In 1998, ALS researchers worked mostly in isolation, often even competing against one another. Project ALS transformed this approach, recruiting world-leading scientists from different fields to work on ALS together, meet regularly, and share data openly. Due to this shift, there has been more advancement towards a cure for ALS disease in the past 20 years than in the previous 100.
Over 50 New ALS Genes Identified.
In 1998, two genes had been linked to ALS, but today, scientists have identified over 50 that contribute to not only familial ALS, but sporadic, which comprises 90% of ALS disease cases. Project ALS has funded studies that investigate the top gene offenders in ALS, including C9orf72, FUS, TDP-43, TBK-1, Optineurin, and SOD-1. Identifying these genes has allowed researchers to build models of the disease that better mimic individuals’ cases of ALS, so now researchers can test drugs on tiny, more accurate representations of the human disease. Genetics breakthroughs have also implicated cellular pathways that are involved in ALS. By identifying cellular pathways scientists can better target therapies.
Built a Stem Cell Toolbox.
Project ALS was the first organization to use stem cell and patient-based models in ALS drug testing; this practice is now used worldwide. Once a laborious process that depended on limited samples from ALS patients, ALS drug screening has since become faster and more efficient using stem cell models. Project ALS also opened the Jenifer Estess Laboratory for Stem Cell Research, the first privately funded stem cell laboratory for ALS research.
Leader in Basic Science
Project ALS is a leader in basic science studies in fields such as neuroscience and biology; these studies clarify aspects of brain disorder and explore the mysteries of ALS. For example, Project ALS currently funds a study that explores ocular motor neurons—the motor neurons controlling eye movement—and why they remain functional in ALS while motor neurons throughout the rest of the body are compromised. Project ALS has also made headway in the ambiguities of brain circuitry and the roles of different cell types in human movement. This provides insight into the ways movement is affected in ALS. Without furthering our knowledge of basic information on the brain, it will be impossible to solve a brain disease as complicated as ALS.
By attacking and further understanding genetics, stem cell research, cellular biology and basic science, we can now 1) identify and create better ALS drugs to test, and 2) better assess these drugs through improved tests and models of the disease. While scientists’ approach to ALS drug testing was once “a shot in the dark”, Project ALS researchers now have the knowledge and technology to more comprehensively and accurately test promising drugs at the pre-clinical level, and subsequently, in human clinical trial.