--:-- Sean Rameswaram: Something truly remarkable happened in Philadelphia recently. Here's a sack by this defense. Not the collapse of the Philadelphia Eagles, though, ouch. An 11-year-old Moroccan boy traveled to Philly via Spain for a procedure. Aysam Dam was born deaf, totally deaf, but he went to the Children's Hospital of Philadelphia for a clinical trial. So this will be where the gene therapy drug went to that tubing there. A new gene therapy offered something miraculous. It allowed ISOM to hear for the very first time. Yes, if you heard that, tell me again. That's going to be pretty cool. On Today Explained, we're going to hear all about it, but we're also going to ask whether deafness is something that needs to be treated in the first place.
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--:-- Sean Rameswaram: This is Today Explained. Antonio Regalado is a biotech reporter at MIT Technology Review. Not so long ago, he was contacted by some scientists in China who said they could make deaf kids hear for the very first time. And he had to know more. Well, they had pursued a gene therapy to reverse deafness, congenital or inborn deafness in children. And so they had been working on a gene therapy that would treat basically one type of inherited deafness, just one type. But it's really cool. And what was cool about the story is we got to interview the mother of a child who had the treatment and heard about the amazing results.
--:-- Antonio Regalado: A girl who couldn't hear, and then within a month of getting this gene therapy into her ear that was injected, she could hear out of the treated ear. I can tell you the story about a little girl named Yi Yi who lives in China and who is one of the kids that got this treatment. She's really cute. Her mother, Quinn, makes lots of videos about her and puts them on the Internet. And they're really remarkable videos that demonstrate that this girl who was born deaf can now hear her mother.
--:-- Antonio Regalado: Yi? Hmm? You heard the sound? I heard. My voice is not loud. Uh, not loud. A couple of years ago, Yi did get a cochlear implant in one ear. So that's a device that helps her hear out of that ear. But that kind of hearing is not that natural. It has a kind of a metallic sound. You don't hear details like the wind through the trees. So the family was glad to try gene therapy in Yi's other ear. And that worked.
--:-- Antonio Regalado: How many people have received this kind of gene therapy where they may have been deaf or hard of hearing and can now hear? Well, this is extremely new. The first cases we heard about were from China this fall. And at that time, that group had treated about 10 children and they were ready to report on the results in five. And then just recently, just a couple weeks ago, we heard about another case in the U.S. with similar results. An 11-year-old boy received the experimental treatment back in October. The boy who was born deaf in both ears now only has mild to moderate hearing loss in the ear that was treated.
--:-- Antonio Regalado: That we know of, six children so far treated in this way for deafness in the world, right? All these studies, they've just started to launch recently within the last year. And it happens that there's several groups doing it. So they're all in a bit of a race to be first and to demonstrate these incredible results. There's at least three teams doing this particular kind of gene therapy.
--:-- Antonio Regalado: We've been working in the field long enough to know we have come a long way. Now it's really a tipping point. So we are going to have some spectacular success as a field. And also, it's an incentive for us to work even harder. And what is this experience like for a child to go from not hearing to all of a sudden having this fifth sense?
--:-- Antonio Regalado: So I really wanted to know what her experience was. Now, this girl's only five or six, so it's hard to get, you know, even her mother has trouble getting her to express to her clearly exactly what it's like. But, you know, there are a few details that were compelling. For instance, they live in this apartment block in a noisy area of town, and the little girl has started to complain that she hears sound at night, right? It's too noisy at night, she says, which she wouldn't have said before because she wouldn't have heard the noise.
--:-- Antonio Regalado: Another story was, you know, when she went to school in her little grammar school, they would have a nap time, and when the nap time was over, a bell would ring, you know, a little bell, and say, nap time's over, kids. And before, Yi would, you know, her friends had to tell her that the bell had rung. But now she can hear it.
