A recent article in the Wall Street Journal (WSJ) posed this question, “Death or Deaf?” amid ongoing trials for an experimental drug, cyclodextrin, designed to cure children of a deadly genetic disease. Cyclodextrin, a sugar-based substance, might possibly extend the lives of children with Niemann –Pick Type C, or NPC, a cholesterol metabolism disorder, such as in the case of 15-year old Andrew Marella from Maryland. However, use of this drug may leave him deaf.
The National Institutes of Health launched this trial about two years ago to test cyclodextrin in children with NPC, which has been diagnosed in over 200 cases in the US.
At the present state of drug testing, researchers say that unfortunately, almost all of the children participating in this drug trial will lose some hearing as a side effect. In addition, there is a positive correlation between the dose and the risk, putting children on high doses of cyclodextrin at a much greater risk of losing their hearing beyond repair.
However, researchers maintain, the drug is effective and animals given the drug in the early stages of drug testing significantly outlived those untreated. Therefore, this trade-off is a weighty one, rife with implications for many parents. For them, weighing their options between extending their children’s lives against the near-certainty of significant side effects is still full of hesitation.
Dr. King one of the researching involved in the study, notes the importance of hearing and asserts that it enables people to connect to the world and those around them. Indeed, Helen Keller noted that while blindness cuts people off from things, deafness cuts them off from people. Daily activities we take for granted can be greatly impaired by deafness as children miraculously live through adolescence and transition into adulthood; such as using telephones to communicate, being aware that someone is talking, or hearing alarms.
While low doses cause hearing loss that can be combated with hearing aids, higher doses of the drug will likely yield damage beyond repair. And while research is ongoing to find treatments that will mitigate hearing loss in the future, these high doses are still necessary based on observations taken from animal testing.
Ultimately, however, in an interview with the WSJ, Andrew’s father states that the family is willing to take the risk. He deems it a “logical trade-off.” And the FDA and NIH seem to agree. They have allowed the drug trial to proceed despite the risks of hearing loss since they claim that the benefits of cyclodextrin outweigh the risks. As Karla Cook who comments on this article in the WSJ writes, “I have worn hearing aids since I was almost six years old, practically a life time. I would say that hearing aids and or sign languages is a small price to pay for life. “