We sat down for an interview with Professor Botond Roska, MD PhD, one of the world’s leading experts in the study of vision and the retina and last year’s recipient of the Körber Prize, to discuss the ground-breaking therapy he and his team have developed and already successfully used to return some vision to a handful of blind patients.
Can you tell us about the exact nature of your research for which you received the Körber Award?
It is sometimes difficult to know exactly what you get an award for, but I think I received it for two distinct but related scientific pursuits: firstly, my research on understanding vision, and secondly, for developing a treatment called optogenetic vision restoration therapy, through which a completely blind person can be sensitized to light, allowing them to see again.
FactProfessor Botond Roska, MD PhD, is a biomedical researcher and one of the foremost experts on vision, the retina, and treating diseases that cause blindness. He is a founding director of the Institute for Molecular and Clinical Ophthalmology Basel (IOB) in Switzerland and a Professor at the University of Basel. In 2019, he was awarded the Order of Saint Stephen, the highest national honor bestowed by Hungary, as well as the Louis-Jeantet Prize for Medicine. In 2020, he won the Körber European Science Prize for his research on a gene therapy that could be utilized to restore some vision to the fully blind, which he successfully implemented with his team for the first time this May.
What exactly does this procedure you developed entail?
It is a so-called two-component therapy. We inject a gene therapy vector into the eye, which is a small, virus-like particle with a DNA that encodes a light-sensitive protein. We target this protein at some elements of the blind retina. The vector also has a goggle that records the world, and then projects a picture onto the retina in a very specific way, in a particular color.
How effective is it now, and how effective can it potentially become?
The therapy restores a certain amount of vision, but not full sight. Based on the few patients we have had so far, the first of whom we recently published a paper about, the therapy allows patients to recognize objects, but it does not enable them to read, for instance.
However, there is a lot of room to improve. This is only the very first step, the very first optogenetic therapy.
Never before has a gene therapy been used to return some of a completely blind person’s sight. We will never be able to restore perfect vision, but we will improve quite a bit.
What kinds of blindness or visual impairment can it help or cure?
It is useful in cases when someone is fully blind as a result of photoreceptor dysfunction, but their retina is still connected to their brain via an intact optic nerve.
How profound an effect do you think being able to heal people’s vision at a large scale might have on society?
Blindness is one of the conditions that people name as the worst affliction to have. In fact, in a recent survey conducted in the U.S., participants named it as the worst condition they could suffer from out of a whole host of common health problems, ranking it above cancer, Alzheimer’s, and other truly devastating diseases.
It is a major problem for society, partly because we humans are highly visual, but especially since we rely increasingly heavily on visual cues in our daily lives as a result of technological development.
Our whole life is spent looking at phones and computers. Particularly during the pandemic, we could have almost no social interactions or opportunities to work without these, so that blind people were essentially cut off from the world apart from what little interaction they could have with it through hearing.
Therefore, we hope that such therapies, once they become widely available – since they are in the clinical trial phase at the moment – will improve many people’s lives tremendously.
Where do you think your research might lead you in the future?
There are three main directions. The first one is simple; as we discussed, we need to keep researching and improving optogenetic vision therapies, expanding them to target other cell types in the retina.
The second one is to do with the fact that if the optic nerve is missing, we cannot provide any therapies at the moment. This is something we are working on at my institute together with researcher Dániel Hillier, who is leading these efforts. We aim to find ways of restoring vision when there is no optic nerve.
The third is that most visual impairment is partial blindness, in which the method we developed cannot be used, so we are interested in looking into very large diseases affecting a lot more people. We want to try to slow down the degeneration or restore more vision to those with partial sight loss.
Why did you choose to dedicate your career to understanding human vision at the hardware level?
It was a chain of random events. I did not plan to be a vision researcher. I just wanted to understand things. When I finished my medical school, I was quite sure I did not want to treat patients for a living, I was more interested in understanding the human body. I decided to go into research, and I met someone who was researching the retina. I started my research in this field, and came up against an increasing number of questions that I found fascinating.
I went from topic to topic, physiology, virology, the molecular biology of the eye, then towards the end of my graduate studies, I read a paper that said it was possible to make cells light-sensitive using molecules form other organisms. At that time, I understood the retina quite well, and thought that I could combine my knowledge with these findings to try to design a therapy. My lab and myself are both interested in gaining scientific insight as well as designing therapies, and that is the path I plan to continue on for the rest of my career.
What is the worst and best part of the publicity you’ve received as a result of your success?
The best part is certainly that blind people can become informed that we are working on a therapy, which can provide them with some hope. It is also important for my institute, which can more easily recruit brilliant scientific minds who will come up with even better therapies down the line.
On the other hand, while there is nothing really bad about publicity per se, it can sometimes be time-consuming. Our paper about our first patient came out in May, and it has been downloaded around 90,000 times, with just about every country in the world reporting on our findings. And of course, all of the press outlets in all these countries bombarded our inboxes with requests. We were basically paralyzed for almost a month.
As someone who is called upon with increasing frequency to explain your cutting edge research, how do you approach the issue of science communication?
Communication is very important. It is part of our lives. In Switzerland specifically, it is taken very seriously, and it is part of our jobs as researchers to explain things to the public. We also have professionals to help us navigate the world of public relations.
The difficulty today is finding the balance between our work and its communication to the world. Because our research reaches a tremendous number of people extremely quickly thanks to the global nature of social media and other platforms, we get a lot of requests for interviews and articles. The difficulty for us is balancing our responsibility to inform the public of our progress with actually making some. Sometimes, it feels very much like we are in the eye of the storm.
Many people consider you a likely future Nobel laureate. Do you believe you might receive the award?
I don’t think about any of that. It does not occupy any of my time or of my imagination. At this moment, there are still great challenges ahead of us if we want to make the therapies as good as they can be. That is what I concentrate on. We also need to focus on innovating and coming up with new tools to help realize our scientific vision. This is also a costly and time-consuming endeavor.
What would you tell aspiring scientists, how can they best succeed in their scientific endeavors?
I think that the key is to come to science with an incredible desire to solve interesting problems. I never wanted to be successful in the public eye, just accomplished at solving scientific questions. For some reason, some scientists are more present in the media, and some of them win prizes, while others win prizes and choose not to have a public presence. Often, the most brilliant and prolific scientists do not even win prizes.
The key is to really be interested, and put an immense amount of energy into it. The main determinant of scientific success is how much energy and time one is willing to invest in their research, and how obsessed they are with understanding the world around them.
Photos by Dr Roska’s Office, Institute for Molecular and Clinical Ophthalmology Basel