In it’s simplest form, gene therapy involves replacing disease-causing variants of genes with healthy ones, in order to reduce or eliminate the symptoms of the condition. “Simple” in this context is extremely deceptive, however, as the process is extremely complex and occasionally dangerous.
At least, it was in the early days of the field. That’s why human gene therapy trials had to pass extensive and stringent review processes before they could be approved. In May, however, the US National Institute of Health ruled that the field of gene therapy had progressed far enough to streamline the review process. Now, only protocols with unusually high risk will need to pass a second layer of scrutiny before they can be tested on patients.
This is good news. A lot of rare genetic diseases strike during childhood, are severe or fatal, and before the possibility of gene therapy, had no effective treatments. Since new treatments can take decades to develop, anything that speed up the process, while protecting safety of trial participants, is a big step forward.
More information on these types of conditions is available from the National Organization for Rare Disorders. You can also check out my book, Gene Therapy, for more info on unusual conditions and the development of a new field of medical science.
Do you know someone with a rare disorder, or someone who’s participated in a medial trial? How do you feel about medical research, and the time it takes to develop treatments? Share your stories in the comments.