Gene Therapy Breakthrough Has Restored Hearing in Children

Gene Therapy Breakthrough

China has successfully used gene therapy to rewrite the chapters of young children born with genetic hearing loss. This groundbreaking treatment is the first time that gene therapy has successfully restored hearing, and the first recipients of it have been children.

Of all the things we take for granted, our 5 senses top the list. Imagine being born without the gift of hearing, unable to enjoy the beauty of music or the liveliness of laughter. Imagine living your life in complete silence and missing out even on the gentle swoosh of wind blowing through trees. 

4 in every 1000 children are born with complete hearing loss and 20% lose their sense of hearing by the tender age of 12 owing to illnesses or trauma. This brilliant advancement has reached heights nobody thought it would.

“Any improvement in a patient’s hearing is a complete victory. It is remarkable if we can do anything to help patients reduce hearing loss. We understand this is a first step in their clinical research and also a very important step,” mentioned Lawrence Lustig, a medical expert at Columbia University in the United States.

Gene Therapy for Hearing loss

Gene therapy works by replacing a defective or missing gene with a functional and healthy gene added externally into the cells of the patient. The study of gene therapy is used to treat both acquired and inherited genetic disorders.

The gene therapy breakthrough from China is effective in individuals who have defects in a specific protein known as otoferlin. Otoferlin plays an important role in the inner part of the ear which contains almost 16,000 hair cells. 

These hair cells, which look like a comb, vibrate upon receiving different frequencies of sound and send a signal to the brain which helps in perceiving and processing sound. Each hair cell is tuned to a different frequency, pertaining to 16,000 different frequencies, which allows us to differentiate between the wide range of sounds we hear from our environment. 

Without otoferlin, these hair cells cannot produce the chemicals needed to convey auditory information to our brains, leaving those affected in a world of complete silence, unable to perceive the rich tapestry of sound that surrounds us.

The new treatment adds a functional version of the defective otoferlin gene to patients, enabling them to regain their ability to hear. While the treatment is only done for these rare cases, many are confident that this breakthrough could be the start of many.

YIYI’S MIRACLE: A 5-YEAR-OLD’S HEARING RESTORED THROUGH GENE THERAPY BREAKTHROUGH

Of the couple of children who have undergone this groundbreaking gene therapy, Yiyi’s story shines the brightest. Yiyi was born with a complete genetic hearing loss and had been heavily reliant on an electronic hearing device, but her journey represents the transformative power of this innovative treatment.

Once Yiyi’s parents heard about the new treatment, they enrolled her in gene therapy. Doctors used a virus to replace the DNA cells in Yiyi’s inner ear and it resulted in a miraculous recovery of the otoferlin protein. 

In less than a month, Yiyi could hear for the first time. Once relying on her friends to wake her up, Yiyi can now hear the bell chime indicating the end of nap time. The young girl can also hear the sound of her mother’s voice without relying on lip-reading or electronic hearing devices. 

Is Gene Therapy on the Rise?

Gene therapy is the next step in medicine. Between 1989 and 2015 there were 2335 gene therapy trials either completed or in the drug development pipeline worldwide. And now, there are over 5000. This means that the amount of gene therapy research has more than doubled in the last 8 years from the last 26 consecutive years before that.

China became the first country in history to approve the commercial production of gene therapy by creating Gendicine, the first approved gene therapy product ever, and a therapy that has been successful in treating cancer. Now, the current $9 billion market is expected to reach $24 billion by 2028.