When Yilai Shu was training to be an otolaryngologist in Shanghai, in the mid-2000s, he often met parents with congenitally deaf kids.
“They always asked me, ‘Do you have any drugs to treat our kids?’ said Shu, who is hearing and a professor at Fudan University in China. “That’s what really inspired me to think about developing a drug.”
Decades later, Shu is closer than ever to such a drug. In October, he presented extraordinary data at a conference: His team had successfully enabled hearing in four congenitally deaf children using gene therapy. The work is the culmination of decades of genetic research and has spurred companies like Regeneron and Eli Lilly to swoop in and back other groups developing similar treatments.
The hearing gains are modest and the technology is currently limited to a type of deafness that only affects 1% to 8% of deaf people globally, but some experts suggest that gene therapy could eventually apply to as many as half of all congenitally deaf people. These treatments would have massive implications for the 12,000 kids born with hearing loss in the United States every year.
“This has been the dream of our field for 30-plus years to come up with a molecular treatment to give people real, natural hearing, that they were originally intended to have,” said Larry Lustig, a hearing otolaryngologist at Columbia University who is leading a Regeneron-backed trial showing promising results using gene therapy to treat deafness. “This is going to be transformative.”
But this transformative technology faces a problem: not all deaf people want it.
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