Last Updated Jun 1, 2009 7:31 PM EDT
The poor folks in gene therapy haven't had it easy. Their approach has yet to gain FDA approval, so there's no regulatory precedent. Toxicity issues -- including the much-publicized death of Jesse Gelsinger in 1999 -- have tarnished the technology's reputation. Neither of these factors is particularly appealing to investors.
Yet the concept of replacing a mutated gene with a functioning one, thus potentially curing debilitating genetic conditions with a single injection, is nothing short of miraculous.
At the ASGT meeting, Maria Grazia Roncarolo of the San Raffaele Telethon Institute for Gene Therapy in Italy presented an update from her ongoing clinical trial in children with ADA-SCID, also known as "bubble boy" disease. The condition essentially deprives children of an immune system and is rapidly fatal without treatment. Yet the 15 children treated with gene therapy in Roncarolo's study are alive and well - some as long as eight years after their procedure.
Roncarolo lamented that "industry is not really interested in [funding] these types of studies." ADA-SCID is a rare disorder, and the gene therapy treatment only costs about $100,000 -- most of which is attributable to the hospital stay following the procedure.
In contrast, the only available drug to treat ADA-SCID -- Enzon Pharmaceuticals' Adagen (pegadamase bovine) -- must be taken weekly for life and costs between $250,000 and $500,000 per year. (No, that's not a typo. How do you people sleep at night? I suppose by knowing you're saving children's lives... but still, half a million dollars a year?)
ASGT President David Bodine said the gene therapy field is largely dependent on limited funding from government and nonprofit groups. Industry has backed gene therapy approaches to some broad indications like cancer, but the rare genetic diseases just don't offer much profit potential.
The pharmaceutical industry is a business. But is there ever a case where a medical advance is so important that it must be pursued, regardless of the return on investment? Maybe not. Maybe it isn't the drug industry's responsibility to fund gene therapy for rare diseases... but then whose responsibility is it?