnering with Drs. Bachmann and Bupp, at Michigan State University and Corewell Health respectively, to help advance BABS research, find more patients that could benefit from this treatment, and navigate access and regulatory pathways so more patients can receive this treatment. “Every Cure is… opening doors that we never would have been able to crack open,” said Dr. Bupp. “It’s a hopeful and exciting time for all of us and, more importantly, our patients.”
(Photo courtesy Jameson’s family)
More information: Scientists explore new treatment of drug for rare disorder (Michigan State University)
Learn more about Jameson’s family’s story.