We have been talking about stem cells for decades now. And often with excessive tones, both for better and for worse. The therapeutic opportunities offered by these undifferentiated cells, progenitors of all the tissues of our organism, are in fact so evident as to be almost obvious. But as always in medicine, moving from an intuition to a safe and effective therapy takes time, a lot of scientific work, and billions in investments. Without it, we risk a wild west that puts the health of the sick at risk, who are often rightly willing to do anything for the hope of an opportunity for treatment. And unfortunately, despite the promises of unscrupulous clinics and self-styled luminaries, the stem-based therapies that have completed the approval process in Europe and in our country are still very few, and limited to specific areas of medicine.
The first therapeutic application of stem cells is almost fifty years old: it is the transplant of hematopoietic stem cells, commonly known as marrow transplant. It is used for the treatment of various tumors and hematological pathologies, and can use cells collected from a donor or, but only in the treatment of some blood tumors, from the patient himself.
The first marrow transplants date back to the end of the 1960s, and therefore it is now a more than tested procedure, used both as a form of immunotherapy (the stem cells taken from a donor help to attack and destroy the patient’s tumor cells), and in in the field of regenerative medicine, to replace the patient’s marrow cells when they are not functioning properly. In all these cases, the therapy is regulated as a transplant in our country, and therefore follows a different procedure from that required for the approval of advanced therapy medicines, which instead includes treatments in which stem cells are manipulated to modify the their biological characteristics and obtain a therapeutic effect. Of this second type of stem cell-based therapies, as we were saying, there are currently very few that have completed the approval process in Europe and Italy.
In fact, there are only two advanced stem cell-based therapies available in Europe. One is a pride of Made in Italy: Holoclar, a tissue engineering product developed by Holostem of Modena, which uses autologous stem cells to reconstruct the epithelium covering the surface of the cornea in patients with severe ocular burns, and which has also been the first stem cell-based therapy ever approved in the West. The second is Alofisel, a cell therapy based on adult mesenchymal stem cells, indicated for the treatment of perianal fistulas in patients with Crohn’s disease which, although it was approved by the EMA in 2018, is currently not reimbursed by the healthcare system in Italy. National.
“This is the situation in Europe – confirms Francesca Ceradini, scientific director of the Advanced Therapies Observatory – while in the United States things go differently, because they have less stringent rules on the use of stem cells, which means that in American territory there are many more cell therapies or tissue engineering products approved. In many cases they are treatments based on hematopoietic stem cells taken from umbilical cords, which in Europe are not yet considered for clinical use as the therapeutic effects are still unclear. Finally, it is now known that in the United States – but also in Europe – there is a dangerous flourishing of private clinics that infuse stem cells with procedures and therapeutic indications that have never actually been approved”.
The risks of health tourism
In short, the two large pharmaceutical markets on the planet move on different tracks in this area. The more stringent European rules represent in some way a brake on the approval of new stem cell-based drugs and therapies. But at the same time, they are a guarantee of safety for patients.
by Letizia Gabaglio
“When you have a relative with a very serious illness, the temptation to turn to anyone who offers hope is understandable – concludes Ceradini – Everyone obviously decides for themselves, but it is best to at least get all the information possible before deciding, from official sources and reliable. We must absolutely be wary of therapies that promise to cure any disease with stem cells. And even in the case of approved therapies, being treated outside the EU could mean spending a lot of money for the treatment, for moving abroad at least for the duration of the therapy, and dealing with any side effects which can also be very serious. Furthermore, treatment with advanced therapies requires a follow-up of several years and if the therapy carried out abroad is not authorized in Italy it becomes difficult for the patient to be followed adequately in her country. This is why we, as the Advanced Therapies Observatory, warn patients and families against health tourism and have created an “anti-scam” handbook on stem cell-based therapies to help all citizens make informed decisions”.