According to CNBC, Judy and Courtney Stecker are trying to secure treatment for their son Wheeler, who was diagnosed with a form of Batten disease at four weeks old and is now losing his vision. The family says a gene therapy developed for CLN3 Batten disease could help, but Wheeler has not received it, and Neela Therapeutics is discussing with the FDA how to design a late-stage trial and treat additional patients.
The article says Amicus Therapeutics previously pursued the therapy after acquiring rights from Nationwide Children’s Hospital, but the FDA told the company in late 2021 that its proposed late-stage study was not sufficient and required a five-year trial with at least 50 patients, including some on placebo. John Crowley, then Amicus CEO and now head of Biotechnology Innovation Organization, said the requirements were too costly and that the system has failed children with rare diseases. Amicus returned the gene therapy to Nationwide Children’s in 2024.
Neela’s chief scientific officer, Kathrin Meyer, said the therapy showed remarkable disease stabilization in three of four treated patients, including one who can read and see colors more than six years after treatment. Wheeler may not qualify for Neela’s upcoming clinical trial because he takes miglustat, so his mother has pushed for expanded access, though the company has been reluctant because other patients may also be waiting.
Kikyo
