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Scientists from around the world are now able to help children born with inherited deafness to hear thanks to gene therapy.
According to a study published Wednesday, January 24, scientists successfully restored hearing in five of six kids treated in China thanks to the technology, Knewz.com has learned.
On Tuesday, the Children's Hospital of Philadelphia announced similar success in an 11-year-old boy. And Chinese researchers produced a study with similar results in two other children earlier this month.
“Gene therapy for hearing loss is something that we physicians and scientists in the world of hearing loss have been working toward for over 20 years, and it is finally here,” said John A. Germiller, MD, PhD, an attending surgeon and Director of Clinical Research in the Division of Otolaryngology at CHOP, and Associate Professor at Perelman School of Medicine at the University of Pennsylvania. “While the gene therapy we performed in our patient was to correct an abnormality in one, very rare gene, these studies may open the door for future use for some of the over 150 other genes that cause childhood hearing loss.”
According to The Associated Press, the experimental treatments target only one rare condition, however.
Still, scientists hope that learning more from the technology will allow them to restore hearing in children with other forms of deafness caused by genes.
According to the World Health Organization, 34 million kids have deafness or hearing loss, and 60 percent of the cases are due to genetics.
“No treatment could reverse hearing loss … That’s why we were always trying to develop a therapy,” said Zheng-Yi Chen of Boston’s Mass Eye and Ear, a senior author of the study published Wednesday in the journal The Lancet, according to AP. “We couldn’t be more happy or excited about the results.”
For the study, the scientists enrolled six children in the program that involves AAV1-hOTOF gene therapy. They captured the patients' progress in videos over 26 weeks.
One video shows a baby who couldn't hear at all before the treatment appear to hear a doctor speaking to her, according to AP. Another clip shows a little girl saying "I love you" back to her father, mother, grandmother and sister 13 weeks after her treatment.
The study was done at Fudan University in Shanghai and was co-led by Dr. Yilai Shu.
The scientists were unsure why the gene therapy didn't work in one of the patients. But they said the other five now hear approximately 60 percent to 70 percent of the normal levels. They noted that there were no negative results from the therapy.
According to AP, preliminary results from other studies have been positive as well.
New York's Regeneron Pharmaceuticals announced in October that a child under the age of 2 showed improvement after being treated with Decibel Therapeutics for six weeks.
Meanwhile, the Philadelphia hospital noted that its patient, Aissam Dam of Spain, began hearing sounds after being treated in October. The sounds are muffled, but he can hear his father's voice and cars driving by, according Germiller, the leader of the study there.
“It was a dramatic improvement,” Germiller said. “His hearing is improved from a state of complete and profound deafness with no sound at all to the level of mild to moderate hearing loss, which you can say is a mild disability. And that’s very exciting for us and for everyone. ”
Though there is still plenty of work to do, the scientists are encouraged by the early results.
“This is real proof showing gene therapy is working,” Chen said. “It opens up the whole field.”
