The illustration shows a tau protein (red) becoming detached from a microtubule (blue). A new compound discovered by researchers at UC San Diego and University of Pennsylvania may be able to treat Alzheimer’s disease by normalising microtubule structure and function. Credit: The Trustees of the University of Pennsylvania.

Researchers at the University of Pennsylvania’s (Penn Medicine) Perelman School of Medicine and University of California (UC) San Diego have secured a $6.9m grant from the National Institutes of Health’s (NIH) National Institute on Aging (NIA) unit to advance clinical trials of CNDR-51997 for Alzheimer’s disease.

The funding is expected support the development of CNDR-51997, a tau-targeting drug candidate, through the necessary investigational new drug (IND)-enabling studies.

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In a study in mouse models, the drug could reverse Alzheimer’s disease symptoms.

CNDR-51997 was detected under a drug discovery programme, jointly conducted by Penn Medicine and UC San Diego, with initial funding support from the NIA.

With this additional funding, the team will conduct formal safety studies as required by the US Food and Drug Administration (FDA) before human testing can commence.

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The three-year grant period is expected to culminate in the submission of an IND application to the FDA.

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Approval of this application would help to prepare for Phase I clinical trials.

In preclinical tests, CNDR-51997 has shown potential in reducing amyloid-beta (Aβ) plaques and tau pathology, which are the key aspects of Alzheimer’s disease in the brain.

The implications of this research extend beyond Alzheimer’s, potentially offering therapeutic benefits for a range of tauopathies.

These include conditions such as traumatic brain injury, frontotemporal lobar degeneration, chronic traumatic encephalopathy (CTE), progressive supranuclear palsy, corticobasal degeneration, and Pick’s disease.

University of Pennsylvania Perelman School of Medicine research professor Kurt Brunden said: “Our findings that CNDR-51997 reduces both Aβ plaques and tau inclusions in mouse models suggest that the compound holds considerable promise for Alzheimer’s disease. However, there is also a great unmet need for disease-modifying drugs for the other tauopathies.

“The potential of CNDR-51997 to address tau-related diseases beyond Alzheimer’s is another important aspect of its therapeutic promise.”