Acute lymphoblastic leukemia (ALL) is a type of blood cancer affecting adults and children. In the case of infants diagnosed with ALL, the prognosis is inferior, especially for those with a specific genetic mutation known as KMT2A rearrangement. Despite the best efforts of intensive chemotherapy, the prognosis for these infants has remained dismal in recent decades.
However, a promising new treatment option has emerged in the form of immunotherapy with the drug blinatumomab. Clinical trials have shown that adding blinatumomab to chemotherapy significantly improves survival rates and fewer side effects for infants with this aggressive form of ALL.
As per research published in The New England Journal of Medicine, immunotherapy with blinatumomab has been shown to significantly improve the survival rate of babies with an aggressive form of acute lymphoblastic leukemia (ALL), according to an international clinical trial led by researchers at the Princess Máxima Center for pediatric oncology in the Netherlands.
The trial, which ran from 2018 to 2021 and involved 30 children across nine countries, found that babies who received one month of immunotherapy in addition to chemotherapy had a survival rate of 93% two years after diagnosis, compared to 66% of those who had only received chemotherapy in the past.
The KMT2A rearrangement, a particular fault in the DNA of leukemia cells, is present in three-quarters of babies with ALL, leading to a poor prognosis. Despite intensifying chemotherapy, the prognosis for these babies has not improved in recent decades, with half of the children experiencing a recurrence within two years or dying from the disease or therapy side effects. The trial results showed that blinatumomab will now become the standard treatment worldwide for babies with this aggressive form of leukemia.
Blinatumomab binds to leukemia cells on one side and immune cells on the other, clearing up the leukemia cells. While the therapy has already been offered to some adults and older children with ALL, it was still determined whether it was well-tolerated and effective in babies. The trial results indicate that blinatumomab significantly improves survival rates and has fewer side effects.
Dr. Inge van der Sluis, the pediatric oncologist and clinical pharmacologist who led the clinical study, said that adding immunotherapy to chemotherapy leads to much better survival and fewer side effects for babies with ALL. While this was a small study, the precise results mean that all babies with this form of leukemia will now receive immunotherapy as part of standard treatment.
A more extensive study with more children is planned to confirm the effect of blinatumomab. Researchers will investigate whether babies benefit from two courses of blinatumomab and a reduction in chemotherapy to improve their quality of life even further.
According to Prof. Dr. Rob Pieters, the medical director and pediatric oncologist at the Princess Máxima Center for pediatric oncology, who led the research group, the cure rate for babies with ALL has lagged behind the cure rate for older children, with babies being much more likely to have an aggressive form of the disease. The addition of immunotherapy to worldwide standard treatment represents a significant improvement in the prognosis for these young children.