UAlbany Scientists Shorrock and Cleary Pursue Single Treatment for Multiple Neurological Diseases

UAlbany Shorrock and Cleary

Two University at Albany researchers are taking a different approach by developing a single compound that could potentially treat dozens of rare neurological diseases in many patients worldwide. Their work has recently been supported by a $50,000 award from the SUNY Technology Accelerator Fund which will help move the research closer to future clinical trials.

The research is led by Assistant Professor Hannah Shorrock of the Department of Biological Sciences and John Cleary, Assistant Director of Research at the RNA Institute, whose work has identified common genetic links shared by many neurological diseases. One well-known example is Huntington’s disease, which is caused by an abnormal repetition of a short DNA sequence known as CAG. This repeated CAG sequence leads to the production of harmful proteins that gradually damage brain cells. Over time, people affected by these neurological diseases such as Huntington’s may lose the ability to walk, speak, or perform everyday tasks, and in later stages have difficulty swallowing or breathing. 

Rather than treating each neurological disease separately, the UAlbany researchers are working to reduce the harmful effects caused by excessive CAG repeats in the body by developing one compound that could be used to treat many neurological diseases that have the dysfunctional CAG repeat. To date, Drs. Shorrock and Cleary have developed a compound that helps reduce the damage caused by these abnormal DNA repeats. However, the compound does not dissolve well in liquids that are safe for long-term patient use. Because some laboratory solvents can cause side effects, the team has created 40 new versions of the compound in hopes of identifying one that is both safe and effective. 

Funding from the SUNY Technology Accelerator Fund will allow the team to test these new versions of the compound and identify the most promising treatment. If successful, this step would move the research closer to clinical trials of which is a major milestone before any treatment could be approved for patients suffering from CAG-related neurological diseases. Past support for Drs. Shorrock and Cleary’s research includes the National Ataxia Foundation, the National Institutes of Health, New York State, and State University of New York.

Together, this research highlights how UAlbany scientists from the Department of Biological Sciences and the RNA Institute are advancing innovative solutions with the potential to improve the lives of people affected with neurological diseases.