NeuroBANK™ as an Accelerated Clinical Research Environment: Development, Deployment and Services to ALS Research Community Research Summary Principle Investigators: Merit Cudkowicz MD & Alex Sherman Successful implementation of NeuroBANK™ will allow for a standardized patient-centric approach to clinical research in ALS with information linked across studies, locations and modalities. Standard Common Data Elements, standard operating procedures and GUID technology will lead to accelerated studies' review, approval, deployment and patient enrollment. NeuroBANK™ is a patient-centric clinical research platform that allows capture and aggregation of clinical and clinical research data from simultaneously running research projects and links these data with biospecimen repositories, [...]

Imaging Axonal Transport Research Summary Principle Investigator: Robert Brown D.Phil., MD The field of ALS needs a tool, often called a biomarker, by which the activity of this disease can be gauged. This is potentially important not only for diagnosing the disease, but also for identifying factors causing ALS and for gauging the efficacy of putative ALS treatments. One such biomarker is to quantify a parameter that directly reflects the health of the motor neuron. One property that is promising in this regard is axonal transport, the process by which a nerve cell moves cargoes such as proteins and organelles up [...]

Application of MultiOmyx to iPSC Models of ALS Research Summary Principle Investigator: Clive Svendsen PhD Induced pluripotent stem cell technology (iPSC) holds great promise for accelerating our understanding of the molecular mechanisms and pathways leading to motor neuron degeneration in ALS. In vitro cellular model systems generated from patient-derived iPSC lines can recapitulate many aspects of in vivo cellular pathology, and beyond basic disease research may serve as a powerful test bed to screen potential therapies. Therapeutic screening on iPSC-derived models will likely be complementary to, and in some aspects superior to other approaches, including animal models in terms of [...]

Using Novel Imaging Agents as a Biomarker for ALS Progression in the fALS RAT Research Summary Principle Investigator: Clive Svendsen PhD Assessing the progression of ALS in both animal models and the human condition would provide the ideal biomarker for testing novel compounds rapidly and efficiently. However, while MRI and other imaging modalities exist, they have not been fine-tuned for this neurological condition. Our rationale is that through better imaging of disease progression in ALS within individual patients, drug therapy throughput and success will be increased. The novel imaging marker project is in collaboration with GE and asks whether a [...]

Biomarker Studies to Accelerate Diagnosis and Identify Therapeutic Targets Northeast ALS Consortium (NEALS) Biorepository Infrastructure Improvement and Expansion Protocol Research Summary Principle Investigators: James Berry MD, Nazem Atassi MD & Merit Cudkowicz MD The exact cause of amyotrophic lateral sclerosis remains unknown, and there are no tests to diagnose the disease or follow its progression. Such tests, known as biomarkers, would be invaluable to speed the development of novel therapies for ALS. In recent years, ALS scientists have begun to identify promising potential biomarkers in the blood and spinal fluid of people with the disease. The next step to build [...]

TRACK-ALS: Imaging and Biofluid Biomarkers in Amyotrophic Lateral Sclerosis Research Summary Principle Investigators: Nazem Atassi MD, Joseph Masdeu MD, PHD & A. Nadeem Ishaque PhD This is a very exciting collaborative project among industry, academia and the ALS Association (ALSA). The overall goal of this project is to identify new imaging markers of ALS that can be used to accelerate ALS diagnostic timelines and the pace of ALS drug development. We plan to compare innovative MRI signals between 50 people with ALS and 50 healthy volunteers, and to study these MRI signals over time in relationship with ALS clinical presentation, [...]