Research Spotlight: Amyotrophic Lateral Sclerosis Biomarkers for Drug Product Registration: A Systematic Review

In this week’s Research Spotlight, we hear from David Farber, a first year US Medical/Medical Director: Multiple Sclerosis and Neurology fellow with Sanofi. David has spent the first months of his fellowship working on this project and we’re so excited to learn more about it!

Can you tell us about your project?

Amyotrophic Lateral Sclerosis (ALS) is a debilitating, devastating, degenerative disease with a short anticipated lifespan post-diagnosis. In some past clinical trials, there have been reductions in various biomarkers without associations to functional scoring systems. The goal of this project is to characterize recent biomarkers in clinical development and their association to functional ability. Hopefully, this new generation of therapies and biomarkers will accurately predict/correlate with disease progression with specificity to ALS.

Why did you decide to pursue this topic for your research project?

My current fellowship is in neurology and ALS is a neurological disorder with few treatment options with variable efficacy. Biomarkers within ALS are a “hot” topic with top the top neurologists in the country/world looking to address this gap. Personally, I wanted to research a topic with high potential for widespread impact and relate to my fellowship.

Did you experience any hurdles while working on the project?

This project required hours and hours of background research. Because ALS has so few efficacious therapies, there are many entities in development. Each company/academic center will often research one or two unique biomarkers to try to find a correlation between current functional scoring systems and potential biomarkers. I also must understand the background of these biomarkers and in vitro/vivo assays that have led to their inclusion in clinical trials.

Have you presented your research project? If so, where?

Not yet. Updates to come!

Do you have any advice for fellows or prospective fellows who want to pursue a research project?

I think that it is important to find a project that you are passionate about and you think will make an impact. It’s also a bonus if it’s in an area related to your work!