text_1: In a First, Gene Therapy Halts a Fatal Brain Disease
text_2: This story is one of two that we are reviewing (the other is from the Associated Press) that focuses on a recent paper published in the New England Journal of Medicine. At issue is a novel gene therapy used to limit or eliminate symptoms related to the disease adrenoleukodystrophy (ALD). The New York Times story does a good job of describing everything that led up to the study, does a fair job of describing the study itself (and its results), and highlights the potential price tag of this treatment. However, the story doesn’t make clear how far this treatment is removed from widespread clinical use or the outstanding questions that remain about the treatment — such as the fact that we don’t know if the benefits are temporary. Left untreated, ALD often leads to death within 1-10 years of diagnosis. And there are precious few treatment options available. What’s more, the primary treatment, bone marrow transplantation, relies on the availability of a donor — and poses significant health risks of its own. All this makes new treatment options incredibly important for ALD patients and their loved ones. However, the fact that we are talking about the life and well-being of patients — usually children — also makes it incredibly important to place the work in context. Limitations, availability and unanswered questions are always key points worth making, and especially so when many parents are eager for information about something that may affect the health and well-being of their children.