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“Encouraging” results from a study with an experimental drug offer hope to patients with Niemann-Pick disease type C1 (NPC1), a fatal neurologic disease that affects children and adolescents.
Symptoms of NPC1 result from cholesterol building in brain cells. The drug VTS-270 showed signs of improving cholesterol metabolism in neurons. After treatment, a molecule derived from cholesterol metabolism in neurons had increased, and 2 proteins indicative of brain injury had decreased.
In a phase 1/2a clinical trial, 14 participants received the experimental drug once a month for 12 to 18 months. Another 3 participants received the drug every 2 weeks for 18 months. After observing that the drug was safe and well tolerated, the researchers increased dosing for all participants. Their progress was compared with that of 21 participants in a previous study of NPC1.
The researchers also evaluated the drug’s effectiveness using a neurologic severity score (higher scores indicated more severe disease effects). Participants treated with VTS-270 had lower scores in cognition, speech, and mobility, indicating that the drug can stabilize or slow disease progression.
No one was observed to have serious adverse outcomes, but earlier studies had shown that the treatment carries the risk for hearing loss. After treatment in this study, participants, most of whom had already lost some hearing due to the disease, had further loss, for which they compensated with hearing aids.
“Encouraging” results from a study with an experimental drug offer hope to patients with Niemann-Pick disease type C1 (NPC1), a fatal neurologic disease that affects children and adolescents.
Symptoms of NPC1 result from cholesterol building in brain cells. The drug VTS-270 showed signs of improving cholesterol metabolism in neurons. After treatment, a molecule derived from cholesterol metabolism in neurons had increased, and 2 proteins indicative of brain injury had decreased.
In a phase 1/2a clinical trial, 14 participants received the experimental drug once a month for 12 to 18 months. Another 3 participants received the drug every 2 weeks for 18 months. After observing that the drug was safe and well tolerated, the researchers increased dosing for all participants. Their progress was compared with that of 21 participants in a previous study of NPC1.
The researchers also evaluated the drug’s effectiveness using a neurologic severity score (higher scores indicated more severe disease effects). Participants treated with VTS-270 had lower scores in cognition, speech, and mobility, indicating that the drug can stabilize or slow disease progression.
No one was observed to have serious adverse outcomes, but earlier studies had shown that the treatment carries the risk for hearing loss. After treatment in this study, participants, most of whom had already lost some hearing due to the disease, had further loss, for which they compensated with hearing aids.
“Encouraging” results from a study with an experimental drug offer hope to patients with Niemann-Pick disease type C1 (NPC1), a fatal neurologic disease that affects children and adolescents.
Symptoms of NPC1 result from cholesterol building in brain cells. The drug VTS-270 showed signs of improving cholesterol metabolism in neurons. After treatment, a molecule derived from cholesterol metabolism in neurons had increased, and 2 proteins indicative of brain injury had decreased.
In a phase 1/2a clinical trial, 14 participants received the experimental drug once a month for 12 to 18 months. Another 3 participants received the drug every 2 weeks for 18 months. After observing that the drug was safe and well tolerated, the researchers increased dosing for all participants. Their progress was compared with that of 21 participants in a previous study of NPC1.
The researchers also evaluated the drug’s effectiveness using a neurologic severity score (higher scores indicated more severe disease effects). Participants treated with VTS-270 had lower scores in cognition, speech, and mobility, indicating that the drug can stabilize or slow disease progression.
No one was observed to have serious adverse outcomes, but earlier studies had shown that the treatment carries the risk for hearing loss. After treatment in this study, participants, most of whom had already lost some hearing due to the disease, had further loss, for which they compensated with hearing aids.