The study found that the antibody-based drug, briquilimab, did not cause toxicity or side effects like chemotherapy and radiation. 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 three children with Fanconi anemia, a rare genetic disorder that can cause cancer. Each child received only one briquilimab infusion 12 days before the stem cell transplant. The results showed that after 30 days, healthy cells from the donor had almost completely covered the children's bone marrow.
The team is now conducting mid-stage trials with more children with Fanconi anemia, and plans to test it on genetic diseases that require bone marrow transplants. Co-lead author Agnieszka Czechowicz, MD, said the treatment not only eliminates the need for radiation and chemotherapy, but also delivers superior results.
Source: https://www.sggp.org.vn/thuoc-thay-hoa-tri-va-xa-tri-post810910.html
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