The ALS Association

ALS Ice Bucket Challenge Progress
Carrie Munk
The ALS Association
(571) 319-3047



New Clinical Research Fellowships are Announced


Washington, D.C. (April 19, 2016)— The ALS Association and the American Academy of Neurology (AAN) are proud to announce this year’s recipients for the Clinical Research Training Fellowship and Clinician-Scientist Development Award in ALS Research to creatively address the gaps in ALS clinical research. These awards are designed to recruit talented, promising young clinicians who propose innovative clinical ALS studies and to foster their development to make significant contributions to ALS clinical research. This year Cindy Ly, M.D., Ph.D., from Washington University in St. Louis under the mentorship of Timothy Miller, M.D., Ph.D., won the Clinical Research Training Fellowship in ALS Research and Lindsey Hayes, M.D., Ph.D., from Johns Hopkins University in Baltimore under the mentorship of Jeffrey Rothstein, M.D., Ph.D., won the Clinician Scientist Development Award in ALS research. Dr. Ly and Dr. Hayes will receive their fellowship awards during the American Academy of Neurology’s 68th Annual Meeting in Vancouver, Canada from April 15 - 21, 2016. The AAN Annual Meeting is the world’s largest gathering of neurologists, with 12,000 attendees and more than 2,700 scientific presentations on the latest research advances in brain disease.

ALS is a progressive neurodegenerative disease that affects nerve cells in the brain and the spinal cord. Eventually, people with ALS lose the ability to initiate and control muscle movement, which often leads to total paralysis and death within two to five years of diagnosis. For unknown reasons, veterans are twice as likely to develop ALS as the general population. There is no cure, and only one drug approved by the U.S. Food and Drug Administration (FDA) modestly extends survival.

Dr. Cindy Ly’s research project focuses on investigating the immune system 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 pathogenesis 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,” stated Dr. Ly.

For the Clinician Scientist Development Award, Dr. Hayes’ project focuses on the development of pharmacodynamic biomarkers to use in antisense oligonucleotide (ASO) therapy against C9orf72, the most common cause of ALS. ASO technology works to eliminate protein by preventing it from being created. 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) (the fluid that surrounds the brain and spinal cord) 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,” stated Dr. Hayes.

About The ALS Association
The ALS Association is the only national non-profit organization fighting Lou Gehrig’s Disease on every front.  By leading the way in global research, providing assistance for people with ALS through a nationwide network of chapters, coordinating multidisciplinary care through certified clinical care centers, and fostering government partnerships, The Association builds hope and enhances quality of life while aggressively searching for new treatments and a cure.  For more information about The ALS Association, visit our website at

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