Drug for rare disease disappoints in key trial

Science | AAAS | 11/9/2018 | Staff
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A treatment for Niemann-Pick type C (NPC), an extremely rare and ultimately fatal neurodegenerative disease, performed no differently than placebo in a pivotal trial in 56 children and youths, its corporate sponsor announced on Tuesday. Perplexingly, though, the disease did not progress in either the treatment or placebo groups during the 1-year study, the company said. Normally, the condition, a result of impaired cholesterol metabolism, inexorably worsens, causing loss of balance, difficulty swallowing, seizures, and cognitive disabilities.

The drug, VTS-270, a doughnut-shaped sugar molecule called a cyclodextrin, “did not show a statistically significant separation from placebo,” Steven Romano, Mallinckrodt Pharmaceuticals’s executive vice present and chief scientific officer told investors on a conference call on Tuesday. “But importantly, neither did [patients in the active or placebo arms of the trial] show disease progression as would have been anticipated in the neurodegenerative condition over 52 weeks of observation.” The drug was given by spinal injection into the cerebrospinal fluid, which circulates to the brain.

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Evans - Family - Practitioner - Leeds - UK

Will Evans, a family practitioner in Leeds, U.K., is chair of the charity Niemann-Pick UK. His 11-year-old son, Sam, was in both an earlier trial to determine safe doses of the drug and in the most recent trial. “Many families, ours included, have invested several precious years in this trial—taking our children fortnightly to trial sites to undergo invasive procedures—and across the community, so much hope has been hung on the outcomes of this trial,” Evans says. “For these families to receive this news via the reporting of a financial call on Twitter is incredibly disappointing and hurtful. It has caused enormous upset for many families.”

“It’s devastating news,” adds Chris Hempel of Reno, Nevada, whose twin daughters, Addi and Cassi, 14, have received spinal injections of...
(Excerpt) Read more at: Science | AAAS
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