2016 Clinical Fellowship Program Recipients

Clinical Research Training Fellowship in ALS Research

The 2016 awardee of the  Clinical Research Training Fellowship in ALS Research is Cindy Ly, M.D., Ph.D., from Washington University in St. Louis under the mentorship of Dr. Timothy Miller. 

Project Summary: Dr. Ly’s research project focuses on investigating innate immunity and autophagy (a type of degradation system in cells) in TANK-binding kinase 1 (TBK1)-associated ALS. TBK1 is a novel gene found in both sporadic and familial ALS suggesting a risk-conferring and potentially causative role in disease. The mechanism by which TBK1 disruption leads to motor neuron loss in ALS is largely unknown. This study will investigate whether TBK1 mutations impair autophagy and disrupt inflammatory responses by examining the fidelity of key protein interactions and their impact on these cellular processes. They will also explore whether TBK1 is misregulated more broadly in sporadic ALS. The goal is to provide valuable insights into the role of TBK1 in ALS disease and clarify its potential as a therapeutic target. 

“I am truly honored to receive this award and am grateful to the AAN Foundation and The ALS Association for supporting my development as a physician-scientist as well as research efforts to expand our understanding of this devastating disease.” 

Clinician-Scientist Development Award in ALS Research 

The 2016 awardee of the  Clinician-Scientist Development Award in ALS Research is Lindsey Hayes, M.D., Ph.D., from Johns Hopkins University in Baltimore under the mentorship of Dr. Jeffrey Rothstein. 

Project Summary: Dr. Hayes’ project focuses on the development of biomarkers to use in antisense oligonucleotide (ASO) therapy against C9orf72, the most common genetic cause of ALS. ASO therapy has shown promising results in the laboratory and the goal is to translate this therapy to patients. To accomplish this, they propose to develop a cerebral spinal fluid (CSF) biomarker to verify that the ASO drug is reaching its target and having the desired effect. This study will generate the preclinical data necessary to move C9orf72 ASO therapy forward to the clinic. 

“I am very honored to receive this award and grateful to The ALS Association, American Academy of Neurology, and American Brain Foundation for their support. This award will provide critical early funding for me as I begin my career as a physician-scientist, and allow me to pursue research that I hope will advance therapy development for ALS.” 

Learn more  about Dr. Hayes’ exciting research project and get to know the person beyond the science.