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International gene therapy trial for 'bubble boy' disease shows promising early results

negative impact on cognitive function. Seeking to explore whether transplants and specifically the intensity of transplant-related chemotherapy and radiation might be associated with cognitive decline, investigators conducted a prospective study, measuring cognitive function in transplant patients and healthy controls at similar intervals. In addition, the investigators measured the relative length of telomeres (protective protein structures on the ends of human chromosomes) in allogeneic HSCT recipients before transplant, hypothesizing that HSCT recipients with shorter telomeres would demonstrate reduced cognitive function after transplant.

To test their hypothesis, researchers assessed several cognitive parameters (e.g., multi-tasking, visual memory, recall) before transplant and at six, 12, and 24 months post-transplant in HSCT recipients and at the same intervals in the control group and found a significant association between shortened telomeres and reduced cognitive ability. Researchers also observed that those HSCT recipients who received full-intensity HSCT reported worse cognitive function than those who received reduced-intensity HSCT. In fact, the cognitive function in patients with reduced-intensity transplants remained comparable to that in the healthy controls. The cognitive decline observed was specifically linked to the shortening effect of these treatments on the length of telomeres.

"This study strongly indicates that patients who receive reduced-intensity transplants are generally spared from significant impact on their cognitive abilities, while full-intensity conditioning contributes to meaningful changes in multiple cognitive domains," said study author Alysia Bosworth of City of Hope in Duarte, Calif. "As our results isolate a specific group of patients who would be considered 'high risk' for cognitive impairment, including those who receive full-intensity transplants, it will be important for clinicians to evaluate and o

Contact: Irene Sege
Dana-Farber Cancer Institute

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International gene therapy trial for 'bubble boy' disease shows promising early results
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