New type of gene therapy could be “gateway” to treat deafness
New type of gene therapy could be “gateway” to treat deafness
MIT Technology Review reported that some deaf children in China can hear after gene therapy treatment. Its article highlighted a girl named Li “Yiyi” Xincheng, who was born entirely deaf. After she received a new type of gene therapy where a virus was used to add replacement DNA to Yiyi’s inner ear cells, she could begin hearing in less than a month in the treated ear. Her other ear has a cochlear implant.
Here is a video clip.
[Video clip showing Yiyi correctly answering questions from her mother, who covers her mouth while speaking] Credit: MIT Technology Review
This medical procedure was done at Fudan University in Shanghai and led by a surgeon named Yilai Shu. Shu said he has treated five deaf children and that four of them gained hearing abilities but one did not.
The article explained that the treatment applies only to one specific cause of deafness at birth, a defect in a gene that produces a protein called otoferlin. This condition is the case of around 1% to 3% of cases of deafness at birth around the world, so it is rare. But researchers say this treatment could be a “gateway” to develop more ways to treat various types of deafness. In the U.S. and Europe, there are at least three companies that are working on clinical trials to treat deaf patients with the otoferlin condition.
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Perspectives from Dr. Mirus and Dr. Burke
I’ve done interviews with Dr. IV Mirus to get a medical perspective and Dr. Teresa Blankmeyer Burke to get a bioethical perspective.
[Begin interviews]
Daily Moth (Alex): There seems to be a “successful” treatment of the OTOF gene in the ear through a virus. Can you explain how the science works to “fix” this?
Dr. IV Mirus, ER Doctor: The technology they use is called gene therapy. Gene therapy has been around and it’s something that is heavily researched. It is not only for deafness but it helps things like cystic fibrosis or sickle cell diseases. These are major illnesses that are caused by defects in only one gene. But it causes a multitude of ailments. So gene therapy potentially could help these people. But for — what gene therapy really is — it’s when you get a virus, a virus is a particle that has its own DNA or RNA. The virus’ DNA makes new viruses. So when it goes into a cell, the virus’ DNA and her DNA will combine together and then the cell will make a new strand of DNA to make more viruses. The cell will divide to make more viruses. That’s how a virus works. So the idea is what if a virus has its DNA removed and replaced with what we want. For example, the otoferlin gene, the “right” one, is put in there and it gives the virus, when it goes into a person, the ability to read the DNA and make a new kind of otoferlin protein. So it is using a virus to introduce a new kind of “blueprint” to make a different kind of protein. It is very complicated and it doesn’t always work. It really depends on what kind of genetic defects you are trying to correct. It may work for some people. It is a really new kind of technology. It is very expensive and most people cannot afford it. It is not a common treatment. It is being used for research purposes and it is being fine-tuned to improve in the future. For now.
My suspicion is that we may identify a way to fix a specific cause of deafness. Otoferlin is first and then what’s next? And so forth. It will happen slowly over time. Will that cause deaf people to reduce in size? That is a concern, right? It’s a big concern. That is the debate about gene therapy. Where do we as, not only medical professionals but as a society and community, establish boundaries on where you cannot cross with gene therapy?
[End Dr. Mirus interview, begin Dr. Burke interview]
Dr. Teresa Blankmeyer Burke, Philosophy Professor & Bioethicist:
The big picture is… when I started my work here at Gallaudet, my PhD was about this topic, genetic engineering. What would its impact be on the Deaf community? I thought that it would probably take about 20 to 25 years. I’ve been here at Gallaudet for 20 years now. So my prediction that it would take 20 years is accurate. What can the Deaf community do right now? Firstly, I think the most important thing is to get educated about this technology. What does it mean for the Deaf community? 1% to 3% is a small percentage but at the same time, this is the first step. What is next?
My concern is that people will find it easy to move forward without thinking about the impact on the Deaf community, on threatening sign languages, and on the sense of valuing the community itself. It will take time. If this starts to roll out, it will probably be 40, 50 years before we see a real impact on the number of children being born deaf. It will be much less. What would that mean for Deaf schools? We have seen the results of cochlear implants and immunizations. It’s reduced the number of Deaf schools and some have closed down. We see more deaf people who have cochlear implants who don’t sign. Their perspectives are different from those who have signing skills. Whatever is important, valued, and needed in the community is split in numbers between two groups. Less numbers means less attention from institutions, government, social services and NGOs, which all will impact this community. What I think is important is that we often have public meetings hosted by the FDA that oversees this process. We also have the NIH, the National Institutes of Health, which is a different government agency, that has a responsibility to have oversight over technologies and how they are being applied in treatments and clinical trials. All of that is run by our government agencies. We can tell them that we have more things to talk about, and that there is a threat to the existence of our community. That is important.
I predict that we’ll see the numbers of deaf children being born deaf and remaining deaf reduce in the future, in 50 years. My concern is whether we will have a critical mass, enough Deaf people to continue this community and continue this culture? Of course, many Deaf families will continue to have children and they will probably continue. But what about people like me? My family is all hearing. I don’t know. I don’t know. And I think the time is now for Deaf people to think through this scenario. What happens in the future?
[End interviews]
Alex: Thank you so much, Dr. Mirus and Dr. Burke, for your expertise. So this is something to ponder for all of us as we face a new frontier that could eventually lead to a drastic reduction in the number of deaf children that are born in countries that have access to advanced medical care.