CAR-T looks like a new sports car model, but instead, is a new anti-tumor therapy. Born in the United States, it’s also being used at the Bambino Gesù Children’s Hospital in Rome. And it’s even been improved!

For some years now we’ve officially entered the era of oncological immunotherapy. What is it?

It’s a therapy that uses the same immune cells as the cancer patient to fight and defeat the tumor. The technique consists of extracting the cells of the patient’s immune system (the T lymphocytes) and reprogramming them genetically by inserting an antigen called Car (Chimeric Antigen Receptor) that enables them to recognize and attack the cancer cells again. In other words, no medication is needed because it’s the patient’s immune system itself that fights the disease.

Which tumors can be treated with Car-T?

To date, the tumors concerned are:

  • Acute Lymphoblastic Leukemia (ALL) B-Cell leukemia in children and young adults up to 25 years of age;
  • Diffuse Large B-cell Cell Lymphoma (DLBCL) in adults.

The most important thing to know is that this therapy, at the moment, can be used on those patients who have had experienced no improvement with chemotherapy treatments, and also after stem cell transplantation. In other words, those who have lost all other hope.

Fortunately, the number of people at this stage is not large, but it’s still a significant number and most importantly, they’re mostly children.

Before receiving treatment, patients must undergo a short course of chemotherapy, in order to eliminate T lymphocytes that are not able to fight the tumor and give “more space” to the new T lymphocytes; always coming from the patient but that are genetically modified and able to selectively recognize tumor cells. It’s as if you were receiving a transplant, using yourself as the donor. The new T lymphocytes are then simply infused via IV into the patient, and the treatment is effective — even if performed only once.

But what role does the “Bambino Gesù” Children’s Hospital play?

As we’ve stated earlier, a considerable number of patients are children, who often spend years at the hospital, undergoing continuous chemotherapy treatments and live in semi-insulated conditions to prevent infections, due to the suppression of their immune systems. The Bambino Gesù Children’s Hospital in Rome provides excellence in the care of children and has also been selected by the Ministry of Health as one of the first Italian hospitals to have adopted the therapy. However, Dr. Franco Locatelli, Director of the Department of Pediatric Onco-Ematology, Cellular and Gene Therapy at the hospital, did not just adopt the protocol, but he has committed both himself and his team to improve it, since some side effects such as cytokinic release syndrome (high fever, low blood pressure, respiratory difficulties and kidney failure — and above all, a 5% mortality rate) and neurotoxicity had been detected. To reduce these side effects, the Italian researchers added a so-called “suicidal” gene, which is activated in the case of non-response to drug therapies against cytokinic release syndrome or neurotoxicity, thus causing the elimination of the Car-T cells.

The Car-T cell treatment of acute B-Cell Lymphoblastic Leukemias and large B-Cell Lymphomas at the Bambino Gesù Hospital indicated important efficacy in the 15 patients treated, with remission rates above 80%.

This is a wonderful example of a Centre of Excellence that applies Precision and Personalized Medicine, because the treatment can only be performed on the same patient to whom T lymphocytes have been genetically modified.

What may happen in the future?

Studies are in progress for the use of the same technique on other tumors, such as Neuroblastoma (unfortunately, a widespread childhood brain tumor), chronic Lymphatic Leukemia, Mantle Cell Lymphoma and Multiple Myeloma (also known as Kahler’s disease), the latter amounts to about four thousand new cases each year and is a typical tumor of the elderly. Other studies will begin soon as immunotherapy is proving to be very promising and could potentially become a treatment for several forms of cancer.

I recommend that you watch the video relating to Medicine and Information on the cases treated at the Bambino Gesù Children’s Hospital, and listen to the passion that Dr. Locatelli uses when explaining the technique and the successes achieved. These are the people who should be rewarded every day, because every day they fight for our health!

This post is also available in: Italiano

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I've always been passionate about science and have a Degree in Biological Sciences and a PhD in Molecular and Cellular Biology. After six years of basic and applied research, I joined the company that provided the DNA sequencers that led Celera Genomics to complete the sequencing of the first human genome shortly before the same result was achieved by the "Human Genome Project" international public consortium. Subsequently, I became interested in human and animal diagnostics, and the development of molecular techniques from research-to-clinic. Science4Life represents the next stage of my personal journey, a stage in which I will make my experience and knowledge accessible to everyone.

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