LSU Health New Orleans and Takeda Collaborate to Advance Novel Research for Rare Neurological Disorders

LSU Health New Orleans entered into an exclusive option and license agreement with Takeda Pharmaceutical Company Limited (“Takeda”) granting Takeda the exclusive right to develop further a technology invented at LSU Health New Orleans with the aim to discover potential therapeutics to treat rare neurological diseases caused by DNA repeat expansion.

In DNA repeat expansion disorders, a segment of repeated DNA expands within a gene to cause disease, and these repeats can number in the dozens or even hundreds. The repeated DNA sequence itself may continue to increase in size over time, which can impact the severity of the disorder, leading to earlier onset or more rapid disease progression. There are more than 30 progressive neurological disorders caused by DNA repeat expansion.

“Imagine that you have a ticking time bomb within your body that will go off in the future and destroy you,” explains Ed Grabczyk, Ph.D., Associate Professor of Genetics at LSU Health New Orleans School of Medicine, who invented the technology. “That is essentially the situation faced by people who inherit a gene carrying a DNA repeat expansion. Our novel therapeutic approach targets a central mechanism that is shared by all repeat expansion diseases.”

Dr. Grabczyk’s research focuses on a novel therapeutic approach to DNA repeat expansion disorders. He received a grant from LSU’s proof-of-concept fund to validate his research and as a result of that work, received a Harrington Rare Disease Scholar Award from the Harrington Discovery Institute at University Hospitals in Cleveland, Ohio, which provided additional research funding and translational support. In April 2018, Dr. Grabczyk and LSU Health New Orleans began a collaboration with Takeda as part of a broader Collaborative Grant Agreement with Harrington Discovery Institute. The exclusive option and license agreement will now allow Takeda to develop further this important field of research.

“Takeda Neuroscience is committed to developing treatments for patients suffering from devastating neurological diseases, and a key part of our strategy is to follow the best science early on and access innovation across the globe,” said Ceri Davies, Ph.D., Head of the Neuroscience Drug Discovery Unit at Takeda. “We look forward to building upon the research we initiated with LSU Health New Orleans through Takeda’s alliance with Harrington Discovery Institute. By combining the early discovery efforts of Dr. Grabczyk and his laboratory with Harrington’s translational and development capabilities, we have an opportunity to help rapidly advance the next generation of treatments to patients suffering from rare neurological disorders.”

“The success of Dr. Grabczyk’s program is a testament to the transformative impact achieved by close collaboration between committed partners like Takeda and LSU Health New Orleans. We are committed to identifying and de-risking breakthrough discoveries that help us achieve our mission to address the world’s most pressing, unmet medical needs. We are excited about the potential of this new innovation to help thousands of patients,” said Jonathan S. Stamler, M.D., President, Harrington Discovery Institute, Robert S. and Sylvia K. Reitman Family Foundation Distinguished Professor of Cardiovascular Innovation and Professor of Medicine and of Biochemistry at University Hospitals and Case Western Reserve University.

This kind of public/private partnership leverages resources to carry leading edge therapies from the university research lab to the bedside where they can bring hope to patients with devastating medical conditions.

“The agreement paves the way for future development of transformative therapeutics for people with repeat expansion disorders,” says LSU Health New Orleans Interim Chancellor Dr. Steve Nelson. “We’re grateful to Harrington Discovery Institute and Takeda for their partnership in helping us to progress a major research discovery.”

Visit lsuhsc.edu to learn more.