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2024 Barber ALS Research Award Winners

California ALS Research Summit

Notable ALS advocate Jim Barber was an instrumental voice in the ALS community who advocated for ALS research. A major outcome of his work was the formation of the California ALS Research Network in 2010 and the creation of the annual California ALS Research Summit in 2011. Jim was deeply passionate about advancing scientific achievement by fostering early-career researchers and developing new ideas. Sadly, he died from ALS in 2016.

In 2017, the Barber family and ALS Network created the Barber ALS Research Awards, which are given annually to scientific poster presenters at the California ALS Research Summit. Submissions are evaluated by the Chair of the ALS Network along with former Barber Award honorees. The three top poster presenters receive a special memento and a cash prize of  $1000 each to support their work in ALS research in recognition of their accomplishments. 

The 7th annual Barber ALS Research Awards winners were announced during the 2024 California ALS Research Summit, where they gave a brief public presentation of their work.

Frederick (Eric) Arnold, PhD, UC Irvine

Frederick (Eric) Arnold, PhD began studying biology as an undergraduate at Haverford College. He has researched neuromuscular disease since 2013 as a graduate student at Thomas Jefferson University. For the last five years, his research has been entirely focused on ALS, both to understand the cellular processes that lead to ALS and to identify new therapy targets. In 2022, the American Brain Foundation honored him with their Richard Olney Clinician-Scientist Development Award in ALS.

What motivates Frederick, known by his friends as “Eric” in his ALS research is the challenge of finding targets to treat sporadic ALS. He is continuing ALS research as a postdoctoral fellow at the La Spada Lab at the University of California, Irvine.

ALS is only caused by a known inheritance in 10% of cases. The other 90% of cases are “sporadic” due to an unknown combination of risk factors, but almost 97% of ALS patients exhibit TDP-43 pathology, suggesting a shared disease mechanism. TDP-43 works to regulate multiple steps in RNA processing.

His work is focused on another aspect of RNA processing called alternative polyadenylation (APA), which is known to be regulated by TDP-43. Through this research, Eric hopes to find new genetic risk factors for ALS, which will help the development of effective treatments for sporadic ALS and help doctors slow the progression of the disease.

“I started in grad school working on a rare neuromuscular disease – spinal and bulbar muscular atrophy. I fell in love with that research topic and community, and I knew I wanted to continue that for the rest of my research career.  The thing that’s particularly interesting and challenging about ALS is …. the sporadic nature of the disease, which makes treatments challenging, unfortunately. The biology is … like a puzzle, which also keeps me engaged. … The double-edged sword of that has me …  in ALS research. I’m happy to be a part of the ALS research community.”

Caiwei Guo, PhD, Stanford University

Caiwei Guo, PhD, received her doctoral degree in Neuroscience from Baylor College of Medicine and her BS in Biology from Peking University, China. She was honored in 2022 with the Milton Safenowitz Postdoctoral Fellowship for ALS Research. She is currently a postdoctoral fellow in Dr. Aaron Gitler’s lab at Stanford University. Their research combines cell models, animal models, and human sequencing data to unravel the genetic and molecular mechanisms of ALS, aiming to identify potential new therapeutic targets.

Caiwei discussed how TDP-43 pathology triggers cryptic RNA splicing of synaptic genes in ALS/FTD for her poster presentation. Before genes are turned into proteins, the DNA has transcribed into RNA and processed in various ways. One of those processes is the RNA splicing process where some parts are spliced in or out. Normal RNA splicing removes non-coding regions from the mature RNA to make functional, accurate RNAs and proteins. When TDP-43 is mislocalized from the nucleus, it becomes dysfunctional. As a result, produced RNA molecules contain extra snippets of inappropriate sequences called cryptic exons. This leads to defective protein production and impaired neuronal transmission – the communication portal between two nerves or between a nerve and muscle. Caiwei’s study aims to identify genes affected by this abnormal RNA splicing process due to TDP-43’s loss of function. Caiwei and her team are developing a new therapeutic strategy to target these cryptic splicing events.

“I’ve been attending the California ALS Research Summit for three years, from virtual sessions during the pandemic to in-person meetings. This conference and the entire ALS research community have been tremendously welcoming and supportive. I particularly enjoy participating in the Barber poster discussion, where I can chat with smart scientists from different backgrounds, hear brilliant ideas that I’ve never thought about, and – even better- establish new collaborations.”

“I’m very interested in RNA biology and how RNA disruption leads to disease. I think ALS has the most exciting research, so I want to apply my background knowledge to this field and find potential therapies for this devastating disease.”

Kevin Rhine, PhD, University of California San Diego

Kevin Rhine, PhD, received his BA in Biochemistry & Molecular Biology and MA in Biotechnology from Boston University. He then earned his PhD in Biology at Johns Hopkins University. In 2022, Kevin joined Dr. Gene Yeo’s lab. He was honored with the 2022 Milton Safenowitz Postdoctoral Fellowship for ALS Research.

As a postdoctoral fellow in the Department of Cellular and Molecular Medicine at UC San Diego, Kevin focused his research on how aging contributes to ALS. Aging is one of the major risk factors for ALS. He studied old neurons from people without ALS and noticed that TDP-43 mislocalized. This mislocalization leads to changes in splicing at cytoskeleton proteins important for neuronal function. In addition, there is a molecular stress response where the older neurons are stressed out. Kevin and his team are trying to reverse the effect of aging on neurons and find potential treatments that could help with ALS.

“I’ve been working on ALS research since grad school. I focused on FUS mutations and how they caused changes in aggregation potential, so it felt natural to keep working on ALS. My grandfather had Parkinson’s Disease, so I know what it feels like to have this kind of difficult diagnosis. I want to try to help patients have more options and, at least, be able to know more about the disease. It’s a really important problem… and I hope to generate more potential therapeutics that will be helpful to real people. ”

You can also watch the Barber ALS Awards winners discuss their presentations with Clive Svendsen, PhD, Chair of the California ALS Network, as a part of our webinar, ASK ME ”ALS Research and Care in 2024”.

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