Mention of gnvc partnered w/Novartis trial pri.org
Dr. Lawrence Lustig, professor and chair of the department of otolaryngology at Columbia University Medical Center, is currently recruiting patients for a Novartis-sponsored clinical trial to find out whether his gene therapy will work on humans. The study involves injecting patients’ inner ears with a harmless virus. The virus is stocked with a gene essential to the development of sound-sensing “hair cells” in the cochlea, in the hope that the introduced gene will stimulate the growth of new hair cells and, eventually, restore some hearing capacity.
“If we could re-grow the hair cells of the inner ear of humans, there's no question that the hearing would be better than it would with an implant,” Lustig says. “It was known for years that birds can regenerate the hair cells. ... It’s really taken us a long time to figure out all the molecular steps that occur to allow us to sort of rewire what we do to our own inner ear to allow us to regrow hair cells in mammals.”
Dr. Lustig’s technique is aimed at patients who have lost their hearing due to years of loud noise or music, or the toxic effects of certain drugs. But a different genetic therapy, still being tested in animals, aims to treat the ears of those who have genetic hearing loss. In one experiment earlier this year, led by otolaryngologist Jeffrey Holt, mice with genetically induced hearing loss were able to regain partial hearing after treatment.
“What we're doing is really trying to fix cells that are broken,” Holt says, “These are the sensory cells that are not functioning properly. And if we can introduce the correct DNA sequence, we can make those cells functional again. We've demonstrated that with one gene in particular, and it works quite well for that gene. Now we're trying to tackle some of the other forms of genetic deafness.” |
