They were the most future future. The horizon of hope. The new advancing, yes, but slowly, it was thought. But here is the acceleration: within months the so-called cell therapies have gone from being complex procedures suitable for very few patients with niche diseases, to being one of the main streams of the war on cancer, and not only, on which Big Pharma points already today. Super complex therapies, which involve taking a certain number of immune system cells, the T lymphocytes, of a patient, and genetically modifying them in order to make them able to fight the tumor and the subsequent reinfusion in the patient. A technique called CarT and travels alongside other techniques of other cell therapies against rare diseases, things that have been reported in recent years relating to a few cured, people in advanced stages of serious diseases. After all, experimental tests of the new world: decades will pass before it becomes reality in hospitals, it was said., And instead, a Big like Bristol Myers Squibb points to them as the hot front of the next few years to the audience of investors called to decide on the goodness of ‘ company, opens an ad hoc plant in Leiden, the Netherlands, already has two FDA approved treatments, against lymphoma, and against multiple myeloma. What happened? Have cell therapies really become today? We asked Giovanni Caforio, president and CEO of BMS in Princenton.
Doctor Caforio. You have indicated cell therapies as a development front for your company, ready to replace drugs with expiring patents on the market. Yet, these are techniques that are nowadays intended for a few very serious patients. And many feel that they will remain so for a long time.
“It’s true: for a long time there have been skeptics about cell therapies and the role that a company like ours could have in this sector. But Bms is a biopharmaceutical company, biotechnologies are the heart of our work. On the one hand, we have the resources, size and capabilities of a great global leader. On the other hand, we are focused on revolutionary innovations, on drugs that represent a breakthrough in therapies. And cell therapy is coming to clinical practice very quickly. So it can happen that we are the first to have two products already registered by the FDA this year. Both in the case of multiple myeloma and lymphoma we find efficacy in patients who, as often happens in oncology, have already used various lines of treatment and are therefore in an advanced stage of the disease in which there are no alternatives.
But, in a recently concluded research, for example, we have seen that cell therapy is more effective against large B cell lymphoma than standard treatments (chemotherapy + autograft) even in less advanced stages of the disease. With this we are arriving at larger populations of less seriously ill patients. Not only that, given these results we are already working on a new generation of cell therapies, which can use two techniques and hit two targets in the same treatment to have even greater efficacy. In short, it is only the beginning, but precisely in a phase like this, the investment capacity of a big player company is needed. In recent years, cell therapy has been especially important for small biotechs who have experimented with the techniques and seen which ones could be promising. Today is the time for clinical development, to verify their effectiveness and bring them to the patient’s bed ”.
Multiple myeloma, the first CAR-T therapy arrives
by Tiziana Moriconi
Two therapies approved by the FDA in the USA, what times are there for Europe and Italy?
“Short times, we hope. One of our drugs has received a marketing authorization from the European Agency for multiple myeloma patients who have already been treated with three previous therapies which unfortunately have not halted the advance of the cancer. And the other is being studied by the Agency “.
Everything you ever wanted to know about CAR-T
by Anna Lisa Bonfranceschi
Will cell therapies change the natural history of cancer?
“The potential is there. And it requires us to work in two directions together. The first is to bring these treatments to an earlier stage of the disease and to do so we must, first of all, minimize the many secondary effects that they entail; we are working on it and certainly the new generations of products will be tolerated much better. When it comes to being able to treat less severe patients, then we will need to have combinations of therapies in hand that can attack the disease more vigorously, aiming for higher survival rates. Second, we need to develop cell therapies that are easier and faster to produce. Now the matter is very complex, it takes weeks of work on the patient’s cells. If we are successful on both fronts, cell therapies will be just as important in the future as immunoncology has been in the last 10 years ”.
Oncology is once again the terrain of great innovations, as it has been in the last 50 years. Because? Should we also expect breakthrough therapies for other diseases that have a similar, if not greater, impact on people than cancer?
“We have focused enormously on cancer therapies. But now, thanks to the developments in genetics, we know we can broaden our gaze. Let’s think of a therapeutic area such as cardiovascular: until now it has been considered less interesting, and certainly not for the number of people affected but because it was seen with the approach of basic medicine. Genetics has changed the cards; it has allowed us to identify genetically determined cardiomyopathies that can be treated with specific drugs for small populations of patients who have specific characteristics. It is another revolution thanks to which we will come to develop therapies for more targeted pathologies, such as heart failure for example, but also others, for which there were no effective answers. And such a picture also opens up for areas such as immunology, thanks to the great progress made in understanding how the immune system works, and neurology, thanks to the understanding of the causes of some degenerative diseases that paved the way for the study of effective therapies also against pathologies up to now substantially unresponsive such as Alzheimer’s or Parkinson’s ”.
And covid?
“Bms has no experience in researching vaccines. Therefore we have chosen a focused approach. And we are working, together with Rockefeller University in New York, to develop an antibody that could be used in people at risk to prevent the disease. But we are still in an early stage of development. And we’ll see “
What do you see for the future?
“I expect a lot from a whole new industry with huge potential for big diseases. It’s one that investigates protein homeostasis as a whole different way of attacking a target and developing a drug. Keep an eye on it. It will give us great. satisfactions “.
Health Festival, Giovanni Caforio: the therapies of the new millennium
.