--:-- Antonio Regalado: Is her hearing at 100%, 50%, 25%? What? Well, of course, the doctors and scientists involved are measuring her hearing. And the way they expressed it to me was that her hearing is at about 60 to 65 percent so it close to normal speech. It's like having moderate hearing loss. Now it can differ at you know the frequency you know, high frequency or low frequency noises, but the amazing thing is that, you know, she would not have heard anything sitting in the movie theater with the loudest sound possible before. And now she can hear you know essentially a normal voice but she might not hear a whisper.
--:-- Antonio Regalado: Will her hearing continue to improve or is this it? How does it how does it work? I think that's one of the unknowns. Will it continue to improve? Um, maybe not. Uh, But it's an incredible success that you can hear natural sounds already. That's a big success. I don't think it's going to improve that much further, but that's my guess.
--:-- Antonio Regalado: Can you tell us what this gene therapy is doing? One of the cool things about writing this story is you learn about the ear. I don't really think about what's going on in my ear that much, but it's really amazingly cool little organ. Inside of it are these hair cells. They're not actually hairs. They just kind of look like hairs because they have these little extensions. And there's about 16,000 of these cells. And each one is sort of tuned to a different frequency.
--:-- Antonio Regalado: So the sounds that come into your ear, they vibrate these little hair-like structures. They vibrate them, and that information is turned into a chemical signal, and then an electrical signal, and goes to your brain, where you experience the sensation of hearing. Hi, hello. But it's actually because of these little wiggly antennas that you can hear it all.
--:-- Antonio Regalado: People have a mutation in a gene that makes a crucial part of that system. It's a molecule that is basically the chemical transmitter of the signal. And so in this form of hearing loss, the problem is people just don't have this chemical. And the gene therapy basically puts new DNA for this gene into people's ear cells. And so, with that copy of the gene, the cell can then make this molecule.
--:-- Antonio Regalado: And when people hear gene therapy, I think they think CRISPR, but this isn't CRISPR. This is not CRISPR. This is, yeah, you can think of gene therapy as adding a new gene, a whole copy of a gene. And in fact, this gene is pretty big. Whereas when people talk about CRISPR, that's gene editing. That's sort of, that's putting in some molecules that could change a gene. Often break a gene, which could also be useful.
--:-- Antonio Regalado: In this case, you have to add the gene back, and you really couldn't even do that with CRISPR at this point. So this is more classic gene therapy. And would this classic gene therapy be able to help any deaf person, or is it better suited to kids? This gene therapy would be suitable for only a small fraction of the people who are born deaf, because only 1% to 3% of those people nobody's quite sure because it's rare enough, have this particular genetic mutation.
--:-- Antonio Regalado: Like, there's lots of different genetic mutations that cause deafness. And some of them are much more difficult to deal with. This one happens to be the sort of most straightforward, the easiest one to deal with. And this would account for about one to three cases of inborn deafness or congenital deafness, not counting people who go deaf for other reasons.
--:-- Antonio Regalado: But you did mention that there's more trials going on. There's at least one in the United States as well. Does that bode well for the sort of spread of this treatment, the more wide application of it? I think what this does is it's going to inspire and excite scientists who are working on kind of harder gene therapies for other forms of deafness, right? There's going to be more money, more excitement, more interest now that they've done this.
--:-- Antonio Regalado: I should say that a lot of gene therapies, as miraculous as they are, they fail in the marketplace. And the reason they fail is that there's too few patients. These are for ultra rare diseases. So it's just, you can imagine, you have a company, a biotech company of 50 people, and there's only 50 patients in the world. So it doesn't make economic sense. So a lot of these gene therapies, as amazing as they are, they undergo a form of market failure. They actually can't be sold. Some of them are retired and patients can't get them.
--:-- Antonio Regalado: So with this one for hearing, we'll have to see. There's still – there's just not that many people who can benefit. It's interesting. So there's a bunch of competitors entering this market, which you say doesn't have, you know, super broad potential. Why are there so many competitors trying to get in on a small market? A lot of people think gene therapy is the future, and it may be. I mean, it is dramatic in its essence, right? I mean, you are changing or adding DNA to human cells. I mean, that is kind of profound because DNA is the molecule of all life.
