Gene Therapy Makes a Debut
Explore this issue:
June 2015Dr. Staecker is also the investigational leader of the Novartis-sponsored CGF166 trial, the first to offer gene therapy to adult patients with hearing loss. The protocol was developed by GenVec, a Maryland biotech firm, and involves introducing a gene known as Atoh1 into the inner ear. Atoh1 has been shown in animal models to trigger the formation of inner ear sensory hair cells (PloS One 2014;9:e102077). The hope is that in humans, supporting cells in the inner ear will absorb Atoh1 and transform into functioning hair cells that could mitigate or even reverse hearing loss, Dr. Staecker said.
Although positive anecdotal results have appeared in the lay press, “it’s really too soon to tell” how the four patients who have received the gene therapy at his center ultimately will fare, he said. “We plan on going back to the FDA in the fall with our clinical data, and we anticipate some positive further developments at that time.”
As for the overall direction of otologic drug and gene therapy, Dr. Staecker echoed the importance of pursuing a very focused approach. “Our animal models of tinnitus and hearing loss show that these conditions are extremely heterogeneous and pose very different challenges,” he said. “That’s why we need to develop targeted treatments for different aspects of inner ear disease.”
Disclosure: Dr. Staecker is a member of the surgical advisory board for MedEl Gmbh, and has received grant support from Novartis for animal studies related to gene therapy for inner ear disease.
David Bronstein is a freelance medical journalist based in New Jersey.