In Drug Trial, Parents Weigh Life and Loss

[WSJ/MSN] —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.”

The National Institutes of Health launched the trial two years ago to test cyclodextrin in children with Niemann-Pick Type C—a cholesterol metabolism disorder known as NPC that has been diagnosed in about 200 people in the U.S., including Andrew. The trial, chronicled by The Wall Street Journal, initially tests the drug’s safety.

All the children participating will likely lose some hearing, said Forbes D. Porter, the NIH doctor running this part of the drug trial. The higher the dose, the greater the risk, researchers found. But animals with NPC that were given cyclodextrin lived significantly longer than those untreated. For the parents of the 14 patients in the trial, every infusion requires balancing the possibility of extending life against the near-certainty of a side effect that may hamper their children’s few remaining years.

The views, opinions and positions expressed by these authors and blogs are theirs and do not necessarily represent that of the Bioethics Research Library and Kennedy Institute of Ethics or Georgetown University.