Deaf or Death? In Drug Trial, Parents Weigh Life vs. Hearing Loss
An experimental treatment could let children with a rare genetic disease live longer, but it may make them deaf
BETHESDA, Md.—While waiting for an infusion of a drug that might save his life, 15-year-old Andrew Marella gripped the controls of an NFL videogame, the hand-held version of a sport he played when he could still run without fear.
Andrew is in a clinical drug trial of cyclodextrin, a sugar-based substance that scientists hope will stop or slow the progress of a rare genetic disease that kills most patients by the time they are old enough to vote.
There is a good chance cyclodextrin will extend Andrew’s life. But his parents worry this will be the dose that leaves him deaf.
Families in the drug trial must decide whether to permit the higher doses of cyclodextrin that research shows might arrest the disease. Hearing loss is one side effect. “Deaf or death, what are our options?” said Andrea Marella, Andrew’s mother. “We have to keep moving forward.”