UCSD Scientist Among Those Granted $6.9M For Alzheimer's Study

SAN DIEGO, CA — A UC San Diego scientist is among a team granted $6.9 million from the National Institute on Aging to prepare a potential disease-modifying Alzheimer's treatment for future clinical trials, it was announced Tuesday.

A multidisciplinary team of researchers led by Carlo Ballatore at UCSD and Kurt Brunden at the University of Pennsylvania recently published a study about a compound, called CNDR-51997, finding it was effective in restoring brain health in mouse models of Alzheimer's disease.

The new grant is intended help the researchers demonstrate the drug's safety in formal studies required by the U.S. Food and Drug Administration prior to the initiation of human testing. By the end of the three-year grant period, the researchers hope to submit an Investigational New Drug application to the FDA that, if approved, would allow for Phase 1 clinical studies, according to UCSD.

"Alzheimer's is a devastating disease with very few treatment options, so we are eager to advance CNDR-51997 through the drug development process," said Ballatore, a professor at UCSD's Skaggs School of Pharmacy and Pharmaceutical Sciences. "This compound has been designed to combat tau- mediated neurodegeneration and our preclinical data suggest that it could be beneficial for the treatment of Alzheimer's and related dementias."

According to the scientists, Alzheimer's is characterized by abnormal deposits of two types of protein in the brain: amyloid beta and tau. The currently available disease-modifying treatments for Alzheimer's target amyloid beta deposits in the brain, with no approved therapies that target pathological tau. In mice, the researchers found that CNDR-51997 was able to reduce both types.

The research suggests the compound could have implications in treatment for frontotemporal lobar degeneration, progressive supranuclear palsy, corticobasal degeneration, Pick's disease, traumatic brain injury and chronic traumatic encephalopathy -- which can also see increased tau deposits.

"Our findings that CNDR-51997 reduces both [amyloid beta] plaques and tau inclusions in mouse models suggest that the compound holds considerable promise for Alzheimer's disease," said Brunden, a research professor in the Perelman School of Medicine and director of drug discovery at Penn's Center for Neurodegenerative Disease Research. "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."

One of tau's functions is to stabilize microtubules, tube-like structures that help give cells their shape. In Alzheimer's disease and other related maladies, tau becomes detached from microtubules, which causes them to become disorganized -- leading to loss of brain cells.

"This is a unique compound with desirable properties, and Dr. Brunden and I are grateful to the NIA for their continued support and the opportunity to develop this compound further through IND-enabling studies, which if successful, will lead to an IND submission," Ballatore said.

— City News Service