DEVELOPING THERAPIES FOR ATAXIA AND TREMOR

Sheng-Han Kuo, M.D.

Principal investigator

Director, Initiative for Columbia Ataxia and Tremor 

Dr. Kuo is a physician-scientist conducting research in the disease of the cerebellum, including ataxia and tremor. The Kuo lab uses diverse methodologies to investigate the disease pathophysiology, including postmortem patient brains, preclinical models, and clinical trials, to collectively advance the knowledge of the cerebellar function and to develop therapies for ataxia and tremor. Dr. Kuo is the recipient of the American Academy of Neurology Research Fellowship Award, the Parkinson's Disease Foundation Lucien Côté Early Investigator Award in Clinical Genetics, Louis V. Gerstner Jr Scholar Merit Award, and American Academy of Neurology Jon Stolk Award in Movement Disorders.

Mission

The goal of our lab is to study the role of the cerebellum in neurological disorders. In particular, we focus on ataxia and tremor, two very disabling symptoms leading to uncoordinated hand movements and gait imbalance. We use multiple approaches to study the disease mechanism of these disorders, including brain pathology, preclinical models, human physiology, and stem cell-derived human neurons. Therefore, we can rapidly translate basic scientific knowledge into clinical applications. In addition, our goal is to develop personalized strategies to treat these disabling diseases and symptoms. Not only do we shed light onto the disease mechanisms, but we also advance the fundamental understanding of how movements are controlled by the cerebellum.

Research Interests 

Ataxia

 

Ataxia is caused by cerebellar dysfunction, which leads to imbalance, loss of hand dexterity, slurred speech, and vision problems. There are multiple causes of cerebellar ataxia, including genetics, immunological disturbances, and nutritional deficiencies. We are interested in the genetic and immunological causes of cerebellar ataxia. We study preclinical models and ataxia patients with an overarching aim to understand the disease mechanism. In addition, we actively participate in the natural history and clinical trials of spinocerebellar ataxias (SCAs) in collaboration with other ataxia centers across the world. Collectively, we hope to discover new therapies for ataxia.

Tremor

 

Tremor is the most common form of abnormal movement. People with tremor suffer from discoordination of the hands, head, and voice. Tremor can impair one’s ability to perform daily activities, such as writing, drawing, drinking, and eating. In addition, tremor can cause embarrassment. Essential tremor and Parkinson’s disease are the two most common tremor disorders and it is estimated that millions of Americans are suffering from these disorders. In our lab, we study the pathology of essential tremor and Parkinson’s disease, focusing on the cerebellum and related brain regions. We further establish preclinical models with these pathological alterations to investigate how the morphological changes in the cerebellum can lead to disturbed cerebellar physiology and tremor. By understanding the disease mechanisms and physiology, we will be able to rationally design and test medications that can dampen the tremor.