Ono Pharma Grant Fuels Innovative Research on Lipid Modifications in Disease

In a significant advancement for biomedical research, Cornell University has secured a $1 million grant from the Ono Pharma Breakthrough Science Initiative to explore the vital role of lipid modifications in cellular processes, particularly in the context of disease progression. This funding will enable researchers to investigate how these chemical alterations influence autophagy, a crucial mechanism for cell health and survival. The research, led by Jeremy Baskin, an associate professor in the Department of Chemistry and Chemical Biology and the Weill Institute for Cell and Molecular Biology, aims to unveil new therapeutic pathways to combat debilitating diseases such as Alzheimer's, cancer, and Parkinson's.

Understanding the core processes that dictate cell survival and death is paramount in developing effective treatments for various diseases. Autophagy, derived from the Greek term meaning "self-eating," is the cellular mechanism through which cells eliminate damaged components, thereby sustaining their health. During this process, cells envelop damaged materials in double-layered lipid membranes called autophagosomes, which subsequently fuse with lysosomes for degradation and recycling.

Despite its importance, the therapeutic targeting of autophagy has proven challenging. Many existing drugs aimed at enhancing autophagic processes lack efficacy or induce adverse side effects, primarily due to incomplete knowledge of the intricate molecular mechanisms involved. As Baskin explained, "This research could help us understand how cells take out their trash—and what happens when that process breaks down. If we can control this process, we may one day be able to treat many serious diseases more effectively."

The Ono Pharma Foundation, known for supporting pioneering scientific inquiries, selected Baskin's project for its potential to lead to groundbreaking discoveries in disease treatment. "What's exciting to me about the support we're getting from the Ono Foundation is that it's enabling us to exploit our expertise in chemical biology methods like bioorthogonal chemistry, which features highly selective chemical tagging reactions inside of living systems without interfering with native biochemical processes," Baskin noted. This innovative approach will facilitate a deeper investigation into the post-translational lipidation of proteins, a modification that influences autophagy.

Currently, only a limited number of proteins associated with autophagy are known to undergo lipid modifications. However, Baskin's research group has developed precise chemical tools to identify additional proteins affected by this modification, revealing promising leads for further study. Their findings could provide insights into how manipulating these lipid modifications might enhance therapeutic strategies. For instance, activating autophagy could help eliminate toxic proteins that accumulate in neurodegenerative diseases, while inhibiting this process in cancer cells may render them more susceptible to treatment.

As Baskin remarked, "Post-translational lipidation could be a general control knob for autophagy. If we learn how to turn it, we could provide new pathways for therapies to treat some really devastating diseases."

The implications of this research extend beyond basic science; they could reshape approaches to treating complex diseases that currently lack effective interventions. The focus on lipid modifications may pave the way for more targeted and efficient therapies, mitigating the side effects commonly associated with broader autophagic interventions.

With the support of the Ono Pharma Foundation, Cornell researchers are poised to make transformative contributions to our understanding of cell biology and its applications in medicine. This collaboration exemplifies the importance of funding innovative scientific research that has the potential to unlock new horizons in disease management and treatment. As Baskin and his team embark on this promising journey, the scientific community and patients alike remain hopeful for the breakthrough discoveries that may emerge from their work.