Dr. Rachita Sumbria has been awarded an NIH R01 grant from the National Institute of Aging, receiving $1.71 million over the next 5 years for research on Alzheimer’s disease (AD) therapeutics. This project is founded on data from her lab’s earlier successes in modifying erythropoietin (EPO) to cross the blood-brain barrier (BBB) with promising results in treating AD.

EPO’s ability to modulate AD-related pathways and its neuroprotective effects provide the rationale for its development, although its limited BBB penetration and hematopoietic side effects pose challenges. The proposed solution involves fusing EPO with a monoclonal antibody (MAb) against the transferrin receptor (TfR) to facilitate BBB penetration and reduce side effects.

The funds will be used to conduct dose-escalation and safety studies in various AD models, and to investigate the molecular pathways modulated by TfRMAb-EPO, building on prior NIH R21-funded proof-of-concept studies. Preliminary data indicate that this BBB-penetrating EPO has better therapeutic and hematologic indices than EPO alone. EPO’s neuroprotective effects and ability to modulate AD-related pathways justify its development, despite challenges like limited BBB penetration and hematopoietic side effects. The findings may establish a clinically feasible method for sustainable EPO delivery to the AD brain.

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