If everything 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 this condition from insulin injections. disease. Unlike type 2, which has to do with lifestyle, this is a dysfunction that has no known causes. The discoveries of Melton, 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 in honor of Dr. Juan Abarca, sponsored by the HM Foundation. He collected the award last night at the Ritz hotel in Madrid where, a few hours before, this interview took place.
Ask. I think she started researching diabetes because she had a son with the disease.
Answer. My son Sam was six months old and was diagnosed with type 1 diabetes, something I knew nothing about. A diabetic has to inject insulin and measure blood sugar, and you can imagine how difficult that is for a baby. My wife told me: you are a scientist, why don’t you do something? Until then I was researching how frogs develop and decided to change to look for a cure for diabetes.
Q. Frogs?
R. I have been interested in them since I was little. I was wondering how a frog egg knows to become a frog. This is what is called developmental biology.
Q. And he abandoned his study.
R. Yes, it’s what any parent would do. When something happens to your child, you try to fix it.
Q. How is Sam now?
R. Well, study in the M.I.T. and has graduated in applied mathematics and physics.
Q. Are you still taking insulin?
R. Yeah.
Q. Until when will 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 much 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 it could be a new medicine for people with type 1 diabetes.
Q. Have you gotten patients in the trials off insulin?
R. A couple of patients have not injected insulin for a year. And the other subjects in the study are going the same way, they need less and less quantity.
Q. How does your treatment work?
R. People with type 1 diabetes cannot create insulin. And you need this hormone to harness the energy from food. Cells cannot take nourishment from glucose without it. That’s why you have to inject it. With the new treatment we ask ourselves: why inject insulin and then measure the blood sugar level if there are cells in the pancreas that do both? They measure sugar and generate the precise amount of insulin. With the treatment, what we do is manufacture those insulin-producing cells that the patient does not have so that they have their own factory.
Q. Do we know why some people are not able to produce it?
R. We don’t know, and the number is growing. There are already three million in North America and Europe. We know that it is not the result of a single gene. But I don’t work in prevention. There are many diseases whose origin we do not understand, such as Alzheimer’s. I would like to know the causes, but in the meantime, what I want is a cure.
Q. What was the path that led you to that cure?
R. The idea is quite simple. There are some cells that the patient is missing and we knew that stem cells existed, which can form any part of the body. So the challenge was knowing how to tell a cell to become one that produces this type, and not bones, muscles or blood. I had no idea how to do it and I didn’t have any bright ideas. It was just persistence: studying how the pancreas works and trying things with mice using that information. If I started over now it would be much faster, because I did a lot of stupid tests that didn’t work.
Q. And how was the change in area of study? What doors did you knock on to leave the frogs and start diabetes?
R. I was very lucky, because I was a professor at Harvard. I had very good students and I was able to attract funding thanks to this. It wasn’t something I came up with at home and made in my garage. I believe that in all this time, at least 50 students have been involved in the research. So it would be wrong to say that I have done this. There is a team behind it.
Q. Suppose that in a few years your treatment becomes generalized. What is your next step?
R. I haven’t thought about it much. 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 live cells to the patient. But it would be great if when a patient went 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 sense that in principle it will be expensive.
R. I am not an economist, but I would say that the price at first will be high. However, as there are so many patients, it will become cheaper. You also have to keep in mind that it will be a one-time outlay, which compared to using insulin for the rest of your life can be a bargain. In any case, it will decrease, as happens with other cutting-edge technologies as time passes after its launch.
Q. Type 2 diabetes is a different disease and its pharmacological treatment goes through other means. Can cell replacement benefit those who suffer from it in some way?
R. Type 2 diabetes is related, but different. The body demands insulin, but cannot respond adequately to it. I think one possibility is that these patients do not have enough producer cells. So it would be possible that at some point the treatment could be used. But I would like to emphasize that many people with type 2 diabetes can benefit from improving their diet and exercising. Somewhat selfishly, I don’t pay the attention I probably should to this disease; I’m really focused on type 1. Every morning when I wake up I think about her. This morning a new experiment occurred to me.
Q. Can you tell me?
R. I want to use genetic modification so that the cells are not rejected by the immune system. Now, our cells tell the body they are transferred to that they are a problem, because they are not its own, so it needs immunosuppression to tolerate them. When you are inside your mother, when you are a fetus, she should reject you, because you express cells from your father. Why doesn’t she do it herself? It’s an interesting question, and it was what she was reflecting on this morning.
Q. What implications could their advances have on treatments for 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 make dopamine that fail. If you could convert a stem cell into one that makes dopamine, and put it in the brain, something not easy, there could be a cure.
Q. And in the more distant future, 30 or 40 years, what do you imagine can be achieved with stem cells?
R. Our body is constantly generating cells that become muscles, skin or blood. That’s why if we donate blood we don’t live the rest of our lives in deficit. I think that in that period of time we will know how to stimulate the cells to regenerate. We take it for granted that aging is inevitable, but perhaps it is not, and we can reach 80 years of age in perfect health thanks to this tissue regeneration.
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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