Hearing management

09 July 2015

Gene therapy for deafness moves a few steps closer

Gene therapy to improve hearing in those with genetic deafness has shown promise in a new trial - a treatment that could greatly improve the quality of life of children born with hearing impairments.

0

Gene therapy for deafness is moving closer to reality, with new research on Wednesday showing the technique for fixing faulty DNA can improve responses in mice with genetic hearing loss.

Separately, a clinical trial backed by Novartis is under way to help a different group of people who have lost their hearing through damage or disease.

After missteps in the late 1990s and early 2000's, when safety scares set back research, gene therapy is enjoying a renaissance, with positive clinical results recently in conditions ranging from blood diseases to blindness.

"We are somewhat late in the auditory field but I think we are getting there now," said Tobias Moser of the University Medical Center Gottingen, Germany, who was not involved in the new research. "It's an exciting time for gene therapy in hearing."

A key element in current optimism is the development of better and safer viral delivery systems for getting corrective genes into the body. In the case of deafness, this involves injecting a gene-carrying engineered virus into the inner ear.

Read: Leaving childhood deafness untreated can be dangerous

Tackling hearing loss

There are currently no approved disease-modifying treatments for disabling hearing loss, which affects some 360 million people, or 5 percent of the world's population, according to the World Health Organisation.

Much of the hearing loss in older people is noise-induced or age-related, but at least half of deafness that occurs before a baby learns to speak is caused by defects in one of more than 70 individual genes.

It is these infants Swiss and U.S. researchers hope to help, after showing that replacing a mutated gene improved the function of hair cells of the inner ear and partially restored hearing in deaf mice.

Scientists from the Ecole Polytechnique Federale de Lausanne and the Boston Children's Hospital, who reported their work in the journal Science Translational Medicine, tested hearing in newborn mutant mice by seeing how high they jumped when startled by a noise.

Read: Is your child hearing you?

TMC1 gene is key

The team focused on a gene called TMC1, which is a common cause of human genetic deafness, accounting for 4 to 8 percent of cases. But other forms of hereditary deafness could also be fixed using the same strategy.

Jeffrey Holt of Boston Children's said the technique still needed "tweaking" but he hopes clinical trials will start within five to 10 years.

Work at Novartis is more advanced, with the first patient treated last October in an early-stage clinical trial that will recruit 45 subjects in the United States, with results due in 2017. 

The Swiss company's product, acquired in a 2010 deal with GenVec worth up to $214 million, delivers a gene called Atoh1 that acts as a master switch for turning on the growth of inner ear hair cells that are central to hearing.

Novartis research head Mark Fishman describes it as a "spare parts" approach to fixing ageing-related frailty.

The process offers hope to adults whose hair cells have been damaged by excessive noise, disease or exposure to certain drugs, including some antibiotics. But it will not help the one to three babies per 1,000 born with severe genetic hearing loss in both ears.

"There are a big range of deafness types needing different approaches," said Moser.

Read more:

1.1 billion youths risk hearing loss due to loud music

Can hearing aids reduce forgetfulness?

What are the symptoms of hearing deficiency?

 

Read Health24’s Comments Policy

Comment on this story
0 comments
Comments have been closed for this article.

Ask the Expert

Hearing Expert

Francis Slabber is a Speech & Language Therapist and Audiologist who has owned and run The Hearing Clinic in Wynberg, Cape Town for the last 17 years. Francis and her team have extensive experience in fitting and supplying hearing aids as well as assistive living devices. Francis has served as the Western Cape Chairperson for the South African Association of Audiologists for three years and has given many talks on the topic of hearing loss and amplification. The Hearing Clinic has a special interest in adult and geriatric hearing impairment, hearing aid fittings and hearing rehabilitation.

Still have a question?

Get free advice from our panel of experts

The information provided does not constitute a diagnosis of your condition. You should consult a medical practitioner or other appropriate health care professional for a physical exmanication, diagnosis and formal advice. Health24 and the expert accept no responsibility or liability for any damage or personal harm you may suffer resulting from making use of this content.

* You must accept our condition

Forum Rules