Medical breakthrough helps children with bone marrow transplants avoid toxic chemotherapy

Scientists removed immune cells from the donated marrow, to avoid the risk of the recipient's body attacking the transplanted cells.

According to a recent research report published in the journal Nature Medicine, an experimental antibody drug developed by Jasper Therapeutics has paved the way for children needing stem cell transplants to avoid extremely toxic chemotherapy and radiation regimens.

In bone marrow transplants, the first step is always to “cleanse” the diseased marrow with chemotherapy or radiation therapy - a procedure that causes a series of severe side effects such as nausea, hair loss, immunosuppression, and even long-term sequelae such as infertility, liver and kidney damage.

However, the study showed that the antibody drug briquilimab did this without causing toxicity. Briquilimab targets CD117 - a protein found on blood stem cells that controls their growth.

The trial was conducted at Stanford Medical School (California, USA) on 3 children with Fanconi anemia - a rare genetic disorder. Each patient only needed one infusion of briquilimab 12 days before the stem cell transplant.

Results showed that after 30 days, healthy donor cells had covered almost all of the children’s bone marrow. Initially, the team’s goal was to achieve 1% coverage – meaning 1% of the cells in the marrow came from the donor. But tests two years later showed that all three children had nearly 100% donor cells and are still healthy.

Notably, in all three cases, the stem cells were donated from the patient’s own father or mother. This is impossible because parents are often not a perfect genetic match to their child, which can easily lead to transplant rejection.

To overcome this, scientists removed immune cells from the donated marrow to avoid the risk of the recipient's body attacking the transplanted cells.

The team is now conducting mid-stage trials in more children with Fanconi anemia, and plans to test the drug in other genetic diseases that require bone marrow transplants.

Meanwhile, another research team at Stanford School of Medicine is also testing the possibility of applying briquilimab to elderly cancer patients - those who are too weak or have many underlying diseases to endure full doses of chemotherapy or radiation therapy./.

(Vietnam+)

Source: https://www.vietnamplus.vn/buoc-ngoat-y-hoc-giup-benh-nhi-ghep-tuy-tranh-duoc-hoa-tri-doc-hai-post1058498.vnp