The abnormal accumulation of the protein TDP-43 occurs in both FTD and amyotrophic lateral sclerosis (ALS), suggesting that disruptions in TDP-43 function may be at the heart of both disorders. Over the last decade, researchers have made remarkable progress in piecing together the complex biology of TDP-43, which involves the coordinated activity of many other proteins and the regulation of hundreds of genes. But just as knowing the many parts that make up a car doesn’t explain how those parts work together in a moving vehicle, knowing all the proteins and genes that interact with TDP-43 doesn’t explain how it functions in healthy brain cells or how this network breaks down in FTD and ALS.

A field of research called systems biology could help “fit the pieces together.” Utilizing tools and techniques from bioinformatics, computational biology, and statistics, systems biology can allow researchers make sense of large datasets to better understand how cells function in health and disease. In other diseases like cancer and autoimmune disorders, systems biology-approaches are not only leading to advances in the biology of disease but have also pointed to new targets for drug and biomarker development.

The state-of-the-art computational tools used in systems biology could result in similar breakthroughs for FTD and ALS – if only cell and molecular biologists working on neurodegeneration had more opportunities to interact with experts in computational biology. On April 23-25, AFTD partnered with the Robert Packard Center for ALS at Johns Hopkins University to co-host a unique interdisciplinary workshop, “Understanding the TDP-43 Ecosystem in ALS and FTD: A Systems Biology Perspective,” at the Four Seasons Hotel in Baltimore to provide such an opportunity. A total of 31 experts on TDP-43 biology and computational biology attended the workshop to brainstorm strategies for applying systems biology tools and techniques to gain a deeper understanding of FTD and ALS data.

“We hope the energy and enthusiasm demonstrated at this workshop will be the starting point for further collaboration,” said Debra Niehoff, PhD, AFTD’s Director of Research and Grants. “Systems biology is an example of how ‘big data’ analytics can advance the development of new treatments for both FTD and ALS.”