Going Beyond Machines
Most patients with end-stage renal disease (ESRD) are consigned to life on dialysis. Most undergo in-center hemodialysis three times a week, greatly diminishing their quality of life. Moreover, dialysis patients have a relatively high mortality. In 2003, three-year survival of dialysis patients was 56%, according to the U.S. Renal Data System.
Researchers have come up with incremental approaches to improve the way dialysis is delivered, such as nocturnal home dialysis that patients undergo five to seven days a week. And, as highlighted in this issue (page 1), investigators are working to create portable and wearable (and potentially implantable) devices dubbed artificial kidneys. They have the potential to improve patients' quality of life by obviating the need to go to dialysis centers, but a commercially available artificial kidney is at least several years off.
I applaud the technological ingenuity and its potential benefits, but an artificial kidney will not be adequate to address
One strong candidate for that shift is gene therapy. I've long believed it will be the next truly great revolution in medicine. Imagine if clinicians had the ability to reverse kidney disease by simply injecting a gene that, for example, stimulates production of nephrons. To date, no FDA-approved gene therapy product exists; this promising treatment remains experimental. With the completion of a map of the human genome in 2003, however, it is likely just a matter of time before researchers master the secrets of human genes—beginning with how to turn them on and off—and figure out how to replace defective or nonfunctioning genes with those that function properly.
Gene therapy holds promise for a great many ailments that currently defy treatment. If we must prioritize, I say gene therapy for renal disease should top the list. Yes, we should look forward to the debut of artificial kidneys, but not forget about other promising modalities.