“Last year we celebrated the 100 years of insulin, which was a real revolution because it transformed a disease that was deadly such as diabetes at its onset into a disease that is now chronic, degenerative, such as type 1 diabetes which, however, still steals 12 years of life expectancy from people, despite the best therapies we can put in place. We are now working hard for an insulin-free world. We would like November 14 “in the not too distant future” to no longer be the celebration of Diabetes day, but that it becomes the day we wake up and no longer need insulin. For type 1 diabetes, the most important achievements in this last year have been mainly related to regenerative medicine approaches “. A world free from insulin is the “dream” of Lorenzo Piemonti, director of the Diabetes Research Institute (Dri) and of the newly established Regenerative Medicine and Transplantation Unit of the Irccs San Raffaele hospital in Milan, and of the specialists who deal with the ‘sweet blood’ disease.
Lthe way to reach this goal could pass through what are called “living drugs”, he explains to time.news Salute. “Type 1 diabetes is characterized by the loss of insulin secreting function by the cells that produce it, which are destroyed by the immune system. After years of trying to rebuild these cells using organ donors as a source , in recent years a new technology has developed that allows to overcome the existing limitations, that is, not having enough donors to treat all patients with diabetes and necessarily having to use immunosuppression “, because the organ donor has a different system of express proteins and therefore rejection must be avoided.
“The big news started about 14 years ago in the laboratory, and arrived 6 years ago at the first attempts in humans. In the last year it has been possible to demonstrate that it is possible to obtain insulin independence and therefore recovery from diabetes, by implementing of cells that produce insulin – explains the expert – derived from pluripotent stem cells. It is a potential turning point, because the last mile is always the most difficult to travel. But today we have all the tools and the clinical preparation to apply the therapy to diabetes “.
This, remembers Piemonti, “was the year in which the transplantation of pancreatic islets was included in the ministerial guidelines in some disease conditions, which is a cell therapy for diabetes now standardized. On this basis, we began to fundamentally change the source of the cells. And an important result has been obtained: the first patient in the USA treated with these cells has become insulin-independent. similar can be extended to all patients with type 1 diabetes and also in a part of those with type 2 diabetes “.
Onimmunosoppressione, he continues, “there are more approaches that can be used: some are in the pipeline for the first clinical trials and presumably within a year we will also have results on this, but it is an area that still needs evidence of efficacy in humans. not only in animal models and in vitro. To overcome the need for immunosuppression, one strategy, for example, is to put these cells inside some ‘containers’ that prevent the immune system from recognizing them as foreign. Or they can be added or removed. in the cells some genes that allow the immune system to recognize it. It is as if the immune system had a barcode reader, like the ones we use in the supermarket, and in every cell there was a barcode. does not recognize that code, it attacks the cell. But today this code can be modified in order to deceive the immune system. n a cell and all those that will derive from it will bring the same modification “.
The time horizon for the promise of regenerative medicine to materialize? “It is difficult to define it – he reflects – but I believe that in the next 5 years we will have concluded the first phase of experimentation in humans, which will tell us how much this type of approach is actually able to cure. We will then move on to phase 2 studies- 3, with a greater number of people. It will also be understood who can be cured. And if these studies lead to the confirmation of the validity of the approach, then extending it to the population with diabetes will only be a question linked to the production capacity, but who has invested in this type of regenerative medicine model, it is already equipping itself. Because it is the most interesting horizon for the next few years: to be able to apply living drugs not only to some rare diseases, but also to pathologies that can have a greater impact “.
Subtraction the cost node. “Cell therapies in general still have very high costs. Those revenues on the market range from a few tens of thousands of euros to more than one million euros. At the moment, being rare indications, one can imagine spending a lot, but if we want to apply this type of treatment on a large scale just thinking that type 1 diabetics would be 200-300 thousand people, no small number. These aspects will be a bet, which depends on the success that these living drugs will demonstrate. We are investing in it “, he explains Piemonti. So much so that, he points out, “today we have decided to build a real clinic unit dedicated to regenerative medicine and transplantation, with the aim of implementing this type of approach. I believe it is a duty of the scientific community to start predicting that in the coming years we will have to deal with diseases treated more and more with this type of drugs that require a different ‘expertise’ than the traditional ones and also a different organizational model – observes the expert – Today we are used to having the patient’s bedside doctor, but when you work with these therapies there is also the stem cell biologist, the bioengineer, he is a much larger and multidisciplinary community. We strongly believe in this perspective. And if the therapy is successful without immunosuppression, in my opinion it will take little to start producing on a large scale “.
Science, Piemonti concludes, “may indicate that there is a way” to tackle a problem. The speed and the possibility of pursuing it depend on much wider choices, which have to do with economic models that support this mode indicated by science, and cultural models that do the same. It must be an alliance of all the actors, not just scientists or doctors, but the whole socio-cultural, political and economic system. Everyone must think in a way that allows in part to get out of the dynamic in which the disease is always chased, and you go to treat the patient in a phase in which you have already ‘lost the battle’, and instead begin to think for solve these diseases, preventing or treating them definitively. Removing that label of chronicity which today is becoming an unbearable burden for those with chronic disease – in psychological terms and quality of life – but also for health systems that no longer hold up today. This is the bet – the scientist remarks – behind this cultural transition of using regenerative therapies “.