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The Science

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White Matter Injury (WMI)

is the most common brain injury in preterm infants. It is caused by inflammation and hypoxia, and leads to the loss of Oligodendrocyte Progenitor Cells (OPCs). This, in turn, disrupts myelination in the developing infant brain, and causes permanent motor and cognitive deficits - exemplified in severe cases by cerebral palsy.

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No Treatment, Lifelong Impact

Around 1 in 10 babies are born prematurely and at-risk for WMI. Yet there is currently no treatment available. Affected children face a lifetime of physical and cognitive disability, while parents face care costs of over $1.5M per child.

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Nature Presents a Solution

Scientists at Duke University and TellusTx have discovered a family of compounds in maternal breast milk that stimulate Neural Stem Cells, restore OPC levels, and promote myelination - effectively restoring lost motor function after brain injury in neonatal animal models (see Chao, Benner et al. in References)

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New Hope for the NICU

Tellus is set to take one such compound, TT-20, into human clinical trials. If successful, TT-20 will be the first treatment for WMI, to the benefit of patients and clinicians in Neonatal Intensive Care Units (NICUs) worldwide.

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Future Applications

Promising new research suggests that oxysterol compounds similar to TT-20 could show great benefit in treating neonatal conditions beyond White Matter Injury, along with adult de-myelinating and neuro-inflammatory conditions such as multiple sclerosis.

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References & Further Reading

DeMarco, S, Neonatal drug development comes out of the Dark Ages. Drug Discovery World, Volume 19: Issue 7 (2023) LINK

Chao, Agnes S., Benner, E., et al. 20-αHydroxycholesterol, an oxysterol in human breast milk, reverses mouse neonatal white matter injury through Gli-dependent oligodendrogenesis. Cell Stem Cell 30, Issue 8, 1054-1071 (2023) LINK

Epstein A, Benner E, et al. Subventricular zone stem cell niche injury is associated with intestinal perforation in preterm infants and predicts future motor impairment. Cell Stem Cell Volume 31 Issue 4, 467-483.e6 (2025) LINK

Garg et al. Brain Injury in Preterm Infants with Surgical Necrotizing Enterocolitis – Clinical and Bowel Pathological Correlates. Pediatric Res. 29; 91(5): 1182-1195 (2021) LINK

Novak et al. Early, Accurate Diagnosis and Early Intervention in Cerebral Palsy Advances in Diagnosis and Treatment. JAMA Pediatr. 171, Issue 9, 919. (2017) LINK

Wadhawan et al. Neurodevelopmental outcomes of extremely low birth weight infants with spontaneous intestinal perforation or surgical necrotizing enterocolitis. J Perinatol. 34(1):64-70. (2014) LINK

Hintz SR, et al. Neurodevelopmental and growth outcomes of extremely low birth weight infants after necrotizing enterocolitis. Pediatrics 115(3):696-703 (2005) LINK