--:-- Antonio Regalado: And now scientists can change it and add to it and engineer it. And they can treat all kinds of diseases that they couldn't treat before. So it's just exciting. And the technology makes it possible. And so that animates biotech companies. But for the scientists involved, it's just super exciting, not only from a technological point of view, but also from a medical point of view. Many of them are doctors, you know, to be able to cure someone, to be able to help someone in a disease that has never before.
--:-- Antonio Regalado: Like, there's no treatment for deafness using pharmaceuticals or pills. There is a cochlear implant that you can get, but there is no pill that you can swallow that will help restore hearing. None. If you're a doctor, that is something profound. So that is another reason to do it.
--:-- Sean Rameswaram: Can you hear me now? Good. Hello, my name is Teresa Lankmeyer-Burke, and I am full professor of philosophy at Gallaudet University. Gallaudet University is a bilingual institution with American Sign Language and English language in use for instruction, and we also have deaf and hard of hearing and hearing students.
--:-- Teresa Lankmeyer-Burke: Professor Blankmeyer-Burke is deaf herself. She signed this interview, and her responses were voiced by an American Sign Language interpreter. And my work in gene therapy has the potential to impact ethics and certainly the deaf community themselves. And my work, my research for the last 20 years has involved consultancy in any number of different groups, such as the United Nations, the World Federation of the Deaf, the National Association of the Deaf of this country, the NAD, and others.
--:-- Teresa Lankmeyer-Burke: So this is a topic that is my jam. Well, I'm so glad because I think when people read the news that science has advanced to the point that gene therapies can restore hearing for children who have never fully or even partially been able to hear anything, they think it's nothing short of a miracle. But I want to ask how you, someone for whom this is their jam, someone who thinks about these questions on a philosophical level, react to such news.
--:-- Teresa Lankmeyer-Burke: There are two stories that were reported in the New York Times last week about the 11-year-old boy from Morocco. There were also six children in China. One thing which we have seen from the reports, as well as a review of the study, is that these seven children taken together who had gene therapy are all still hard of hearing or deaf. And these children need support and access to a language.
--:-- Teresa Lankmeyer-Burke: As an ethicist, I think that it is hugely important for those children. Yes, gene therapy is taking place. From the perspective of the public, it's seen as a panacea, but that is not anywhere near the case. So I think it's the nature and expectation of people who don't have the lived experience within the deaf community to think, oh, it is miraculous. And I would also add that I am not against gene therapy in and of itself.
--:-- Teresa Lankmeyer-Burke: I think it's important for us to consider all of the potential impacts of this technology. One thing that's part of the history in the signing deaf community is the experience of miracle cures that are imposed upon us and a history of people who see a development. A recent example might be cochlear implants and how that can impact the lives of children.
--:-- Teresa Lankmeyer-Burke: These developments are of a concern in the deaf community because the priority, generally of deaf people and for a deaf child would be to say you're okay and that you do have a language. You have access to your language. Sometimes what happens with medical interventions and technologies is that you would have an issue of, say, treating a child with whatever that intervention might be, but there's very little attention given to what might happen to that child if that intervention or therapy doesn't work.
--:-- Teresa Lankmeyer-Burke: We see that within the deaf community, certainly. It is a source of concern because children need to have full access to language from the get-go, of course, for the purposes of learning. And in these scenarios, they don't. And they experience what is known as language deprivation. And language deprivation is a term of art that we see in the literature surrounding the deaf community.
--:-- Teresa Lankmeyer-Burke: And it is the notion that people who don't have access to language during the critical window of development or language acquisition will never really gain full access to a language. They might have the ability to communicate gesturally in some way, but as far as their critical thinking ability, their interpersonal ability, there are permanent deficits.
--:-- Teresa Lankmeyer-Burke: So if I'm understanding you correctly, for you it's less of a miracle, it's less of an end of a story and more of a midpoint where okay yes these children have had some hearing restored but how are they communicating are they still learning language?
