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NewsFor the first time, a randomized controlled trial is testing whether a nasal spray can treat the root cause of cerebral palsy in young children. Dompé's Galassia trial is evaluating cenegermin — a nerve growth factor already FDA-approved as an eye drop — delivered intranasally to about 60 children ages 2–6 with spastic CP across Italy. There are currently no proven treatments that address the underlying brain injury in CP.
For the first time, a randomized controlled trial is testing whether a nasal spray can treat the root cause of cerebral palsy (CP) in young children. Dompé has enrolled its first patient in the Galassia phase 2 trial, which is evaluating intranasal cenegermin — a recombinant nerve growth factor (NGF) already FDA-approved as an eye drop for a rare corneal disease — in children with spastic CP. Currently, no treatments exist that address the underlying brain injury driving CP-related disability.
The science behind the approach is compelling: NGF is a naturally occurring protein critical to the growth, survival, and repair of developing nerve cells (its discoverers won the 1986 Nobel Prize). Dompé's intranasal formulation is designed to bypass the blood-brain barrier by targeting olfactory neurons, delivering the therapy non-invasively to the central nervous system.
By the Numbers:
Why it matters: CP affects millions of children worldwide, and families have long had no options beyond symptom management. If cenegermin proves safe and effective via the intranasal route, it could represent the first therapy to meaningfully alter the disease's course — a potential breakthrough for one of the most common causes of childhood disability.
