Innovative treatment of lymphoblastic leukemia (ALL)

Lymphoblastic leukemia (ALL) of B cells – New treatment  FDA approved

Seventeen patients in Israel have so far received the experimental genetic engineering treatment recommended by the FDA experts, and the success rate is amazing for doctors. Dr. Michal Besser, Director of the Laboratory that performs the treatment: “These are tremendous news, terminally ill patients are coming to life.”

Leading Doctors for Leukemia – Consultation Online

What is the treatment, how long does it last and what are the side effects?

“Patients on the brink of death got on their feet”: a day after FDA experts announced their intention to approve the world’s first genetically engineered treatment to cure critically ill cancer patients and the medical world in turmoil. More and more evidence suggests that groundbreaking treatment could make a real difference in the fight against cancer.

The revolutionary treatment, in which genes are transplanted directly into the patient’s blood cells, making them violent so that they can completely eliminate the cancer, has been carried out for the past year in Israel at the Sheba Medical Center. So far, 17 patients have received B white blood cell cancer, The experts were astounded here as well: 75% of patients who were in critical condition and no longer responded to any treatment succeeded in overcoming the disease.

“One of the cases I remember was that of a child who was confined to a wheelchair, her condition deteriorated and she had only a few weeks left to live,” says Dr. Michal Besser, director of the Institute for Immunology, at the Sheba Medical Center. After the infusion the patient is already skating on rollerblades throughout the corridor of the hospital. This treatment is a tremendous news for the medical world, and is expected to save hundreds of Israelis each year. It’s hard not to get excited when you look at the patients who were sure that their end was coming, and suddenly they were coming to life. ”

What is included in innovative treatment, how genes are inserted into the human body, how long treatment lasts and what are the dangers?

What did the FDA said on July 12, 2017?

A panel of FDA experts unanimously recommended that the new treatment be approved for Novartis’s CTL019, which changes the patient’s genetics and causes the body’s cells to fight leukemia, turning them into a “living drug” that causes the immune system to eliminate the patient’s tumor.

In most cases, the FDA tends to automatically approve the recommendations of its expert panels. Novartis has announced that it is working on a similar treatment for other types of cancer.

Who is the treatment for?

The treatment will be approved for children and young adults aged 3 to 25, who have severe lymphoblastic leukemia (ALL) of B cells that have been resistant to treatment or have had a recurrence of the disease, often ending in death. The treatment is intended for about 15% of leukemia patients. In Israel, the treatment was given in an experimental framework at the Sheba Medical Center, which has treated 4 patients a month in the past year.

What is the treatment principle?

The treatment takes millions of T white blood cells from the patient’s body and converts them, through genetic engineering techniques, into cells that can kill cancer cells. The action is carried out through a type of HIV virus that is “paralyzed,” meaning that it does not cause the disease, but is able to penetrate into the T cells and reprogram them. After this stage, the T cells are able to attack the white blood cells of type B of the leukemia patient.

The programmed T cells, called “chemical antigenic receptor cells,” are transfused into the patient’s blood, where they multiply and begin to attack the cancer. Dr. Carl John, head of the University of Pennsylvania’s team of scientists who developed the revolutionary treatment, called these cells “serial cancer killers.” Such programmed T cells can destroy up to 100,000 cancer cells.

How is the treatment done in practice?

The patient arrives at the hospital and undergoes a process called plasma-paresis, in which a blood stream is drawn and at the same time the white blood cells are separated from T. The rest of the blood is returned to the body. Next, the T cells undergo a process of “reprogramming” that changes their properties. In the laboratory, genes that “educate” the T cells to destroy cancerous tumors are inserted into the cells. After this “education series,” the T cells are returned to the patient’s body, multiplying and destroying the cancer cells.

Does treatment require hospitalization

Initial treatment of cell extraction does not require hospitalization. The patient arrives at the department, undergoes the plasma process for about an hour, and returns home while the cells are being treated in Sheba laboratories. After about 10 days, the patient is returned to the hospital, where the transfusion is carried back to the T cells that are activated to the body, where they multiply and begin to destroy the cancer cells. This time the treatment includes hospitalization for several weeks.

What is genetic engineering in therapy?

T cells learn to attack cancer cells aggressively and specifically. They do it thanks to the genes that are inserted into them. The genes are inserted through the use of parts of the HIV virus, the same virus that causes AIDS. But do not worry: this is not the whole HIV virus, but parts of its envelope that serve as “buses” and are able to penetrate the cells of the immune system. Thus, they carry the necessary genes on their backs.

Is the treatment intended only for children?

In the United States, the treatment was approved for ages 3 to 25. In Israel, as part of the Sheba trial, treatment is also performed in patients aged up to 50 who suffer from B-cell blood cancers. Treatment is intended for patients who have not responded to any conventional treatment.

What are the side effects of the new treatment?

This is the major problem of the new treatment: Side effects are considered to be extremely difficult and even life-threatening, and include, among other things, the collapse of blood pressure, high fever and pulmonary problems that have nearly killed some patients. It is therefore possible to transfusion the blood cells that are activated in the hospital only.

Will the treatment be suitable for other cancers or only for leukemia?

The developer said it has begun trials to test the efficacy of other cancers, including brain cancer.

⇒ Leukemia

⇒ Hematology

⇒ Israeli hematologists