Researcher Spotlight: Dr. Lindsey Hayes

What is your academic background?
I grew up in Kansas and went to Emory University in Atlanta for my undergraduate degree in Neuroscience and Behavioral Biology. I stayed on at Emory to complete the M.D.-Ph.D. program, and then moved to Baltimore in 2011 to pursue my internship and neurology residency at Johns Hopkins. Currently, I am a fellow at Hopkins, completing subspecialty training in neuromuscular medicine. It has been a busy year, but one that I have really enjoyed. In July, I am thrilled to be staying on as faculty. Thanks in large part to The ALS Association Clinical Fellowship, the majority of my time will be dedicated to research, and I will also be able to continue seeing neuromuscular patients in clinic.

What has the support from The ALS Association meant to you and how will your Clinical Research Fellowship push your project forward?
I am very grateful for this fellowship. It comes during a critical time when I am focused on generating data and applying for funding to try to become established as an investigator. This support will allow me to remain focused on ALS and ongoing work on C9orf72 therapy development.

Do you have a message for the donors that support your award?
Thank you very much for this generous award and for the outpouring of support I have received. It has been an amazing source of motivation and encouragement as I near the end of my training.

How and why did you get involved in ALS research?
My grandmother developed ALS while I was in high school. The idea that there was a fatal disease that we couldn’t treat and couldn’t even explain made a major impression on me. Sadly, she passed away soon after I went to college. At that time, I was studying neuroscience, and thought I wanted to go to medical school but did not have a clear sense of direction. I approached Jonathan Glass, the Director of the Emory ALS Center, about joining his lab. He got me involved on a project looking at the time course of pathology in the SOD1 mouse, and things took off from there. I loved working in the lab. Dr. Glass’s enthusiasm for translational research was what really convinced me to pursue a career as a physician-scientist. The ALS field has changed considerably since I finished graduate school, with the discovery of C9orf72 and other new genes, so it is a fast-paced and challenging environment. But for me, it also goes back to my family connection. Even though I am the only scientist in my family, who are all talented musicians, my family can relate to what I am doing on a personal level and that has made this journey even more meaningful.

Do you play any musical instruments?
Yes, I play the flute and the piano. My parents spent a lot of time and money taking me to music lessons when I was growing up. I have apologized to them many times for not following through! But I still play on occasion.

What makes Johns Hopkins a great place to work and study ALS?
The Neuromuscular Division at Hopkins has many amazing faculty who are experts in their fields. I have been very lucky to be able to train with such great mentors both clinically and in the lab. There is also a large group of basic and translational scientists focused on ALS and an active ALS clinic team. Having that depth of resources and collaboration really helps to move things forward quickly.

Speaking of mentors, is there a mentor that stands out to you?
Jeff Rothstein has been instrumental in helping me get my current work off the ground and has been very supportive as I look forward to the next step in my career.

Jonathan Glass, my graduate advisor, is really the one who convinced me to be a clinician-scientist. The amount of training and time involved seemed daunting – more than 12 years of medical school, Ph.D., and residency training – but he convinced me that it would be worth it. Seeing him going back and forth between clinic and the lab, finding both worlds equally challenging and rewarding, made a big impression. Hopefully, I will be able to balance all of this with as much skill and enthusiasm.

In layman’s terms please describe your C9orf72 antisense project that was funded by our fellowship.
The project I am working on is focused on therapy development for C9orf72-mediated ALS and frontotemporal dementia (FTD). The C9 repeat expansion was discovered about 5 years ago and has come to be recognized as the most common genetic cause of ALS and FTD. This is the first time this type of genetic expansion has been identified in ALS, and it gives us a druggable target for therapies such as antisense oligonucleotides (ASOs) and small molecules. ASOs can turn off C9 by not allowing the toxic C9 protein to be made. Although these treatments have shown promise in lab models, in order to bring them to clinical trial we need accurate ways of monitoring them in patients. My work is focused on developing biomarkers that can serve as a therapeutic readout to ensure that these drugs are reaching the target and having the maximum effect. Over the past year, in collaboration with the Petrucelli lab at Mayo Jacksonville, we have validated candidate biomarkers for ASO therapy using induced pluripotent stem cell (iPSC) neurons. This award from The ALS Association will help move this work forward, as we develop novel methods to monitor turnover of these markers, first in model systems and then in patients.

What are biomarkers and why do you think they are so important?
Biomarker is a broad term referring to tools used to diagnose or follow progression of disease. I am focused on pharmacodynamic markers, which provide readouts of drug efficacy. Ideally, pharmacodynamic markers change in parallel with drug activity and can be readily measured either in blood or cerebral spinal fluid (CSF) [the fluid that surrounds the brain and spinal cord].

What is the overall impact of your research on the ALS field and how can it lead to potential ALS treatments?
Our research is really providing the bridge between laboratory models and patients. Without having biomarkers to follow treatment, you are flying blind. So having these biomarkers will let us know that ASOs are getting to their target and working efficiently. This will really allow us to titrate the therapy and get maximum benefit from it. It is a critical step to bring these therapies to clinical trial.

Do you work with people living with ALS in the clinic? Is there an experience in the clinic that stands out to you?
As a fellow, I work with faculty in a number of different clinics and in the electromyography (EMG) lab to learn how to diagnose and manage ALS and other neuromuscular disorders. It is always difficult any time someone is diagnosed with ALS, regardless of his or her age. However recently, I have interacted with a number of young patients, some younger than myself, who are facing this diagnosis. These stories serve as a powerful source of motivation to keep pushing forward.

What qualities make for a good scientist?
Great scientists are innately curious and able to find joy in the journey. Experiments often fail or produce unexpected results, which can be discouraging. But often these are important clues.

What else do you think is exciting in ALS research? What are the bright spots on the horizon?
The Answer ALS initiative is really exciting. This is the largest coordinated research effort in ALS to date to collect comprehensive data from 1000 patients, build iPS cell lines and systematically comb through all of the biological data. Hopefully, this will help clarify different subtypes of ALS and also uncover new mechanistic and therapeutic targets.

What do you do for fun?
When I get a chance, I love to spend time outside with my husband and our beagle, Lucy. There are a lot of great hiking trails around Baltimore, and we also enjoy just hanging out at the park.

Who are your heroes in real life?
ALS patients and their caregivers are really my heroes. Those of us in clinic or in the lab get to go home at the end of the day, but this is 24/7 for them and constantly becoming more challenging. I really hope that we are on our way to treatments that can slow or stop this terrible disease.

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