ALS doesn’t stop and neither do we. The reality is, people with ALS can’t wait for treatments and a cure, and just as importantly, the tireless researchers working together around the world can’t wait to make the next breakthrough. Researchers like our 2019 Milton Safenowitz Postdoctoral Fellowship Program recipients.
Our Milton Safenowitz Postdoctoral Fellowship Program continues to support young scientists and is the only program of its kind specifically funding early ALS postdoctoral fellows eagerly searching for a cure.
The awards were founded in memory of Mr. Safenowitz by the Safenowitz family through The ALS Association Greater New York Chapter. The program encourages young scientists to enter and, importantly, to remain in the ALS field.
We are proud that 76 percent of the postdoctoral fellows we fund go on to start their own labs to continue studying ALS and mentor other young ALS researchers. The rest of our Safenowitz fellowship program graduates go on to careers in the biomedical industry, nonprofits, and medical writing, with many still staying in the ALS space.
This year, we are supporting six new postdoctoral fellows out of a highly competitive applicant pool. Over the next few weeks, we will highlight each fellow, their dedication and unique contributions to ALS research, as well as their interests outside of the lab.
We recently talked with Dr. Gerbino from the Maniatis Lab at Columbia University to learn about her unique research project focused on identifying how mutations in TBK1, one of the genes associated with ALS, differentially affect the cells of the spinal cord involved in the pathogenesis of ALS.
Valeria Gerbino, Ph.D.
Maniatis Lab at Columbia University
Project: The cell autonomous and non-cell autonomous role of TBK1 in disease progression in a mouse model of ALS.
Mentor: Tom Maniatis, Ph.D.
Can you briefly describe your academic background?
I did my undergraduate and graduate studies at the University of Rome Tor Vergata (Italy). I carried out my PhD studies in the lab of Professor Maria Teresa Carri’ in Italy, where I started to investigate molecular mechanisms underlying Amyotrophic Lateral Sclerosis. In particular, at that time, I was interested in how ALS-associated mutations in the FUS/TLS gene cause problems to the splicing machinery. For my post-doctoral studies, I joined the lab of Tom Maniatis at Columbia University, where I am currently pursuing the role of TBK1, a protein involved in autophagy and immunity, in ALS.
It is said that every 90 minutes, someone is diagnosed with ALS and every 90 minutes someone dies from the disease. Time is not on the side of those who are diagnosed, and no matter what issues we are all currently facing in the world, ALS won’t stop, so neither will we. What are you doing to address the urgency our ALS community is feeling?
I have been committed to ALS research for the past 10 years. Important goals have recently been achieved in the field with the advent of gene therapy. This progress was allowed by decades of basic research that led to the discovery of cellular and molecular mechanisms underlying disease in some of its genetic forms. My commitment and contribution in this sense is to continue to study this devastating disease as more genes are associated to it. This will hopefully open the way to the discovery of novel therapeutic approaches in the future.
What are the goals of your funded research project?
The ALS Association funded project I am working on seeks to identify how mutations in TBK1, one of the genes associated with ALS, differentially affect the cells of the spinal cord involved in the pathogenesis of ALS. I am using genetic manipulation of mouse models to test the hypothesis that mutations in TBK1 that cause loss of function of the protein that it encodes for, differentially affect the neurons and the immune cells of the spinal cord. I anticipate that the results from this experiment will broaden the little understanding that we have to date of the role of TBK1 in neurodegenerative disease progression and will set the basis for future work aimed at identifying therapeutic targets for patients with mutations in this gene.
Why did you decide to study ALS over other diseases?
I have always been fascinated by the perfection and complexity of the nervous system. Neurodegenerative diseases relentlessly disrupt this perfection. During my master’s degree I met Professor Carri’, who had built her career studying SOD1 mutations in ALS. She introduced me to ALS research when I joined her lab for my PhD, and I inherited from her the passion for understanding and studying this devastating disease. With her sudden death 2 years ago, the ALS community has lost an important contributor. I owe her my passion for what I study, and I’ll always be thankful to her for teaching me how to be a rigorous scientist.
How might your work impact the ALS community?
The ultimate goal of my research is to help discover new pathways that can slow ALS disease progression in mouse models of the disease. Hopefully this can give insights into potential approaches for the human disease.
Where can people get more details about your research project?
Part of the research funded in 2019-2020 has just been published in the journal “Neuron”, for the rest…stay tuned!
It is often said that ALS is one of the most complex diseases to understand. Yet, you go to work every day to tackle the challenges of your research. What gives you hope that there will someday be a world without ALS?
The progresses that the field has made in recent years, not only on ALS, but on other motor neuron diseases such as Spinal Muscular Atrophy, for example. Neurodegenerative diseases that were considered incurable 10 years ago are now treatable. It’s a long process unfortunately, but I have hope that ALS will get there too.
What do you like to do when you aren’t in the lab?
I am an avid aerial dancer; you can easily find me hanging on aerial silks or aerial hoops in the studio when I am not in the lab.
Is there anything else you’d like to add?
I would like to thank the Milton-Safenowitz fellowship for supporting my ALS studies, and my mentor Tom Maniatis, from whom I learned (and continue to learn) a lot, including fundamental skills that will be much needed in my career path to independence in the ALS field.