USC receives UChicago Medicine grant to advance $1,100 grant in Huntington’s disease research

The University of Chicago Medicine has received a $1,100,000 grant from the National Institute of Neurological Disorders and Stroke (NINDS) for the advanced study and development of a novel form of treatment for Huntington’s disease (HD). The $1,100,000,000-funded project builds upon prior NINDS grants for neurodegenerative diseases of the central nervous system. With this grant, the University of Chicago Medicine joins the ranks of ten other institutions from Yale University, Case Western Reserve University, and the National Institutes of Health (NIH). The University of Chicago’s National Institutes of Health (NIH) leadership, supported by the National Institutes of Health are leading efforts from the National Institutes of Health (NIH)/National Institute of Neurological Disorders and Stroke (NINDS) to develop new and improved treatments for brain disorders and disorders of the brain and nervous systems.

Huntington’s disease (HD) is a neurodegenerative disorder that causes sloughing away of the brain’s protective layer, called the neocortex. Over time, the disease’s pathology can cause severe confusion and cognitive dysfunction, leading to serious problems with movement, speech, and swallowing. None of the therapies approved by the FDA currently target HD, but scientists hope that the researchers’ advanced mouse model of the disease will help them develop better clinical tools.

“Huntington’s disease is a devastating neurodegenerative disease. We hope this innovative grant will spur scientists to further develop therapies for both disease management and treatment,” said the study’s principal investigator, Robert S. Lukes, PhD, UM1, a principal investigator of the NINDS grant. Additional investigator and lead investigator on the project is Sunil S. Sood, MD, vice dean for research and graduate studies at UM1.

The $1,000,000 grant will support the phase I clinical trial. By combining two existing clinical and preclinical experiments, pilot project investigators will explore combining the use of a zinc-based drug delivery method, delivering the targeted medication through implanted subcutaneous electrodes and carrying the drug locally, with high safety and tolerability.

Led by Peter Hoelzle, PhD, the lead investigator at the medical innovation office of the University of California at San Francisco, the pilot project will recruit 2 newly diagnosed HD patients and 1 cognitively normal control group to undergo high-dose cytolysis therapy at Walter Reed National Military Medical Research Center in Bethesda, Maryland. Initial results from the trial are expected to be available in the summer of 2020.

A primary goal of the pilot project is to document the safety and tolerability of the drug therapy and determine whether ingesting the drug via implanted subcutaneous electrodes will alleviate pain and reduce DSC related neurotoxicity. A second goal is to determine whether the combination of drugs will maximize activity of the brain’s N-glycoshiplase (NGL) enzymes and increase brain neurogenesis (brain cells that produce and maintain new nerve cells).

Gary Freund, MD, PhD, professor in the departments of neurology and neurology surgery at the University of Michigan, is the study’s senior investigator. He and co-principal investigator Luying Zhang, PhD, a former graduate student of Dr. Freund and an instructor of neurology at Case Western Reserve School of Medicine, are co-principal investigators for the pilot project.