If all goes according to his plans, Professor Douglas Melton (Chicago, 70 years old) is about to revolutionize the world of type 1 diabetes. His research could free the more than nine million people who suffer from the disease from insulin injections. Illness. Unlike type 2, which is lifestyle-related, this is a dysfunction with no known causes. The discoveries of Melton, a biologist and co-director of the Harvard Stem Cell Research Institute, have earned him the third edition of the Abarca Prize, the International Medical Sciences Award honoring Dr. Juan Abarca, sponsored by the HM Foundation. He accepted the award last night at the Ritz Hotel in Madrid, where this interview took place a few hours earlier.
Questions. I believe she began researching diabetes because she had a son who suffered from the disease.
Answer. My son Sam was six months old and was diagnosed with type 1 diabetes, which I didn’t know about. A diabetic has to inject insulin and check blood sugar, and you can imagine how difficult this is for a baby. My wife told me: You’re a scientist, why don’t you do something? Until then, I researched how frogs develop and decided to start looking for a cure for diabetes.
Q Frogs?
R. I’ve been interested in them since I was a child. I was wondering how a frog egg becomes a frog. This is called developmental biology.
Q And he gave up his studies.
R. Yes, any parent would do that. If something happens to your child, try to fix it.
Q How is Sam now?
R. Well, he studies at MIT and has degrees in applied mathematics and physics.
Q Are you still taking insulin?
R. Yes.
Q By when do you have to do it?
R. Good question, because when he sees awards like this or reads about my research, he wonders when he will benefit from all this.
Q What is your answer?
R. It’s a lot closer than I might have thought a while ago. I think in a few years. The clinical trials are going very well and everything indicates that this could be a new drug for people with type 1 diabetes.
Q In the studies, did you get patients to stop insulin?
R. Some patients have not injected insulin for a year. And the other subjects in the study follow the same path, they need less and less amount.
Q How is your treatment going?
R. People with type 1 diabetes cannot produce insulin. And you need this hormone to harness the energy from food. Without glucose, cells cannot absorb nutrition from glucose. That’s why you have to inject it. With the new treatment, we ask ourselves: Why inject insulin and then measure blood sugar levels when there are cells in the pancreas that do both? They measure sugar and produce the exact amount of insulin. During the treatment, we create the insulin-producing cells that the patient does not have, so they have their own factory.
Q Do we know why some people are unable to produce it?
R. We don’t know, and the number is growing. In North America and Europe there are already three million. We know that it is not the result of a single gene. But I don’t work in prevention. There are many diseases whose origins we do not understand, such as Alzheimer’s. I would like to know the causes, but in the meantime I want a cure.
Q What path led you to this healing?
R. The idea is very simple. There are some cells that the patient is missing and we knew that there are stem cells that can form any part of the body. So the challenge was to tell a cell to become a cell that produces that type, rather than bone, muscle or blood. I had no idea how to do it and I had no good ideas. It was simply persistence: studying how the pancreas works and using that information to try things out in mice. If I started over now it would be a lot faster because I did a lot of stupid tests that didn’t work.
Melton, a few hours before the Abarca Prize for Medical Sciences was awarded. Jaime Villanueva
Q And what was it like changing your field of study? What doors did you knock on to get off the frogs and start diabetes?
R. I was very lucky because I was a professor at Harvard. I had very good students and was therefore able to attract funding. It wasn’t something I thought up at home and did in my garage. I believe that at least 50 students were involved in the research during this time. So it would be wrong to say that I did that. There is a team behind it.
Q Let’s assume that your treatment becomes generalized in a few years. What is your next step?
R. I didn’t think much about it. I would say the first thing I would do is treat my children [otra hija de Melton también tiene diabetes]. And we would have a great party. Then I would think about a way to freeze the cells. Now we have to administer the living cells to the patient. But it would be great if, when a patient goes to a clinic in Spain, the doctor had these frozen cells available and could inject them directly.
Q Would that make the treatment cheaper? Because I feel that it will generally be expensive.
R. I’m not an economist, but I would say the price will be high initially. But since there are so many patients, it becomes cheaper. You also need to keep in mind that this is a one-time expense that can be a bargain compared to using insulin for life. In any case, as with other cutting-edge technologies, it will decline over time after its introduction.
Q Type 2 diabetes is a different disease and drug treatment is done differently. Can cell replacement help those who suffer from it in any way?
R. Type 2 diabetes is related but different. The body needs insulin, but cannot respond to it adequately. I think one possibility is that these patients don’t have enough producer cells. It is therefore possible that the treatment could be used at some point. However, I want to emphasize that many people with type 2 diabetes can benefit from improved diet and exercise. Somewhat selfishly, I don’t give this illness the attention I should; I really focus on Type 1. Every morning when I wake up I think about her. This morning I thought of a new experiment.
Q Can you tell me?
R. I would like to use genetic modifications so that the cells are not rejected by the immune system. Now our cells tell the body they are being transferred to that they are a problem because they do not belong to them and therefore it needs immunosuppression to tolerate them. If you are in your mother, if you are a fetus, she should reject you because you are expressing cells from your father. Why do not you do it? That’s an interesting question and one I was thinking about this morning.
Q What impact might their advances have on the treatment of other diseases?
R. There are several, not many, that have to do with missing cells. The best example is Parkinson’s. There are cells in the central part of the brain that produce dopamine, but they are failing. If you could convert a stem cell into a dopamine-producing cell and insert it into the brain—which isn’t easy—there could be a cure.
Q And what do you think can be achieved with stem cells in the more distant future, in 30 or 40 years?
R. Our bodies are constantly creating cells that become muscles, skin, or blood. That’s why when we donate blood, we don’t live in a deficit for the rest of our lives. I think that during this time we will know how to stimulate the cells to regenerate. We assume that aging is inevitable, but perhaps it is not, and thanks to this tissue regeneration we can live to be 80 years old in perfect health.
Q Would this open the door to virtual immortality?
R. Maybe it was possible, but it’s not interesting. I don’t think anyone wants to be immortal. But if you’ve seen your elders age, you’ve seen how brutal the end of life can be. Healthy aging is a realistic goal.
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