New therapy helped save 64% of patients with incurable cancer

As reported by IFLScience, the first person to bravely undergo the experimental treatment was Alyssa Thelwell from the British city of Leicester. In May 2021, she was diagnosed with the dreadful T-cell acute lymphoblastic leukemia. This is an aggressive type of blood cancer that, in approximately 20% of cases, does not respond to either chemotherapy or bone marrow transplantation.

This was exactly what happened to Alyssa. Standard protocols did not work, and doctors had to admit that no treatment options remained. It was then that her family was offered participation in a clinical trial. Alyssa agreed, reasoning that even if the new method didn't save her, it might help other people in the future.

Fortunately, the risk paid off. After treatment, the disease receded, and now, several years later, the girl (now 16) has returned to a full life. She attends school, participates in sailing, and plans to become a scientist to one day make discoveries in medicine herself.

What is the essence of the method?

We are talking about the so-called BE-CAR7 — the world's first "universal" CAR-T therapy based on precise gene editing.

Typically, for similar treatments, doctors have to take the patient's own blood, modify their cells, and return them. This is time-consuming, very expensive, and sometimes the patient's cells are simply not of sufficient quality. Scientists from Great Ormond Street Hospital and University College London made a breakthrough: they learned how to use cells from healthy third-party donors, transforming them into a medicine suitable for everyone.

First, healthy donor leukocytes are modified to make them "universal." This is achieved by removing surface receptors and neutralizing the cells by removing an immune marker called CD7. This procedure is necessary to prevent the destruction of the new cells by the recipient's immune system.

Next, another surface protein, CD52, is removed. This is done to hide the donor cells from the effects of powerful immunosuppressive drugs prescribed to the patient.

Finally, a new DNA sequence is introduced into the cells using a modified virus. This allows them to produce a chimeric antigen receptor (CAR). It is this mechanism that enables donor cells to recognize and attack cancerous T-cells within the patient's body.

The ideal treatment cycle takes four weeks, after which the patient undergoes a bone marrow transplant to restore their own immune system.

Results that offer hope

At the annual meeting of the American Society of Hematology, the research team presented the results of the first phase of trials, which included eight children and two adults.

The numbers are impressive: 82% of patients achieved deep remission, allowing them to undergo life-saving bone marrow transplants.

Moreover, 64% of participants remain absolutely healthy to this day, with some living without signs of cancer for over three years.

Hematologists note that they are observing an impressive response in clearing leukemia that was previously considered incurable.

However, scientists warn: the treatment is very difficult and intensive. Unfortunately, not all patients selected for participation in the trial survived. Side effects can be serious, and after a bone marrow transplant, it takes a long time for the immune system to recover, during which any infection is life-threatening.

Nevertheless, for people with such aggressive forms of the disease, where survival chances were minimal, this method has become a true breakthrough. Thanks to the success of the trials, charitable foundations have already allocated funds for the treatment of ten more patients, offering hope for the further development of the technology.

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