How would you describe Advanced Therapies?
In short, I would say that they are the medicine of the future. Let me explain. The term Advanced Therapy is generally used as a synonym for stem cell therapies, and this is not true. Advanced Therapies are a much broader concept, because they include gene therapy, tissue engineering… What is certain is that it is this new medicine, these new treatments that have been under development at the preclinical level for a long time and which, in a very short time, have turned current medicine upside down. As far as the patient is concerned, these new therapies are currently offering treatment to pathologies that barely 5 or 10 years ago would not have had the possibility of a therapeutic alternative. It is true that we must be cautious and that we are at the beginning of an enormous development, but I believe that in a short time great strides have been made.
It is clear that great strides have been made, judging by the course of the NC1.
They are live medicinal products, due to which the conditions and requirements are much more special. It is important to have a little patience because when you implement a therapy in the National Health System, as in this case, it gives the feeling that we have already reached the solution. And I always try to be cautious. This is an important step, both from the scientific point of view and for the patient, but let us look at the case of organ transplants. We have been transplanting organs for more than 50 years and, unfortunately, they are still failing due to chronic rejection. Well, the case we are dealing with is a little bit the same, we are at the beginning of a new medicine. We have come a long way in transplantation, but we still have a long way to go because we still have a lot to learn. We have not yet learned how to avoid rejection.
In Advanced Therapies, we still have a lot to learn from these live medicinal products, both in gene therapy and in cell therapy and tissue engineering. There are still many aspects to be perfected, although we have come a long way.
What we can surely say is that we know it is a safe treatment for the patient. We have learned a lot about safety, we still have a lot to learn about how they work: how to preserve them, how to transport them so that they do not lose their biological characteristics, in other words, we still have those years we have given to organ transplantation to make it a reality. We know that, in terms of medicine, ten, fifteen years of development are very little. That is why we have to give that time and that margin to advanced therapies.
Today we talk about a kidney transplant, a heart transplant, a lung transplant, and we consider it a reality, but it has taken more than fifty years of studies. It seems that sometimes we ask Advanced Therapies for miracles, sometimes possibly due to the fault of the professionals themselves, who are perhaps not very cautious when it comes to issuing results. I always say that even from failures we learn, because we are aware that we are at the beginning. For the moment, we have learned something very good: that it is safe for the patient.
Being clear that the NC1 treatment is safe for patients is a big step forward from a clinical point of view….
Of course! And for us it is a success. For the patient it’s not as big a success as they would like it to be, but in clinical terms it’s a success. A few more years of development are now needed for this success to go hand in hand with greater efficacy. Logically, efficacy has been demonstrated, otherwise we would not be here, but probably not as much as we would all wish for. That is why we need a little bit more time for that safety to be combined with an efficacy that really translates to the patient in terms of a spectacular improvement. And we will get there. I know we will. I’m confident. If we know how to do things…
What does the medicinal product NC1 consist in?
It is an Advanced Therapy medicinal product, which is constituted, like any other medicinal product, by two parts. On the one hand, the active ingredient (which, in this case, are mesenchymal cells from the patient’s own bone marrow stroma; on the other, the excipient, which is made up of part of the patient’s plasma, so that the medicinal product itself is an autologous medicinal product, meaning, it is manufactured from the patient’s own blood and bone marrow, two of the patient’s own raw materials. Its entire development and indication at this time is targeted at the treatment of patients with traumatic spinal cord injury that is chronically established. Meaning, patients who are in a situation, from the neurological point of view, stable. There is no possibility of the patient being able to recover on their own, from the neurological point of view, as may occur in patients who are in an acute phase.