Home Mind & Brain Autologous Bone Marrow Cells Show Promise in Treating Severe Paediatric Brain Injuries

Autologous Bone Marrow Cells Show Promise in Treating Severe Paediatric Brain Injuries

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A new clinical trial has shown that the use of autologous bone marrow mononuclear cells (BMMNCs) may significantly improve outcomes for children suffering from severe traumatic brain injuries (TBI). This innovative treatment has demonstrated its potential to reduce the duration of intensive care and preserve crucial brain structures, offering hope for improved recovery in affected children. The findings were published in the journal Brain.

The research, conducted by teams at McGovern Medical School, UTHealth Houston, and Phoenix Children’s Hospital, explored the safety and efficacy of BMMNCs in treating children with severe TBI. The study was designed as a randomised, double-blind, placebo-sham-controlled Bayesian dose-escalation trial. Participants aged 5 to 17 years with severe TBI (Glasgow Coma Scale score ≤ 8) were enrolled from two children’s hospitals in Houston and Phoenix.

Within 48 hours of injury, the children were randomised to receive either BMMNCs or a placebo. The primary endpoints included quantitative brain volumes assessed via MRI and the microstructural integrity of the corpus callosum, evaluated at 6 months and 12 months post-injury. Secondary outcomes included long-term functional outcomes and metrics such as ventilator days, intracranial pressure monitoring days, and ICU duration.

Out of 47 patients randomised, 37 completed a one-year follow-up. The results were encouraging. Children treated with BMMNCs experienced a nearly three-day reduction in ventilator days, a one-day reduction in intracranial pressure monitoring days, and a three-day reduction in ICU stay. Importantly, white matter volume was significantly better preserved in the BMMNC group compared to the placebo group.

Quantitative MRI showed that the decrease in white matter volume was much less pronounced in the BMMNC group (decrease of 19,891 mm³) than in the placebo group (decrease of 40,491 mm³). This preservation of white matter is crucial, as it is associated with better long-term cognitive and functional outcomes.

The number of corpus callosum streamlines, which indicates connectivity within the brain, was also better preserved in the BMMNC group. The placebo group saw a reduction of 431 streamlines, whereas the BMMNC group only lost 37 streamlines. Although this finding did not reach statistical significance due to variability, it suggests a trend towards preserved brain connectivity.

The study confirmed that administering BMMNCs is both safe and feasible. No serious adverse events or significant infusion-related toxicities were reported. This is consistent with earlier phase 1 trials, which also demonstrated the safety of BMMNC infusion in paediatric TBI patients.

The underlying mechanism by which BMMNCs exert their beneficial effects is believed to involve the modification of the inflammatory response following brain injury. By controlling neuroinflammation, BMMNCs help preserve brain structure and function. This aligns with preclinical studies showing that BMMNCs can modulate immune responses and promote tissue repair.

The findings from this phase 2 trial suggest that BMMNC therapy could become a critical component in the treatment of severe paediatric TBI. The reduction in ICU days and the preservation of white matter and corpus callosum connectivity could lead to better long-term outcomes for children, potentially reducing the burden of lifelong disabilities.

Future research will focus on larger phase 3 trials to confirm these results and further explore the therapeutic potential of BMMNCs. These studies will also aim to establish standardised protocols for the administration of BMMNCs and evaluate their long-term benefits more comprehensively.

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