STAT+: Obscure vertigo drug could bring rare hope for patients with devastating neuron disease

A patient community that has endured a series of crushing setbacks may soon get help from a modified version of an old French vertigo pill.

Feb 1, 2024 - 18:00
STAT+: Obscure vertigo drug could bring rare hope for patients with devastating neuron disease

About a decade ago, Tatiana Bremova-Ertl’s graduate adviser was studying an obscure, 1950s-era French vertigo drug, probing its effects on people with balance disorders, when she thought of another, very sick group of patients. 

A graduate student and medical resident at the German Center for Vertigo and Balance Disorders, in Munich, Bremova-Ertl often saw patients with Niemann-Pick Type C, a rare, genetic disease that slowly kills neurons. 

NPC has a range of manifestations. When symptoms appear in early childhood, it is often fatal before adulthood. When it manifests  later, it can be milder. But it’s always degenerative and leads to a cluster of challenges: cognitive decline, difficulty with speech and swallowing, enlarged liver, low muscle tone, and, notably, difficulty with balance and muscle control. Researchers and a fervent group of parents were working to develop medicines, but little had yet worked. 

Continue to STAT+ to read the full story…

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