text_1: GENE THERAPY MIGHT BE THE BEST, AND PERHAPS ONLY, CHANCE AT CURING BRAIN DISEASES
text_2: This in-depth story takes a look at the work of scientists who are trying to repair gene-deficient mice using “gene therapy.” These researchers hope to one day apply these still-experimental treatments to people who suffer from a variety of serious neurological conditions, but the focus in this story is mostly on a rare fatal condition known as Canavan disease. The heart-rending narratives that dot this account make for vivid reading, and the story does an admirable job of reflecting the hope and enthusiasm of some of the scientists on the front lines of this work. It offers readable explanations of how these researchers are modifying gene delivery strategies to overcome tragic problems uncovered by earlier clinical studies that led to a wholesale halt in work with humans. But the bottom line here is that nearly all of the promising research of the present has been conducted in mice, making the enthusiasm that permeates this story premature. Instead of deeply discussing the challenges in moving from animal data into medical interventions, the story spends more time on the growing financial interest among investors. The first quote in the story is from a venture capitalist, for example. While it’s important to discuss the biotech buzz surrounding gene therapy research, it’s also important to provide clarity on how challenging it will be to turn the research into actual treatments, even when experiments are amply funded. Genetic diseases of the brain are difficult to treat. If gene therapy offers an avenue for intervention, it would offer hope where there currently is little.