--:-- Teresa Lankmeyer-Burke: And your chief concern here is sign language. For me, I think it's the most important thing that the deaf children get a language full stop. It's certainly nice for deaf children to have both, to be sure. Access to a signed language, I can use myself as an example, having grown up in a mainstreamed environment educationally, learning to speech read, use what hearing I had, and figuring out how to navigate the hearing world.
--:-- Teresa Lankmeyer-Burke: When I came to the sign language community, for the first time in my life, I noted that I had full access to a language and I was no longer playing guessing games. Of course, now when you're learning a new language, you are playing guessing games, but that all evened out. So the experience was actually understanding and understanding the feeling of not having to struggle to understand.
--:-- Teresa Lankmeyer-Burke: It was just there. So part of my question remains, for those children, maybe they will acquire spoken language, which is all well and good. But for other children, that might not happen. And we have to consider what would happen for every and all deaf children in this environment of gene therapy.
--:-- Teresa Lankmeyer-Burke: Whatever happens, I'm more concerned about shining a light of ethics on people who are deaf and my overriding concern about the decisions made about whether to administer gene therapy or not. I think that one thing that really warrants close attention is the word serious conditions.
--:-- Teresa Lankmeyer-Burke: This includes things such as sickle cell anemia, cystic fibrosis, Tay-Sachs, and other things. These conditions cause a tremendous amount of pain and suffering or could result in premature death. And this is the cohort that is always within the discussion of prioritization to receive gene therapies.
--:-- Teresa Lankmeyer-Burke: I think you're leaning towards something I want to ask you about, which is the simplest way to think about this treatment is as one that is curing deafness. And I imagine you don't think deafness is a thing that needs to be cured.
--:-- Teresa Lankmeyer-Burke: I think for people whose experience is within the hearing world, and they don't have an experience in a signing deaf context in that community, I think that it would be a matter of course that they would think they'd like to see this particular sensory disability cured. Cured.
--:-- Teresa Lankmeyer-Burke: At the same time, I think that it is important to note that we've had conversations about difference. I see the signing deaf community as a thriving cultural linguistic community that carries their own ethical norms, their own cultural norms, their own cultural contributions to art, to history, to any number of domains, the sciences.
--:-- Teresa Lankmeyer-Burke: This community is part of our human diversity. I am always concerned when I see a challenge or an attempt to dilute human diversity. I would think, yes, that people think it's easier. People who are hearing would think that it is easier to not be deaf.
--:-- Teresa Lankmeyer-Burke: But within the community, I know many people who are deaf, who have intergenerational deaf families, and they have purchase in a part of community with deep roots and a deep reverence for that experience and that community. I recognize that, of course, there are tensions between parental right to make a decision for their children and a community who wants to preserve itself.
--:-- Teresa Lankmeyer-Burke: And the parents' decisions ultimately impact the ability of that community to maintain itself and indeed thrive going forward. So where we are now is, well, I've been thinking about this problem for 20 years, and I still don't have an easy answer.
--:-- Teresa Lankmeyer-Burke: Yes, we need to support parents in making decisions for their children. However, at the same time, I think it's important that parents are fully informed before they make such a decision. And it's important for people to understand the value of a signing deaf community and what that brings to the world in terms of human diversity.
--:-- Sean Rameswaram: Our program today was produced by DC's Victoria Chamberlain. We were edited by Matthew Collette, fact-checked by Halima Shah, and Avishay Artsy. Engineered by Patrick Boyd, and hosted by Sean Rameswaram.
--:-- Sean Rameswaram: The rest of our team includes Amina Alsadi, Hadi Mawagdi, Amanda Llewellyn, Miles Bryan, Laura Bullard, and Jesse Alejandro Cottrell. We use music by Breakmaster Cylinder. Our executive producer is Miranda Kennedy. My co-host is Noelle King. I'm Sean Rameswaram. Today, Explained is distributed by WNYC. The show is a part of Vox, which is totally free. Thanks in part to contributions from our listeners. Join us at Vox.com slash give. And thank you.
