Unlocking the Therapeutic Potential of Tears

Kellogg is also home to one of the nation’s most robust and diverse oculoplastics research portfolios, including numerous basic, clinical and translational research grants, clinical trials, and patent applications.

Dr. Aakalu in Principal Investigator on two NIH R01 grants. The latest aims to develop a novel treatment approach for the fatal disease Niemann-Pick Type C (N-P). The project leverages his groundbreaking work deriving therapeutic agents from the peptides found in tears.

N-P is a rare, genetic neurodegenerative disorder characterized by the body’s inability to transport cholesterol and other fatty substances (lipids). As a result, lipids build up within brain and other cell types.

In earlier research into lipid and small molecule therapies to treat dry eye and corneal injuries,

Dr. Aakalu and his colleagues discovered that the peptide they were studying had something in common with N-P; namely, the protein receptor TMEM97.

“The TMEM97 receptor has been shown to impact the protein that is damaged in N-P,” explains Dr. Aakalu. “We found that TMEM97 can, in turn, be impacted by the peptide-based formula we developed for dry eye. With that knowledge, we applied those peptides to models of N-P and found that they yielded improvements in some features of the disease.”

The grant will fund future studies to advance a potential peptide-based treatment for N-P.

While N-P is an unusual target in ophthalmology research, the development of peptide-based therapeutics represents an exciting new frontier for treating a range of ocular disorders.

“Our group is at the forefront of the emerging study of tear biology,” Dr. Aakalu says. “We hope that mining the molecular components of tears may yield novel biomarkers for new therapies.”

Header image caption: Vinay Aakalu, M.D., M.P.H.