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Advancing Hope for Patients with Huntington's Disease

Posted on July 18, 2023 in Center for Rare Diseases

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Harrington Scholar Dr. Ed Grabczyk’s remarkable discovery has advanced one step closer to a potential treatment for patients living with Huntington’s disease and other rare neurological disorders, such as Friedreich ataxia, through a licensing agreement with LSU Health New Orleans and Takeda.

Harrington Discovery Institute Provides Key Resources to Advance Huntington’s Disease Therapeutic

In a significant step forward for the treatment of rare neurological disorders, LSU Health New Orleans has announced an exclusive licensing agreement with Takeda, a global pharmaceutical company. The agreement focuses on early-stage technology designed to slow down the progression of DNA repeat expansion disorders, particularly in Huntington's disease. Huntington's disease, along with over 40 other related neurological disorders, affects more than 100,000 individuals in the United States alone.

Repeat expansion disorders are a group of more than 40 inherited neurological conditions that affect approximately one person in every 3,000. DNA contains repeating sequences, and while most repeats don't cause any issues, in certain areas, having a large number of repeats can lead to these disorders. The most well-known repeat expansion disorder is Huntington's disease, where the expanded DNA makes a harmful protein that damages the brain and causes problems with movement and thinking.

Dr. Ed Grabczyk, Professor of Genetics at LSU Health New Orleans School of Medicine, and a 2018 Harrington Rare Disease Scholar, has made a breakthrough discovery that offers potential for addressing the continuous growth of DNA repeat expansions within genes. This growth is a key factor in the development of various neurological disorders, including Huntington's disease. By targeting a specific DNA mismatch repair protein responsible for DNA repeat expansion, Dr. Grabczyk's research offers hope for slowing down or even stopping the harmful effects caused by this mechanism.

Recognizing the significance of Dr. Grabczyk's work, Harrington Discovery Institute awarded him the Harrington Rare Disease Scholar Award in 2018. This recognition not only provided additional research funding but also facilitated access to industry experts via Harrington’s Therapeutics Development Center who contributed invaluable insights, enabling further progress in the development of this potential treatment.

Collaboration has been a key driving force behind these advancements. The partnership between Takeda, LSU Health New Orleans, and Harrington Discovery Institute has played a crucial role in supporting Dr. Grabczyk's groundbreaking research. By combining their expertise and resources, these dedicated partners have fostered an environment conducive to innovation and breakthroughs.

Dr. Jonathan S. Stamler, President of the Harrington Discovery Institute, highlights the institute's commitment to supporting groundbreaking research that has the potential to make a tangible impact on patients' lives. Their assistance, combined with the expertise and dedication of the research team, has propelled this project forward.

“The success of Dr. Grabczyk’s program is a testament to the extraordinary impact that can be achieved through close collaboration between committed partners like Takeda and LSU Health New Orleans,” said Jonathan S. Stamler, MD, 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. “We are pleased to have had the opportunity to support Dr. Grabczyk’s work.”

The licensing agreement exercised by Takeda underscores the potential value of Dr. Grabczyk's discovery in the treatment of Huntington's disease and related disorders.

As this promising breakthrough unfolds, there is renewed hope for individuals affected by Huntington's disease and other DNA repeat expansion disorders. The collaborative efforts of passionate scientists, visionary partners, and the support Harrington Discovery Institute have paved the way for potential advancements in treatments that could transform the lives of countless individuals and their families. Together, we are driving progress towards a brighter future in the fight against devastating neurological diseases.

**Watch this excerpt of Dr. Grabczyk’s presentation at the 2023 Harrington Symposium where he explains the guidance the Harrington Therapeutics Development Center provided for his project.**

About the Scholar

Ed Grabczyk

Musculoskeletal, Neuroscience, Rare/Orphan

Ed Grabczyk, PhD

LSU Health Sciences Center
Harrington Rare Disease

More about Ed Grabczyk

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Harrington Scholar Advances Technology for Potential Rare Neurological Disorders

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