Two infants diagnosed with a relapsed form of childhood cancer who had previously exhausted all other treatment options remain disease-free after receiving a first-in-human experimental therapy that uses genetically engineered T cells, a new analysis reports.

Such cell-based approaches have been difficult to implement in young children, a population in which B cell acute lymphoblastic leukemia (ALL) is highly prevalent, representing 25% of all pediatric cancer cases in the United States. Several options exist for treating ALL in children between the ages of one and 10, but infants less than one year old generally have much poorer prognoses.

While modified or chimeric antigen receptor (CAR) T cells have proven to be highly effective in tackling ALL, these cancer-killing cells must be generated from each individual patient, which isn’t feasible for those who do not have sufficient quantities of healthy T cells to start, like very young children. In search of a better alternative, Waseem Qasim and colleagues developed a novel approach that overcomes this significant obstacle to current T cell therapies. Instead of fine-tuning the infants’ own T cells to fight their cancer, the researchers made a “universal” T cell that eradicated the leukemia from both subjects’ blood.

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Dr. Stegall’s Comments: Although pediatric oncology is not my specialty (I only treat adults), I do like to stay on top of the research involving children since therapies which are effective in children could also apply to adults. While the topic of genetically engineered treatments is a controversial one, the results of the cases in the article are impressive. I look forward to more research in this area.